BioMarin has an ongoing commitment to the hemophilia community, which includes our growing understanding of valoctocogene roxaparvovec (BMN 270), through ongoing registries and research.
Please click on the trial numbers to find out more information, where available.
Phase 1/2 Dose Escalation Study
Enrollment ongoing (701)
Global Seroprevalence Study
Phase 3 Study with 6 x 1013vg/kg dose
Phase 3 Study with 4 x 1013vg/kg dose
AAV5+ Phase 1/2 Titer Escalation Study with 6 x 1013vg/kg dose
6 x 1013vg/kg dose with Prophylactic Corticosteroids Study
Active or Prior Inhibitor Phase 1/2 Study with 6 x 1013vg/kg dose
Long-term follow up for patients who received valoctocogene roxaparvovec
aBioMarin announces its decision to cease development of the 4 x 1013 dose (GENEr8-2) in August 2019
KSA, Kingdom of Saudi Arabia; AAV, adeno-associated virus