AllStripes, Taysha Gene Therapies partner on rare disease gene therapy program

By The Science Advisory Board staff writers

January 4, 2021 -- AllStripes and Taysha Gene Therapies have entered a multiyear collaboration focused on advancing the development of TSHA-104, an adeno-associated virus 9 (AAV9)-based gene therapy for the treatment of surfeit locus protein 1 (SURF1)-associated Leigh syndrome.

Under the collaboration, AllStripes will use its technology platform to unify otherwise scattered and fragmented SURF1-associated clinical data, allowing researchers to uncover new insights into patient history and to better inform clinical studies.

Leigh syndrome is a severe neurological rare disease that primarily affects infants. Mutations in the SURF1 gene prevent mitochondria from producing enough energy for cells to function normally, leading to Leigh syndrome. Approximately 10%-15% of people with Leigh syndrome have a SURF1 mutation and there are currently no targeted treatments or cures for SURF1-associated Leigh syndrome.

TSHA-104 has been granted rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA). Taysha plans to submit an investigational new drug application for TSHA-104 in SURF1-associated Leigh syndrome to the FDA in 2021.

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Taysha nabs rare pediatric disease, orphan drug status
Taysha Gene Therapies has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration for TSHA-104,...
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