Category Archives: Stell Cell Research


Human-derived proteins used in DREAM tool – Drug Target Review

CRISPR-DREAM tool used to activate insufficiently expressed genes and convert skin cells to induced pluripotent stem cells (iPSCs).

Published in Nature Methods, Rice University bioengineers developed a tool that activates silent or insufficiently expressed genes using human-derived proteins termed mechanosensitive transcription factors. These naturally enable cells to switch on specific genes in response to mechanical cues.

Called CRISPR-DREAM, which stands for CRISPR-dCas9 recruited enhanced activation module or DREAM for short, the tool is smaller, more effective and less toxic to medically useful cell types compared to other technologies used to control gene expression. It may enable safer cell and gene therapies and more accurate disease models to address haploinsufficiency disorders which cause several difficult-to-treat conditions like epilepsy, some forms of cancer, immunodeficiency and Alzheimers disease (AD).

Dr Isaac Hilton, Assistant Professor of Bioengineering and Biosciences at Rice University, said: Many human diseases are driven by problems with too little of a gene being produced. He continued: You encounter these health issues where people dont make enough of a certain protein or gene product, and in those cases, unfortunately there are often few therapeutic options.

To address this problem, researchers have utilised CRISPR-based targeting systems to create cutting-edgesynthetic transcription factors. The majority of these tools are built with materials of nonhuman origin, which may come with unwanted side effects.

One challenge with some of the current technologies is that they are built using viral proteins that have evolved to reprogram how our cells work, and they do so in ways that are of course not necessarily beneficial, Hilton said. And even though virally derived elements can be engineered to work for the host cells benefit, we and others have observed in our research that they can still cause some toxicity in human cells.

Instead of relying on virally derived proteins, Barun Mahata, a postdoctoral researcher in Hiltons lab and the studys lead author, endeavoured to employ transcription factors that human cells already produce and use. Mahata fused the specific parts of these proteins responsible for activating genes to CRISPR-based programmable delivery platforms with a method intended to enhance their transcriptional powers.

We harnessed the natural ability of human-derived transcription factors, or proteins responsible for gene synthesis in the cells of our body, said Mahata. The transcription activation units we built function in a very precise way. They induce gene activity where we target them and can activate very rapid and robust transcription.

They also help tackle another disadvantage of current synthetic gene-activation platforms, which are often too large for efficient delivery to human cells.

What we did was look for small segments of human proteins that we could leverage to apply these technologies in human cells more effectively, said Hilton. When we started this project of building synthetic transcription factors with human-derived proteins, we wanted to identify the ideal source material with compactness being one key factor.

Human mechanosensitive transcription factors proteins that our cells and organs use to respond to mechanical forces met the researchers criteria, being relatively small, quick-acting and widely used by nearly all human cell types.

Additionally, the team activated up to 16 different locations on the genome simultaneously, a record number for synthetic transcription factors.

The reason why that capability is particularly important is because when our cells perform a function, its not just that they turn on a single gene, Hilton explained. Instead, they typically turn on whole constellations or networks of genes in concert. And we can now use these synthetic transcription factors to mimic and engineer what our cells do naturally.

The researchers showed that the DREAM tool can be used to convert skin cells to induced pluripotent stem cells (iPSCs) in a dish as a proof of concept exercise, a feat that Hilton says carries tremendous biomedical utility in the future.

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Human-derived proteins used in DREAM tool - Drug Target Review

Global Culture Media Market Size to Reach USD 15.84 Billion in … – GlobeNewswire

Vancouver, Oct. 16, 2023 (GLOBE NEWSWIRE) -- Current Market Scenario (Market estimates)

The global Culture Media Market size was USD 5.43 billion in 2020, and is expected to register a revenue CAGR of 9.3% between 2021 and 2032. Steady market revenue growth is driven by rising need for monoclonal antibodies, growing emphasis on personalized medicine, increasing prevalence of infectious diseases, rising investment in research & development of innovative cell culture products, rising awareness about vaccines based on cell culture, and high demand for single-use technologies.

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Drivers: Increased Investment in Research & Development of Innovative Cell Culture Products

Increased investment in research & development of innovative cell culture products is a key factor driving culture media market revenue growth. Cell culture media is an important component in producing cultivated meat. Cell culture media is necessary to keep the cells healthy and alive. Currently, most of these media are very expensive and oftentimes deliver inconsistent outcomes. A limited number of species-specific formulations of commercial culture media exists in case of cultivated meat firms dealing with fish species. For instance; in September 2020, GFI announced providing a two-year grant to a research project focused on development of a serum-free, high-quality fish cell culture media, which is an essential move in making cultivated seafood to reach market. Researchers at Virginia Tech are developing a formulation for open-source media improved for growing fish cells. This research project deploys artificial neural networks and Response Surface Methodology (RSM) to optimize cell culture media for better thriving of fish cells.

Restraints: Cost Prohibitive Culture Media and Contamination Risks

Cost prohibitive culture media and contamination risks may hamper market revenue growth over the forecast period. Culture media comprise various ingredients such as serum and nutrients for cell growth, which makes the product very costly. Also, issues associated with specificity, variability, and standardization may also impact market revenue growth negatively. Sometimes, ingredients procured from poor sources can lead to contamination risks of cell culture media. This factor would also restrain demand for culture media.

Growth Projections

The global culture media market size is expected to reach USD 15.84 billion in 2032 and register a revenue CAGR of 9.3% over the forecast period, attributed to growing population, especially geriatric population, and rising prevalence of infectious diseases. Increasing prevalence of infectious diseases and rising need for development of more efficient drugs to combat resulting conditions are driving market revenue growth. Infectious diseases are considered to be the foremost cause of mortalities across the globe, particularly in young children living in low-income countries. As per the World Health Organization (WHO), diarrheal diseases and lower respiratory infections were included in the top 10 leading causes of death worldwide in 2019. Culture technologies are considered crucial for identification of infectious diseases, despite significant increase in molecular testing, as pathogenic organisms causing disease are required to be separated from other microbes in mixed cultures. In addition, occurrence of an organism is necessary for assessing the probability that a specific organism is responsible for a said disease, unlike a culture.

COVID-19 Direct Impacts

COVID-19 pandemic has boosted demand for culture media, as many biotechnology firms are conducting in-vitro R&D for vaccines and antivirals. In-vitro assessment of vaccines normally requires a culture media for identifying and analyzing the response and growing targeted microbes. Increasing emphasis on research & development of vaccines by various pharmaceutical companies to curb spread of COVID-19 virus is also propelling market revenue growth.

Current Trends and Innovations

Increasing trend of single-use technologies plays a pivotal role in driving market revenue growth. In the biotechnology industry, use of single-use technologies has become a common practice. Engineers and researchers are utilizing plastic components as an alternative to stainless steel items in biomanufacturing processes. In cell culture production, adoption of single-use is quite essential and these cell growth systems may be wave-type bioreactors, plastic bioreactors, or plastic linings present in stainless-steel support. Reusable or disposable probes are present in all systems that protrude through an interior sleeve or attach to the outside. Majority of the connections depend on separate systems having aseptic/plastic connectors. Single-use systems are pre-cleaned and pre-sterilized, generally via gamma irradiation. Hence, there is no requirement for cleaning, sterilization, or sanitization steps. It saves money on use of chemicals for cleaning, as well as power and equipment needed to produce pure water and steam.

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Geographical Outlook

Culture media market in Asia Pacific is expected to register fastest revenue CAGR during the forecast period, attributed to increasing geriatric population in countries such as Japan and China and increase in prevalence of chronic diseases. In addition, increasing prevalence of contagious diseases, high demand for personalized medication, and presence of biotechnology firms such as Daiichi Sankyo Company Limited and large population base in countries in the region are also contributing to market growth.

Strategic Initiatives

In December 2018, Fujifilm acquired IS Japan (ISJ) and Irvine Scientific Sales Company (ISUS). Both companies have expertise and technological know-how on cell culture media. Irvine Scientific Sales Company distributes its products mostly in Europe and the US, whereas IS Japan distributes its products primarily in Japan and various other Asian countries. Both of these firms offer culture media to bio-ventures, pharmaceutical companies, and academia. Fujifilm is a photography and imaging firm in Japan. It has entered into stock purchase contract worth approximately USD 800.0 million.

Scope of Research

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Major Companies and Competitive Landscape

The global culture media market is moderately fragmented, with a large number of small- and medium-sized companies accounting for a major revenue share. Major companies have well-established facilities and enter in acquisitions & mergers, strategic agreements, and engage in various research & development activities and initiatives to develop & deploy new and more efficient technologies & products in the culture media market. Some major players operating in the culture media market are:

Strategic Development

In December 2019, Sartorius, which is a life science research firm, made an announcement about signing an agreement for the acquisition of Biological Industries. The latter is an Israel-based firm involved in cell culture media development and production. Sartorius would purchase 50% shares of Biological Industries for worth over USD 52.0 million.

Some Key Highlights From the Report

Browse a Detailed Summary of the Research Report @ https://www.emergenresearch.com/industry-report/culture-media-market Emergen Research has segmented global culture media market on the basis of type, research type, application, end-use, and region:

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Global Culture Media Market Size to Reach USD 15.84 Billion in ... - GlobeNewswire

Overcoming Cancer Therapy Obstacles With Epitope Editing – The Scientist

Acute myeloid leukemia (AML) is a deadly disease that presents unique treatment challenges. The cancerous hematopoietic stem and progenitor cells (HSPC) can be treated using chimeric antigen receptor (CAR) T cell therapy, but not in a targeted manner; the AML markers recognized by these therapies are also expressed by healthy HSPC and differentiated myeloid cells. This makes it impossible for current treatments to selectively kill the cancer cells, which causes significant toxicity and prevents successful HSPC transplant.1

In a study published in Nature, researchers from the Dana-Farber Cancer Institute and Harvard Medical School addressed this key obstacle.2 Editing specific epitopes of receptors expressed on the surfaces of healthy donor HSPC rendered the cells resistant to CAR T cell therapy.

Prior to this study, it was not very well characterized that its possible to abrogate the binding of therapeutic antibodies or CAR T cells with just one amino acid change in the extracellular domain of a protein, said hematologist and coauthor Gabriele Casirati of the Dana-Farber Cancer Institute. That was one of our main discoveries.

Casirati and his colleagues selected three well characterized AML surface markers that are currently being targeted with CAR T cell therapy: FMS-like tyrosine kinase 3 (FLT3/CD135), KIT (CD117), and the subunit of the IL-3 receptor (IL-3RA; also known as CD123). These proteins associate with almost all cases of AML and their overexpression on AML cells correlates with relapse and low survival rates, but they are also expressed in healthy cells at various stages of hematopoiesis.

The research team extensively screened cells for possible mutation sites in the genes encoding the surface markers. They hoped to identify sites that prevented the antibody portion of the CAR T cells from binding to them without interfering with the genes other critical cellular functions.

For each gene, they identified several such possible mutation sites. We were lucky enough that at least some of these mutations could be inserted by adenine base editing, Casirati explained. We tried conventional nuclease-based editing, but avoiding double-strand breaks is fundamental [for safety] so I wanted to go with the least toxic possible gene editing technology.

Using this information, the team performed adenine base editing of the epitopes in healthy human HSPC, creating a stealth version of each receptor.3 While the majority of nonedited HSPC were killed by CAR T cells directed against the markers in in vitro assays, epitope-edited cells survived. Each line of edited cells also maintained its stemness and differentiation capacity.

To minimize the chance of tumor escape and prevent relapse, CAR T cell therapies can target more than one AML marker. This led the team to produce cells expressing multiple edited epitopes. Casirati emphasized that the success of multiplexed editing is a key finding of the paper; using a mouse model, he and his team found that dual-edited FLT3 and CD123 HSPC survived in the presence of bispecific CAR T cell therapy targeting both markers. This approach fully eradicated AML cells in the tested tissues.

This paper is state-of-the-art in terms of doing epitope editing of these hematopoietic stem and progenitor cells that will still preserve their normal hematopoietic function, said Jeffrey Taub, a pediatric oncologist at Wayne State University who was not involved in the research. Definitely also, in terms of being able to target multiple genes simultaneously for AML with immunotherapies, [that] would be much more effective than only targeting one gene.

Casirati said that the study has implications that reach far beyond the treatment of AML. In the broader sense, [our work] is one of the first papers that shows how we can manipulate healthy tissues to endow them with new functions, he concluded.

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Overcoming Cancer Therapy Obstacles With Epitope Editing - The Scientist

Singamaneni to develop advanced protein imaging method – The … – Washington University in St. Louis

Cell-secreted proteins, such as antibodies, hormones and neurotransmitters, play a crucial role in maintaining overall health and well-being. They are also key components in disease research and in developing diagnostic tools and personalized medicines. However, current methods for studying these proteins are limited to observing large groups of cells together, which makes it difficult to discern individual cell behaviors and differences.

With a $450,000 grant from the National Science Foundation, Srikanth Singamaneni, the Lilyan & E. Lisle Hughes Professor in the McKelvey School of Engineering at Washington University in St. Louis, will develop a method called Plasmon-Enhanced Expansion FluoroSpot (PEEFS) to address these limitations. PEEFS combines a very bright fluorescent nanoparticle with expansion microscopy to image secreted proteins with high sensitivity and precision and accurately measure differences between cells.

The project represents a potentially transformative advance, particularly in immunology, oncology, stem cell biology and other life-science disciplines. With PEEFS, researchers will be able to image and quantify protein secretion at extremely high resolutions down to the level of a single cell revealing cell-to-cell variability and interactions and the spatial and temporal dynamics of cell-secreted proteins.

This story was originally published on the McKelvey School of Engineering website.

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Singamaneni to develop advanced protein imaging method - The ... - Washington University in St. Louis

The Pershing Square Foundation Is Accepting Applications for Its … – Yale School of Medicine

The Pershing Square Foundation has announced the opening of applications for the 2024 MIND (Maximizing Innovation in Neuroscience Discovery) Prize. Since the prize launched in 2023, it has been awarded to seven multidisciplinary investigators from institutions across the country. The annual prize awards $250,000 per year for three years ($750,000 total) to at least six scientists looking to uncover a deeper understanding of the brain and cognition. The prize is meant to enable the most talented early-to-mid-career investigators to pursue bold, creative projects that could have transformative impacts on the field of brain research.

Neurodegenerative diseases are like the Eldredge knot, nearly impossible to untie. Stemming from compromised cellular pathways, neural circuit dysfunctions, genetic risk factors, pathological epigenetic landscapes; these networked ailments are tightly coupled and profoundly intertwined, said Pershing Square Foundation Co-trustee Neri Oxman, PhD.

The MIND Prize calls for the creation of a road map to enable the understanding of related pathologies at a cellular and molecular level; to develop a single yet holistic theory blending genetics, environmental insults, and viruses, and to comprehend the bodys natural defenses against NDDs. Through MINDand its communitywe seek to disentangle cause-and-effect and offer radically novel insights into disease prevention and treatment. We believe in unforeseen visions made possible by interdisciplinary and trans-modal approaches that combine genetics with mobility, applied physics with neurodegeneration, memory and AI, and more.

The Pershing Square Foundation remains fueled by the urgency of this vast problem, and the individualsacross ages and disease stateswhose lives this work could and should transform. We remain enthusiastic, hopeful and proud of the bold work set forth by our first cohort of grantees, and the next, and the next, continued Oxman.

Applicants must have between one and ten years of experience running their own laboratories by the award start date (May 2024), hold a PhD, MD, or MD-PhD (or degree equivalent), and be affiliated with a research institution in the United States of America. The deadline to submit a Letter of Intent is November 13, 2023 at 5:00pm EST. For more details on the MIND Prize and the application process, including the full eligibility criteria, a link to FAQs, and a link to the application submission platform, please visit: https://pershingsquarefoundation.org/portfolio-organization/mind-prize/.

The highly competitive MIND Prize will catalyze novel and daring interdisciplinary and multidisciplinary work by facilitating collaborations across academic departments and institutions and amongst the academic, biomedical industry, philanthropic, and business communities. These breakthroughs in basic, fundamental research will help augment the toolkit for, and knowledge of, neurodegenerative and neurocognitive disorders. Projects may range from the invention of novel tools, techniques, and technologies for mapping and analyzing the brain to bold approaches that demonstrate extraordinary therapeutic potential.

Our first year of the MIND Prize impressed us with the remarkable talent across the country, said Olivia Tournay Flatto, PhD, president of the Pershing Square Foundation and co-founder and executive director of the Pershing Square Sohn Cancer Research Alliance. This year, we are looking forward to receiving more bold proposals from researchers who arent afraid to tackle an old problem in a new way, that compel us to find new paths forward in the fields of neurobiology, immunology, engineering, computational biology, and more. We are grateful to our Scientific Advisory Board, which includes experts spanning across scientific disciplines, ready to help The Pershing Square Foundation uncover transformative and novel projects from investigators ready to change the field as we know it.

"The MIND Prize is allowing me to start projects that otherwise I wouldn't be able to do with traditional funding sources. said 2023 MIND Prize winner Sergey Stavisky, PhD, assistant professor at the University of California, Davis, It's letting me do something that's very hard, that's risky, that hasn't been done before, and to do it at a much earlier stage of my career."

The MIND Prize is proud to rely on the guidance of a highly accomplished scientific advisory board:

About The Pershing Square Foundation

The Pershing Square Foundation (PSF) is a family foundation established in 2006 to support exceptional leaders and innovative organizations that tackle important social issues and deliver scalable and sustainable global impact. PSF has committed more than $600 million in grants and social investments in target areas including health and medicine, education, economic development and social justice. Bill Ackman and Neri Oxman are co-trustees of the foundation. For more information visit: http://www.pershingsquarefoundation.org.

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The Pershing Square Foundation Is Accepting Applications for Its ... - Yale School of Medicine

Israel Cancer Research Fund and Cancer Research Institute … – Newswise

Newswise NEW YORK, October 11, 2023 -- The Israel Cancer Research Fund (ICRF) and the Cancer Research Institute (CRI) have awarded a collaborative grant to Michael Berger, PhD, of the Hebrew University of Jerusalem in Israel. This is the fifth time the two organizations have worked together to sponsor innovative research in immunotherapy. The project grant will provide funding of $180,000 over three years.

Professor Bergers project, Improving Solid Tumor Immunotherapy Through Rewiring of T-cells Mitochondrial Metabolism, will use a biosynthesis process and block production of a specific protein to reenergize T-cells, a type of immune cell, that have become impaired by the tumor microenvironment, to make them more effective in fighting cancers.

While immunotherapy first emerged as a form of FDA-approved cancer treatment in the late 1980s, it is only within the past few years that this class of therapy has begun to deliver significant survival benefit to patients, bringing it to the forefront of public attention. New immunotherapeutic approaches have been shown in clinical trials to effectively treat patients with bladder, head and neck, kidney, and lung cancers as well as leukemia, lymphoma, and melanoma, with clinical trials under way for more than 25 other types of cancer.

Building on our history of collaboration, CRIs partnership with the Israel Cancer Research Fund punctuates our commitment to supporting cutting-edge science and life-saving discoveries in the field of cancer immunotherapy across the globe. CRI has pioneered the field of cancer immunotherapy for 70 years. It is only through relationships such as this that we can one day create a world immune to cancer, said CRIs CEO and Director of Scientific Affairs, Jill ODonnell-Tormey, PhD.

Commenting on the partnership, Beryl P. Chernov, National Executive Director of ICRF said, Immunotherapy stands as one of the beacons of hope in the landscape of cancer treatment, offering new avenues for patients to battle this terrible disease. ICRF's partnership with CRI represents a profound commitment from both organizations to advancing science and medicine in our collective fight against cancer.

About the Cancer Research Institute

The Cancer Research Institute (CRI), established in 1953, is the preeminent U.S. nonprofit organization dedicated exclusively to saving more lives by fueling the discovery and development of powerful immunotherapies for all cancers. Guided by a world-renowned Scientific Advisory Council that includes four Nobel laureates and 33 members of the National Academy of Sciences, CRI has invested over $517 million in support of research conducted by immunologists and tumor immunologists at the worlds leading medical centers and universities and has contributed to many of the key scientific advances that demonstrate the potential for immunotherapy to change the face of cancer treatment. To learn more, go to cancerresearch.org.

About Israel Cancer Research Fund

Israel Cancer Research Fund (ICRF) was established in 1975 by a group of scientists, physicians, and philanthropists in the United States and Canada to support the best and brightest scientists conducting groundbreaking cancer research in Israel. Its goal is to end the suffering caused by cancer. To date, ICRF has provided more than $92 million in funding for over 2,800 grants to support innovative cancer researchers from leading institutions throughout Israel.Today, ICRF is the largest nongovernmental source of cancer research funding in Israel. ICRF-funded scientists have been instrumental in the development of innovative FDA-approved drugs Gleevec, Doxil and Velcade and include the first two Israeli Nobel Prize Laureates in Chemistry. ICRF grantees continue to make major breakthroughs and are at the forefront of cancer discoveries in nanomedicine, immunotherapy, stem cell research and targeted therapies. For more information, visit https://www.icrfonline.org.

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Israel Cancer Research Fund and Cancer Research Institute ... - Newswise

Pharmaceutical and Biotechnology Companies Lead the … – PR Newswire

DUBLIN, Oct. 16, 2023 /PRNewswire/ -- The"Microcarrier Market, Size, Global Forecast 2023-2030, Industry Trends, Growth, Share, Outlook, Impact of Inflation, Opportunity Company Analysis" report has been added to ResearchAndMarkets.com's offering.

The global Microcarrier Market is poised to achieve a substantial valuation of US$ 3.43 billion by 2030, according to a comprehensive market analysis.

Microcarriers, vital in the growth of adherent cells in bioreactors, are revolutionizing biopharmaceutical and cell therapy production. This versatile technology supports the cultivation of virus-generating or protein-producing adherent cell populations, playing a pivotal role in large-scale biologics and vaccine manufacturing.

Key Market Drivers:

Market Opportunities:

Microcarrier Market Expected to Grow at a CAGR of 8.85% from 2022 to 2030

The Microcarrier Market is experiencing significant growth, driven by the need to enhance vaccine production yields, reduce costs, and address a wide range of diseases. Biologics and regenerative medicine are witnessing substantial demand, with a focus on innovative treatments and constant technological advancements in cell biology research.

Key Market Challenges:

Despite these challenges, the Microcarrier Market is growing, having reached USD 1.74 billion in 2022.

Microcarrier Beads: The Leading Consumables Fostering Growth

Microcarrier Market's Consumables segment includes Microcarrier Beads and Media & Reagents. Microcarrier beads take the lead, thanks to their pivotal role in biotechnology and cell culture. These beads provide a flexible platform for cell attachment and growth, facilitating the production of biopharmaceuticals, vaccines, and cell-based therapies. Their ability to support high cell densities and scalability in bioprocessing makes them indispensable for efficient and cost-effective solutions.

Cell Therapy: Fastest-Growing Segment

Cell therapy is the fastest-growing segment in the Microcarrier Market, driven by regenerative medicine and the expanding use of cell-based treatments. Microcarriers provide an essential platform for cultivating and expanding cells used in cutting-edge therapies, including stem cell-based cures, immunotherapies, and tissue engineering applications.

Pharmaceutical and Biotechnology Companies: Market Leaders

Pharmaceutical and biotechnology companies lead the Microcarrier Market, relying extensively on microcarriers for various applications. They play a crucial role in the production of biopharmaceuticals, vaccines, and cell-based treatments. Continuous investments in research and development, along with innovation in microcarrier technology, further bolster the demand for microcarriers.

The United States: Driving Innovation in the Microcarrier Industry

The United States stands out in the Microcarrier Market, known for its innovation and technological advancements. With a robust research and development environment, a vast pool of biotech and pharma companies, and strategic collaborations with academic institutions, the U.S. Microcarrier industry leads in areas such as stem cell therapy, vaccine production, and biopharmaceutical manufacturing.

Key Players:

The Microcarrier Market continues to evolve, driven by the growing demand for biologics, regenerative medicine, and innovative cell-based therapies. As the industry expands, it promises advancements in healthcare and biopharmaceuticals.

Key Topics Covered:

1. Introduction

2. Research& Methodology

3. Executive Summary

4. Market Dynamics 4.1 Growth Drivers 4.2 Challenges

5. Global Microcarriers Market

6. Global Microcarriers Market - Share Analysis 6.1 By Product Type 6.1.1 By Consumables 6.2 By Application 6.3 By End User 6.4 By Countries

7. Product Type-Global Microcarriers Market 7.1 Consumables 7.1.1 Microcarrier Beads 7.1.2 Media & Reagents 7.2 Equipment

8. Application- Global Microcarriers Market 8.1 Cell Therapy 8.2 Vaccine Manufacturing 8.3 Others

9. End User- Global Microcarriers Market 9.1 Pharmaceutical & Biotechnology Companies 9.2 Contract Research Organizations & Contract Manufacturing Organizations 9.3 Academic & Research Institutes

10. Countries- Global Microcarriers Market 10.1 North America 10.1.1 United States 10.1.2 Canada 10.2 Europe 10.2.1 France 10.2.2 Germany 10.2.3 Italy 10.2.4 Spain 10.2.5 United Kingdom 10.2.6 Belgium 10.2.7 Netherland 10.2.8 Turkey 10.3 Asia Pacific 10.3.1 China 10.3.2 Japan 10.3.3 India 10.3.4 South Korea 10.3.5 Thailand 10.3.6 Malaysia 10.3.7 Indonesia 10.3.8 Australia 10.3.9 New Zealand 10.4 Latin America 10.4.1 Brazil 10.4.2 Mexico 10.4.3 Argentina 10.5 Middle East & Africa 10.5.1 Saudi Arabia 10.5.2 UAE 10.5.3 South Africa 10.6 Rest of the World

11. Porter's Five Forces Analysis- Global Microcarriers Market

12. SWOT Analysis- Global Microcarriers Market

13. Key Players Analysis

For more information about this report visit https://www.researchandmarkets.com/r/o18ciu

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Pharmaceutical and Biotechnology Companies Lead the ... - PR Newswire

Monkeys With Transplanted Pig Kidneys Survive for Up to Two Years – Smithsonian Magazine

In the new study, long-tailed macaques, or crab-eating macaques, received kidney tranplants from genetically edited pigs. One of the monkeys survived for just over two years after the transplant. Reinhard Dirscherl / ullstein bild via Getty Images

Results from a new study published this week give hope to the goal of solving the world's kidney crisis.In the United States alone, 92,000 people are awaiting a new kidney on the national transplant waiting list, according to the American Kidney Fund. Donated kidneys cannot meet support the demandas of 2020, 5,000 people were dying each year while waiting for a kidney transplant, per the University of Pennsylvania.

One strategy for addressing this organ shortage is using kidneys from pigs, whose organs are of a similar size to human organs.

The global burden of kidney disease is staggering, Mike Curtis, CEO of eGenesis, tells Wireds Emily Mullin. Cross species transplantation offers the most sustainable, scalable, and feasible approach for delivering new sources of organs.

The new study, published Wednesday in the journal Nature, reported that a new experiment transplanting kidneys from genetically engineered pigs into monkeys showed that the primates could survive up to two years. Researchers used the gene editing technology CRISPR to make tweaks to Yucatan miniature pig genes, then removed the kidneys from 21 crab-eating macaques and transplanted the pigs' organs into the monkeys.

Some of the monkeys received kidneys just with three edits to prevent their immune systems from attacking the donated organ, per Wired.The monkeys that received the kidneys that were only designed to evade rejection survived for only between four and 50 days, with a median of 24 days.Others received kidneys with seven additional edits to make the pig cells behave a little bit more like human cells, Qin tells Scientific Americans Shi En Kim. Those monkeys survived times longer for a median of 176 days, with one surviving for 758 days.

The results bring researchers closer to testing pig kidney transplants in human trials, the study authors write.

The study is a proof of principle in non-human primates to say our [genetically engineered] organ is safe and supports life, Wenning Qin, a co-author of the study and a molecular biologist at eGenesis, a biotech company that conducted the research, tells Nature News Max Kozlov.

The research is a groundbreaking achievement, but there is still a long way to go before this strategy could be used in clinical trials, Dusko Ilic, a stem cell scientist at Kings College London who did not contribute to the findings, tells the Guardians Ian Sample and Anna Bawden.

A number of transplants involving pig organs have taken place in recent years. Last April, scientists reported transplanting a genetically edited pig kidneys into a brain-dead human patient, and the kidneys remained viable for the duration of the 74-hour experiment. Two other brain-dead human patients received pig kidneys that remained functional during a 54-hour experiment. And a human patient with no treatment options remaining received a pig heart transplant that functioned normally for 49 days.

Adam Griesemer, a transplant surgeon at New York University who contributed to research on transplants in brain-dead humans, tells Wired that the studies and brain-dead humans together demonstrate that pig kidneys can be tested in clinical trials. But these trials will be different from the monkey experiments in part because humans weigh much more and have higher blood pressure than the monkeys, Jayme Locke, a transplant surgeon at the University of Alabama at Birmingham, says to Nature News.

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Pharmacological Interventions and TBI: What Does the Future Hold? – PharmiWeb.com

Traumatic brain injuries (TBIs) often have devastating effects. However, in the future, pharmacological interventions could play a pivotal role in mitigating the long-term consequences of TBIs, offering positive outcomes to individuals affected by this life-altering condition. Ongoing research and promising developments suggest the future holds immense potential for pharmacological interventions in TBI treatment.

The Centers for Disease Control and Prevention (CDC) estimates that approximately 1.5 million Americans survive a TBI annually, while 230,000 are hospitalized. TBIs can result from various causes, such as accidents, falls, sports injuries, or combat situations.

They encompass a broad spectrum of severity, ranging from mild concussions to severe brain damage. While 75% of brain injuries are closed-head injuries, the effects can still be severe.

According to the experts at Pam Health, Mild and moderate injuries are more likely to have temporary symptoms, while severe injuries are more likely to have long-term or permanent symptoms. People with a minor concussion from bumping into a pole will likely have no long-term effects, while a bullet that penetrates the skull could drastically alter the life of the victim

Regardless of the initial traumas intensity, TBIs can lead to debilitating physical, cognitive and emotional impairments. In the United States, 30% of injury-related deaths result from a TBI, and innovative solutions are required to treat the effects of these injuries.

TBI treatment primarily revolves around supportive care, rehabilitation, and symptom management. While significant progress has been made in these areas, there is still a profound need for therapies targeting the root causes of TBI-related damage, like inflammation, oxidative stress and neuronal dysfunction.

While minor brain injuries require rest and monitoring, sometimes surgery is needed to treat a brain bleed, remove a blood clot, repair fractures, or relieve skull pressure.

Healthcare providers often use medication to prevent secondary brain damage after the injury. These medications include diuretics, anti-seizure drugs, and even coma-inducing drugs. Some current interventions for TBI include the following.

TXA is an antifibrinolytic medication that works by inhibiting the breakdown of blood clots. Its used to control bleeding, and researchers have studied it as a TBI treatment. TXA reduces the risk of death in individuals with mild to moderate TBIs if given within three hours. However, it doesnt reduce the risk of death in patients with a severe TBI.

Coagulopathies, or blood clotting disorders, can complicate TBI management. Treatment may involve blood products such as fresh frozen plasma (FFP), platelets, or clotting factor concentrates to correct coagulation abnormalities and prevent or control brain bleeds.

Diuretics increase the excretion of excess fluid from the body through urine. Diuretics are sometimes used to manage elevated intracranial pressure, which can occur due to brain swelling. By reducing fluid retention in the brain and around the injury site, diuretics can help lower the pressure.

Commonly used diuretics include mannitol and hypertonic saline. However, their use is carefully considered, and the potential risks and benefits are weighed based on the patients specific condition and treatment response.

TBIs can increase the risk of seizures, especially in the days and weeks following the injury. Anti-seizure drugs, also known as antiepileptic drugs (AEDs), are prescribed to prevent or control seizures in these patients.

Seizures can further damage brain tissue and worsen outcomes, so healthcare providers often administer AEDs prophylactically to reduce the risk. Medications like phenytoin and valproic acid are commonly used.

Anesthetics and sedatives are often used in TBI management, particularly in the intensive care unit (ICU). They help control agitation, pain, and intracranial pressure. Sedation may be necessary for patients requiring mechanical ventilation, while anesthetics can be used during neurosurgical procedures to protect the injured brain.

The primary goal of TBI treatment is to optimize a patients physical, cognitive, and emotional recovery while minimizing the long-term impact of the injury.

This multifaceted approach involves several key objectives, including stabilizing the patients medical condition, preventing secondary brain injury, managing symptoms like pain and cognitive deficits, and promoting neural repair and regeneration.

Additionally, rehabilitation helps individuals regain independence through physical therapy, occupational therapy, speech therapy, and other important interventions. The overarching goal is to maximize the individuals functional abilities and enhance their overall well-being to the greatest extent possible.

The future of TBI treatment may lie in developing pharmacological interventions that address these underlying issues. Here are some promising avenues the medical system is exploring.

Inflammation plays a significant role in the brains response to injury. Researchers are exploring drugs that target specific inflammatory pathways to reduce brain inflammation after a TBI.

Theyve studied steroids and non-steroidal anti-inflammatory drugs (NSAIDs) for their potential to mitigate the damaging effects of inflammation. However, finding the right balance between suppressing harmful inflammation and preserving necessary immune responses remains challenging.

Neuroprotective agents, one of the promising pharmacological interventions for TBI, aim to protect brain cells from further damage after injury. Clinical trials are ongoing to assess the effectiveness of substances like erythropoietin in humans.

Growth factors such as nerve growth factor (NGF) promote brain cell growth, survival, and maintenance. Researchers are investigating ways to deliver these growth factors directly to the injured brain to encourage neuronal regeneration and recovery, however, several case studies have reported positive results following the delivery of NGF in children.

Researchers are also considering other growth factors, such as hepatocyte growth factor (HGF) and brain-derived neurotrophic factor (BDNF), for TBI treatment.

Neurotrophic factors, like glial cell-derived neurotrophic factor (GDNF) and BDNF, show potential to support damaged neurons and promote functional recovery. Healthcare providers can administer them through gene therapy.

Stem cell research offers the possibility of replacing damaged brain tissue with healthy cells. Several types of stem cells, including induced pluripotent stem cells (iPSCs) and mesenchymal stem cells (MSCs), are being investigated for their potential in TBI treatment. Early-stage clinical trials are exploring the safety and efficacy of these approaches.

Progesterone, a naturally occurring hormone, has emerged as a promising TBI intervention. Research suggests progesterone may offer neuroprotective effects by reducing inflammation, limiting brain swelling, and mitigating excitotoxicity, all of which can contribute to secondary brain injury.

Clinical trials have shown varying degrees of success, with some studies reporting improved outcomes in TBI patients who received progesterone treatment, particularly when administered early after the injury.

While more research is needed to define optimal dosages and treatment protocol, progesterone represents an intriguing avenue to enhance TBI recovery and reduce the long-term consequences of brain injuries.

Determining the best treatment for traumatic brain injury is complex and highly individualized. Treatment depends on the severity of the injury, the specific symptoms and deficits, and the patients unique circumstances.

However, an effective TBI treatment typically involves a multidisciplinary approach. This approach may encompass internal stabilization in an acute care setting, followed by intensive medical management to prevent secondary brain injury. Rehabilitation is the cornerstone of treatment, focusing on physical, occupational, and speech therapies to address motor skills, cognitive function, and communication.

Healthcare providers may prescribe medications, such as those targeting symptoms like pain and seizures, as part of the treatment plan. The goal is to tailor the treatment to the patients needs, combining medical interventions, therapies, and support to maximize their recovery while limiting long-term complications.

As promising as these interventions may be, the path forward isnt without its challenges. Researchers must contend with rigorous clinical trials, ethical concerns surrounding experimental treatments, and the need to balance risks and benefits for patients. Additionally, theres a need for comprehensive long-term studies to evaluate the safety and effectiveness of these interventions.

Challenges of TBI Treatment

Clinical trials

Conducting rigorous clinical trials to test the safety and efficacy of these interventions in humans is essential. This process can be time-consuming and costly and may cause ethical dilemmas when enrolling patients, especially those with severe TBIs.

Ethical considerations

Experimental treatments for TBIs often involve risks, and obtaining informed consent from individuals with compromised decision-making capacity can be challenging. Balancing the potential benefits against the risks and respecting autonomy are crucial ethical considerations.

Long-term effects

Understanding the long-term effects of pharmacological interventions is essential. Longitudinal studies are necessary to assess the immediate outcomes and the impact on patient lives years or decades after treatment.

Safety concerns

Ensuring the safety of pharmacological interventions is paramount. Some medications may have unforeseen side effects or complications, necessitating careful monitoring and post-market surveillance.

Supporting someone with a brain injury requires patience, empathy, and a deep understanding of their needs and challenges. Its important to offer emotional support by actively listening and validating their feelings.

Practical assistance, such as helping with daily tasks, transportation, and medication management, can also be invaluable. Encouraging engagement in rehabilitation therapies and attending appointments together demonstrates your supportiveness in their recovery.

Additionally, creating a safe and calm environment with minimal sensory overload can aid in their comfort and cognitive function. Educating yourself about TBI and connecting with support groups or professionals specializing in brain injury can help you provide more informed and effective support.

In the not-so-distant future, pharmacological interventions could revolutionize the way we treat traumatic brain injuries. While we may not have all the answers just yet, the relentless pursuit of scientific knowledge and innovative research is paving the way for a promising future.

As researchers strive to test and develop new interventions, the future offers the possibility of better outcomes and improved quality of life.

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Pharmacological Interventions and TBI: What Does the Future Hold? - PharmiWeb.com

Functional Type 1 diabetes cure research enjoys breakthrough as … – Notebookcheck.net

Vertex Presents Positive, Updated VX-880 Results From Ongoing Phase 1/2 Study in Type 1 Diabetes at the European Association for the Study of Diabetes 59th Annual Meeting

-All patients treated with VX-880 in Parts A and B have follow-up data beyond Day 90 and have demonstrated islet cell engraftment and glucose-responsive insulin production -

-All patients showed improvement across all measures of glucose control, including decreases in HbA1c, increases in blood glucose time-in-range, and reduction or elimination of insulin use -

-The two patients with at least 1 year of follow-up met the criteria for the primary endpoint of elimination of severe hypoglycemic events (SHEs) and HbA1c <7.0% -

-VX-880 was generally well tolerated -

-Part C concurrent dosing well underway -

BOSTON--(BUSINESS WIRE)--Oct. 3, 2023--Vertex Pharmaceuticals Incorporated(Nasdaq: VRTX) today presented longer-term data on patients dosed in Parts A and B of its Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated islet cell therapy in people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). Prior to VX-880 treatment, all six patients enrolled had long-standing T1D with no endogenous insulin secretion, required an average of 34.0 units of insulin per day, and had a history of recurrent severe hypoglycemic events (SHEs) in the year prior to screening.

All patients in Part A and B now have more than 90 days of follow-up and have demonstrated islet cell engraftment and endogenous glucose-responsive insulin production on the Day 90 mixed-meal tolerance test (MMTT). All patients demonstrated improved glycemic control across all measures, including decreases in HbA1c, improved time-in-range on continuous insulin monitoring, and reduction or elimination of exogenous insulin use.

The two patients with at least 12 months of follow-up after VX-880 infusion, who were therefore evaluable for the studys primary efficacy endpoint, met the criteria for the primary endpoint of elimination of SHEs between Day 90 and Month 12 with an HbA1c <7.0%. The first patient achieved insulin independence at Day 270 through Month 24. This is a patient who has had T1D for nearly 42 years and prior to trial enrollment was on 34 units of daily exogenous insulin. The second patient achieved insulin independence at Day 180 through Month 12. This is a patient who has had T1D for 19 years and prior to trial enrollment was on 45.1 units of daily exogenous insulin. Starting at Month 15, this patient was started on four units of basal insulin daily, at the investigators discretion. After the data cut-off date, a third patient achieved insulin independence at Day 180.

VX-880 has been generally well tolerated in all patients dosed to date. The majority of adverse events (AEs) were mild or moderate, and there were no serious AEs related to VX-880 treatment. As previously reported, one subject had SHEs in the perioperative period. There have been no other SHEs in the study.

We continue to marvel at the impressive data from the VX-880 program as evidenced by the improvements in all treated patients across all glycemic measures, saidTrevor Reichman, M.D.,Department of Surgery,University of Toronto. This represents an incredibly promising investigational therapy, one with far-reaching potential.

These data are particularly meaningful in the context of our overall investigational T1D program, as these same VX-880 cells are the foundation for our VX-264 cells-plus-device program, and our hypoimmune islet cell program, saidCarmen Bozic, M.D., Executive Vice President,Global Medicines Developmentand Medical Affairs, and Chief Medical Officer atVertex. We are moving with urgency to bring these potentially transformative therapies to patients who are waiting.

These data were presented during theEuropean Association for the Study of Diabetes59th Annual Meeting onOctober 3, 2023, inHamburg, Germanyas an oral presentation, Glucose-Dependent Insulin Production and Insulin-Independence in Patients with Type 1 Diabetes Infused with Stem Cell-Derived, Fully Differentiated Islet Cells (VX-880) (abstract/publication #449).

AboutVertexT1D Programs in Clinical Development

About VX-880VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using proprietary technology. VX-880 is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 has the potential to restore the bodys ability to regulate glucose levels by restoring pancreatic islet cell function, including glucose responsive insulin production. VX-880 is delivered by an infusion into the hepatic portal vein and requires chronic immunosuppressive therapy to protect the islet cells from immune rejection. The VX-880 trial has expanded to additional sites that are currently active and enrolling in theU.S.,Canada,U.K.,Germany,Norway,Switzerland,Italy,Netherlands, andFrance.

VX-880 was recently granted PRIME designation by theEuropean Medicines AgencyinMarch 2023, in addition to Fast Track Designation by theU.S.FDA inMarch 2021. PRIME designation is granted to innovative new therapies that have demonstrated the potential to significantly address an unmet medical need.

About the VX-880 Phase 1/2 Clinical TrialThe clinical trial is a Phase 1/2, multi-center, single-arm, open-label study in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. This study is designed as a sequential, multi-part clinical trial to evaluate the safety and efficacy of VX-880. Approximately 17 patients will be enrolled in the clinical trial. Enrollment in Part C of the study is ongoing and multiple patients have been dosed.

About VX-264VX-264 is an investigational cell therapy in which allogeneic human stem cell-derived islets are encapsulated in a channel array device designed to shield the cells from the bodys immune system. VX-264 is designed to be surgically implanted and is currently being evaluated for patients with T1D.

About the VX-264 Phase 1/2 Clinical TrialThe clinical trial is a Phase 1/2, single-arm, open-label study in patients who have T1D. This will be a sequential, multi-part clinical trial to evaluate the safety, tolerability, and efficacy of VX-264. Approximately 17 patients will be enrolled in the global clinical trial. Enrollment is ongoing in this study.

About Type 1 DiabetesT1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, leading to loss of insulin production and impairment of blood glucose control. The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood glucose levels. High blood glucose can lead to diabetic ketoacidosis and, over time, to complications such as kidney disease/failure, eye disease (including vision loss), heart disease, stroke, nerve damage, and even death.

Due to the limitations and complexities of insulin delivery systems, it can be difficult to achieve and maintain balance in glucose control in people with T1D. Current standards of care do not address the underlying causes of the disease, and there are limited treatment options beyond insulin for the management of T1D; there is currently no cure for diabetes.

AboutVertexVertexis a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF,Vertexhas a robust clinical pipeline of investigational small molecule, mRNA, cell and genetic therapies (including gene editing) in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes, and alpha-1 antitrypsin deficiency.

Founded in 1989 inCambridge, Mass.,Vertex's global headquarters is now located inBoston'sInnovation Districtand its international headquarters is inLondon. Additionally, the company has research and development sites and commercial offices inNorth America,Europe,Australia, andLatin America.Vertexis consistently recognized as one of the industry's top places to work, including 13 consecutive years onScience magazine'sTop Employers list and one of Fortunes 100 Best Companies to Work For. For company updates and to learn more aboutVertex's history of innovation, visitwww.vrtx.comor follow us on Facebook, Twitter, LinkedIn, YouTube, and Instagram.

Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, (i) statements byCarmen Bozic, M.D., andTrevor Reichman, M.D., in this press release, (ii) our plans, expectations for, and the potential benefits of VX-880 and VX-264, and (iii) our plans for dosing and enrollment of patients. WhileVertexbelieves the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's research and development programs may not support registration or further development of its compounds due to safety, efficacy, and other risks listed under the heading Risk Factors inVertex's most recent annual report and subsequent quarterly reports filed with theSecurities and Exchange Commissionatwww.sec.govand available through the company's website atwww.vrtx.com. You should not place undue reliance on these statements, or the scientific data presented.Vertexdisclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

View source version onbusinesswire.com:www.businesswire.com/news/home/20231003786678/en/

Vertex Pharmaceuticals Incorporated Investors: Susie Lisa, +1 617-341-6108 Or Manisha Pai, +1 617-961-1899 Or Miroslava Minkova, +1 617-341-6135

Media: [emailprotected] or U.S.: +1 617-341-6992 or Heather Nichols: +1 617-839-3607 or International: +44 20 3204 5275

Source:Vertex Pharmaceuticals Incorporated

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