原始出處紀錄
重大申請已送件
Taysha Gene Therapies：TSHA-102 BLA 支援性 PPQ 活動已啟動
Taysha 於 2026 年 4 月使用商業製造流程啟動 TSHA-102 的 BLA 支援性 Process Performance Qualification (PPQ) 活動，預計於 2026 年 Q4 完成。FDA 在 2026 年 1 月的 Type C 會議後核准 PPQ 活動策略。此 CMC 里程碑使製造活動與 TSHA-102 在 Rett 症候群的 BLA 提交時程保持一致。
關鍵事實
來源摘錄
EX-99.1 2 d99032dex991.htm EX-99.1 EX-99.1 Exhibit 99.1 Taysha Gene Therapies Reports First Quarter 2026 Financial Results and Provides Corporate Update Reaffirmed FDA alignment on TSHA-102 BLA pathway, including pivotal trial design, endpoints and potential to submit for approval based on REVEAL pivotal trial 6-month interim analysis, following recent breakthrough therapy Type B meeting Further advanced dosing in the REVEAL pivotal trial, with enrollment in the ASPIRE trial ongoing across multiple sites; on track to complete dosing in REVEAL and ASPIRE in Q2 2026 Maintained favorable tolerability profile with no treatment-related SAEs or DLTs in REVEAL Phase 1/2 and REVEAL pivotal trials as of May 2026 data cutoff; plan to present longer-term safety and efficacy data from Part A of R