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Open-label phase 1/2 study of REGV131-LNP1265 gene insertion therapy in adults with hemophilia B
2023-507260-40-00Phase 2Authorised
- Sponsor
- Regeneron Pharmaceuticals
- Start
- Apr 2024
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Small Molecules, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Countries
- 9
01
Overview
Phase 2, open-label trial of REGV131 and LNP1265 in hemophilia B (HB) (4 arms), sponsored by Regeneron Pharmaceuticals. Primary endpoints: Occurrence and severity of treatment-emergent adverse events, Factor IX functional activity and 3 more. Planned start Apr 2024; status: authorised.
02
Drugs
REGV131LNP1265
03
Conditions
hemophilia B (HB)
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