An insightful perspective on what biological factors may have been the cause of a patient’s death after receiving a blood-brain-barrier crossing AAV treatment. It’s crucial for the field to think about this carefully as we move forward.
Brief disclosure: I am a named inventor on patents and author on publications related to AAV capsid engineering and CNS gene delivery, developed during my time at the Broad Institute. I now operate independently. This post does not represent any prior employer, current advisory client, or collaborator. The mechanistic analysis presented here is my own scientific interpretation of publicly available data. Full disclosures at the bottom.