原始来源记录
决策级顶线结果
Taysha 完成 REVEAL 关键剂量；6 个月主要数据和 FDA 反馈预计 2027 年上半年
REVEAL 关键试验中 17 名患者的给药现已完成，触发 6 个月中期分析，该分析将构成计划中的 BLA 提交基础。A 部分长期数据显示，100% 的患者获得或重新获得 ≥1 项发育里程碑，并在 ≥12 个月内保持持久且不断加深的功能改善。关键 6 个月分析的主要结果及 FDA 对 BLA 路径的反馈预计将于 2027 年上半年公布。
关键事实
来源摘录
EX-99.1 2 d20817dex991.htm EX-99.1 EX-99.1 Exhibit 99.1 Taysha Gene Therapies Announces Completion of Dosing in REVEAL Pivotal Trial and Reports Longer- Term Clinical Data from Part A of REVEAL Phase 1/2 Trials Evaluating TSHA-102 for Rett Syndrome Completed dosing of 17 patients in REVEAL pivotal trial; topline data from 6-month interim analysis and FDA feedback on next steps toward BLA submission pathway expected 1H 2027 TSHA-102 was generally well-tolerated with no treatment-related SAEs or DLTs reported as of the June 2026 data cutoff across REVEAL Phase 1/2 and pivotal trials (N=29) 100% of REVEAL Part A patients (N=12, 6-21 years) gained/regained ≥ one developmental milestone by 12 months post-TSHA-102, with consistent responses across ages and disease severity Longer-term f