What is hemophilia B and Hemgenix gene therapy? Hemophilia B is a life-threatening disorder caused by a mutation on the F9 gene, resulting in low levels of functional clotting factor IX.

More information: Ulrike M. Reiss et al, Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B, New England Journal of Medicine (2025). DOI: 10.1056/NEJMoa2414783 ...

Pfizer's hemophilia therapy, Hympavzi, met the main goal of a late-stage study in patients with certain types of antibodies, ...

The first patient in the UK has been treated by the NHS with CSL Behring’s haemophilia B gene therapy Hemgenix (etranacogene ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children’s Research Hospital and University College ...

Hemophilia B, an X-linked recessive bleeding disorder, arises from mutations in the gene F9 that lead to reduced production of functional coagulation factor IX protein.

Primary Source New England Journal of Medicine Source Reference: Reiss UM, et al "Sustained clinical benefit of AAV gene therapy in severe hemophilia B" N Engl J Med 2025; DOI: 10.1056/NEJMoa2414783.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the most undervalued US stocks according to analysts. On June 24, ...

Approved eight months ago for a certain class of patients with both hemophilia A or B, Pfizer’s Hympavzi has now shown its ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children’s Research Hospital and University College London ...

J.R. Landry became one of only a handful in the nation, and the first in Rhode Island, to get what doctors believe will be ...

uniQure and CSL have become the first pharma companies to get regulatory approval for a gene therapy for haemophilia B anywhere in the world, after the FDA cleared their Hemgenix therapy for ...