On January 7, 2021 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, reported its expected 2021 milestones and the following strategic priorities (Press release, Intellia Therapeutics, JAN 7, 2021, View Source [SID1234573644]):

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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; and
Platform 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 therapeutic potential."

Anticipated 2021 Milestones:

NTLA-2001 for ATTR: NTLA-2001 is the first systemically delivered CRISPR-based therapy dosed in a patient, and could potentially be the first curative treatment for ATTR. By applying the Company’s in vivo LNP technology, NTLA-2001 offers the possibility of halting and reversing the disease with potent, lifelong transthyretin (TTR) protein reduction after a single course of treatment. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party, and Regeneron Pharmaceuticals, Inc. (Regeneron).
Intellia is continuing to enroll patients in the global Phase 1 study of NTLA-2001 in adults with hereditary ATTR with polyneuropathy (hATTR-PN) in order to establish an optimal dose. Later this year, the Company plans to provide guidance around timing of the first expected data readout, with the goal of demonstrating clinical proof-of-concept for its modular LNP delivery platform.
Intellia intends to evaluate NTLA-2001 in a broader ATTR population of both polyneuropathy and cardiomyopathy patients following its Phase 1 safety assessment and dose optimization.

NTLA-5001 for AML: NTLA-5001 is a potential best-in-class engineered T cell therapy designed to treat all genetic subtypes of acute myeloid leukemia (AML). This investigational candidate is a T cell receptor (TCR)-T cell therapy targeting the Wilms’ Tumor 1 (WT1) antigen utilizing Intellia’s proprietary cell engineering process.
Intellia plans to submit an Investigational New Drug (IND) or equivalent regulatory application for NTLA-5001 in mid-2021. The first-in-human trial is expected to evaluate the safety and activity of NTLA-5001 in patients with persistent or recurrent AML who have previously received first-line therapies.
The Company is also evaluating the potential use of NTLA-5001 to treat WT1-positive solid tumors in preclinical studies.

NTLA-2002 for HAE: NTLA-2002 aims to prevent attacks and eliminate the current, significant treatment burden for people living with hereditary angioedema (HAE) after a single course. Intellia is applying its modular LNP delivery system to develop NTLA-2002 to knock out the KLKB1 gene in the liver to permanently reduce plasma kallikrein activity.
Intellia plans to submit an IND or equivalent regulatory application in the second half of 2021.
The Company is applying insights gained from NTLA-2001 to expedite clinical development of NTLA-2002.

Pipeline Expansion: Intellia is focused on advancing its differentiated genome editing, delivery and cell engineering strategies to broaden in vivo and ex vivo applications for wholly owned and partnered programs. The Company continues to progress its robust research efforts and modular platform to develop new therapeutic candidates for genetic diseases requiring removal and/or restoration of a protein, as well as the next generation of engineered cell therapies for cancers.
Intellia reported plans to nominate at least one additional development candidate in 2021.
Intellia, in partnership with lead party Regeneron, is also continuing to advance hemophilia A and B therapeutic programs toward IND-enabling studies using their jointly-developed in vivo targeted insertion technology.
The Company plans to present at upcoming scientific conferences, with updates on multiple in vivo targets in the liver and other tissues and an allogeneic solution that enables the next generation of engineered cell therapies.
Cash Position and Financial Guidance

Intellia ended the fourth quarter of 2020 with approximately $597 million in cash, cash equivalents and marketable securities. Intellia expects that its cash, cash equivalents and marketable securities as of December 31, 2020 will enable the Company to fund its anticipated operating expenses and capital expenditure requirements for at least the next 24 months. This expectation excludes any strategic use of capital not currently in the Company’s base-case planning assumptions.