火箭制药公司撤回了美国Fanconi贫血病基因治疗申请,将重点放在心血管方案和更明确的监管路径上。 Rocket Pharmaceuticals withdrew its U.S. gene therapy application for Fanconi anemia to focus on cardiovascular programs and clearer regulatory paths.

火箭制药公司已自愿撤回其美国生物许可证申请RP-L102,这是Fanconi贫血症的基因疗法,并列举了将重点放在心血管方案的战略转变和更明确的监管途径。 Rocket Pharmaceuticals has voluntarily withdrawn its U.S. Biologics License Application for RP-L102, a gene therapy for Fanconi anemia, citing strategic shifts to focus on cardiovascular programs and clearer regulatory paths. 该决定基于商业考虑,而不是基于安全或效能,遵循了该疗法有希望的第二阶段成果,并事先撤回了欧洲应用。 The decision, based on business considerations rather than safety or efficacy, follows the therapy's promising Phase 2 results and prior withdrawal of its European application. 该公司停止了内部发展,解雇了约30%的工作人员,在FDA停机后恢复试验后,现在将达农病治疗(RP-A501)列为优先事项。 The company ceased internal development, laid off about 30% of staff, and is now prioritizing its Danon disease therapy, RP-A501, after resuming trials post-FDA hold lift.