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Intellia Therapeutics Highlights Strategic Priorities and Anticipated Development Milestones for 2021
Continued advancement of global Phase 1 study of NTLA-2001, a potentially curative single-course therapy for transthyretin amyloidosis (ATTR)Anticipates
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"Continued advancement of global Phase 1 study of NTLA-2001, a potentially curative single-course therapy for transthyretin amyloidosis (ATTR)Anticipates submitting an IND or IND-equivalent in mid-2021 for NTLA-5001 for the treatment of acute myeloid leukemia (AML)Expects to submit an IND or IND-equivalent in 2H 2021 for NTLA-2002 for the treatment of hereditary angioedema (HAE)Plans to nominate at least one new development candidate in 2021 from broad research efforts and continued platform innovation Ended 2020 in strong financial position with $597M in cash CAMBRIDGE, Mass., Jan. 07, 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 outlined its expected 2021 milestones and the following strategic priorities: Clinical validation: Evaluate the clinical profile of NTLA-2001 as a single-course therapy for transthyretin amyloidosis (ATTR) and Intellia’s in vivo non-viral, lipid nanoparticle (LNP)-based CRISPR/Cas9 delivery system as a platform for achieving clinically-relevant protein reduction for patients;Full-spectrum pipeline advancement: Rapidly progress in vivo and engineered cell therapy candidates for genetic diseases and cancers towards the clinic; andPlatform innovation: Extend Intellia’s continued scientific leadership across genome editing, delivery and cell engineering capabilities. “Since our founding, we set out to develop modular platform components that could serve as the engine powering an expansive portfolio of curative therapeutics. We have paved a rapid and reproducible development path for both in vivo and engineered cell therapies to address serious genetic diseases and cancers,” said Intellia President and Chief Executive Officer John Leonard, M.D. “Over the next 12 months, we will evaluate the clinical profile of NTLA-2001, both as a one-time treatment option for ATTR patients and as a validation of our non-viral approach to in vivo delivery. In addition, we anticipate first-in-human regulatory submissions for NTLA-5001 and NTLA-2002, at least one new development candidate and new platform innovations to create the next wave of genomic medicines. These priorities for 2021 reflect our long-term vision for Intellia: to unlock genome editing’s full therape...