Alnylam Pharmaceuticals, Inc., reports positive interim results from a part of its ongoing Phase 1 study with fitusiran in patients with hemophilia A and B with inhibitors.
Gene therapy clinical trials eliminate infusions in some patients and improve quality of life.
Trials of Sangamo's gene therapy called SB-525 show promising results in animals with hemophilia A.
WILATE®, a human-derived von Willebrand factor/coagulation factor VIII therapy, will be used in two upcoming clinical trials slated to launch in December 2016.
Gene editing therapy will be used in clinical trials first in people with hemophilia B, then in those with hemophilia A.
The program encourages innovative research and educational initiatives that benefit individuals with hemophilia worldwide.
Preliminary findings show psychosocial impact on individuals and families dealing with hemophlia B, including mild and moderate forms.
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.
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