Researchers make ‘exciting first step’ to better understanding development and treatment of autism – BioPharma-Reporter.com

The authors of a paper, published in the journalScience, in an attempt tostudy the function of genes implicated in autism spectrum disorders (ASDs), applied a gene-editing and single-cellsequencing system, Perturb-Seq, to knock out 35 ASD candidate genes in multiple mice embryos.

They described how the Perturb-Seq method they developed can investigate the function of many different genes in many different cell types at once.

Directing the large-scale method to the study of dozens of genes that are associated with ASD, they identified how specific cell types in the developing mouse brain are impacted by mutations.

"The field has been limited by the sheer time and effort that it takes to make one model at a time to test the function of single genes. Now, we have shown the potential of studying gene function in a developing organism in a scalable way, which is an exciting first step to understanding the mechanisms that lead to autism spectrum disorder and other complex psychiatric conditions, and to eventually develop treatments for these devastating conditions," co-senior author Paola Arlotta, the Golub Family Professor of Stem Cell and Regenerative Biology at Harvard.

The method is also broadly applicable to other organs, enabling scientists to better understand a wide range of disease and normal processes, she said.

The study was also led by co-senior authors Aviv Regev, who was a core member of the Broad Institute during the study and is currently executive vice president of Genentech research and early development, and Feng Zhang, a core member of the Broad Institute and an investigator at MIT's McGovern Institute.

"Through genome sequencing efforts, a very large number of genes have been identified that, when mutated, are associated with human diseases. Traditionally, understanding the role of these genes would involve in-depth studies of each gene individually. By developing Perturb-seq for in vivo applications, we can start to screen all of these genes in animal models in a much more efficient manner, enabling us to understand mechanistically how mutations in these genes can lead to disease," said Zhang, who is also the James and Patricia Poitras Professor of Neuroscience at MIT and a professor of brain and cognitive sciences and biological engineering at MIT.

According to the World Health Organization (WHO), the global burden of ASD is continuously growing, with a current prevalence rate of 1 in 160 children.

Reported prevalence rates vary widely from country to country though, according to apaper published in Nature.

Data from the US Centers for Disease Control and Prevention shows that about 1 in 68 children in the US had been identified with some form of ASD, with more than 3 million people affected.A study referenced in the Nature report estimates that the prevalence of ASD in the US in 20142016 was 2.47% among adolescents and children, while in the UK, the annual prevalence rate for children aged 8 years between 2004 and 2010 was 3.8/1000 for boys and 0.8/1000 for girls.

That paper also indicated recent studies showing the pooled ASD prevalence estimate in Asia is 0.36%, including data from nine countries: China, Korea, India, Bangladesh, Lebanon, Iran, Israel, Nepal and Sri Lanka, while the prevalence of ASD in the Middle East region was documented to be 1.4 per 10,000 children in Oman, 4.3 per 10,000 children in Bahrain, and 1/167 in Saudi Arabia.

Moreover, ASD incidence is four to five times greater in males than in females, according to the Nature report.

To investigate gene function at a large scale, the researchers said they combined two powerful genomic technologies. They used CRISPR-Cas9 genome editing to make precise changes, or perturbations, in 35 different genes linked to autism spectrum disorder risk. Then, they analyzed changes in the developing mouse brain using single-cell RNA sequencing, which allowed them to see how gene expression changed in over 40,000 individual cells.

By looking at the level of individual cells, the researchers could compare how the risk genes affected different cell types in the cortex - the part of the brain responsible for complex functions including cognition and sensation. They analyzed networks of risk genes together to find common effects.

"We found that both neurons and glia - the non-neuronal cells in the brain - are directly affected by different sets of these risk genes," said Xin Jin, lead author of the study and a Junior Fellow of the Harvard Society of Fellows. "Genes and molecules don't generate cognition per se - they need to impact specific cell types in the brain to do so. We are interested in understanding how these different cell types can contribute to the disorder."

To get a sense of the model's potential relevance to the disorder in humans, the researchers compared their results to data from post-mortem human brains. In general, they found that in the post-mortem human brains with autism spectrum disorder, some of the key genes with altered expression were also affected in the Perturb-seq data.

"We now have a really rich dataset that allows us to draw insights, and we're still learning a lot about it every day," Jin said. "As we move forward with studying disease mechanisms in more depth, we can focus on the cell types that may be really important."

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Researchers make 'exciting first step' to better understanding development and treatment of autism - BioPharma-Reporter.com

Research Antibodies and Reagents Market to Reach $6.32 Billion by 2027 With COVID-19 Impact, at a CAGR of 5.6% from 2020- Meticulous Research Analysis…

December 01, 2020 05:00 ET | Source: Meticulous Market Research Pvt. Ltd.

London, Dec. 01, 2020 (GLOBE NEWSWIRE) -- In its latest publication, titled Research Antibodies and Reagents Market by Product {Antibodies [Type (Primary, Secondary), Production, Source, Research Area (Oncology, Neurology)], Reagents}, Technology (ELISA, Western Blot), Application, End User (Pharma, Academia) - Global Forecast to 2027, Meticulous Research analyses that the research antibodies and reagents market is expected to grow at a CAGR of 5.6% from 2020 to reach $6.32 billion by 2027.

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Traditionally, antibodies were obtained from blood and serum. However, these antibodies were produced in a limited number and may cause cross-reactivity. This led to an increase in their cost, along with decreasing use in basic research. To overcome these drawbacks and decrease the outgrowth of various diseases, researchers developed different types of antibodies using sophisticated technologies that can produce a large number of antibodies within less time with high specificity and affinity than traditional ones. Thus, to promote research activities, advanced technologies using research antibodies and reagents products are widely used by various end-users.

Factors such as the increasing proteomics and genomics research studies, increased funding for research activities, and growing industry-academia collaborations are majorly driving the research antibodies and reagents market. Also, growing economies, rising protein-based research, and increasing biomarker discovery provide opportunities for the growth of the market. In addition, the growing growing number of research activities due to increasing COVID-19 cases all over the globe is further driving the adoption of research antibodies and reagents by various end-users.

To provide efficient analysis, Meticulous Research has segmented this market by product {antibodies [type (primary antibody, secondary antibody), production type (monoclonal antibody, polyclonal antibody, and antibody fragments), source (mouse, rabbit, and others), research area (oncology, cardiovascular disease, infectious diseases, immunology, neurology, stem cell research, and others)], reagents [sample preparation reagents (media and serum, stain and dyes, probes, buffers, and solvents), antibody production reagents (enzymes and proteins), other research reagents}, technology (western blot, immunofluorescence, ELISA, multiplex immunosorbent assay, flow cytometry, immunohistochemistry, immunoprecipitation, and others), application (proteomics, drug discovery and development, and genomics), end user (pharmaceutical and biotechnology industry, academic and research institutes, and contract research organizations), and geography (Asia-Pacific, Europe, North America, the Middle East & Africa, and Latin America).

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In 2020, geographically, North America is projected to command the largest share of the research antibodies and reagents market, closely followed by Europe and Asia-Pacific. However, Asia-Pacific region is expected to grow at the fastest rate due to growing R&D investment in proteomics research, rising middle-income people, growing focus on the pharma sector, and improving healthcare industry.

Based on product, the reagents segment is projected to grow at the fastest growth rate of the overall research antibodies and reagents market due to increasing focus on understanding the molecular basis of diseases and routine use in target-based assays during the basic research and huge demand for various reagents in many routine assays.

In 2020, the flow cytometry segment is expected to command the largest share of the overall research antibodies and reagents market. Growing focus on biomedical research for improving diagnosis and therapy developments and growing focus on biomarker discovery and cell-based research are the key factors driving the growth of this segment.

In 2020, on the basis of application, the proteomics segment is poised to command the largest share of the overall research antibodies and reagents market. The upsurge in proteomics research is attributed to the rising need to design more effective drugs through protein-based disease profiling, rising uptake of research antibodies in the significantly growing proteomics market, and increasing public & private sector spending on proteomic research.

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On the basis of end user, in 2020, the pharmaceutical and biotechnology industry segment is estimated to command the largest share of the research antibodies and reagents market due to the highest adoption of antibodies and reagents by this end-user for increasing research activities.

The report includes a competitive landscape based on an extensive assessment of the product portfolio offerings, geographic presence, and key strategic developments adopted by leading market players in the industry over the past four years (2016-2019). The key players profiled in the research antibodies and reagents market are GE Healthcare (U.S.), Merck KGaA (Germany), Thermo Fisher Scientific Inc. (U.S.), F. Hoffmann La-Roche AG (Switzerland), Rockland Immunochemicals Inc. (U.S.), Johnson & Johnson (U.S.), Agilent Technologies, Inc. (U.S.), Eli Lily and Company (U.S.), Becton Dickinson and Company (U.S.), Danaher Corporation (U.S.), PerkinElmer, Inc. (U.S.), GenScript Biotech Corporation (U.S.), Lonza (Switzerland), Bio-Techne Corporation (U.S.), Bio-Rad Laboratories, Inc. (U.S.), Teva Pharmaceutical Industries Limited (Israel), Santa Cruz Biotechnology, Inc. (U.S.), and BioLegend, Inc. (U.S.) among others.

To gain more insights into the market with a detailed table of content and figures, click here:https://www.meticulousresearch.com/product/research-antibodies-reagents-market-5055/

Scope of the Report:

Research Antibodies And Reagents Market, by Product Type

Research Antibodies And Reagents Market, by Technology

Research Antibodies And Reagents Market, by Application

Research Antibodies And Reagents Market, by End User

Research Antibodies And Reagents Market, by Geography

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Amidst this crisis, Meticulous Researchis continuously assessing the impact of COVID-19 pandemic on various sub-markets and enables global organizations to strategize for the post-COVID-19 world and sustain their growth. Let us know if you would like to assess the impact of COVID-19 on any industry here-https://www.meticulousresearch.com/custom-research.php

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The name of our company defines our services, strengths, and values. Since the inception, we have only thrived to research, analyze and present the critical market data with great attention to details. With the meticulous primary and secondary research techniques, we have built strong capabilities in data collection, interpretation, and analysis of data including qualitative and quantitative research with the finest team of analysts. We design our meticulously analyzed intelligent and value-driven syndicate market research reports, custom studies, quick turnaround research, and consulting solutions to address business challenges of sustainable growth.

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Research Antibodies and Reagents Market to Reach $6.32 Billion by 2027 With COVID-19 Impact, at a CAGR of 5.6% from 2020- Meticulous Research Analysis...

Nobel Prize history from the year you were born – Morganton News Herald

Nobel Prize history from the year you were born

Since 1901, Nobel Prizes have honored the worlds best and brightest and showcased the work of brilliant and creative minds, thanks to Swedish businessman Alfred Nobel, who made his fortune with the invention of dynamite.

The Prize in Physiology or Medicine often honors those whose discoveries led to medical breakthroughs, new drug treatments, or a better understanding of the human body that benefit us all.

The Prize in Literature celebrates those skilled in telling stories, creating poetry, and translating the human experience into words. The Prizes in Chemistry and Physics remind most of us how little we understand of genetics, atomic structures, or the universe around us, celebrating the scientists who further knowledge. A later addition to the award roster, the Nobel Memorial Prize in Economic Sciences is not an original Prize, but was established by the Central Bank of Sweden in 1968 as a memorial to Alfred Nobel. It applauds those who can unravel the mysteries of markets, trade, and money.

The Peace Prize celebrates, in Nobels words, the person who shall have done the most or the best work for fraternity between nations, the abolition or reduction of standing armies and for the holding and promotion of peace congresses, sometimes risking their lives to do so.

So precious are the awards that the medals of German physicists Max von Laue and James Franck, stored away for safekeeping in Copenhagen during World War II, were dissolved in acid to keep them away from approaching Nazi troops. After the war, the gold was reconstituted from the acid and recast into new medals.

But Nobel history has not been entirely noble. In 1939, British Prime Minister Neville Chamberlain, known for his policy of appeasement toward Nazi Germany, was nominated for the Peace Prize. In an act of irony and protest, members of the Swedish Parliament nominated Adolf Hitler. That nomination was withdrawn. Some recipients have ordered oppressive crackdowns on their own people or ignored genocides, either before or after receiving the Prize. The 1918 Nobel Prize in Chemistry was given to Germanys Fritz Haber, who invented a method of producing ammonia on a large scale, which was helpful in making fertilizer. But the same chemist helped develop the chlorine gas that was used as a chemical weapon in World War I.

Stacker looked at facts and events related to the Nobel Prizes each year from 1931 to 2020, drawing from the Nobel Committees recollections and announcements, news stories, and historical accounts.

Take a look, and see what was happening with the Nobel Prizes the year you were born.

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Erik Axel Karlfeldt was the first Nobel winner to be awarded posthumously. The Swedish poet was alive during the nomination and deliberation process but died six months before the Literature Prize was announced. As of 1974, the rules were changed so that awards can only be given posthumously if the winner dies after the announcement but before the formal award is bestowed.

John Galsworthy, author of The Forsyte Saga, was awarded the Nobel Prize in Literature. The chronicle of English life became a hugely popular miniseries in 1967 on American public television.

Ivan Bunin, the first Russian writer to win the Nobel Prize in Literature, departed Russia after the 1917 revolution and settled in France as a permanent exile. His books were banned by Soviet authorities due to his anti-Bolshevik writing. To accept his Nobel Prize in Stockholm, Bunin had to travel through Germany, where he was arrested by the Nazis and falsely accused of smuggling jewels. The Nazis forced him to drink a bottle of castor oil before letting him go.

Three Americans shared the Nobel Prize in Physiology or Medicine for their research into anemia, when the bodys red blood cell count is low. George Whipple found dogs formed new blood cells by eating diets of liver, kidney, meat, and apricots, and George Minot and William Murphy applied those findings to humans with pernicious anemia. They also delved into the cause of pernicious anemia: a shortage of vitamin B12.

The 1935 Peace Prize was awarded to Carl von Ossietzky, a journalist and critic of Nazi Germany who was being held in a concentration camp. Hitler would not allow him to accept the prize. He died in 1938 in a prison hospital. Also in 1935, married couple Frdric Joliot and Irne Joliot-Curie won the Nobel Prize in Chemistry. Joliot-Curie was the daughter of Marie Curie and Pierre Curie, also Nobel Prize winners.

The Norwegian royal family chose not to attend the Nobel ceremony following the controversial choice of German pacifist Carl von Ossietzky to win the Peace Prize. Critics said the prize decision would provoke Germany. The royal family offered no official explanation for skipping the ceremony, but it was widely believed that Norway wanted to distance itself from the prize selection.

Contributions to understanding how the human eye functions earned the Nobel Prize in Physiology or Medicine for a scientific trio. Finnish scientist Ragnar Granit researched the types of cones responsible for seeing color; American Keffer Hartline analyzed how the eye processes contrasts; and American George Wald studied the role of light in visual impressions.

The Nobel Memorial Prize in Economic Sciences was established in 1968 by Sveriges Riksbank, the central bank of Sweden. It was created to mark the banks 300th anniversary. The first recipients would be awarded in 1969.

The first Nobel Memorial Prize in Economic Sciences was shared by Norwegian economist Ragnar Frisch and Dutch economist Jan Tinbergen, who helped develop the study of economics based upon mathematics. Tinbergen created a model of macroeconomics, placing economic variables in mathematical relationships to each other. His younger brother, Nikolaas Tinbergen, was one of three scientists awarded the 1973 Nobel Prize in Physiology or Medicine for their studies of animal behavior, particularly how animals communicate and care for their young.

Swedens Ulf von Euler discovered the bodys neurotransmitter norepinephrine, which is involved in the fight-or-flight process, and was awarded the Nobel Prize in Physiology or Medicine. His father, Hans von Euler-Chelpin, won the Nobel Prize in Chemistry in 1929 for his research in the fermentation of sugar and the enzymes involved in the process.

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Physicist Dennis Gabor won the Nobel Prize in Physics for inventing the holograph, and German Chancellor Willy Brandt won the Peace Prize for helping ease relations between East and West Germany. Under his administration, West Germany signed a nuclear weapons non-proliferation treaty, reached a nonviolence deal with the Soviet Union, and laid the groundwork for making family visits easier in the divided city of Berlin.

In a controversial decision, the 1973 Nobel Peace Prize was awarded jointly to U.S. Secretary of State Henry Kissinger and Le Duc Tho of North Vietnam for the Vietnam ceasefire negotiations. But the North Vietnamese leader refused to accept the prize, on grounds that the war was ongoing and the United States violated terms of the agreement. Kissinger did not travel to Norway to accept the prize and said he wanted to return it but was told he could not do so.

Free-market economist Friedrich von Hayek, a critic of central planning, shared the Nobel Prize with Swedish economist Gunnar Myrdal. Hayek argued for a decentralized market system with open competition and disagreed with the use of government fiscal policy to moderate movements of the economy as promoted by economist John Maynard Keynes.

Danish physicist Aage Bohr won the Nobel Prize for his experiments on the structure of atoms. His father, Niels Bohr, won the Nobel Prize in Physics in 1922, also for work on atomic structure. The elder Bohr created a theory that explained how moving electrons cause atoms to emit light.

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American economist Milton Friedman was awarded the Nobel Memorial Prize in Economic Sciences for his work on monetarypolicy. Friedman advocated for free markets and opposed government economic intervention. His views influenced the conservative fiscal policies of U.S. President Ronald Reagan and British Prime Minister Margaret Thatcher. He was a major proponent of school vouchersusing public tax funds to pay for students to attend private schoolsa system that remains hugely controversial to this day.

The Camp David Agreement, which laid out a framework for peace in the Middle East, earned the Nobel Peace Prize for Egyptian President Mohamed Anwar al-Sadat and Israeli Prime Minister Menachem Begin. U.S. President Jimmy Carter was to have been a third recipient, but a technicality prevented him from being nominated within the Committees deadline. But he won the Peace Prize nevertheless in 2002.

Mother Teresa of the Missionaries of Charity was given the Nobel Peace Prize for her work assisting the poor in Kolkata, especially its orphans, lepers, and terminally ill. Pope Francis declared the Albanian nun a saint in 2016. She started the Missionaries of Charity with a dozen nuns and it had nearly 5,000 at the time of her death in 1997.

Britains Frederick Sanger won his second Nobel Prize in Chemistry, having won his first in 1958. The first prize honored his research in the composition of insulin molecules, and the second prize recognized his work in mapping human genomes and developing a method used in DNA sampling and identification.

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The Office of the United Nations High Commissioner for Refugees won its second Nobel Peace Prize. It was awarded its first Peace Prize in 1954 for its relief to refugees in post-war Europe. The Nobel Committee noted that more recently, refugees were coming from developing countries, especially Vietnam, Afghanistan, and Ethiopia, and that the UNHCR accomplished its humanitarian work despite political obstacles and challenges.

Alva Myrdal, along with Mexicican diplomat Alfonso Garca Robles, won the Nobel Peace Prize for working toward nuclear disarmament. Myrdal was a Swedish scientist, government official, and diplomat, and she was married to Gunnar Myrdal, a Swedish economist who was awarded an Economics Nobel in 1974.

Best known for Lord of the Flies, British author William Golding won the Nobel Prize in Literature. The Nobel Committee said his novels, with the perspicuity of realistic narrative art and the diversity and universality of myth, illuminate the human condition in the world of today."

The Nobel Peace Prize was given to Desmond Tutu for his role in ending apartheid in South Africa. The Nobel Committee said it selected the Anglican bishop for his advocacy of using non-violence to counter the system of racial separation. The award to Tutu was influential in the global advocacy for the economic sanctions that pressured South Africa to dismantle its brutal system.

In awarding the Peace Prize to the International Physicians for the Prevention of Nuclear War, the Nobel Committee singled out its Soviet founder Yevgeny Chazov and American founder Bernard Lown to accept the award because of their cooperative influence. The IPPNW consists of tens of thousands of medical professionals, now in 64 countries, who banded together in hopes of preventing and averting nuclear war.

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Holocaust survivor, activist, and author Elie Wiesel won the Nobel Peace Prize for his message of peace, atonement and dignity, the Nobel Committee said. At 16, Wiesel was imprisoned in Buchenwald. His mother, father, and younger sister were killed in the camps.

Costa Rican President Oscar Arias Snchez is credited for his leadership in encouraging five presidents in Central America to sign a peace agreement ending the regions civil wars. The peace plan signed by Costa Rica, Guatemala, El Salvador, Honduras, and Nicaragua called for human rights safeguards, free elections, and an end to interference by other countries.

The winners of the Prize in Physiology or Medicine brought relief to millions with their drug discoveries. The work of Sir James Black led to the use of receptor-blocking drugs to treat heart disease, hypertension, and peptic ulcers, and collaborators Gertrude Elion and George Hitchings made discoveries that led to drugs for the treatment of leukemia, malaria, gout, and herpes virus infections.

Marking the end of the Cold War, the Nobel Committee presented the Peace Prize to Soviet President Mikhail Gorbachev. It honored Gorbachev for his efforts at economic and political reform known as perestroika and dtente with the United States. In a speech delivered by an aide sent to accept the prize, the Soviet leader said it was a recognition of what we call perestroika and innovative political thinking, which is of vital significance for human destinies all over the world.

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Myanmars human rights activist Aung San Suu Kyi was under house arrest when she was awarded the Peace Prize. In 2015 she was released and elected to national office, but she has come under strong criticism for her countrys treatment of its Muslim Rohingya minority and its possible genocide. Calls were made for her Nobel Prize to be revoked, but the head of the Nobel Foundation said it would not be withdrawn for events that took place after it was awarded.

Marking the 500th anniversary of Christopher Columbus landing in the Americas, Mayan Rigoberta Mench Tum of Guatemala was hailed for her campaign for human and Indigenousrights. Taking a global approach, she facilitated the use of international intermediaries in negotiations between guerrilla forces and the government, culminating in a 1996 peace agreement that ended a 36-year civil war.

South Africa took the spotlight when the Nobel Peace Prize was awarded to Nelson Mandela and Frederik Willem de Klerk for their roles in ending the apartheid system of racial separation. Mandela was imprisoned 27 years for his political activism, and de Klerk, the president of South Africa, released him in 1990. They went on to work together to end apartheid and design a new constitution that allowed universal voting rights, regardless of race.

American John Nash was one of three winners of the Nobel Memorial Prize in Economic Sciences for his work on game theory. Nashs struggle with schizophrenia was portrayed in the 2001 Academy Award-winning film A Beautiful Mind, starring Russell Crowe as the Princeton-educated mathematician. His work, which became known as the Nash equilibrium, is used in understanding the processes of chance and decision-making.

Joseph Rotblat, a physicist and longtime opponent of nuclear weapon development, won the Nobel Peace Prize 50 years after atom bombs were dropped on Hiroshima and Nagasaki. He withdrew in 1943 from the Manhattan Project, which was producing nuclear weapons. He worked with the anti-nuclear Pugwash movement, a series of conferences with which he shared the Peace Prize.

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Southeast Asias East Timor was highlighted when the Peace Prize was awarded to Carlos Belo and Jos Ramos-Horta. Ramos-Horta was a leader of resistance in East Timor to the occupation by Indonesia and helped build international support for self-determination, and Belo, a Roman Catholic bishop, demanded the United Nations and the United Nations Commission on Human Rights take notice. The Peace Prize is considered a major factor in East Timor achieving independence in 2002.

Citing the more than 100 million anti-personnel landmines estimated to be strewn around the world, the Nobel Committee gave the Peace Prize to the International Campaign to Ban Landmines and to its coordinator Jody Williams for their accomplishments in banning and clearing mines. Their work culminated in the United Nations Mine Ban Convention, adopted in 1997, that prohibited the stockpiling and use of landmines and required countries to clear mines.

Two political leadersDavid Trimble and John Humein Northern Ireland shared the Peace Prize for helping bring about the Good Friday Agreement that laid out plans for governance of Northern Ireland. Key to the agreement were the Protestant Ulster Unionist Party, led by Trimble, and the Catholic Social Democratic and Labour Party, led by Hume.

The Nobel Committee applauded Mdecins Sans Frontires, known as Doctors without Borders in English, for its extensive humanitarian work across several continents. It said the organization maintained a high degree of independence, helped build public opinion in opposition to humanitarian abuses, and helped forge contacts between sides in conflicts.

Understanding our relation to money earned the Nobel Prize for two American economists. James Heckman researched factors that affect statistical sampling, and his findings have been used to understand how early life experiences influence earnings potential and economic status. Daniel McFadden studied how people make decisions, developing so-called discrete choice models that can explain and predict behavior and are applied to such real-life uses as public transportation systems and senior housing.

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Americans George Akerlof, Michael Spence, and Joseph Stiglitz were awarded the Nobel Memorial Prize in Economics for their work on markets with asymmetric informationmarkets in which one side has better information than the other. Akerlof looked at the consequences of such markets in areas like developing world lending and medical insurance, Spence demonstrated how market participants convey information, and Stiglitz showed how asymmetric markets work in areas like unemployment and credit.

Jimmy Carter was commended for decades of untiring effort to find peaceful solutions to international conflicts, to advance democracy and human rights, and to promote economic and social development. The Committee cited his foreign policy achievements including the Panama Canal treaties, Camp David Middle Eastern accord, and SALT II treaty with the Soviet Union as well as his founding afterward of The Carter Center, which specialized in international conflict mediation and election monitoring.

The creators of magnetic resonance imagining, or MRI, took home the Nobel Prize in Physiology or Medicine. Paul Lauterbur of the United States and Britains Sir Peter Mansfield researched the behavior of atoms and molecules in a magnetic field to develop interior imagery of the human body.

Wangari Maathai, the first female professor in Kenya, became the first African woman to win the Nobel Peace Prize. Already active in the nations democratic movement, Maathai launched a grassroots movement that mobilized women to plant trees to fight deforestation. Called the Green Belt Movement, it spread elsewhere in Africa and led to the planting of more than 30 million trees.

Winners of the Nobel Prize in Physiology or Medicine, Australians J. Robin Warren and Barry Marshall discovered the bacterium that causes gastritis and peptic ulcer disease. Their discovery ran counter to the commonly held belief that peptic ulcer disease was caused by lifestyle and stress. The revelation influenced research into the causes of other chronic inflammatory conditions and the links between chronic infection, inflammation, and cancer.

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The winner of the Nobel Prize in Chemistry, American Roger Kornberg, researched the structure and role of an enzyme called RNA polymerase in a genetic process essential to building and maintaining molecular and cell structure. His father Arthur Kornberg won the 1959 Prize in Physiology or Medicine for his pioneering work in genetics.

Doris Lessing, author of dozens of books including the novel The Golden Notebook, became the oldest winner of the Nobel Prize in Literature at age 88. She told reporters from her doorstep in London that she was not that surprised because her name had been under consideration for decades. Either they were going to give it to me sometime before I popped off or not at all, she said.

The New York Times columnist Paul Krugman won the Nobel memorial Prize in Economic Sciences for his work in international trade patterns and economic geography. Krugman was instrumental in the development of New Trade Theory, which concerns factors in international market patterns such as economies of scale and the network effect, when goods become more valuable with wider use.

Sir John Gurdon of England and Shinya Yamanaka of Japan, winners of the Nobel Prize in Physiology or Medicine, researched the way cells and organisms develop. They discovered how mature specialized cells could be reprogrammed to become immature cells, a major medical breakthrough applicable to stem cell research.

The three American economists who shared the Nobel Prize made discoveries that help predict the long-term prices of stocks and bonds. Eugene Famas research was used in the development of stock index funds, Robert Shiller discovered certain dynamics about stock prices and dividends, and Lars Hansen looked at theories of risks and returns that are used in asset pricing.

At age 17, Malala Yousafzai was the youngest Nobel Laureate when she was awarded the 2014 Peace Prize. Yousafzai, an outspoken advocate for girls education and rights, survived an attempted assassination in 2012 by the Taliban in Pakistan. She shared the Peace Prize with Indian activist Kailash Satyarthi, who was honored for his work fighting child labor.

American physicist Clinton Davisson won the Nobel Prize partly by accident. When he was testing the hypothetical relation between particle velocity and wavelength, he bombarded a nickel block with electrons and measured how they scattered. But when the nickel was baked at high heat after accidental contamination, the structure of its atoms changed, as did the patterns of the electrons, proving the hypothesis.

Germanys Adolf Butenandt won the Nobel Prize in Chemistry for his research into sex hormones that would be used in the development of oral contraception. Like Richard Kuhn in 1938, he was forced by the Nazis to decline the prize but was able to accept the certificate and medal in 1949. Germanys Gerhard Domagk, whose research led to the development of antibiotics, also had to turn down the Nobel Prize in Physiology or Medicine but got it after the war.

No Nobel prizes were awarded in 1940 due to World War II. Norway was occupied by German forces, and Adolf Hitler was angry about the 1936 Peace Prize awarded to Carl von Ossietzky, a journalist who disclosed that Germany had been secretly rearming, a violation of the Treaty of Versailles that ended World War I. Ossietzky was tried and convicted of treason.

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Not only were no Nobel Prizes awarded while the world was locked in war, but a significant brain drain was underway that would see Europe lose many of its brilliant thinkers. By 1941, a dozen Nobel-winning scientists had left for England and the United States, including physicists Niels Bohr, a winner in 1922, Albert Einstein, who won in 1921, and Enrico Fermi, who won in 1938.

While no Nobel Prizes were awarded, 1942 was the year that the name United Nations was coined by President Franklin Roosevelt in a declaration by 26 nations to stand together against the Axis powers. Founded three years later, the United Nations, along with its agencies, programs, and staff, has been awarded the Nobel Peace Prize a dozen times. The U.N.s High Commissioner for Refugees (UNHCR) has won it twice, and most recently, the U.N.s World Food Programme won in 2020.

Henrik Dam and Edward Doisy shared the Nobel Prize in Physiology or Medicine for their work in the discovery of vitamin K, which the human body uses for clotting and healing. Dam determined that Vitamin K is needed for blood to coagulate, and Doisy found ways to produce it artificially, which was useful in stopping bleeding in small children.

The Nobel Peace Prize was awarded to the International Committee of the Red Cross for the work it had done during the war on behalf of humanity. It was the first Peace Prize bestowed in five years; the prizes were not given out from 1939, the year Germany invaded Poland at the start of World War II, to 1943.

Two American activists shared the Nobel Peace Prize. One was Emily Greene Balch, co-founder of the Womens International League for Peace and Freedom and a professor at Wellesley College for 22 years until she was fired for her activism. John Raleigh Mott, head of the Young Mens Christian Association, promoted international youth programs, worked with relief programs for prisoners of war, and was an outspoken critic of colonial oppression and race discrimination.

Gerty Cori, the first woman to win the Nobel Prize in Physiology or Medicine, was honored with her husband Carl Cori for their work in understanding the metabolism process. When the couple moved to America in 1922 from Austria, Carl Cori was hired as a biochemist at a New York research institute. Gerty Cori could only find work as an assistant pathologist, despite having the same degrees and research experience, because she was a woman. She was finally allowed a position equal with her talent and experience at Washington University in St. Louis in 1938.

Indian leader Mahatma Gandhi was nominated five times for the Nobel Peace Prize, the final time being in 1948 not long before he was assassinated. That year, the Nobel Committee chose not to bestow any award, declaring there was no suitable living candidate.

The winner of the Nobel Prize in Medicine, Portugals Egas Moniz, invented the lobotomy. He discovered how a surgical incision into the brains prefrontal lobe could alter behavior. Lobotomies were used widely to treat mental illness in the 1940s and 1950s until they were widely recognized as dangerous, and medication for mental illness became more commonly prescribed.

Ralph Bunche was principal secretary of the United Nations Palestine Commission when he was awarded the Peace Prize for his role in mediating the 1949 cease-fires between Israel and Arab states after the partition of Palestine. Bunche, an academic and U.S. State Department advisor, was the first Black person to receive a Nobel Prize.

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Max Theiler of South Africa was honored for his research on yellow fever, a deadly disease found in subtropical and tropical South America and Africa and spread primarily by mosquitoes. Theiler discovered how to transmit the yellow fever virus to mice, helping produce weaker forms of the virus that could be used as a vaccine for humans.

For his role in the discovery of streptomycin, Selman Waksman was awarded the Nobel Prize in Physiology or Medicine. Streptomycin was the first antibiotic found to be effective against tuberculosis. Waksman studied how the bacteria that causes tuberculosis interacted with microorganisms in soil and found that a bacterium called Streptomyces griseus blocked its growth.

British Prime Minister Winston Churchill won the Nobel Prize in Literature for his works that included an autobiography, several volumes about the First and Second World Wars, and his notable speeches during World War II. From 1946 to 1953, Churchill was nominated in seven years for the Literature Prize and twice for the Nobel Peace Prize.

Although the Nobel Prize in Literature is considered to honor a writers body of work, the Nobel Academy singled out The Old Man and the Sea when it chose American author Ernest Hemingway. It said the novel demonstrated his mastery of the art of narrative.

The winner of the Chemistry Prize, Vincent du Vigneaud, studied biochemical sulphur compounds, including oxytocin, a hormone involved in sexual intimacy and reproduction. Sometimes called the cuddle or love hormone, it is released when people bond, including as couples or parents with children. Vigneaud isolated oxytocin, calculated its chemical composition, and determined how to produce it artificially.

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The king of Sweden goodnaturedly reprimanded John Bardeen for leaving most of his family homehis children were in schoolwhen he was awarded the Nobel Prize in Physics. The American physicist told the king he would bring his family the next time. Sure enough, Bardeen won a second Nobel Prize in 1972, and he took his whole family to theceremony.

Ending conflict in the Middle East was the focus of Canadian historian and diplomat Lester Pearson, who won the Peace Prize for his role in ending violence that erupted in 1956 over control of the Suez Canal. The conflict among the major superpowers could have had severe global consequences. Thanks to Pearsons efforts, a United Nations Emergency Force was dispatched to oversee a cease-fire.

Russias Boris Pasternak was awarded the Nobel Prize in Literature, which he at first accepted but later was forced to turn down by Soviet authorities who banned his novel Doctor Zhivago. The only novel the poet wrote, Dr. Zhivago was deemed to be anti-Soviet and remained forbidden until the late 1980s.

Biochemists Severo Ochoa and Arthur Kornbergs work on DNA and RNA earned them the Nobel Prize in Physiology or Medicine. Ochoa discovered how to create RNA, and Kornberg, formerly a student of Ochoas, found ways of making DNA. Kornbergs son, Roger, a chemical physicist, also conducted genetic research and won the Nobel Prize in Chemistry in 2006.

The awarding of the Peace Prize to Dag Hammarskjld, the second Secretary-General of the United Nations, was only the second time a Nobel was given posthumously. The first occasion was the posthumous literature prize awarded to Erik Axel Karlfeldt in 1931. The rules were changed in 1974 so that prizes could not be awarded posthumously unless the winner dies after the announcement but before the award ceremony.

The winner of the Nobel Prize in Physiology or Medicine, to American James Watson, would later sell it at auction at Christies in 2014 to raise money for charity. Russian tycoon Alisher Usmanov paid $4.7 million, then said he was returning the award to the scientist. Watson won for discovering the structure of DNA along with Francis Crick.

Frances Jean-Paul Sartre was awarded, and declined, the Nobel Prize in Literature.He explained that he always declined official honors and that as a writer, he felt he should remain distinct from any institution.

The United Nations Childrens Fund, better known as UNICEF, was awarded the Nobel Peace Prize. UNICEF started out in 1946 providing food, clothes, and medicine to children and mothers but expanded to promote nutrition, school attendance, and health care in developing countries. The Nobel Committee honored UNICEF for its effort to enhance solidarity between nations and reduce the difference between rich and poor states.

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Peyton Rous won the Nobel Prize in Physiology or Medicine when he was 87, the oldest winner ever in the category. The American studied the role of viruses in cancer cells and transmission. His work was based on research begun in the early 1910s.

In awarding the Prize in Literature, the Nobel Committee honored German writer Heinrich Bll, saying his a broad perspective on his time and a sensitive skill in characterization has contributed to a renewal of German literature." After World War II, Bll was part of the Gruppe 47, a number of influential writers who would meet over the course of three decades as they dealt with the wars destruction and the aftermath of the Holocaust.

Winners of the Nobel Prize in Physiology or Medicine, Roger Guillemin and Andrew Schally used pig brains and lamb brains in their research on hormonal roles and structure. They shared the prize with Rosalyn Yalow, a nuclear physicist who developed radioimmunoassay, a method that can measure extremely small amounts of bodily substances. It was used in helping determine the cause of type 2 diabetes.

When the 14th Dalai Lama was awarded the Nobel Peace Prize, he was living in exile for his opposition to the Chinese occupation of Tibet. Chinese authorities said the award was an act of interference in its internal affairs and that it hurt the Chinese peoples feelings. The Dalai Lama used the occasion to present a plan for Tibet to be a demilitarized zone, an idea the Chinese government rejected.

Originally posted here:
Nobel Prize history from the year you were born - Morganton News Herald

Environmental Factor – December 2020: Aging and cancer interplay explored at NIH workshop – Environmental Factor Newsletter

Researchers discussed how aging influences cancer progression and how cancer accelerates aging, at National Institutes of Health event.

By Arif Rahman

The population of older individuals is increasing globally, and most cancers arise as people age, according to experts at an Oct. 26-27 National Institutes of Health (NIH) virtual conference: Age-Dependent Changes in Cancer Biology. NIEHS, the National Cancer Institute (NCI), and National Institute on Aging (NIA) jointly organized the meeting.

Invited speakers discussed common ground in the biology of cancer and aging and identified areas for further research efforts.

Cancer is rare among people younger than 45. There is a sharp rise after that, peaking between 65 to 74 years, according to NCI Director Ned Sharpless, M.D.

Among the possible explanations for this association, one factor stands out. It is becoming increasingly clear that time itself is a potent mutagen, he said. Mutagens promote errors in DNA replication that can lead to cancer.

Sharpless discussed key causes of age-related cancers, including the following.

He pointed to two critical areas that need more attention from researchers in basic science. The first issue is that many cancer drugs contribute to aging directly, he said. Secondly, therapies that are beneficial in young adults can be difficult to use in older adults because of the age-associated reduced resilience of the host.

NIA Scientific Director Luigi Ferrucci, M.D., Ph.D., echoed Sharpless during his keynote talk. Nearly half of the cancer risk factors are unknown to this day, Ferrucci said. He suggested that studying the role of aging in cancer development would significantly fill that knowledge gap.

Les Reinlib, Ph.D., moderated a session on the overlap between environmental carcinogens and gerontogens, or agents that quicken the aging process. Reinlib is a health scientist administrator in the NIEHS Exposure, Response, and Technology Branch.

Studies suggest that gerontogens such as air pollutants, cigarette smoke, and arsenic may cause cancer by promoting cellular senescence, or cell death. Although senescence gets rid of old or damaged cells, it also promotes cancer development by changing the cellular microenvironment.

Susan Neuhausen, Ph.D., from the City of Hope Comprehensive Cancer Center, noted that the U.S. Environmental Protection Agency has registered approximately 85,000 synthetic chemicals for commercial use, only 10% of which have been tested for effects on human health.

She emphasized the need for more human-relevant testing of these chemicals.

One unifying theme among the talks was that aging and cancer share common biological mechanisms.

By slowing down the aging process, it should be possible to reduce cancer incidence. One way to do that is to promote a healthy cellular microenvironment via proper nutrition, according to Trygve Tollefsbol, Ph.D., from the University of Alabama at Birmingham.

Tollefsbol said that certain dietary guidelines could help curb the high prevalence of cancer, as well as obesity, in developed countries.

Due to the rapid global rise in the population of older adults, experts at the meeting expressed the belief that it is more cost effective to prevent diseases like cancer in the elderly population than to manage them.

(Arif Rahman, Ph.D., is a visiting fellow in Division of the National Toxicology Program Toxicoinformatics Group.)

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Environmental Factor - December 2020: Aging and cancer interplay explored at NIH workshop - Environmental Factor Newsletter

CURE Media Group Announces the 2020 MPN Heroes Winners – Business Wire

CRANBURY, N.J.--(BUSINESS WIRE)--CURE Media Group, the industry-leading multimedia platform devoted to cancer updates and research that reaches more than one million patients, has named the winners of the MPN Heroes Recognition Program. The virtual celebration will take place tonight from 6 to 7:30 p.m. CST.

Keynote speaker Devon Still, former professional athlete, entrepreneur, motivational speaker, author and advocate for childhood cancer awareness, will join in this years annual evening of gratitude and celebration honoring these eight remarkable heroes who have gone above and beyond, making a difference in the field of rare blood cancers known as myeloproliferative neoplasms (MPNs).

The following individuals will be recognized in the Commitment to the Individual category:

The second category honors MPN specialists whose efforts have helped the broader MPN community:

These incredible heroes have truly dedicated themselves to improving the lives of people with myeloproliferative neoplasms, said Mike Hennessy Jr., president and CEO of MJH Life Sciences, parent company of CURE Media Group. This year marks the eighth anniversary of the MPN Heroes Recognition Program, and we look forward to an amazing celebration this evening recognizing these champions who have made a difference in MPN care.

The MPN Heroes Recognition Program is sponsored by Incyte and CURE Media Group, publishers of CURE magazine. Incyte partners with CURE Media Group to support the program, with CURE hosting the annual MPN Heroes Celebration Event. The honorees were nominated by colleagues, patients and caregivers for their heroic contributions and dedication. The selected MPN Heroes were chosen by an independent judging panel comprised of patient advocates and healthcare professionals. Individuals or entities supported through funding or directed by Incyte were not eligible for consideration or recognition. Supporting the MPN community is an ongoing priority for Incyte, a global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development, and commercialization of proprietary therapeutics.

About CURE Media Group

CURE Media Group is the leading resource for cancer updates, research, and education. It features a full suite of media products, including the industry-leading website CUREtoday.com; innovative video programs such as CURE Connections; a series of widely attended live events; CURE magazine, which reaches more than 1 million readers; and the dynamic website for oncology nurses, OncNursingNews.com, and its companion publication, Oncology Nursing News. CURE Media Group is a brand of MJH Life Sciences, the largest privately held, independent, full-service medical media company in North America, dedicated to delivering trusted health care news across multiple channels.

About Incyte

Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.

MPN Heroes is a registered trademark of Incyte. Other trademarks are the property of their respective owners.

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CURE Media Group Announces the 2020 MPN Heroes Winners - Business Wire

John Theurer Cancer Center (JTCC) Researchers to Present 52 Abstracts across Hematological Malignancies at the 2020 American Society of Hematology…

Newswise December 4th, 2020 Hackensack, NJClinical investigators from Hackensack MeridianHealthJohn Theurer Cancer Center (JTCC), amember of the Georgetown Lombardi Comprehensive Cancer Center consortium, are to present updates on treatment advances in multiple myeloma (MM), mantle cell lymphoma (MCL), and other types of B-cell lymphoma (BCL) as well as leukemia at the 62ndAmerican Society of Hematology (ASH) Annual Meeting and Exposition, to be held virtually from December 5-8, 2020.

Once again, and despite COVID, our team has a full presence at ASH 2020, being part of 52 abstracts. This shows our commitment to clinical science and collaboration with other leading institutions, from new studies on CAR-T cells, bispecific T cell engager antibodies, and other new small molecules in leukemia, lymphoma, and myeloma, said Andre Goy, MD, MS, Chairman and Director of John Theurer Cancer Center (JTCC) at Hackensack University Medical Center. It is not just a requirement for our patients but a privilege to contribute to the phenomenal acceleration of cancer medicine.

The 51 abstracts (listed in the table below) cover three of the main themes of ASH this year:

CAR-T cells have emerged over the last few years as a game-changing therapy for patients withhematologicmalignancies. Anti-CD19 CAR-T cells are approved for use in adults with aggressive B-cell non-Hodgkin lymphoma (NHL) or MCL,but also in B-cell acute lymphocytic leukemia (ALL, up to 25years ofage). At ASH,JTCC is part of the team presenting an update from the ZUMA 2 pivotal trial in MCL, in which CAR-T therapy was associated with a durable response (40% of the first28patients still in remission at 32 months follow-up), an unprecedented response in that setting.(Abstract 1120; Dr. AndreGoy).Also, there was a consistent benefitacross all patients with relapsed/refractory MCL, even in those with high-risk features such as blastoid variant or with a high proliferation rate(Abstract 1126; Dr.AndreGoy).

CAR-T cell therapy is also being tested in other blood cancers and will be featured in other presentations at ASH focusing onMM and indolent BCL.

CAR-T cell therapy directed against B-cell maturation antigen (BCMA) has shown promising results in the treatment of relapsed/refractory multiple myeloma (RRMM), with very high response rates (>90%), but unfortunately a significant proportion of patientshave relapsed, potentially due to the lack of sustainability of CAR-T cells over time. To address this, bb21217 is an anti-BCMA CAR-T cell therapy that uses the same CAR molecule as idecabtagene vicleucel (called bb2121; see below) but adds the PI3K inhibitor molecule bb007 during theex vivomanufacture/T cells culture to enrich the drug product for memory-like T cells, thereby reducing the proportion of highly differentiated or senescent T cells. The rationale is to make CAR-T cells more sustainable and to help prolong treatment responses. The results from a Phase I dose-escalation trial of bb21217(Abstract 130; Dr. DavidSiegel)in which patients with RRMM had received 3 prior regimens, are promising, with no added toxicity. The presence of T cell markers associated with memory-like T cells and the absence of T cell markers associated with differentiation/senescence correlated positively with the T cells peak expansion in the patients and the duration of response, consistent with the investigators hypothesis.

Also, an update on BCMA CAR-T therapy(Abstract 131; Dr.DavidSiegel)confirmed a high response rate of 76% (39% with a complete response [CR]) and a median duration of response of 10.3 months. Similarly, a BCMA CAR-T update from the KarMMA trial (NEJM 2019) showed the same benefit in older patients(65-70 years old), even in those who were heavily pretreated(Abstract 1367; Dr. DavidSiegel)or presenting very high-risk features(Abstract 3234; Dr.DavidSiegel).

Finally, JTCC will be a co-presenter of the ZUMA-5 trial, providing the firstCAR-T therapy data in indolent BCL (146 patients), using an anti-CD19 CAR-T and showing a very high response rate (>90% overall response rate [ORR] and >80% CR).These data are very impressive based on the efficacy -- even in double-refractory patients -- but also because of the favorable toxicity profile(Abstract 700; Dr. Lori Leslie).

Alternative options to generate cell therapies are emerging, including natural killer (NK) cell therapygenerated from a pluripotent stem cell (iPSC) line, which can be used as a renewable source for the mass production of off-the-shelf NK cells for broad patient access.Early data on FT538, an investigational, first-of-its-kind, multiplexed engineered NK cell therapy, will be presented in both myeloma and acute myeloid leukemia (AML) at ASH(Abstract 1449; Dr. David Siegel).

The BITE antibodieshelp force the normal immune system to reengage and to fight cancer cells. They are definitely a big topic at ASH, as they continue to show great activity and unlike CAR-T cells, do not necessitate a complex process of cell collection and manufacturing. They may thus offer a form of off-the shelf cell therapy. However, as with CAR-T cell therapy, BITE antibodies can be associated as in CAR-T cells therapy - with cytokine release syndrome (CRS) and/or neurotoxicity both related to the rapid activation and amplification of T cells in patients after infusion. In order to address this, JTCC investigators and colleagues explored the subcutaneous (SC) administration of mosunetuzumab a lead product among BITEs (targeting anti CD3 and CD20) to patients with B-cell relapsed/refractory lymphomas. Results showed an ORR of 60% in heavily pretreated patients with aggressive lymphoma (DLBCL), including in patients who failed prior CAR-T therapy. In addition,researchers observed much less toxicity andhigh bioavailability (>75%), supporting the use of SC dosing for CRS mitigation(Abstract 2096; Dr. AndreGoy).

Usage of both CAR-T cells and BITEs will expand dramatically in the future across many cancers and JTCC has a robust ongoing comprehensive portfolio of CAR-T cells and BITE-based trials, including, off-the shelf CAR-T cells and NK cells, dual/multi-target CAR-T cells, as well as combinations with small molecules to enhance the activity and/or durability of these modifiedT cells.

Another emerging form of immunotherapy involvescheckpoint inhibitors,which also help unleash the immune system and have been approved in many cancers. At ASH a new study using an anti-CD47 checkpoint inhibitor showed very promising results(Abstract 646; Dr. TatyanaFeldman).CD47 is an innate immune checkpoint that binds signal regulatory protein alpha (SIRP) and delivers a"do not eat"signal to suppress macrophage phagocytosis. Overexpression of CD47 can allow tumor cells to escape immune surveillance. TTI-621 (SIRPFc) is a fusion protein consisting of the CD47 binding domain of human SIRP linked to the Fc region of human immunoglobulin G1 (IgG1), designed to enhance phagocytosis and antitumor activity by blocking the CD47-SIRP interaction between malignant cells and macrophages.

Finally, the standard of care of patientswith relapsed Hodgkin disease is salvage with high-dose therapy, followed by stem cell transplantation. A subset of these patientsstill relapse and experience a poor outcome. The ability to use immunotherapy as consolidation post-transplantation (brentuximab + nivolumab) was illustrated in a study presented at ASH(Abstract 472; Dr. TatanyaFeldman), in whichonly one of 59 enrolled patientsrelapsed, leading to a remarkable 98% survival rate and no major toxicity issues.

A number of presentations at ASH will focus on novel emerging agents in several diseases with persistent unmet need, including the following examples.

Acute myeloid leukemia (AML):

The FMS-like tyrosine kinase 3 (FLT3) inhibitor gilteritinib (Gilt) improves survival compared with standard salvage chemotherapy in relapsed/refractory (R/R)FLT3-mutated (FLT3mut+) AML. However, relapses are common and long-term survival remains poor. Combination therapies of FLT3 inhibitors with agents that induce apoptosis have demonstrated preclinical activity and synergy againstFLT3mut+clones and may delay or prevent drug resistance. This was the subject of a study of Gilt + venetoclax (Ven) in patients with refractory AML, with most having previously failed FLT3 inhibitor therapy (Abstract 333; Dr. JamesMcCloskey),suggesting substantially greater antileukemic activity from Gilt + Ven than with single-agent Gilt in a very difficult-to-treat patient population.

Chronic myeloid leukemia (CML):

CML is a disease literally transformed by kinase inhibitors (TKIs)over the last 20 years.However, patients can develop resistance against these targeted therapies, leading to the development of second-, third-, and fourth-generation kinase inhibitors, offering new options, albeit often with new or different toxicities encountered over time. The OPTIC trial was a randomized Phase 2 trial evaluating different dosing schedules of ponatinib in patients with CP-CML who were resistant/intolerant to 2 TKIs or with a T315I mutation. The results of this large landmark trial will help refine dosing in this population, while minimizing toxicities(Abstract 48; Dr. JamesMcCloskey).

B-cell lymphoma (BCL)

CA-4948 is a novel oral small molecule inhibitor of interleukin-1 receptor-associated kinase 4 (IRAK4). IRAK4 is part of the Myddosome signaling pathway (a group of proteins forming a complex involved in innate immunity) and is essential for downstream signaling of tolllike receptors (TLRs) and the interleukin-1 receptor (IL-1R) family in immune cells including B lymphocytes. Dysregulated signaling in these pathways is frequently observed in certain types of BCL, particularly in ABC-subtype of DLBCL and Waldenstrm macroglobulinemia (WM). In this early trial CA-4948 demonstrated a good safety profile, desirable pharmacokinetic properties, and preliminary clinical activity. This offers a new venue to target B-cell signaling in BCLs and provides a rationale for combination therapy with BTK and/or BCL2 inhibitors, expanding the portfolio of emerging non-chemotherapy options for these patients(Abstract 703;Dr. LoriLeslie).

Epstein-Barr virus (EBV) can play a role in several subtypes of lymphoma including Hodgkin, B and T cell lymphomas. EBV-positive (EBV+) lymphomas are generally associated with poor clinical outcomes, particularly for patientswho have relapsed or are refractory (R/R) to standard therapies. Targeting EBV by using the antiviral valganciclovir and the histone deacetylase (HDAC) inhibitorNstat showed promising results, with an ORR up to 80% (50% CR) in T/NK cell lymphoma (a very difficult-to-treat subtype) and 60% in aggressive BCL, providing a potential new approach, as there are currently no approved therapies for these EBV+lymphomas(Abstract 1154; Dr. TatyanaFeldman).

Multiple myeloma (MM):

Proteasome inhibitors were the firstnew class of drugs approved in myeloma; they typically disrupt the system responsible for protein recycling inside the cells and have become the backbone of MM therapy. Attempts to refine these drugs have led to new compounds including IBER, an oral, potent novel cereblon E3 ligase modulator (CELMoD) agent, which has shown marked synergistic tumoricidal and immune-stimulatory effects in combination with bortezomib or daratumumab in preclinical models.CC-220-MM-001 is a phase 1/2 study evaluating dose escalations of IBER with different treatment combinations in independent cohorts in patients with RRMM, expanding the combination of biological agents and providing new options in this population(Abstract 724; Dr. DavidSiegel).

Selinexor (SEL) is a novel, first-in-class oral selective inhibitor of nuclear export (SINE), which blocks XPO1, forcing the nuclear retention and activation of tumor suppressor proteins, ultimately causing apoptosis in cancer cells.STOMP is a multicenter, open-label, phase 1b/2 study ofcombination therapy with SEL, pomalidomide, and dexamethasone in patients with RRMM who previously failed lenalidomide and proteasome inhibitor therapy, two of the key therapies in myeloma. This combination almost doubled the response rate (58% vs 31%) and led to a durable response in these heavily pretreated patients(Abstract 726; Dr. NoaBiran).

Stem cell transplantation:

Despite prophylaxis, graft versus host disease (GVHD) remains a significant cause of morbidity and non-relapse mortality after allogeneic hematopoietic cell transplantation (HCT). ITA is a potent, selective Janus kinase (JAK) 1 inhibitor that has been combined safely with steroids in patients with acute GVHD. Results from a proof-of-concept study evaluating ITA + a calcineurin inhibitor (CNI) for GVHD prophylaxis, co-presented by the JTCC Bone Marrow Transplantation team, were promising, showing reduced incidence of severe GVHD, a crucial complication post-transplantation(Abstract 356; Dr. ScottRowley).

Clinical trials help move the field and establish new standards with more and more options for multiple cancer types, raising the complexity of treatment decisions. Real-world evidence (RWE) can help confirm a clinical benefit observed in a given trial and refine potential toxicities but also can help inform the best sequence of care among the many available options -- one of the biggest current challenges in oncology. Several abstracts at ASH report on this specific issue.

JTCC was part of the largest series to date comparing the first-line use of chemoimmunotherapy (CIT) to targeted therapy (e.g., ibrutinib [IBR]) in more than 500 high-risk patients with chronic lymphocytic leukemia (CLL) (Abstract 372, Dr. LoriLeslie). Results showed that patients treated with single-agentIBRtherapy had significantly longer responses and longer time to next therapy than those treated with CIT, despite similar demographics and clinical characteristics. This RWE study also demonstrated that IBR therapy provided sustained clinical benefit regardless of risk status and supports the use of IBR in the first-line setting.

JTCCs Euro-oncology group was part of a RWE study in patients with primary central nervous system lymphoma (PCNSL). Thetreatment of older patients with PCNSL is challenging due to the prevalence of comorbidities, frailty, and complexities with delivery of chemotherapy (CT). Investigators from this large series showed that outcomes in these older patients are suboptimal, and the use -- when possible -- of high-dose methotrexate truly makes a difference (as in younger patients)(Abstract 476, Dr. SamuelSinger),helping guide oncologists in this rare disease.

Burkitt lymphoma is a rare lymphoma which carries the most aggressive features and requires very intensive therapy. Authors from several institutions including JTCC reported on a new prognostic model and its impact of modern therapy(Abstract 706; Dr. TatanyaFeldman)as well as the outcome in HIV-infected patients where this lymphoma subtype is more common(Abstract 705; Dr. TatanyaFeldman).

Finally, JTCC was part of two studies reported at ASH on the impact of COVID-19 on cancer patients. The first, a global observation of more than 400 patients with CLL who were hospitalized with COVID, which showed that advanced age was the highest risk factor for mortality(Abstract 1590, Dr.LoriLeslie andDr.Michael Koropsac), while CLL treatment did not seem to have a consistent impact across cohorts. Another study looked at 89 evaluable BCL patients with COVID, with the goal of determining the impact of immunosuppression due to the lymphoma itself and/or therapies received.Survival was poor in lymphoma patients with hypertension, diabetes, or age 70 years. Interestingly, the rate of seropositivity (i.e., developing antibodies to COVID) in patients treated with monoclonal CD20 antibody therapy was significantly lower (12.5% vs 55%). Patients exposed to anti-CD20 therapy also required significantly more days to clear viral shedding (median 56 days vs 14 days). Such patientsmight be candidates for plasma therapy to help control virus expansion and induce its clearance(Abstract 2553; Dr. AndrewIp and Dr. TatanyaFeldman).

The full set of ASH data presentations by JTCC researchers is as follows:

Abstract #

Type

Title of Abstract

Authors

Presenting (PST)

48

Oral

Outcome By Mutation Status and Line of Treatment in Optic, a Dose-Ranging Study of 3 Starting Doses of Ponatinib in Patients with CP-CML

James K. McCloskey, MD

Saturday, December 5, 2020: 8:15 AM

130

Oral

Updated Results from the Phase I CRB-402 Study of Anti-Bcma CAR-T Cell Therapy bb21217 in Patients with Relapsed and Refractory Multiple Myeloma: Correlation of Expansion and Duration of Response with T Cell Phenotypes

David S. Siegel, MD

Saturday, December 5, 2020: 9:45 AM

131

Oral

Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Directed CAR T Cell Therapy, in Patients with Relapsed and Refractory Multiple Myeloma: Updated Results from Phase 1 CRB-401 Study

David S. Siegel, MD

Saturday, December 5, 2020: 10:00 AM

333

Oral

Efficacy and Safety of Venetoclax in Combination with Gilteritinib for Relapsed/RefractoryFLT3-Mutated Acute Myeloid Leukemia in the Expansion Cohort of a Phase 1b Study

James K. McCloskey, MD

Sunday, December 6, 2020: 10:15 AM

356

Oral

A Single-Arm, Open-Label Phase 1 Study of Itacitinib (ITA) with Calcineurin Inhibitor (CNI)Based Interventions for Prophylaxis of Graft-Versus-Host Disease (GVHD; GRAVITAS-119)

Scott D. Rowley, MD

Sunday, December 6, 2020: 10:15 AM

372

Oral

Clinical Outcomes Among Real-World Patients with Chronic Lymphocytic Leukemia (CLL) Initiating First-Line Ibrutinib or Chemoimmunotherapy (CIT) Stratified By Risk Status: Results from a US Retrospective Chart Review Study

Lori A. Leslie, MD

Sunday, December 6, 2020: 10:00 AM

472

Oral

Consolidation with Nivolumab and Brentuximab Vedotin after Autologous Hematopoietic Cell Transplantation in Patients with High-Risk Hodgkin Lymphoma

Tatyana A. Feldman, MD

Sunday, December 6, 2020: 2:30 PM

476

Oral

Real World (RW) Outcomes and Prognostication of Older Patients with Primary Central Nervous System Lymphoma (PCNSL) in the Contemporary Era

Samuel Singer, MD,

Samuel Goldlust, MD

Sunday, December 6, 2020: 2:00 PM

646

Oral

Updates from Ongoing, First-in-Human Phase 1 Dose Escalation and Expansion Study of TTI-621, a Novel Biologic Targeting CD47, in Patients with Relapsed or Refractory Hematologic Malignancies

Tatyana A. Feldman, MD

Monday, December 7, 2020: 12:00 PM

677

Oral

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Stacey Rifkin-Zenenberg, DO

Monday, December 7, 2020: 1:30 PM

700

Oral

Primary Analysis of Zuma-5: A Phase 2 Study of Axicabtagene Ciloleucel (Axi-Cel) in Patients with Relapsed/Refractory (R/R) Indolent Non-Hodgkin Lymphoma (iNHL)

Lori A. Leslie, MD

Monday, December 7, 2020: 1:30 PM

703

Oral

Safety, Pharmacokinetics and Activity of CA-4948, an IRAK4 Inhibitor, for Treatment of Patients with Relapsed or Refractory Hematologic Malignancies: Results from the Phase 1 Study

Lori A. Leslie, MD

Monday, December 7, 2020: 2:15 PM

705

Oral

Continued here:
John Theurer Cancer Center (JTCC) Researchers to Present 52 Abstracts across Hematological Malignancies at the 2020 American Society of Hematology...

Global Gene Therapy Market Report 2020: Non-COVID-19 Care Delivery Bears the Brunt of the Pandemic – Gene Therapy Market Slumps by -13.6% -…

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.

In a Major Setback to the Healthcare System, Non-COVID-19 Care Delivery Bears the Brunt of the Pandemic. Gene Therapy Market Slumps by -13.6%

The global market for Gene Therapy is expected to decline by -13.6% in the year 2020 and thereafter recover and grow to reach US$3.3 billion by the year 2027, trailing a post COVID-19 CAGR of 19.5% over the analysis period 2020 through 2027.

Governments worldwide are focusing all healthcare resources on fighting the global pandemic. Billions of dollars have poured into researching COVID-19 drugs, therapies and vaccines. Over US$8 billion globally excluding the U.S. has been pledged only for vaccine development. The U.S. has independently pumped billions of dollars into COVID-19 research and response. The massive reallocation of funds and reprioritization of efforts has left a glaring gap in other sectors of healthcare.

Gene therapy which holds promise for treating cancer, cystic fibrosis, heart disease, diabetes, hemophilia & AIDS, is slumping due to lack of research funds & reduced footfall of patients seeking treatment. Given the complex and fragile manufacturing and delivery system along with funding models of the industry, COVID-19 has emerged as a black swan event. Various players still find it challenging to ensure timely delivery of gene therapy to patients and clinical sites.

There are concerns regarding administration of cell and gene therapies. The chances of virus transmission, mainly to people in the high-risk group, coerced hospitals to delay or cancel appointments. In addition, travel restrictions and stay-at-home orders discouraged patients from visiting to treatment centers. Treatments intended to be delivered into ICUs are being impacted by bed reservations made for patients with COVID-19 infection.

R&D and preclinical activities are also affected by supply shortages as a result of strong demand for consumables like reagents and PPE from COVID-19 laboratories. The clinical development segment suffered the most due to concerns regarding recruitment of patients and suspension of trial enrollments for protecting participants from the risk of infection. These issues are delaying activation of new sites, prompting players to postpone new clinical trials.

However, the intensity of disruptions for cell and gene therapy trials was less in comparison to the pharmaceutical industry due to association of the former with rare and serious medical conditions, enabling participants to continue trials. While companies targeting paediatric diseases suspended trials, others dealing with oncology maintained the pace. COVID-19 has also impacted patient assessment and has made it difficult for companies to perform follow-up evaluations for trial participants. These issues are attributed to confluence of various factors like travel ban, withdrawal of several services from healthcare sites and the risk of virus transmission.

In addition, these disruptions are anticipated to threaten existence of certain cell and gene therapy companies, particularly small-scale biotech players that are in pre-commercial phase and rely on external funding. As governments, stakeholders, pharmaceutical companies and venture capitalists invest in these players on the basis of research milestones, pipeline progress and data readouts, ability of these companies to secure future funding will also be affected.

In the post COVID-19 period, growth will be led by therapy indications in the field of oncology. Gene therapies hold promise to improve the condition of patients where traditional cancer treatments such as radiation and chemotherapy are not effective. Blood and lymphatic cancers hold huge potential as gene therapies can manipulate the genetic information to target the cancerous proteins, thereby enabling the body to fight against the cancers. Oncology will remain the key area of focus for gene therapy applications. Cancer therapies represent the leading category, as is gauged through robust rise in the number of molecules being tested across numerous clinical trials.

Novartis which recently bagged the U.S. FDA approval for Kymriah, a gene therapy designed for the treatment of hematological cancer, is seeking to gain commercial approval in established and emerging countries. Similarly, Kite Pharma, the developer of YESCARTA, the first CAR T-cell therapy approved for certain types of non-Hodgkin lymphoma in adults, has formed a separate team to provide end-to-end support for its Yescarta customers including hospitals and clinics. Such efforts by developers would augment the use case of gene therapies in treatment of large B-cell lymphoma and acute lymphoblastic leukemia (ALL), the high potential cancer treatment verticals.

More developmental focus will also be shed on monogenic rare diseases which have clearer genomic targets and the unmet need in smaller patient populations. Majority gene therapies so far have come to market through accelerated review pathways of regulatory authorities. In the year 2018 alone, over 150 applications for investigational new drugs for gene therapies were filed.

In the coming years, there will be significant improvement in the number of approvals for new gene therapies. The growth is anticipated to emerge from different modalities including RNAi, ASOs and CRISPR gene editing based therapeutics which offer long term opportunities for growth. These technologies are generating much excitement for investors.

Competitors identified in this market include, among others:

Key Topics Covered:

1. MARKET OVERVIEW

2. FOCUS ON SELECT PLAYERS

3. MARKET TRENDS & DRIVERS

4. GLOBAL MARKET PERSPECTIVE

For more information about this report visit https://www.researchandmarkets.com/r/cz3rpc

Read this article:
Global Gene Therapy Market Report 2020: Non-COVID-19 Care Delivery Bears the Brunt of the Pandemic - Gene Therapy Market Slumps by -13.6% -...

Global Gene Therapy Market Report 2020-2027: Market is Projected to Reach a Revised $3.3 Billion – GlobeNewswire

November 27, 2020 04:18 ET | Source: Research and Markets

Dublin, Nov. 27, 2020 (GLOBE NEWSWIRE) -- The "Gene Therapy - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.

In a Major Setback to the Healthcare System, Non-COVID-19 Care Delivery Bears the Brunt of the Pandemic. Gene Therapy Market Slumps by -13.6%

The global market for Gene Therapy is expected to decline by -13.6% in the year 2020 and thereafter recover and grow to reach US$3.3 billion by the year 2027, trailing a post COVID-19 CAGR of 19.5% over the analysis period 2020 through 2027.

Governments worldwide are focusing all healthcare resources on fighting the global pandemic. Billions of dollars have poured into researching COVID-19 drugs, therapies and vaccines. Over US$8 billion globally excluding the U.S. has been pledged only for vaccine development. The U.S. has independently pumped billions of dollars into COVID-19 research and response. The massive reallocation of funds and reprioritization of efforts has left a glaring gap in other sectors of healthcare.

Gene therapy which holds promise for treating cancer, cystic fibrosis, heart disease, diabetes, hemophilia & AIDS, is slumping due to lack of research funds & reduced footfall of patients seeking treatment. Given the complex and fragile manufacturing and delivery system along with funding models of the industry, COVID-19 has emerged as a black swan event. Various players still find it challenging to ensure timely delivery of gene therapy to patients and clinical sites.

There are concerns regarding administration of cell and gene therapies. The chances of virus transmission, mainly to people in the high-risk group, coerced hospitals to delay or cancel appointments. In addition, travel restrictions and stay-at-home orders discouraged patients from visiting to treatment centers. Treatments intended to be delivered into ICUs are being impacted by bed reservations made for patients with COVID-19 infection.

R&D and preclinical activities are also affected by supply shortages as a result of strong demand for consumables like reagents and PPE from COVID-19 laboratories. The clinical development segment suffered the most due to concerns regarding recruitment of patients and suspension of trial enrollments for protecting participants from the risk of infection. These issues are delaying activation of new sites, prompting players to postpone new clinical trials.

However, the intensity of disruptions for cell and gene therapy trials was less in comparison to the pharmaceutical industry due to association of the former with rare and serious medical conditions, enabling participants to continue trials. While companies targeting paediatric diseases suspended trials, others dealing with oncology maintained the pace. COVID-19 has also impacted patient assessment and has made it difficult for companies to perform follow-up evaluations for trial participants. These issues are attributed to confluence of various factors like travel ban, withdrawal of several services from healthcare sites and the risk of virus transmission.

In addition, these disruptions are anticipated to threaten existence of certain cell and gene therapy companies, particularly small-scale biotech players that are in pre-commercial phase and rely on external funding. As governments, stakeholders, pharmaceutical companies and venture capitalists invest in these players on the basis of research milestones, pipeline progress and data readouts, ability of these companies to secure future funding will also be affected.

In the post COVID-19 period, growth will be led by therapy indications in the field of oncology. Gene therapies hold promise to improve the condition of patients where traditional cancer treatments such as radiation and chemotherapy are not effective. Blood and lymphatic cancers hold huge potential as gene therapies can manipulate the genetic information to target the cancerous proteins, thereby enabling the body to fight against the cancers. Oncology will remain the key area of focus for gene therapy applications. Cancer therapies represent the leading category, as is gauged through robust rise in the number of molecules being tested across numerous clinical trials.

Novartis which recently bagged the U.S. FDA approval for Kymriah, a gene therapy designed for the treatment of hematological cancer, is seeking to gain commercial approval in established and emerging countries. Similarly, Kite Pharma, the developer of YESCARTA, the first CAR T-cell therapy approved for certain types of non-Hodgkin lymphoma in adults, has formed a separate team to provide end-to-end support for its Yescarta customers including hospitals and clinics. Such efforts by developers would augment the use case of gene therapies in treatment of large B-cell lymphoma and acute lymphoblastic leukemia (ALL), the high potential cancer treatment verticals.

More developmental focus will also be shed on monogenic rare diseases which have clearer genomic targets and the unmet need in smaller patient populations. Majority gene therapies so far have come to market through accelerated review pathways of regulatory authorities. In the year 2018 alone, over 150 applications for investigational new drugs for gene therapies were filed.

In the coming years, there will be significant improvement in the number of approvals for new gene therapies. The growth is anticipated to emerge from different modalities including RNAi, ASOs and CRISPR gene editing based therapeutics which offer long term opportunities for growth. These technologies are generating much excitement for investors.

Competitors identified in this market include, among others:

Key Topics Covered:

1. MARKET OVERVIEW

2. FOCUS ON SELECT PLAYERS

3. MARKET TRENDS & DRIVERS

4. GLOBAL MARKET PERSPECTIVE

For more information about this report visit https://www.researchandmarkets.com/r/x4ed1e

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Link:
Global Gene Therapy Market Report 2020-2027: Market is Projected to Reach a Revised $3.3 Billion - GlobeNewswire

2020 Top 10 Innovations – The Scientist

We know the old saw: necessity is the mother of invention. Well, 2020 has shown us that a global pandemic is one serious mother. Typically, our Top 10 Innovations competition focuses on laboratory technologies, tools designed to plumb the mysteries of basic biology. But as biologists turned their sights to understanding SARS-CoV-2, the innovation landscape changed accordingly, with new tools developed and existing technologies bent to address the pandemic. So this year at The Scientist, our annual contest incorporates inventions aimed at understanding and ultimately solving the COVID-19 problem.

Among our independent judges picks for 2020s Top 10 Innovations were core laboratory technologiessuch as a single-cell proteome analyzer and a desktop gene synthesizeralongside pandemic-focused products, including a rapid COVID-19 test, a tool that can capture antibody profiles from the blood plasma of convalescing coronavirus patients, and a platform for characterizing glycans in the spike protein that studs the surface of SARS-CoV-2. The competition among stellar submissions was so steep that this years Top 10 actually contains 12 products, thanks to a couple of ties.

As challenging as 2020 has been for all of us, this tumultuous year has given birth to promising products and approaches for elucidating the complex world of biology. And even more than that, 2020 has shown that the scientific community, when faced with a shared problem, can rise to the challenge and come together to refocus, research, and innovate. Here, The Scientist presents the tools and technologies that make up this years Top 10 Innovations.

In late March, biotech firm AbCellera hosted a call with 40 researchers to review the data theyd collected on potential antibodies against SARS-CoV-2. Using AbCelleras high-throughput microfluidics and single-cell analysis tools to probe samples of COVID-19 patients, the companys team had deciphered the genetic sequences encoding hundreds of antibodies that might treat the disease. Sifting through all of that data by hand was tedious, though, so the team fed it into Celium, a data visualization tool that intersects more than a million high-quality data points for those antibodies to reveal which ones might work best in patients as a potential therapy. In real time, on the call, the researchers used Celium to probe those relationships and home in on the LY-CoV555 antibody that, months later, entered clinical trials as a possible COVID-19 treatment, says Maia Smith, lead of data visualization at AbCellera and creator of Celium. I think that kind of says it all.

Before Celium came on the market in 2017, scientists working with AbCellera to find antibodies would get back complex spreadsheets of data that were difficult to navigate, and it was hard to know where to start, Smith says. Using Celium, data are presented in a visual format and the tool helps you identify the right molecule for your needs, Fernando Corra, a protein engineer at Kodiak Sciences in Palo Alto, California tells The Scientist. Hes partnered with AbCellera to identify antibodies to treat retinal diseases, and says the companys package of microfluidics, single-cell analysis, and data visualization tool streamlines the process of antibody discovery in a user-friendly manner.

KAMDAR:"AbCelleras response to the pandemic underscores the real power of the Celium platform at the intersection of biology and AI to make new antibody discoveries at a blazing speed."

Since 2014, Abbotts ID NOW system has helped physicians detect influenzas A and B, strep A, respiratory syncytial virus (RSV), and most recently SARS-CoV-2, in less than 15 minutes. The toaster-size device works by heating nasal samples in an acidic solution that cracks open the envelope of theviruses, exposing their RNA, which ID NOW amplifies at a constant temperature instead of the heating and cooling cycles that PCR machines use. Gaining emergency authorization from the US Food and Drug Administration in late March, the COVID-19 ID NOW test was one of the first tests accessible to the US public.

Norman Moore, Abbotts director of scientific affairs for infectious diseases, says the tests short turnaround time is critical to stopping viral spread. Youre the most infectious early onand if we dont have that result in that timely fashion, what does it help if a molecular test comes back two weeks later? he tells The Scientist.

With more than 23,000 ID NOW devices in use in the US, mainly in urgent care clinics and pharmacies, Moore says his team is developing tests compatible with the platform for other infectious diseases, such as sexually transmitted infections.

J.D. Zipkin, chief medical officer of GoHealth Urgent Care, which partnered with San Francisco International Airport to administer the ID NOW COVID-19 test to travelers, calls the test a game changer. [Abbott] took a platform thats already really good at detecting very specific disease states and applied it to the biggest pandemic need that we have in this country, he says.

The ID NOW platform costs $4,500 and each COVID-19 test costs $40.

CRUICKSHANK-QUINN: The ability to receive COVID-19 test results from a throat or nasal swab in under 15 minutes can provide hospitals, schools, or any other institution with the ability to quickly test persons to determine those who would need to self-isolate at home. Since it is light-weight and portable it can be used in the field and at mobile sites like drive-thru testing locations."

In 2017, researchers from the New York Genome Center published a new approach called CITE-seq that allows scientists to assess proteins in individual cells at the same time they are doing single-cell transcriptomics. CITE-seq works by linking antibodies with oligonucleotides that can eventually be sequenced to reveal whether target proteins were present and joined to their corresponding antibodies. Life science company BioLegend licensed CITE-seq and developed the TotalSeqTM-C Human Universal Cocktail v1.0, a collection of 130 oligo-linked antibodies for massive screening of the cell-surface proteins of individual cells, for use on a single-cell sequencing platform from 10X Genomics.

In contrast to proteomics approaches based on visual assessment of tagged proteins, theres no theoretical limit anymore as to how many proteins you can [screen for], says BioLegends Head of Proteogenomics Kristopher Kit Nazor, adding that the company is already working to expand the number of antibodies included in the cocktail. That increases the opportunity for unbiased discovery massively.

Its groundbreaking in many ways, says immunologist and genomicist Alexandra-Chlo Villani of Massachusetts General Hospital, Harvard Medical School, and the Broad Institute of MIT and Harvard University. Like many researchers, Villani, who is one of the coordinators of the immune cell segment of the Human Cell Atlas, pivoted this year to studying COVID-19. She has already used BioLegends cocktail, launched in early August at a price of $5,350 for five single-use vials, to analyze blood samples from nearly 300 patients who tested positive for SARS-CoV-2.

When you have surface protein and RNA in the same cell, it really helps us to derive a more granular definition of the immune cells involved in response to infection, says Villani. I actually know a lot of colleagues across the United States and Europe that have used this same panel to analyze their COVID cohorts . . . which means well be able to combine all of our data and compare. And thats incredible.

MEAGHER:This is a really nice merging of next-gen sequencing as a digital readout for sequence barcodes and single-cell barcoding technology to enable single-cell quantitative proteomics."

The release of the human reference genome in 2013 was a tremendous leap forward for biology, but as far as actually representing humanity, it fell quite short. Our genomes are rife with variants not present in the reference genome, which was built from a small sampling of individuals, primarily of European descent. To account for human genetic diversity, bioinformatics firm Seven Bridges has developed a genomic analysis platform called GRAF that attempts to include all possible iterations of genetic sequences at any given locus. The resulting GRAF/Pan Genome Reference is a graph of the known variants at particular points in the genome, rather than a linear reference sequence. When genomes are aligned to the GRAF reference, any deletions, insertions, single nucleotide polymorphisms, or other variations are therefore not missed as they might be when aligned to the linear reference genome.

With the goal of boosting the presence of underrepresented groups in genomic research, Seven Bridges announced in June that access to its GRAF Germline Variant Detection Workflow and GRAF/Pan Genome Reference would be free to academic researchers. This is the first production-grade workflow that incorporates ancestry information and diversity of the human genome to provide improved variant calls and alignment, says the companys chief scientific officer, Brandi Davis-Dusenbery.

The hope is that, by accounting for that complexity in the analysis, you will see things you were missing, says Bruce Gelb, the director of the Mindich Child Health and Development Institute at the Icahn School of Medicine at Mt. Sinai. Thats been an idea floating around for a few years, but nobody prior to what Seven Bridges is doing implemented a graph-based approach that is practical. Theyre the first to do that.

Gelb has been using the GRAF platform to search for variants related to congenital heart defects and comparing those variants to what turns up when he uses traditional sequence analyses. So far, he says, it appears that GRAF is identifying some variants that would otherwise have been overlooked.

CRUICKSHANK-QUINN: The fact that SevenBridges GRAF is being made freely available to academic institutions will certainly pave the waytowards precision medicine by allowing research advancement in under-represented populations without thestruggle of cost to academic researchers."

A central challenge to delivering gene therapies to patients cells is the cost of making adeno-associated virus (AAV), a common vector for genes of interest, says Ryan Cawood, CEO of UK-based biotech company OXGENE. The first AAV gene therapy product that was approved in the EU cost a million pounds per dose, he says. If you wanted to treat a disease [with a therapy targeting a large organ] that you could apply to thousands of people, you just simply couldnt make enough of it at a cost that would make it viable.

Currently, Cawood says, batches of cultured human cells are transfected with multiple plasmids to induce them to make the AAV vectors containing a selected gene. But the plasmids are expensive to make, and the transfection process isnt very efficient. By contrast, infection with adenoviruses naturally induces cells to activate replication of AAVs. The problem is, the adenoviruses also replicate themselves and contaminate the resulting AAV product. To get around this issue, OXGENE devised a genetic switch that shuts down an adenoviruss activity halfway through its life cycle within a cell, so that it programs the cell to churn out AAV particles but not to make adenovirus. When the virus goes in, you only get AAV coming out; you dont get any more of the adenovirus coming back out, Cawood says. The company began selling its research-grade viral vector, which it calls TESSA, in September, and plans to begin offering clinical-grade material next year, he adds. The cost for the research-grade vector starts at 5,000, and depends on the size of the batch of cells to be infected.

BLAINEY:"Supports translation of gene therapies. Demonstrates the biotechnical value of biological engineering."

Biotech firm Codex DNA released the BioXp 3250 system in August 2020 as a follow-up to BioXp 3200, released in 2014. The automated platform for on-demand DNA assembly and amplification allows researchers to synthesize genes and genomes faster than ever, with the potential to accelerate the development of vaccines, diagnostics, and treatments, says Peter Duncan, director of product management at Codex DNA. The equipment can be used on cancer cells or a variety of infectious agents, including SARS-CoV-2.

Without BioXp 3250 or its predecessor, labs that want to synthesize DNA fragments, clones, or whole genomes have to send samples out to be processed by a third party. In addition to having to deal with transit, such processing could take weeks or months. With the BioXp 3250, priced at $100,000, DNA sequences up to 7,000 base pairs in length can be assembled in a matter of days, with the push of a button.

Rather than having to code genetic script on a computer for specific experiments, customers can order a module that comes in about two days, ready to go. The module has a barcode containing all the necessary information; when scanned by the device, instructions for synthesizing the desired DNA are uploaded. A lab technician merely needs to insert the module into the device and press start, Duncan says.

The BioXp has enabled us to perform simple subcloning steps hands-free, Mark Tornetta, VP of Biologics Discovery at Tavotek Biotherapeutics, tells The Scientist in an email, describing how the lab uses the device to generate NGS libraries. All of these methods [that are run] on the BioXP save us time and cost to perform.

BLAINEY:"Democratizing gene synthesis by placing capability in individual labs for faster turnaround and lower costs at high throughput."

The Single-Cell Intracellular Proteome solution from IsoPlexis grew out of several labs at Caltech, all seeking better ways to monitor protein-protein interactions in cancer cells with the goal of developing targeted treatments. With traditional methods such as Western blot, mass spectrometry, and flow cytometry, only a couple of protein types can be tracked at a given time. With Isoplexiss system, launched in July, researchers can monitor 30 or more protein pathways, with results available on the same day.

With previous technology, phosphorylation was used to identify the function of the individual proteins, with no insight as to how they work together. The Single-Cell Intracellular Proteome reveals the function the same way, but is also able to provide the context of entire protein signaling pathways, uncovering how the network operates as a whole.

Understanding the entire network of cellular pathways allows researchers to better understand the downstream effects of aberrant cells, says Sean Mackay, CEO and cofounder of IsoPlexis. In cancers, he adds, this approach helps evaluate the efficacy of targeted treatments such as antibody therapies or small-molecule drugs.

Such pathways basically define how cells are activated, [which] is particularly important for cancer, where activated phosphoprotein signaling is not only a hallmark of cancer, says James Heath, who ran the Caltech lab that created the technology eight years ago, but is a major focus of targeted inhibitors.

MEAGHER:"The Single-Cell IntracellularProteome solution uses innovative microfluidics to scale down what looks like well-established ELISA chemistry down to the level of single cells."

Scientists have used intravenous immunoglobulin (IVIG) to treat immunodeficient or immunosuppressed patients and convalescent plasma to treat infectious diseases for more than a century. And plasma is one of many treatments now being tried for COVID-19. But biological samples drawn from donors are not the most standardized therapeutics. Enter GigaGens Surge platform, which uses single-cell sequencing to capture and recreate libraries of antibodies from blood donors. To create these libraries, the company runs donors blood samples through the Surge platform to isolate individual antibody-producing B cells into microdroplets and extract the RNA that encodes the antibodies. From these genetic sequences they can create a blueprint of that persons immune system, says GigaGen CEO David Johnson.

Company researchers then select some of those antibodies to engineer in mammalian cells to create a recombinant antibody treatment, which they say is much more potent than convalescent plasma or IVIG, based on in vitro experiments and tests in animal models. GigaGen does not currently plan to sell Surge, but rather has been using the platform to develop treatments for cancers, immunodeficiency disorders, and, most recently, COVID-19. GigaGen hopes to start clinical trials on their COVID-19 treatment, which uses more than 12,500 antibodies from 16 donors, in early 2021. The goal of Surge is to tease apart the complexity of the immune system, says Johnson, and then tailor antibody treatments that elicit the strongest response.

Fred and Vicki Modell, who founded the Jeffrey Modell Foundation after their son Jeffrey died at 15 due to complications from primary immunodeficiency, say they have been searching for an alternative to IVIG, which is sometimes in short supply and can lead to side effects in many patients. [GigaGen] is giving the greatest gift of alltheyre giving hope to [immunodeficient] patients, Fred Modell says.

CRUICKSHANK-QUINN: "By combining single-cell emulsion droplet microfluidics technology, genomics, and protein library engineering, this antibody drug therapy, if successful, could revolutionize COVID-19 treatment as well as treatments for many different diseases."

A few years ago, 10X Genomics launched an assay, ATAC-seq, to identify regions of open chromatin in single cells; the product won a spot in The Scientists 2019 Top 10 Innovations. According to product marketing manager Laura DeMare, it wasnt long before customers were clamoring for more, with feedback to the effect of, This is great, but wed really love to get the gene expression information and the ATAC-seq information in the same cell. In September, 10X rolled out Chromium Single Cell ATAC + Gene Expression, which harvests both epigenetic and gene expression data from individual nuclei.

The platform tags mRNA and open chromatin fragments from each nucleus with DNA barcodes, DeMare explains, and the nucleic acids are then amplified and analyzed. You can begin to actually link which regulatory elements in the genome are turning on or off genes, she says. It costs approximately $2,400 per reaction for the reagents and a microfluidic chip.

Ansu Satpathy, an immunologist at Stanford University School of Medicine and a former postdoc of ATAC-seq codeveloper Howard Chang, tells The Scientist that hes using the new assay to investigate the effects of epigenetic changes associated with T cell exhaustion in tumor samples biopsied from cancer patients. When exhausted, T cells become less effective at battling cancer, and what were doing now with the RNA and ATAC method combined is asking, How do each of those molecular switches regulate genes that lead to this dysfunctional outcome in the cell? Satpathy says.

KAMDAR:"This approach allows, for the first time, the simultaneous profiling of the epigenome and transcriptome from the same single cell, enabling a better understanding of cell functionality."

Over the last several years, single-cell transcriptomics has provided a wealth of gene expression information for individual cells and cell types. Now, 10X Genomics advances the newer technology of spatial transcriptomics, which provides whole transcriptome data for just one or a few cells, and reveals exactly where in a tissue sample that gene expression is taking place. The Visium Spatial Gene Expression Solution, launched in October 2019, exposes 55-micrometer areas at 5,000 locations within a tissue sample to mRNA-binding oligonucleotides, and overlays the resulting gene expression data with histological images.

The technology was developed and originally marketed by Swedish company Spatial Transcriptomics, which 10X Genomics acquired in 2018. Then 10X developed the product further before launching Visium last year. The Visium Spatial Gene Expression Solution, which sells for $1,000 per sample, has smaller and more densely packed spotsand five times more of themthan it did when the company inherited it, says Nikhil Rao, 10X Genomicss director of strategic marketing for the spatial platform. This increases resolution, he explains. We also improved the sensitivity of the assay dramatically, being able to pick up tens of thousands of unique molecular identifiers per spot.

Rao says that many of Visiums users focus on neuroscience, studying neurodegenerative diseases, for example. But the product is also being used in developmental biology, oncology, and immunology. Johns Hopkins University computational biologist Elana Fertig has used Visium to understand how a cancer can resist treatment. By virtue of having the spatial information of these cells, you can really figure out the molecular mechanisms where they interact directly, because you can see if the cells are interacting physically, she explains.

MEAGHER: "This is another frontier in biology: not just single-cell or few-cell gene expression, but now collecting gene expression data with spatial resolution at the level of a few cells."

While CRISPR-based genome editing has become a widely used technique in labs all over the globe, there are research questions that require a scale of nucleotide tinkering that can be cumbersome, if not prohibitive, for some labs. Inscripta Inc.s Onyx Digital Genome Engineering Platform offers a solutionfully automated genome-engineered libraries with hundreds of thousands of single edits in microbial genomes. The benchtop device, which launched in October 2019 and sells for $347,000, allows users to plant desired variants in the DNA of E. coli bacteria and S. cerevisiae yeast, and the instrument takes care of the rest.

The platform combines everything from the algorithms for optimizing the editing process to the microfluidics for handling cells to the reagents themselves. Biologists dont have to worry about the technical optimization anymore and can go ahead and focus on any problem in biology now, says Nandini Krishnamurthy, the vice president of applications development at Inscripta.

Shelley Copley, a molecular biologist at the University of Colorado Boulder, is an early tester of Onyx. Shes using it to examine the effects of synonymous mutations, those that dont change the resulting protein, on fitness inE. coli. The high-throughput part of it is critical to be able to address this, she says. Rather than attempt to engineer each mutation she wants to examine one by one, Onyx enables Copley to generate all 50,000 variants. Her team can then move straight to the fitness assays. I dont know of any other technology that can do it.

KAMDAR:"CRISPR is a powerful tool for editing genomes and allowing functional assessments that can elucidate causality and improve our understanding of genome biology. But those outcomes will not be achieved without overcoming a number of the technical and scalability challenges. This is what the Onyx Digital Genome Engineering Platform enables."

John McLean, a bioanalytical chemist at Vanderbilt University, wants to know exactly whats in a puff of gas, down to a vaporized blood or tissue samples very last lipid molecule. For years, he has used mass spectrometry to catalog compounds in a sample by weight. Sometimes different molecules can have the same mass and the same atomic composition, making it hard to distinguish them. Ion mobility separation runs gas samples down meter-long tubes to differentiate molecules by shape and structure, getting around the mass issue. But because the technique was designed decades ago, it hasnt achieved the same resolution as mass spectrometry. To achieve a similar resolution, the ion separation instrument would need a 13-meter tube.

Making a linear tube that length is impractical due to constraints on lab space. So a few years ago, Richard Smith of Pacific Northwest National Laboratory and colleagues began brainstorming ways to get ions to turn corners. That discussion led to the development of MOBILions SLIM, or Structures for Lossless Ion Manipulation, an instrument with a 13-meter track cut as switchbacks in two circuit boards that fit in a 3-meter-long box; the device provides data on the size and shape of compounds in samples in minutes. SLIM reveals the unseen, says Laura Maxon, MOBILions head of business development and corporate strategy, without the sacrifice of time. This first iteration of SLIM, which MOBILion began deploying as a Beta version to early adopter collaborators the second quarter of 2020, is built for scientists in a pharmaceutical or clinical research academic environment. The price is competitive with existing technologies, she notes, and the company plans to design the instrument for use in the clinic to identify biomarkers of disease.

What were seeing today, from MOBILion on SLIM, is just the tip of the iceberg, McLean says. Theres a lot of untapped potential . . . from an analytical standpoint, so people should really expect huge advances for these technologies.

BLAINEY: "Ion-selective chromatography is central to biochemistry. Nice integration of microelectronic technology with biotechnology. "

Paul Blainey

Associate professor of biological engineering at MIT and a core member of the Broad Institute of MIT and Harvard University. The Blainey lab integrates new microfluidic, optical, molecular, and computational tools for application in biology and medicine. The group emphasizes quantitative single-cell and single-molecule approaches, aiming to enable studies that generate data with the power to reveal the workings of natural and engineered biological systems across a range of scales. Blainey did not consider products submitted by 10X Genomics in his rankings due to his financial ties to the company.

Charmion Cruickshank-Quinn

Application scientist at Agilent Technologies.Previously, she was a postdoctoral fellow at the University of Colorado Denver - Anschutz Medical Campus, a research fellow at National Jewish Health in Denver, and a graduate student at the State University of New York at Buffalo, where she worked in the instrument center.

Kim KamdarManaging partner at Domain Associates, a healthcare-focused venture fund creating and investing in biopharma, device, and diagnostic companies. She began her career as a scientist and pursued drug-discovery research at Novartis/Syngenta for nine years.

Robert Meagher

Principal member of Technical Staff at Sandia National Laboratories. His main research interest is the development of novel techniques and devices for nucleic acid analysis, particularly applied to problems in infectious disease, biodefense, and microbial communities. Most recently this has led to approaches for simplified molecular diagnostics for emerging viral pathogens which are suitable for use at the point-of-need or in the developing world. Meaghers comments represent his professional opinion but do not necessarily represent the views of the US Department of Energy or the United States government.

Editors Note: The judges considered dozens of entries submitted for a variety of life science products by companies and users. The judging panel evaluated submissions with only basic instructions fromThe Scientist, and its members were invited to participate based on their familiarity with life science tools and technologies. With the exception of Paul Blainey, who has financial ties to 10X Genomics and therefore did not consider that firm's products in his rankings, they have no financial ties to the products or companies involved in the competition. In this issue ofThe Scientist, any advertisements placed by winners named in this article were purchased after our independent judges selected the winning products and had no bearing on the outcome of the competition.

Corrections (December 1): The original version of this story stated that GigaGen's Surge platform captured antibodies from samples that came from plasma donors. They were, in fact, blood donors. Changes were also made to clarify the title of AbCellara's Maia Smith and the nature of Celium and collaborations surrounding the tool.The Scientistregrets these errors.

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2020 Top 10 Innovations - The Scientist

Global Regenerative Medicine Market to Rise Impressively at 26.1% CAGR and Hit USD 151,949.5 Million by 2026; Integra’s Latest Product Offerings to…

December 01, 2020 02:25 ET | Source: Fortune Business Insights

Pune, India, Dec. 01, 2020 (GLOBE NEWSWIRE) -- The global regenerative medicine market size is likely to expand considerably in the coming years due to growing applications in the treatment of chronic diseases. The market was valued at US$ 23,841.5 Million in 2018. Fortune Business Insights states that the market will reach US$ 151,949.5 Million by the end of 2026, thereby exhibiting a CAGR of 26.1%. North America generated maximum revenue of US$ 9,128.2 Million in 2018 and is expected to dominate the global regenerative medicine market throughout the forecast period. Due to presence of substantial number of key market players based in U.S., presence of research institutes involved in development of novel therapeutics and availability of advanced technologies are attributive to the high number of clinical trials in North America.

Key Industry Developments:

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Increasing Investment in R&D of Regenerative Medicine to Enable Growth

The growing investment in research and development of regenerative medicine has made a significant contribution to the growth of the global market. The increasing investment from private as well as government organizations has had a positive impact on the global market. IN March 2018, SanBio collaborated with Hitachi Chemical Advanced Therapeutics Solutions for developing regenerative medicine across a wide range of applications.

Novartis Receives EU Approval for Luxturna

The severity of chronic diseases has led to the demand for efficient medicines. The ability of regenerative medicine to treat severe life-threatening diseases in an efficient manner has created a huge demand for the products across the world. Increasing drug approvals have contributed to the rising uptake for regenerative medicines.

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In 2018, Novartis received usage approval from the European Union for its latest regenerative medicine Luxturna. The drug was used to treat and restore sight for people with vision impairment. Luxturna was widely useful in treatment of rare retinal diseases.

Integra LifeSciences Latest Product Offering Will Favor Market Growth

The advancements in regenerative medicine have fueled their demand across the world. Increasing product launches have contributed to the rising uptake of regenerative medicine across the world. In 2017, Integra LifeSciences announced the launch of Integra Dermal Regeneration Template Single Layer Thin.

Fortune Business Insights some of the leading companies that have made significant growth contributions to the global market. Besides this, the report identifies some of the attractive business strategies that have been adopted by renowned companies in the world.

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List of the leading companies that are operating in the Regenerative Medicine Market:

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Global Regenerative Medicine Market Segmentation:

By Product

Cell Therapy

Gene Therapy

Tissue Engineering

Platelet Rich Plasma

By Application

Orthopedics

Wound Care

Oncology

Others

By Distribution Channel

Hospitals

Clinics

Others

By Geography

North America (USA and Canada)

Europe (UK, Germany, France, Italy, Spain, Scandinavia and Rest of Europe)

Asia Pacific (Japan, China, India, Australia, Southeast Asia and Rest of Asia Pacific)

Latin America (Brazil, Mexico and Rest of Latin America)

Middle East & Africa (South Africa, GCC and Rest of Middle East & Africa)

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SECONDARY DATA SOURCES THAT WE REFER TO:

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Global Regenerative Medicine Market to Rise Impressively at 26.1% CAGR and Hit USD 151,949.5 Million by 2026; Integra's Latest Product Offerings to...