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Taysha Gene Therapies Announces First Patient Dosed with TSHA-102 in the REVEAL Phase 1/2 Trial Under Investigation for the Treatment of Rett Syndrome
The Phase 1/2 REVEAL trial is a first-in-human, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"The Phase 1/2 REVEAL trial is a first-in-human, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in adults with Rett syndrome TSHA-102 utilizes novel miRARE technology, designed to regulate cellular MECP2 levels Initial available clinical safety data from Phase 1/2 REVEAL trial will be reported at Taysha’s upcoming R&D Day on June 28, 2023, at 10:00 AM Eastern Time DALLAS, June 05, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-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), today announced that the first patient has been dosed with TSHA-102 in the Phase 1/2 REVEAL trial evaluating the safety and preliminary efficacy of TSHA-102 in adult patients with Rett syndrome. TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy that utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform designed to regulate cellular MECP2 expression. The study is being conducted at CHU Sainte-Justine, the Université de Montréal mother and child university hospital centre in Montreal, Canada. “Dosing of the first adult patient marks the beginning of clinical evaluation of TSHA-102 in the Phase 1/2 REVEAL trial, and, to our knowledge, the first time a gene therapy has ever been evaluated in a clinical setting for the treatment of Rett syndrome,” said Sukumar Nagendran, M.D., President, and Head of R&D. “By targeting the regulation of gene expression on a cell-by-cell basis, we believe our miRARE technology has the ability to enable safe expression of MECP2, which may help address the risks associated with both under and overexpression resulting from the mosaic pattern of MECP2 silencing. This is a significant milestone that furthers our quest to bring a potentially transformational gene therapy to patients and families living with Rett syndrome. We look forward to sharing initial available clinical safety data from the Phase 1/2 REVEAL trial at our R&D Day on June 28, 2023.” The Phase 1/2 REVEAL trial is a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in adult females with Rett syndr...