New Era of Medicine: Researchers Publish First Positive Clinical Data for In Vivo Genome Editing in Humans

Researchers from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners have published the first-ever clinical data that support the safety and efficacy of in vivo CRISPR genome editing in humans.

The team of researchers based in the U.S., the U.K., and New Zealand showed in a study that the companies’ lead in vivo genome editing candidate NTLA-2001 generated a dose-dependent sustained reduction of protein linked to transthyretin (ATTR) amyloidosis following a single dose in six patients living with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).