- Taysha Gene Therapies announced it raised $30 million in funding and signed a strategic partnership with UT Southwestern
Taysha Gene Therapies, a company that launched with a mission to eradicate severe and life-threatening monogenic diseases of the central nervous system (CNS) by advancing its pipeline of 15 AAV (adeno-associated virus) gene therapy programs, announced it has raised $30 million in seed financing co-led by PBM Capital (the first institutional investor in AveXis) and Nolan Capital (the investment fund of former AveXis CEO Sean Nolan). And Taysha Gene Therapies reunites former investors and executives from AveXis while accelerating its discovery and development efforts with the UT Southwestern Gene Therapy Program and Department of Pediatrics, one of the largest pediatric neurology residency programs in the U.S.
Taysha’s Board of Directors played an integral role in the formation of the company and is comprised of Sean Nolan (Chairman of the Board); Paul Manning of PBM Capital; Claire Aldridge, Ph.D., of UT Southwestern; and RA Session II, President, CEO and Founder of Taysha.
Taysha and UT Southwestern entered into a strategic partnership to rapidly and efficiently translate novel AAV gene therapies from bench to bedside. The UT Southwestern Gene Therapy Program is led by Steven Gray, Ph.D., Director of the Viral Vector Core and Assistant Professor in the Department of Pediatrics, and Berge Minassian, M.D., Division Chief of Child Neurology. UT Southwestern developed a GMP viral vector manufacturing facility with the capacity to support Taysha’s broad preclinical and clinical development programs.
And under the partnership, UT Southwestern will conduct discovery and preclinical research, lead IND-enabling studies, provide clinical GMP manufacturing, as well as execute natural history studies. Taysha will lead all clinical development, regulatory strategy, commercial manufacturing, and commercialization activities. The collaboration will be governed by a joint steering committee composed of key leadership members from Taysha and UT Southwestern.
Taysha’s approach is to combine the speed, scale, and expertise of the UT Southwestern Gene Therapy Program with the experience of a proven management team in the gene therapy space. And the company is developing an extensive pipeline of potentially curative therapies for monogenic CNS diseases in both rare and large-market indications that are centered on proven AAV technology. In addition, Taysha is developing a novel AAV capsid platform that utilizes machine learning, DNA shuffling, and directed evolution to improve targeted delivery. The company is also developing an AAV redosing platform that facilitates redosing by subverting the humoral immune response through delivery to the vagus nerve.
Taysha currently has 15 AAV gene therapy programs in its pipeline with options to an additional four programs. TGTX-101, a gene replacement therapy for GM2-Gangliosidosis, is expected to advance into clinical development later this year. In total, Taysha is planning to file four Initial New Drug (IND) applications by the end of 2021, including indications for SURF1 deficiency, SLC6A1 haploinsufficiency, and Rett syndrome.
Key Quotes:
“Gene therapies have proven to deliver transformational benefit to patients who suffer from devastating diseases with significant, unmet medical need. Our mission at Taysha is to build upon these advancements to eradicate monogenic CNS diseases for the thousands affected. In joining forces with UT Southwestern—home to some of the brightest minds in gene therapy—we will advance our deep pipeline of potentially curative medicines with speed and scale. At Taysha, we are ushering in a new era of gene therapy drug development, one in which we can rapidly translate early discoveries into the clinic and beyond.”
-RA Session II
“Together with Taysha, we are merging cutting-edge translational research, hands-on clinical care, and proven regulatory and commercial expertise – ultimately creating an engine for new cures,” said Claire Aldridge, Ph.D., Taysha Board Member and Associate Vice President of Commercialization and Business Development at UT Southwestern Medical Center. “In a short amount of time, I’ve already witnessed how quickly and efficiently we can leverage our collective expertise and resources to advance new gene therapies to the patients who so desperately need them.”
-Claire Aldridge, Ph.D., Taysha Board Member and Associate Vice President of Commercialization and Business Development at UT Southwestern Medical Center
“Taysha is leveraging the proven safety and heritage of AAV technology—combining it with an experienced management team and the world-class capabilities of UT Southwestern—to rapidly accelerate the research and development of multiple new therapies at a scope we haven’t seen before. This is an exciting time for gene therapy, and Taysha’s approach brings promise to patients suffering from many devastating CNS diseases.”
-Sean Nolan, Chairman of Taysha and former CEO of AveXis
