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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
CAMBRIDGE, Mass. and TARRYTOWN, N.Y., June 26, 2021 /PRNewswire/ -- First-ever clinical data supporting safety and efficacy of in vivo CRISPR genome editing
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"CAMBRIDGE, Mass. and TARRYTOWN, N.Y., June 26, 2021 /PRNewswire/ -- \nFirst-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. 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. \n\"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 to treating a wide array of other genetic diseases with our...