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Intellia and Regeneron Announce Landmark Clinical Data Showing Deep Reduction in Disease - Causing Protein After Single Infusion of NTLA - 2001 , an Investigational CRISPR Therapy for Transthyretin ( ATTR ) Amyloidosis In ir.intelliatx.com/news-releases/news-release-details/intellia-and-regeneron-announce-landmark-clinical-data showing Jun 26 , 2021 • First - ever clinical data supporting safety and efficacy of in vivo CRISPR genome editing in humans • Interim readout in ongoing Phase 1 trial finds single 0.3 mg / kg dose of NTLA - 2001 led to 87 % mean reduction in serum TTR , with a maximum 96 % serum TTR reduction by day 28 , with dose - dependent response Encouraging safety profile ; no serious adverse events observed in the first six patients by day 28 • Data published in The New England Journal of Medicine and presented at Peripheral Nerve Society Annual Meeting ( PNS ) • Intellia to host investor event on Monday , June 28 at 8:00 a.m. E.T. CAMBRIDGE , Mass . and TARRYTOWN , N.Y. , June 26 , 2021 ( GLOBE NEWSWIRE ) -- Intellia Therapeutics , Inc. ( NASDAQ : NTLA ) and Regeneron Pharmaceuticals , Inc. ( NASDAQ : REGN ) today announced positive interim data from an ongoing Phase 1 clinical study of their lead in vivo genome editing candidate , NTLA - 2001 , which is being developed as a single - dose treatment for transthyretin ( ATTR ) amyloidosis . The Phase 1 study , run by Intellia as the program's development and commercialization lead , is evaluating NTLA - 2001 in people living with hereditary transthyretin amyloidosis with polyneuropathy ( ATTRv - PN ) . NTLA - 2001 is the first CRISPR / Cas9 - based therapy candidate to be administered systemically , via intravenous infusion , for precision editing of a gene in a target tissue in humans . NTLA - 2001 is designed to inactivate the TTR gene in liver cells to prevent the production of misfolded transthyretin ( TTR ) protein , which accumulates in tissues throughout the body and causes the debilitating and often fatal complications of ATTR amyloidosis . The interim data were presented today at the 2021 Peripheral Nerve Society ( PNS ) Annual Meeting and published in The New England Journal of Medicine ( nejm.org/doi/full/10.1056/NEJMoa2107454.)¹ . " These are the first ever clinical data suggesting that we can precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of CRISPR . The interim results support our belief that NTLA - 2001 has the potential to halt and reverse the devastating complications of ATTR amyloidosis with a single dose , " said Intellia President and Chief Executive Officer John Leonard , M.D. " Solving the challenge of targeted delivery of CRISPR / Cas9 to the liver , as we have with NTLA - 2001 , also unlocks the door 1/9