Category Archives: Stell Cell Research

$500K Grant Supports Research on Muscle Growth in Pigs, With Broader Health Implications – Maryland Today

A $500,000 award from the U.S. Department of Agriculture National Institute of Food and Agriculture is supporting University of Maryland-led research to enhance pork production through improved muscle growth in pigs.

While early life nutrition is especially important for how muscles grow and develop, less is known about how these benefits can be passed from mother to offspring during pregnancy. For the $20 billion U.S. pork industry, increased and faster muscle growth would result in healthier animals, less feed and waste to raise that animal, and ultimately a more competitive and sustainable pork industry.

In partnership with the Uniformed Services University of the Health Sciences (USUHS), this work could also have future applications beyond the pork industry to optimize human performance and treat wounded service members.The key to these applications could lie in the epigenetic changes (or changes to how genes are expressed) and stem cell activity caused by a simple supplementbutyric acid.

Over the last decade, weve published some nutritional work in this area showing the impact of butyric acid and other dietary components on the activity of tissue-specific stem cells, and feeding butyric acid to pigs resulted in faster muscle growth, said Chad Stahl, professor and chair in the Department of Animal and Avian Sciences who is leading the research. If we are able to make the muscle fibers grow bigger because of the activity of these muscle stem cells, we want to see what happens if we are giving these compounds to the pregnant sow during fetal development.

Stahl conducted previous research in this area with his former student, Robert Murray Ph.D. 18. Now an assistant professor with USUHS and a lieutenant in the U.S. Navy, he is a co-investigator on this grant.

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$500K Grant Supports Research on Muscle Growth in Pigs, With Broader Health Implications - Maryland Today

EU approves bluebird bio’s CALD gene therapy Skysona – PMLiVE

The European Commission (EC) has approved bluebird bios gene therapy Skysona for the rare inherited neurological disease cerebral adrenoleukodystrophy (CALD).

The EC has cleared Skysona (elivaldogene autotemcel) for the treatment of early CALD in patients under the age of 18 years old with an ABCD1 gene mutation who do not have a matched sibling blood stem cell donor.

The approval is supported by data from the phase 2/3 Starbeam study as well as the ongoing phase 3 ALD-104 study.

In the phase 2/3 Starbeam study evaluating Skysona, 90% of CALD patients met the month 24 major functional disability- (MFD) free survival endpoint as of the last data cutoff date.

MFDs are the six severe disabilities commonly attributed to CALD, which have the most severe effect on a patients ability to function independently.

In addition, 26 out of 28 evaluable patients maintained a neurologic function score (NFS) less than or equal to one until month 24, with 24 of those patients having no change in their NFS.

All the patients who completed the Starbeam study enrolled for long-term follow-up in the LTF-304 study. The majority of patients that enrolled in LTF-304 96.3% - remained alive and maintained their MFD-free status through their last follow-up on study.

The median duration of follow-up was 3.2 years and 14 patients reached at least their year five follow-up visit.

bluebird bio was founded with the mission of developing a therapy to recode CALD on the genetic level, and todays announcement represents over twenty years of research and development that has laid the groundwork for future gene therapies to be possible, said Andrew Obenshain, president of severe genetic diseases at bluebird bio.

CALD is a progressive and fatal neurodegenerative disease that overwhelmingly affects males. It involves the breakdown of myelin the protective sheath of nerve cells in the brain that is responsible for muscle control and thinking.

The condition is caused by mutations in the ABCD1 gene that affect the production of ALDP which eventually causes damage to the adrenal cortex and white matter of the brain and spinal cord.

Skysona is designed to add functional copies of the ABCD1 gene into a patients hematopoietic stem cells (HSC).

Once this functional gene is added to a CALD patients stem cells, the patient's body can produce the adrenoleukodystrophy protein (ALDP), which is believed to allow for the breakdown of very-long-chain fatty acids that build up to toxic levels in the brain.

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EU approves bluebird bio's CALD gene therapy Skysona - PMLiVE

Secrets and pies: the battle to get lab-grown meat on the menu – The Guardian

Not a week goes by without Elliot Swartz receiving at least one request from researchers asking him where they can find cell lines (a cell culture developed from a single cell) for use in cellular agriculture an essential tool for creating lab-grown meat. One of the most important things that cell lines offer is that they enable researchers to just get started in this new field, says Swartz, who works in New York as a senior scientist at the Good Food Institute (GFI) a nonprofit focused on advancing cellular agriculture and bringing its products to our shelves and stomachs as quickly as possible. Helping researchers is a core part of his role. In the case of cell lines, however, theres very little he can do.

Swartzs response to the researchers is unfortunately always the same: at the moment, publicly available cell lines relevant for cellular agriculture dont really exist. That doesnt mean that theyre nowhere to be found. Upside Foods (previously Memphis Meats) has submitted several patents to protect cell lines it has developed, and companies such as Cell Farm Food Tech have built a business around selling cell lines for profit. Keeping discoveries behind closed doors is a pattern of behaviour found in private companies across the industry, which many believe is slowing down innovation.

Cellular agriculture is the use of animal cells or microbes to grow animal products, such as meat or milk, in bioreactors. The field gained prominence after Dutch scientist Mark Post unveiled the first cultured meat burger in 2013. Since then, cultured meats have been touted as a sustainable alternative to livestock farming, which is the leading cause of habitat destruction. Global demand for burgers and bacon is to increase over the coming decades, meaning more ecosystems will be bulldozed to accommodate the expanding market. This, in turn, will increase the risk of future pandemics, as biodiversity loss is linked to the emergence of new diseases. Moreover, efforts to cut carbon emissions will also fall short of Paris targets if we dont reduce our meat consumption, according to a special report published by the Intergovernmental Panel on Climate Change in 2019.

There is some progress. In Singapore last year, Eat Just became the first cultured meat company to gain regulatory approval to sell its product. But many technological, social, and economic hurdles remain before our supermarkets are filled with a variety of cultured cutlets. To surpass these hurdles, organisations including the GFI are pushing for a more public exchange of data, tools and ideas. As it stands, most research in the field is done by private companies which seem keen to protect their intellectual property.

Swartz says the lack of publicly available cell lines is a gatekeeper in getting people into the field, even though theres a lot of interest, adding that this isnt really an issue in other industries. Scientists looking for stem cells for research or clinical purposes can go to the government-funded UK Stem Cell Bank, and across the Atlantic, the nonprofit American Type Culture Collection hosts a reserve of cell lines that are mainly open access. Although repositories like this do include animal cells, that doesnt mean theyre suitable for generating meat.

What makes cell lines themselves so useful is that they are immortal and can multiply indefinitely, so they can be used as a standard model across the industry. Were not going to understand if our findings are true if different groups are using different cells with different features, Swartz continues. So cell lines are the first piece of the puzzle for getting cultivated meat to become an actual field of study. The GFI is filling the cell-line-shaped hole in cellular agriculture by funding the creation of lines that will be openly accessible, and making a repository to store them in. Kerafast a Boston-based bioresearch company will maintain this repository. Researchers not involved with the GFI are welcome to deposit cell lines too, as are private companies; anyone looking to use the cells must pay a small fee to cover the costs of storing and maintaining them. So far, only one academic group has deposited a cell line. The lines being worked on in academic groups are still in development, which is why we havent got that many yet, Swartz says.

The reluctance of private companies to share their cell lines may in part be because of how they are financed a GFI report found that of the $366m invested in cultured meat in 2020, only around $12m came from public sources. Controlling the vast majority of the capital in the industry means that the private sector can comfortably dictate the pace and direction of innovation, which the Breakthrough Institutes food and agriculture analyst Saloni Shah sees as an issue. With the government and public sector funding research you can set criteria and standards, and make sure the right kinds of technologies get funded so that the development of the sector accelerates and improves, says Shah.

The complaint that governments need to start investing in more sustainable food options is echoed by Isha Datar, the executive director of New Harvest another nonprofit focused on advancing cellular agriculture. She thinks one of the reasons the field lacks government funding is that it is a mix of tissue engineering, which is medically oriented, and food science. Cellular agriculture is kind of homeless and so it falls in between the cracks of the existing pillars of funding and how we think about science being separated, she says. Swartz also agrees that more public funding is needed, but he thinks it will only come after the technology has been scaled up. Does this industry scale? is going to be the key to opening the floodgate for governments funding this technology, he says. Open source research is going to be really important for bringing new ideas on how to scale this technology or lower costs.

Swartz also complains that secrecy is holding up the industry-wide adoption of other cheaper, more efficient materials. For example, all of the nutrients needed for animal cells to grow into chunks of meat are contained in the cell culture medium, but the industry standard foetal bovine serum is expensive, and must be extracted from the foetus of a slaughtered cow. Many startups claim to have developed alternatives, but they remain trade secrets. Companies tend not to patent these things, because by patenting a cell culture medium you have to include everything thats in there, which is open sourcing what the ingredients are, says Swartz.

Even if the cell line problem were solved, there would still be technological hurdles holding the field back from large-scale commercialisation. Using computer modelling to address these hurdles and accelerate the intensification of cultured meat production is a central goal of the Cultivated Meat Modeling Consortium (CMMC).

Modelling is a useful tool that allows researchers to simulate experiments before entering a laboratory. This helps to save on time and resources. In order to run more complicated simulations, however, modellers first need data from simpler experiments that detail the fundamental biological processes behind cultured meat production to understand the sum of the whole, we must first analyse the parts. Were experiencing quite some difficulty in getting the information we need to actually build models, says Jaro Camphuijsen, a researcher associated with the CMMC. Private companies they work with have shown resistance to sharing data and running certain experiments. We have been talking to a cultivated meat company quite a lot, and we often ask: What happens if you do this experiment? The answer is usually: We dont know, and We arent going to do that because the cells will die, Camphuijsen explains. But failed experiments, he says, can provide useful data points that often reveal more than successful tests. Experiments that go wrong actually provide lots and lots of information if you want to find out how these tiny systems of cells are behaving.

When asked to respond to accusations that industry secrets were slowing down innovation in the field, Uma Valeti, the CEO of Upside Foods, wrote in an email that the firm kickstarted the cultured meat movement when we were founded in 2015. Without that, the industry wouldnt be in the place it is today, where there are hundreds of companies, NGOs, academic groups and government institutions focusing on cultured meat, across every continent but Antartica. He adds that Upside is actively supportive of more open access research on cultured meat, and it has actively supported the development of public research institutions like the Cultured Meat Consortium.

What's the point of lab-grown meat when we can simply eat more vegetables? | Jenny Kleeman

Responding to the same accusations, Robert E Jones, head of public affairs at Mosa Meat, wrote: Few companies have done more than Mosa Meat to contribute to the open advancement of cellular agriculture. He adds that Mosa hoped the 2013 burger would trigger a moonshot level of public investment in research, and that there is something to be said for an innovation ecosystem that includes both private capital and public investments for a challenge as big as reforming the food system.

The idea that governments need to start investing in more sustainable food options is echoed by Datar. She has concerns about a field that lacks an academic basis and publicly accessible information. It means cellular agriculture is going to have to be more transparent than other industries, says Datar. I think we need a lot more data sharing and a lot more transparency if we are to create a better food system. Will private companies heed this call for more transparency and build on their claims that they are supportive of more open access research, or will they follow the approach in other sectors where financial gain has been prioritised over societal benefits? Campaigners hope the answer is one that puts the planet before profit margins.

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Secrets and pies: the battle to get lab-grown meat on the menu - The Guardian

Biomedical Warming and Thawing Devices Market Revenue to Cross USD 308 Mn by 2027: Global Market Insights Inc. – Markets Insider

SELBYVILLE, Del., July 20, 2021 /PRNewswire/ -- According to the latest report "Biomedical Warming and Thawing Devices Market by Product (Manual, Automatic), Sample (Blood Products, Ovum/Embryo, Semen), End-use (Hospitals, Research Laboratories, Pharmaceutical Industry, Blood Banks and Transfusion Centers), Regional Outlook, Price Trends, Competitive Market Share & Forecast 2027", by Global Market Insights Inc., the market valuation of biomedical warming and thawing deviceswill cross $308 million by 2027.

Increasing volume of research in the biotechnology sector for development of regenerative medicine, precision surgery, personalized therapeutics and immunotherapy among others is augmenting the demand for thawing devices and is expected to grow significantly. According to a recently published report, in 2019 and 2020, the biotech sector witnessed double-digit growth related to fundraising from venture capitalists and co-developments, joint ventures and partnerships among biotech companies is further increasing the research activities. As per the Lancet journal, the global biomedical research expenditure is projected to reach around a quarter of a trillion U.S. dollars annually in the coming years. Increased funding and financial support for biotech research activities will further promote the use of cryopreserved samples.

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Biotechnology research incorporates cryopreserved cells, tissues and other products on a high scale. For instance, cryopreserved primary neuronal cells and cardiomyocytes are frequently utilized in neuroscience and cardiology research. Cryopreserved biologics are extensively deployed in biotech research in multiple fields such as stem cell research, discovery science, diagnostics development and genomics. These products are essential for supporting several cell-based applications, including stem cell therapy, assisted reproduction and tissue engineering. Ongoing developments in these areas have increased the clinical demand of cryopreserved products.With the growing utilization of cryopreserved cells, the demand for biomedical warming and thawing devices is anticipated to surge at an accelerated rate in the coming years. Moreover, the rising developments in bioengineered products intended to facilitate therapeutics in several diseases are further poised to fuel the biomedical warming and thawing devices market growth.

The automatic segment in the biomedical warming and thawing devices market was valued at over USD 59 million in 2020. The use of automated warming and thawing devices reduces several drawbacks of water-bath-based and conventional manual approaches. These devices incorporate mechanical heating mechanism, and computerized control and monitoring, thereby eliminating user-to-user variability and offers consistent process that exterminates the need of intervention from user. Automated thawing devices have an extensive range of applications and can be applied successfully to be used in non-cellular therapeutic materials including plasma samples. In addition, considering the quality assurance aspect in biomedical warming and thawing, automated systems offer standardized, rapid warming rate and temperature read-out of the thawing process in certain cases for monitoring traceability are driving the rising preference for automated systems.

The biomedical warming and thawing devices market for ovum/embryo segment will showcase 9.3% growth rate through 2027 led by the growing infertility rates and increasing adoption of in vitro fertilization (IVF) and embryo transfer. According to the Centers for Disease Control and Prevention (CDC), around 6% of married women in the U.S. belonging to the 15 to 44 years age group face infertility concerns and around 12% of women face difficulty carrying a pregnancy to term. The growing prevalence of infertility has led to acceptance of treatment measures such as in vitro fertilization (IVF) or embryo transfer that incorporates cryopreserved or thawed embryos. According to the National Embryo Donation Academy of U.S., there are approximately 1,000,000 embryos in storage in the country as of 2021 and the number has almost doubled with the number of embryos around 500,000 in 2011. Furthermore, the high success rate of embryo cryopreservation, the rising use of vitrification that provides enhanced outcomes and identical IVF pregnancy rates are further slated to promote the product demand. Patients are increasingly receiving embryo cryopreservation for a broad range of indications, and their number has increased significantly.

The pharmaceutical end-use segment in the biomedical warming and thawing devices market is anticipated to reach USD 35 million by 2027 on account of the increasing research spending and studies in the pharmaceutical sector. Stem cells, blood components and other tissues host a great potential for wide range of applications in pharmaceutical and medical research. Advancements in diagnostic technologies have facilitated growing utilization of cell-based functional assays in drug development and discovery process in the pharmaceutical industry. Screening assays that incorporate cryopreserved cells reduces day-to-day variation, eliminates passage effects that is hampering the research and improvises the precision and consistency of cell-based assay outcomes. Additionally, the launch of advanced products in the biomedical warming and thawing devices market that offers clinical benefits in research is further set to promote the product adoption rate.

Brazil's biomedical warming and thawing devices market is estimated to grow at 8.4% CAGR by 2027 owing to the rising incidence of accidental injuries across the country. According to the World Health Organization (WHO), the burden of road accidents and associated mortality is more than 1.2 million people on yearly basis and nearly over 90% of these accidents occur in low- and middle-income economies such as Brazil. Brazil's road accident mortality rate per 100,000 people is over 20 and is significantly higher than surrounding countries. As per the World Health Rankings, around 43,698 deaths were reported associated with road traffic accidental mortalities in Brazil, accounting for 3.96% of total deaths in 2018. The increasing incidence of accidents and accidental injuries would require blood transfusion for managing the patient's health, thereby fostering the demand for cryopreserved blood and blood components.

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Some of the major players operating in thebiomedical warming and thawing devices market are Helmer Scientific, Brook Life Sciences, Sartorius AG, Sarstedt AG & Co. KG, Boekel Scientific, Barkey, Cytiva, Cardinal Health, and BioLife Solutions. These companies are implementing several strategies such as product launches, research collaborations, distribution partnerships to strengthen their industrial positioning.

Table of Contents (ToC) of the report:

Chapter 3Biomedical Warming and Thawing Devices Market Insights

3.1 Industry segmentation

3.2 Industry landscape, 2016 - 2027 (USD Million)

3.3 Industry impact factors

3.3.1 Growth drivers

3.3.2 Industry pitfalls & challenges

3.4 Growth potential analysis

3.4.1 By product

3.4.2 By sample

3.4.3 By end-use

3.5 COVID-19 impact analysis

3.6 Porter's analysis

3.7 Competitive landscape, 2020

3.8 PESTEL analysis

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https://www.gminsights.com/toc/detail/biomedical-warming-and-thawing-devices-market

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Global Market Insights Inc., headquartered in Delaware, U.S., is a global market research and consulting service provider, offering syndicated and custom research reports along with growth consulting services. Our business intelligence and industry research reports offer clients with penetrative insights and actionable market data specially designed and presented to aid strategic decision making. These exhaustive reports are designed via a proprietary research methodology and are available for key industries such as chemicals, advanced materials, technology, renewable energy, and biotechnology.

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biomedical-warming-and-thawing.png Biomedical Warming and Thawing Devices Market Growth Predicted at 8.5% Through 2027: GMI Major biomedical warming and thawing devices market players include Helmer Scientific, Brook Life Sciences, Sartorius AG, Sarstedt AG & Co. KG, Boekel Scientific, Barkey, Cytiva, Cardinal Health, and BioLife Solutions.

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Biomedical Warming and Thawing Devices Market Revenue to Cross USD 308 Mn by 2027: Global Market Insights Inc. - Markets Insider

A Recovery for All Of Us: New York City Invests $1 Billion in Life Sciences – Brooklyn Daily Eagle

Editors note: Job development in retail and high-tech is blooming along Brooklyns waterfront. From Brooklyn Navy Yard to Industry City and Brooklyn Army Terminal (BAT) literally billions of dollars are being invested in job growth. This EXPLAINER from the Citys EDC in June helps set the stage for a new announcement in the news about the latest development at BAT.

Mayor de Blasio and the NYC Economic Development Corporation (NYCEDC) announced on June 9 a plan to double the Citys $500 million investment in life sciences to $1 billion as part of LifeSci NYC, a commitment launched in the Mayors State of the City address to create jobs and establish New York City as the global leader in life sciences. This expanded initiative is expected to generate 40,000 jobs.

Mayor de Blasio kicked off this next chapter of the citys support for this industry by announcing aRequest for Proposals(RFP) to help advance the commercial research and development of new medicines, medical devices, diagnostics, materials, and research tools. The City will provide up to $112 million in City capital to award $20 million to support one or more innovation projects. Multiple awardees can access up to $20 million each.

New York City can do more than just fight back COVID-19. We can invest in fast-growing sectors like the life sciences to stop the next pandemic before it starts and become the public health capital of the world, said Mayor Bill de Blasio.This expansion will accelerate the growth of local researchers and businesses inventing the cures for whatever comes next. Its the key to our economic and public health recovery, and it will produce more effective and more equitable health outcomes for New Yorkers across the five boroughs.

A recovery for all requires making the City healthier giving every neighborhood and every household access to the best preventive health care and treatment available. To do that, we are committing today to make New York City the public health capital of the world. Todays investment will foster life sciences research, innovation and manufacturing, making the City the place where diagnostics, therapeutics and improvements in health care delivery are invented, tested and made available to the world, all while providing good-paying jobs to our incredibly talented, well-educated, and driven workforce, said Deputy Mayor for Housing and Economic Development Vicki Been. By doubling our original investment, we ensure both that health care will be fairer for all New Yorkers, and that New York City will bethe incubator for public health innovation full stop.

Strengthening our commitment to LifeSci NYC bolsters our pipeline of job opportunities in life sciences innovation and supports the creation of construction jobs as we build new infrastructure, saidSenior Advisor for Recovery Lorraine Grillo. This expanded investment in life sciences affirms New York Citys leadership in advancing public health, developing treatments, finding cures, and ensuring a recovery for all of us.

Building a healthier city means ensuring the life science sector is equipped with the greatest potential for cutting-edge technologies and treatments for all New Yorkers, saidRachel Loeb, president and CEO of the New York City Economic Development Corporation. With a diverse talent pool, a network of premier academic and medical institutions, New York City is positioned to grow as a global leader in life sciences research and innovation. Were thrilled to expand LifeSci NYC by investing in more talent, companies, and innovative spaces to help us recover and build a stronger economy for all.

Over the next decade, New York City will expand its investment to $1 billion to develop the life sciences industry by launching new commitments as part of LifeSci NYC. The program will invest an additional:

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I applaud Mayor de Blasio and NYCEDC for investing in the future of New York City with an additional investment of $500M to the citys life science initiative, LifeSci NYC, saidRep. Carolyn B. Maloney(NY-12). New York City has long been a national leader in life science innovation. This new investment will cement our city as a leader in life science innovation, critical to our preparedness for future pandemics, everyday illnesses, and other maladies affecting human health.

This pandemic has shown the need to invest in research and development of life sciences. The Bronx, the entire City and State of New York were drastically impacted by COVID-19, saidBronx Borough President Ruben Diaz Jr.Doubling the initial investment of $500 million to $1 billion dollars, will ensure New York City will stay at the forefront of cutting-edge discoveries, cures and treatments. In order to stay ahead we need to have secured infrastructure. As the Bronx Borough President, I know the need for up-to-date equipment and the announcement of the RFP for $20 million in capital to one or more recipients will assist them tremendously.

COVID-19 has had devastating consequences for Queens and the entire City, but we can prevent future health emergencies from having a similar impact by investing now in potentially life-saving research and development, saidQueens Borough President Donovan Richards Jr.We are proud that Long Island City in Queens is leading in life sciences innovation, and the investments announced today will help us go even further in the areas of pandemic prevention and preparedness. This funding shows our City is committed to the growing life sciences sector and will not let the lessons of COVID-19 go unheeded.

I applaud the forward thinking investments that the City will make as part of the LifeSci initiative to expand our capacity as a world leader in fighting pandemics and increase life sciences research and development, said Council Member Paul A. Vallone, Chair of the Committee on Economic Development, It is more important now than ever to ensure that we have a diversified economy in our city that spurs new research and creates jobs that will prevent future public health catastrophes.

Recovery means investing in long-term economic opportunities that will grow and physically stay in New York, and we continue to hear how the life science industry views our city as an important partner and geographic foothold into the future. The LifeSci NYC vision for the next decade fosters new businesses and equitably expands technical talent among our residents, which will help keep our city at the forefront of the economy of tomorrow, saidCouncil Member Carlina Rivera.

By taking a multi-factorial approach attentive to real estate, taxes, talented workers, academic collaborations, and cash the NYCEDC has dramatically expanded the life science industries in our city over the past few years, said Harold Varmus, MD, Weill Cornell Medicine. The Mayors decision to extend his successful initiative will sustain the progress already made and encourage further growth as the City recovers from the pandemic and increasingly recognizes the importance of the health-supporting sciences in its future economy.

It has been exciting for the Advisory Council to contribute to the excellent progress that New York City has made since 2016 in its journey to build a world class biotechnology ecosystem, saidVicki Sato, PhD, Professor of Management Practice, Harvard Business School. With Mayor de Blasios commitment to economic growth and the focused work of the EDC, we have visibility to 2M square feet of new laboratory space coming online by 2023, we have seen growth to over $2Bn of venture investment in 2020, we have launched a vibrant internship program that helps young people compete for important jobs, and most important, we have seen the creation of many new companies committed to improving healthcare. We look forward to continuing the work.

The Lasker Foundation joins leaders across New York City and beyond in celebrating the inspiring vision of a thriving life science ecosystem in our community, saidClaire Pomeroy, President, Albert and Mary Lasker Foundation. Building on our outstanding academic research and healthcare institutions, the increasing presence of biomedical companies and innovative start-ups, experienced investment institutions, and diverse engaged stakeholders, New York City is an ideal place for further growth and expansion of life sciences. As companies specializing in biotechnology, diagnostics, therapeutics, digital health and more choose the City as their preferred location, we see New York City as the home to future innovative breakthroughs that will support better health for all.

At Kallyope, we have witnessed first hand the benefits of being headquartered in New York City, saidNancy Thornberry, CEO, Kallyope.As an early-stage biotechnology company focused on pursuing novel therapeutics for diseases of high unmet need in a fundamentally new way, access to the Citys extraordinary talent pool, technologies, and academic centers has proven invaluable in our ability to deliver on our mission. We strongly encourage leaders across the industry to consider building their next life sciences venture in New York City.

New Yorks biotech ecosystem is unique in its diversity in the kinds of discoveries being made, in the kinds of scientists involved, and in the kinds of impacts that these will potentially have on the world, saidKevin Gardner, Director of Structural Biology Initiative, CUNY Advanced Science Research Center (ASRC). LifeSci NYC has played a transformational role in recognizing the strength of this diversity, and in turn ensuring that these great ideas and people get their chances to realize their potentials all while staying here in New York. I cant imagine a better investment for our city, our scientists and students, and our ideas.

LifeSci NYC has established New York as a hub for the life sciences and biotech industry, putting the city on the map for entrepreneurs starting new businesses, while spurring a flood of private sector investment, saidMaria Gotsch, President and CEO of the Partnership Fund for New York City. Last year, the industry hit a record high in jobs and venture capital funding, demonstrating New Yorks value as a central access point to other industries and commercial activity. The citys decision to double down on this initiative sends a strong message that New Yorks life science community is a smart bet and will play an essential role in the regions economic recovery.

About LifeSci NYC With a diverse talent pool, more than 100 disease-specialty foundations, 370 federally qualified health centers, 50 hospitals, and nine world-leading academic medical centers, New York City is home to one of the largest concentrations of life sciences research. Building on these advantages, NYCEDC established Lifesci NYC in 2016 to form industry partnerships, create thousands of good-paying jobs, and drive key life sciences investments in New York City. The program has helped cement New York Citys place on the map in life sciences, with pharmaceutical and biotech companies attracting more than $1 billion in annual venture investment in 2020 up from $130 million in 2016. The city has unlocked two million square feet of new life sciences innovation space, provided 400 students with paid internships at top life sciences companies, and opened six new incubators yielding 150 start-up companies every two to three years.

The Citys network of life sciences companies, institutions, and industry partners helped throughout the COVID-19 pandemic with broad response for testing, treatment, and vaccination programs. This included working directly with NYCEDC on the development of local supplies for PPE and test kits, the launch of the Pandemic Response Laban award for the local development of a rapid test at Columbia University and the ongoing development of a Pandemic Response Institute.

LifeSci NYC has invested $38 million in city capital to fundapplied research and development facilities at four of New York Citys leading scientific research institutions Columbia University, Einstein College of Medicine + Montefiore Medical Center, the New York Stem Cell Foundation, and Rockefeller University. In addition, the program has launched BioLabs @ NYULangone, the citys largest wet-lab incubator, and partnered withDeerfield Management and King Street Propertiesto develop more than 500,000 square feet of new lab space. The City will continue to build out research and development to establishLifeSci Avenue stretching from the Pandemic Response Institute in Kips Bay in the South through East Harlem in the North. This corridor will anchor the Citys public health vision with neighborhood clusters across the City Long Island City, Sunset Park, Central Brooklyn, Hudson Square, Manhattans West Side, West Harlem, Upper Manhattan, and Morris Park to build a complete network of life science innovation. To learn more about LifeSci NYC, visit lifesci.nyc.

About NYCEDC New York City Economic Development Corporation creates shared prosperity across New York Citys five boroughs by strengthening neighborhoods and creating good jobs. NYCEDC works with and for communities to provide them with the resources they need to thrive, and we invest in projects that increase sustainability, support job growth, develop talent, and spark innovation to strengthen the Citys competitive advantage. To learn more about our work and initiatives, please visit us onFacebook,Twitter, orInstagram.

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A Recovery for All Of Us: New York City Invests $1 Billion in Life Sciences - Brooklyn Daily Eagle

The growing global "infodemic" around stem cell therapies – Axios

An industry centered around unproven stem cell therapies is flourishing due to misinformation.

Why it matters: Stem cells offer a tantalizing potential to address a large number of diseases, like Parkinson's, ALS, cancers and bodily injuries. But only a small number of therapies have been found safe and effective through clinical trials, while misinformation continues to proliferate.

The latest: The Pew Charitable Trusts issued a brief in early June that describes a rising number of reported adverse events.

Background: Clinics with unregulated stem cell products or therapies began emerging in the early 2000s all over the world, "taking advantage of the media hype around stem cells and patients hope and desperation," says Mohamed Abou-el-Enein, executive director of the Joint USC/CHLA Cell Therapy Program at USC's Keck School of Medicine.

Regulatory agencies like the FDA need to crack down on these misinformation campaigns, several experts say.

What they're saying: Turner says in that period the FDA contacted about 400 businesses to warn of noncompliance and issued several warning letters, but adds that was "probably of very little consequence. ... A one-year period could be justified, but three years is basically like a security guard walking away from the post, and you can guess what's going to happen."

The big picture: This is a global threat as well, Master and Abou-el-Enein say. In a recent perspective in the journal Stem Cell Reports, they argue for the WHO to establish an expert advisory committee to explore global standards.

What's next: Researchers are still hopeful stem cell therapies can be effective but emphasize the need for more research into how stem cells work and how they can be manipulated for therapies.

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The growing global "infodemic" around stem cell therapies - Axios

NanoString Launches nCounter Stem Cell Characterization Panel to Advance the Development of Stem Cell Therapy – Yahoo Finance

SEATTLE, Jun 22, 2021--(BUSINESS WIRE)--NanoString Technologies, Inc. (NASDAQ: NSTG), a leading provider of life science tools for discovery and translational research, today announced the launch of the nCounter Stem Cell Characterization Panel for the analysis and optimization of stem cell lines used in the development of potential novel therapeutics.

Recent breakthroughs in stem cell therapy, regenerative medicine, and CRISPR engineering have advanced the development of promising new treatments for debilitating diseases across a broad range of research areas, including neurological and cardiovascular disease, vision loss, and certain types of cancers. However, one of the biggest challenges with stem cell research is the high variability found within the development and manufacturing process that impacts the ability of the stem cells to differentiate and function. The new nCounter Stem Cell Characterization panel measures the eight essential components of stem cell biology and provides a novel, standardized assay for evaluating factors that influence and determine viability, functionality, and pluripotency.

"The simple, automated workflow and highly reproducible, digital results make the nCounter system an excellent fit for all types of stem cell applications," said Chad Brown, senior vice president of Sales and Marketing at NanoString. "With this panel, researchers have a powerful new tool that can quickly assess stem cell health to advance development efforts and optimize stem cell production, achieving robust results in less than 24 hours."

"The Process Development team at ARMI-BioFabUSA is very excited to use the nCounter Stem Cell Characterization panel across a number of our projects where we are developing human tissues composed of mature cells differentiated from stem cells. The Stem Cell Characterization Panel will give us greater insight into the differentiation status of our cells and the success of our current process development and manufacturing runs," said Damian Hile, senior process development scientist at Advanced Regenerative Manufacturing Institute-BioFabUSA (ARMI-BioFabUSA).

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The novel 770 gene panel is available for humans and mice and was designed at NanoString with input from leading stem cell experts. To learn more about the nCounter Stem Cell Characterization Panel, visit NanoString at the virtual 2021 ISSCR Conference June 21-26. In addition, NanoString is sponsoring the Cellular Identity: Pluripotency Dynamics session, with Joseph Beechem, Ph.D., chief scientific officer at NanoString.

To learn more about the panel and how the development of the panel can expedite stem cell research, visit the Brief nCounters stem cell experience.

About ARMI-BioFabUSA

The Advanced Regenerative Manufacturing Institute (ARMI), headquartered in Manchester, NH, is an organization funded by the United States Department of Defense. ARMI's mission is to make practical the large-scale manufacturing of engineered tissues and tissue-related technologies to benefit existing industries and grow new ones. ARMI brings together a consortium of over 150 partners from across the industry, government, academia and the non-profit sector to develop next-generation manufacturing processes and technologies for cells, tissues and organs. For more information on ARMI-BioFabUSA, please visit http://www.ARMIUSA.org.

About NanoString Technologies, Inc.

NanoString Technologies is a leading provider of life science tools for discovery and translational research. The company's nCounter Analysis System is used in life sciences research and has been cited in more than 4,300 peer-reviewed publications. The nCounter Analysis System offers a cost-effective way to easily profile the expression of hundreds of genes, proteins, miRNAs, or copy number variations, simultaneously with high sensitivity and precision, facilitating a wide variety of basic research and translational medicine applications, including biomarker discovery and validation. The company's GeoMx Digital Spatial Profiler enables highly-multiplexed spatial profiling of RNA and protein targets in a variety of sample types, including FFPE tissue sections.

For more information, please visit http://www.nanostring.com.

NanoString, NanoString Technologies, the NanoString logo, GeoMx, and nCounter are trademarks or registered trademarks of NanoString Technologies, Inc. in various jurisdictions.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210622005265/en/

Contacts

Doug Farrell, NanoString Vice President, Investor Relations & Corporate Communications dfarrell@nanostring.com Phone: 206-602-1768

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NanoString Launches nCounter Stem Cell Characterization Panel to Advance the Development of Stem Cell Therapy - Yahoo Finance

Catalent to Acquire RheinCell Therapeutics, Strengthening a Path Towards Industrialization of Induced Pluripotent Stem Cell-based Therapies – Yahoo…

Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, today announced that it has reached an agreement to acquire RheinCell Therapeutics GmbH, a developer and manufacturer of GMP-grade human induced pluripotent stem cells (iPSCs). Upon completion, the acquisition will build upon Catalent's existing custom cell therapy process development and manufacturing capabilities with proprietary GMP cell lines for iPSC-based therapies. The deal will enable Catalent to offer the building blocks to scale iPSC-based cell therapies while reducing barriers to entry to the clinic for therapeutic companies and is expected to close before the end of 2021, subject to customary conditions. Financial details of the transaction have not been disclosed.

SOMERSET, N.J., June 24, 2021 /PRNewswire-PRWeb/ -- Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, today announced that it has reached an agreement to acquire RheinCell Therapeutics GmbH, a developer and manufacturer of GMP-grade human induced pluripotent stem cells (iPSCs). Upon completion, the acquisition will build upon Catalent's existing custom cell therapy process development and manufacturing capabilities with proprietary GMP cell lines for iPSC-based therapies. The deal will enable Catalent to offer the building blocks to scale iPSC-based cell therapies while reducing barriers to entry to the clinic for therapeutic companies and is expected to close before the end of 2021, subject to customary conditions. Financial details of the transaction have not been disclosed.

iPSCs are cells that can be differentiated into various cell types to address a wide range of therapeutic indications. Founded in 2017, RheinCell has undertaken significant research and development of full GMP human leukocyte antigen (HLA)-matched cell banks with superior genomic integrity, as well as investing in development-scale operational capabilities. RheinCell is based in Langenfeld, near Dsseldorf, Germany. Upon closing, RheinCell's current employees will join Catalent's Cell & Gene Therapy business.

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"We formed RheinCell based on our deep scientific and regulatory expertise in the promising field of cell-based therapies," commented Juergen Weisser, Chief Executive Officer, RheinCell Therapeutics. He added, "We are convinced Catalent will be able to substantially accelerate RheinCell's future growth and help to support customers around the globe that are interested in our GMP-grade iPSC lines and iPSC-based services to feed their development pipelines in this exciting and highly demanding new therapeutic field."

"By offering a renewable, and standardized, source of cells for further product development, iPSCs have the potential to be a disruptive technology that could fuel the development of the next generation of cell therapies and substantially enhance the ability to manufacture at scale," said Julien Meissonnier, Vice President and Chief Scientific Officer, Catalent. He added, "Catalent is committed to building a full-scale value chain for emerging modalities and accelerating their path to market through expertise and innovation. This acquisition further strengthens Catalent's position in these new therapeutic areas, by pioneering tools and techniques to substantially advance scale-up to meet the demands of clinical and commercial manufacturing."

"This latest acquisition fuels the extraordinary growth of Catalent Cell & Gene Therapy, and the expertise and deep knowledge in iPSC cell lines that RheinCell brings will immediately boost our cell therapy portfolio, allowing us to offer iPSC banks to our customers as a premium source for their therapeutic development pathway," said Manja Boerman, Ph.D., President, Catalent Cell & Gene Therapy. She added, "The addition of the RheinCell team to our growing cell therapy network will create an opportunity to share cutting-edge expertise across our global centers of excellence."

Since 2020, Catalent has invested in its cell therapy capabilities with four strategic expansions at its Gosselies, Belgium, campus the location of its European Center of Excellence for cell and gene therapy. Together with its U.S. cell and gene therapy facilities across Texas and Maryland, Catalent continues to increase its clinical and commercial-scale manufacturing capabilities across the full range of cell and gene therapy activity.

About RheinCell Therapeutics GmbH RheinCell develops and manufactures GMP-grade human induced pluripotent stem cells (iPSCs) for the next generation of cell therapies. Its production pipeline focuses on high immune compatibility and low rejection potential, with a spotlight on solutions for off-the-shelf, allogenic therapeutics. RheinCell provides exclusive access to clinically approved and consented cord blood cells, proprietary cell reprogramming protocols, state-of-the-art cleanroom and cell culture facilities, GMP-compliant manufacturing processes, and a first-class community of iPSC workflow experts who also develop GMP-compliant differentiation protocols in close cooperation with customers. For more information, visit http://www.rheincell.de

About Catalent Cell & Gene Therapy Catalent Cell & Gene Therapy is an industry-leading technology, development, and manufacturing partner for advanced therapeutics. Its comprehensive cell therapy portfolio includes a wide range of expertise across a variety of cell types including CAR-T, TCR, TILs, NKs, iPSCs, and MSCs. With deep expertise in viral vector development, scale-up and manufacturing for gene therapies, Catalent is a full-service partner for plasmid DNA, adeno-associated viral (AAV), lentiviral and other viral vectors, oncolytic viruses, and live virus vaccines. An experienced and innovative partner, Catalent Cell & Gene Therapy has a global network of dedicated, small- and large-scale clinical and commercial manufacturing facilities, including an FDA-licensed viral vector facility, and fill/finish capabilities located in both the U.S. and Europe.

About Catalent Catalent is the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products. With over 85 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable global clinical and commercial product supply. Catalent employs over 15,000 people, including approximately 2,400 scientists and technicians, at more than 45 facilities, and in fiscal year 2020 generated over $3 billion in annual revenue. Catalent is headquartered in Somerset, New Jersey. For more information, visit http://www.catalent.com

More products. Better treatments. Reliably supplied.

Media Contact

Chris Halling, Catalent, +447580041073, chris.halling@catalent.com

Richard Kerns, Northern Exposure Public Relations, +441617285880, chris.halling@catalent.com

SOURCE Catalent

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Catalent to Acquire RheinCell Therapeutics, Strengthening a Path Towards Industrialization of Induced Pluripotent Stem Cell-based Therapies - Yahoo...

Immusoft Announces Formation of Scientific Advisory Board – Business Wire

SEATTLE--(BUSINESS WIRE)--Immusoft, a cell therapy company dedicated to improving the lives of patients with rare diseases, announced today the formation of its Scientific Advisory Board (SAB) composed of world-renowned experts to provide external scientific review and high-level counsel on the Companys research and development programs.

The SAB will work closely with the Immusoft leadership team to advance and expand its leadership position in B cells as biofactories for therapeutic protein delivery, a novel approach that Immusoft has pioneered. The Company is currently preparing for the near-term clinical development of its lead investigational drug candidate ISP-001, a first-in-class investigational treatment for Hurler syndrome, the most severe form of mucopolysaccharidosis type 1 (MPS I), a rare lysosomal storage disease.

We are excited and privileged to have the opportunity to work with this group of rare disease and cell therapy experts, on the development of our pipeline, said Sean Ainsworth, Chief Executive Officer, Immusoft. These thought leaders bring tremendous understanding of rare diseases, as well as extensive experience in drug development from discovery through to late-stage clinical trials. We look forward to their continued contributions at Immusoft as we enter a new stage in advancing ISP-001 into clinical trials this year."

Members of the Immusoft Scientific Advisory Board are as follows:

Robert Sikorski, M.D., Ph.D., is Head of the SAB and consulting Chief Medical Officer at Immusoft. Dr. Sikorski currently serves as the Managing Director of Woodside Way Ventures, a consulting and investment firm that helps biotechnology companies and investors advance lifesaving technologies through clinical development. Prior to that, he was Chief Medical Officer of Five Prime Therapeutics (acquired by Amgen). Earlier in his career, he played a leading role in building MedImmunes oncology portfolio through partnering and acquisition efforts. Before joining Medimmune, he led late-stage clinical development and post-marketing efforts for several commercial drugs and drug candidates at Amgen. Dr. Sikorski began his career as a Howard Hughes Research Fellow and Visiting Scientist at the National Cancer Institute and the National Human Genome Research Institute in the laboratory of Nobel Laureate Harold Varmus. Additionally, he has served as an editor for the journal Science and Journal of the American Medical Association. Dr. Sikorski obtained his MD and PhD degrees as a Medical Scientist Training Program awardee at the Johns Hopkins School of Medicine.

Paula Cannon, Ph.D., is a Distinguished Professor of Molecular Microbiology and Immunology at the Keck School of Medicine of the University of Southern California, where she leads a research team that studies viruses, stem cells and gene therapy. She obtained her PhD from the University of Liverpool in the United Kingdom, and received postdoctoral training at both Oxford and Harvard universities. Her research uses gene editing technologies such as CRISPR/Cas9, to develop treatments for infectious and genetic diseases of the blood and immune systems. In 2010, her team was the first to show that gene editing could be used to mimic a natural mutation in the CCR5 gene that prevents HIV infection, and which has now progressed to a clinical trial in HIV-positive individuals.

Michael C. Carroll, Ph.D., is a Senior Investigator at Boston Children's Hospital and Professor of Pediatrics, Harvard Medical School. His recent research focuses on two major areas, i.e. neuroimmunology and peripheral autoimmunity. Using murine models of neuro-psychiatric lupus, his group is testing their hypothesis that interferon alpha from peripheral inflammation enters the brain and mediates synapse loss and symptoms of cognitive decline observed in patients. Following-up on a large genetic screen in schizophrenia patients, they recently reported that over-activation of a process known as complement-dependent, microglia-mediated synaptic pruning in novel strains of mice can induce psychiatric symptoms of schizophrenia. In a murine lupus model, his lab has identified that self-reactive B cells evolve with kinetics similar to that of foreign antigen responding B cells providing a novel explanation for epitope spreading. Dr. Carroll received his PhD from UT Southwestern Medical School and his postdoctoral training with the Nobel Laureate, Professor Rodney R. Porter at Oxford University. He is a recipient of awards from the Pew Foundation, American Arthritis Foundation and the National Alliance for Mental Health.

Hans-Peter Kiem, M.D., Ph.D. is the Stephanus Family Endowed Chair for Cell and Gene Therapy at Fred Hutchinson Cancer Research Center. He is a world-renowned pioneer in stem-cell and gene therapy and in the development of new gene-editing technologies. His focus has been the development of improved treatment and curative approaches for patients with genetic and infectious diseases or cancer. For gene editing, his lab works on the design and selection of enzymes, known as nucleases, which include CRISPR/Cas. These enzymes function as molecular scissors that are capable of accurately disabling defective genes. By combining gene therapys ability to repair problem-causing genes and stem cells regenerative capabilities, he hopes to achieve cures of diseases as diverse as HIV, leukemia and brain cancer. He is also pioneering in vivo gene therapy approaches to make gene therapy and gene editing more broadly available and accessible to patients and those living with HIV, especially in resource-limited settings. He received his M.D. and Ph.D. at the University of Ulm, Germany.

Bruce Levine, Ph.D., Barbara and Edward Netter Professor in Cancer Gene Therapy is the Founding Director of the Clinical Cell and Vaccine Production Facility in the Department of Pathology and Laboratory Medicine and the Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania. First-in-human adoptive immunotherapy trials include the first use of a lentiviral vector, the first infusions of gene edited cells, and the first use of lentivirally-modified cells to treat cancer. Dr. Levine has overseen the production, testing and release of 3,100 cellular products administered to more than 1,300 patients in clinical trials since 1996. Dr. Levine is a recipient of the William Osler Patient Oriented Research Award, the Wallace H. Coulter Award for Healthcare Innovation, the National Marrow Donor Program/Be The Match ONE Forum 2020 Dennis Confer Innovate Award, serves as President of the International Society for Cell and Gene Therapy, and on the Board of Directors of the Alliance for Regenerative Medicine. Dr. Levine received a B.A. in Biology from the University of Pennsylvania and a Ph.D. in Immunology and Infectious Diseases from Johns Hopkins University.

Peter Sage, Ph.D., is an Assistant Professor of Medicine at Harvard Medical School and an Associate Immunologist at Brigham and Womens Hospital. Dr. Sage is also a member of the Committee on Immunology (COI) at Harvard Medical School. Dr. Sage obtained his PhD in Immunology from Harvard Medical School in 2013, during which he received the Jeffrey Modell Prize. He completed a post-doctoral fellowship in the laboratory of Dr. Arlene Sharpe in the Department of Immunology at Harvard Medical School in 2017. Dr. Sage started his independent laboratory in 2017 at the Transplantation Research Center in the Division of Renal Medicine of Brigham and Womens Hospital. Dr. Sages laboratory focuses on studying how the immune system controls B cell and antibody responses in settings of health and disease.

About Immusoft

Immusoft is a cell therapy company focused on developing a novel therapies for rare diseases using a sustained delivery of protein therapeutics from a patients own cells. The company is developing a technology platform called Immune System Programming (ISP), which modifies a patients B cells and instructs the cells to produce gene-encoded medicines. The B cells that are reprogrammed using ISP become miniature drug factories that are expected to survive in patients for many years. The company is based in Seattle, WA. For more information, visit http://www.immusoft.com.

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Immusoft Announces Formation of Scientific Advisory Board - Business Wire

Jasper Therapeutics and Aruvant Announce Research Collaboration to Study JSP191, an Antibody-Based Conditioning Agent, with ARU-1801, a Novel Gene…

REDWOOD CITY, Calif. and NEW YORK and BASEL, Switzerland, June 21, 2021 /PRNewswire/ --Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, andAruvant Sciences, a private company focused on developing gene therapies for rare diseases, today announced that they have entered a non-exclusive research collaboration to evaluate the use of JSP191, Jasper's anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning agent with ARU-1801, Aruvant's investigational lentiviral gene therapy for sickle cell disease (SCD). The objective of the collaboration is to evaluate the use of JSP191 as an effective and more tolerable conditioning agent that can expand the number of patients who can receive ARU-1801, a potentially curative treatment for SCD.

"This research collaboration with Aruvant is the first to use a clinical-stage antibody-based conditioning agent and a novel clinical-stage gene therapy, giving this combination a clear advantage by moving beyond the harsh conditioning agents currently used for gene therapy and establishing this next-generation potentially curative treatment as a leader in sickle cell disease," said Kevin N. Heller, M.D., executive vice president, research and development of Jasper. "Our goal is to establish JSP191 as a potential new standard of care conditioning agent, broadly in autologous gene therapy and allogeneic hematopoietic stem cell transplantation."

Gene therapies and gene editing technologies generally require that a patient's own hematopoietic stem cells first be depleted from the bone marrow to facilitate the engraftment of the new, gene-modified stem cells through a process called conditioning. Other investigational gene therapies and gene editing approaches in SCD use a high-dose chemotherapy such as busulfan for the conditioning regimen, which can place patients at prolonged risk for infection and bleeding, secondary malignancy and infertility. ARU-1801 is currently the only gene therapy that has demonstrated durable efficacy using both a lower dose of chemotherapy and a different agent than busulfan with a more limited side effect profile. The Aruvant-Jasper partnership is focused on evaluating the potential of using JSP191, a highly targeted anti-CD117 (stem cell factor receptor) monoclonal antibody agent, as the foundationof a novel conditioning regimen for use in combination with ARU-1801 to further reduce the negative side effects while maintaining efficacy.

"The unique attributes of ARU-1801 enable us to bring a potentially curative one-time therapy to individuals with sickle cell disease that can be delivered in the safest way possible," said Will Chou, M.D., Aruvant chief executive officer. "By partnering with Jasper to evaluate the use of JSP191 with ARU-1801, we are one step closer to developing a next-generation definitive therapy with an even more patient-friendly conditioning regimen. We believe that this combination may be able to further expand the number of patients who can benefit from ARU-1801 in the future, including potentially those with more moderate disease."

About JSP191 JSP191 is a humanized monoclonal antibody in clinical development as a conditioning agent that blocks stem cell factor receptor signaling leading to clearance of hematopoietic stem cells from bone marrow, creating an empty space for donor or gene-corrected transplanted stem cells to engraft. While hematopoietic cell transplantation can be curative for patients, its use is limited because standard high dose myeloablative conditioning is associated with severe toxicities and standard low dose conditioning has limited efficacy. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients. It is currently enrolling in two clinical trials for myelodysplastic syndromes (MDS)/acute myeloid leukemia (AML) and severe combined immunodeficiency (SCID) and expects to begin enrollment in four additional studies in 2021 for severe autoimmune disease, sickle cell disease, chronic granulomatous disease and Fanconi anemia patients undergoing hematopoietic cell transplantation.

About ARU-1801 ARU-1801 is designed to address the limitations of current curative treatment options, such as low donor availability and the risk of graft-versus-host disease (GvHD) seen with allogeneic stem cell transplants. Unlike investigational gene therapies and gene editing approaches which require fully myeloablative conditioning, the unique characteristics of ARU-1801 allow it to be given with reduced intensity conditioning ("RIC"). Compared to myeloablative approaches, the lower dose chemotherapy regimen underlying RIC has the potential to reduce not only hospital length of stay, but also the risk of short- and long-term adverse events such as infection and infertility. Preliminary clinical data from the MOMENTUMstudy, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, demonstrate continuing durable reductions in disease burden.

The MOMENTUM Study Aruvant is conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time potentially curative investigational gene therapy for patients with SCD. This Phase 1/2 study is currently enrolling participants, and information may be found at momentumtrials.comwhich includes a patient brochure, an eligibility questionnaireand information for healthcare providers.

About Jasper Therapeutics Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, a first-in-class anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic and autologous hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical engineered hematopoietic stem cell (eHSC) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

About Aruvant Sciences Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an active research program with a lead product candidate, ARU-1801, in development for individuals suffering from sickle cell disease (SCD). ARU-1801, an investigational lentiviral gene therapy, is being studied in a Phase 1/2 clinical trial, the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. The company's second product candidate, ARU-2801, is in development to cure hypophosphatasia, a devastating, ultra-orphan disorder that affects multiple organ systems and leads to high mortality when not treated. Data from pre-clinical studies with ARU-2801 shows durable improvement in disease biomarkers and increased survival. For more information on the ongoing ARU-1801 clinical study, please visit http://www.momentumtrials.comand for more on the company, please visit http://www.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciencesand on Instagram @Aruvant_Sciences.

About Roivant Roivant's mission is to improve the delivery of healthcare to patients by treating every inefficiency as an opportunity. Roivant develops transformative medicines faster by building technologies and developing talent in creative ways, leveraging the Roivant platform to launch Vants nimble and focused biopharmaceutical and health technology companies. For more information, please visit http://www.roivant.com.

SOURCE Aruvant Sciences andJasper Therapeutics

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Jasper Therapeutics and Aruvant Announce Research Collaboration to Study JSP191, an Antibody-Based Conditioning Agent, with ARU-1801, a Novel Gene...