Spark Therapeutics, Inc. has announced the initiation of a phase 1/2 trial of SPK-8011, the company’s investigational gene transfer candidate for hemophilia A.
The therapy entails a one-time intravenous infusion of SPK-8011, designed to trigger the production of therapeutic levels of factor VIII, a protein that is normally deficient in individuals with hemophilia A. Spark Therapeutics’ proprietary bioengineered adeno-associated viruses (AAVs) act as delivery vehicles, or vectors, to carry the genetic codes that prompt the FVIII production. The approach being tested in this trial uses a modified novel AAV vector to deliver the corrected FVIII gene into liver cells where the protein is normally made.
In collaboration with Pfizer, Spark Therapeutics has been conducting an ongoing AAV-based gene therapy for hemophilia B known as SPK-9001. In December 2016, Spark Therapeutics and Pfizer announced updated preliminary data from the first nine participants in its ongoing phase 1/2 clinical trial of SPK-9001. These data were presented at the 58th American Society of Hematology Annual Meeting in San Diego, CA, on December 4, 2016.
While the SPK-8011 study for hemophilia A is currently only open for recruitment at the Children’s Hospital of Philadelphia, there are plans for additional trial sites to open in the upcoming months. To learn more about this study or for specific inclusion/exclusion criteria go to clinicaltrials.gov and search by trial ID#: NCT03003533.
Source: Spark Therapeutics, Inc.
