This agreement allows Shire to conduct research on the use of Rani Pill™ technology for the oral delivery of factor VIII therapy for patients with hemophilia A.
Spark provided clinical trial updates on the company’s investigational gene therapies for hemophilia A and B, SPK-8011 and SPK-9001.
Genentech recently reported results from the phase III HAVEN 3 study, which showed that the prophylactic use of HEMLIBRA® led to significantly reduced bleeds in adult and adolescent patients with h
Alnylam is developing the therapy to treat patients with hemophilia A and B, both with and without inhibitors.
People who used the therapy for nearly three years saw regular improvements in their joint health.
The BLA was submitted in light of positive data yielded from a phase 2/3 clinical study.
The breakthrough status was granted in light of positive results of phase 1/2 clinical study.
If effective, the therapy prompts the sustained production of FVIII and enhanced protection from bleeds.
UniQure announced plans to “expeditiously advance” its investigational hemophilia B gene therapy AMT-061 into a study in 2018.
The company stated subcutaneous injections of the therapy boosted FIX in hemophilia B patients to therapeutically significant levels.
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