Gene Therapy is on everyone’s mind these days. We wonder about opportunities, clinical sites, neutralizing antibodies and most recently details of a small number of Serious Adverse Events (SAE) and what these learnings might mean to the inclusion criteria of gene therapy studies.

Over the last weeks, we have been gathering questions from the community about all of these issues expressing concern. Time is the factor for the community as every family weighs time and progression vs opportunity to participate.

We shared  our list of questions with  Pfizer and  a request – an urgent need – to understand what they are learning and the rationale for any changes to their current protocol as well as potential next steps, planned studies.

Our thanks to Dr. Beth Belluscio and to Pfizer for this update.