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DALLAS – (BUSINESS WIRE) – December 22, 2021–

Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centered, pivotal stage gene therapy company focused on the development and commercialization of AAV-based gene therapies for the treatment of monogenic diseases of the nervous system central (CNS) in both cases and large patient populations, today announced preliminary clinical safety data for constructing first generation in CLN7 disease. Preliminary clinical data on efficacy and safety 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). In addition, 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 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 who received a dose of 1.0 x 10 15 vg in total, which is the highest dose ever safely administered intrathecally in l man for gene therapy. The DSMB supported dose escalation from the initial dose of 5.0 x 10 14 to the high dose of 1.0 x 10 15. It is important to note that no major adverse events were observed. We look forward to Dr Ben Greenberg’s presentation of preliminary clinical data on efficacy and safety at the upcoming 18th Annual WORLDSymposium in February, ”said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, 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-generation construct. generation. With human proof of concept clinical data to benchmark, we remain on track to advance next-generation construction in a pivotal trial scheduled for 2022. “

UTSW Continues to Enroll Patients in Investigator-Sponsored Clinical Trial at Children’s Medical Center in Dallas for First Generation AAV9 Gene Replacement Therapy Given Intrathecally for Treatment of Childhood CLN7 Disease . The primary endpoint of the trial is safety and tolerability. Secondary efficacy endpoints include the Clinical Global Impression scales, assessments of neuropsychological and neurodevelopmental progression, ataxia and motor function, and quality of life. UTSW retains a financial interest in Taysha.

CLN7 disease is a rare, fatal, rapidly progressive neurodegenerative disease that is a form of Batten disease. CLN7 is caused by autosomal recessive mutations in the MFSD8 gene that causes lysosomal dysfunction and accumulation of abnormal material in the lysosomes of cells. The onset of the disease occurs around the age of two to five, with death often occurring in young adolescence. Patients experience progressive loss of nerve cells in parts of the brain and typically experience seizures, vision loss, speech disturbances, 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 dedicated to eradicating monogenic CNS disease. By focusing singularly on the development of curative drugs, we aim to rapidly translate our treatments from the laboratory to the bedside. We have combined our team’s proven track record in gene therapy drug development and commercialization with UT Southwestern’s world-class gene therapy program to create an extensive AAV gene therapy pipeline focused on rare and rare indications. at large market. Together, we are leveraging our fully integrated platform, an engine for potential new treatments, with the goal of dramatically improving 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”, “plans” “And” the 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 to alter the course of disease in the patients we seek to treat, our plans of research, development and regulation for 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 distributed and successfully marketed, 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. Therefore, these forward-looking statements are not guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. The risks relating to our business are described in detail in our filings with the Securities and Exchange Commission (“SEC”), including in our annual report on Form 10-K for the 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 website at Additional information will be made available in other documents that we file from time to time with the SEC. These risks may be magnified 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 such statements, except as required by law.

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SOURCE: Taysha Gene Therapies, Inc.

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PUB: 12/22/2021 7:00 a.m. / DISC: 12/22/2021 7:01 a.m.

Copyright Business Wire 2021.