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Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal stage gene therapy company focused on the development and commercialization of AAV-based gene therapies for the treatment of monogenic system disorders central nervous system (CNS) in both cases and large patient populations, today announced that it has been granted an exclusive option from UT Southwestern (UTSW) to license worldwide rights to a clinical stage AAV9 gene therapy replacement program for the treatment of CLN7 disease. The company has also entered into a research collaboration with UTSW to develop a next-generation construct for the treatment of CLN7 disease, which is expected to improve potency, safety profile, packaging efficacy and manufacturability compared to to first generation construction. Next-generation construction design is expected to be completed by the end of 2021, with commercial grade GMP materials expected in 2022.

The first generation construction of the CLN7 program was developed in the lab of Steven Gray, Ph.D., Associate Professor at UT Southwestern Medical Center and Chief Scientific Advisor to Taysha, with financial support from Mila’s Miracle and Batten Hope , the main CLN7 patient advocacy groups. The CLN7 program is currently in a Phase 1 proof-of-concept clinical trial conducted by UTSW, and Taysha expects preliminary clinical data for human proof-of-concept and construction efficacy. genes are available by the end of 2021. Taysha intends to launch a planned pivotal trial using a next-generation construct in 2022, with reference to human proof-of-concept clinical data generated from first generation construction.

Additionally, Taysha provided a grant to Batten Hope to support patient awareness, disease education and newborn screening initiatives.

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. 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.

Preclinical data in rodents supported the advancement of the first generation construct in a Phase 1 clinical proof of concept study in patients with CLN7 disease. In a in vivo efficacy study, intrathecal (IT) administration of the first generation construct to MFSD8 knockout mice with high or low doses resulted in clear age and dose effects with early intervention and dose high to obtain the best therapeutic benefits. A high IT dose of the first generation construct in younger knockout mice resulted in: 1) generalized expression of MFSD8 mRNA in all tissues tested; 2) almost complete normalization of altered free field and rotarod performance at 6 and 9 months after injection; 3) more than doubled the median life expectancy (16.82 months versus 7.77 months in untreated knockout mice); and 4) maintaining a healthy body weight for an extended period. Toxicological studies in wild-type rodents have demonstrated the safety and tolerability of computer administration of the first generation construct. These preclinical data will be presented by Xin Chen, Ph.D., assistant professor, Department of Pediatrics at UT Southwestern, during an oral presentation at the 17th Annual International Ceroid Neuronal Lipofuscinosis Congress on October 8, 2021.

“The CLN7 program is a strategic addition to our gene therapy pipeline focused on monogenic CNS diseases. Encouraging preclinical data generated in relevant rodent models suggests that the first generation construct has the potential to reduce overall disease pathology, preserve motor function, and ultimately prolong survival, ”said RA Session II, President, Founder and CEO of Taysha. “The first generation construct is currently in a Phase 1 proof of concept clinical trial with two patients treated to date, and we look forward to the availability of preliminary data by the end of the year. . With human proof-of-concept clinical data to benchmark, we plan to advance a next-generation construct in a pivotal trial scheduled for 2022, which is expected to improve potency, safety, packaging efficacy and manufacturability by compared to the first generation construction. Importantly, we are also pleased to announce our grant to Batten Hope, the leading nonprofit patient advocacy organization for CLN7 disease, to support patient awareness, disease education and screening initiatives. neonatal. We believe that a gene therapy approach has the potential to address a significant unmet need in approximately 4,000 patients worldwide.

Gina Hann, Founder, President and Treasurer of Batten Hope, added, “Our mission is to support families with children with end-stage and rapidly progressive neurodegenerative diseases like CLN7. We are honored to receive Taysha’s support to raise awareness, increase newborn screening, and help patients access potentially life-changing treatments that offer hope and therapeutic advancements for conditions with significant unmet needs.

UTSW is currently enrolling patients in a researcher-sponsored open-concept Phase 1 clinical trial at Children’s Hospital in Dallas for AAV9-based gene replacement therapy administered intrathecally for treatment of childhood disease CLN7. The primary endpoint of the trial is safety and tolerability based on the incidence and severity of serious treatment-related adverse events. Secondary efficacy endpoints include overall clinical impression, neuropsychological, ataxic and motor assessments, and quality of life. The rationale for the design and a discussion of the outcome measures of this clinical trial will be presented in poster form at the upcoming 17th Annual International Congress on Neuronal Ceroid Lipofuscinosis. To date, one patient has received a dose of 5×1014 total vg and a second patient has received a dose of 1×1015 total vg as measured by the qPCR method. UTSW continues to enroll patients in this Phase 1 study at 1 x 1015 yd total and plans to assay additional patients in the short term. Preliminary safety and efficacy data are expected by the end of 2021.

With the addition of the CLN7 program, Taysha expects to have five clinical stage programs by the end of the year. As such, the TSHA-104 program for the treatment of Leigh’s syndrome associated with SURF1 will pass to company collaborators at UTSW to complete studies enabling IND, followed by an investigator-planned clinical trial. by the end of 2022. Taysha will continue to support the SURF1 natural history study in partnership with UTSW.

Financial terms of the agreements were not disclosed.

Webcast information

Taysha management will host a webcast today at 8:00 am ET / 7:00 am CT to discuss today’s news. To participate, please access the following link: The live webcast and replay can also be viewed by visiting Taysha’s website at An archived version of the webcast will be available on the website for 60 days.

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’s world-class Southwestern Gene Therapy program to create an extensive AAV gene therapy pipeline focused on rare and very expensive indications. 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 “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements regarding the potential of our product candidates, as well as the CLN7 program, to have a positive impact on quality of life and to alter the course of disease in the patients we seek to treat, our expectations regarding the benefits of a next generation build for CLN7, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or regulatory agencies foreign equivalents, and whether, if approved, these product candidates will be successfully distributed and 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 these 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 June 30, 2021, both available on the SEC website at Additional information will be available in other documents filed 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.

Company details :

Kimberly Lee, DO

Senior Vice-President, Corporate Communications and Investor Relations

Taysha’s gene therapies

[email protected]

Media contact:

Carolyn hawley

Canale Communications

[email protected]

Source: Taysha Gene Therapies

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