St. Jude, UCL gene therapy cuts bleeding in hemo B trial

Patients receiving scAAV2/8-LP1-hFIXco, a gene therapy developed by St. Jude Children's Research Hospital and University College London (UCL), maintained higher Factor IX levels and had markedly fewer spontaneous bleeding episodes in a Phase I dose-escalation trial to treat severe hemophilia B. The New England Journal of Medicine published the results on Nov. 20.

Six patients in the study's high-dose group who received single infusions of the modified adeno-associated virus (AAV) 8 vector encoding the Factor IX gene achieved circulating Factor IX levels averaging 5.1% of normal after one year, up from a baseline of <1%. All 10 patients receiving infusions had Factor IX levels 1-6% of normal, and maintained elevated levels for a median of 3.2 years. ...