CSL and uniQure's Hemgenix shows sustained efficacy in hemophilia B, reducing bleeding rates by 90% and eliminating ...

The frequency of mutations in the factor VIII or IX gene is consistent. However, it turns out that anyone can be affected by hemophilia. Hemophilia could potentially develop in every ten ...

These Gene Editing Treatments Could Come Next ... Generally, patients must receive monthly infusions of Factor IX, the missing protein. But Pfizer says Beqvez is a one-time treatment that will ...

Four years after receiving Hemgenix in a Phase 3 trial, bleeding rates and the use of replacement therapy are low for men ...

The adeno-associated virus (AAV) vector-based therapy is given as a one-shot intravenous infusion, delivering a gene for Factor IX, a clotting factor that is deficient in haemophilia B.

“The gene you need — for factor VIII or factor IX — is surrounded by the viral capsid. The viral vector product is administered to the recipient via peripheral IV over 1-2 hours.” Infusion ...

Fidanacogene elaparvovec is a novel, investigational vector containing a bio-engineered adeno-associated virus capsid and a high-activity human coagulation factor IX gene. A lack of the gene ...

Avigen is investigating the use of AAV vectors to deliver the factor IX gene for the treatment of hemophilia B and the factor VIII gene for hemophilia A. Preclinical efficacy and safety studies in ...

CSL Behring has shared positive four-year results from a late-stage study of its haemophilia B gene therapy Hemgenix ...

The study shows that 94% of patients eliminated factor IX prophylaxis and maintained near-normal factor IX levels through ...

CSL Behring's gene therapy Hemgenix four years post-infusion data continue to show sustained efficacy and safety in adults with haemophilia B: King of Prussia, Pennsylvania Monday ...