Researchers who created the bespoke CRISPR treatment for Baby KJ published their FDA interactions and described plans to translate the single‑patient emergency program into a replicable regulatory pathway for ultra‑rare, bespoke gene therapies. The team (University of Pennsylvania and Children’s Hospital of Philadelphia) laid out documentation and agency feedback that could guide academic and small‑biotech sponsors. Post‑hoc interviews with the Baby KJ investigators detailed operational lessons—from rapid vector design to regulatory submissions—aimed at converting one‑off investigational treatments into an approvable, reimbursable paradigm. The researchers emphasized close FDA collaboration and standardized dossiers as central to wider adoption. Their disclosures could accelerate clinical access strategies for individualized gene editing but will also amplify ethical, manufacturing, and reimbursement debates as regulators weigh how to evaluate highly personalized biologics at scale.