News

CSL Behring has closed an agreement giving it global commercialization and licensing rights to AMT-061, an experimental gene therapy for hemophilia B now being tested in a Phase 3 clinical trial. The therapy’s developer, uniQure, will receive an upfront cash payment of $450 million from CSL…

Bayer’s Jivi safely and effectively prevents bleeds during and after minor surgery in children, adolescents, and adults with severe hemophilia A, according to interim data from the Phase 2/3 PROTECT VIII and the Phase 3 PROTECT VIII Kids clinical trials. Most of the included patients were on…

Hemophilia A patients with factor VIII (FVIII) inhibitors show deficiencies in regulatory B- and T-cells — immune cells that typically dampen immune and inflammatory responses — but their levels can be restored with successful immune tolerance induction (ITI), a study shows. These findings shed light on how ITI — given…

Preventive, or prophylactic, treatment with Hemlibra (emicizumab) safely and effectively managed bleeding in adults and children with severe hemophilia A, a real-world study has found. The study, “Emicizumab prophylaxis: Prospective longitudinal real‐world follow‐up and monitoring,” was published in the journal Haemophilia. Originally developed by Roche’s subsidiary…

Screening for clinical trials in gene therapy can exclude the vast majority — 92% — of severe hemophilia patients, for reasons too often due to potentially modifiable factors such as socioeconomic status and location, a study of patients at a treatment center in Belgium reported. Patients’ unwillingness to…

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on uniQure’s gene therapy program for hemophilia B, which includes AMT-061 (etranacogene dezaparvovec), the company announced. According to uniQure, the FDA found it was “very unlikely” that AMT-061 had contributed to a case of liver cancer in a patient who…

Healthcare costs can be 25 times higher for hemophilia B patients than for individuals who do not have a bleeding disorder, according to a recent study. These findings reflect both direct costs of hemophilia treatment and a greater need for other healthcare resources, researchers noted. The study, “…

An investigational gene therapy for hemophilia B, called CB 2679d-GT, increased factor IX (FIX) levels and significantly reduced bleeding in a mouse model of the disease, a preclinical study demonstrated. The clotting activity of this potential therapy, being developed by…

Scientists found the immune signaling protein BAFF can promote the formation of neutralizing antibodies, or inhibitors, against blood clotting factor VIII (FVIII), which can lower the effectiveness of FVIII replacement therapies used to treat people with hemophilia A.  They also discovered that hemophilia A…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…