TSHA - Taysha Gene Therapies Shares Jump On Initial Data From First Pediatric Patient With Rare Neurodevelopmental Disorder | Benzinga
Tuesday, Taysha Gene Therapies Inc (NASDAQ:TSHA) shared initial clinical data from the first pediatric patient from the REVEAL Phase 1/2 adolescent and adult trial of TSHA-102, an investigational gene therapy, in adult females with Rett syndrome.
Rett syndrome is a rare neurodevelopmental disorder characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities, and shortened life expectancy.
Results from the first patient and second patient with late motor deterioration stage four Rett syndrome dosed with TSHA-102 in the low dose cohort (5.7×1014 total vg) showed:
- Generally well-tolerated with no treatment-emergent serious adverse events as of ...