CRISPR Therapeutics

Stories

One-time CRISPR therapy keeps cholesterol lower for a full year in early trial

Reported Phase 1b CTX310 data on Nov. 9; additional Phase 1b cohorts ongoing

CRISPR Therapeutics' CTX310 cut triglycerides by 63% and ANGPTL3 by 65% a month after one infusion in 10 patients with severely high triglycerides, the company reported Nov. 9 at the American Heart Association meeting. The Phase 1b results follow a Phase 1a trial in which the same one-time treatment kept LDL cholesterol about 52% lower for a year.

Updated 2 days ago

The six-month cure: gene editing goes bespoke

New Capabilities

As of May 2026: Commercializing Casgevy globally

In August 2024, KJ Muldoon was born with a death sentence encoded in his DNA—a single broken letter among three billion that left his body unable to process protein. Six months later, he walked out of Children's Hospital of Philadelphia, cured by a gene-editing therapy designed, manufactured, approved, and delivered in 180 days.

Updated May 19