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Taysha Gene Therapies Receives Rare Pediatric Disease and Orphan Drug Designations for TSHA-103 for the Treatment of Epilepsy Caused by SLC6A1 Haploinsufficiency

DALLAS–(BUSINESS WIRE)–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”

“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 conditions, including approval prior to December 11, 2022 unless changed by legislation. If issued, a sponsor may redeem a priority review voucher for priority review of a subsequent marketing application for a different product candidate, or the priority review voucher could be sold or transferred to another sponsor.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to investigational treatments that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the United States. The program was developed to encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset some development costs as well as eligibility for market exclusivity for seven years post approval.

About Taysha Gene Therapies

Taysha 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 to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients’ lives. More information is available at

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates, including TSHA-103, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential benefits of rare pediatric disease designation and orphan drug designation to our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management’s current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (“SEC”) filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SEC’s website at Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.


Company Contact:
Kimberly Lee, D.O.
SVP, Corporate Communications and Investor Relations
Taysha Gene Therapies

Media Contact:
Carolyn Hawley
Canale Communications

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