Intellia Therapeutics’ Investigational CRISPR Treatment NTLA-2001 Receives European Union Orphan Drug Designation for ATTR Amyloidosis - Seite 2

About NTLA-2001
NTLA-2001 is the first experimental CRISPR therapy to be administered systemically, or through a vein, to edit a gene 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. Intellia’s 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 dose of treatment. 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 Amyloidosis (ATTR)
Transthyretin amyloidosis, or ATTR, is a rare, progressive and fatal disease. Hereditary ATTR (hATTR) occurs when a person is born with a specific DNA mutation in the TTR gene, which causes the liver to produce a protein called transthyretin (TTR) in a misfolded form and build up in the body. hATTR can manifest as polyneuropathy (hATTR-PN), which can lead to nerve damage, or 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). 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 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 treatment course, 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 intelliatx.com. Follow us on Twitter @intelliatweets.