other material
confidence high
sentiment positive
materiality 0.65
First pediatric patient dosed with TSHA-102 in REVEAL Phase 1/2 trial for Rett syndrome
Taysha Gene Therapies, Inc.
- First pediatric patient (female, 5-8 years, stage 3 Rett syndrome) dosed at RUSH University Medical Center.
- MHRA authorized UK CTA for TSHA-102 in pediatric patients, expanding REVEAL into the UK.
- Initial safety and efficacy data for cohort one (low dose, n=3) expected in mid-2024.
- TSHA-102 uses miRARE technology to mediate MECP2 expression on a cell-by-cell basis.
- TSHA-102 also has FDA Fast Track, Orphan Drug, and Rare Pediatric Disease designations.