Category Archives: Gene therapy

THOUSAND OAKS, Calif., Dec. 7, 2021 /PRNewswire/ --Capsida Biotherapeutics Inc., an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders, today announced the appointment of Peter Anastasiou as the company's Chief Executive Officer (CEO) and the promotions of Capsida co-founders Nicholas Flytzanis, Ph.D., to Chief Scientific Officer (CSO) and Nick Goeden, Ph.D., to Chief Technology Officer (CTO).

Gene therapy is still in its infancy and has yet to achieve its full potential. First-generation gene therapies have been challenged by safety issues due to their inability to target cells and organs without also penetrating non-targeted cells and organs, especially the liver. Capsida's proprietary, targeted, non-invasive gene therapy technology allows more selective targeting of specific tissues and cells, overcoming many of the problems associated with first-generation gene therapies, specifically off-target cell and organ activity. In addition, it allows the gene therapy to be delivered non-invasively through intravenous (IV) administration. The company's already strong leadership team is poised to actualize the promise of gene therapy with the addition of Mr. Anastasiou and the promotions of Drs. Flytzanis and Goeden.

"I can't imagine a more exciting time to join this organization," said Mr. Anastasiou. "Capsida is enabling gene therapy to become what the industry, physicians, and patients have been dreaming it will be. Our patent-protected technology allows the targeting of cells and organs while limiting the negative impact on non-targeted areas, and can be applied across multiple therapeutic areas. Another important benefit of our technology is that we are able to deliver the gene therapy non-invasively through IV administration. I'm honored to lead this talented team to achieve Capsida's potential and to improve and even save patients' lives."

Mr. Anastasiou joins Capsida from Lundbeck, where he was an executive vice president and a member of the executive committee, reporting to the CEO. As the president of Lundbeck's U.S. and Canadian business operations, Mr. Anastasiou has built organizations from the ground up. He brings significant leadership experience managing diverse organizations and bringing them together to achieve common goals. He led as many as 1,200 employees and achieved net revenues of $1.5 billion. During his 12-year tenure at Lundbeck, Mr. Anastasiou held several progressive leadership positions, playing a pivotal role in developing and launching multiple products and building the company's cross-functional capabilities. Mr. Anastasiou serves on the Board of PhRMA and the global advisory board for the Healthcare Businesswomen's Association. Mr. Anastasiou begins his new role with Capsida on January 3, 2022.

"We're thrilled to welcome Peter as Capsida's new CEO," said Beth Seidenberg, M.D., founding managing director at Westlake Village BioPartners, one of the company's lead investors, and Capsida board member. "Peter has deep industry expertise, a broad network, and significant public company experience, which will be valuable as Capsida grows. In addition, his strong track record of success demonstrates he is a visionary leader who will be able to deliver on the promise of targeted non-invasive gene therapy to help underserved patients and achieve business success."

"During his tenure at Lundbeck, Peter has created significant shareholder value, creating and leading organizations and successful blockbuster product launches," said Clare Ozawa, Ph.D., managing director at Versant Ventures, one of Capsida's lead investors, and Capsida board member."Under Peter's leadership, we will continue to build Capsida as the industry's leading targeted, non-invasive gene therapy company with the ability to transform the lives of patients with life-threatening genetic disorders."

Prior to Lundbeck, Mr. Anastasiou held management roles at Neuronetics, Inc., Bristol-Meyers Squibb Company, and Eli Lilly and Company. He holds an MBA from the Kelley School of Business at Indiana University, and a B.A. in economics and management from Albion College.

Capsida co-founders Nicholas Flytzanis, Ph.D., promoted to CSO and Nick Goeden, Ph.D., promoted to CTO

In addition to Mr. Anastasiou's appointment, Capsida announced that Dr. Flytzanis has been promoted toCSO and Dr. Goeden has been promoted to CTO.

"The promotions of Drs. Flytzanis and Goeden are in recognition of the significant contributions they have made since co-foundingCapsida in 2019," said Mr. Anastasiou. "Their steadfast commitment to delivering on the promise of Capsida's differentiated, non-invasive gene therapy platform has been a key driver behind many of the company's early achievements."

"Drs. Flytzanis' and Goeden's strong scientific and technical expertise and know-how have already delivered results in the startup of Capsida based on Caltech'sbasic research on targeted non-invasive gene delivery to the brain," said Capsida co-founder Viviana Gradinaru, Ph.D. "Their promotions are timely as Capsida enters the phase of delivering from the lab and for the patients."

Prior to co-founding Capsida, Dr. Flytzanis served as scientific director of the CLOVER research center at the California Institute of Technology (Caltech), leading an interdisciplinary team to develop and disseminate emerging technologies focused on the cross-section of neurological research and gene therapy. His research spans the fields of tissue clearing and imaging, optogenetics and rodent behavior, and adeno-associated virus (AAV) engineering and gene therapy, with collaborations across multiple institutions. During his Ph.D., Dr. Flytzanis applied protein engineering and directed evolution across biological modalities, with a focus on developing AAVs as therapeutic tools for neurological disease.

Dr. Flytzanis holds a Ph.D. in biology from Caltech and a B.S. in biology from the Massachusetts Institute of Technology.

Prior to co-founding Capsida, Dr. Goeden led a team developing the novel adeno-associated virus (AAV) engineering technology underlying Capsida's biologically driven gene therapy platform. During his tenure as a postdoctoral fellow in Dr. Gradinaru's lab at Caltech, he developed high-throughput methods for screening combinatorial libraries to explore the AAV fitness landscape and engineered novel AAVs with high efficiency and specificity for the rodent and primate brain. During his Ph.D., Dr. Goeden developed a novel organ bioreactor to study real-time metabolomics in diseased states, exploring the relationship between gene expression and the pathophysiology of neurodevelopmental disorders.

Dr. Goeden holds a Ph.D. in neuroscience from The University of Southern California and a B.S. in biology from Caltech.

About Capsida Biotherapeutics

Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of life-threatening genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology. Visit us at http://www.capsida.com to learn more.

View original content to download multimedia:https://www.prnewswire.com/news-releases/capsida-biotherapeutics-poised-to-capitalize-on-industry-leading-gene-therapy-technology-with-new-ceo-cso-and-cto-301438459.html

SOURCE Capsida Biotherapeutics

Link:
Capsida Biotherapeutics Poised to Capitalize on Industry-leading Gene Therapy Technology With New CEO, CSO, and CTO - BioSpace

The CGT Catapult was established to advance the growth of cell and gene therapies in the UK by bridging the gap between scientific research and full-scale commercialisation. As it prepares to open a new facility in the Edinburgh BioQuarter next summer, we put questions to its chief clinical officer Dr Jacqueline Barry.

How effective a bridge between academia and industry has CGT Catapult been?

When we were set up in 2012, there wasnt really a strong cell and gene therapy (CGT) industry. The UK is now the largest cluster for cell and gene therapies outside the United States. About 30 per cent of all CGT companies in Europe are in the UK, and the UK has representation in 12 per cent of global clinical trials. So were now becoming quite a mature industry, and the UK is known and respected globally for advanced therapies.

Our role is to create powerful collaborations which overcome challenges to the advancement of the sector. Id say weve done pretty well in bridging the gap between industry and academia, including creating new collaborations, supporting the creation of spin-out from universities and facilitating progress of companies towards commercialisation.

We continue to focus on this as a core activity for Cell and Gene Therapy Catapult.

What areas of academic need was CGT Catapult able to address?

It depends who the academics are. Some have already spun-out successful companies like Autolus Therapeutics, which announced a $250 million investment by Blackstone this month.

Another is Resolution Therapeutics, founded from a collaboration between Edinburghs Centre for Regenerative Medicine, the Scottish National Blood Transfusion Service, and Syncona Investment Management.

The company is based in the Centre for Regenerative Medicine on the Edinburgh Royal Infirmary campus. A further example would be Purespring Therapeutics, a spin-out from the University of Bristol, which secured one of the largest single investments to date for a new UK university biotech company.

While others are still relatively early in their product development path, we can use our facilities and expertise to accelerate them through the translation pathway.

We provide support through collaborative grants, for example, support of the design of a non-clinical testing programme, and provide commercialisation of research support for really promising technology or therapies to help them secure investment for their research.

How was CGT Catapult able to help industry to bring therapies closer to the market?

We try to anticipate barriers and then act to break them down. For example, a number of years ago we identified there wasnt enough cleanroom manufacturing space for late-stage clinical trials and early market release. In response to this barrier, we established our Manufacturing Innovation Centre in Stevenage, 30 miles north of London. This is a unique collaborative model, where we provide the support in the form of facility licensure, quality and warehouse management systems, environmental monitoring etc, while our collaborators can develop their processes and expertise within their own manufacturing module using their staff and processes.

Our collaborators having such space to build expertise and in-house knowledge is really valuable for them, and it cements their ability to manufacture and supply here in the UK.

In addition, we help with projects 100+ a year of different sizes and complexity, providing technology and process innovation solutions, or helping groups navigate the regulatory and reimbursement challenges and barriers.

How has CGT Catapult helped to foster a culture of innovation?

Innovation can mean so many different things. Technology and process innovation is important, and we help groups with process and analytical solutions. For example, weve taken processes with say 1,000 manual steps and automated the manufacture, increasing the security of the product.

Another could be in the clinical space. The Industrial Strategy Challenge Fund made funds available for the Advanced Therapy Treatment Centre network. This is truly innovative. Were working with 65 industry partners alongside the NHS to come up with solutions for these innovative but disruptive products for patients. Working hand-in-hand with industry and the NHS, we are innovating together, producing practical solutions for both parties.

What are the challenges for the NHS with these kinds of products?

These are living therapies, its disruptive and difficult to deliver these products. In addition, there is an avalanche of products coming with different product types for different indications and different patient groups.

Specialists might not be familiar with these new products. There are often complex referral pathways, so theyre only delivered from particular hospitals. There are specific regulatory and reimbursement conditions placed on the manufacturers. All these things together add complexity and require innovative solutions to not increase the burden for the NHS.

The CGT Catapult aims to help cell and gene therapies to be safer, more effective, scalable and affordable. How do you maintain research integrity and best practice in the face of that constant demand to do things better, faster, cheaper?

Were all scientists and we know this is a young field which has great promise. I think its just in everybodys DNA to ensure that your data integrity is as solid as it possibly can be.

These are quite unusual products which are designed to treat patients who are either at the end of their treatment regimen for example, treatment of a blood cancer or for the treatment of rare genetic disorders, and you want to catch their symptoms before they start impacting on their day-to-day life. So you have to act quickly, but be really confident that your data supports the use of these products.

Why is CGT Catapult coming to Edinburgh [in summer 2022]? Whats it adding?

A lot of cell and gene therapy work is currently focused in southern England, where we have also seen the third-largest cell and gene therapy cluster developing around Stevenage. There are, however, opportunities for growth and further cluster development across the UK, creating jobs and offering equity of access for patients through the UK.

The CGT Catapult will have offices and labs based in the Institute for Regeneration and Repair in the Bioquarter, Edinburgh. The University of Edinburgh and Scottish Blood Transfusion Service have considerable expertise in the development of cell and gene therapy products. Between the Scottish Centre for Regenerative Medicine and the Institute for Regeneration and Repair [currently under construction in the BioQuarter], the University of Edinburgh will have 500 stem cell scientists. Thats the biggest accumulation of stem cell scientists in Europe, and possibly the world.

Pluripotent stem cells [cells with the capacity to develop into all cell types] offer new possibilities for off-the-shelf products. The Cell and Gene Therapy Catapult will work with these scientists to develop their products and accelerate them through clinical trials and become investable propositions, whether through spin-out companies or investment by big pharma.

In addition, we want to work with the NHS, academics, industry and the whole life sciences community to make the best potential of the wealth of experience in the Central Belt of Scotland and use it for the advantage for all of the UK.

Whats the future vision for CGT Catapult?

Our vision is a thriving industry delivering life changing advanced therapies to the world. For the UK to remain one of the most important players globally for these advanced cell and gene therapies.

We want the UK to be at the forefront of manufacture and supply of these living therapies. We want our NHS to be able to adopt them quickly and ensure they get to the right patients as quickly as possible. The UK, thanks to its favourable ecosystem including CGT Catapults activities and continued impact on it, and the continuous support by government for innovation, can stay at the forefront of that.

Visit link:
Bridging the cell and gene therapy gap - The Scotsman

AviadoBio has come flying out the traps surrounded by A-list names. Incubated at F-Prime Capital and Johnson & Johnsons JJDC, AviadoBio exited stealth with $80 million from backers such as New Enterprise Associates (NEA) and a C-suite led by Novartis veteran Lisa Deschamps.

The investors and executives have coalesced around the work Christopher Shaw, Youn Bok Lee, Ph.D., and Do Young Lee, Ph.D., have carried out at King's College London. Shaws group is focused on optimizing AAV9 vectors to deliver genes to the central nervous system for the treatment of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS).

AviadoBio is going after the same indications. The London-based biotech has said the most about its work on FTD, which is spearheaded by a near-clinical AAV gene therapy that is designed to deliver a functional copy of the progranulin gene to slow or stop disease progression.

Researchers identified mutations on the gene as a major cause of familial FTD more than a decade ago, but getting a payload safely to the brain to fix the problem is a potential challenge. Alector, in partnership with GlaxoSmithKline, is studying an intravenous FTD gene therapy in a phase 3 clinical trial, but other groups think a different delivery method is needed.

RELATED: Passage Bio pulls off $216M IPO, blowing past original goal

Passage Bio has come up with a potential solution, injecting its FTD gene therapy into the cisterna magna, one of the spaces surrounding the brain. That prospect moved into phase 1 early this year. AviadoBio is working on a rival prospect, AVB-PGRN, that is given via intrathalamic delivery, enabling it to concentrate the gene therapy on the cells where it is needed.

The series A funding will equip AviadoBio to take AVB-PGRN into the clinic. NEA co-led the round with Monograph Capital. LSP and seed investors Advent Life Sciences, Dementia Discovery Fund, F-Prime, JJDC and LifeArc rounded at the syndicate.

Responsibility for spending the $80 million will fall on Deschamps, who joined AviadoBio as CEO after a 25-year career at Novartis that culminated in her serving as the chief business officer for its gene therapy unit. In addition to AVB-PGRN, AviadoBio is working on earlier-stage gene knock-down assets given via an undisclosed delivery method to treat ALS and FTD.

Continued here:
Gene therapy startup bursts forth from incubation at J&J and F-Prime, graduating with $80M and a Novartis veteran at the helm - FierceBiotech

Cell & gene therapies promise innovative new treatments for severe diseases like cancer, autoimmune disease, muscular dystrophy, and hemoglobinopathies. Several cell & gene therapies products have been approved by the FDA, and more than 1000 are in clinical trials. Because they treat the underlying cause of diseases rather than just the symptoms, cell & gene therapies are attractive alternatives to traditional drug-based therapies.

To study and develop new cell & gene therapies, ProteinSimple, a Bio-Techne brand, offers Simple Western, which is a fully automated capillary immunoassay for the specific detection of target proteins in complex samples. The key value propositions of Simple Western include small sample size requirements (as little as 3 L), high throughput, quick time to results (as little as 3 hours), highly quantitative data, and total protein detection.

This paper highlights how Simple Western and Single-Cell Western are instrumental in developing cell & gene therapies for Duchenne muscular dystrophy, lupus, cystic fibrosis, and sickle-cell disease. We also include references to other noteworthy cell & gene therapy publications that harness the power of Simple Western and Single-Cell Western.

See the original post here:
Advancing Cell Gene Therapies With Simple Western And Single-Cell Western - BioProcess Online

At home, Daphne had started to crawl up some stairs to the upper level where her parents, Lindsey and Brice, were. A sudden seizure left her sprawled across the first step.

At first Lindsey and Brice thought Daphne was simply exhausted from a fun day of playing and had fallen asleep in mid-crawl on the stairs. As they got closer to pick her up, they realized to their horror that she was acting differently than she ever had.

Her body was convulsing, she was turning blue from lack of air to the lungs, and her eyes had more fear in them than anything (we) had ever seen before, Lindsey wrote in a post on the website Caring Bridge.

The incident was the initial indication that something was seriously wrong, but it gave scant warning of the terrifying reality that was to envelop them.

A call to 911 and a trip to the emergency room confirmed Daphne had suffered a seizure the first of many more frightening seizures, more ER visits, more hard falls to the ground, more tremors and body shakes, more fear-filled eyes, Lindsey said. And many more questions with unknown answers.

In July 2019, after a consultation with doctors and genetic testing at the Minnesota Epilepsy Group, Lindsey and Brice received the worse-case scenario answer that had eluded them for months: Daphne has Batten Disease-CLN2, a rare disease wherein her brain doesnt produce an important enzyme needed for proper brain function.

They were told that every motor skill Daphne had acquired in her three years of life along with her ability to dance and play, run and walk would be taken from her. The disease would claim her ability to breathe and, eventually, her life.

We were told, as fact ... your child is going to deteriorate and die, Lindsey said.

Children diagnosed with this genetic disease have a life expectancy of 8 to 12 years.

So we mourned the beautiful, fun, loving, sassy girl that we were still able to see, hold and talk to, Lindsey said. To mourn a child still alive is a situation I cant describe.

We gave ourselves two days to cry, she said. After two days passed with us crying and holding the child we were told would be taken from us very soon in the years to come, she and Brice set out to learn what they could do here and now to give her the best possible life and to fight the brutal prognosis.

They immediately made arrangements for Daphne to undergo surgery to implant a port in her brain to begin the enzyme replacement treatment that slows the deterioration.

For nearly half her life, Daphne, now 6, has been battling the cruel neurodegenerative, or brain, disease that steadily steals from her. While other young children are gaining words, skills, talents and abilities, Daphne loses these things at a heart-breaking rate, Lindsey said.

Every two weeks, she receives an enzyme-replacement treatment, approved by the FDA in 2017, to slow the disease progression.

Daphne undergoes the treatment by Altru doctors and nurses who have been specially trained to administer it. The procedure, which takes nearly five hours, delivers the drug Brineura to the brain via a port. Brineura is a synthetic form of the enzyme that her brain does not produce.

If she didnt have this treatment, she would die, Lindsey said.

Brineura is a huge blessing that slows deterioration of several functions but does not affect the deterioration that starts in the retina of the eye, she said.

(Daphne) doesnt walk very well. She has lost most of her words, and she is beginning to lose her sight, Lindsey said. Time is not on her side.

Daphne has regressed developmentally, from a 3-year-old to a 1-year-old.

The treatment gives the Enger family, including Daphnes brother Oliver, 9, the gift of time to be with our daughter and more time for a cure to be found, Lindsey said.

And so Daphne fights. She has been fighting every single day for two and a half years. She has lost (so much), she fights with everything she has every minute of every day not to lose more. I am in awe of how strong she is.

Through it all, she keeps her smile her smile and her sass, Lindsey said. As the parents of such a brave and strong girl, we are in awe of her resilience and determination.

The Engers and other parents around the country who are facing this same terrible situation are worried that FDA officials harbor a misconception that the drug Brineura is helping more than it is, Lindsey said. It slows the deterioration, but you have to understand how fast this disease progresses. (The drug) is not able to slow it enough.

Children with the disease are still deteriorating at a very fast rate, said Lindsey, who is part of a 200-member worldwide Facebook group of parents and others who have been impacted by the disease.

These parents have started a petition and are determined to gather as many signatures as possible to urge the FDA to look carefully at the positive pre-clinical trial results of gene therapy for Batten Disease and approve clinical trials.

The gene therapy would alter Daphnes genes to make her brain develop the enzyme it needs for proper brain function. The therapy has been shown to be effective in not only stopping disease progression, but restoring the functions that have been lost, Lindsey said.

Results of preclinical trials have been 100% successful that really gets us going, she said.

These parents want the FDA to fast track the therapy, she said. We know they can fast track it. And they believe that, given the impact of the petition signatures, the FDA will be persuaded to quickly move the gene therapy forward.

We understand the rarity of the (Batten) disease, and that there are many worthy causes, Lindsey said, but we are not grasping at straws.

We need the FDA to see that these childrens lives have potential, they have meaning, she said. (These children) have the right to live the same full and long life everyone else is promised. These children, including Daphne, deserve to live.

We need to give our children every possible chance to save their lives, she said, noting that companies working on the therapies are ready to go.

If the FDA approves clinical trials, it would give hope to many others who are afflicted with Batten Disease.

If trials are approved, she and Daphne are ready at a moments notice to go wherever they must out of state or out of country to participate.

We are almost at the finish line, she said. We just need to get the FDAs attention now.

Dealing with this crisis has been really hard, Lindsey said. It has changed us; its made us stronger. We have a lot of faith, a lot of hope, a lot of love.

We have not and will not give up hope and faith for a cure for Daphne before it is too late, she said, adding that her daughter is inspirational.

Shes so happy all the time, Lindsey said. I dont want to lose that smile, that laughter.

To sign a petition that urges the FDA to approve gene therapies for children with Batten Disease, go to https://chng.it/wRZjqXZk9G , and share it with others to sign, so we can get the attention of the FDA please and save our children, said Lindsey Enger. You dont have to donate. We just ask that you please sign the petition and share it for Daphne.

Link:
Parents of Grand Forks girl fighting rare disease push for FDA approval of gene therapy that could save her life - Grand Forks Herald

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced a late-breaking abstract and poster presentation by Dr. Rachel Bailey, Assistant Professor at UT Southwestern Medical Center on positive preclinical data for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy at the American Epilepsy Society Annual Meeting on December 6th 2021.

SLC13A5-related epilepsy results from a mutation in the SLC13A5 gene that prevents citrate from being taken up into neurons in the brain. Affected children present with seizures beginning within a few days of birth and as they continue to grow, have motor progression and difficulty with speech and language development. Some children never achieve walking independently, said Rachel M. Bailey, Ph.D., Assistant Professor with the Center for Alzheimer's and Neurodegenerative Diseases and Pediatrics at UT Southwestern. Currently there are no treatments for SLC13A5 deficiency that target the underlying cause of disease. In knockout mouse models of SLC13A5 deficiency, treatment with TSHA-105 resulted in normalized citrate levels, reduced seizure activity and improved survival regardless of age. We are highly encouraged by the positive therapeutic response and absence of toxicity in these preclinical models and look forward to further exploring the possible utility of TSHA-105 as a treatment for SLC13A5 deficiency.

Dr. Bailey, whose research is supported in part by Taysha, and UT Southwestern maintain financial interests in Taysha Gene Therapies due to their development of the intellectual property that serves as the basis for TSHA-105.

These preclinical results suggest that TSHA-105 can demonstrate functional improvements with intervention at any age in a potentially safe and tolerable manner which would be expected to translate into a meaningful benefit to patients with SCL13A5 deficiency, said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. These highly encouraging preclinical data further support our plan to submit an IND/CTA filing in 2022.

TSHA-105 is a self-complementary vector encoding a codon-optimized human SLC13A5 gene that was evaluated in an SLC13A5 knockout (KO) mouse model recapitulating the increased plasma citrate levels, electroencephalogram (EEG) abnormalities and an increased susceptibility to seizure induction seen in patients. CSF-delivered TSHA-105 significantly decreased plasma citrate levels in SLC13A5 KO mice and reduced epileptic activity. Increased seizure susceptibility in SLC13A5 KO mice measured by the Racine scale, a well-established methodology for assessing seizure severity in preclinical models, was attenuated with TSHA-105 treatment in both age groups. No adverse findings were detected following CSF delivery of TSHA-105. An IND/CTA filing for TSHA-105 for the treatment of SLC13A5-related epilepsy is expected in 2022. TSHA-105 previously received orphan drug designation from the U.S. Food and Drug Administration and from the European Commission.

SLC13A5 deficiency is a form of infantile epilepsy caused by mutations in the SLC13A5 gene. As an autosomal recessive disorder, two copies of the mutated gene must be inherited for an infant to be affected. This type of epilepsy manifests as developmental delay, and seizures beginning within the first few days of life. SLC13A5 deficiency is a rare disorder, with an estimated prevalence of 1,900 patients in the United States and in Europe. Current standards of care include anti-seizure medications which only target the symptoms and do not address the underlying cause of the disease.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our 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, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including TSHA-105, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, TSHA-105s eligibility for accelerated approval in the United States and Europe, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020 and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, both of which are available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

Read more:
Taysha Gene Therapies Announces Late-Breaking Abstract and Poster Presentation on Positive Preclinical Data For TSHA-105 Demonstrating Therapeutic...

MINNEAPOLIS and SAN FRANCISCO, Dec. 08, 2021 (GLOBE NEWSWIRE) -- Be The Match BioTherapies, an organization offering solutions for companies developing and commercializing cell and gene therapies (CGTs), and Vineti, the provider of the leading digital enterprise platform for cell and gene therapy supply chains, today announced a collaboration to develop joint solutions that simplify and scale supply chain management of cell and gene therapies.

The non-exclusive collaboration will bring together industry leaders in workflow and logistics solutions -- Be The Match BioTherapies cellular therapy supply chain management services and Vinetis Personalized Therapy Management (PTM) platform. The collaboration will enable the Be The Match BioTherapies Cell Therapy Supply Chain Managers and Logistics Coordinators to utilize the PTM platform on behalf of shared biopharmaceutical clients with greater efficiency and simplicity.

The organizations leverage complementary strengths in starting material collection, cell therapy supply chain and managed logistics, Chain of Identity (COI) and Chain of Custody (COC), and enterprise-grade digital solutions for end-to-end value chain management. The collaboration will provide unique integrated solutions for these mission-critical components of CGT operations, and will simplify workflows, speed time to treatment, and provide the flexibility that CGT development requires.

This is a message to the industry that both parties are committed to improving our clients experience, and to improving outcomes for patients by building a combined solution that eliminates unnecessary complexity, said Amy Ronneberg, CEO of the National Marrow Donor Program (NMDP)/Be The Match and Be The Match BioTherapies.

By combining each teams core expertise, the organizations aim to create innovative solutions that blend Be The Match BioTherapies best in class cell therapy supply chain services with Vinetis proven expertise in advanced therapy management and enterprise grade digital technology to deliver next-generation therapy management, automated traceability, and digitized compliance for CGTs.

Story continues

Were very honored to partner with Be The Match BioTherapies, said Amy DuRoss, CEO and Co-founder of Vineti. Both organizations share a focus on driving transformative outcomes for patients that wouldnt be possible without cell and gene therapies. Well be able to offer a solution that reduces timelines to the clinic, while allowing CGT developers to simplify and scale operations with best-in-case expertise, services, and digital tools.

The Be The Match BioTherapies-Vineti solution will be available from both organizations Business Development teams. This news follows the recent Be The Match BioTherapies webinar on scalable and efficient cell collection networks as well as Vinetis recent partnership announcement with Autolomous.

About Be The Match BioTherapies

Be The Match BioTherapies is the only cell and gene therapy solutions provider with customizable services to support the end-to-end cell therapy supply chain. Backed by the industry-leading experience of the National Marrow Donor Program/Be The Match, and a research partnership with the CIBMTR (Center for International Blood and Marrow Transplant Research), the organization designs solutions that advance the development of cell and gene therapies across the globe.

Be The Match BioTherapies is dedicated to accelerating patient access to life-saving cell and gene therapies by providing high-quality cellular source material from the Be The Match Registry, the worlds most diverse registry of more than 22 million potential blood stem cell donors. Through established relationships with apheresis, marrow collection, and transplant centers worldwide, the organization develops, onboards, trains, and manages expansive collection networks to advance cell therapies. Be The Match BioTherapies uses a proven infrastructure consisting of regulatory compliance and managed logistics experts and cell therapy supply chain case managers to transport and deliver regulatory-compliant life-saving therapies across the globe successfully. Through the CIBMTR, Be The Match BioTherapies extends services beyond the cell therapy supply chain to include long-term follow-up tracking for the first two FDA-approved CAR-T therapies.

For more information, visit http://www.BeTheMatchBioTherapies.com or follow Be The Match BioTherapies on LinkedIn or Twitter.

About Vineti

Vineti offers the first commercial, configurable cloud-based platform to expand patient access to life-saving cell and gene therapies. Vineti was co-founded by GE and the Mayo Clinic to solve the key challenges that patients, medical providers, biopharmaceutical companies, and regulators face in the delivery and commercialization of individualized therapies. Now a fully independent company, Vineti offers a digital platform of record to integrate logistics, supply chain management, manufacturing, and clinical data for personalized therapies. The Vineti Personalized Therapy Management (PTM) platform aligns and orchestrates the advanced therapy process and improves product performance overall, supporting the full continuum of patient-specific therapies, including personalized cancer vaccines and autologous and allogeneic cell and gene therapies. Vineti is currently serving patients, healthcare providers, and researchers in hundreds of leading medical centers and manufacturing centers world-wide on behalf of a growing number of biopharmaceutical partners. The World Economic Forum has honored Vineti as a World Economic Forum Technology Pioneer. Vineti is headquartered in San Francisco, California, with teams based in the Washington, D.C. area and Yerevan, Armenia. For more information, please visit http://vineti.com.

Contact Information:

Bonnie Quintanilla, Clarity Quest (for Be The Match BioTherapies)bonnie@clarityqst.com

Dan Budwick, 1AB Media (for Vineti)dan@1abmedia.com

See the original post:
Be The Match BioTherapies and Vineti collaborate to develop innovative, integrated supply chain management solutions for cell and gene therapies -...

EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular diseases, today announced the first authorization of a Clinical Trial Application (CTA) in Europe for ATA-100, its gene therapy for the treatment of the fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9). This authorization was granted by the United Kingdom Medicines & Healthcare products Regulatory Agency (MHRA). Additional CTAs were filed in France and Denmark.

We are thrilled to obtain our first CTA approval in the U.K. for the devastating LGMD2I/R9 disease, said Dr Sophie Olivier, Chief Medical Officer of Atamyo. Atamyo plans to initiate dosing in patients for ATA-100 in the first half of 2022.

LGMDR9 is a severe muscular dystrophy with progressive symptoms for which there is currently no approved treatment, said Pr John Vissing, Director of the Copenhagen Neuromuscular Center at the National Hospital, Rigshospitalet (Denmark), and principal investigator of this trial. It is a great motivation to know that the work we are doing has the potential to make a life-changing difference for the patients affected by this disease.

We are eager to start treating the first European patient and mark this as a milestone for the field in advancing a potential one-time treatment for patients with LGMD-R9, said Pr Volker Straub, Professor of Medicine and Director of the John Walton Muscular Dystrophy Research Centre, Newcastle University (UK).

This is an important step in our mission to bring to patients suffering from limb-girdle muscular dystrophies (LGMD) a new generation of safe and effective gene therapies, after only one year of activity, said Stphane Degove, CEO of Atamyo Therapeutics.

LGMD2I/R9 is a rare genetic disease caused by mutations in the gene that produces fukutin-related protein (FKRP). It affects an estimated 5,000 people in the US and Europe. Symptoms appear around late childhood or early adulthood. Patients suffer from progressive muscular weakness leading to loss of ambulation. They also are prone to respiratory impairment and myocardial dysfunction. There are currently no curative treatments for LGMDR9.

ATA-100, a gene therapy candidate for LGMD21/R9, delivers a normal copy of the gene for production of FKRP proteins. The therapy is based on the research of Atamyo Chief Scientific Officer Isabelle Richard, Ph.D., Research Director at CNRS who heads the Progressive Muscular Dystrophies Laboratory at Genethon.

In preclinical mice models, ATA-100 demonstrated its tolerability and capability to correct symptoms and biomarkers of the pathology at unprecedented low doses for systemic AAV-mediated gene transfer addressing muscle diseases.

About Atamyo Therapeutics

Atamyo Therapeutics is focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyos most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD). The name of the company is derived from two words: Celtic Atao which means Always or Forever and Myo which is the Greek root for muscle. Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments. For more information visit http://www.atamyo.com

Go here to read the rest:
Atamyo Therapeutics Obtains First Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-100, its Gene Therapy to Treat Limb-Girdle...

SparingVision Strengthens Leadership Team with the Appointment of Dr Mehdi Gasmi as Chief Operating Officer

Raffaella Toso, PhD, joins as VP Corporate Development & Alliance Management and Florence Paliargues appointed as VP Portfolio Project Management

Paris, December 9, 2021 SparingVision, a genomic medicine company developing vision-saving treatments for ocular diseases, today announces the appointment of Dr Mehdi Gasmi, PhD, as Chief Operating Officer (COO), as well as other senior appointments, further strengthening the SparingVision team.

Mehdi Gasmi is a seasoned executive with over 25 years of experience across the gene therapy drug development field, including in ophthalmology indications. His deep experience in the design and translational development of gene therapy drug candidates has been garnered through a multitude of posts, most recently as a Board member and, prior to that, as President and Chief Scientific Officer at Adverum Biotechnologies Inc. (Nasdaq: ADVM) in California, USA. During his time as CSO of Adverum, Gasmi led the companys R&D and translational operations for AAV-based gene therapy products, specifically ADVM-022, a treatment for ocular VEGF-driven diseases.

Prior to Adverum, Mehdi Gasmi held numerous roles at gene therapy companies both in the US and in Europe, including Genethon, Ceregene, Cell Genesys and Chiron. In addition to his corporate experience, he worked on the development of HIV-based vectors for gene delivery at academic institutions including City of Hope and the University of California, San Diego. Mehdi Gasmi obtained his Ph.D. in Biochemistry from Claude Bernard University, Lyon in 1996.

Stphane Boissel, President and Chief Executive Officer of SparingVision, said: Following our recently announced transformational deal with Intellia Therapeutics, I am delighted to strengthen our management team with the appointment of Mehdi Gasmi to further support our corporate expansion and acceleration as a leader in ophthalmic genomic medicine. Mehdi brings extensive expertise in the gene therapy drug development space, particularly in ophthalmology.

Story continues

Dr Mehdi Gasmi, PhD, commented: SparingVisions disruptive approach to combat retinal disease is inspiring and I am excited to be joining such a visionary team. Encompassing both gene agnostic therapies and CRISPR-Cas9 approaches, SparingVision really has the worlds most exciting and broad pipeline to potentially bring significant change to patients in areas of huge unmet need.

SparingVision is also delighted to announce the appointments of Dr Raffaella Toso, PhD, as VP Corporate Development and Florence Paliargues, Pharm. D, as VP Portfolio Project Management. Raffaella Toso is joining SparingVision from Spark Therapeutics where she most recently held the role of Search and Evaluation Lead. Raffaella Toso is based in Philadelphia, PA. Florence Paliargues has more than 15 years of experience in drug development and program leadership and was most recently Program Leader at biopharmaceutical company Cellectis. She is based in Paris, France.

Stphane Boissel, President and Chief Executive Officer of SparingVision, said: I am delighted to welcome Raffaella and Florence, who both have strong backgrounds in genomic medicines are a further demonstration of the exceptional talent we are bringing into the Company to support us in our mission.

**ENDS**

Contacts:

SparingVision

Stphane Boissel

President and CEO

Nathalie Trepo

Investor Relations nathalie.trepo@sparingvision.com

Consilium Strategic Communications

Amber Fennell, Genevieve Wilson, Davide Salvi

+44 (0)20 3709 5700sparingvision@consilium-comms.com

NOTES TO EDITORS:

About SparingVisionSparingVision is a genomic medicines company with a mission to translate pioneering science into vision saving treatments. Leveraging its unparalleled understanding of retinal diseases, SparingVision has built the worlds most compelling portfolio of synergistic cutting-edge gene therapy and genome editing treatments for inherited retinal diseases (IRDs). Both of its most advanced products, SPVN06 and SPVN20 look to go beyond single gene correction therapies to deliver new mutation agnostic treatments for Retinitis Pigmentosa (RP), a group of IRDs which are the leading cause of blindness globally. The Company also has a strategic collaboration with Intellia Therapeutics (NASDAQ:NTLA) to develop novel genome editing-based treatments for ocular disease utilizing CRISPR-Cas9 technology.

SparingVision is backed by high-quality international investors including 4BIO Capital, Advent France Biotechnology, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, Intellia Therapeutics, UPMC Enterprises, Jeito Capital, Ysios Capital.

Visit http://www.sparingvision.com for more and follow us on LinkedIn and Twitter @SparingVision.

See the original post:
SparingVision Strengthens Leadership Team with the Appointment of Dr Mehdi Gasmi as Chief Operating Officer - Yahoo Finance

Groundbreaking gene therapy treatments have tremendous potential to provide treatment to the around one in ten Americans living with rare diseases. But these game-changing therapies also bring with them new challenges, including how the individuals who need them most will get access to and be able to pay for these expensive treatments.

To tackle this issue, we convened a panel of health leaders with ample experience in rare disease treatment and gene therapy to talk through how our health system can approach paying for gene therapy. In our latest 5 Slides Were Watching conversation, Jennifer Hodge, PhD, US Rare Neurology Medical Team lead at Pfizer, Angela Ramirez Holmes, founder and president of Cal Rare, and Ryan Fischer, chief advocacy officer at Parent Project Muscular Dystrophy, discuss potential solutions to this complex issue.

Get the latest state-specific policy intelligence for the health care sector delivered to your inbox.

Hodge brought a slide that illustrated the benefits of gene therapy, including the potential for one-time treatments that can eliminate the need for ongoing therapies and reduce the long-term economic impact of treating rare diseases. She also listed some of the challenges to adopting widespread use of gene therapy, such as the need for rare disease specialists to deliver care and ensuring patients get appropriate and timely care. She said 95% of individuals living with rare diseases have limited or no approved treatment options.

It really makes the rare disease population, collectively, one of the most underserved communities in medicine today. And that, frankly, is unacceptable to me.

Holmes focused on Medi-Cals role in rare disease treatment. Her slide cited that 51% of births in California are funded by Medi-Cal and 53% of the states children under five receive Medi-Cal coverage. She said Medi-Cals significant share of state health care coverage indicates the state needs to prioritize policies that make treatment accessible for Medi-Cal recipients with rare diseases.

With nearly four million rare disease patients in California, we know that policy solutions will be needed. And if we dont address the people that are covered by Medi-Cal, we will leave behind that piece of access for patients.

Fischer shared a slide that detailed ways California can advance rare disease treatments: continuing to engage with rare disease experts, leveraging investments in data collection, and continuing patient-focused drug development (PFDD).

[Its important] that we recognize that were really part of one drug development ecosystem. Its essentially a community of stakeholders living in conjunction with other components and their environment, all interacting as one system. We have to recognize that and recognize the needs of all stakeholders within that ecosystem.

Go here to see the original:
Video: Gene therapy and the promise for rare disease - State of Reform