Three years after receiving the one-time gene therapy Roctavian (valoctocogene roxaparvovec) at trial, the majority of men with severe…
Articles by Marisa Wexler, MS
One-time treatment with the investigational gene therapy SPK-8011 led to sustained low bleed rates for people with hemophilia A…
A committee of the European Medicines Agency (EMA) has issued a positive opinion recommending the one-time gene therapy etranacogene…
The U.S. Food and Drug Administration (FDA) has scheduled its inspection of the facility in California where BioMarin Pharmaceutical…
Two new gene therapy vectors designed for liver diseases show promising effects in a mouse model of hemophilia B,…
The U.S. Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) from BioMarin Pharmaceutical requesting approval…
The U.S. Food and Drug Administration (FDA) has cleared the biotechnology company TeraImmune to launch a Phase 1/2a clinical trial…
Men with moderate-to-severe hemophilia A are again being enrolled in the Phase 3 AFFINE trial, which is testing the…
Hemophilia A patients who develop inhibitors — neutralizing antibodies made by immune cells against clotting factors — have more…
Prophylactic, or preventive, treatment with an experimental replacement therapy called TQG202 safely maintained a low bleeding rate in people with…