In a May 27th letter to the hemophilia community, it was confirmed that Spark Therapeutics has been fully integrated into the Roche Group. Spark first became a member of the Roche Group in 2019 with the aim of continuing to advance potential gene therapies for hemophilia and many other genetic diseases.
As part of this development, U.S. Genentech (a current member of the Roche Group) will now be the point of contact for the hemophilia A gene therapy program. This includes dirloctocogene samoparvovec (SPK-8011), an investigational therapy that was developed by Spark and continues to be evaluated in phase I/II clinical trial of hemophilia A patient participants.
Administered via a one-time intravenous infusion, SPK-8011 is designed to elicit the production of therapeutic levels of the factor VIII (FVIII) protein in individuals with hemophilia A. Bioengineered adeno-associated virus (AAV) vectors are used to carry the genetic codes that help generate FVIII. The approach being tested in this trial uses a modified novel AAV vector genome to deliver the corrected FVIII gene into liver cells where the protein is normally generated.
Long term monitoring and study of these phase I/II trial participants will be ongoing and individuals with questions are encouraged to contact a participating HTC trial site.
The letter also reminded the community of the companies’ decision to introduce an enhanced function factor VIII variant (SPK-8011QQ) into the hemophilia A gene therapy program. This “next generation” gene therapy is planned to be studied in a Phase 2b clinical trial sponsored by both Roche and Genentech. This study will be focused on gathering safety data in the lead up to a larger phase III trial.
Any questions about the Spark integration and/or implications for these hemophilia gene therapy programs can be sent to infoadvocacyrelations-d@gene.com.
NBDF will continue to report any new developments relevant to these and other investigational hemophilia gene therapies.
