In May 2025, clinicians at Children’s Hospital of Philadelphia and Penn Medicine successfully performed a personalized CRISPR-based therapy on a newborn diagnosed with CPS1 deficiency—a rare genetic disorder impairing ammonia clearance in the liver. Built on an adenine base-editing platform, the treatment was developed and administered within six months of diagnosis, stabilizing the infant’s condition and marking reportedly the first known case of a bespoke in vivo gene-editing intervention in a single patient.
While the CHOP–Penn therapy was built on established molecular and delivery technologies, it highlighted the pace that targeted interventions can now reach. For many, the next question is how such approaches might scale beyond bespoke development. One path involves increasing reliance on computational tools to guide design and reduce iteration time. Reflecting on that broader possibility in late 2024, CRISPR co-inventor Jennifer Doudna noted:
“It’s still early days, but the potential to appropriately harness the joint power of AI and CRISPR, arguably the two most profound technologies of our time, is clear and exciting—and it’s already started.”
In this article: The Cradle of Gene Editing—CRISPR Arrives—Life after CRISPR—AI-Powered Applications—Startups Applying AI—DNA Cleavage Layer—DNA-Base Editing — RNA Editing—Epigenetic Rewiring—CRISPR-Customising Layer—Policy, Funding, and the Future