Intellia to Present Phase 1 Data on Nex-Z for ATTR Amyloidosis at 2024 AHA Sessions

Intellia Therapeutics, Inc., a pioneer in the gene editing field, has announced that it will present new interim data from its Phase 1 study of nexiguran ziclumeran (nex-z), also known as NTLA-2001. This investigational therapy utilizes CRISPR technology for the treatment of transthyretin (ATTR) amyloidosis. The data will be shared during a late-breaking oral presentation at the 2024 American Heart Association (AHA) Scientific Sessions, scheduled for November 16-18 in Chicago, Illinois.

Nex-z aims to be a groundbreaking single-dose treatment for ATTR amyloidosis, a severe and fatal condition caused by the accumulation of abnormal transthyretin (TTR) protein. The development and commercialization of this therapy are being spearheaded by Intellia in collaboration with Regeneron. The upcoming presentation will cover various aspects including safety, serum TTR reduction, biomarkers of disease progression, and functional capacity in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM).

The presentation is titled "Nexiguran ziclumeran (nex-z, also known as NTLA-2001), an investigational in vivo CRISPR-based therapy for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM): interim report of the Phase 1 study." It will be part of the Late-Breaking Science 1 session and will be delivered by Dr. Marianna Fontana, a distinguished Professor of Cardiology and Honorary Consultant Cardiologist at the University College London Centre for Amyloidosis.

Nex-z is built on the Nobel Prize-winning CRISPR/Cas9 technology and has shown promising results in the interim Phase 1 clinical data. The study revealed that administering nex-z led to consistent, deep, and long-lasting reductions in TTR levels. This therapy represents the first investigational CRISPR treatment designed to edit genes inside the human body systemically. 

ATTR amyloidosis is a rare and progressively debilitating disease. It can be hereditary (ATTRv) or acquired (ATTRwt). Hereditary ATTR amyloidosis stems from mutations in the TTR gene, leading to the production of structurally defective TTR proteins by the liver. These misfolded proteins aggregate as amyloid deposits, causing severe complications in various organs, including the heart and nerves. ATTRv amyloidosis commonly manifests as polyneuropathy (ATTRv-PN) or cardiomyopathy (ATTRv-CM). On the other hand, non-mutated but unstable TTR proteins can also lead to wild-type ATTR (ATTRwt) amyloidosis, mainly affecting the heart. Current medications for ATTR amyloidosis only aim to slow down the accumulation of these misfolded proteins, with no known cure available.

Intellia Therapeutics is a frontrunner in the clinical-stage gene editing arena, with a mission to revolutionize medical treatments using CRISPR-based therapies. Their in vivo programs target disease-causing genes directly within the body, while ex vivo programs modify human cells outside the body to treat conditions like cancer and autoimmune diseases. Intellia’s expertise in scientific, technical, and clinical development is setting a new standard in the field of gene editing. The company continues to innovate and expand its CRISPR-based platform to harness the full potential of gene editing.

The forthcoming presentation at the AHA Scientific Sessions is eagerly anticipated and represents a significant milestone in Intellia’s efforts to advance gene editing therapies for serious conditions like ATTR amyloidosis.

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