New Gene Therapy Vectors Better at Targeting Liver: Hem B Mouse Study
Two new gene therapy vectors designed for liver diseases show promising effects in a mouse model of hemophilia B,…
Articles by Marisa Wexler, MS
FDA Decision Expected in Early 2023 on Hem A Gene Therapy Roctavian
The U.S. Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) from BioMarin Pharmaceutical requesting approval…
FDA Clears Trial of TI-168 Therapy to Eliminate Hemophilia A Inhibitors
The U.S. Food and Drug Administration (FDA) has cleared the biotechnology company TeraImmune to launch a Phase 1/2a clinical trial…
Phase 3 Trial of SB-525, Gene Therapy for Hem A, Again Enrolling
Men with moderate-to-severe hemophilia A are again being enrolled in the Phase 3 AFFINE trial, which is testing the…
Activity of Inflammatory Genes Increased in Patients with Inhibitors
Hemophilia A patients who develop inhibitors — neutralizing antibodies made by immune cells against clotting factors — have more…
Hemophilia A Replacement Therapy TQG202 Safely Controls Bleeds: Trial
Prophylactic, or preventive, treatment with an experimental replacement therapy called TQG202 safely maintained a low bleeding rate in people with…
FDA Grants Priority Review of Efanesoctocog Alfa for Hemophilia A
The U.S. Food and Drug Administration (FDA) has granted priority review for the approval of efanesoctocog alfa, with a…
Roctavian, Hem A Gene Therapy, Given Conditional Approval in EU
The European Commission has granted conditional marketing authorization to BioMarin’s one-time gene therapy Roctavian (valoctocogene roxaparvovec) as a treatment…
In Trial, Hemlibra Improves Joint Health for Younger Hem A Patients
Treatment with Hemlibra (emicizumab) improved joint health in people with hemophilia A participating in the Phase 3 HAVEN…
Older Male Patients Report Higher Rates of Diabetes, Depression, Anxiety
Older men with hemophilia report higher rates of anxiety, depression, and diabetes compared with the general U.S. population, a…