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 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
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