Business
Intellia Therapeutics Announces Third Quarter 2019 Financial Results
On track to submit in mid-2020 an investigational new drug application for NTLA-2001 for the treatment of transthyretin amyloidosis Expects to nominate its
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"On track to submit in mid-2020 an investigational new drug application for NTLA-2001 for the treatment of transthyretin amyloidosis Expects to nominate its first T cell receptor-directed engineered cell therapy development candidate for the treatment of acute myeloid leukemia by the end of 2019 Presented preclinical data including first demonstration of consecutive in vivo gene knockout and insertion for the treatment of alpha-1 antitrypsin at the 2019 European Society of Gene and Cell Therapy Annual MeetingEnds quarter with strong cash position of $296 million CAMBRIDGE, Mass., Oct. 31, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), reported operational highlights and financial results for the third quarter ended September 30, 2019.\n “In 2019, we continued to leverage the breadth of our genome editing platform to advance our in vivo and engineered cell therapy programs. We have demonstrated that we can knock out a disease-causing gene as well as introduce a functional gene to restore normal protein production. Now, we have achieved consecutive editing in vivo by combining both these edit types, further highlighting the versatility of our modular platform,” said Intellia President and Chief Executive Officer, John Leonard, M.D. “Our full-spectrum strategy and platform capabilities are enabling Intellia’s development of a robust pipeline to address a range of severe diseases. We look forward to the planned nomination of our first engineered cell therapy development candidate for acute myeloid leukemia by year-end and the submission of our first IND application for NTLA-2001 for the treatment of transthyretin amyloidosis in mid-2020.” Third Quarter 2019 and More Recent Operational Highlights ATTR Program: Intellia remains on track to submit in mid-2020 an investigational new drug (IND) application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR). NTLA-2001 is anticipated to be the first systemically delivered CRISPR/Cas9 therapy to enter the clinic. As part of an ongoing durability study of its lead lipid nanoparticle (LNP) formulation in support of NTLA-2001, Intellia has demonstrated 10 months of durable liver editing with sustained reduction of circulating transthyretin (TTR) protein (average reduction >95%) following a single dose in non-human primates. In preparation for the submission...