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Taysha Gene Therapies Announces Initiation of Clinical Development of TSHA-102 in Rett Syndrome
Clinical Trial Application (CTA) approved by Health Canada in March 2022 TSHA-102, which utilizes novel miRARE platform to regulate transgene expression
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nClinical Trial Application (CTA) approved by Health Canada in March 2022\n\nTSHA-102, which utilizes novel miRARE platform to regulate transgene expression genotypically on a cell-by-cell basis, is the first-and-only gene therapy in clinical development for Rett syndrome\n\nTSHA-102 improved survival, respiratory and motor function assessments in mouse models of Rett syndrome; biodistribution in non-human primates (NHP) supports miRARE mechanism of action; six-month NHP GLP toxicology data support the favorable safety and tolerability profile of TSHA-102\n\nPreliminary Phase 1/2 clinical data for TSHA-102 expected by year-end 2022\n\n DALLAS--(BUSINESS WIRE)--\n\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-102 for the treatment of Rett syndrome under a recently approved Clinical Trial Application (CTA) by Health Canada. Sainte-Justine Mother and Child University Hospital Center in Montreal, Quebec, Canada has been selected as the initial clinical site under the direction of Dr. Elsa Rossignol, principal investigator. The company also announced positive preclinical data from IND/CTA-enabling studies including a pharmacology study in the Rett knockout mouse model (n=252) that assessed the efficacy of TSHA-102 and a Good Laboratory Practices (GLP) toxicology study of TSHA-102 in nonhuman primates (NHPs) (n=24) that explored biodistribution and mechanism of action, both of which supported authorization of the CTA.\n\n“Initiation of clinical development is a significant milestone for the TSHA-102 program and Rett syndrome community,” said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. “Treating Rett syndrome by gene replacement therapy requires an approach that can safely regulate transgene expression in a genotypic manner on a cell-by-cell basis without causing deleterious effects associated with overexpression. TSHA-102’s robust preclinical data package supports and validates the safe and controlled regulation of transgene expression using miRARE, a novel miRNA-...