Business
Intellia Therapeutics Announces First Quarter 2021 Financial Results
Plan to report initial safety and activity data from Phase 1 study of NTLA-2001, a potentially curative single-dose therapy for transthyretin amyloidosis
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"Plan to report initial safety and activity data from Phase 1 study of NTLA-2001, a potentially curative single-dose therapy for transthyretin amyloidosis (ATTR), in mid-2021 On track to submit an IND or IND-equivalent in mid-2021 to advance NTLA-5001 for acute myeloid leukemia (AML) Expect to submit an IND or IND-equivalent in 2H 2021 for NTLA-2002 for hereditary angioedema (HAE)Ended the quarter with a strong cash position of $600.8 million CAMBRIDGE, Mass., May 06, 2021 (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 first quarter ended March 31, 2021. “Intellia continues to build momentum as we progress our full-spectrum pipeline towards key upcoming milestones this year. In mid-2021, we anticipate reporting initial clinical data for NTLA-2001 for ATTR, the first-ever systemically delivered CRISPR-based therapy. The company also plans on submitting first-in-human regulatory filings for NTLA-5001 for AML and NTLA-2002 for HAE, and remains on track to nominate at least one additional development candidate from our research efforts,” said Intellia President and Chief Executive Officer John Leonard, M.D. “During the first quarter, we shared important advancements that expanded the breadth of our modular platform, such as introducing our proprietary base editor and establishing preclinical proof-of-concept for systemic in vivo editing in bone marrow. We believe our platform innovations will drive future pipeline growth and expand our ability to develop potentially curative treatments for patients with a range of severe diseases.” First Quarter 2021 and Recent Operational Highlights NTLA-2001 for ATTR: NTLA-2001 is the first systemically delivered CRISPR-based therapy dosed in a patient and could potentially be a curative treatment for ATTR. By applying the Company’s in vivo lipid nanoparticle (LNP) delivery technology, NTLA-2001 offers the possibility of halting and reversing the disease with potent, lifelong transthyretin (TTR) protein reduction after a single administration. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party, and Regeneron Pharmaceuticals, Inc. (Regeneron). T...