Encouraging results from a phase I/II clinical trial of SPK-9001, the company’s novel investigational hemophilia B gene therapy.
Preliminary data from a low-dose group were presented at the 21st Congress of the European Hematology Association in Copenhagen, Denmark.
Combined company is expected to be the global leader in the treatment of many rare diseases.
Chugai's novel prophylactic therapy for people with hemophilia A is administered through a subcutaneous injection.
Gene therapy trials in lab animals show significant increases in production of factor VIII and IX.
Early clinical trial results in patients with hemophilia A show increased clotting factor levels in all subjects.
New agent will provide another option for promoting blood clotting in patients with bleeding disorders undergoing dental procedures.
The collaboration will focus on developing adeno-associated viruses (AAVs), delivery vehicles for carrying genetic material, including those for hemophilia.
Alnylam Pharmaceuticals announces Phase 1 study of ALN-AT3, a subcutaneous therapy being tested on patients with hemophilia.
BioMarin announces enrollment of the first patient in clinical trial of BMN 270, a gene therapy to treat people with hemophilia A.
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