Adverse Events Consortium


Duchenne gene therapy developers and scientific experts discuss AAV in DMD: Antitransgene SAEs. Please watch this webinar to learn more about recent concerns in gene therapy clinical trials.


There have been some severe adverse events (SAEs) in a small number of participants in AAV-micro-dystrophin gene therapy trials in DMD.  These complications are due to patients having an immune response to the treatment, and do not appear related to a specific AAV serotype, nor to a particular mini/micro-dystrophin transgene. 

In response, some DMD gene therapy trial protocols are excluding participants with specific types of mutations which might make them more susceptible to the immune complications.  In addition, the companies currently conducting gene therapy trials in DMD are working together to share safety data and identify preventative strategies. 

The organizers of this collaborative effort and the sponsors of the DMD gene therapy trials will be holding a webinar for the DMD community, in which they will present the information about the recent SAEs and share more details of how the various stakeholders are working together.  The webinar will be recorded for viewing by all of the DMD community, and will cover:

  • Summary of the SAEs
  • Overview of the mechanisms underlying the immune complications
  • Steps taken to make participants safe and reasons behind exclusion of specific genotypes
  • Ongoing scientific collaboration to improve our understanding on the implications of these SAEs for the field
  • Efforts to move towards a plan to eventually include all patients in studies

If you have specific questions you’d like to hear addressed in the webinar, we have the opportunity to submit them to the webinar participants in advance. We invite your input.

Please share your questions or concerns with us at

Related Posts

Share This Page

Make an Impact

You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.