Category Archives: Stem Cell Treatment


Global Stem Cell Therapy Market 2020 Industry Analysis, Size, Share, Growth, Trend and Forecast to 2025 – re:Jerusalem

MarketsandResearch.biz has published the latest market research study on Global Stem Cell Therapy Market 2020 by Company, Type and Application, Forecast to 2025 which investigates a few critical features of the market such as industry condition, division examination, market insights. The report studies the global Stem Cell Therapy market share, competition landscape, market share, growth rate, future trends, market drivers, opportunities and challenges, sales channels. The report has referenced down to earth ideas of the market in a straightforward and unassuming way in this report. The research contains the categorization of the market by top players/brands, region, type, and end-user. The report exhaustive essential investigation of current market trends, opportunities, challenges, and detailed competitive analysis of the industry players in the market.

The research report has comprehensively included numbers and figures with the help of graphical and pictorial representation which embodies more clarity on the global Stem Cell Therapy market. Then the report delivers key information about market players such as company overview, total revenue (financials), market potential, global presence, as well as market share, prices, production sites and facilities, products offered, and strategies adopted by them. Market status and outlook of global and major regions, from angles of players, countries, product types, and end industries have been analyzed.

NOTE: Our report highlights the major issues and hazards that companies might come across due to the unprecedented outbreak of COVID-19.

DOWNLOAD FREE SAMPLE REPORT: https://www.marketsandresearch.biz/sample-request/95817

Key strategic manufacturers included in this report: Osiris Therapeutics, Molmed, JCR Pharmaceutical, NuVasive, Anterogen, Chiesi Pharmaceuticals, Medi-post, Pharmicell, Takeda (TiGenix)

Market Potential:

Key market vendors have been predicted to obtain the latest opportunities as there has been an increased emphasis on spending more on the work of research and development by many of the manufacturing companies. Also, many of the market contenders are forecasted to make a foray into the emerging economies to find new opportunities. The global Stem Cell Therapy market has gone through rapid business transformation by good customer relationships, drastic and competitive growth, significant changes within the market, and technological advancement in this market.

Geographically, this report is segmented into several key countries, with market size, growth rate, import and export of in these countries from 2015 to 2020, which covering: North America (United States, Canada and Mexico), Europe (Germany, France, United Kingdom, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), Middle East & Africa (Saudi Arabia, UAE, Egypt and South Africa)

The market can be segmented into product types as: Autologous, Allogeneic

The market can be segmented into applications as: Musculoskeletal Disorder, Wounds & Injuries, Cornea, Cardiovascular Diseases, Others

ACCESS FULL REPORT: https://www.marketsandresearch.biz/report/95817/global-stem-cell-therapy-market-2020-by-company-type-and-application-forecast-to-2025

Reasons for Buying Market Report:

Customization of the Report:

This report can be customized to meet the clients requirements. Please connect with our sales team (sales@marketsandresearch.biz), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

About Us

Marketsandresearch.biz is a leading global Market Research agency providing expert research solutions, trusted by the best. We understand the importance of knowing what global consumers watch and buy, further using the same to document our distinguished research reports. Marketsandresearch.biz has worldwide presence to facilitate real market intelligence using latest methodology, best-in-class research techniques and cost-effective measures for worlds leading research professionals and agencies. We study consumers in more than 100 countries to give you the most complete view of trends and habits worldwide. Marketsandresearch.biz is a leading provider of Full-Service Research, Global Project Management, Market Research Operations and Online Panel Services.

Contact Us Mark Stone Head of Business Development Phone: +1-201-465-4211 Email: sales@marketsandresearch.biz Web: http://www.marketsandresearch.biz

See the article here:
Global Stem Cell Therapy Market 2020 Industry Analysis, Size, Share, Growth, Trend and Forecast to 2025 - re:Jerusalem

Taking Gene Editing to the Brain, the Immune System, and More – Tufts Now

One of the most remarkable recent advances in biomedical research has been the development of highly targeted gene-editing methods such as CRISPR that can add, remove, or change a gene within a cell with great precision. The method is already being tested or used for the treatment of patients with sickle cell anemia and cancers such as multiple myeloma and liposarcoma, and today, its creators Emmanuelle Charpentier and Jennifer Doudna received the Nobel Prize in chemistry.

While gene editing is remarkably precise in finding and altering genes, there is still no way to target treatment to specific locations in the body. The treatments tested so far involve removing blood stem cells or immune system T cells from the body to modify them, and then infusing them back into a patient to repopulate the bloodstream or reconstitute an immune responsean expensive and time-consuming process.

Building on the accomplishments of Charpentier and Doudna, Tufts researchers have for the first time devised a way to directly deliver gene-editing packages efficiently across the blood brain barrier and into specific regions of the brain, into immune system cells, or to specific tissues and organs in mouse models. These applications could open up an entirely new line of strategy in the treatment of neurological conditions, as well as cancer, infectious disease, and autoimmune diseases.

A team of Tufts biomedical engineers, led by associate professor Qiaobing Xu, sought to find a way to package the gene editing kit so it could be injected to do its work inside the body on targeted cells, rather than in a lab.

They used lipid nanoparticles (LNPs)tiny bubbles of lipid molecules that can envelop the editing enzymes and carry them to specific cells, tissues, or organs. Lipids are molecules that include a long carbon tail, which helps give them an oily consistency, and a hydrophilic head, which is attracted to a watery environment.

There is also typically a nitrogen, sulfur, or oxygen-based link between the head and tail. The lipids arrange themselves around the bubble nanoparticles with the heads facing outside and the tails facing inward toward the center.

Xus team was able to modify the surface of these LNPs so they can eventually stick to certain cell types, fuse with their membranes, and release the gene-editing enzymes into the cells to do their work.

Making a targeted LNP takes some chemical crafting.

By creating a mix of different heads, tails, and linkers, the researchers can screen first in the laba wide variety of candidates for their ability to form LNPs that target specific cells. The best candidates can then be tested in mouse models, and further modified chemically to optimize targeting and delivery of the gene-editing enzymes to the same cells in the mouse.

We created a method around tailoring the delivery package for a wide range of potential therapeutics, including gene editing, said Xu. The methods draw upon combinatorial chemistry used by the pharmaceutical industry for designing the drugs themselves, but instead we are applying the approach to designing the components of the delivery vehicle.

In an ingenious bit of chemical modeling, Xu and his team used a neurotransmitter at the head of some lipids to assist the particles in crossing the blood-brain barrier, which would otherwise be impermeable to molecule assemblies as large as an LNP.

The ability to safely and efficiently deliver drugs across the barrier and into the brain has been a long-standing challenge in medicine. In a first, Xus lab delivered an entire complex of messenger RNAs and enzymes making up the CRISPR kit into targeted areas of the brain in a living animal.

Some slight modifications to the lipid linkers and tails helped create LNPs that could deliver into the brain the small molecule antifungal drug amphotericin B (for treatment of meningitis) and a DNA fragment that binds to and shuts down the gene producing the tau protein linked to Alzheimers disease.

More recently, Xu and his team have created LNPs to deliver gene-editing packages into T cells in mice. T cells can help in the production of antibodies, destroy infected cells before viruses can replicate and spread, and regulate and suppress other cells of the immune system.

The LNPs they created fuse with T cells in the spleen or liverwhere they typically resideto deliver the gene-editing contents, which can then alter the molecular make-up and behavior of the T cell. Its a first step in the process of not just training the immune system, as one might do with a vaccine, but actually engineering it to fight disease better.

Xus approach to editing T cell genomes is much more targeted, efficient, and likely to be safer than methods tried so far using viruses to modify their genome.

By targeting T cells, we can tap into a branch of the immune system that has tremendous versatility in fighting off infections, protecting against cancer, and modulating inflammation and autoimmunity, said Xu.

Xu and his team explored further the mechanism by which LNPs might find their way to their targets in the body. In experiments aimed at cells in the lungs, they found that the nanoparticles picked up specific proteins in the bloodstream after injection.

The proteins, now incorporated into the surface of the LNPs, became the main component that helped the LNPs to latch on to their target. This information could help improve the design of future delivery particles.

While these results have been demonstrated in mice, Xu cautioned that more studies and clinical trials will be needed to determine the efficacy and safety of the delivery method in humans.

Mike Silver can be reached at mike.silver@tufts.edu.

Read this article:
Taking Gene Editing to the Brain, the Immune System, and More - Tufts Now

Antibody cocktail given to Trump is controversial, and not only because its still under trial – ThePrint

Text Size: A- A+

New Delhi: US President Donald Trump was given an array of drugs to treat his Covid-19 symptoms. These included remdesivir, steroid dexamethasone, and an experimental cocktail of antibodies by biotech major Regeneron Pharmaceuticals.

While remdesivir and dexamethasone have been known to be used for mild and severe cases of Covid, the eight grams of experimental cocktail Trump was given generated controversy partly because it is still under trial, but also because it uses a method of research that has stirred Trumps voter base in the past: embryonic stem cell research.

In a statement on the research in April, the company said: Regeneron uses a wide variety of research tools and technologies to help discover and develop new therapeutics. Stem cells are one such tool. It added that there are limited research efforts employinghuman embryonic stem cells.

Trump has repeatedly identified himself as pro-life, a belief that nothing should harm life once it has been conceived. This is in line with the conservatives, who are often anti-abortion because of their Christian beliefs and make up a chunk of the Republican vote. In 2019, Trump even clamped down on the use of foetal tissue in scientific research.

In March this year, the Washington Post reported that a scientist was forced to abandon his research into a coronavirus treatment because of Trumps foetal tissue laws.

Also read:Trump leaves hospital but White House to restrict access to him during recovery

Embryonic stem cell research involves using cells at the embryo stage, when the human egg has been fertilised by sperm. The fertilised egg is referred to as an embryo till the 9th week, after which it is called a foetus.

Embryonic stem cells are sought after in scientific research because they can develop into all cell types of the body. While embryonic cells from abortions are commonly used, Regeneron clarified that it uses embryos that have been created solely through in vitro fertilization.

In 2001, the George Bush administration restricted federal funding for embryonic stem cell research. Bush, a conservative and Christian, said at the time, At its core, this issue forces us to confront fundamental questions about the beginnings of life and the ends of science.

This was changed only after Barack Obama came to power in 2008.

After Trump took the Oval Office in 2016, a ban or restriction on the use of embryonic and foetal tissue research was always on the cards. In 2019, the government officially imposed restrictions on the federal funding of research requiring foetal tissue.

According to the new rules, scientists must detail why exactly they require fetal tissue and how it will be obtained.

In its statement, Regeneron said it uses embryonic cells that are approved for research use by the National Institutes of Health and adheres to federal and state laws and regulations.

The cocktail given to Trump includes a mix of two potent antibodies directed against a spike protein found on the novel coronavirus surface. The therapy is still under trial. Regeneron released preliminary results last month saying it was safe, reduces viral load, and eases symptoms.

While recovering this week, Trump put out a spate of Tweets about the therapy. One of them said, PRO LIFE! VOTE!.

But Twitter users were quick to point out the hypocrisy.

Also read:Trump intends to be ready to debate Biden next week but will oppose a mute button

Subscribe to our channels on YouTube & Telegram

Why news media is in crisis & How you can fix it

India needs free, fair, non-hyphenated and questioning journalism even more as it faces multiple crises.

But the news media is in a crisis of its own. There have been brutal layoffs and pay-cuts. The best of journalism is shrinking, yielding to crude prime-time spectacle.

ThePrint has the finest young reporters, columnists and editors working for it. Sustaining journalism of this quality needs smart and thinking people like you to pay for it. Whether you live in India or overseas, you can do it here.

Support Our Journalism

Read more here:
Antibody cocktail given to Trump is controversial, and not only because its still under trial - ThePrint

The Global Stem Cell Therapy Market Growth To 2023 Will Be Driven By Increasing Prevalence Of Chronic Diseases – Press Release – Digital Journal

"Global Stem Cell Therapy Market, Forecast Market Size, 2019 2023, $ Billion"

The Business Research Companys Global Stem Cell Therapy Market Report 2020-30: Covid 19 Growth And Change; Segments Covered: 1) By Type: Allogeneic Stem Cell Therapy, Autologous Stem Cell Therapy 2) By Cell Source: Adult Stem Cells, Induced Pluripotent Stem Cells, Embryonic Stem Cells 3) By Application: Musculoskeletal Disorders, Wounds and Injuries, Cancer, Autoimmune Disorders, Others 4) By End-User: Hospitals, Clinics

The global stem cell therapy market is expected to decline from $8.73 billion in 2019 to $8.62 billion in 2020 at a compound annual growth rate (CAGR) of -1.24%. The decline is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The global stem cell therapy market is then expected to recover and reach $14.76 billion in 2023 at a CAGR of 19.62%.

The rising prevalence of chronic diseases contributed to the stem cell therapy market growth. Long working hours, limited physical activity, and unhealthy eating and drinking habits contribute to the prevalence of chronic diseases among people, thus driving the need for stem cell therapy. According to a United Nations article, by 2030, the proportion of global deaths due to chronic diseases is expected to increase to 70% of total deaths. The global burden of chronic diseases is expected to reach about 60%. The rising prevalence of chronic diseases is expected to drive the global stem cell therapy market growth.

Companies in the stem cell therapy market are increasingly investing in strategic partnerships. The strategic partnership is a mutually beneficial agreement between two companies that do not compete directly with each other. For instance, in September 2018, CRISPR Therapeutics, a biotechnology company that develops transformative medicine using the gene-editing platform for serious diseases, and ViaCyte, a California-based regenerative medicine company, collaborated for the development and commercialization of allogeneic stem cell therapies for diabetes treatment.

The high cost of stem cell therapy is expected to limit the growth of the stem cell therapy market. The pressure to contain costs and demonstrate value is widespread. Political uncertainty and persistent economic stress in numerous countries are calling into question the sustainability of public health care funding. In less wealthy countries, the lack of cost-effective therapies for chronic diseases has impacted the health conditions of the population and has led to a low average life expectancy. According to the DVCSTEM, the average cost of stem cell therapy in the USA is between $20,000 to $25,000, in Mexico, it is $33,000, in Central America, it is $30,000, and in Asia, it is $50,000, thus restraining the growth of the market.

The stem cell therapy market consists of the sales of stem cell therapy and related services by entities (organizations, sole traders, and partnerships) that provide stem cell therapy. Stem cell therapy, also known as regenerative medicine, promotes the repair response of a diseased patient, or a dysfunctional or injured tissue using stem cells or their derivatives.

Here Is A List Of Similar Reports By The Business Research Company:

Cell Therapy Market Global Report 2020-30: Covid 19 Growth And Change

Stem Cell/Cord Blood Banking Global Market Report 2020-30: Covid 19 Growth And Change

Plasma Therapy Global Market Report 2020-30: Covid 19 Growth And Change

Interested to know more about The Business Research Company? The Business Research Company is a market intelligence firm that excels in company, market, and consumer research. Located globally it has specialist consultants in a wide range of industries including manufacturing, healthcare, financial services, chemicals, and technology.

Media Contact Company Name: The Business Research Company Contact Person: Oliver Guirdham Email: Send Email Phone: +44 20 7193 0708 Address: City: London State: Greater London Country: United Kingdom Website: https://www.thebusinessresearchcompany.com/

Originally posted here:
The Global Stem Cell Therapy Market Growth To 2023 Will Be Driven By Increasing Prevalence Of Chronic Diseases - Press Release - Digital Journal

Stem cell therapy for blood cancer patients at NIMS – The Hindu

In a big blessing for blood cancer patients requiring stem cell therapy, the State government has established a Centre for Stem Cell and Regenerative Medicine in the Nizams Institute of Medical Sciences (NIMS). The facility will particularly help those from the lower strata of the society who cannot afford corporate medical care.

The centre was inaugurated on Friday by Health Minister Eatala Rajender in the presence of NIMS Director K. Manohar and Superintendent N. Satyanarayana. The centre will provide a ray of hope to blood cancer patients from poor families, those covered under Aarogyashri scheme, requiring stem cell therapy as they would be treated at the centre free of cost, the Minister saidafter dedicating the stem cell and molecular lab as part of the centre to the people.

With the opening of the stem cell therapy centre, NIMS has grown into one of the major hospitals in the country, Mr Rajender stated samples of blood cancer patients which were sent to Delhi for diagnosis earlier can now be done here itself. The success rate of NIMS in stem cell therapy among kidney and heart transplantation patients was very high, he said, but some services were stopped due to rise in COVID-19 cases. However, all services would be resumed within a week with the COVID spread now under control.

Stating that living with COVID-19 would be a new norm as the society was doing with dengue, viral fever, swine flu and malaria, the Minister said and asserted that as per ICMR statistics, about 40 lakh people in Telangana had developed antibodies to COVID and the fleecing of patients families by corporate hospitals in the name of plasma therapy and some costly injections was wrong.

Mr Rajender admitted that COVID treatment in NIMS had impacted the treatment of other patients, particularly those coming in emergency health conditions, and made it clear that outpatient services would be scaled up soon with OP and Critical Care blocks being set up with an investment of 250 crore. Chief Minister K. Chandrasekhar Rao would lay the foundation stone for the new blocks soon to scale up the outpatient services from 2,500 a day in the past to 5,000 a day.

Excerpt from:
Stem cell therapy for blood cancer patients at NIMS - The Hindu

First Man Cured of AIDS Dies From Cancer – The Keystone Newspaper

Featured

By Dylan Adams News Editor

Timothy Ray Brown, the first known person to be cured of HIV, died on Sept. 29 at age 54 after battling cancer.

Timothy Ray Brown, a figurehead in the AIDS and HIV community, passed away surrounded by friends after a five-month battle with leukemia, stated Tim Hoeffgen, Browns partner.

Brown received a positive HIV diagnosis in 1995 while studying in Berlin.

In 2006, Brown was diagnosed with acute myeloid leukemia, which is a cancer that builds in the bone marrow and blood interfering with blood cell production. After bouts of infections from several rough rounds of chemotherapy, Browns leukemia came out of remission.

Due to leukemia in his bones, Brown required a stem cell transplant, a process that allows healthy stem cells to be introduced into a host to stimulate the immune system and healthy bone marrow growth. At the time, the survival rates for stem cell transplant were around fifty percent.

Doctors found a match to Browns genetic type, a donor with the CCR5 Delta 32 mutation, a protein that acts as a doorway to stop the HIV from infecting new cells. Three months after Brown stopped taking his HIV medication, doctors found he no longer had HIV in his blood.

After another round of stem cell treatment in February of 2008, Brown went through several near-death complications, almost going blind and becoming paralyzed but slowly recovering. His body was still successfully fighting off HIV.

In July 2012, the Timothy Ray Brown Foundation was created during the World AIDS Conference in Washington, DC. This foundation was built for Brown to show his support and work with medical institutions and scientists to develop a unifying cure and vaccination against HIV.

Brown would often donate large amounts of blood and tissue samples to researchers in the hope of progressing closer towards an HIV cure. According to his partner, Hoeffgen, Tims lifework was to tell his story about his HIV cure and become an ambassador of hope to those in need.

Doctors have since used Brown as a blueprint to work on a potential cure and vaccine for HIV. Most notably for the second person to ever be cured of HIV the London Patient, Adam Castillejo who went through similar stem cell transplants in 2019 before coming forward to the public.

Like Loading...

See original here:
First Man Cured of AIDS Dies From Cancer - The Keystone Newspaper

Mesoblast hit by FDA rejection, request to run another trial – FierceBiotech

The FDA has issued (PDF) a complete response letter to Mesoblasts request for approval of allogeneic cell therapy remestemcel-L in children with steroid-resistant graft-versus-host disease. FDA officials want to see data from at least one additional clinical trial before granting full authorization, but Mesoblast is still hoping to snag an accelerated approval using its existing data.

Evidence of the FDAs concerns with Mesoblasts remestemcel-L filing emerged in August when the agency released a briefing document to inform a discussion of the application by the Oncologic Drugs Advisory Committee (ODAC). The FDA cited multiple perceived shortcomings of the filing, arguing for example that the ability of the mesenchymal stem cell therapy to reduce inflammation as measured by inflammatory biomarkers in humans receiving the product has not been demonstrated.

Despite the FDAs concerns, ODAC members voted nine to one that the data support the efficacy of remestemcel-L in children with steroid-resistant graft-versus-host disease. The positive ODAC vote sent shares in Mesoblast, which had been depressed by the briefing document, up 50%.

Join a panel of experts as they discuss the rigor involved in optimizing and scaling a reproducible serological assay for COVID-19.

However, the ODAC vote was advisory, and the FDA, in a rare but far from unprecedented action, has chosen to go against the recommendation of the committee. Shares in Mesoblast fell 37% in response to the rejection, sinking back to the low they hit in the wake of the briefing document.

Mesoblast said the FDA has recommended it runs at least one additional randomized clinical trial in adults or children with steroid-resistant graft-versus-host disease before refiling for approval. The recommendation suggests Mesoblast will need to expend time and money to get remestemcel-L to market. The phase 3 trial cited in the briefing document lasted almost three years.

However, Mesoblast is holding out hope that it can get remestemcel-L to some patients without the extra data requested by the FDA. In light of the unmet medical need, Mesoblast plans to request a Type A meeting with FDA to discuss the possibility of an accelerated approval. Mesoblast is hoping the FDA will agree to authorize remestemcel-L on the proviso that it runs a study post-approval.

If Mesoblast is to persuade the FDA to grant remestemcel-L accelerated approval, it may need to address some of the agencys other concerns. Mesoblast said the FDAs response letter identified the need for further scientific rationale to demonstrate the relationship of potency measurements to the products biologic activity.

The comment reflects sections of the briefing document in which the FDA said the quality attributes lack a demonstrated relationship to the clinical performance of specific [drug product] lots. In the absence of evidence of that relationship, the FDA said the attributes may not be sufficient to ensure the manufacturing process consistently produces remestemcel-L lots of acceptable quality.

In disclosing the response letter, Mesoblast said assays measuring the potency of remestemcel-L will continue to be refined to provide further scientific rationale for its use in severe inflammatory diseases with high mortality risk.

See more here:
Mesoblast hit by FDA rejection, request to run another trial - FierceBiotech

FDA Grants Breakthrough Therapy Designation to CD123-Directed Antibody-Drug Conjugate – Targeted Oncology

The FDA has granted a Breakthrough Therapy designation to IMGN632 as treatment of patients with relapsed or refractory blastic plasmacytoid dendritic cell neoplasms (BPDCN), ImmunoGen, Inc., announced in a press release.

We are pleased FDA has granted Breakthrough Therapy designation for IMGN632, our novel CD123-targeted ADC, as it underscores the urgent need for effective and well-tolerated treatments for patients with this rare and aggressive cancer, said Mark Enyedy, president and chief executive officer, ImmunoGen, in a statement. We look forward to continuing to work with FDA to further define the development path for IMGN632 in BPDCN, in addition to pursuing our ongoing evaluation of IMGN632 in AML and other hematological malignancies.

IMGN632, a CD123-targeting antibody-drug conjugate (ADC), is in clinical development for the treatment of hematologic malignancies like BPDCN, acute myeloid leukemia (AML), and acute lymphocytic leukemia in clinical trials. The agent is under evaluation as monotherapy in patients with BPDCN and minimal residual disease (MRD)-positive AML after induction treatment, as well as in combination with venetoclax (Venclexta) plus azacitidine (Vidaza) in patients with relapsed/refractory AML.

BPDCN, a rare blood cancer, is known to have features associated with both leukemias and lymphomas, with characteristic skin lesions, lymph node involvement, and frequent spread to the bone marrow. This is an aggressive cancer that requires intense treatment regimens that are often followed by stem cell transplantation. This represents a patient population of unmet need, particularly those in the relapsed/refractory setting.

The phase 1/2 open-label, multicenter clinical trial of IMGN632 plus venetoclax and azacitidine is investigating the safety and efficacy, as well as assess the antileukemic activity of the combination in patients with relapsed assessed in frontline CD123-positive AML. The antileukemia activity of the monotherapy will also be assessed in patients with MRD-positive AML.

The primary end points of the study include safety and tolerability, preliminary antileukemia activity, and MRD levels. The study is currently recruiting and is exploring several different doses of the ADC. IMGN632 will be assessed in combination and monotherapy across multiple regimen arms. Arm A will assess azacitidine, arm B venetoclax, arm C azacitidine plus venetoclax, and arm D will evaluate IMGN632 monotherapy in patients with MRD+ AML.

For arms A through C, a phase 1b dose-escalation cohort in arms A through C will determine the recommended phase 2 dose, and a phase 2 dose-expansion phase will further evaluate each regimen in arms A through D to characterize the safety profile and assess the antileukemia activity. Arm D will open with a dose-expansion cohort, using the ADC monotherapy dose and schedule based on the findings from the initial phase 2 IMGN632-0801 study, and there will be no dose-escalation portion.

To be included in the trial, patients must be at least 18 years of age, have a confirmed AML diagnosis, and be deemed appropriate for this experimental therapy per the treating physician. Patients must have CD123-positive AML to be included in the study, and they were allowed to have received prior CD123-targeted therapies, as long as they have not received IMGN632. Patients had to have an ECOG performance status 1 and resolution of any prior treatment-related toxicities to either grade 1 or baseline.

Patients cannot have received any anticancer therapy within 14 days before administration of the study drug, prior IMGN632 therapy, a myeloproliferative neoplasm-related secondary AML, or active central nervous system AML. They are also ineligible if they have a history of sinusoidal obstruction syndrome/venous occlusive disease of the liver, myocardial infarction within 6 months of enrollment, or clinically relevant active infection, including known active hepatitis B or C, HIV infection, cytomegalovirus, or other infectious diseases that would make the patient inappropriate for enrollment to this study.

Reference

ImmunoGen announces FDA breakthrough therapy designation for IMGN632 in relapsed or refractory blastic plasmacytoid dendtritic cell neoplasm. News Release. ImmunoGen, Inc. October 5, 2020. Accessed October 5, 2020. https://bit.ly/3ljtSFt

Originally posted here:
FDA Grants Breakthrough Therapy Designation to CD123-Directed Antibody-Drug Conjugate - Targeted Oncology

Avrobio bags gene therapy to join Regenxbio and Sangamo in race – FierceBiotech

Avrobio has licensed a Hunter syndrome lentiviral gene therapy from the University of Manchester in the U.K. The deal positions Avrobio to join Regenxbio and Sangamo Therapeutics in the race to develop gene therapies for use in patients with the rare disorder.

Hunter syndrome, also known as mucopolysaccharidosis Type II, is driven by a mutation that limits a patients ability to break down sugar molecules generated as a byproduct of cell activity. The buildup of the molecules causes progressive damage to organs such as the brain, affecting the development of children with the lysosomal disorder.

Takedas approved drug Elaprase gives Hunter syndrome patients the enzyme they need to break the molecules down, but its inability to cross the blood-brain barrier renders it ineffective against the CNS manifestations of the disease.

Join a panel of experts as they discuss the rigor involved in optimizing and scaling a reproducible serological assay for COVID-19.

Avrobio has become the latest company to identify gene therapy as a potentially better way to treat Hunter syndrome. The biotech, which is active in other lysosomal disorders, has agreed to pay the University of Manchester $8 million upfront for the global rights to a stem cell gene therapy that is set to enter the clinic in the second half of next year.

Brian Bigger, Ph.D., a professor of cell and gene therapy at the U.K. university, developed the candidate and co-authored a paper about it in 2018. The paper describes work on a braintargeted hematopoietic stem cell gene therapy designed to give patients the lysosomal enzyme iduronate-2-sulfatase (IDS).

Avrobio will make the therapy, now called AVR-RD-05, by modifying a patients own hematopoietic stem cells with a transgene for IDS expression and a protein tag intended to improve stability of the enzyme. If AVR-RD-05 works as hoped, the cells will engraft in the bone marrow and make copies of themselves that also carry the transgene.

The differentiation of the cells into components of the immune system could enable AVR-RD-05 to cause a lasting, body-wide increase in the presence of the enzyme at the root of Hunter syndrome. Avrobio will fund a 9.1 million ($11.8 millon) investigator-sponsored phase 1/2 trial to assess whether the gene therapy performs as hoped. The biotech is also on the hook for up to $80 million in milestones.

Avrobio will face competition if it gets AVR-RD-05 all the way to market. Takeda is the incumbent, Regenxbio and Sangamo have gene therapies in the clinicalthough the latter has underwhelmed so farand Denali Therapeutics is developing an IDS enzyme designed to cross the blood-brain barrier.

Read the rest here:
Avrobio bags gene therapy to join Regenxbio and Sangamo in race - FierceBiotech

Global Animal Stem Cell Therapy Market Professional Survey 2020 by Manufacturers, Regions, Types and Applications, Forecast to 2024 – The Daily…

In its newly published report, Global Marketers has provided unique insights about the Animal Stem Cell Therapy Market for the given period. One of the major objectives of this report is to categorize the various dynamics of the market and to offer the newest updates such as mergers and acquisitions, various technological developments, new entrants in the market, which make an impact on different segments of the Animal Stem Cell Therapy market. The report also takes into account the impact of the COVID-19 pandemic on the Animal Stem Cell Therapy market and offers a clear evaluation of the future market demand during the forecast period.

Get a Free Sample Report of Global Animal Stem Cell Therapy Market at:

https://www.globalmarketers.biz/report/life-sciences/global-animal-stem-cell-therapy-market-2019-by-manufacturers,-regions,-type-and-application,-forecast-to-2024/130268#request_sample

The following manufacturers are covered:

Medivet Biologics LLC VETSTEM BIOPHARMA J-ARM U.S. Stem Cell, Inc VetCell Therapeutics Celavet Inc. Magellan Stem Cells Kintaro Cells Power Animal Stem Care Animal Cell Therapies Cell Therapy Sciences Animacel

This is likely to drive the Global Animal Stem Cell Therapy Market over the forecast period. This research report covers the market landscape and its advancement prediction in the near future. After studying key vendors, the report focuses on the new entrant contributing to the enlargement of the market. Most companies in the Global Animal Stem Cell Therapy Market have currently adopted new technological trends in the market.

Segmentation by Region Of Animal Stem Cell Therapy Are:

Animal Stem Cell Therapy Market Leading Competition:In this section, the report provides information on Competitive situations and trends as well as merger and achievement and expansion, market shares of the top players. Readers could also be provided with production, revenue, and average price shares by Key vendors.

Ask for Discount at:

https://www.globalmarketers.biz/discount_inquiry/discount/130268

Animal Stem Cell Therapy Market Segment by Type, covers:

Dogs Horses Others

Animal Stem Cell Therapy Market Segment by Applications, covers:

Veterinary Hospitals Research Organizations

The Animal Stem Cell Therapy market report is the study of various business viewpoints like challenges geographies, divers, restraints, opportunities, and major players. This Animal Stem Cell Therapy research report was aggregated on the grounds of sub-segments and market sections linked to the sector.

Major Highlights of Animal Stem Cell Therapy Market in the Covid-19 pandemic covered in the report:

Market Competition by key manufacturers in the Animal Stem Cell Therapy industry.

Discussed Sourcing strategies, industrial chain information, and downstream buyers data.

Distributors and traders on Animal Stem Cell Therapy marketing strategy analysis focusing on region-wise needs in the COVID-19 pandemic.

Vendors who are providing a wide range of product lines and intensifying the competitive scenario in the Animal Stem Cell Therapy COVID-19 crisis.

Also highlights the key growth sectors of the Animal Stem Cell Therapy market and how they will perform in the coming years.

Inquire Before Buying at:

https://www.globalmarketers.biz/report/life-sciences/global-animal-stem-cell-therapy-market-2019-by-manufacturers,-regions,-type-and-application,-forecast-to-2024/130268#inquiry_before_buying

This Animal Stem Cell Therapy market breakdown also offers the scope of different segments and applications that can in fact impact the market in the future. Thorough information is based on current trends and significant milestones. The Animal Stem Cell Therapy market analysis mentions the volume of sales by region from 2020 to 2024. A thorough evaluation of the limitations included in the market analysis portrays the drivers and gives scope for strategic planning.

Major Highlights:

Detailed overview of parent market

Changing Animal Stem Cell Therapy market dynamics in the industry

exhaustively market segmentation

past, present, and future market size in terms of volume and value

current industry trends and developments

Competitive landscape

Potential and niche segments, geographical regions exhibiting promising growth

Must-have information for market Key vendor to keep going and increase their market footprint

The major key questions addressed through this innovative research report:

Some Points from Table of Content

Global Animal Stem Cell Therapy Market Report 2020 by Key Players, Types, Applications, Countries, Market Size, Forecast to 2024

Chapter 1Report Overview

Chapter 2Global Market Growth Trends

Chapter 3Value Chain of Animal Stem Cell Therapy Market

Chapter 4Players Profiles

Chapter 5Global Animal Stem Cell Therapy Market Analysis by Regions

Chapter 6North America Animal Stem Cell Therapy Market Analysis by Countries

Chapter 7Europe Animal Stem Cell Therapy Market Analysis by Countries

Chapter 8Asia-Pacific Animal Stem Cell Therapy Market Analysis by Countries

Chapter 9Middle East and Africa Animal Stem Cell Therapy Market Analysis by Countries

Chapter 10South America Animal Stem Cell Therapy Market Analysis by Countries

Chapter 11Global Animal Stem Cell Therapy Market Segment by Types

Chapter 12Global Animal Stem Cell Therapy Market Segment by Applications

Chapter 13Animal Stem Cell Therapy Market Forecast by Regions (2020-2024)

Chapter 14Appendix

Access full Report Description, TOC, Table of Figure, Chart, etc.at:

https://www.globalmarketers.biz/report/life-sciences/global-animal-stem-cell-therapy-market-2019-by-manufacturers,-regions,-type-and-application,-forecast-to-2024/130268#table_of_contents

Excerpt from:
Global Animal Stem Cell Therapy Market Professional Survey 2020 by Manufacturers, Regions, Types and Applications, Forecast to 2024 - The Daily...