原始来源记录
决策级顶线结果
Beam Therapeutics：BEAM-302 AATD 顶线数据；关键队列 2026 年下半年
BEAM-302 在 AATD 中的更新 1/2 期顶线数据显示出强劲的 AAT 升高和耐受性良好的特征，导致 60 mg 被选为最佳生物剂量。FDA 反馈支持加速批准途径，计划于 2026 年下半年启动约 50 名患者的 pivotal 队列，以支持未来的 BLA。
关键事实
来源摘录
EX-99.1 2 beam-ex99_1.htm EX-99.1 EX-99.1 Exhibit 99.1 Beam Therapeutics Reports First Quarter 2026 Financial Results and Recent Business Updates Recent BEAM-302 Topline Data in Alpha-1 Antitrypsin Deficiency (AATD) Demonstrate Strong Single-dose Safety and Efficacy Profile, with 60 mg Selected as Optimal Biological Dose; Global Pivotal Cohort Expected to Initiate in Second Half of 2026 Data from Phase 1/2 BEACON Clinical Trial of Risto-cel in Sickle Cell Disease Published in April 1 Issue of the New England Journal of Medicine; U.S. Biologics License Application (BLA) Submission Expected as Early as Year-End 2026 Investigational New Drug (IND) Application for BEAM-304 in PKU and Data from BEAM-301 in GSDIa Anticipated in 2026 Ended First Quarter 2026 with $1.2 Billion in Cash, Cash