EMA Names Investigational Gene Therapy, BMN 270, a Priority Medicine for Hemophilia A
BioMarin Pharmaceuticals is moving forward with BMN 270, an experimental gene therapy for the treatment of hemophilia A, and is…
Articles by Margarida Azevedo, MSc
Uniqure’s Hemophilia B Treatment Granted FDA Breakthrough Therapy Designation
The U.S. Food and Drug Administration (FDA) has granted uniQure’s AMT-060, an investigational gene therapy, breakthrough therapy designation for patients with severe…
Catalyst to Present Data on Clotting Factor Candidates at Hemophilia Congress
Catalyst Biosciences will present three posters with data from its two investigational candidates for hemophilia at the upcoming 10th Annual Congress…
Sangamo Therapeutics Presents Advances in Zinc Finger Nucleases for Gene-Editing Use
Sangamo Therapeutics recently presented what it characterized as key improvements to its technology platform for engineering highly specific…
Xenetic Paid $3 Million to Advance Phase 1/2 Trial of Long-acting Hemophilia Therapy
Xenetic Biociences has received a $3 million milestone payment from its partner Shire for advancing the Phase 1/2 clinical trial of…
FDA Clears Hemophilia A Gene Therapy, SB-525, for Testing in Patients
A Sangamo Therapeutics‘ gene therapy program to potentially treat hemophilia A, called SB-525, has been cleared by the U.S.
FDA Approves Adynovate for use in Surgical Settings and Children Under 12
The U.S. Food and Drug Administration (FDA) has approved Shire’s Adynovate for the treatment of pediatric patients under…
Health Canada Approves Afstyla for Treatment of Hemophilia A
Health Canada, the agency that oversees health decisions and regulations in Canada, has approved Afstyla (lonoctocog alfa) for the treatment…
World AIDS Day Events Focused on Toll Disease Took on Hemophilia Community
More than 1,300 people who have been affected by acquired immune deficiency syndrome (AIDS)Â gathered at the National AIDS…
Octapharma to Launch Two Hemophilia A Clinical Trials Using Wilate
Two clinical trials launching in December will use Octapharma USA’s Wilate, von Willebrand Factor/Coagulation Factor VIII Complex…