Developing a gene therapy typically takes years, but when Baby KJ was diagnosed with a deadly genetic condition, scientists had only months. What followed was a successful collaboration among ...

Casgevy sickle cell gene therapy is now FDA-approved for children as young as two, opening access for 5,500 more U.S. kids.

CRISPR Therapeutics had its first FDA-approved product in December 2023, a treatment for sickle cell disease called Casgevy. It’s also developing treatments for cancer and cardiovascular disease using ...