Business
Intellia Therapeutics Highlights Recent Progress and Anticipated 2020 Milestones
On track to submit IND application for lead candidate, NTLA-2001 for transthyretin amyloidosis, in mid-2020 and dose first patients in 2H 2020Nominated
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"On track to submit IND application for lead candidate, NTLA-2001 for transthyretin amyloidosis, in mid-2020 and dose first patients in 2H 2020Nominated NTLA-5001 for the treatment of acute myeloid leukemia as first WT1-TCR-directed engineered cell therapy development candidate; plan to submit IND in 1H 2021 Announced third development program, which will be for treatment of hereditary angioedema; expects to nominate a development candidate in 1H 2020 Ended 2019 with strong cash position of $284 million; cash runway extended through YE 2021 CAMBRIDGE, Mass., Jan. 09, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, today provided an update on recent progress and the Company’s 2020 priorities and expected milestones.\n “2020 will be a significant year for Intellia, as we execute on our full-spectrum strategy. With milestones anticipated across our pipeline, we are making important progress towards the development of curative treatments for severe diseases. In particular, we expect to dose ATTR patients with the first-ever systemically delivered CRISPR/Cas9-based therapy this year, and we are beginning IND-enabling activities for our newly announced development candidate, NTLA-5001, a WT1-TCR-directed engineered cell therapy, for treatment of AML,” said Intellia President and Chief Executive Officer, John Leonard, M.D. “We are focused on developing a robust platform with modular genome editing capabilities that enable a fast and reproducible path to development. Today’s update reflects this strategy, and it also features the announcement of our third development program, an in vivo knockout approach for HAE. Importantly, this program leverages the infrastructure and insights from NTLA-2001 and underscores our ability to produce a rapid succession of new clinical candidates. We are excited by the strong momentum across our diverse pipeline and look forward to providing updates on our development programs in the upcoming year.” Program Updates and Anticipated 2020 Milestones: 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 treatment of transthyretin amyloidosis (ATTR). NT...