Researchers Develop Custom Therapy for Rare Genetic Mutation in Infant
Earlier this year, a groundbreaking discovery was made by researchers in Philadelphia who successfully developed a custom-made therapy to fix a rare genetic mutation in an infant known as Baby KJ. This remarkable achievement has opened up new possibilities for treating ultra-rare genetic diseases and has the potential to revolutionize the use of CRISPR gene editing technology.
Back in May, Endpoints News shared the inside story of the six-month sprint to create this innovative therapy. Now, the brilliant minds behind this breakthrough, University of Pennsylvania scientist Kiran Musunuru and Children’s Hospital of Philadelphia pediatrician Rebecca Ahrens-Nicklas, have outlined a plan to make this treatment available to more patients.
Join Us for Post-Hoc Live Discussion
Today on Post-Hoc Live at 9:45 a.m. ET, Musunuru and Ahrens-Nicklas will be joining Endpoints Science Correspondent Ryan Cross and myself to discuss their collaboration with the FDA in order to transform Baby KJ’s unique treatment into an approvable and potentially reimbursable procedure. This development brings renewed hope to the field of CRISPR cures.
You can join us on YouTube (click subscribe to receive a reminder when we go live) or set a calendar reminder to tune in.
During the discussion, we will delve into the specifics of Baby KJ’s case, their plans for expanding their approach, and their insights on the future of CRISPR technology.
Embracing the Potential of CRISPR
While CRISPR technology continues to face challenges, Baby KJ’s story and the ongoing work inspired by this toddler underscore the immense potential of correcting genetic diseases at the DNA level. These advancements serve as a powerful reminder of why there was so much excitement surrounding CRISPR in the first place.
We look forward to seeing you there!
— Drew
