Exhibit 99.1
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
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, January 10, 2024 – 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 and families suffering from this devastating disease,” said Sukumar Nagendran, M.D., President, and Head of R&D of Taysha. “The
pediatric trial will build on our ongoing REVEAL adolescent and adult trial, where early data demonstrated improvements across multiple clinical domains in adult patients with the most advanced stage of disease. We also plan to expand our U.S.
pediatric trial into the U.K. following the recent acceptance of our CTA by the MHRA.”
Elizabeth Berry-Kravis, M.D., Ph.D., Professor of Pediatrics,
Neurology and Anatomy/Cell Biology, and Principal Investigator of the REVEAL pediatric trial added, “Designed as a one-time, disease-modifying treatment with the ability to mediate MECP2 expression
on a cell-by-cell basis, TSHA-102 holds the potential to address a significant unmet medical need for the Rett syndrome
community. By intervening early in disease, we believe TSHA-102 may provide significant therapeutic impact for pediatric patients. I look forward to evaluating this promising gene therapy and its impact
on the lives of patients and their caregivers in the clinic.”