News

Hemophilia gene therapy Beqvez reduces bleeds in Phase 3 trial

Hemophilia gene therapy Beqvez (fidanacogene elaparvovec), marketed as Durveqtix in the European Union, results in stable production of factor IX (FIX), the clotting protein that is missing or faulty in hemophilia B, leading to about three times fewer bleeding episodes compared with prophylaxis, or preventive treatment, with regular infusions…

Marstacimab recommended for EU approval for hemophilia A and B

A European Medicines Agency (EMA) committee has issued a positive opinion recommending the approval of marstacimab in the European Union for preventing bleeding episodes in people with severe hemophilia A or hemophilia B without inhibitors. The European Commission will now review the recommendation, made by the…

No real COVID-19 infection impact seen for hemophilia patients

Having a COVID-19 infection had no significant impact on bleeding episodes, nor on the levels of factor replacement therapy used in treatment, for people with hemophilia, a new study from China found. The study did reveal, however, that, compared with adults, children with hemophilia had a significantly lower…

MGX-001 gene-editing therapy shows promise in preclinical study

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…