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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