Orphan Drug and Rare Pediatric status for Taysha’s TSHA-104 in SURF1-associated Leigh syndrome

Taysha Gene Therapies Inc. () announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. FDA for -104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Taysha anticipates it will submit an Investigational New Drug (IND) application to the FDA for -104 in 2021.
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Taysha has secured rare pediatric disease designation and orphan drug designation for multiple programs, including GM2 gangliosidosis (-101), CLN1 (-118), Rett syndrome (-102) and now SURF1 (-104). In addition to these designations, the company also has fast track status for the CLN1 program. 

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 10-15% of people with Leigh syndrome have a SURF1 mutation. 

Press release