A New Chapter in Rare Disease: Inside the First Custom CRISPR Treatment

In 2025, a six-month-old with a fatal genetic disorder became the first person to receive a CRISPR-based therapy tailored to their unique mutation. In this episode of Curie, host Mike unpacks how a team of researchers and clinicians raced against time to design, test, and deliver a one-time treatment that rewrote the rules of genomic medicine. From the science of base editing to the ethical and logistical hurdles, this is the story of what it took to make personalized gene therapy a reality—and what it means for the future of rare disease care.