研究结果显示,dirloctocogene samoparvovec给药后7年的耐受性良好,FVIII持久表达,以及具有临床意义的ABR和AIR减少。 参考来源:Stacy E. Croteau, et al. Long-Term Follow-Up of Participants in the Phase I/II Trial of Dirlocto...
Treatment name: SPK-8011 Administration: Being tested in hemophilia A as a one-time intravenous infusion Clinical testing: Previously tested in a Phase 1/2 clinical trial; Phase 3 clinical trial underwayHow does SPK-8011 work in hemophilia?
Learn more about Spark® Therapeutics’ progress and explore our recent announcements and resources.For additional media inquiries, please contact: communications@sparktx.com or call 1-855-SPARKTX / +1 215-220-9300.Coming Soon: Spark®’s Gene ...
Methods : SPK-8011 is a recombinant AAV vector composed of a bio-engineered capsid (AAV-Spark200) with liver specific enhanced tropism and a codon-optimized expression cassette that encodes the SQ-FVIII variant of a B-domain-deleted (BDD) human F8 gene (Lind 1995). This Phase 1/2 study ...