Intellia to Present First Clinical Data on Redosed CRISPR Therapy at 2024 Peripheral Nerve Society Meeting

CAMBRIDGE, Mass., June 17, 2024—Intellia Therapeutics, Inc. (NASDAQ: NTLA), a frontrunner in clinical-stage gene editing, announced that an abstract featuring redosing data from its Phase 1 study of NTLA-2001 has been accepted for an oral presentation at the Peripheral Nerve Society Annual Meeting, scheduled for June 22-25 in Montreal, Canada. NTLA-2001 is an experimental in vivo CRISPR-based gene editing therapy aimed at providing a single-dose treatment for transthyretin (ATTR) amyloidosis.

In the dose-escalation phase of the Phase 1 study, the first three patients were administered the lowest dose of 0.1 mg/kg, followed by a subsequent dose of 55 mg. This marks the first clinical data on patients who have been redosed with an in vivo CRISPR-based gene editing candidate, offering insights into its safety and pharmacodynamic effects. While repeat dosing is not planned for NTLA-2001 in treating ATTR amyloidosis, the possibility of redosing could be a significant advantage of Intellia's non-viral lipid nanoparticle (LNP)-based delivery platform for future therapies where an additive effect is needed.

The presentation titled "Activity of Follow-On Dosing for an Investigational In Vivo CRISPR-Based LNP Therapy in Transthyretin Amyloidosis" will be part of the Richard A.C. Hughes Symposium on Clinical Highlights, Late Breaking Abstracts, and Clinical Trials. The session is slated for Tuesday, June 25, 2024, from 3:45 p.m. to 4:00 p.m. EST and will be presented by Jorg Taubel, M.D., FFPM, FESC, Visiting Professor at King’s College London and Chief Executive Officer of Richmond Pharmacology.

NTLA-2001 leverages Nobel Prize-winning CRISPR/Cas9 technology and has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. The therapy is designed to inactivate the TTR gene responsible for producing the transthyretin (TTR) protein. NTLA-2001 is the first CRISPR therapy investigated for systemic administration to edit genes within the human body. Interim Phase 1 clinical data have demonstrated that NTLA-2001 administration results in consistent, deep, and long-lasting reductions in TTR levels. Intellia is spearheading the development and commercialization of NTLA-2001 in collaboration with Regeneron.

Transthyretin amyloidosis (ATTR amyloidosis) is a rare, progressive, and fatal condition. Hereditary ATTR (ATTRv) amyloidosis results from genetic mutations in the TTR gene, causing the liver to produce abnormal transthyretin protein that misfolds. These misfolded proteins accumulate as amyloid deposits, leading to severe complications in multiple tissues, including the heart, nerves, and digestive system. ATTRv amyloidosis can manifest as polyneuropathy (ATTRv-PN), which causes nerve damage, or cardiomyopathy (ATTRv-CM), which can result in heart failure. In contrast, wild-type ATTR (ATTRwt) amyloidosis affects individuals without genetic mutations, with non-mutated TTR proteins becoming unstable over time and misfolding into amyloid deposits, primarily impacting the heart. An estimated 50,000 people worldwide live with ATTRv amyloidosis, and between 200,000 and 500,000 people suffer from ATTRwt amyloidosis. Currently, there is no known cure, and existing treatments only slow the build-up of misfolded TTR protein.

Intellia Therapeutics, Inc. is at the forefront of clinical-stage gene editing, aiming to transform medicine through CRISPR-based therapies. The company's in vivo programs utilize CRISPR to precisely edit disease-causing genes within the human body, while its ex vivo programs engineer human cells outside the body for treating cancer and autoimmune diseases. Intellia's extensive scientific, technical, and clinical development expertise, combined with its dedicated team, is setting new standards in medicine. The company is continually expanding its CRISPR-based platform with innovative editing and delivery technologies.