一次情報の記録
決定級申請提出
Beam Therapeutics、risto-celのBLA申請を2026年末までに予定
8-Kは、同社が2026年12月までに鎌状赤血球症に対するristo-celの米国BLAを提出する予定であることを確認し、Sixth Streetとの5億ドルの非希薄化クレジットファシリティにより、2029年半ばまでのキャッシュランウェイを延長し、予想される商業的発売を資金支援する。
主要事実
出典の抜粋
EX-99.1 2 beam-ex99_1.htm EX-99.1 EX-99.1 Exhibit 99.1 Beam Therapeutics Reports Fourth Quarter and Year-End 2025 Financial Results and Announces New Liver-Targeted Genetic Disease Program in Phenylketonuria (PKU) New Program Designed as Platform-based Approach for Direct Correction of Mutations Causing PKU; Investigational New Drug (IND) Filing for BEAM‑304 Anticipated in 2026 Updated Phase 1/2 Data and Next Steps for Pivotal Development for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) on Track for Q1 2026 Strategic Financing Agreement with Sixth Street Provides up to $500 Million in Long-term, Non-dilutive Capital to Fund Anticipated Launch of Risto-cel in Sickle Cell Disease (SCD); U.S. Biologics License Application (BLA) Submission Expected as Early as Year-End 2026 Expected Ca