Intellia Achieves Gene-Editing Breakthrough With First In Vivo CRISPR Therapy

Promise Of Safe And Effective Gene Editing Takes Step Forward

The first use of CRISPR/Cas9 in humans shows promising safety and efficacy against rare disease ATTR, and opens up the possibility of treating many genetic diseases.

Intellia Therpeutics — Intellia's in vivo CRISPR therapies could rival other modalities, such as viral vector-based gene therapies and RNAi therapies. • Source: Intelllia

Landmark results from the first in vivo CRISPR gene-editing therapy have been unveiled by Intellia Therapeutics and Regeneron, and could put them at the forefront of a medical revolution.

Six patients living with hereditary transthyretin amyloidosis with polyneuropathy (hATTRv-PN) were given in the CRISPR/Cas9-based therapy candidate NTLA-2001 which ‘edited...

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