一次情報の記録
重要申請提出
Taysha Gene Therapies:TSHA-102のBLA申請に向けたPPQキャンペーンを開始
Tayshaは2026年4月に商業製造プロセスを用いてTSHA-102のBLA-enabling Process Performance Qualification (PPQ) キャンペーンを開始し、2026年Q4に完了予定。FDAは2026年1月のType CミーティングでPPQキャンペーン戦略を承認した。このCMCマイルストーンは、Rett症候群向けTSHA-102のBLA提出予定タイムラインと製造活動を整合させる。
主要事実
出典の抜粋
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