TSHA Taysha Gene Therapies Inc

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 it will present clinical data on its TSHA-102 program in evaluation for Rett syndrome during an oral poster presentation at the upcoming British Paediatric Neurology Association (BPNA) 2024 Annual Conference, taking place in Bristol, England from January 24-26, 2024.

The presentation will include previously disclosed clinical data from the first two adult patients with stage four Rett syndrome dosed with TSHA-102 in the REVEAL Phase 1/2 adolescent and adult trial in Canada. The Company expects to provide further updates on available clinical data from cohort one (low dose) in the REVEAL adolescent and adult trial in the first quarter of 2024.

Oral poster presentation details are as follows:

Abstract Title: Early safety and efficacy observations following the first use of TSHA-102 gene therapy in patients with Rett Syndrome

Presenter: Meredith Schultz, M.D., M.S., Senior Vice President of Clinical Development and Medical Affairs at Taysha Gene Therapies

Oral Presentation Session Date/Time: Thursday, January 25 at 12:05-12:40 PM GMT

Location: Screen C - Wallace Suite

Poster Number: 056

Additional details on the meeting can be found at the BPNA 2024 Annual Congress website.

About TSHA-102TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. TSHA-102 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. TSHA-102 has received Fast Track designation and Orphan Drug and Rare Pediatric Disease designations from the FDA and has been granted Orphan Drug designation from the European Commission.

About Rett SyndromeRett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene, which is a gene that’s essential for neuronal and synaptic function in the brain. The disorder is characterized by intellectual disabilities, loss of communication, seizures, slowing and/or regression of development, motor and respiratory impairment, and shortened life expectancy. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU and U.K.

About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team’s proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program. Together, we leverage our fully integrated platform with a goal of dramatically improving patients’ lives. More information is available at www.tayshagtx.com.

Company Contact:Hayleigh CollinsDirector, Head of Corporate Communications and Investor RelationsTaysha Gene Therapies, Inc.hcollins@tayshagtx.com

Media Contact:Carolyn HawleyCanale Communicationscarolyn.hawley@canalecomm.com