Exhibit 99.1
Taysha Gene Therapies Receives Rare Pediatric Disease and Orphan Drug Designations for TSHA-103 for the Treatment of Epilepsy Caused by SLC6A1 Haploinsufficiency
Designations provide validation of encouraging preclinical data generated to date
TSHA-103 joins portfolio of rare pediatric disease and orphan drug designated product candidates including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome, TSH-104 for SURF1-associated Leigh syndrome and TSHA-118 for CLN1
Dallas – December 3, 2020 - Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received both rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for TSHA-103, an AAV-9-based gene therapy in development for SLC6A1-related epilepsy.
“We are pleased by the FDA’s acknowledgement of the imperative need to develop therapies for such a severe and life-threatening condition,” said RA Session II, President, Founder and CEO of Taysha. “We are encouraged by the early evidence of our gene therapy approach to potentially treat this devastating disease. These designations in now five programs underscore the critical nature of our work and add momentum for these programs. We remain committed to advancing our pipeline of innovative and potentially transformative product candidates as we aim to eradicate monogenic CNS disease.”
SLC6A1 epilepsy is an autosomal dominant genetic disorder characterized by the loss of function of one copy of the SLC6A1 gene, with clinical manifestations of seizures, epilepsy, language impairment and intellectual disability.
“Haploinsufficiency in the SLC6A1 gene has been identified as a cause of genetic epilepsy, yet there remains a lack of approved disease-modifying therapies,” said Steven Gray, Ph.D., Chief Scientific Advisor at Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. “The designations highlight the innovation of TSHA-103 and the importance of developing a treatment for patients living with this devastating disease.”
“As a mother of a child affected by SLC6A1, Taysha’s dedication to developing a treatment for this community is greatly applauded,” said Amber Freed, Founder of SLC6A1 Connect. “We are delighted that the FDA recognizes the unmet medical need and the role that TSHA-103 may play.”
The FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain
