Earlier this year, researchers in Philadelphia announced they’d developed a custom-made therapy to fix one infant’s rare genetic mutation. The news about Baby KJ opened the possibility of a new model for treating ultra-rare genetic diseases that might crack open the use cases for CRISPR gene editing.
In May, Endpoints News published the inside story of the six-month sprint to make the therapy. Now the researchers behind it — University of Pennsylvania scientist Kiran Musunuru and Children’s Hospital of Philadelphia pediatrician Rebecca Ahrens-Nicklas — have a plan to bring the treatment to more patients.
Today on Post-Hoc Live at 9:45 a.m. ET, Musunuru and Ahrens-Nicklas will talk with Endpoints Science Correspondent Ryan Cross and me about how they’re working with the FDA to turn Baby KJ’s one-off treatment into an approvable, and potentially reimbursable, procedure, bringing new hope to the promise of CRISPR cures.
You can join us on YouTube (click subscribe to get a reminder when we go live) or set a calendar reminder.
We’ll talk about that case, their plans to expand their approach, and what they think comes next for CRISPR.
CRISPR’s growing pains are far from over. But Baby KJ’s story and the further work that the toddler has inspired highlight the power of fixing diseases at the level of DNA and remind us why there was so much hype over CRISPR in the first place.
See you soon!
— Drew