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 individuals with hemophilia B, focusing on finding a safe dose and assessing the therapy’s impact on quality of life, joint health, and the need for factor replacement therapy.
The intervention being tested is a CRISPR/Cas9-based gene insertion therapy, known as REGV131-LNP1265, designed to enable the body to produce clotting factor IX, potentially reducing the need for regular factor replacement therapy.
The study is structured as a non-randomized, sequential interventional trial with no masking, primarily aimed at treatment. It involves dose escalation and confirmation in adults, followed by dose expansion to include adolescents and children.
The study began on September 11, 2024, with the latest update submitted on August 12, 2025. These dates are crucial for tracking the study’s progress and anticipating future updates.
This clinical study could significantly impact Regeneron and Intellia’s stock performance by showcasing their capabilities in gene therapy. Positive outcomes may enhance investor confidence and position these companies as leaders in hemophilia treatment, potentially affecting competitors in the gene therapy and hemophilia markets.
The study is ongoing, with further details available on the ClinicalTrials portal.