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Taysha Gene Therapies Reports Second Administration of TSHA-102 in the REVEAL Phase 1/2 Adult Trial for Rett Syndrome Treatment

Wednesday, September 27, 2023

Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage biotechnology company specializing in gene therapies for monogenic diseases of the central nervous system (CNS), has reported the dosing of the second patient with TSHA-102 in the REVEAL Phase 1/2 adult trial aimed at treating Rett syndrome in Canada.

Dr. Sukumar Nagendran, President, and Head of R&D at Taysha, highlighted the significance of this achievement and the growing interest within the Rett syndrome community for a potential therapy that could modify the course of the disease. The company remains dedicated to continuing the clinical evaluation of TSHA-102 in adults and expanding its investigation to include pediatric patients afflicted by this devastating condition. Taysha intends to provide updates on the first adult patient in the low-dose cohort and share initial clinical data on the second adult patient during its quarterly earnings conference call in mid-November, following a pre-specified IDMC review.

TSHA-102 is currently under assessment in the REVEAL Phase 1/2 adult trial in Canada, which is an open-label, randomized study designed to assess the safety and preliminary efficacy of TSHA-102 in adult females with Rett syndrome caused by MECP2 loss-of-function mutations. This therapy is administered via a single lumbar intrathecal injection, and dose escalation involves the sequential evaluation of two dose levels of TSHA-102. The maximum tolerated dose (MTD) or maximum administered dose (MAD) established will be used in the dose expansion phase. The enrollment for the low-dose cohort is anticipated to conclude in the fourth quarter of 2023 with the administration of the third patient.

The REVEAL adult trial is being conducted at CHU Sainte-Justine, the Université de Montréal mother and child university hospital center in Montreal, Canada, under the supervision of Dr. Elsa Rossignol, M.D., FRCP, FAAP, who serves as the Principal Investigator.

Furthermore, the United States Food and Drug Administration (FDA) has granted clearance for the Investigational New Drug (IND) application for TSHA-102 in pediatric Rett syndrome patients, and Taysha plans to initiate dosing of the first pediatric patient in the first quarter of 2024. Additionally, the company has submitted a Clinical Trial Application to the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) for TSHA-102 in pediatric Rett syndrome patients and anticipates receiving MHRA feedback in the second half of 2023.

TSHA-102 is an investigational gene transfer therapy that employs self-complementary intrathecal AAV9 delivery for the treatment of Rett syndrome. This therapy incorporates a unique miRNA-Responsive Auto-Regulatory Element (miRARE) platform designed to regulate MECP2 levels in the CNS at the cellular level without the risk of overexpression. TSHA-102 has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, as well as Orphan Drug designation from the European Commission.

Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene, which is essential for neuronal and synaptic function in the brain. This disorder is characterized by intellectual disabilities, communication loss, seizures, developmental regression or slowing, motor and respiratory impairment, and reduced life expectancy. Rett syndrome primarily affects females and ranks among the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that target the genetic root cause of this condition. Pathogenic/likely pathogenic MECP2 mutations are estimated to impact between 15,000 and 20,000 patients in the United States, European Union, and United Kingdom.

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