On Jnauary 5, 2023 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, reported its strategic priorities for the upcoming two years as the Company enters its next phase of pipeline execution and platform innovation (Press release, Intellia, JAN 5, 2023, View Source [SID1234625900]).

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2023 – 2024 Strategic Priorities

Initiate global pivotal trials for Intellia’s first two investigational in vivo CRISPR-based therapies, NTLA-2001 for transthyretin (ATTR) amyloidosis and NTLA-2002 for hereditary angioedema (HAE);

Advance new platform capabilities to the clinic, including CRISPR-based in vivo targeted gene insertion and a first-of-its-kind allogeneic cell engineering solution designed to avoid NK cell-mediated rejection;

Lead the development of new gene editing and delivery modalities, compatible with its modular platform, which will extend Intellia’s position of technological leadership and drive future pipeline growth.

"2022 proved to be another outstanding year for Intellia, with several significant clinical milestones achieved across our pipeline, further reinforcing the ability of our modular CRISPR genome editing platform to target a broad range of diseases," said Intellia President and Chief Executive Officer John Leonard, M.D. "These accomplishments reflect steady execution against our core strategy: to harness the immense power of genome editing, both for in vivo and ex vivo applications. As we look ahead, our highest priority will be to prepare for the initiation of global pivotal trials for our first two investigational in vivo CRISPR-based therapies, NTLA-2001 for ATTR amyloidosis and NTLA-2002 for hereditary angioedema. As these programs continue to progress, we believe we are moving closer to setting a new standard of care for people living with these and other serious diseases. In addition, we are advancing the next wave of platform capabilities, such as in vivo gene insertion and our proprietary allogeneic solution. Importantly, while the possibilities to apply our industry-leading genome editing technology are expansive, we are taking a disciplined approach with our portfolio by deploying resources on high-impact opportunities and collaborating with a network of other scientific leaders to expand the applications of our innovative technologies."

Based on these strategic priorities, which will be the Company’s focus over the next two years, Intellia anticipates reaching the following key program milestones in 2023:

In Vivo Programs

NTLA-2001 for ATTR amyloidosis:

Submit an IND application in mid-2023 to enable inclusion of U.S. sites in a pivotal study of NTLA-2001 for patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM).

Present additional clinical data from the ongoing Phase 1 study of NTLA-2001 in 2023.

Initiate a global pivotal NTLA-2001 trial for ATTR-CM by year-end 2023, subject to regulatory feedback.
Prepare for a Phase 3 study of NTLA-2001 for the treatment of ATTR amyloidosis with polyneuropathy (ATTRv-PN), including discussions with regulatory authorities.

NTLA-2002 for HAE:

Initiate Phase 2 portion of the ongoing NTLA-2002 Phase 1/2 study in 1H 2023.

Submit an IND in 1H 2023 to support inclusion of U.S. sites in the Phase 2 study of NTLA-2002.

Present additional clinical data from the ongoing first-in-human study of NTLA-2002 in 2023.

Alpha-1 antitrypsin deficiency (AATD) franchise:

Submit an IND or IND-equivalent application for NTLA-3001, Intellia’s wholly owned insertion candidate in development for AATD-associated lung disease, in 2H 2023.

Complete IND-enabling activities for NTLA-2003, a wholly owned knockout candidate for AATD-associated liver disease, by year-end 2023.

Prevalent diseases:

Progress one new in vivo development candidate, nominated in 2022, for the treatment of an undisclosed prevalent condition.

Ex Vivo Programs

NTLA-6001 for CD30+ Lymphomas:

Identify collaboration opportunities to advance development of NTLA-6001.

Additional ex vivo candidates:

Advance multiple programs, wholly owned or in collaboration with partners, utilizing allogeneic platform.

Platform Innovation

Advance novel gene editing technologies, including DNA writing, and delivery to other tissues outside of the liver.

Cash Position

Intellia ended the fourth quarter of 2022 with approximately $1.3 billion in cash, cash equivalents and marketable securities.