" class="no-js "lang="en-US"> Taysha Gene Therapies Starts Trial of Rett Syndrome Treatment
Wednesday, April 10, 2024

Taysha Gene Therapies Announces First Patient Dosed with TSHA-102 in the REVEAL Phase 1/2 Trial for the Treatment of Rett Syndrome

Taysha Gene Therapies, 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), has 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 syndrome due to MECP2 loss-of-function mutation. Participants will receive a single lumbar intrathecal injection of TSHA-102. Dose escalation will evaluate two dose levels of TSHA-102 sequentially, with an initial dose of 5×1014 total vector genomes (vg) and the second dose of 1×1015 vg. The maximum tolerated dose (MTD) or maximum administered dose (MAD) established will then be administered during dose expansion. Per the protocol, an independent data monitoring committee will review available safety data from the first patient at approximately six weeks post-dosing to determine if the Company can proceed with dosing the second patient. Initial available clinical safety data will be reported at Taysha’s upcoming R&D Day on June 28, 2023. To register for the event, please click here.

Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor Neuroscience and Pediatrics, and Principal Investigator of the REVEAL study added, “Based on its unique and compelling technology targeting the genetic root cause of Rett syndrome, TSHA-102 has the potential to transform care by addressing a significant unmet medical need for patients with this devastating and currently incurable disease. The dosing of the first patient in this important clinical trial represents a critical advancement in evaluating the potential of gene therapy for Rett syndrome. It is a privilege to be part of this important endeavor. In the name of all affected families, I thank Taysha for bringing this potentially transformative therapy from the bench to the bedside.”

Sabrina Millson, President of Ontario Rett Syndrome Association further added, “This is a momentous day for the Rett syndrome community. As a mom to a daughter living with Rett syndrome and the president of the Ontario Rett Syndrome Association here in Canada, I know first-hand how this disease leads to debilitating symptoms, including difficulties in communication, mobility and breathing. The potential for a treatment that addresses the underlying cause of disease and slows progression or potentially prevents the onset of disease with early intervention is truly remarkable. We’re pleased to collaborate with Taysha Gene Therapies in an effort to bring a gene therapy treatment that could meaningfully change the lives of patients and their caregivers.”

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