CRISPRMED27, April 19-22. 2027, Copenhagen, Denmark

AIRNA has taken a different route to correcting the genetic lesion behind alpha-1 antitrypsin deficiency (AATD). Its oligonucleotide AIR-001 restored...

Updated Phase 1 data presented at the 2026 American Society of Gene and Cell Therapy (ASGCT) meeting suggest that Fate Therapeutics’ off-the-shelf CAR...

When clinicians at the Children’s Hospital of Philadelphia (CHOP) treated baby KJ – born with a rare, often-fatal urea-cycle disorder – with a CRISPR...

Intellia Therapeutics has initiated a rolling biologics license application (BLA) with the U.S. Food and Drug Administration for lonvoguran ziclumeran...

A new regulatory framework proposed in February by the US Food and Drug Administration could dramatically reduce the time and cost of bringing...

A genome-wide CRISPR-Cas9 loss-of-function screening platform developed for primary human natural killer cells has identified several intracellular...