Regeneron Pharmaceuticals ((REGN)), Intellia Therapeutics Inc ((NTLA)) announced an update on their ongoing clinical study.
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Study Overview: 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 individuals with hemophilia B, a condition caused by a genetic mutation in the coagulation Factor 9 gene. The primary goal is to determine a safe and well-tolerated dose of the therapy, with secondary objectives including the assessment of drug presence in the blood, antibody formation, and the impact on quality of life and joint health.
Intervention/Treatment: The study tests an experimental gene insertion therapy named REGV131-LNP1265. This CRISPR/Cas9-based treatment is designed to enable the body to produce coagulation factor IX, potentially reducing the need for regular factor replacement therapy.
Study Design: This interventional study is structured in two parts with a non-randomized, sequential intervention model. It is open-label, meaning there is no masking, and its primary purpose is treatment. The study involves dose escalation and confirmation in adults, followed by dose expansion in adolescents and children.
Study Timeline: The study began on September 11, 2024, with the last update submitted on August 21, 2025. These dates are crucial as they indicate the study’s progression and current status, which is actively recruiting participants.
Market Implications: This study update could positively impact Regeneron and Intellia’s stock performance by showcasing their commitment to innovative therapies in the competitive gene therapy market. Successful outcomes could enhance investor confidence and position these companies as leaders in hemophilia treatment, potentially influencing the broader biotechnology sector.
The study is ongoing, with further details available on the ClinicalTrials portal.