原始来源记录
重大申请已提交
Taysha Gene Therapies：TSHA-102 BLA支持性PPQ活动已启动
Taysha于2026年4月启动了TSHA-102的BLA支持性工艺性能确认（PPQ）活动，采用商业化生产工艺，预计2026年第四季度完成。FDA在2026年1月的C类会议后认可了PPQ活动策略。该CMC里程碑将生产活动与TSHA-102在Rett综合征中的计划BLA提交时间表保持一致。
关键事实
来源摘录
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