NIH Working Group to Better Understand Immune Response in Gene Therapy Trials
In March 2022, Dr. Francessco Muntoni, MD, Great Ormond Street Institute of Child Health in London and Dr. Carsten Bonnemann, MD, National Institute of Health, brought together four companies with ongoing microdystrophin gene therapy programs to discuss recent Serious Adverse Events (SAEs). The objective of the working group is to share data and better understand the cause of these SAEs and how we may be able to treat those patients with genetic variants that may be at higher risk for an SAE.