European Agency Grants Orphan Medicinal Status to Experimental Therapy SB-525 for Hemophilia A
The European Medicines Agency (EMA) has granted orphan medicinal product status to SB-525, a clinical stage gene therapy candidate…
Articles by Margarida Azevedo, MSc
Denver’s Hemophilia Hero Chris Bombardier Becomes First with the Disease to Climb Mount Everest
Chris Bombardier, the author of the “Adventures of a Hemophiliac” blog, has become the first person with hemophilia to climb…
Hemophilia A Candidate SHP656 Fails to Meet Once-Weekly Dosing Goal, Partners Announce
The Phase 1/2 trial looking at the Factor VIII protein SHP656 as a long-acting therapeutic to treat hemophilia A did not meet…
CSL Behring Receives NORD 2017 Industry Innovation Award for Hemophilia Therapy Idelvion
The National Organization for Rare Disorders (NORD) has recognized pharmaceutical company CSL Behring for pioneering Idelvion (rIX-FP), its therapeutic medicine against…
FDA Awards Fast-track Status to Sangamo’s Investigational Gene Therapy SB-525 for Hemophila A
The U.S. Food and Drug Administration (FDA) has granted fast-track status to SB-525, a clinical gene therapy for hemophilia…
Dimension to End Development of DTX101 as Gene Therapy for Hemophilia B
Dimension Therapeutics will discontinue clinical development of DTX101, a gene therapy intended to treat moderate/severe-to-severe hemophilia B. The decision comes…
Sangamo, Pfizer Unite to Develop SB-525 Hemophilia A Gene Therapy
Sangamo Therapeutics and Pfizer have entered a global collaboration and license agreement to develop and market gene therapy…
UniQure Making Progress Toward US and European Approval of AMT-060 for Hemophilia B
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 …
FDA Grants Special Designations to Sangamo for Hemophilia Gene Therapies
Two gene therapies from Sangamo Therapeutics for hemophilia A and B have been granted special regulatory designations by the U.S.
BDI Pharma Employees Raise Funds, Awareness on World Hemophilia Day
In recognition of the World Hemophilia Day, BDI Pharma employees company-wide united to raise awareness and money to support those affected…