Roche announces latest developments in development of type A haemophilia Gene therapy to maintain coagulation factor levels

Hemophilia Type A affects the lives of 320,000 patients worldwide, resulting in insufficient levels of coagulation factor VIII in the body due to mutations in the gene that encodes coagulation factor VIII( For them, minor injuries can lead to pain and potentially life-threatening bleedingSevere haemophiliacs usually bleed spontaneously in their muscles and joints, which can cause permanent joint damageAt present, the standard treatment for severe haemophilia type A is preventive intravenous coagulation factor VIIINot only do patients need treatment 2-3 times a week, but even if treated, bleeding events can still occur, which can have a significant impact on their quality of lifeMoreover, some patients because of long-term infusion coagulation factor VIII, external coagulation factor VIII to produce antibodies (coagulation factor VIII inhibitors), so that the coagulation factor VIII alternative therapy failureThe gene therapy SPK-8011 developed by Roche's Spark Therapeutics is a genetically modified gene therapy that uses adeno-associated viral vectors (AAVs) to deliver to the liver a genetically modified gene that expresses coagulation factor VIIIIt is the first time Roche has announced the latest advances in this gene therapy since acquiring Spark TherapeuticsThe maximum follow-up time of the five patients initially treated with gene therapy reached 142 weeks, according to the press releaseThe latest data showed a 91 per cent reduction in the annual haemorrhage rate (ABR) in patients, and no evidence of a decrease in the expression level of coagulation factor VIII over a period of more than two years of follow-upMoreover, no patients were found to have coagulation factor VIII inhibitors, Roche will for the first time announce the results of a phase 3 clinical trial of the company's hemophilia bispecific antibody therapy Hemlibra (emicizumab-kxwh, Amezumab-kxwh) in patients with type A haemophilia in the Asia Pacific regionHemlibra has been approved by China's State Drug Administration (NMPA) for accelerated approval in November 2018This phase 3 test is a post-approval verification testresults showed that hemlibra reached the primary endpoint of the trial in 70 patients with type A haemophilia in the Asia Pacific region who carried clotting factor VIII inhibitorsThe number of bleeding events requiring acute treatment is reduced by 96 per cent (p 0.0001) of hemlibra preventive treatment on a weekly or quadofed basis, compared to those who do not receive preventive treatmentAt the same time, the experiment reached all critical secondary endpointsOverall, the trial data showed that Hemlibra showed good efficacy and tolerance in this patient population"We are pleased to be able to share the progress of the Type A haemophilia research and development program at the ISTH annual meeting," said DrLevi Garraway, Roche's Chief Medical Officer and Head of Global Product Development"