Intellia Therapeutics Inc.

Company Overview

Intellia is a genome editing company focused on the development of therapeutics utilizing the CRISPR-Cas9 system. Intellia intends to advance its pipeline and potentially develop a new drug class by leveraging its scientific and clinical development expertise and its exclusive access to related intellectual property platforms. The company’s intellectual property portfolio includes the foundational CRISPR-Cas9 platform technology, delivery technologies and nucleic acid modifications for human therapeutic use. Intellia is supported by its founding investors Atlas and Novartis, as well as numerous public and private sector funds. In gene editing, the company focuses on the type of edit, which includes knockout, repair or insertion.

Through strategic engagements with partners in industry and academia, the company is broadening and optimizing its pipeline. Intellia’s immediate pipeline opportunities focus on developing treatments for rare diseases such as Transthyretin (ATTR) Amyloidosis, Hereditary Angioedema (HAE), lung disease, hemophilia and primary hyperoxaluria.

Its Novartis partnership accelerates the development of engineered ex vivo CRISPR-Cas9 cell therapies for chimeric antigen receptor T cell (CART) and hematopoietic stem cell (HSC) applications. Its platform development agreement with Caribou Biosciences provides access to ongoing advances in CRISPR-Cas9 technology as the technology continues to mature.

The company’s lead in vivo candidates, NTLA-2001 for treating transthyretin (ATTR) amyloidosis and NTLA-2002 for treating hereditary angioedema (HAE), are the first CRISPR/Cas9-based therapies administered systemically via intravenous (IV) infusion for precise gene editing in a specific tissue in humans. Additionally, Intellia is also progressing several ex vivo programs, both independently and in partnership, targeting immuno-oncology and autoimmune diseases.

Financial Highlights (FY 2023)

Company's Business Segments

• Genome Editing-based Therapies : This segment provides therapeutics using CRISPR/Cas9-based technologies by leveraging the company's modular platform to advance in-vivo and ex-vivo therapies for diseases with high unmeeted needs.