Capsida's Trailblazing Moment: What the Field Owes the Next BBB Program
By Fatma Elzahraa Eid , PhD | TheBioMLClinic 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. A child died. Brain swelling (cerebral edema) within days of receiving a gene therapy that had cleared every preclinical safety bar the field knows how to set. The IND had FDA clearance. The Good Laboratory Practice (GLP) toxicology study showed no adverse histopathology. The capsid achieved greater than 70% neuronal transduction across critical brain areas in nonhuman primates. By every metric the field currently uses, this program was ready. And yet. This post is not about assigning blame. Cap...