A recent announcement from Voyager Therapeutics outlined a shift in the companys strategy towards an exciting new technology for gene therapy delivery. Unfortunately this also means that in the short term, they have dropped previous plans to test an HD gene therapy in people with HD. While this news is disappointing, the decision to embrace a novel approach now could potentially lead to a safer, more accurate, and less invasive HD therapeutic in the longer term.

This news provides an opportunity for the HDBuzz team to talk more about the current landscape and share the latest news from the gene therapy pipeline.

Before we get into the HD gene therapy pipeline, lets review some basic genetics. With the advent of RNA-based COVID vaccines, weve all been hearing a lot about RNA. But how does RNA differ from DNA, and what does it mean if we alter either of these?

You can think of DNA like a blueprint its the master plan at the genetic level for every cell in your body. To ensure that master plan stays in pristine condition, cells make copies of DNA to work from when they make proteins. That copy of the DNA is RNA. Because RNA is just a copy, it can be well-used without much care if it gets a bit tattered. If it does, the cell can just make another RNA copy from the DNA blueprint, and voila! The cell has a fresh RNA copy that can be used to produce more protein.

Scientists have leveraged this knowledge to come up with clever ways to get cells to produce more or less of the proteins theyre interested in.

In the case of Huntingtons disease, were interested in reducing production of the huntingtin protein that damages cells referred to as huntingtin-lowering. That could be done in 2 ways:

1) Destroy the RNA copies as they are produced, but leave the DNA blueprint intact. This is the strategy behind antisense oligonucleotides (ASOs), like those that were being tested in trials by Roche and Wave.

2) Modify the message of the DNA blueprint, so it either cant be copied into RNA or contains new instructions to help destroy the RNA. This approach is what we refer to when we say gene therapy it changes what is made from the blueprint without altering it.

While both of the strategies above ultimately lower huntingtin protein production, they are different for several reasons. The primary difference is that destroying only the RNA copy requires repeated doses. Because the cell still has the original DNA blueprint for the huntingtin protein, it will continue to make more RNA copies. So unless the copy is constantly destroyed, the huntingtin protein will still be produced. While the repeated doses may seem like a nuisance, this type of approach means that the effect of any drug that targets only the RNA will eventually wear off an added safety benefit.

Gene therapy approaches for huntingtin-lowering, like those being pursued by uniQure and Voyager, target huntingtin with a one-time delivery of genetic instructions to cells of the brain. These instructions then tell the cells to continuously produce RNA molecules that can interfere with the making of huntingtin, leading to lower protein levels. This is a one-and-done type of approach no repeated doses necessary. But something to consider is that this approach also means that if there are other effects because of huntingtin-lowering, theres no going back.

Its important to note that even though DNA is being added in these gene therapy approaches, a persons DNA is not being edited. This means that while the gene therapy will have benefits in the person being treated, it wont be passed to future generations. That would require a gene editing strategy like CRISPR.

Current gene therapy strategies for brain diseases like HD would require brain surgery since these DNA-altering drugs cant get past the barrier of the brain. This major limitation is something Voyager wanted to get around.

On August 9th, 2021, Voyager Therapeutics issued a press release about their finances, recent leadership transitions, and importantly, a major shift in their scientific pipeline. The announcement had a lot of corporate and investor information, but the science content centered around an improved gene therapy delivery system and a proprietary discovery platform, which in combination could allow Voyager to develop less invasive methods of delivering gene therapies for rare diseases like HD.

Like previous genetic therapies developed by Voyager (and other companies, like uniQure), delivery involves packaging genetic drugs inside a harmless virus called an AAV. In the field of HD gene therapy, AAVs are used to deliver genetic instructions that cause cells to divert one tiny wing of their machinery towards producing a genetic antidote to the expanded HD gene.

Voyager has developed a proprietary new AAV packaging and has collected evidence from monkeys that these AAVs can be delivered with greater safety, potency, and accuracy. They have also invested in a new discovery system for identifying and improving upon AAVs for additional diseases and drug targets.

Whereas AAV delivery of HD therapies has so far required a brain surgery, drugs developed using Voyagers new platform can be designed for delivery through an injection into the blood, so there is potential for less invasive delivery to the brain.

The press release shared that Voyager will shift its focus to the new technologies and away from older existing ones. The upside is the next-generation technology; the downside is that this means that Voyager will no longerbe pursuing the therapy that they had previously developed for HD. This drug, VY-HTT01, was meant to be the focus of a planned clinical safety trial called VYTAL, which would have begun later this year. No participants had yet been recruited it was still in early planning stages.

Although the loss of a gene therapy that was approaching the clinic is a significant short-term setback, Voyagers shift in focus now to accommodate a new scientific development provides a new and potentially better therapeutic avenue for HD.

Luckily, there are other companies working on gene therapy approaches, who have also provided recent public updates on their ongoing or upcoming trials for Huntingtons disease. Weve provided brief summaries for each of these below; stay tuned for additional updates as these efforts advance.

The first company out of the HD gene therapy gate was uniQure, who are developing a viral therapy known as AMT-130, which has the goal of delivering instructions to brain cells for the making of a special kind of RNA that will find and destroy the RNA for the huntingtin gene. In this way, gene therapy can be used to permanently induce huntingtin-lowering. After many years of careful work in animals, uniQure launched their safety study, and as of this summer they have excitingly been able to complete surgeries for 12 of the planned 26 patients. A strictly regulated schedule has allowed the team to carefully monitor any safety worries, and none have emerged so far.

Additional companies in the preclinical stages of development of virus-based huntingtin-lowering gene therapies include Spark, Sanofi, and AskBio.

Another gene therapy approach to huntingtin-lowering relies on a novel tool known as a Zinc Finger. Weve been writing about this approach at HDBuzz since 2012, and more recently (2019) about a large scale study of the tools in HD mice. Recently, the Japanese drug company Takeda has taken over the HD program from Sangamo Therapeutics, who initially developed the drugs. A key benefit of the Zinc Finger approach for huntingtin-lowering is that it allows selective silencing of just the mutant huntingtin gene, while sparing the normal copy that nearly every HD patient has.

We mentioned the multiple-delivery strategy which was used by Roche and Wave in the trials that concluded unsuccessfully this spring. Despite these setbacks, ASOs and other RNA-based strategies are still being actively developed as HD therapies.

Wave Life Sciences has redesigned the chemistry of their ASO drugs, which could lead to better potency and the ability to use lower doses in people with HD. They have announced plans to launch a safety trial of a new ASO by the end of 2021. The drug is called WVE-003, and it targets the expanded form of huntingtin.

Novartis and PTC Therapeutics are developing drugs called splice modulators that also target huntingtin RNA, but can be delivered by mouth. We covered Novartiss drug, branaplam, in a recent article; a trial in HD patients is planned to begin by the end of 2021.

NeuBase Therapeutics is developing an ASO drug called NT0100 which also aims to target only the expanded form of huntingtin.

At the end of July, a company called Vico Therapeutics received a special rare disease therapeutics status, known as Orphan Drug Designation, to develop their ASO for HD, known as VO659.

Companies like Atalanta and Alnylam/Regeneron are developing ways to lower huntingtin through RNA interference (RNAi) which, similar to ASOs, target copies of RNA and would require multiple deliveries.

There are more strategies in the works, some of which also rely on gene therapy or destroying copies of RNA, like targeting the expansion of CAG repeats, which is being explored by companies like Triplet Therapeutics and LoQus23 Therapeutics.

There are also many approaches to HD drug development that diverge from genetics but focus on addressing other aspects of HD biology, like preserving or boosting connections between neurons, or treating aggression, memory issues, or movement problems. Those already being tested in human we explored in a recent clinical trials roundup. Other companies have pre-clinical programs aimed at strategies like cleaning up existing huntingtin protein that litters brain cells, suppressing inflammation in the HD brain, and more newcomers to HD research are quite frequent (and very welcome)!

Gene therapy for brain diseases is amongst the most cutting edge approaches to trying to fight HD. As with any new field, there are bound to be many ups and downs on the way to a treatment. The recent update from Voyager is a good example of this while its disappointing that theyll not be running their planned trial later this year, its very exciting that theyve developed these new technologies and want to apply them to help HD families. The extensive efforts from other companies in the gene therapy space and beyond suggest that a lot of really exciting strategies are being applied to the problem of HD.

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Unpacking recent gene therapy press - HDBuzz

Dallas biotech firm Taysha Gene Therapies reported a $40.9 million quarterly loss this week as it expands its research and development efforts and its manufacturing footprint.

The company, founded through a partnership with researchers at the University of Texas Southwestern Medical Center, also announced an agreement to borrow up to $100 million from Silicon Valley Bank. The loan adds to the companys $197.4 million in cash on hand to support its growth as it plans to submit one of its drugs for regulatory approval for the first time later this year.

Taysha is one of several biotech companies looking to stake its claim on the gene therapy frontier as the landscape begins to grow crowded with both startups like Taysha and pharma giants like Swiss firms Roche and Novartis. Many gene therapies, including most of Tayshas, seek to treat genetic disorders by giving patients genetically modified, benign viruses that carry healthy copies of the gene needed.

Founded in April 2020, Taysha became publicly traded less than five months later. Its developing its treatments under a license agreement with the gene therapy program at UT Southwestern led by Steven Gray and Dr. Berge Minassian, who both serve as Taysha advisers.

Taysha began with a pipeline of 15 gene therapy programs, which has since expanded to 26 programs.

It develops therapies to treat disorders of the central nervous system, many of which affect children. While many of its programs treat conditions that are only present in a small number of patients, the company is also working on treatments for larger patient populations.

The financial results come amid a period of rapid growth for Taysha, which raised $181 million in its September 2020 IPO. The company has dramatically increased its research and development expenses as it aims to begin as many as three clinical trials before the end of the year.

Tayshas R&D expenses for the first half of this year totaled almost $54.5 million, compared with $8.6 million in the same period last year.

Not only did we make the transition from private to public company last year, but also a preclinical to clinical company, Taysha founder, president and CEO RA Session II told The News in an interview last month. On the heels of that, weve now made the transition from a clinical-stage company to a pivotal-stage company embarking on regulatory discussions around approval pathways for our lead program. So its a really exciting time.

Taysha plans to initiate phase one and two clinical trials in the U.S. for its gene therapy for GM2 gangliosidosis, also known as Tay-Sachs disease, before the end of the year. The program also is currently in a clinical trial at Queens University in Canada. The Canadian clinical trial will release safety and biomarker data in the second half of this year.

The Silicon Valley Bank loan is expected to add an extra infusion of cash as Taysha pursues regulatory approval for TSHA-120, its program for giant axonal neuropathy, a disorder that causes parts of neurons to deteriorate and causes issues with motion and sensation in young children. Taysha has drawn $30 million of the loan.

The terms of this deal are quite attractive, Session said in the companys earnings call on Monday. Its all about being able to move things [that are] best-in-class forward and not necessarily have to slow anything down or make any particular trade-offs as we get into the next year. Because we cant predict whats going to happen in the equity capital markets, we thought this was just a wonderful opportunity to be able to add some additional dry powder to the tank.

The company also made significant personnel moves this quarter with the hiring of Mary Newman as chief development officer and Claire Aldridge as chief of staff and senior vice president of business operations.

Newman was previously senior vice president of regulatory affairs at Astellas Gene Therapies and has over 30 years of biotech experience. Aldridge has long been a fixture of the Dallas biotech world and is the former associate vice president of commercialization and business development at UT Southwestern. She has also previously worked with Dallas philanthropist and biotech investor Lyda Hill as vice president of venture development at Remeditex Ventures, Hills biotech venture capital firm.

In May, Taysha moved into its new headquarters in the Pegasus Park development near Dallas medical district, a 23-acre campus that seeks to bring the business and science sides of the biotech industry together in one place.

The company also plans to complete its 187,000-square-foot manufacturing facility in Durham, N.C., by the end of 2023 to round out the companys manufacturing process and allow it to continue to scale rapidly. The company is investing $75 million in the facility, which will eventually employ over 200 workers.

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Dallas biotech firm Taysha Gene Therapies reports loss as it ramps up research and development - The Dallas Morning News

Aclinical trial testing a novel gene therapy for a rare neurological disease has been put on hold after one of the participants in a Phase 3 trial developed a bone marrow disorder that can lead to cancer. The pause, announced Monday by the trials sponsor, bluebird bio, and mandated by the US Food and Drug Administration (FDA), was taken out of an abundance of caution, the companys president of rare genetic diseases, Andrew Obenshain, said in a recent quarterly call.

The therapy targets cerebral adrenoleukodystrophy, which is caused by a mutation in the gene for an enzyme called adrenoleukodystrophy protein (ALDP) that breaks down fats. The mutation causes fat to build up in the brain, where it breaks down the insulating myelin that allows neurons to communicate with one another. Because the gene is on the X chromosome, women typically have a least one good copy, so the disease primarily strikes men. Left untreated, it causes damage to hearing, vision, cognition, and coordination. It is often fatal.

Bluebirds gene therapy uses an engineered lentivirus to correct the mutation associated with the disease. Lentiviruses belong to the same family as HIVretrovirusesand have been widely used in gene therapies and other medical applications for many years. While other virus-based platforms using retroviruses had previously been linked to cancer among patients, it is only recently that a lentivirus has been implicated in such an outcome: in February of this year, bluebird bio paused another trial, one for a blood disorder, after two patients developed leukemia-like cancer, Sciencereports, although it was later determined that the virus was likely not the cause, and the trial resumed.

Most in the field were hoping that we would not see such an event with lentiviral vectors, Harry Malech, a gene therapy researcher at the National Institutes of Health, tells Science, adding, I dont think anybodys been . . . saying this couldnt happen.

The cerebral adrenoleukodystrophy therapy involves taking samples of a patients bone marrow and treating the stem cells therein with the modified virus that contains a corrected copy of the gene that encodes ALDP. After a round of chemotherapy to reduce the persons bone marrow cells, the treated cells are infused back into the patient. Thereafter, the patients stem cells produce healthy blood cells with a functional copy of the gene for ALDP. The therapy entered the market in Europe last month following a previous safety and efficacy trial that included 32 patients. A second trial, the one that has now been paused, was set to finish in 2024.

Speaking on the call, bluebird bios Chief Scientific Officer Philip Gregory said that one patient in the second trial developed myelodysplastic syndrome (MDS), a blood disorder that sometimes leads to leukemia, and another two had abnormal bone marrow cells that could progress to MDS. When scientists examined their cells, they found lentiviral DNA inserted at a site in the genome that has previously been linked to MDS in retrovirus-based therapies, suggesting that the virus may have caused the changes.

Specifically, Gregory said the issue is likely caused by the virus promoter, the DNA sequence that turns on the therapeutic copy of the gene. To ensure the gene produces enough ALDP in the brain to be an effective treatment, the researchers needed a strong promoter, but as a consequence, the promoter had off-target effects, turning on other genes in the area around the mutation, including cancer genes, Gregory speculated.

Donald Kohn, a gene therapy researcher at the University of California, Los Angeles, who helped design the viral vector, tells Sciencethat in the time since bluebird bio first began developing the therapy, researchers have identified other promoters that might be able to do the job with a lower risk of causing cancer. He adds that this particular incident shouldnt preclude scientists from pursuing other lentivirus treatments, as the issue seems to come down to design, and Kohn doesnt know of any other lentivirus therapies that use the same type of promoter.

Panam Malik, a hematologist at Cincinnati Childrens Hospital who was not involved in the work, similarly tells Science that virus-based platforms should be highlighted for the good they have done. This is a severe adverse event, she says, but adds, we should never lose sight of the fact that so many patients . . . have been helped. Despite this rare incident, the findings could help scientists and researchers design safer and better vectors for the future.

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Participants Diagnosis Halts Gene Therapy Clinical Trial - The Scientist

Three research teams investigating HIV cures received a total of nearly $600,000 in grants from amfAR, The Foundation for AIDS Research. All three groups are exploring cutting-edge gene therapies as a possible way to eradicate the HIV reservoir and cure HIV.

The HIV reservoir is one of the main obstacles to curing HIV. The term refers to virus that hides in latent cells in lymph nodes and other locations. HIV meds cant reach this virus because its not actively replicating, but in the absence of antiretrovirals in the body, HIV in the reservoir can begin making new virus that replicates and circulates, causing destruction.

Gene therapy strategies hold tremendous potential for curing HIV, but we must walk the fine line between optimizing this highly technical approach and making it feasible around the world, said amfAR vice president and director of research Rowena Johnston, PhD, in an amfAR press release. These new awards should get us closer to unleashing the full power of gene therapy against HIV.

Heres a look at the approaches the research teams hope will eliminate the HIV reservoir.

The Food and Drug Administration first approved CAR-T therapywhich stands for chimeric antigen receptor T-cell therapyin 2017. A part of the bodys immune system, T cells are white blood cells that attack foreign substances. CAR-T therapy is used to treat some forms of cancer, but as POZs sister publication Cancer Health has reported, it hasnt been commonly used because it is expensive and must be custom made for each patient.

In the case of cancer treatment, CAR-T therapy involves taking a patients T cells and sending them to a lab where they are genetically modified to recognize and attack the cancer. The resulting cells are then infused back into the individual after the person has received strong chemotherapy to kill off some of their existing immune cells to make room for the new ones.

When applied to HIV treatment, CAR-T therapy has been less successful because the resulting cells are vulnerable to HIV infection. The amfAR grant will help a team of researchers explore a way to get around this problemand to do so in a manner thats much cheaper than the cancer therapy. Scientists led by Anastasios Karadimitris, PhD, MRCP, FRCPath, of Imperial College in London, will combine CAR-T therapy with another type of cell found in the immune system: invariant natural killer T cells, or iNKT cells. Unlike the T cells used in the cancer therapy, these cells are more resistant to HIV. Another advantage is that theyre more uniform throughout the human population, which means that, hopefully, the resulting HIV treatment would not need be customized for each patient.

In related news, last summer the National Institutes of Health awarded a grant to a group of HIV scientists developing and studying CAR-T therapies. For more, see $14M Federal Grant to Research CAR-T Gene Therapy to Cure HIV.

These types of antibodies show an exceptional ability to bind to infected cells and mark them to be destroyed by other parts of the immune system, such as natural killer T cells. bFAbs were identified by a group of researchers led by Todd Allen, PhD, of theRagon Institute of Massachusetts General Hospital, MIT and Harvard. With amfAR funding, the researchers will test bFAbs alone as well as with genetically engineered CAR-T cells, in the hope that the antibodies and CAR-T cells will be able to target and help destroy only cells infected with HIV. The experiments will be conducted on mice with human immune cells.

Late last year, hopeful headlines announced that the CRISPR-Cas9 gene editing tool successfully snipped simian HIV from monkeys cells, including from the viral reservoir. CRISPR (clustered regularly interspaced short palindromic repeats) technology allows scientists to remove or alter specific bits of DNA using the Cas9 protein, an enzyme that cuts the DNA. In this case, its used to remove HIV that has inserted itself into a persons or monkeys DNA.

The idea is straightforward, but the reality is more complicated, in part because areas of the genome (a collection of all our genes, or DNA) are tightly wound and hard for CRISPR to access. New research shows that HIV might be integrated into these hard-to-reach areas. A team of scientists led by Jori Symons, PhD, of University Medical Center Utrecht in the Netherlands, will explore this possibility in addition to drugs that might open up these areas of the genome so that CRISPR/Cas can access it.

Were pleased to be supporting this impressive trio of research teams that are bringing a range of smart, creative approaches to the challenge of curing HIV, said amfAR CEO Kevin Robert Frost in the press release. We hope these investments generate new insights that will help us develop a curative intervention for all people living with HIV.

Since 1985, amfAR has awarded more than 3,300 grants to HIV researchers across the globe, totaling nearly $600 million. In 2010, the organization started the amfAR Research Consortium on HIV Eradication, or ARCHE, specifically to fund strategies to cure HIV as well as the Countdown to a Cure for HIV/AIDS initiative; in 2014, it launched the amfAR Institute for HIV Cure Research.

Last year, the group launched the amfAR Fund to Fight COVID-19. (Dont worry: Money is not diverted from HIV research.) For an example of this funding, see AIDS Group amfAR Awards Two Grants to Research COVID-19. The article includes a video from a series exploring the intersection of the two diseases.

For more POZ articles about the organization, click the hashtag #amfAR, where youll find headlines like Why Women Are a Vital Part of Cure Research [VIDEO] and Does the Coronavirus Affect the HIV Reservoir?

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Can These Three Gene Therapies Get Us Closer to an HIV Cure? - POZ

After Bluebird Bio (BLUE) announced plans this week to end commercial operations in Europe, the move is raising fresh questions about the extent to which selling such pricey treatments is viable for small biotechs in one of the worlds biggest markets.

In explaining the move, the biotech complained that it has become a losing proposition trying to convince European Union member states to make large upfront payments for therapies that can save health care systems much higher costs later. So instead, the company is shifting its focus to the U.S., where it is more likely to get reimbursed at the desired prices.

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As Bluebird backpedals from Europe, gene therapy players face hurdles - STAT

- Founder and CEO Lindsay Androski previously served as a federal prosecutor and an executive at Roivant Sciences

- MIT spinout Sunflower Therapeutics, focused on improving access to medicines through improvements in complex manufacturing, is Roivant Social Ventures' first investment, building upon prior funding from the Bill and Melinda Gates Foundation

- Nature Gene Therapy publication spotlights efforts to expand gene therapy access in India and Africa

NEW YORK, Aug. 17, 2021 /PRNewswire/ -- Roivant Social Ventures (RSV), a not-for-profit social impact organization launched out of Roivant Sciences, today unveiled its executive team and announced its inaugural partnership with Sunflower Therapeutics, a biopharmaceutical company developing simplified manufacturing methods for therapeutic proteins. RSV also announced the publication in Nature Gene Therapy of the Global Gene Therapy Working Group's initiative to expand gene therapy access in Africa and India.

Executive Team

Lindsay Androski, JD, MBA, CFA is the Founder, President and CEO of Roivant Social Ventures. Ms. Androski sits on the MIT board of trustees, the Visiting Committee for the MIT Department of Biology, and the Visiting Committee for MIT Sponsored Research. She also serves as President of Incubate, an organization which educates policymakers on the role of venture capital in the biopharma industry. Ms. Androski joined the founding team at Roivant Sciences in order to build and lead the transaction team primarily responsible for in-licensing and acquiring over 40 therapeutics programs. Earlier in her career she served as an Assistant U.S. Attorney in the Eastern District of Virginia, where she investigated and prosecuted high-profile cybercrime and national security cases.

Rachel Rubin, who serves as Vice President of Programs at RSV, also joins from Roivant Sciences, where she oversaw launch and business operations for several healthcare technology Vants. Prior to Roivant, Ms. Rubin worked at the Michael J. Fox Foundation for Parkinson's Research and spent several years as an investor.

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Saranna Biel-Cohen joins RSV as Vice President of Strategic Development. Ms. Biel-Cohen previously served as Executive Director of the Hermitage Museum Foundation, where she facilitated major funding and programming partnerships, including with the United States federal government and leading corporations.

Partnership with Sunflower Therapeutics

RSV's first partnership, which includes a financial investment and company incubation assistance, is with Sunflower Therapeutics, a public benefit corporation operationalized in 2019 by CEO Dr. Kerry Love and a team of engineers previously involved in the development of the InSCyT (Integrated and Scalable Cytotechnology) Program in the lab of J. Christopher Love at the Koch Institute for Integrative Cancer Research at MIT.

Sunflower's Daisy System is a multi-product manufacturing platform for distributed production of protein biologics, monoclonal antibodies, and subunit vaccines, which enables efficient production of multiple medicines tailored to a region's specific needs. RSV's funding will be used to deploy the Daisy System for the first time and builds upon prior funding from the Bill & Melinda Gates Foundation in 2019 to develop the Daisy System. The system will be deployed to a non-profit partner serving low-or-middle-income countries (LMICs) and used to bolster local capacity to provide advanced therapeutics to underserved populations and reduce reliance on the global supply chain.

"Roivant Social Ventures is truly a one-of-a-kind impact investor, creating productive partnerships with companies like Sunflower that are focused on increasing global health access and equity," said Dr. Kerry Love, CEO of Sunflower Therapeutics. "I am excited to further development of Sunflower's technologies with the goal of ensuring advanced therapeutics are accessible to all members of the global community."

"We are thrilled to play a role in solving supply chain issues that disadvantage LMICs and inhibit their ability to deliver needed therapeutics to their local populations," said Ms. Androski. "Sunflower Therapeutics embodies the core values of Roivant Social Ventures and is a phenomenal first partner in our mission to use advances in science and technology as vehicles to change the ethical norms of healthcare delivery."

Publication in Nature Gene Therapy

RSV is also a founding member of the Global Gene Therapy Initiative (GGTI), a cross-collaborative group with expertise spanning manufacturing, drug development, patient advocacy, clinical practice, regulatory, and diplomacy, focused on a common goal: to bring gene therapy treatments to patients worldwide. Among other operational pillars, the group is using scaled-down and modular manufacturing systems to deliver gene therapies to patients in Uganda and India by 2024, with an initial focus on HIV and sickle cell therapies.

A publication regarding the group's progress, titled "Towards Access for All: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)", is in press in Nature Gene Therapy, with Lindsay Androski and Alex Popovski as Contributing Authors.

"Gene therapy, and other advanced therapies, offer patients hope far beyond most current treatment methods," said Ms. Androski. "We cannot accept as normal a world where advanced therapies are only available to a small number of patients in the wealthiest countries, and we must prevent this from becoming a reality by investing now in ways to bring these treatments to patients worldwide. We are thrilled to help advance the important work of GGTI."

About Roivant Social VenturesRoivant Social Ventures (RSV) is a not-for-profit social impact organization founded by Roivant Sciences focused on improving healthcare access and outcomes for underserved groups. RSV invests in initiatives to expand worldwide access to cell and gene therapies through simplified manufacturing techniques, facilitates research and development on promising deprioritized programs across the biopharma industry, and encourages continued focus on challenging therapeutic areas where significant unmet medical needs remain. RSV applies Roivant Sciences' unique approach to incubating biotech and healthcare technology companies, including providing non-financial assistance to companies and health initiatives, and seeks to maximize impact through strategic collaborations.

About Sunflower TherapeuticsSunflower Therapeutics, PBC is a unique biotech company with a mission to enable more medicines to reach patients worldwide. Sunflower's goal is to transform access to biologic medicines for patients worldwide by creating novel technologies for development and manufacturing with the whole global community in mind. Using its core technologiesan efficient host, data-driven methods for process development, and novel manufacturing facilitiesthe team aims to create efficient, fast and less costly cycles of development and production for many new innovative patient-focused products. Sunflower's research labs are currently located at LabCentral in Cambridge, Massachusetts.

Cision

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Social Impact Nonprofit Roivant Social Ventures Announces Leadership Team, Inaugural Partnership, and Publication in Nature Gene Therapy - Yahoo...

CALGARY, Alberta, Aug. 17, 2021 (GLOBE NEWSWIRE) Zenith Capital Corp. (Zenith or the Company), a clinical stage biotechnology company focused on the development of novel epigenetic combination therapies for the treatment of cancers, announces a publication in the high-impact Nature Publishing Group journal of Cancer Gene Therapy, titled: Combination of ZEN-3694 with CDK4/6 inhibitors reverses acquired resistance to CDK4/6 inhibitors in ER-positive breast cancer.

This important study highlights the potential for ZEN-3694 in combination with CDK4/6 inhibitors as a new therapeutic option for patients with advanced ER+ breast cancer who developed resistance to current therapies. The study shows that the combination can reverse acquired CDK4/6i resistance by targeting key oncogenic pathways involved in cell cycle regulation, cellular growth, proliferation, apoptosis, inammation, and cellular immune response. Additionally, ZEN-3694 down-regulated markers involved in CDK4/6 inhibitor resistance including CDK6, CCND1, CDK4, MYC, ESR1 and others. Notably, the combination led to the synergistic increase of apoptosis and inhibition of proliferation in the resistant ER+ breast cancer models.

The publication can be viewed using the following LINK.

This compelling data highlights the role of ZEN-3694 in the potential treatment of patients with advanced ER+ breast cancer, leading to the reversal of the acquired resistance to the current standard of care therapies, said Donald McCaffrey, President and Chief Executive Officer of Zenith. As there is a significant unmet need in this population, this study reveals the huge potential in this indication and rationale for a future clinical trial.

About ER+ Breast Cancer

According to Cancer Statistics, 2021, Estrogen receptor-positive breast cancer (ER+) is a prevalent disease with over two million global new cases diagnosed in 2020. Approximately 7080% of women diagnosed with breast cancer are ER+. The standard of care for metastatic ER+ breast cancer patients include selective estrogen receptor modulators, estrogen receptor degraders, aromatase inhibitors, as well as a combination of endocrine therapy with inhibitors of cyclin-dependent kinases (CDK4/6 inhibitors, CDK4/6i). Three approved CDK4/6 inhibitors (palbociclib, ribociclib, and abemaciclib) have been shown to signicantly improve progression-free survival in combination with endocrine therapy for the treatment of metastatic breast cancer patients. Although significant advances have been made in treating metastatic ER+ breast cancer, resistance to therapies invariably occurs over time, making it essential to develop new therapies that address resistance.

About Zenith

Zenith Capital Corp. is a biotechnology investment company originally spun out of Resverlogix Corp. (TSX: RVX) in 2013. Zenith Epigenetics Ltd., a wholly-owned subsidiary of Zenith Capital Corp., is a clinical stage biotechnology company focused on the discovery and development of novel therapeutics for the treatment of cancer and other disorders with significant unmet medical need. Zenith Epigenetics is developing various novel combinations of BET inhibitors with other targeted agents. The lead compound, ZEN-3694, is in clinical development for:

For further information, please contact:

Investor Relations & Communications

Zenith EpigeneticsPhone: 587-390-7865Email: info@zenithepigenetics.com Website: http://www.zenithepigenetics.com

This news release may contain certain forward-looking information as defined under applicable Canadian securities legislation, that are not based on historical fact, including without limitation statements containing the words believes, anticipates, plans, intends, will, should, expects, continue, estimate, forecasts and other similar expressions. In particular, this news release includes forward looking information related to the potential role of ZEN-3694 in combination with CDK4/6 inhibitors in the treatment of advanced ER+ breast cancer and a future clinical trial. Our actual results, events or developments could be materially different from those expressed or implied by these forward-looking statements. We can give no assurance that any of the events or expectations will occur or be realized. By their nature, forward-looking statements are subject to numerous assumptions and risk factors including those discussed in our most recent MD&A which are incorporated herein by reference and are available through SEDAR at http://www.sedar.com. The forward-looking statements contained in this news release are expressly qualified by this cautionary statement and are made as of the date hereof. Zenith disclaims any intention and has no obligation or responsibility, except as required by law, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

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Zenith Epigenetics Announces a Publication in the Journal of Cancer Gene Therapy with Compelling Data for the Treatment of ER-Positive Breast Cancer...

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TSHA - Market Data & News

Taysha Gene Therapies Inc (NASDAQ: TSHA) shares fell 8.98%, or $1.56 per share, to close Monday at $15.82. After opening the day at $17.66, shares of Taysha Gene Therapies fluctuated between $17.66 and $15.75. 102,676 shares traded hands an increase from their 30 day average of 84,431. Monday's activity brought Taysha Gene Therapiess market cap to $599,854,423.

Taysha Gene Therapies is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, Taysha aims to rapidly translate our treatments from bench to bedside. Taysha Gene Therapies has combined its teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, Taysha Gene Therapies leverages its fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives.

Visit Taysha Gene Therapies Incs profile for more information.

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To get more information on Taysha Gene Therapies Inc and to follow the companys latest updates, you can visit the companys profile page here: Taysha Gene Therapies Incs Profile. For more news on the financial markets be sure to visit Equities News. Also, dont forget to sign-up for the Daily Fix to receive the best stories to your inbox 5 days a week.

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Taysha Gene Therapies (TSHA) falls 8.98% on Strong Volume August 16 - Equities.com

Dublin, Aug. 13, 2021 (GLOBE NEWSWIRE) -- The "Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market by Scale of Operation, Type of Vector, Application Area, Therapeutic Area, and Geographical Regions: Industry Trends and Global Forecasts, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the rapidly growing market of vector and gene therapy manufacturing, focusing on contract manufacturers, as well as companies having in-house manufacturing facilities. The study presents an in-depth analysis of the various firms / organizations that are engaged in this domain, across different regions of the globe.

Over the past few years, a number of advanced therapy medicinal products, including cell and gene therapies, have been developed and approved for the treatment of a variety of disease indications. In fact, as of 2020, close to 15 such therapeutics have received marketing approval across different regions worldwide. Further, over 1,000 clinical trials focused on the evaluation of cell and gene therapies have been registered globally. It is worth noting that the clinical success of these therapies heavily relies on the design and type of gene delivery vector used (in therapy development and / or administration). At present, several innovator companies are actively engaged in developing / producing viral and / or non-viral vectors for gene therapies. In this context, it is worth mentioning that multiple viral and non-viral vector based vaccine candidates are being developed against the novel coronavirus (SARS-CoV-2). As of January 2021, the WHO revealed that more than 55 such vaccines are under evaluation, while two viral vector based vaccines (AZD1222 and Sputnik V), being developed by AstraZeneca / Oxford University and Gamaleya Research Institute / Acellena Contract Drug Research and Development, have been approved. This is indicative of the lucrative opportunities for companies that have the required capabilities to manufacture vectors and gene therapies.

Vaccine production is a challenging process and dealing with vectors (viral and non-viral) further adds to the complexity. Therefore, outsourcing is a common practice among biopharmaceutical companies when it comes to vector development and / or manufacturing. Several players have developed / are developing versatile technology platforms for designing and manufacturing different types of gene delivery vehicles. Innovation in this segment of the pharmaceutical industry is presently focused on the enhancement of transduction efficiency and improving gene delivery efficiencies. In fact, some vector-related technology providers claim that their proprietary solutions have the ability to enable further improvements in existing genetically modified therapeutic products, and / or optimize affiliated manufacturing processes. The viral / non-viral vectors and gene therapy manufacturing market has also witnessed significant partnership activity in the recent past, especially now that COVID-19 vaccine developers are actively approaching such companies for their services. Given the growing demand for interventions that require genetic modification, the vector and gene therapy manufacturing market is poised to witness substantial growth in the foreseen future.

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION3.1. Chapter Overview3.2. Viral and Non-Viral Gene Transfer Techniques3.3. Viral Vectors Used in Genetically Modified Therapies3.4. Types of Viral Vectors3.5. Types of Non-Viral Vectors3.6. Gene Delivery using Non-Viral Vectors3.7. Applications of Viral and Non-Viral Vectors3.8. Current / Ongoing Trends in Vector Development / Manufacturing3.9. Vector Manufacturing3.10. Future Perspectives

4. VIRAL VECTOR AND GENE THERAPY MANUFACTURERS (INDUSTRY PLAYERS): MARKET LANDSCAPE4.1. Chapter Overview4.2. Viral Vector and Gene Therapy Manufacturers: Overall Market Landscape4.2.1. Analysis by Year of Establishment4.2.2. Analysis by Company Size4.2.3. Analysis by Location of Headquarters4.2.4. Analysis by Type of Product Manufactured4.2.5. Analysis by Location of Vector Manufacturing Facilities4.2.6. Analysis by Purpose of Production4.2.7. Analysis by Scale of Production4.2.8. Analysis by Location of Headquarters and Scale of Production4.2.9. Analysis by Type of Vector Manufactured4.2.10. Analysis by Scale of Production and Type of Vector Manufactured4.2.11. Analysis by Application Area4.2.12. Information on Production Capacity

5. PLASMID DNA AND GENE THERAPY MANUFACTURERS (INDUSTRY PLAYERS): MARKET LANDSCAPE5.1. Chapter Overview5.2. Plasmid DNA and Gene Therapy Manufacturers: Overall Market Landscape5.2.1. Analysis by Year of Establishment5.2.2. Analysis by Company Size5.2.3. Analysis by Location of Headquarters5.2.4. Heat Map: Analysis by Company Size and Location of Headquarters5.2.5. Analysis by Type of Product Manufactured5.2.6. Analysis by Location of Plasmid DNA Manufacturing Facilities5.2.7. Analysis by Purpose of Production5.2.8. Analysis by Scale of Production5.2.9. Analysis by Application Area5.2.10. Information on Production Capacity

6. VECTOR AND GENE THERAPY MANUFACTURERS (NON-INDUSTRY PLAYERS): MARKET LANDSCAPE6.1. Chapter Overview6.2. Vector and Gene Therapy Manufacturers: Overall Market Landscape6.2.1. Analysis by Year of Establishment6.2.2. Analysis by Location of Vector Manufacturing Facilities6.2.3. Analysis by Purpose of Production6.2.4. Analysis by Scale of Production6.2.5. Analysis by Type of Vector Manufactured6.2.6. Analysis by Scale of Production and Type of Vector Manufactured6.2.7. Analysis by Application Area

7. VECTOR AND GENE THERAPY MANUFACTURING TECHNOLOGIES: MARKET LANDSCAPE7.1. Chapter Overview7.2. Vector and Gene Therapy Manufacturing Technologies7.2.1. Analysis by Type of Technology7.2.2. Analysis by Purpose of Technology7.2.3. Analysis by Scale of Production7.2.4. Analysis by Type of Vector7.2.5. Analysis by Application Area7.2.6. Most Active Players: Analysis by Type of Technology7.3. Concluding Remarks

8. COMPANY COMPETITIVENESS ANALYSIS8.1. Chapter Overview8.2. Methodology and Key Parameters8.3. Vector and Gene Therapy: In-House Manufacturers8.3.1. Players based in North America8.3.2. Players based in Europe8.3.3. Players based in Asia-Pacific and Rest of the World8.4. Vector and Gene Therapy: Contract Manufacturing Organizations8.4.1. Players based in North America8.4.2. Players based in Europe8.4.3. Players based in Asia-Pacific and Rest of the World8.5.. Vector and Gene Therapy: Players Engaged in In-house and Contract Manufacturing8.5.1. Players based in North America8.5.2. Players based in Europe8.5.3. Players based in Asia-Pacific and Rest of the World

9. VECTOR AND GENE THERAPY MANUFACTURERS IN NORTH AMERICA9.1. Chapter Overview9.2. Advanced BioScience Laboratories9.3. Aldevron9.4. Audentes Therapeutics9.5. BioReliance / SAFC Commercial (Merck KGaA)9.6. bluebird bio9.7. Brammer Bio9.8. Emergent BioSolutions9.9. FUJIFILM Diosynth Biotechnologies9.10 MeiraGTx9.11. Other Companies9.11.1. MassBiologics9.11.2. Spark Therapeutics9.11.3. Vigene Biosciences

10. VECTOR AND GENE THERAPY MANUFACTURERS IN EUROPE10.1. Chapter Overview10.2. Biovian10.3. Centre for Process Innovation10.4. Cobra Biologics10.5. FinVector10.6. Kaneka Eurogentec10.7. Lonza10.8. MolMed10.9. Novasep10.10. Orchard Therapeutics10.11. Oxford BioMedica10.12. Richter-Helm10.13. Sanofi (CEPiA, Sanofi Pasteur, Genzyme)10.14. uniQure10.15. Vibalogics10.16. VIVEbiotech10.17. Other Companies

11. VECTOR AND GENE THERAPY MANUFACTURERS IN ASIA-PACIFIC11.1. Chapter Overview11.2. Wuxi AppTec11.2.1. Company Overview11.2.2. Financial Information11.2.3. Manufacturing Facilities11.2.4. Manufacturing Experience11.2.5. Recent Developments and Future Outlook11.3. Other Key Players

12. RECENT PARTNERSHIPS12.1. Chapter Overview12.2. Partnership Models12.3. Vector and Gene Therapy Manufacturing: Recent Partnerships12.3.1. Analysis by Year of Partnership12.3.2. Analysis by Type of Partnership12.3.3. Analysis by Scale of Production12.3.4. Analysis by Type of Vector12.3.5. Analysis by Therapeutic Area12.3.6. Most Active Players: Analysis by Number of Partnerships12.3.7. Geographical Analysis12.3.7.1. Intercontinental and Intracontinental Agreements12.4. Other Collaborations

13. RECENT EXPANSIONS13.1. Chapter Overview13.2. Expansions Models13.3. Vector and Gene Therapy Manufacturing: Recent Expansions13.3.1. Analysis by Year of Expansion13.3.2. Analysis by Type of Expansion13.3.3. Analysis by Amount Invested by Key Players13.3.4. Analysis by Scale of Production13.3.5. Analysis by Type of Vector13.3.6. Analysis by Application Area13.3.7. Most Active Players: Analysis by Number of Expansions13.3.8. Geographical Analysis13.3.8.1. Analysis by Location of Expansion Project

14. STRATEGIC PARTNER ANALYSIS14.1. Chapter Overview14.2. Strategic Partner Analysis: Viral Vector based Therapy Developers14.3. Methodology and Key Parameters

15. EMERGING VECTORS15.1. Chapter Overview15.1.1. Alphavirus based Vectors15.1.2. Anc80 based Vectors15.1.3. Bifidobacterium longum based Vectors15.1.4. Cytomegalovirus based Vectors15.1.5. Listeria monocytogenes based Vectors15.1.6. Minicircle DNA based Vectors15.1.7. Myxoma Virus based Vectors15.1.8. Self-Complementary Vectors15.1.9. Sendai Virus based Vectors15.1.10. Sleeping Beauty Transposons15.1.11. Vaccinia Virus and Modified Vaccinia Ankara based Vectors

16. KEY INSIGHTS16.1. Chapter Overview16.2. Vector and Gene Therapy Manufacturers: Analysis by Purpose of Manufacturing, Type of Vector Manufactured and Scale of Operation16.3. Vector and Gene Therapy Manufacturers: Analysis by Company Size and Type of Vector Manufactured16.4. Vector and Gene Therapy Manufacturers: Prominent Geographical Hubs by Type of Organization16.4.1. Contract Manufacturing Organizations16.4.2. In-House Manufacturers16.5. Vector and Gene Therapy Manufacturers: Analysis by Location of Manufacturing Facilities and Type of Vector Manufactured16.5.1. AAV Vector Manufacturers16.5.2. Adenoviral Vector Manufacturers16.5.3. Lentiviral Vector Manufacturers16.5.4. Retroviral Vector Manufacturers16.5.5. Plasmid DNA Manufacturers

17. COST PRICE ANALYSIS17.1. Chapter Overview17.2. Factors Contributing to High Price of Viral Vector and Plasmid DNA based Therapies17.3. Viral Vector and Plasmid DNA based Therapies: Pricing Models17.3.1. On the Basis of Expert Opinions17.3.2. On the Basis of Manufacturing Cost17.3.2.1. On the Basis of Technology Used17.3.2.2. On the Basis of Scale of Manufacturing17.3.2.3. On the Basis of Type of Client17.3.3. Prices of Different Types of Vectors17.4. Concluding Remarks

18. CAPACITY ANALYSIS

19. DEMAND ANALYSIS

20. MARKET SIZING AND OPPORTUNITY ANALYSIS

21. IMPACT OF COVID-19 PANDEMIC ON THE VECTOR AND GENE THERAPY MANUFACTURING MARKET

22. KEY DRIVERS AND CHALLENGES

23. SURVEY ANALYSIS

24. CONCLUDING REMARKS

25. EXECUTIVE INSIGHTS

26. APPENDIX I: TABULATED DATA

27. APPENDIX II: LIST OF COMPANIES AND ORGANIZATIONS

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

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Insights on the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Global Market to 2030 - GlobeNewswire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Be Biopharma (Be Bio), whose mission is to pioneer the emerging new class of engineered B cells as medicines, today announced that Krishnan Viswanadhan, Pharm.D., MBA, has been appointed President and Chief Operating Officer and Brad Hartman has joined as Chief People Officer. They join a rapidly growing team of scientists, drug developers, manufacturing experts, and business leaders whose track records have led to the creation and development of some of the industrys most impactful gene and cell therapies.

I am thrilled to welcome both Krishnan and Brad to Be Bio. Krishnan brings an exceptional track record building, rapidly scaling, and effectively leading operations that have delivered multiple highly impactful cell therapy products to patients, said Joanne Smith-Farrell, PhD, Chief Executive Officer at Be Bio. At Be Bio, we believe people are our most important asset, and Brads track record of building cell and gene therapy enterprises with innovative, inclusive cultures will help us push the boundaries of whats possible to create an unparalleled work experience. added Dr. Smith-Farrell. Both of these industry leaders have come to Be Bio to build something truly unique a fantastic team and a bespoke operating environment designed especially to unleash the breadth and depth of our leading engineered B cell medicines platform on behalf of patients who need options.

Prior to joining Be Bio, Krishnan served as Senior Vice President, Global Cell Therapy Franchise Lead at Bristol Myers-Squibb (BMS). In this role, he was responsible for setting the vision and developing the integrated, enterprise-wide cell therapy franchise strategy, building core capabilities, and driving key investments to support long-term growth. He oversaw the program team leaders responsible for the cross-functional development, registration, and lifecycle management of Breyanzi (liso-cel), a CD19 CAR T in large B cell lymphoma, and Abecma (ide-cel), the first BCMA CAR T in relapsed/refractory multiple myeloma. Prior to BMS, Krishnan held senior roles in Business Development & Global Alliances as well as Global Project Leadership at Celgene Corporation. He co-founded Advyzom, a boutique consulting company, and held multiple roles in leading development teams and global regulatory strategy at Hoffman-La Roche. Krishnan is a registered pharmacist and received his Pharm.D. from Rutgers University and holds a MBA from Cornell University. He currently serves as a non-executive director on the board of JW Therapeutics, a leading cell therapy company in China.

What is remarkable about Be Bio is the unprecedented opportunity afforded by this novel B cell therapy platform to tackle a broad set of therapeutic applications that cannot be addressed by other modalities, said Dr. Viswanadhan. The possibilities for transforming patient lives in such a wide range of serious and life-threatening diseases, the passion of the team, and the focus on patients are truly inspiring. I am honored to join this incredible team to build a portfolio of disruptive therapies for patients and to create a truly special place to come to work each day.

Brad joins Be Bio from FerGene where he served as the Chief People Officer leading the rapid expansion of the company in support of a first product commercialization effort for a novel gene therapy. He has spent the past 15 years building small to mid-sized biotech companies from early research stages through commercialization and has played a significant role in launching multiple innovative therapies, including Kalydeco (cystic fibrosis), Kalbitor (hereditary angioedema), and Incivek (hepatitis C), which was one of the fastest blockbuster drug launches of all time. He also founded and built out his own Executive Search firm, ConnectedSearch. Brad received his BS in Neuroscience from the University of Rochester and worked at both the National Cancer Institute and the Graduate School of Pharmacology at the University of Rochester in cellular pharmacology.

There is a deeply held belief at Be Bio in the paramount importance of people, culture, and service to patients, to each other, and to the community, said Mr. Hartman. I am equally awestruck by the enormous potential of this B cell platform to transform patient lives across a wide variety of serious, life-threatening diseases as I am by this teams character, heart, and values. I am incredibly honored to join this distinguished team in building a remarkable place to work together and endeavoring to reshape the lives of so many patients and their families.

About Be Biopharma

Be Biopharma is a leader in developing B cells as medicines, treating disease with the human bodys native protein factories. We precisely engineer B cells to harness their intrinsic drug-like properties remarkable protein production, selective tissue targeting, and fine control of their cellular environment to forge a new category of cell therapy. These medicines are designed to be durable, allogeneic, re-dosable, and administered without toxic conditioning, creating new avenues to halt or reverse severe diseases like cancer, autoimmune conditions, and enzyme deficiency. Founded by Longwood Fund and B cell engineering pioneers David Rawlings, M.D., and Richard James, Ph.D., Be Biopharma is re-imagining medicine based on the power of B cell therapy. Be Bio was founded in October 2020 by Longwood Fund with a $52 million Series A investment led by Atlas Ventures and RA Capital, joined by Alta Partners and Takeda Ventures. For more information, please visit Be Biopharma.

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Be Bio Appoints Leading Cell & Gene Therapy Executives, Krishnan Viswanadhan as President & Chief Operating Officer, and Brad Hartman as Chief...