08 January 2018

A single gene therapy treatment that produces a clotting factor has safely stopped bleeding in haemophilia B patients in a phase 1/2 clinical trial in the USA.

The intravenous infusion of a novel bioengineered gene therapy treatment resulted in the cohort of ten adult patients terminating prophylactic treatments, say the researchers from the Children's Hospital of Philadelphia.

The gene for Factor IX-Padua (FIX-Padua), a naturally occurring hyperfunctional clotting factor, was delivered to patients via an adeno-associated viral vector.  

Following treatment, the ten study participants maintained factor levels of approximately 33%, which was sufficient to move them out of the severe disease category.

In addition, the patients did not suffer the painful, disabling joint bleeds that previously occurred when their clotting factor levels dropped and the researchers found the clinical benefits continued for up to 78 weeks of follow up.

None of the ten patients had serious adverse events from the gene therapy and eight of the ten did not require external doses of factor. Furthermore, nine of the group did not experience bleeds after the vector infusion.

Dr Lindsey George and colleagues published their findings in the New England Journal of Medicine.

"We look forward to continuing the development of this therapy in a Phase 3 trial," said Dr George. "I am optimistic this study is just the beginning of a true paradigm shift in the treatment of haemophilia."

Source: Lindsey A. George, et al. Hemophilia B Gene Therapy with a High Specific Activity Factor IX Variant. New England Journal of Medicine 7 December 2017.

Link: http://www.nejm.org/doi/full/10.1056/NEJMoa1708538


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