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Aurora magazine

New genetic therapy for type B hemophilia

The first phase of a clinical trial for a new gene therapy against Type B hemophilia is underway. Researchers have also recruited 5 new patients to be given a new version of the therapy. For the time being, the researchers used SPK-9001 on only one patient, who is around the 35th week of follow-up. According to the first data, however, the results obtained from the new process are comparable to those obtained with the old one.

Gene therapy SPK-9001 should help those with type B hemophilia to produce factor IX. For the time being, neither the first 10 participants nor what they added at a later date showed particular side effects. They have also stopped concentrate IX concentrate infusions, which also makes good hope for efficacy.

All patients showed an increase in factor IX levels, making infusions of the same high. Data on the 7 patients who completed a one-year follow-up show a lasting efficacy of therapy. None of them has shown any cases of bleeding, even without the use of preventative prophylaxis. Everyone has reported an improvement in the quality of life, which is good for the future.

The new version of SPK-9001 will be used in phase 3 of the clinical trial. If this stage is fine, gene therapy will be ready for approval and subsequent marketing.