Industry News

Alnylam Pharmaceuticals, Inc. has reported on the death of a hemophilia A patient who had been participating in a Phase 2 study for the company’s investigational therapy fitusiran.

Bayer applied for a Biologics License Application for BAY 94-9027, the company’s long-acting site-specific PEGylated recombinant factor VIII therapy for the treatment of hemophilia A.

Sangamo Therapeutics, Inc. and Pfizer Inc. recently announced that the first patient received treatment in a phase 1/2 clinical trial designed to evaluate SB-525, the company’s gene therapy program for people with hemophilia A, or factor VIII (FVIII) deficiency.

Specifically, Pfizer will expand an 11,000-square-foot plant in Sanford, North Carolina that it acquired last year when it bought Bamboo Therapeutics, a biotech company that specializes in gene therapies for certain rare diseases.

Spark Therapeutics, Inc. recently released preliminary data from a phase 1/2 dose-escalation clinical trial for SPK-8011, the company’s investigational gene therapy candidate for hemophilia A.

New online learning activity entitled, “An Overview of the Background, Inheritance and Physiology of von Willebrand Disease,” is now available.

UniQure N.V. has announced that is has “scaled up” the manufacturing process at its large facility in Lexington, MA.