Regeneron Pharmaceuticals ((REGN)), Intellia Therapeutics Inc ((NTLA)) announced an update on their ongoing clinical study.
Elevate Your Investing Strategy:
- Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence.
- Make smarter investment decisions with TipRanks' Smart Investor Picks, delivered to your inbox every week.
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 without regular replacement therapy. This approach could significantly improve the quality of life for patients with this genetic disorder.
The intervention being tested is a CRISPR/Cas9-based gene therapy, specifically REGV131-LNP1265. This therapy involves inserting a gene into the patient’s DNA to facilitate long-term production of the necessary clotting factor, potentially reducing or eliminating the need for ongoing factor replacement treatments.
The study employs a non-randomized, sequential intervention model with no masking, focusing on treatment as its primary purpose. It includes a two-part adaptive design: Part 1 involves dose escalation and confirmation in adults, while Part 2 expands to include adolescents and children, adjusting doses based on weight.
Key dates for this study include an actual start date of September 11, 2024, with the latest update submitted on July 14, 2025. These dates are crucial for tracking the progress and potential upcoming milestones of the study.
The market implications of this study are significant for Regeneron and Intellia, as successful results could enhance their positions in the gene therapy market, potentially boosting investor confidence and stock performance. This study also positions them competitively against other companies in the hemophilia treatment space.
The study is ongoing, with further details available on the ClinicalTrials portal.