Margarida Azevedo, MSc,  —

The European Medicines Agency (EMA) has granted orphan medicinal product status to SB-525, a clinical stage gene therapy candidate under development for the treatment of hemophilia A, according to Sangamo Therapeutics and Pfizer. The announcement coincides with the opening of patient enrollment for a Phase 1/2…

Chris Bombardier, the author of the “Adventures of a Hemophiliac” blog, has become the first person with hemophilia to climb Mount Everest, the world’s highest peak. That achievement on May 22 means the 31-year-old Denver native has only one mountain to go toward his goal of scaling the tallest…

The Phase 1/2 trial looking at the Factor VIII protein SHP656 as a long-acting therapeutic to treat hemophilia A did not meet a pre-defined once-weekly dosing criterion, according to Xenetic Biosciences and Shire, which are jointly developing the drug. The study demonstrated SHP656’s efficacy and pharmacokinetic data (the drug’s behavior once…

The National Organization for Rare Disorders (NORD) has recognized pharmaceutical company CSL Behring for pioneering Idelvion (rIX-FP), its therapeutic medicine against hemophilia B. “We are proud to present the team at CSL Behring with a 2017 Industry Innovation Award for the important work they are doing to address the needs of patients…

The U.S. Food and Drug Administration (FDA) has granted fast-track status to SB-525, a clinical gene therapy for hemophilia A developed by Sangamo Therapeutics in partnership with Pfizer. SB-525 delivers a human factor VIII cDNA construct and synthetic liver-specific promoter to the cell’s nucleus by using recombinant adeno-associated virus (rAAV). The…

Dimension Therapeutics will discontinue clinical development of DTX101, a gene therapy intended to treat moderate/severe-to-severe hemophilia B. The decision comes after a review of data from a Phase 1/2 clinical trial (NCT02618915), which assessed and the safety and dosing of DTX101. The trial…

Sangamo Therapeutics and Pfizer have entered a global collaboration and license agreement to develop and market gene therapy programs for hemophilia A, including SB-525. SB-525 was cleared by the U.S. Food and Drug Administration (FDA) in January as an investigational new drug (IND) for the…

UniQure has taken major steps this year toward U.S. and European regulatory approval of AMT-060, its gene-therapy treatment for hemophilia B. In January, the U.S. Food and Drug Administration (FDA) gave AMT-060 breakthrough therapy designation for patients with severe hemophilia B. In April the European Medicines Agency (EMA) gave it PRIME designation. The…

Two gene therapies from Sangamo Therapeutics for hemophilia A and B have been granted special regulatory designations by the U.S. Food and Drug Administration (FDA). Sangamo said in a press release that the FDA has granted orphan drug status to SB-525  gene therapy for hemophilia A, providing the company…

In recognition of the World Hemophilia Day, BDI Pharma employees company-wide united to raise awareness and money to support those affected by bleeding disorders. Events were held April 17 at the company’s South Carolina, Texas and Kansas sites, with employees joining remotely to raise money to support the WFH (World Federation of Hemophilia) by wearing…