By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1
A few months back, I wrote an article about Hemophilia A and how Spark Therapeutics’ administration of SPK-8011 in patients with hemophilia A resulted in an acceptable safety profile with no deaths and no FVIII inhibitor development with up to 4 years of follow-up. These data reinforce the ability of AAV gene therapy targeting hepatocytes to achieve stable and durable FVIII expression with an acceptable safety profile. A. Spark states that administration of SPK-8011 in patients with hemophilia A resulted in an acceptable safety profile with no deaths and no FVIII inhibitor development with up to 4 years of follow-up. Two of the 17 participants with over one year of data lost FVIII expression due to a presumed cellular immune response to the AAV capsid that was unresponsive to immunosuppression. Indeed, the company recently announced updated data from the ongoing Phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A during the International Society of Thrombosis and Hemostasis (ISTH) 2021 Virtual Congress. Spark retains global commercialization rights to its SPK-FVIII program includes both SPK-8011 and SPK-8016 for hemophilia A. These data demonstrated that administration of SPK-8011 in patients with hemophilia A resulted in sustained factor VIII (FVIII) expression in 16 of 18 participants with up to 4 years of follow-up, as of the May 3, 2021 data cutoff.
We’ve now released Episode 7 of Cell & Gene: The Podcast, which features Spark’s Dr. Tiffany Chang, Clinical Development Lead, Hematology. Dr. Chang and I spend the episode discussing Spark Therapeutics’ updated data from the ongoing Phase 1/2 clinical trial of investigational SPK-8011 for hemophilia A, the largest Phase 1/2 gene therapy trial in this disease to date. Dr. Chang details the data presented and why the advancements made to date are so critically important for patients with hemophilia
Dr. Chang and I discuss what is next for the trial, and we also discuss some of the misconceptions or unknowns about Hemophilia A. For example, one of the misconceptions Dr. Chang discusses is that while Hemophilia A is a genetic disorder, it is not always caused by an inherited, genetic trait. One third of Hemophilia A cases are new, and do not stem from family history.
Finally, Dr. Chang outlines what she believes her role as Clinical Development Lead, Hematology will look like in 3 to 5 years based on evidence and data.
Speaking of Cell & Gene: The Podcast, be sure to subscribe if you haven’t already. Each episode, I sit down with subject matter experts in cell and gene therapy to discuss what they’re working on why it matters to the sector at large, and most importantly, the patient. To date, we’ve released seven podcasts featuring luminaries from Enzyvant, University of Pennsylvania Health System, Cartesian Therapeutics, SwanBio, as well as Penn’s Dr. Bruce Levine and The CLL Society’s Bob Levis. Future episodes will include decision makers from Novartis Gene Therapies, AVROBIO, BridgeBio, and many, many more.