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Taysha Gene Therapies Announces Initiation of Clinical Development of TSHA-118 for the Treatment of CLN1 Disease
CTA approved by Health Canada in November 2021; IND approved and open in 2019 Queen’s University selected as initial clinical site under the direction of Dr.
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nCTA approved by Health Canada in November 2021; IND approved and open in 2019\n\nQueen’s University selected as initial clinical site under the direction of Dr. Jagdeep Wahlia, principal investigator\n\nPPT1 enzyme activity of 5% or greater normalizes survival and significantly improves clinical phenotype based on natural history data\n\nPreliminary clinical safety and PPT1 enzyme activity data expected in the first half of 2022\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 the initiation of clinical development of TSHA-118 for the treatment of CLN1 disease under a recently approved Clinical Trial Application (CTA). Queen’s University in Ontario, Canada has been selected as the initial clinical site under the direction of Dr. Jagdeep Wahlia. There is also an open investigational new drug application (IND) in the United States for TSHA-118 in CLN1 disease.\n\n“CLN1 disease is caused by mutations in the CLN1 gene, which encodes the soluble lysosomal enzyme palmitoyl-protein thioesterase-1, or PPT1,” said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. “Introduction of a functional CLN1 gene with TSHA-118 treatment offers a potentially effective therapeutic approach that addresses the root cause of the disease. In preclinical models of CLN1, TSHA-118 significantly extended survival and improved behavior. We are highly encouraged by the therapeutic potential of TSHA-118 and expect PPT1 activity of 5% or greater to normalize survival and significantly improve clinical phenotype based on natural history data.”\n\nTSHA-118 is a self-complementary AAV9 gene replacement therapy designed to express a human codon-optimized CLN1 transgene for the treatment of CLN1 disease. The global trial is a single arm, open-label Phase 1/2 trial evaluating TSHA-118 for the treatment of CLN1 disease utilizing commercial grade material. The initial clinical site is Queen’s University under the direction of principal investigator, Jagdeep S. Walia, MBBS, FRCPC, FCCMG, Clinical Geneticist and Associate Pro...