RARE Daily

Taysha Gene Therapies Launches, Targets Monogenic CNS Diseases

April 29, 2020

Rare Daily Staff

Taysha Gene Therapies has launched with $30 million in seed financing and a strategic partnership with the University of Texas Southwestern Gene Therapy program to develop gene therapies to treat monogenic diseases of the central nervous system, in both rare and large-market indications. 

The Dallas, Texas-based biotech reunites former investors and executives from gene therapy pioneer AveXis, which also got its start in Texas. Taysha plans to use the new funding to advance its pipeline of 15 AAV-based gene therapy programs, with options to an additional four programs, while accelerating its discovery and development efforts in collaboration with UT Southwestern’s Gene Therapy program and department of Pediatrics, one of the largest pediatric neurology residency programs in the United States.

PBM Capital and Nolan Capital co-led the financing. PBM was the first institutional investor in AveXis, and Nolan Capital is the investment fund of former AveXis CEO Sean Nolan, who is chairman of Taysha’s board of directors.

“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,” said RA Session II, founder, president, and CEO of Taysha.

Concurrent with the launch, Taysha and UT Southwestern have entered into a strategic partnership to rapidly and efficiently translate novel AAV gene therapies from bench to bedside. UT Southwestern has developed a state-of-the-art, GMP viral vector manufacturing facility with the capacity to support Taysha’s broad preclinical and clinical development programs.

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, and 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. In addition to developing its extensive pipeline of AAV gene therapies targeting monogenic CNS diseases, 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’s TGTX-101, a gene replacement therapy for the rare disease 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 haplo-insufficiency and Rett syndrome.

“This is an exciting time for gene therapy, and Taysha’s approach brings promise to patients suffering from many devastating CNS diseases,” said Nolan.

Photo: Sean Nolan, chairman of Taysha

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