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REGV131
- Development stage
- Phase 2
- Lead developer
- Regeneron Pharmaceuticals
- Modality
- Small Molecules, CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
REGV131 is an investigational gene therapy for Hemophilia B that uses a CRISPR/Cas9-based approach to insert a functional human Factor IX (F9) gene into the patient's genome. The therapy consists of two components—REGV131 and LNP1265—which are administered sequentially. This treatment aims to enable the patient’s liver cells to produce clotting factor IX endogenously, potentially providing a long-term or lifelong solution for individuals with Hemophilia B who have mutations in their F9 gene. The therapy is delivered via intravenous infusion and is currently being evaluated in Phase 1/2 clinical trials for safety and efficacy in adults, adolescents, and children with Hemophilia B. Developed by Regeneron Pharmaceuticals and Intellia Therapeutics.
- Brand names
- REGV131-LNP1265REGV-131-LNP1265REGV 131-LNP1265
- Other names
- REGV131-LNP1265REGV-131-LNP1265REGV 131-LNP1265
02
Targets
F9 (Coagulation Factor IX)
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