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 ...

involves a one-time infusion of a gene therapy medicine called Hemgenix, which contains a modified virus carrying a functional copy of the factor IX gene. The virus carries the functional copy to ...

Global biotechnology leader CSL Behring (ASX:CSL; USOTC: CSLLY (OTC:CSLLY)) announced the four-year results from the HOPE-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 ...

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 ...

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 ...

UC San Diego announced that it is offering a newly Food and Drug Administration-approved gene therapy for hemophilia B, a ...