TSHA-104

TSHA-104 is an AAV9-based gene therapy developed to deliver a functional copy of the SURF1 gene to treat patients with SURF1-associated Leigh syndrome, a rare monogenic mitochondrial disease that affects the central nervous system[1][6][5]. The therapy uses an adeno-associated virus serotype 9 (AAV9) vector to target central nervous system cells, with the goal of restoring SURF1 function and mitochondrial respiratory chain complex IV activity. TSHA-104 was originally developed by Taysha Gene Therapies and received rare pediatric disease designation and orphan drug designation from the FDA for this indication[1][5][6]. As of early 2024, further development by Taysha was discontinued and rights were transferred back to the originating institution, with future development status uncertain[7][9][10].

Other names

SURF1 gene therapySURF-1 gene therapySURF 1 gene therapyAAV9-SURF1AAV-9-SURF1AAV 9-SURF1