Taysha Gene Therapies Collaborates with AllStripes on Leigh Syndrome

Rare Daily Staff

Rare disease gene therapy developer Taysha Gene Therapies and AllStripes, formerly known as RDMD, have entered into a multi-year collaboration in which Taysha will leverage AllStripes’ data platform to inform its understanding of SURF1-associated Leigh syndrome natural history and burden of disease, as well as patients’ diagnostic journeys.

Leigh syndrome is a severe neurological disorder that usually presents in the first year of life. It is characterized by progressive loss of mental and movement abilities that can result in death within two to three years. Approximately 10 to 15 percent of people with Leigh syndrome have a SURF1 mutation. There are currently no approved therapies to treat SURF1-associated Leigh syndrome.

The collaboration will focus on advancing the development of TSHA-104, an AAV9-based gene therapy product candidate in development for the treatment of SURF1-associated Leigh syndrome. TSHA-104 is a recombinant AAV9 vector with engineered transgene encoding the human SURF1 protein. TSHA-104 has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration.

Taysha will utilize AllStripes’ clinical database to uncover new insights into disease progression and better inform selection of endpoints for clinical studies.

“This collaboration will allow us to leverage the AllStripes technology platform to optimize our therapeutic strategy and to potentially accelerate the development of TSHA-104 in SURF1-associated Leigh syndrome,” said RA Session, II, president, founder and CEO of Taysha. “We remain committed to developing a safe and effective gene therapy for patients suffering with this devastating disease, and data generated from this unique collaboration could bring us one step closer to our goal.”

AllStripes has developed a technology platform that generates FDA-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in treatment research online and benefit from their own medical data.

Taysha Gene Therapies expect to submit an Investigational New Drug (IND) application for TSHA-104 in SURF1-associated Leigh syndrome to the FDA in 2021.

Photo: RA Session, II, president, founder and CEO of Taysha