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周日报道,REGENX比奥强调2026年催化剂,因为RGX-202显示出持久的18个月功能益处,预计FDA PDUFA、BLA备案和多个百分比读数
2026-01-12 16:17
- New Phase I/II RGX-202 functional data demonstrates long-term, durable treatment effect at pivotal dose at 18 months
- Robust patient enrollment in confirmatory trial continues, expect majority of patients to be dosed by planned BLA filing, mid-year
- Expecting FDA PDUFA decision and multiple pivotal top-line data readouts in 2026 to support potential commercial launches 2026-2028
- In-house manufacturing and strategic global partnerships driving commercial readiness
- Presentation at 44th Annual J.P. Morgan Healthcare Conference Wednesday, January 14
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