Action required: Please refresh your browser
We have recently implemented some changes that require a hard refresh of your browser: Please hold down the CTRL-key and press the F5 key.
After a successful hard refresh, this message should not appear anymore.
More details about this topic are available here »
Taysha Gene Therapies Announces Updates to TSHA-102 Clinical Program in Rett Syndrome | ||
By: GlobeNewswire - 29 Feb 2024 | Back to overview list |
|
REVEAL adolescent and adult trial will proceed to high dose cohort following Independent Data Monitoring Committee approval of Company’s request to initiate dose escalation; dosing of first patient in cohort two (high dose, n=3) expected in Q2 2024 REVEAL adolescent and adult trial will now expand into the U.S. following submission to FDA (age 12+ protocol) Dosing of second patient in cohort one (low dose) of REVEAL pediatric trial expected in Q1 2024 following Independent Data Monitoring Committee approval Update on available clinical data from completed cohort one (low dose, n=2) in REVEAL adolescent and adult trial expected in Q1 2024; initial data from cohort one in REVEAL pediatric trial expected mid-2024; initial data from cohort two (high dose) in both trials expected in second half of 2024 DALLAS, Feb. 29, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha” or “the Company”), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of severe monogenic diseases of the central nervous system (CNS), today announced updates to its TSHA-102 program in clinical evaluation for the treatment of Rett syndrome. The Independent Data Monitoring Committee (IDMC) approved the Company’s request to proceed to dose escalation in the REVEAL Phase 1/2 adolescent and adult trial, enabling earlier advancement to the high dose cohort of 1x1015 total vg. The IDMC also approved the dosing of the second patient in cohort one (low dose) of 5.7x1014 total vg in the REVEAL Phase 1/2 pediatric trial. The decisions follow IDMC review of available clinical data from the patients dosed with TSHA-102. The Company also announced the expansion of its ongoing REVEAL Phase 1/2 adolescent and adult trial in Canada into the United States (U.S.) following submission of the adolescent and adult trial protocol to the U.S. Food and Drug Administration (FDA). “We have been quite encouraged by the initial safety and efficacy data demonstrated to date with TSHA-102 and are pleased that we are cleared to dose the second patient in our REVEAL Phase 1/2 pediatric trial, and importantly, that the IDMC approved our request to proceed to dose escalation in our REVEAL Phase 1/2 adolescent and adult trial earlier than planned. Advancing to the high dose cohort accelerates our ability to further inform our clinical development and regulatory plan for Part B of the study by at least a quarter,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “Additionally, we believe expanding our ongoing REVEAL adolescent and adult trial in Canada into the U.S. will further support our goal to evaluate TSHA-102 across a broad range of ages and stages of patients with Rett syndrome. We are pleased with the progress we’ve made and remain focused on evaluating TSHA-102 as a potential treatment option for patients and families living with Rett syndrome.” REVEAL Phase 1/2 adolescent and adult trial: a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in adolescent and adult females aged 12 years and older with Rett syndrome due to MECP2 loss-of-function mutation. The trial is taking place in Canada and the U.S. TSHA-102 is administered as a single lumbar intrathecal injection. Dose escalation will evaluate two dose levels of TSHA-102 sequentially. The maximum tolerated dose (MTD) or maximum administered dose (MAD) established in Part A will then be administered during dose expansion in Part B of the study.
REVEAL Phase 1/2 pediatric trial: a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in pediatric females with Rett syndrome due to MECP2 loss-of-function mutation. The trial is taking place in the U.S. and cleared in the U.K. TSHA-102 is administered as a single lumbar intrathecal injection. Part A of the study will focus on determining MAD and MTD in patients aged 5-8 years old. Part B is the dose expansion phase and will evaluate TSHA-102 at the MAD or MTD in two age cohorts (5-8 years and 3-5 years).
About TSHA-102 About Rett Syndrome About Taysha Gene Therapies Forward-Looking Statements Company Contact: Media Contact: |
||
|
||
Copyright 2024 GlobeNewswire | Back to overview list |