The trial is Spark's investigational gene transfer candidate for hemophilia A.
A statement from Genentech/Roche regarding the death of a participant in the HAVEN 1 clinical trial.
AbbVie recently announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) and granted priority review for glecaprevir/pibrentasvir (G/P).
As of February 1, 2017, the new company, known as Bioverativ, became an independent global biotechnology company focused on hemophilia and other rare blood disorders.
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.
eNewsletter Sign Up
Steps for Living
Victory for Women
Unite for Bleeding Disorders Walk
Donate Your Car