Intellia Therapeutics to Present Interim Clinical Data from Ongoing Phase 1 Study of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis at the 2021 Peripheral Nerve Society Annual Meeting

CAMBRIDGE, Mass. – Intellia Therapeutics, Inc.(NASDAQ:NTLA) today announced that a late-breaking abstract featuring interim Phase 1 clinical data from NTLA-2001, the Company’s lead CRISPR/Cas9 in vivo therapy in development as a single-dose, systemically administered treatment for transthyretin (ATTR) amyloidosis, has been selected for an oral presentation at the 2021 Peripheral Nerve Society (PNS) Annual Meeting, taking place this month.

The presentation will include interim data from the ongoing dose-escalation portion of Intellia’s Phase 1 trial evaluating NTLA-2001 in people living with hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN). Following safety assessment and dose optimization, Intellia intends to further evaluate NTLA-2001 in a broader population of people with ATTR amyloidosis, including those with cardiomyopathy.

“These findings represent Intellia’s first clinical data readout and an important step forward in our commitment to develop breakthrough genome editing treatments for people living with severe diseases, such as transthyretin (ATTR) amyloidosis,” said Intellia President and Chief Executive Officer John Leonard, M.D. “By knocking out the disease-causing gene, NTLA-2001 is designed to halt progression and potentially reverse the disease with a single dose, offering the potential of meaningful improvement over the standard of care, which requires chronic, lifelong administration. These interim data will provide a view of NTLA-2001’s safety and activity profile as the dose-escalation portion of our study progresses, as well as insight into the promise of our modular platform to develop other systemically delivered in vivoCRISPR/Cas9 therapies across a range of diseases with unmet need.”

About the NTLA-2001 Clinical Program

Intellia’s global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). The trial’s primary objectives are to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2001. Patients receive a single dose of NTLA-2001 via intravenous administration. The study will enroll up to 38 participants (ages 18-80 years) and consists of a single-ascending dose phase in Part 1 and, following the identification of an optimal dose, an expansion cohort in Part 2. Visit (NCT04601051) for more details.

Enrollment is ongoing at global clinical trial sites, as Intellia is submitting additional regulatory applications in other countries as part of its development strategy. After completion of the Phase 1 trial, the company is planning to rapidly move to pivotal studies for both polyneuropathy and cardiomyopathy patients.

About NTLA-2001

Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially be the first curative treatment for ATTR. NTLA-2001 is the first experimental CRISPR therapy candidate to be administered systemically, or through a vein, to edit genes inside the human body. Intellia’s proprietary non-viral platform utilizes lipid nanoparticles designed to deliver to the liver a simple, two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 protein. Our robust preclinical data showing deep and long-lasting transthyretin (TTR) reduction following knockout of the target gene in vivo support NTLA-2001’s potential as a single-administration therapeutic. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead development and commercialization party, and Regeneron Pharmaceuticals, Inc.

About Transthyretin (ATTR) Amyloidosis

Transthyretin amyloidosis, or ATTR, is a rare, progressive and fatal disease. Hereditary ATTR (hATTR) amyloidosis occurs when a person is born with DNA mutations in the TTR gene, which causes the liver to produce transthyretin (TTR) protein in a misfolded form and build up in the body. hATTR amyloidosis can predominantly manifest as polyneuropathy (hATTR-PN), which can lead to nerve damage, and cardiomyopathy (hATTR-CM), which involves heart muscle disease that can lead to heart failure. In addition, non-mutated, or wild-type TTR protein, can also accumulate in the body, leading to wild-type ATTR (wtATTR) amyloidosis. There are an estimated 50,000 hATTR patients worldwide and between 200,000 and 500,000 people with wtATTR.

About Intellia Therapeutics

Intellia Therapeutics is a leading clinical-stage genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single administration, and creating enhanced engineered cells that can treat oncological and immunological diseases. Intellia’s combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts it in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create new classes of therapeutic products. Learn more about Intellia and CRISPR/Cas9 at Follow us on Twitter @intelliatweets.

Intellia Contacts:

Lina Li
Director, Investor Relations
[email protected]


Julie Ferguson
(Interim) Head of External Affairs & Communications
[email protected]
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