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The novel gene therapy BAX 335 did not induce sustained factor IX expression among patients with hemophilia B, according to an interim analysis of a phase 1/phase 2 dose-escalation study published in Blood.
Patients with hemophilia, particularly in the most severe form, suffer from recurrent, sometimes life-threatening bleeding events, long-term disability and reduced life expectancy Bruce M. Ewenstein, MD, scientist at Brigham and Womens Hospital and part-time lecturer on medicine at Harvard Medical School, told HemOnc Today. Despite the significant progress in the development of factor VIII and factor IX [FIX] replacement products, as well as nonfactor products that partially overcome the hemostatic defects in hemophilia, there is widespread belief that gene therapy, by enabling persistent endogenous expression of the missing coagulation factors, has the potential to provide long-term benefit after a single administration. Early clinical trials demonstrated the feasibility of gene therapy for hemophilia B but failed to produce sustained therapeutic levels of FIX.
BAX 335 (Baxalta Inc.; Baxalta Innovations GmbH, Takeda) is an investigational gene therapy compromising an adeno-associated virus serotype 8 (AAV8) vector with the naturally occurring Padua human FIX variant gene.
The open-label, dose-escalation...