Category Archives: Gene therapy

Marianne De Backer,Head of Business Development & Licensing in Bayers Pharmaceuticals Division, pictured above. Photo courtesy of Bayer.

In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies.

The launch of the C> Platform is supported by a number of collaborations and acquisitions the Germany-based company made over the past year, including the October acquisition of AskBio's AAV-based gene therapy pipeline, as well as its 2019 deal for BlueRock Therapeutics and that companys induced pluripotent stem cells (iPSC) platform.

Also in December, the company partnered with Atara Biotherapeutics to develop off-the-shelf T-cell immunotherapies for high mesothelin-expressing tumors.While these are three companies considered key to the future of Bayers C> Platform, the company is not done fleshing out this new area. Marianne De Backer, Head of Business Development & Licensing in Bayers Pharmaceuticals Division, said the core pieces of the platform are in place, but noted there are other areas left to explore in the rapidly evolving space.

In an interview with BioSpace, De Backer outlined Bayers thought process for opening its purse strings and diving into the deep end of the cell and gene therapy space.

The roots of Bayers platform began in 2014, when the company stepped into the gene therapy space through a collaboration for hemophilia A with Dimension Therapeutics, which was later acquired by Ultragenyx in 2017. Although that never panned out as hoped, Bayer still saw significant potential in cell and gene therapy. De Backer said last year, the company decided it was time to heavily invest in this area.

We analyzed where we wanted to play to become a leader in the field, De Backer said from Germany. There is an incredible unmet need in some diseases that can only be addressed through cell and gene therapies.

As a result, the company settled on four areas of focus iPSC, allogenic CAR-T, genome mutations and gene editing. In order to make a big impact in these areas, De Backer said it was important to find pioneers in the field and bring them into the fold. The company flexed its M&A muscle and has done precisely that with its three announced partnerships. Each of the companies met strict criteria that includes assets in clinical development, an industry-leading platform and in-house manufacturing capabilities.

We have set the bar high. We wanted to come up with a deal that had pioneers in the field, De Backer said.

She added she was incredibly happy to secure deals with these companies, noting that the core pieces are in place for the C> Platform. However, she said the company wants to strengthen its position in gene editing.

The platform is already bearing fruit. Last week, BlueRock announced the U.S. Food and Drug Administration cleared an Investigational New Drug application for a Phase I study of pluripotent stem cell-derived dopaminergic neurons in advanced Parkinsons disease.

Although Bayer has been and will continue to seek out companies to acquire and forge strategic partnerships with, De Backer said part of the companys strategy is to keep the companies at arms length, rather than fold them into Bayers own operations.

We want them to focus on their science and make sure they keep their entrepreneurial culture, De Backer said, describing the company as a docking station for its subsidiaries and partners.

Collaborative agreements will be a key for the companys strategy in this space. De Backer said the past 10 months of the global pandemic has shown how important a cooperative landscape is to the development of promising new treatments. She touted the speed of vaccine development this past year and said that type of laser-like focus has proven drug development is prime for this kind of disruption.

The silver lining of this pandemic has shown that we can do things differently, De Backer said.

In addition to cell and gene therapy, De Backer said digital technology will also be a key focus for Bayer. Machine learning and artificial intelligence will bolster drug design programs and help spur that kind of innovation in drug discovery De Backer anticipates. An ongoing partnership with Recursion Pharmaceuticals is expected to lead to new development for fibrotic disease treatments.

De Backer said Recursions purpose-built artificial intelligence-guided drug discovery platform is the kind of tool Bayer hopes to harness to benefit patients across multiple indications. Despite all of the cutting-edge technologies now at in its arsenal, De Backer said the patients remain the companys priority. It is the patient who they ultimately serve, she said.

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Bayer Continues Eying Potential Partnerships in Cell and Gene Therapy Space - BioSpace

Special delivery: Stem cells can be modified to produce a therapeutic protein in the brain.

Laguna Design / Science Photo Library

Two unpublished studies detail improved methods for delivering gene therapies to the brain: One involves a type of stem cell that can produce gene-altering proteins on-site; the other taps an engineered virus to target neurons efficiently and noninvasively.

Researchers presented the work virtually on Monday and Tuesday at the 2021 Society for Neuroscience Global Connectome.

One of the biggest hurdles for targeted gene therapy is getting enough treatment to the right spot. In the first study, researchers overcame this obstacle by developing stem cells that produce a therapeutic protein inside the brain.

The team is using the approach to develop a treatment for Angelman syndrome, which is caused by mutation in or deletion of the maternal copy of the gene UBE3A. Because the paternal copy of the gene is typically silent, loss of the maternal copy results in an absence of UBE3A protein. People with Angelman syndrome usually have intellectual disability and motor impairments, and many are autistic.

The researchers had previously used modified stem cells to produce a protein that can activate the paternal copy of UBE3A. Transplanting the cells into the brains of Angelman syndrome model mice boosts levels of UBE3A protein, they found. However, the treatment required multiple direct injections into the animals brains.

In the new work, they instead tried injecting the cells into a pocket of cerebrospinal fluid at the base of the skull an approach that is less invasive and can be performed multiple times. They compared the results with direct injection into the animals hippocampus. In both cases, the mice had UBE3A expression in the brain for up to three weeks.

Mice that received direct injection of the stem cells had fewer Angelman syndrome traits than controls, as measured by their motor skills.

This suggests that though the new route is effective, it may not provide a high enough dosage, says Peter Deng, a postdoctoral researcher inKyle Finkslab at the University of California, Davis, who presented the work. And because the transplanted cells produce protein for only a limited period of time, the effects are temporary a limitation the team is addressing.

Deng and his colleagues also found that monkeys treated with the stem cells had the therapeutic protein throughout their brain and spinal cord three weeks after injection, which suggests the approach has potential for treating people.

The second approach presented at the conference improves the delivery of a more permanent form of gene therapy that uses adeno-associated viruses (AAVs).

Researchers typically inject these viruses directly into the brain, and the viruses usually only affect cells immediately surrounding the injection site.

Youre required to use a ton of the virus to penetrate the whole brain, says Jerzy Szablowski, assistant professor of neuroengineering at Rice University in Houston, Texas, who presented the work.

One potential workaround is to inject the AAV into the blood and use focused ultrasound to temporarily open up the blood-brain barrier, allowing the AAV to cross into the brain. Sometimes with this approach, however, the virus also inserts itself into other organs.

In their new work, the team developed AAVs that more easily cross the blood-brain barrier and more selectively target neurons than previous versions do. As a result, the new AAVs can be given in lower doses, reducing the amount of tissue affected outside the brain, Szablowski says.

To identify the most efficient AAV, Szablowski and his colleagues designed 2,100 new viruses, injected them all into the bloodstream of mice and applied focused ultrasound to the animals skulls. The mice had been engineered so that AAVs that successfully inserted themselves into a neuron got tagged with a marker. The team performed genomic sequencing on the mouse brains a few weeks later and read out the levels of viruses.

Compared with the previously most effective AAV, the top five newly identified AAVs targeted twice as many cells in the brain (including more neurons), and nearly half as many cells outside the brain, the researchers found.

The approach could be used to more efficiently deliver treatments for conditions such as Angelman syndrome or Parkinsons disease, the team says.

Read more reports from the 2021 Society for Neuroscience Global Connectome.

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New gene therapy methods deliver promise - Spectrum

An innovative public-private partnership took a big step in its plan to open a center next year that aims at boosting advances in cell and gene therapy in the region, signing a 10-year lease for a 40,000-square-foot facility in Watertown.

Project participants refer to the facility, which has not been formally named, as the center for advanced biological innovation and manufacturing (CABIM). The goal is to increase availability of materials like genetically altered cells that are essential to advancing discoveries from the lab to clinics for use in treating patients.

There has been great progress in developing pharmaceuticals small-molecule drugs to treat a wide range of diseases, said Harvard Provost Alan Garber, who has led the effort. But many conditions resist treatment with conventional pharmaceuticals. Cell-based therapies offer biological approaches that are complementary to and sometimes far more effective than chemistry-based treatments.

Scientists say that a bottleneck in manufacturing such biological materials is slowing the development of cutting-edge advances in gene therapy, stem cell science, regenerative medicine, CRISPR/Cas9 gene editing, and cancer immunotherapy. An array of treatments based on those and similar technologies such as those involving RNA, peptides, and oligonucleotides are in development, in clinical trials, and in some cases already in the clinic.

This facility will help turn scientific findings into approved therapies by making these resources available to early-stage companies and labs.

Alan Garber, Harvard provost

The center, whose creation was announced in late 2019, is led by institutions from both academia and industry. It will contain both manufacturing and innovation space to boost the supply of materials for late-stage research and early clinical trials and provide space to develop ideas that have left the lab but are not yet ripe for corporate investment. It will also emphasize training in the operation of advanced equipment used in cell manufacturing as a way to increase the pool of workers with such critical skills in the region.

The promise of cell-based therapies has been proven, Garber said, pointing to recent gene-therapy trials to treat sickle cell anemia, which showed significant improvement. He also cited stem-cell-based work to treat diabetes by implanting insulin-producing beta cells, developed in the Harvard lab of Xander University Professor Douglas Melton.

The development of tools like CRISPR and progress in stem-cell science are among the advances that have given us hope that we will soon be able to treat cancer, immunological diseases, neurological conditions, and many other inherited conditions far better, Garber said. This facility will help turn scientific findings into approved therapies by making these resources available to early-stage companies and labs.

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Center for cell and gene therapy to open next year - Harvard Gazette

Cell and gene therapies achieved record growth in 2020, surpassing prior high points in terms of financings and approvals. Janet Lambert, CEO of the Alliance for Regenerative Medicine (ARM), painted a rosy picture despite a few challenges at the 2021 Cell & Gene State of the Industry Briefing during this years Biotech Showcase, held virtually January 11-15.

2020 was a record-shattering year for financing, with $19.9 billion raised in 2020, up from the $9.8 billion raised in 2019 and the $13.3 billion raised in 2018 the previous record, she said.

Follow-on financings, at $6.8 billion, accounted for the largest portion of funding, followed by venture capital at $5.6 billion and IPOs at $3.7 billion. Upfront payments from partnerships totaled $3 billion and private placements totaled $1.2 billion.

The number of large financings exceeding $200 million grew noticeably. Sana Bios $700 million private financing, announced last June, topped the field. Iovance and bluebird bio gained the most from follow-on financing, with fundings of $604 million and $575 million, respectively. Legend Bio led the list of IPOs, raising $487 million last May.

Large pharma continued to buy-in to biotechs for research, development, commercialization, and licensing agreements focuses largely on oncology and CNS disorders, Lambert said. She cited Sangamo, with two major collaborations with Biogen ($350 million) and Novartis ($75 million), though adding, Bayer is especially active.

In the stock market, Regenerative medicine companies outperformed the NASDAQ Biotech Index, she continued. Although stock prices for virtually everything plummeted in mid-March, biotech stocks rebounded. Cell based immuno-oncology (IO) share prices increased 80% from their January 1 levels. Gene therapy was close behind, with a 70% increase, and all publicly traded regenerative medicine companies saw a 50% increase.

Given the overall environment, it seems safe to predict that 2021 will be another good year for regenerative therapies.

Currently, there are approximately 1,100 gene, cell and tissue-based therapeutic developers throughout the world. Of those, the overwhelming majority are in North America, with 543. We saw a lot of growth in China, Japan, and Korea, she said, with 295 companies. Europe boasts 209.

Globally, 1,220 clinical trials are underway for regenerative medicine.

More than 100 clinical trials commended in the fourth quarter alone, Lambert said. Roughly, they are divided evenly among cell, gene, and IO. With 152 trials in phase III and nearly half of those in gene therapy, this offers strong support for predictions by both the FDA and EMA that there will be 10 to 20 advanced therapies approved each year through 2025.

What people often forget, Lambert said, quoting Amy Price, a mother of two gene therapy recipients, is that cell and gene therapies arent some fantastical futuristic thing. Two of the Price children received gene therapy in a clinical trial 10 years ago for metachromatic leukodystrophy (MLD), a historically fatal disease. That drug, Libmeldy, by Orchard Therapeutics, was approved in Europe in 2020, making it one of the most significant milestones of the year.

The benefits of cell and gene therapy have expanded beyond experimental treatments.

Patients are continuing to benefit from innovative therapies, Lambert said. We saw a significant number of gene therapy approvals in 2020. In addition to Libmeldy, she cited approvals of Zolgensma (by Novartis Gene Therapies) in Europe, Japan, and Canada; Tecartus (by Kite, a Gilead company) in the U.S.; and Luxturna (by Spark/Roche) in Canada.

Looking forward, oncology, and particularly IO, dominates the regenerative medicine landscape. Some 554 oncology trials are underway.

Investors have invested heavily in this space for some time, and IO comprises 50% of Phase I trials in cell and gene therapy, Lambert noted. Focus is increasing on allogeneic therapies as well as gene editing.

Central nervous system therapeutics are the second most popular therapeutic indication for regenerative therapies for the second year in a row, with 94 trials. 2020 saw promising data from the first-ever attempt iPSC therapy for Parkinsons disease.

Gene editing continues to advance in the clinic, she added. For the first time, a patient was treated with CRISPR therapy in vivo and, later, systemically with CRISPR. CRISPR Therapeutics and Vertex Pharmaceuticals shared data (during J.P. Morgan week) from a sickle cell trial of 20 patients.

Despite these scientific advances and investor enthusiasm, cell and gene therapies face challenges in terms of dosing and delivery, and chemistry, manufacturing and controls (CMC), Lambert admitted. Gene therapy represents almost half of the Phase III pipeline, so we expect to learn a lot quite soon.

While it goes without saying that 2020 was a challenging year, many of the repercussions of the COVID-19-related disruptions remain to be seen. They extend not only to operational and clinical disruptions but also to regulatory backlogs and the politicization of diagnostics and therapeutics, all set against the usual challenges of fast-moving science.

None-the-less, Lambert pointed out, There were some positive developments.

As she said, Medicare approved a new diagnosis related group (DRG) for CAR T cell therapy and promulgated a new rule for outcomes-based therapies, thus enabling a new payment model that ARM deems essential for cell and gene therapies. In Europe, the European Commissions pharmaceutical strategy now recognizes the importance of cell and gene therapy. We are encouraged that we can build on that starting point with the Commission, Lambert said. ARM also is expecting progress on n-of-one therapies for ultra-orphan indications this year.

Looking ahead to 2021, Lambert identified six regenerative therapies on the FDAs docket from Mallinckrodt, bluebird, BMS, PTC Bio, and Gensight Bio. All indications are that 2021 will be a fantastic year of scientific, technological, and clinical progress in this sector, Lambert predicted.

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Cell and Gene Therapies Shatter Prior Records, with Continued Growth Expected - BioSpace

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Shares of the gene-therapy biotech bluebird bio have tumbled in recent months, with the stock dropping 46.4% over the past year as the S&P 500 Health Care sector index has climbed 14.2%.

Now, the company is trying something entirely different in an effort to stop the downward spiral. In a move announced Monday morning, bluebird (ticker: BLUE) said it planned to split into two public companies by the end of the year. One will focus on rare diseases and the other will work on treating cancer.

After careful strategic review, it is clear to us that the two businesses are best served by independent leadership and teams to drive distinct strategic and operational objectives, said the companys CEO, Nick Leschly, in a statement.

Bluebird shares were up 5.1% in premarket trading.

Bluebirds gene therapy Zynteglo, which treats the blood disorder beta-Thalassemia, has been approved in Europe, though treatments have not yet begun due to the Covid-19 pandemic, according to the Wall Street Journal. The gene therapy, one of the first in the world to achieve regulatory approval, does not yet have approval from the U.S. Food and Drug Administration.

The news came in the course of an unsettling few days for the gene-therapy field.

Shares of Sarepta Therapeutics (SRPT) fell 51.3% on Friday on news that its gene therapy for Duchenne muscular dystrophy had failed in a trial to improve muscle strength in patients. And data from BioMarin Pharmaceutical (BMRN) on Sunday night about its Phase 3 trial of its gene therapy valoctocogene roxaparvovec in patients with severe Hemophilia A, while positive, failed to rouse enthusiasm among investors. Shares were down 0.1% in early trading on Monday.

The news is likely raise to questions about whether investors are running out of patience for gene therapy, after decades of investment and only a couple of marketed products to show for it.

In its statement, bluebird said that the new severe genetic disease company would focus on therapies for the blood diseases beta thalassemia and sickle cell disease, and a rare disease called cerebral adrenoleukodysrophy. It said the company would work on expanding access and reimbursement for its gene therapy Zynteglo in Europe.

The new cancer company, it said, would focus on commercialization of its CAR T cell therapy ide-cel, on which it is collaborating with Bristol Myers Squibb (BMY). The product is under FDA review. It said its pipeline would focus on non-Hodgkin lymphoma, acute myeloid leukemia, and other cancers.

Despite its stumble in recent months, the stock remains popular on the Street: Of the 24 analysts who cover bluebird tracked by FactSet, 16 rate it Buy or Overweight, and eight rate it a Hold.

The company scheduled a conference call to discuss the announcement for 8 a.m. Eastern.

Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com

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Gene-Therapy Firm Bluebird Bio Is Spinning Off Its Cancer Programs - Barron's

BETHESDA, Md., Jan. 14, 2021 /PRNewswire/ --Precision Medicine Group ("PMG") announced today it has acquired Project Farma, a patient-focused bioengineering services firm that supports life science innovators in the manufacturing and scale-up of advanced therapies. Project Farma provides Precision a market-leading expertise in the manufacturing of life-changing medicines, with a unique focus in the rapidly growing area of cell and gene therapy.The acquisition adds Project Farma's unique expertise to Precision's suite of end-to-end cell and gene therapy capabilities to support development and commercialization.

Led by executive leaders Anshul Mangal, John Khoury, and Tony Khoury, Project Farma has distinguished itself as a global leader in planning and implementing complex biomanufacturing strategies and facilities for clients that range from start-up biotechnology companies to global biopharmaceutical companies and academic medical centers. Project Farma has led more than a dozen manufacturing facility builds with capital investments greater than $1 billion (including some of the leading FDA-approved cell and gene therapy medicines).

Mark Clein, CEO of Precision Medicine Group, commented on the acquisition, "Since 2013, Precision has supported over 70% of the FDA- approved cell and gene therapies and is now even better positioned to support the hundreds of therapeutic new entrants coming to market. With our acquisition of Project Farma, Precision is the only life-science services company with true end-to-end capabilities in cell and gene therapy."

Project Farma will be part of Precision for Medicine, PMG's research and development services arm. Commenting on the acquisition, Precision for Medicine's president Chad Clark explains, "In today's highly competitive and scrutinized advanced therapies marketplace, ensuring a safe, scalable, and high-quality manufacturing capability can be the difference between success and failure. Project Farma are the experts at defining and developing the optimal manufacturing strategy and leading implementation of a customized solution. This essential capability is now expanded and leveraged by the comprehensive cell and gene therapy services available through Precision ADVANCE."

Anshul Mangal shared, "We are excited to join Precision's cell and gene therapy collective, leveraging interconnected services to accelerate the development, manufacturing, and commercialization of advanced therapies." Added Tony Khoury, "We realize Precision ADVANCE presents an unbelievable opportunity to further next generation medicines and to further Project Farma's and Precision's shared mission to positively impact as many patients and families as possible."

To find out more about Project Farma's innovative biomanufacturing and validation services, please visit the website at projectfarma.com.

To find out more about Project Farma and the end-to-end capabilities of Precision ADVANCE, the cell and gene therapy collective, please visit: precisionmedicinegrp.com/advance.

About Precision Medicine GroupFormed in 2012, Precision Medicine Group is a specialized services company supporting next generation approaches to drug development and commercialization. Precision provides an integrated infrastructure that supports pharmaceutical and life science companies as they develop new products in the age of precision medicine. The company is headquartered in Bethesda, Maryland with offices throughout North America and Europe. For more information, visitprecisionmedicinegrp.com.

About Precision for MedicinePrecision for Medicine is the first biomarker-driven clinical research services organization supportinglife sciences companies in the use of biomarkers essential to targeting patient treatments more precisely and effectively. Precision applies novel biomarker approaches to clinical research that integrate clinical trial execution with deep scientific knowledge, laboratory expertise and advanced data sciences. This convergence of trials, labs and data sciences is driving faster clinical development and approval. Precision for Medicine is part of Precision Medicine Group, with over2,100 people in 35 locations in the U.S.,Canada, andEurope. For more information, visitPrecisionForMedicine.com.

About Project FarmaProject Farma is a consulting firm providing biomanufacturing strategy and execution. Project Farma's services include providing technical operations strategy such as pipeline evaluation and mobilization, make vs buy, and GMP infrastructure and startup. They also include strategy execution by managing capital projects, CDMO selection and management, tech transfers and facility builds and providing engineering services in automation, validation, quality, reliability and maintenance. Project Farma clients include startups, established life science corporations, advanced therapy organizations, universities, hospitals, government agencies, financial institutions, not-for-profits, and CROs/CMOs. For more information, visit projectfarma.com.

About Precision ADVANCE, the cell & gene therapy collectivePrecision ADVANCE is a focused collection of interconnected services and teams uniquely positioned to address the complexities of clinical, regulatory, manufacturing, and commercial needs to successfully bring a cell or gene therapy to market. For more information, visit precisionmedicinegrp.com/advance.

Media Contact: Louis Landon, Precision Medicine Group Media Relations310-984-7707 [emailprotected]

SOURCE Precision Medicine Group

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Precision Medicine Group Significantly Expands Cell And Gene Therapy Expertise With Acquisition Of Project Farma - PRNewswire

R.A. Session II, CEO and co-founder of Taysha Gene Therapies [Background image: Olena Yepifanova via iStock]

Dallas-based biotech startup Taysha Gene Therapies and UT Southwestern Medical Center have launched a new innovation fund dedicated to discovering and developing novel gene therapy candidates and improving next-gen technologies that could be used to treat monogenic diseases of the central nervous system.

The fund will exclusively support the discovery efforts of a team of researchers from the UTSW Gene Therapy Program, the company told us via email. The amount in the fundmanaged by reps from both Taysha and UTSWis undisclosed.

Fitting with the new fund, Taysha is a patient-centric gene therapy company that focuses on the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations. The team ultimately aims to end severe, life-threatening CNS diseases that are caused by variation in a single gene, also known as monogenic. Their solution is to develop and commercialize a pipeline of extensive, AAV (adeno-associated viruses) gene therapies.

Taysha initially emerged from stealth last April with a $30 million Series A funding round and a strategic partnership with UT Southwestern. The company launched with a pipeline of 15 adeno-associated virus gene therapy programs, along with options to four others.

Now, Taysha expects to file four IND (Investigational New Drug) applications in 2021. A big part of achieving that feat comes from the partnership with UTSW.

The majority of Tayshas product candidates originated from UTSW, but all are being developed to treat monogenic CNS diseases. For instance: TSHA-101 is being developed for the treatment of GM2 gangliosidosis, where there are no approved treatments.

Tayshas team, which has proven experience in gene therapy drug development and commercialization, works with the UT Southwestern Gene Therapy Program, led by Steven Gray, director of the Viral Vector Core and Assistant Professor in the Department of Pediatrics, and Berge Minassian, division chief of Child Neurology.

The UTSW Gene Therapy Program supports Tayshas wide range of preclinical and clinical development programs.

Under the partnership, UT Southwestern, which has developed a state-of-the-art viral vector manufacturing facility, runs discovery and preclinical research, as well as lead studies, provide manufacturing, and execute natural history studies.

Together, the two leverage Tayshas fully integrated platform, which it calls an engine for potential new cures, to meet the goal of dramatically improving patients lives.

Our relationship with the UTSW gene therapy program has produced over 18 novel product candidates, including TSHA-101 in GM2 gangliosidosis and TSHA-118 in CLN1, which are currently in clinical development, RA Session II, the president, founder and CEO of Taysha, said in a statement. We are pleased by the significant progress our partnership has achieved and are excited to build on that foundation and momentum to bring additional compelling innovation to the clinic.

The new innovation fund is an expansion of that partnership.

UTSWs discovery efforts to facilitate the translation of promising discoveries from bench to clinic will be supported, per a news release, and Taysha will get an exclusive option on new programs and intellectual property that is associated with, or arising from, the research conducted under the agreement.

Researchers from UTSWs Gene Therapy Program will explore gene therapy targets in new disease areas, and will work to create gene therapy tech platforms that address current limitations.

We are excited to expand our alliance with UTSW to accelerate the discovery and development of novel gene therapy candidates and next-generation technologies for patients with monogenic CNS diseases, Session said. We believe that the combination of UTSWs translational research expertise in gene therapy and strong track record of innovation and our experience in drug development and GMP manufacturing will create opportunities to reach more patients with unmet medical needs.

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Taysha Gene Therapies, which was been operating in stealth with UT Southwestern until this April, plans to use the financing to advance its initial cohort of lead programs into its clinic. By the end of 2021, Taysha expects to file four Investigational New Drug applications.

Taysha Gene Therapies, which has been operating in stealth with UT Southwestern, is off to a fast start with a pipeline of 15 gene therapy programs. Together with UTSW, the combined platform could be"an engine for new cures."

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Rising to the top of the list is 7-Eleven's fifth Evolution Store in Lake Highlands. The location will serve as an experiential testing ground for patrons to try new shopping technology and products.

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Taysha and UTSW Launch Innovation Fund to Advance the Development of New Gene Therapies - dallasinnovates.com

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BioMarin Pharmaceutical has been running the worlds largest clinical trial of a gene therapy in hopes of sparing hemophilia patients from frequent bleeding and chronic drug infusions. But the company suffered a rebuke last August, when U.S. regulators demanded a lengthier study before approval would be considered.

On Sunday, BioMarin (ticker: BMRN) released data on the first year of the continuing study of its Roctavian therapy. The data looked good. In 112 participants, the product was safe and a single treatment with the gene therapy reduced annual bleeding rates by 84%. That is better than the performance of todays standard therapy, which periodically infuses a patient with artificial versions of the clotting factor that their defective genes cant make. Like other gene-augmentation therapies, Roctavian adds working versions of the gene to a patients cells, to supply the correct instructions for production of the clotting protein.

But Sundays data from BioMarin didnt clear an issue that has worried the U.S. Food and Drug Administration and that led the agency to rebuff last years approval request and demand a longer follow-up. The levels of clotting factor produced in Roctavian recipients decline over time. While remaining above levels needed to prevent bleeding, the worry is that a continued decline might end the treatments usefulness after a number of years. Gene therapies were originally hailed as potential cures.

In early trading Monday, BioMarin stock was off 8%, to $83, while the Nasdaq Composite index was off 1%.

The new data will continue to fuel the debate, concluded RBC Capital Managements Kennen MacKay in a Monday note. He called the new information positive, though likely controversial.

MacKay observed that the data in BioMarins press release showed wide variability in how patients responded to the gene therapy. The treated patients median blood levels of clotting factor tapered from 39 units-per-deciliter at six months after treatment, to 24 units at one year. A subset of 17 patients treated earlier showed continued declines to 16 units at 18 months, then 15 units at two years.

BioMarin has said that it plans to talk with the FDA in hopes of persuading the agency to consider approval based on one-year datainstead of requiring two years of follow-up on trial participants. European regulators have shown a willingness to consider approval based on one year of data.

Eun Yang, at Jefferies, described the new data as positive, but remained doubtful that the FDA would relax its demand for longer follow-up of the gene therapy patients.

A majority of analysts surveyed by FactSet, including Jefferies Yang, rate BioMarin a Buy with price targets for the stock that average $111. Even after recent setbacks, the stock isnt cheap by standards that prevail outside of the biotech bubble. The consensus forecast at Sentieo.com sees BioMarin breaking even in 2021, then earning about $1.35 in 2022, $3.00 in 2023, and $5.00 in 2024.

RBCs Mackay has a Hold and a $90 price target. He worries that delays in launching the companys gene therapies will leave revenue gaps as BioMarins existing drugs lose their exclusivity.

We anticipate continued debate, despite this clinical de-risking, MacKay says, of the latest gene therapy data.

Write to Bill Alpert at william.alpert@barrons.com

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BioMarins One-Year Data on Its Hemophilia Gene-Therapy Looked Good. Can It Win FDA Approval? - Barron's

SAN RAFAEL, Calif., Jan. 10, 2021 /PRNewswire/ --BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants. All participants in the study received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up.

Data from the GENEr8-1 Phase 3 study with a mean follow-up of 71.6 weeks showed that in the pre-specified primary analysis for Annualized Bleeding Rate (ABR) a single dose of valoctocogene roxaparvovec significantly reduced ABR by 84% from a prospectively collected 4.8 (median 2.8) at baseline to 0.8 (median 0.0) bleeding episodes per year (p-value <0.0001), among a pre-specified group of prior participants in a non-interventional baseline observational study (rollover population; N=112). 80% of participants were bleed-free starting at week five after treatment.

Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII in the rollover population by 99% from 135.9 (median 128.6) to 2.0 (median 0.0) infusions per year (p-value <0.0001).

Table 1: Mean/Median Annualized Bleeding Rate (ABR) and FVIII Infusion Rate in Phase 3 GENEr8-1 Study Rollover Population (N=112) from Week 5 Through Week 52 at Nov. 2020 Cut Off

Phase 3

Rollover Population*

On Factor VIII prophylaxis, before valoctocogene roxaparvovec infusion

N=112

Phase 3

Rollover Population*

After valoctocogene roxaparvovec infusion

N=112

Mean (SD)

Median (IQR)

Mean (SD)

Median (IQR)

Annualized

Bleeding Rate

(bleeding

episodes per

4.8 (6.5)

2.8 (0.0, 7.6)

0.8 (3.0)

0.0 (0.0, 0.0)

Annualized

FVIII Infusion

135.9 (52.0)

128.6 (104.1, 159.9)

2.0 (6.4)

0.0 (0.0, 0.9)

*See study descriptions for patient population information.

At the end of the first year post-infusion with valoctocogene roxaparvovec, participants in the modified intent-to-treat (mITT) population (N=132) had a mean endogenous Factor VIII expression level of 42.9 (SD 45.5, median 23.9) IU/dL, as measured by the chromogenic substrate (CS) assay, supporting the marked clinical benefits observed with abrogation of bleeding episodes and Factor VIII infusion rate. Factor VIII expression declined at a slower rate compared to the Phase 1/2 study, and remained in a range to provide hemostatic efficacy. In a subset of the mITT population that had been dosed at least two years prior to the data cut date (N=17), Factor VIII expression declined from a mean of 42.2(SD 50.9, median 23.9) IU/dL at the end of year one to a mean of 24.4 (SD 29.2, median 14.7) IU/dL at the end of year two with continued hemostatic efficacy demonstrated by a mean ABR of 0.9 (median 0.0) bleeding episodes per year.

Table 2: Factor VIII Activity Levels in 6-Month Intervals

Median Factor

VIII Activity,

IU/dL

Phase 3 Rollover

Population

(N=112)

Mean (SD)

Median

Phase 3 mITT

Subset

Population

(N=17*)

Mean (SD)

Median

Phase 1/2

6e13 vg/kg

Cohort

(N=7)

Mean (SD)

Median

Phase 1/2

4e13 vg/kg

Cohort

(N=6)

Mean (SD)

Median

Week 26

55.1 (57.4)

38.6

43.9 (42.1)

33.8

71.0 (41.6)

61.2

18.0 (8.7)

18.0

Week 52

43.6 (45.3)

24.2

42.2 (50.9)

23.9

63.6 (36.5)

60.3

21.1 (12.3)

23.8

Week 76

27.9 (30.6)

15.8

53.9 (31.2)

50.2

20.6 (15.4)

21.3

Week 104

24.4 (29.2)

14.7

36.4 (26.3)

26.2

12.3 (8.2)

Excerpt from:
BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy...

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the Companys plans for the buildout of its new Research and Development (R&D) and Chemistry, Manufacturing and Controls (CMC) operation which will also serve as the Companys new headquarters in Cranbury, New Jersey. This new 103,720 ft2 facility will support clinical development of Rockets growing pipeline of lentivirus (LV) and adeno-associated virus (AAV) gene therapies from discovery through pivotal trials, with space for potential future expansion and commercialization. This buildout comes on the heels of the Companys recent successful capital raise of approximately $300 million that provides a cash runway into the second half of 2023.

Investing in R&D and manufacturing innovation, talent, and capacity through this new world-class facility will allow us to deliver on our mission of bringing five curative gene therapies to rare disease patients by 2025, said Gaurav Shah, M.D., President and Chief Executive Officer of Rocket. With data on five clinical programs expected this year, including two that are in registration-enabling trials, these new capabilities will enable us to work with continued urgency and purpose towards bringing transformational therapies to patients.

We are collaborating with some of the best scientists and innovators worldwide. This new facility is instrumental in bolstering Rockets gene therapy capabilities to rapidly advance multiple platforms and programs efficiently and effectively. Producing clinical drug product will enable greater control of supply, cost, quality, and timing to pave a smoother path toward commercialization, added Kinnari Patel, Pharm.D., MBA, Chief Operating Officer and Head of Development.

Approximately one-half of the facility is being scaled for AAV Current Good Manufacturing Practice (cGMP) production. The other half features state-of-the-art R&D labs to support the expanding pipeline and Quality Control (QC) laboratories to support CMC development for process and analytics.

Rocket recently reported positive interim Phase 1 results for its first AAV-based gene therapy, RP-A501 for the treatment of Danon Disease. The first cGMP production at this facility will be initiated in 2021 and will be used in a planned Phase 2 registrational study evaluating RP-A501, following the completion of the current Phase 1 trial.

Approximately $300 million secured in public equity offering

On December 14, 2020, Rocket closed an upsized underwritten public offering of 6,035,714 shares of its common stock, inclusive of greenshoe, at the public offering price of $56.00 per share. The offering was ~7.3x oversubscribed based on the initial deal size of $175 million. Rocket intends to use the net proceeds from this offering to further fund the development of its pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the U.S. and Europe, accelerate the buildout of in-house manufacturing capabilities, and for general corporate purposes. This capital raise extends Rockets cash runway to the second half of 2023.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia, and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the preclinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, filed November 8, 2019 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Buildout of R&D and Manufacturing Facility to Support Development of Innovative Gene Therapy Pipeline - Business Wire