Freeline gives detailed look at hemophilia gene therapy results

 An experimental gene therapy for hemophilia B boosted blood clotting protein levels in a small group of patients with a severe form of the rare bleeding condition, allowing all but one to discontinue regular preventive treatment, study results published Thursday in The New England Journal of Medicine show. 

After a median follow-up of more than two years, nine of the ten men who were treated in the clinical trial had sustained clotting protein activity, including five whose levels were within what’s considered a normal range. One participant initially saw his levels rise, but had to resume receiving other treatment a year later because the gene therapy’s effects waned. 

Eight participants experienced fewer bleeding episodes following treatment than before, according to the results. Bleeding episodes, even minor ones, can be dangerous in people with hemophilia B who lack or have low levels of the protein, called factor IX, that helps clot blood. 

Prior to their publication Thursday, data from the study were disclosed last December by the gene therapy’s maker, Freeline Therapeutics, and presented then at a prominent conference for research on blood disorder treatments. Study authors wrote that the results seen thus far warrant further investigation of the therapy to determine the best dose and immune-suppressing regimens. Freeline, a London-based drugmaker, has advanced its therapy, dubbed FLT180a, into another early-stage study after choosing a target dose in the middle of the four it tested previously. 

While promising, the results also point to questions that still need to be answered. All patients received drugs meant to limit their immune system’s response to treatment, which is delivered via an inactivated synthetic virus. These medicines have side effects, and investigators observed immune responses after their withdrawal. The one study participant who needed to resume preventive treatment also received another round of immunosuppressive drugs following an increase in liver enzyme counts. 

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