Particle.news
Download on the App Store
5 ARTICLES
·
last wk.

Personalized CRISPR Therapy Sends Baby KJ Home from CHOP

FDA fast-tracked the six-month custom CRISPR therapy that corrected KJ’s CPS1 mutation, enabling normal protein metabolism with reduced drug support.

Image
Image
Image
Image

Overview

  • KJ Muldoon, a 10-month-old with severe carbamoyl phosphate synthetase 1 deficiency, went home from Children’s Hospital of Philadelphia on June 3 after a 307-day stay.
  • A multidisciplinary team at CHOP, the University of Pennsylvania and partner institutes designed a bespoke CRISPR-Cas9 base editor in six months to target his unique genetic mutation.
  • Following two lipid nanoparticle infusions at seven and eight months, KJ’s ammonia levels stabilized, allowing higher protein intake and lower medication doses.
  • His treatment was detailed in the New England Journal of Medicine and presented at the American Society for Gene and Cell Therapy in May.
  • Partners at CHOP, Penn, the Innovative Genomics Institute and Danaher secured FDA approval within one week and are developing similar bespoke therapies for other ultra-rare disorders.