This Breakthrough designated therapy is put on a fast-track approval program from the FDA and could dramatically reduce the frequency of bleeding episodes in people with hemophilia B.
Collaboration will enhance the development of gene therapy tools for people with hemophilia A and B.
New therapy is intended for patients with hemophilia A or B with inhibitors.
The green light allows Sangamo to move forward with clinical development and means investigational therapy on adults can get underway in 2017.
Results of the ongoing clinical trial of BMN 270 in human subjects provide encouraging outcome so far.
Emicizumab trial shows significant improvements in reducing bleeds in people with hemophilia A and inhibitors.
Results of recombinant product on previously untreated patients provide data on inhibitor development.
Significant improvements seen in the patients in the trial, some of whom were followed for one year.
Experimental hemophilia B therapy shows evidence of better bleed control when the trough level is higher.
Recombinant extended half-life therapy stretches infusion intervals and improves quality of life across different ages tested.
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