FVIII Gene Therapy Directly into Joints May Best Protect Against Damage
Gene therapy delivering the blood clotting factor VIII (FVIII) — whose lack causes hemophilia A — into the joints did…
Articles by Patricia Inácio, PhD
Kovaltry Safe and Effective Prophylactic for Hemophilia A, Long-term Study Shows
Preventive treatment with Kovaltry (octocog alfa) is safe and effective at preventing bleeds, including joint bleeds, for up…
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
Light Chain, Takeda Reach Key Milestone in Partnership Developing Bispecific Antibodies for Treating Hemophilia A
Light Chain Bioscience, a unit of Swiss biotech Novimmune, has completed the discovery phase of its collaborative program with…
Generation Bio Raises $110 Million for Non-viral Gene Therapy for Hemophilia A, Other Diseases
Generation Bio said it received $110 million in funding to bring its innovative, non-viral gene therapy for lead…
Pfizer Taking over Development of Potential Gene Therapy SB-525, Advancing into Phase 3 Trials
Sangamo Therapeutics has handed over the development of SB-525, a gene therapy for hemophilia A, to…
Bayer, Children’s Hospital of Philadelphia Working on Non-replacement Hemophilia Therapies
Bayer has entered into a three-year research partnership with the Children’s Hospital of Philadelphia to develop small molecule…
Professional Help Essential for Parents Telling Daughters of Hemophilia Carrier Risk, Study Reports
An interview-based study from Japan found professional support was necessary when mothers inform their daughters they might be carriers for…
Bone Defects Detected at Birth, After Injury, in Mouse Models of Hemophilia A and B, Study Shows
Bone defects were seen since birth — and following injury — in mouse models of hemophilia A and B,…
DalcA, Under-skin Injectable Factor IX Prophylactic, Showing Potential in Phase 2 Trial, Catalyst Says
A preventive treatment for hemophilia B called dalcinonacog alfa (DalcA) led to an increase of more than 12% in the levels of…