Business
Intellia Therapeutics to Present Interim Clinical Data from Ongoing Phase 1 Study of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis at the 2021 Peripheral Nerve Society Annual Meeting
Data to offer insight into safety and pharmacodynamics of NTLA-2001, the first-ever systemically administered in vivo CRISPR therapy candidateLate-breaking
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"Data to offer insight into safety and pharmacodynamics of NTLA-2001, the first-ever systemically administered in vivo CRISPR therapy candidateLate-breaking abstract selected for oral presentation on June 26 CAMBRIDGE, Mass., June 04, 2021 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA) today announced that a late-breaking abstract featuring interim Phase 1 clinical data from NTLA-2001, the Company’s lead CRISPR/Cas9 in vivo therapy in development as a single-dose, systemically administered treatment for transthyretin (ATTR) amyloidosis, has been selected for an oral presentation at the 2021 Peripheral Nerve Society (PNS) Annual Meeting, taking place this month. The presentation will include interim data from the ongoing dose-escalation portion of Intellia’s Phase 1 trial evaluating NTLA-2001 in people living with hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN). Following safety assessment and dose optimization, Intellia intends to further evaluate NTLA-2001 in a broader population of people with ATTR amyloidosis, including those with cardiomyopathy. “These findings represent Intellia’s first clinical data readout and an important step forward in our commitment to develop breakthrough genome editing treatments for people living with severe diseases, such as transthyretin (ATTR) amyloidosis,” said Intellia President and Chief Executive Officer John Leonard, M.D. “By knocking out the disease-causing gene, NTLA-2001 is designed to halt progression and potentially reverse the disease with a single dose, offering the potential of meaningful improvement over the standard of care, which requires chronic, lifelong administration. These interim data will provide a view of NTLA-2001’s safety and activity profile as the dose-escalation portion of our study progresses, as well as insight into the promise of our modular platform to develop other systemically delivered in vivo CRISPR/Cas9 therapies across a range of diseases with unmet need.” Presentation DetailsTitle: “In vivo CRISPR/Cas9 Editing of the TTR Gene by NTLA-2001 in Patients with Transthyretin Amyloidosis”Session: Platform Session II Date and Time: Saturday, June 26, 2021 from 11:15-11:30 a.m. E.T.Presenter: Dr. Julian Gillmore, M.D, Ph.D., FRCP, FRCPath, Professor of Medicine, National Amyloidosis Centre, UCL Division of Medicine, Royal Free Hospital, U.K...