ISTH 2023 | Improved joint health following dirloctocogene samoparvovec gene therapy in patients with hemophilia

So SPK-8011 or dirloctocogene samoparvovec is an AAV based drug for hemophilia A. We’ve previously presented and published the initial safety and efficacy data demonstrating sustained factor VIII levels in the mild range and marked reduction in the annualized bleeding rate both in participants that were on on-demand treatment prior to gene therapy but also on participants that were previously on prophylaxis with factor VIII replacement. And so the presentation at ISTH 2023 was focused on the changes in joint health after a gene therapy. And so we use two standardized instruments to look at joint health. So one is the Hemophilia Joint Health Score or HJHS, and the second is the Hemophilia Activities List or HAL. So the HJHS measures joint health in the bleeds most associated with hemophilia so the elbows, knees and ankles and then the HAL is a self-reported marker instrument to look at functional ability. And what we observed was that in most patients the HJHS stayed the same or got better, though there were a few outliers, and similarly in the HAL most participants stayed the same or got better. And interestingly for the HAL, the patients whose baseline was a little bit lower showed more marked improvement. And so we’re very excited that not only do you have a decrease in bleeding, but that patients with joint disease have the potential to get better after gene therapy. So gene therapy is part of the expanding armamentarium for hemophilia care, I think it has the potential to really change some patients’ lives and how they experience hemophilia. I don’t think it’s going to be right for everyone and it’s not going to be available to everyone, but I think it’s a really important piece in our therapeutic arsenal.