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Taysha Gene Therapies Announces First Pediatric Patient Dosed with TSHA-102 in REVEAL Phase 1/2 Pediatric Trial in Rett Syndrome
Initiation of REVEAL pediatric trial in the U.S. broadens the clinical evaluation of TSHA-102 to female patients 5-8 years old with stage three Rett syndrome
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"Initiation of REVEAL pediatric trial in the U.S. broadens the clinical evaluation of TSHA-102 to female patients 5-8 years old with stage three Rett syndrome MHRA authorized the CTA for TSHA-102 in pediatric patients with Rett syndrome, enabling expansion of ongoing U.S. REVEAL pediatric trial into the U.K. TSHA-102 clinical program now includes broad evaluation across pediatric, adolescent and adult patients in three countries including the U.S., U.K. and Canada Initial safety and efficacy data for cohort one (low dose, n = 3) in the REVEAL pediatric trial expected in mid-2024 DALLAS, Jan. 10, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that the first pediatric patient has been dosed with TSHA-102 in the REVEAL Phase 1/2 pediatric trial in the United States (U.S.) evaluating the safety and preliminary efficacy of TSHA-102 in stage three female patients 5-8 years of age with Rett syndrome. The Company also announced the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has authorized the Clinical Trial Application (CTA) for TSHA-102 in pediatric patients, enabling expansion of the ongoing U.S. REVEAL pediatric trial into the U.K. TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy that utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. Initial dosing in the REVEAL Phase 1/2 pediatric trial took place at RUSH University Medical Center in Chicago under Principal Investigator Elizabeth Berry-Kravis, M.D., Ph.D., Professor of Pediatrics, Neurology and Anatomy/Cell Biology at RUSH University Medical Center. “Dosing the first pediatric patient with Rett syndrome marks an important step forward in our efforts to broaden the clinical evaluation of TSHA-102 to younger patients with earlier stages of Rett syndrome. We are pleased with our progress on expanding the study of TSHA-102 across a broad population of ages and stages of Rett syndrome to bring a potentially transformative treatment option to all patients an...