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New Data Boost For Intellia’s Pioneering In Vivo Gene-Editing Therapy

Next Step: Pivot To Cardiomyopathy To Take On Pfizer

 
• By Andrew McConaghie

New data show that a high dose of Intellia’s in vivo CRISPR-Cas9 based therapy is safe and effective for up to six months, which could unlock a new era in genetic disease treatment.

Intellia HQ
Intellia looks to have a potentially curative ATTR amyloidosis gene therapy on its hands, but must replicate the early results in a pivotal study. • Source: Alamy

Updated results from Intellia Therapeutics and Regeneron’s CRISPR Cas9-based in vivo gene-editing candidate NTLA-2001 have boosted confidence that it could be a major breakthrough in treating genetic diseases.

NTLA-2001 is the first CRISPR-based therapy candidate to be administered systemically to patients to permanently edit

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