News

September 22, 2017September 22, 2017

Bioverativ, Bicycle Therapeutics to Jointly Develop New Therapies for Rare Blood Disorders

Bioverativ and Bicycle Therapeutics will jointly discover, develop and market new therapies to treat hemophilia and sickle cell disease, the two companies announced. These future therapies will be based on ... Read more

September 20, 2017

Catalyst’s CB 2679d Was Shown to Outperform BeneFix in Phase 1/2 Trial in Hemophilia B

News

Results of the first group of patients with hemophilia B treated with Catalyst Biosciences’ lead candidate CB 2679d in a Phase 1/2 clinical trial demonstrated its improved stability and activity compared ... Read more

September 18, 2017September 18, 2017

Regenxbio Likely to Add Dimension’s DTX201 for Hemophilia A to Its Pipeline

News

Dimension Therapeutics may soon become a wholly owned subsidiary of Regenxbio, following the terms of an agreement between the two companies. The merger agreement, if finalized, will add Dimension’s ... Read more

September 11, 2017September 11, 2017

Alnylam Suspends All Trials of Fitusiran After Hemophilia A Patient Dies in Phase 2 Study

News

Alnylam Pharmaceuticals has suspended patient dosing in all ongoing studies of its RNA therapy fitusiran to treat hemophilia A or B, after a patient died of swelling in the brain in ... Read more

September 8, 2017September 8, 2017

Bayer Asks FDA to Approve BAY94-9027, Long-acting Treatment for Hemophilia A

News

Bayer has filled a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) requesting marketing approval for its long-acting human Factor VIII therapy, BAY94-9027, to treat hemophilia ... Read more

September 6, 2017

Bioverativ to Partner with Invicro on Ultrasound Imaging of Hemophilia Joint Disease

News

Bioverativ will collaborate with the Boston imaging company Invicro to use ultrasound imaging to improve the diagnosis and management of joint disease in hemophilia patients. “Through this innovative collaboration with Invicro, we hope ... Read more

September 1, 2017September 1, 2017

Shire and MicroHealth Working on App to Improve Care of Hemophilia Patients Resistant to Treatment

News

Shire announced it is working with MicroHealth to develop a free and secure digital monitoring mobile app for hemophilia A and B patients with antibodies, known as inhibitors, that infer with ... Read more

August 30, 2017

Pfizer Debuts Innovative Video Game and Wristband for Children with Hemophilia

News

Pfizer Rare Disease has developed a new video game and wearable wristband to help hemophilia patients learn more about their condition and promote activity tracking in the U.S. The video ... Read more

August 28, 2017August 31, 2017

FDA Approves New, More Convenient Vial Strengths for Hemophilia A Therapy Nuwiq

News

The U.S. Food and Drug Administration has approved new vial strengths for Octopharma’s hemophilia A therapy Nuwiq, which means patients will need fewer vials per treatment session. Octapharma USA said single-dose strengths ... Read more

August 26, 2017August 28, 2017

SB-525 Gene Therapy Phase 1/2 Trial Begins for Hemophilia A Patients

News

The first patient in a Phase 1/2 clinical trial (“the Alta trial”) designed to assess safety and tolerability of SB-525 gene therapy has received treatment. Sangamo Therapeutics, in a ... Read more