Hemophilia News Today

June 20, 2022June 20, 2022

New Agreement Set to Scale Up Production of Hem A Gene Therapy

ASC Therapeutics has joined forces with Charles River to scale the manufacturing of ASC618, its second-generation virus-based gene therapy for hemophilia A. The agreement expands upon their current collaboration, begun in ... Read more

June 17, 2022June 17, 2022

Portraits of Life With Hemophilia Showcased in Multimedia Exhibit

Hemophilia News Today

An immersive multimedia exhibit, which features portraits by renowned photographer Rankin, seeks to educate about life with hemophilia and the disease community’s hope for the future. The exhibit, called “Portraits ... Read more

June 15, 2022June 15, 2022

2nd Hem B Patient Group Begins Dosing in Gene Therapy Trial

Hemophilia News Today

Dosing has begun in a second group of hemophilia B patients enrolled in a Phase 1/2 dose-confirmation trial of the experimental gene therapy FLT180a, the treatment’s developer, Freeline, announced. The ... Read more

June 13, 2022June 13, 2022

Study Reveals Risk Factors for Future Bleeds in Hemophilia Patients

Hemophilia News Today

The levels of blood clotting factors, physical activity, and bleeding history are all potential risk factors for future bleeds in hemophilia patients under preventive therapy, a small review study from ... Read more

June 10, 2022June 11, 2022

Elocta Found to Reduce Elbow, Knee Bleeds in Severe Hem A Patients

Hemophilia News Today

One year of treatment with Elocta (efmoroctocog alfa) — an extended half-life replacement therapy known as Eloctate in the U.S. — reduced the frequency of bleeds in the elbows and ... Read more

June 8, 2022June 9, 2022

Behavioral Problems in Young Patients Tied to Age, Disease Severity

Hemophilia News Today

Children and young adults with hemophilia A and hemophilia B have several behavioral problems, ranging from depression and anxiety to aggressive behaviors, a small study suggests. Age, disease severity, and joint disease ... Read more

June 6, 2022June 6, 2022

Low-dose Preventive Therapy Yields Variable FVIII Levels, Study Reports

Hemophilia News Today

Preventive treatment with a low dose of factor replacement therapy in children with hemophilia A led to sufficient levels of the clotting factor in most patients, but the time in ... Read more

June 3, 2022June 3, 2022

Efanesoctocog Alfa Earns Breakthrough Therapy Status

Hemophilia News Today

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to efanesoctocog alfa, an experimental replacement therapy for hemophilia A designed for once-weekly dosing. This designation is given ... Read more

June 1, 2022June 1, 2022

Benefits of Roctavian, Hem A Gene Therapy, Holding for Up to 6 Years

Hemophilia News Today

A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) prevents bleeds and the need for preventive treatment, or prophylaxis, for up to six years in men with severe ... Read more

May 27, 2022May 27, 2022

BBM-H901, Gene Therapy for Hem B, Shows Efficacy at 1 Year in Pilot Trial

Hemophilia News Today

The gene therapy BBM-H901 increased factor IX activity in 10 men with moderate to severe hemophilia B, while reducing bleeds and the use of FIX replacement therapy over one year, according ... Read more

May 25, 2022May 25, 2022

1st Gene Therapy for Hem B Granted Priority Review by the FDA

Hemophilia News Today

The U.S. Food and Drug Administration (FDA) has accepted — under priority review — a marketing application for EtranaDez (etranacogene dezaparvovec), an investigational gene therapy for adults with hemophilia B. ... Read more

May 23, 2022May 23, 2022

Extended Half-life Products Prevent Surgery Bleeds in Hemophilia A, B

Hemophilia News Today

Extended half-life (EHL) blood-clotting factor products are safe and effective at preventing bleeds during minor and major surgeries in people with hemophilia, according to a real-world study in Nordic countries. ... Read more

May 20, 2022May 20, 2022

Anxiety, Depression Need Better Management in Hemophilia: Survey

Hemophilia News Today

Symptoms of pain, anxiety, and depression generally increased with disease severity in people with hemophilia and were also associated with a lower quality of life, according to a recent survey involving ... Read more

May 18, 2022May 18, 2022

Blood Vessel Abnormalities May Explain Gene Therapy Limitations

Hemophilia News Today

The effectiveness of liver-directed, viral-based gene transfer — the most used experimental gene therapy approach for hemophilia A — is significantly reduced in mouse models of hemophilia A relative to ... Read more

May 16, 2022May 16, 2022

Gene Therapy Researcher Honored by Children’s Hospital of Philadelphia

Hemophilia News Today

Katherine High, MD, has received the gold medal from Children’s Hospital of Philadelphia (CHOP) in recognition of her work to advance gene therapies for hemophilia and other genetic disorders. In ... Read more