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Taysha Gene Therapies Announces Positive Clinical Data Across Adult and Pediatric Patients from Low Dose Cohort in Ongoing REVEAL Phase 1/2 Trials Evaluating TSHA-102 in Rett Syndrome
Durable improvements across consistent clinical domains in both adult and pediatric patients, including motor skills, communication/socialization, autonomic
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"Durable improvements across consistent clinical domains in both adult and pediatric patients, including motor skills, communication/socialization, autonomic function, seizures, and an encouraging safety profile seen across adult (up to 52 weeks) and pediatric (up to 22 weeks) patients with different genetic mutation severity Longer-term data from both adult patients showed sustained and new improvements across multiple efficacy measures and clinical domains following the completion of steroid taper (patient one: sat unassisted for first time in over a decade, normalized sleep, stabilized seizures; patient two: improved hand stereotypies and breathing, seizure-free for 8.5 months at 25% lower anti-seizure medication) Initial data from first two pediatric patients showed improvements across multiple efficacy measures and clinical domains, with early evidence of developmental gains (patient one: improved hand function, grasp and gross motor coordination, gained visual reception and receptive language skills; patient two: gained ability to stand up from chair and walk up a stair, increase in seizure-free days) IDMC approved Company’s request for early advancement to cohort two (high dose) in the REVEAL pediatric trial; dosing expected in Q3 2024 following IDMC review of initial safety data from the first high dose patient in the adolescent and adult trial Company will host webcast today at 8:00 AM Eastern Time DALLAS, June 18, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced positive longer-term clinical data from the ongoing REVEAL Phase 1/2 adolescent and adult trial and initial clinical data from the REVEAL Phase 1/2 pediatric trial evaluating TSHA-102 in Rett syndrome. “We are highly encouraged by the safety profile and broad clinical response observed across multiple domains in both the adult and pediatric patients with different genetic mutation severity treated with the low dose of TSHA-102,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “The longer-term follow up data indicate a durable response with sustained and new improvements across multiple clinical domains in both adult patients, and imp...