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 designed to treat hemophilia B by enabling the body to produce clotting factor IX naturally, potentially reducing the need for regular factor replacement therapy.
The intervention involves the use of two drugs, REGV131 and LNP1265, administered via intravenous infusion. This experimental therapy uses CRISPR/Cas9 technology to insert a functional gene into the patient’s DNA, aiming to provide a long-term solution for hemophilia B.
The study employs a non-randomized, sequential intervention model with no masking, primarily focusing on treatment. It is structured in two parts: dose escalation and confirmation in adults, followed by dose expansion to include adolescents and children.
The study began on September 11, 2024, with the last update submitted on July 14, 2025. These dates are crucial as they mark the progression and current status of the study, which is still recruiting participants.
This study could significantly impact Regeneron and Intellia’s market positions by potentially offering a groundbreaking treatment for hemophilia B. Positive results may boost investor confidence and stock performance, especially given the competitive landscape in gene therapy.
The study is ongoing, and further details can be found on the ClinicalTrials portal.