Margarida Azevedo, MSc,  —

BioMarin Pharmaceuticals is moving forward with BMN 270, an experimental gene therapy for the treatment of hemophilia A, and is preparing new clinical trials expected to begin this year. The European Medicines Agency (EMA)’s Committee for Advanced Therapies (CAT) and its Committee for Medicinal Products for Human Use (CHMP) agreed that BMN 270…

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 hemophilia B. The regulatory agency based its decision on data from the ongoing, dose-ranging Phase 1/2 clinical trial (NCT02396342) evaluating AMT-060 for hemophilia B. The study’s estimated completion date…

Catalyst Biosciences will present three posters with data from its two investigational candidates for hemophilia at the upcoming 10th Annual Congress of the European Association of Haemophilia and Allied Disorders (EAHAD) Feb. 1-3 in Paris, France. The first poster presentation is for a next-generation factor VIIa, marzeptacog alfa (activated), that showed…

Sangamo Therapeutics recently presented what it characterized as key improvements to its technology platform for engineering highly specific zinc finger nucleases (ZFNs), which are used for targeted editing of the human genome. This technique has broad applicability, namely for the treatment of hemophilia B and other disorders. The…

Xenetic Biociences has received a $3 million milestone payment from its partner Shire for advancing the Phase 1/2 clinical trial of PSA-recombinant SHP656, a factor VIII protein, evaluating the drug’s effectiveness as a long-acting therapy for hemophilia A. Shire’s main objective behind the agreement is to develop a novel FVIII…

A Sangamo Therapeutics‘ gene therapy program to potentially treat hemophilia A, called SB-525, has been cleared by the U.S. Food and Drug Administration (FDA) for clinical testing in adult patients. The company announced that the FDA approved its Investigational New Drug (IND) application for SB-525, a necessary first step…

The U.S. Food and Drug Administration (FDA) has approved Shire’s Adynovate for the treatment of pediatric patients under 12 years of age with hemophilia A. Adynovate also was approved for use by both adults and children undergoing surgical procedures, to control bleeding episodes. Adynovate is a recombinant Factor…

Health Canada, the agency that oversees health decisions and regulations in Canada, has approved Afstyla (lonoctocog alfa)  for the treatment and prevention of bleeding in adults and children with hemophilia A. In May, the U.S. Food and Drug Administration (FDA) approved Afstyla for the same indications. Developed by CSL Behring, Afstyla is…

More than 1,300 people who have been affected by acquired immune deficiency syndrome (AIDS) gathered at the National AIDS Memorial in San Francisco on Dec. 1 to commemorate World AIDS Day. During two days of events, family and friends paid tributes to those lost to the disease, while inspiring remembrance…

Two clinical trials launching in December will use Octapharma USA’s Wilate, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection, in patients with hemophilia A. Hemophilia A is an X-linked hereditary bleeding disorder that results from either reduced or absent levels of…