Taysha Gene Therapies, Inc. (NASDAQ: TSHA) has announced positive longer-term clinical data from the ongoing REVEAL phase 1/2 adolescent and adult trial, as well as initial clinical data from the REVEAL phase 1/2 pediatric trial evaluating TSHA-102 in Rett syndrome.
In the adult trial, the first patient, a 20-year-old with a large MECP2 deletion, and the second patient, a 21-year-old with a missense MECP2 mutation, both saw sustained and new improvements across multiple clinical domains as of 52 weeks post-treatment for the first patient and 36 weeks post-treatment for the second patient. Both patients showed improvements in motor skills, communication/socialization, autonomic function, and seizures.
In the pediatric trial, initial results from the first pediatric patient, a 6-year-old with an MECP2 deletion, and the second pediatric patient, a 7-year-old with a missense MECP2 mutation, showed initial improvements across multiple clinical domains as of 12 weeks post-treatment for the first patient and 8 weeks post-treatment for the second patient. Both patients demonstrated improvements in motor skills, communication/socialization, autonomic function, and seizures.
The company has completed dosing in cohort one (low dose) and dosed the first patient in cohort two (high dose) in the second quarter of 2024. Initial available safety and efficacy data from cohort two are expected in the second half of 2024.
The longer-term follow-up data indicate a durable response with sustained and new improvements across multiple clinical domains in both adult patients. The initial improvements in both adult and pediatric patients further reinforce the potential of TSHA-102 to be transformative for a broad range of patients with Rett syndrome.
TSHA-102 has received regenerative medicine advanced therapy, fast track, and orphan drug and rare pediatric disease designations from the FDA, orphan drug designation from the European Commission, and innovative licensing and access pathway designation from the Medicines and Healthcare products Regulatory Agency.
The company will host a webcast to discuss the clinical data for TSHA-102 in Rett syndrome.
The details of the data will also be presented at the 2024 International Rett Syndrome Foundation Rett Syndrome Scientific Meeting by principal investigators of the REVEAL trial.
Taysha Gene Therapies is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. The market has reacted to these announcements by moving the company's shares -28.2% to a price of $2.9. Check out the company's full 8-K submission here.
