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.

Regulatory Roundup: Designations come through before year's end
This week's Regulatory Roundup covers activities from November 30 to December 4 and is filled with breakthrough, orphan, and rare disease designations...

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,...

Taysha gets $95M in financing to advance gene therapies
Taysha Gene Therapies has closed on $95 million in series B financing to advance gene therapies for monogenic central nervous system diseases.