Regeneron ex vivo CRISPR Factor IX gene editing therapy

Regeneron ex vivo CRISPR Factor IX gene editing therapy is an experimental gene-edited cell therapy being developed by Regeneron for the treatment of Hemophilia B. The program utilizes CRISPR/Cas9 technology to perform ex vivo genetic modification of patient-derived cells to restore the production of functional Coagulation Factor IX (FIX). Hemophilia B is a rare genetic bleeding disorder caused by mutations in the F9 gene, leading to deficient clotting factor activity. While Regeneron and its partner Intellia Therapeutics are also advancing an in vivo CRISPR therapy (REGV131), this ex vivo approach involves the extraction, modification, and subsequent re-infusion of cells to provide a durable therapeutic effect. As of 2024, Regeneron is the lead developer of the Hemophilia B program, with Intellia Therapeutics remaining eligible for future milestones and royalties after opting out of co-development.

Other names

Regeneron-Factor IX-ex vivo CRISPR gene editing-gene editing-Hemophilia BRegeneron Hemophilia B ex vivo programIntellia Hemophilia B ex vivo program