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First Positive Data of In Vivo CRISPR-Based Genome Editing in Humans Reported June 28, 2021 A team of researchers based in the U.S., the U.K., and New Zealand from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners 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). The study is the first ever to support the safety and efficacy of in vivo CRISPR genome editing in humans. The New England Journal of Medicine and in a presentation that day at the 2021 Peripheral Nerve Society (PNS) Annual Meeting the researchers reported that a single 0.3 mg/kg dose of NTLA-2001 led to an 87% mean reduction in serum transthyretin (TTR) protein concentration in three of the ATTRv-PN patients by day 28, with individual re

New zealandJohn evansDavidr liuGeorged yancopoulosMani forooharMaury raycroftJulian gillmoreJohn leonardPeripheral nerve societyUniversity college londonRegeneron pharmaceuticalsBroad instituteDepartment of neurologyNational amyloidosis centreAlnylam pharmaceuticalsDivision of medicine