by Molly Coddington – Technology Networks –
CRISPR gene therapy is changing the future of rare genetic disorder treatments.
In May, news broke that a 10-month-old baby, KJ Muldoon, was doing well after receiving 3 doses of the world’s first personalized CRISPR base-editing therapy.
Baby KJ had been diagnosed with carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after his birth in August 2024. In a mere six months, scientists across academia and industry developed and manufactured a novel, custom therapy that will hopefully help KJ avoid a liver transplant long-term. It may also provide a roadmap for transforming CRISPR therapies for other inborn errors of metabolism.
