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Taysha Announces Exclusive Option from UTSW to License Worldwide Rights to Clinical-Stage AAV9 Gene Therapy Program for CLN7 Disease, a Research Collaboration with UTSW to Develop Next-Generation Construct for CLN7 and a Grant Award to Batten Hope
CLN7 program currently in Phase 1 clinical proof-of-concept trial with preliminary data anticipated by year-end 2021 Intrathecal dosing of the high dose
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nCLN7 program currently in Phase 1 clinical proof-of-concept trial with preliminary data anticipated by year-end 2021\n\nIntrathecal dosing of the high dose first-generation construct resulted in nearly complete normalization of impaired open field and motor function and more than doubled median life expectancy in MSFSD8 knockout mice; data to be presented at upcoming 17th Annual International Congress on Neuronal Ceroid Lipofuscinosis\n\nTaysha also enters into a research collaboration with UT Southwestern to develop next-generation construct for CLN7 disease, which is expected to improve potency, safety profile, packaging efficiency and manufacturability over first-generation construct\n\nInitiation of a planned pivotal CLN7 clinical trial with next-generation construct anticipated in 2022, with reference to human proof-of-concept data generated from first-generation construct\n\nProvides a grant to Batten Hope, the leading CLN7 patient advocacy group, to support patient awareness, disease education and newborn screening initiatives\n\nEstimated prevalence of CLN7 disease is 4,000 patients worldwide\n\nTaysha expected to have five clinical stage programs by year-end 2021\n\nWebcast today at 8:00 AM Eastern Time\n\n DALLAS--(BUSINESS WIRE)--\nTaysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has obtained 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 efficiency and manufacturability over the first-generation construct. Completion of the next-generation construct design is anticipated by year-end 2021, with commercial-grade GMP material expected in 2022.\n\nThe first-generation construct for the CLN7 program was developed in the laboratory of Steven Gray, Ph.D., Associate Professor at UT Southwestern Medical Center and Chief Scientific Advisor for Tay...