Regeneron and Intellia’s Gene Therapy Study for Hemophilia B: A Potential Game Changer

Regeneron Pharmaceuticals ((REGN)), Intellia Therapeutics Inc ((NTLA)) announced an update on their ongoing clinical study.

Regeneron Pharmaceuticals and Intellia Therapeutics are conducting a study titled A Two-Part Open-Label Study of REGV131-LNP1265, A CRISPR/Cas9 Based Coagulation Factor IX Gene Insertion Therapy in Participants With Hemophilia B. The study aims to evaluate the safety and effectiveness of a gene insertion therapy for hemophilia B, focusing on finding a safe and well-tolerated dose while assessing various health impacts on participants.

The intervention being tested is a CRISPR/Cas9-based gene insertion therapy named REGV131-LNP1265. It is designed to enable the body to produce clotting factors independently, potentially reducing the need for regular factor replacement therapy.

This interventional study is non-randomized and follows a sequential model without masking. It primarily aims to treat hemophilia B by determining the recommended dose for expansion through dose escalation and confirmation in adults, followed by dose expansion in younger participants.

The study began on September 11, 2024, with an estimated completion date yet to be announced. The last update was submitted on August 12, 2025, indicating ongoing recruitment and progress.

This study could significantly impact Regeneron and Intellia’s stock performance by showcasing their innovative approach to gene therapy. Success in this study may enhance investor sentiment and position these companies as leaders in the hemophilia treatment market, potentially influencing competitors in the biopharmaceutical industry.

The study is ongoing, with further details available on the ClinicalTrials portal.