Bioverativ, Bicycle Therapeutics to Jointly Develop New Therapies for Rare Blood Disorders
Bioverativ and Bicycle Therapeutics will jointly discover, develop and market new therapies to treat hemophilia and sickle cell disease, the…
Articles by Alice Melão
Catalyst’s CB 2679d Was Shown to Outperform BeneFix in Phase 1/2 Trial in Hemophilia B
Results of the first group of patients with hemophilia B treated with Catalyst Biosciences’ lead candidate CB 2679d in a…
Regenxbio Likely to Add Dimension’s DTX201 for Hemophilia A to Its Pipeline
Dimension Therapeutics may soon become a wholly owned subsidiary of Regenxbio, following the terms of an agreement between the…
Alnylam Suspends All Trials of Fitusiran After Hemophilia A Patient Dies in Phase 2 Study
Alnylam Pharmaceuticals has suspended patient dosing in all ongoing studies of its RNA therapy fitusiran to treat hemophilia A or…
Bayer Asks FDA to Approve BAY94-9027, Long-acting Treatment for Hemophilia A
Bayer has filled a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) requesting marketing approval…
Biomarin Plans to Test Two Doses of BMN 270 in Phase 3 Trials for Treatment of Hemophilia A
BioMarin Pharmaceutical will add an additional Phase 3 study to the clinical development of its investigational gene therapy BMN 270…
Hemophilia B Gene Therapy AMT-060 Moves to Large-scale Production, Says UniQure
Pharmaceutical firm uniQure N.V. has developed an optimized large-scale process to manufacture its lead gene, therapydate, to treat hemophilia…
Phase 1 Trial of Potential RNA Therapy Fitusiran Shows Promise for Hemophilia A and B
Once-monthly injection of the investigational RNA therapy Fitusiran reduced the levels of the protein antithrombin, a natural inhibitor of several…
Idelvion Shown to Benefit Both Adult and Pediatric Patients With Hemophilia B
The latest results of CSL Behring’s recombinant coagulation factor IX (FIX) Idelvion, also known by its…
Shire Asks FDA for a Designation That Will Lead to More Clinical Trials of Its Hemophilia A Therapy SHP654
Shire has asked the U.S. Food and Drug Administration to grant Investigational New Drug status to its hemophilia A treatment…