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DALLAS–(BUSINESS WIRE)–Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on the development and commercialization of AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in rare and important patient populations, today announced preliminary clinical data on the safety of the first-generation construct in CLN7 disease. Preliminary clinical efficacy and safety data will be presented at the 18th Annual WORLDSymposium in February 2022 by Dr. Ben Greenberg, Vice President of Clinical and Translational Research and Professor in the Departments of Neurology and Pediatrics at the UT Southwestern (UTSW). Additionally, UTSW has completed the design of a next-generation construct, which is expected to further improve potency, packaging efficiency, and manufacturability, as well as reduce the risk of immunogenicity compared to the first-generation construct. .

“We are very pleased that the first generation construct was well tolerated and that preliminary data from the ongoing clinical trial supports a favorable safety profile. To date, three patients with CLN7 disease have been treated, including two patients receiving a 1.0×10 dose15 vg total, which is the highest dose ever safely administered intrathecally in humans for gene therapy. DSMB supported dose escalation from 5.0×1014 initial dose at 1.0×1015 high dose. It is important to note that no major adverse events were observed. We look forward to Dr. Ben Greenberg’s presentation of preliminary clinical efficacy and safety data at the next 18and Annual Global Symposium in February,” said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, SWOT, Chief Medical Officer and Head of Research and Development at Taysha. “In addition, UTSW has finalized the design of a next-generation CLN7 construct, which is expected to improve potency, packaging efficiency and manufacturability, as well as reduce the risk of immunogenicity compared to the first-in-class construct. generation. With human proof-of-concept clinical data at baseline, we remain on track to advance the next-generation construct into a pivotal trial scheduled for 2022.”

UTSW continues to enroll patients in the investigator-sponsored clinical trial at Children’s Medical Center Dallas for first-generation intrathecal-dose AAV9-based gene replacement therapy for the treatment of childhood CLN7 disease. The primary endpoint of the trial is safety and tolerability. Secondary efficacy endpoints include clinical global impression scales, assessments of neuropsychological and neurodevelopmental progression, ataxia and motor function, and quality of life. UTSW maintains a financial interest in Taysha.

CLN7 disease is a rare, fatal and rapidly progressive neurodegenerative disease that is a form of Batten disease. CLN7 is caused by autosomal recessive mutations in MFSD8 gene that lead to lysosomal dysfunction and the accumulation of abnormal material in the lysosomes of cells. The disease begins around the age of two to five, with death often occurring in the early teens. Patients experience progressive loss of nerve cells in parts of the brain and typically experience seizures, loss of vision, speech impairment, and mental and motor regression. Currently, there are no approved therapies to treat CLN7 disease, which affects approximately 4,000 patients worldwide.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a particular focus on the development of curative drugs, 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 rare and large-scale indications. . Together, we are leveraging our fully integrated platform – a powerhouse for potential new treatments – to dramatically improve the lives of patients. 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 regarding the potential of our product candidates, including the CLN7 program, to positively impact quality of life and alter disease course in the patients we seek to treat, our plans research, development and regulatory frameworks for our product candidates, the possibility that these product candidates will receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be distributed and commercialized successfully, and the potential market opportunity for these product candidates. 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 are not guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks relating to our business are described in detail in our filings with the Securities and Exchange Commission (“SEC”), including our Annual Report on Form 10-K for the fiscal year ended December 31, 2020, and our report quarterly on Form 10-Q for the quarter ended September 30, 2021, both available on the SEC’s website at Additional information will be made available in other documents filed from time to time with the SEC. These 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 assume no obligation to update these statements except as required by law.