Business
Intellia Therapeutics Announces Fourth Quarter and Full-Year 2019 Financial Results
On track to submit an IND application for NTLA-2001 for the treatment of transthyretin amyloidosis in mid-2020 and to dose first patients in 2H 2020Plans to
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"On track to submit an IND application for NTLA-2001 for the treatment of transthyretin amyloidosis in mid-2020 and to dose first patients in 2H 2020Plans to submit an IND application for NTLA-5001, a WT1-directed TCR-T cell therapy, for the treatment of acute myeloid leukemia in 1H 2021Expects to nominate a development candidate for the treatment of hereditary angioedema in 1H 2020Ends 2019 with a strong cash position of $284 million; cash runway through YE 2021 CAMBRIDGE, Mass., Feb. 27, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, today reported operational highlights and financial results for the fourth quarter and year ended December 31, 2019.\n “In 2019, we advanced our full-spectrum strategy, guiding both our in vivo and ex vivo lead programs toward the clinic. We also continued to build on our genome editing and delivery capabilities to enable a rapid succession of candidates,” said Intellia President and Chief Executive Officer, John Leonard, M.D. “We are off to a productive start in 2020. We announced the nomination of NTLA-5001, a WT1-directed TCR-T cell therapy for the treatment of AML, and plan to select our third development candidate in the first half of this year, which will be for the treatment of HAE. In addition, in the second half of the year, we expect to begin dosing ATTR patients with NTLA-2001, a potential single-course treatment for ATTR patients. This is anticipated to be the first-ever systemically delivered CRISPR/Cas9-based therapy to enter the clinic, representing an important milestone in our mission to deliver potentially curative therapies from our proprietary modular platform.” 2019 and Recent Operational Highlights ATTR Program: Intellia remains on track to submit an investigational new drug (IND) application in mid-2020 for its lead in vivo candidate, NTLA-2001, for the treatment of transthyretin amyloidosis (ATTR). In December 2019, Intellia completed a 12-month durability study of its lead lipid nanoparticle (LNP) formulation in support of NTLA-2001, maintaining an average reduction of >95% of serum transthyretin (TTR) protein and sustained liver genome editing after a single dose in non-human primates (NHPs). NTLA-2001 is anticipated to be...