BEYOND-9 hemophilia B gene therapy clinical trial launched in four Canadian study sites
Montreal, February 5, 2026 – Four Canadian bleeding disorder treatment centres are participating in the Phase 1 clinical trial of Regeneron’s CRISPR/Cas9-based targeted gene insertion therapy for hemophilia B. The trial is entitled “A Two-Part Open-Label Study of REGV131-LNP1265, A CRISPR/Cas9 Based Coagulation Factor IX Gene Insertion Therapy in Participants with Hemophilia B” and is registered as NCT06379789.
The centres are the McGill University Health Centre (Montreal Children’s Hospital), The Ottawa Hospital (Ottawa Regional Adult Bleeding Disorders Program), Hamilton Health Sciences Centre, and the University of Alberta Hospital (Northern Alberta Bleeding Disorders Clinic).
REGV131-LNP1265 uses lipid nanoparticles and AAV8 to insert functional DNA to the liver.
The CRISPR technology has two potential advantages over the first-generation hemophilia B gene therapy currently approved and marketed in many countries, which is based on gene addition. Because the factor IX gene is inserted into the genome …
- the factor IX expression has the potential to be permanent, and not decrease over time, as is possible with gene addition.
- this gene therapy could potentially be used in children, as the effect will not be diluted as the cells in a child’s growing liver cells divide.
Research in mice and non-human primates has shown robust and stable expression of factor IX.
Eligibility criteria for Phase 1 of the study include:
- confirmed diagnosis of severe or moderately severe hemophilia B with medical history of FIX functional activity ≤2% or documented genotype known to produce severe hemophilia B;
- currently taking FIX prophylaxis and previous experience with FIX therapy;
- 18 years of age and older.
Those interested in the clinical trial should speak with their bleeding disorder treatment centre team or directly contact one of the four participating centres. You can access the treatment centre directory at www.hemophilia.ca/treatment-centres-by-province.
For more information about the BEYOND-9 clinical trial, see …
hemophilia-beyond-9.com/en-us/about-beyond-9
clinicaltrials.gov/study/NCT06379789?cond=Hemophilia%20B&term=BEYOND-9&rank=1#study-overview