BioMarin to Resubmit Gene Therapy Roctavian for EU Approval in June
The European Medicines Agency (EMA) has accepted BioMarin Pharmaceutical’s request for accelerated assessment of Roctavian, the company’s investigational…
Articles by Marta Figueiredo, PhD
Hemophilia B Gene Therapy AMT-061 Safe, Effective, Trial Data Show
AMT-061 (etranacogene dezaparvovec), uniQure‘s investigational gene therapy for hemophilia B, is safe and effective — even in patients with…
1st Patient Dosed in Phase 3 Trial of Injectable MarzAA
The first participant has been dosed in a Phase 3 clinical trial testing marzeptacog alfa activated (MarzAA), an experimental under-the-skin…
CSL Behring Acquires AMT-061, Hem B Gene Therapy in Phase 3 Trial
CSL Behring has closed an agreement giving it global commercialization and licensing rights to AMT-061, an experimental gene…
Jivi May Prevent Minor Surgery Bleeds in Severe Hemophilia A Patients
Bayer’s Jivi safely and effectively prevents bleeds during and after minor surgery in children, adolescents, and adults with…
Immune Deficiencies With FVIII Inhibitors Evident, But Can Be Restored
Hemophilia A patients with factor VIII (FVIII) inhibitors show deficiencies in regulatory B- and T-cells — immune cells that…
FDA Lifts Hold on uniQure’s Gene Therapy AMT-061 for Hemophilia B
The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on uniQure’s gene therapy program for hemophilia B, which includes …
Long-term Jivi Continues to Safely Prevent Bleeds in Severe Hemophilia A
Long-term treatment with Jivi safely and effectively reduced the number of bleeding episodes in adolescents and adults with severe…
Continuous Advate IV Effectively Prevented Bleeds in Surgery
Continuous into-the-vein (intravenous) infusions of Advate immediately before and after major orthopedic surgery is as safe and effective as standard,…
Hemophilia B Gene Therapy AMT-061 Unlikely to Have Caused Liver Tumor
AMT-061 (etranacogene dezaparvovec), uniQure’s investigational gene therapy for hemophilia B, likely did not cause the case of hepatocellular carcinoma (HCC)…