Small UK biotech touts long-term data for hemophilia B gene therapy as it attempts a comeback
As it continues pushing its hemophilia B gene therapy toward the FDA, small biotech Freeline Therapeutics is hoping new long-term data will boost its case.
A single shot of Freeline’s gene therapy, called FLT180a, led to durable responses for at least two years, according to researchers from Freeline and the University College London. Nine of 10 patients, all adult men, maintained their factor IX activity at a median follow-up of 27.2 months, with one patient seeing a response as far out as 42.4 months. Researchers published their findings in the New England Journal of Medicine.
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