Stay informed with the latest updates for the inheritable blood disorders community.

Industry
DNA Gene Helix
FDA Accepts Priority Review BLA for Hemophilia B Gene Therapy
Industry
First Patient Dosed in Freeline’s Dose-Confirmation Trial for Hemophilia B Gene Therapy
First Patient Dosed in Freeline’s Dose-Confirmation Trial for…
Industry
BioMarin Provides Update Including Next Steps for Investigational Gene Therapy
BioMarin Provides Update Including Next Steps for Investigational Gene…
Industry
Sigilon Announces a Priority Shift in Light of Clinical Trial Setbacks
Sigilon Announces a Priority Shift in Light of Clinical Trial Setbacks
Industry
uniQure and CSL Behring Announce Trial Updates for Hemophilia B Gene…
Industry
Sigilon Reports Trial Updates for Investigational Hemophilia A Therapy
Industry
Spark Announces Multiyear Data Update for Investigational Gene Therapy
Spark Announces Multiyear Data Update for Investigational Gene Therapy
NBDF & Community
Washington D.C. - Capitol Building
NHF Applauds Implementation of the Hemophilia SNF Access Act
Medical
Prophylaxis Indication Restored for BeneFIX®
Industry
Spark Provides Trial Updates for Investigational Gene Therapy SPK-8011
Spark Provides Trial Updates for Investigational Gene Therapy SPK-8011
Industry
FDA Places Clinical Hold on Sigilon’s Investigational Hemophilia Therapy
FDA Places Clinical Hold on Sigilon’s Investigational Hemophilia Therapy
Industry
Female scientist conducting research in lab with goggles and gloves
BioMarin Presents Positive Safety and Efficacy Data for their…
Industry
Gene Therapy Trial Data Suggests Efficacy in the Presence of Antibodies
Gene Therapy Trial Data Suggests Efficacy in the Presence of Antibodies
Industry
Gene Therapy Licensing Agreement Between uniQure and CSL Now in Effect
Gene Therapy Licensing Agreement Between uniQure and CSL Now in Effect
Industry
Gene Therapy Helix
uniQure Announces End of Clinical Hold on Hemophilia B Gene Therapy…