Roctavian’s Effectiveness Tied to Molecules that Aid FVIII Production
Factor VIII (FVIII) levels in the bloodstream of men with severe hemophilia A treated with the investigational gene therapy…
Articles by Steve Bryson, PhD
Rebinyn Compares Well to Two Other FIX Therapies: Real-life Study
Hemophilia BÂ patients who switched to Rebinyn (nonacog beta pegol), an extended half-life factor IX (FIX) replacement therapy, had…
Risk of Inhibitor Development Tied to High BAFF Protein Levels
Elevated levels of the immune signaling protein BAFF were found in the bloodstream of boys who developed neutralizing antibodies against…
Enrollment Complete for GENEr8-3 Trial of Roctavian Plus Steroids
BioMarin Pharmaceutical has completed enrollment in GENEr8-3, a Phase 3b study evaluating its investigational gene therapy Roctavian (valoctocogene…
Preventive Esperoct Plus Surgery Reduces Joint Bleeds, Aids Mobility
Preventive treatment with Esperoct (turoctocog alfa pegol) plus joint surgery significantly decreased annual joint bleeding rates, reduced pain, and…
Low-dose ITI Successful in 80% of Hemophilia A Children, Study Shows
Low-dose immune tolerance induction (ITI) — a type of treatment that can be used to eliminate inhibitors that limit the…
2seventy Bio, Novo Nordisk Expand Partnership to Develop Gene Editing
2seventy Bio has expanded its collaboration with Novo Nordisk to develop a gene-editing approach to treat hemophilia…
Trial of Hemophilia B Gene Therapy BBM-H901 Begins Dosing in China
The first man with hemophilia B has been dosed in a clinical trial in China to evaluate Belief BioMed’s…
Fitusiran Curbs Bleeds in Hemophilia A, B With Inhibitors, Data Show
When given monthly to people with hemophilia A or B…
Phase 3 Trial of Gene Therapy SB-525 for Hemophilia A on FDA Hold
The U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Phase 3 trial evaluating SB-525…