Following earlier reporting, researchers aim to broaden access to the Baby KJ therapy by working with the FDA to convert the one-off CRISPR treatment into an approvable, potentially reimbursable procedure.

Viewers can watch the update live on YouTube or set a reminder, with related Endpoints coverage outlining plans to extend the approach to more children.

This development underscores CRISPR’s promise for ultra-rare genetic diseases while highlighting regulatory and development hurdles as it shifts from a case study toward broader clinical use.

The discussion will air on Post-Hoc Live at 9:45 a.m. ET, featuring Endpoints Science Correspondent Ryan Cross and the author to explore regulatory steps and expansion plans.

The therapy was developed by researchers from the University of Pennsylvania and Children’s Hospital of Philadelphia, Kiron Musunuru and Rebeca Ahrens-Nicklas, who crafted a custom CRISPR treatment for Baby KJ’s rare mutation.