Regeneron Pharmaceuticals ((REGN)), Intellia Therapeutics Inc ((NTLA)) announced an update on their ongoing clinical study.
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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 dose and assessing the therapy’s impact on quality of life and joint health.
The intervention involves a CRISPR/Cas9-based gene insertion therapy, REGV131-LNP1265, designed to enable the body to produce clotting factor IX, potentially reducing the need for regular factor replacement therapy.
The study is interventional, non-randomized, and follows a sequential model with no masking. Its primary purpose is treatment, focusing on dose escalation and confirmation in adults, followed by dose expansion in younger age groups.
The study began on September 11, 2024, with an estimated completion date yet to be announced. The latest update was submitted on July 22, 2025, indicating ongoing recruitment.
This study could significantly impact Regeneron and Intellia’s market performance by showcasing the potential of gene therapy in treating genetic disorders. Success could enhance investor confidence and position these companies as leaders in innovative treatments, amidst growing competition in the gene therapy sector.
The study is ongoing, with further details available on the ClinicalTrials portal.