Intellia Therapeutics Achieves Normal Human Alpha-1 Antitrypsin Protein Levels in Non-Human Primates Through Targeted Gene Insertion for the Treatment of AAT Deficiency - Seite 2

The findings being presented today reinforce recent data showing the use of the same proprietary insertion technology for targeted gene insertion of Factor 9 resulted in circulating human Factor IX, a blood-clotting protein that is missing or defective in hemophilia B patients, that ranged from normal levels (50-150%)¹ to supratherapeutic levels in a six-week NHP study. Intellia and Regeneron, the lead party, are co-developing potential hemophilia A and B CRISPR/Cas9-based treatments using their jointly developed targeted transgene insertion capabilities. Intellia is continuing to develop its proprietary platform to advance its wholly owned research programs, such as AATD. Click here to register for the Alpha-1 Foundation’s virtual workshop and here to view Intellia’s presentation on the company’s website.

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¹ National Hemophilia Foundation

About Alpha-1 Antitrypsin Deficiency and Intellia’s Genome Editing Treatment Approach

The SERPINA1 gene normally encodes the alpha-1 antitrypsin (AAT) protein produced in the liver that is then secreted to protect the lungs. SERPINA1 mutations can cause AAT deficiency (AATD), a rare, genetic disease that commonly manifests in lung dysfunction, as well as in liver disease in some patients. Intellia’s targeted in vivo insertion platform uses a hybrid delivery system combining a non-viral lipid nanoparticle (LNP), which encapsulates CRISPR/Cas9 components, with an adeno-associated virus (AAV) carrying a donor DNA template to enable therapeutic protein production. One of the editing strategies Intellia is studying as a potential single-course AATD treatment is using the company’s SERPINA1 gene insertion approach to restore normal human AAT protein levels. Intellia also is investigating a consecutive genome editing approach, in which the PiZ allele, the most prevalent disease-causing mutation of SERPINA1, is knocked out and the normal human SERPINA1 gene is inserted.

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.