A groundbreaking gene therapy for Hemophilia B, known as REGV131-LNP1265, is currently in clinical trials, aiming to enable patients' bodies to produce clotting factors independently and potentially reduce the need for regular factor replacement treatments.

The study, which began on September 11, 2024, and is actively progressing as of August 21, 2025, involves multiple phases of participant recruitment and is structured to assess safety, dosing, and effectiveness.

Developed by Regeneron Pharmaceuticals and Intellia Therapeutics, this innovative approach uses CRISPR/Cas9 technology to insert a functional copy of the coagulation Factor IX gene, marking a significant advancement in gene editing for hemophilia.

The trial is divided into two parts: an initial dose escalation and confirmation in adults, followed by a dose expansion in adolescents and children, with ongoing recruitment of participants.

Successful results from this study could position Regeneron and Intellia as leaders in gene therapy for hemophilia B, potentially boosting their stock performance and influencing investor confidence in the biotech sector.

The non-randomized, sequential trial continues to recruit participants as of August 2025, with no announced completion date yet.

The intervention aims to enable the body to produce coagulation factor IX, which could significantly reduce the reliance on regular factor replacement therapy for patients.

The study's primary goals include evaluating the safety and tolerability of the therapy, with secondary objectives such as measuring drug presence, immune response, and improvements in patients' quality of life and joint health.