A gene therapy designed to provide hemophilia A patients with a highly functional version of factor VIII (FVIII), the blood clotting protein they lack, could offer more sustained bleed control than existing gene therapies, preclinical research suggests. The current approach in hemophilia A gene therapy is to provide a…
MGX-001, a gene-editing therapy being developed by Metagenomi for hemophilia A, has shown promising safety and durability in an ongoing preclinical study in three nonhuman primates, the company has announced. Results showed that after one year, two animals had normal (82%) or nearly normal activity levels (41%) of…
A team of U.S. researchers has developed a new gene-editing strategy that could make the approach safer and more efficient for treating certain diseases, such as hemophilia. In preclinical studies, the gene-editing technique was shown to enable fast and efficient delivery of healthy genetic material to cells, while bypassing…
Prescriptions for extended half-life products to hemophilia A patients in Japan increased from 2016 to 2020, according to a new study that analyzed real-world data from a hospital database. This was accompanied by an increase in healthcare costs seen for patients switching from standard half-life factor replacement therapies to…
Long-term prophylaxis or preventive treatment with Eloctate (efmoroctocog alfa) helps to ease pain and improve health-related life quality for adults and children with hemophilia A, according to an analysis of three Phase 3 clinical trials. “These analyses highlight the relevance of pain, physical health, and overall wellbeing as…
More than 1 in 10 men with hemophilia reported being restricted in their sexual functioning, which, in turn, had a negative impact on their quality of life, per data from a new survey-based study conducted in the Netherlands. The study also found that this…
A single dose of Roctavian (valoctocogene roxaparvovec-rvox) nearly zeroed out the number of bleeds and the need for prophylaxis, or preventive therapy, for as long as seven years — the longest follow-up to date for any hemophilia A gene therapy clinical trial. While most of the 12 patients…
The incidence of acquired hemophilia A (AHA) was nearly twice as high in Hong Kong than in Caucasian populations previously observed, a study found. Patients in Hong Kong tended to be older and have co-existing medical conditions, with age being a significant predictor of mortality and failure to achieve…
Brazilian hemophilia A patients who are 19 or older had significantly higher median care costs per patient per year than younger patients, a study reported. The study also revealed an increase in median costs for patients with more severe disease — that is, those who have less than 1%…
In an effort to cut costs and boost profitability, Biomarin Pharmaceutical is limiting commercial development of hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox) to three countries: the U.S., Germany, and Italy. The company has struggled to turn a profit from the gene therapy, which was approved in…
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