Intellia Therapeutics, Inc. (NASDAQ: NTLA)

Intellia Therapeutics, Inc., a clinical-stage gene editing company, is at the forefront of developing potentially curative genome editing treatments using CRISPR/Cas9-based technologies. The company's primary business activities involve developing gene editing therapies for various diseases, including genetic liver diseases, hereditary angioedema, and alpha-1 antitrypsin deficiency.

Intellia's main business activities are centered on gene editing therapies, with a particular focus on in vivo research programs. The company's lead in vivo candidates, NTLA-2001 and NTLA-2002, are the first CRISPR/Cas9-based therapy candidates to be administered systemically for precision editing of a gene in a target tissue in humans. NTLA-2001 is being developed for the treatment of transthyretin (ATTR) amyloidosis, a progressive and fatal disorder resulting from deposition of insoluble amyloid fibrils into multiple organs and tissues. Intellia is currently enrolling patients in a global, pivotal Phase 3 MAGNITUDE trial to evaluate the efficacy and safety of NTLA-2001 in adults with ATTR-CM. NTLA-2002 is being developed for the treatment of hereditary angioedema (HAE), a rare genetic disease characterized by severe, recurring, and unpredictable inflammatory attacks in various organs and tissues. The company has completed enrollment and dosing in the Phase 2 portion of the Phase 1/2 study and plans to initiate a global pivotal Phase 3 study in the second half of 2024.

Intellia's revenue is generated through the development and commercialization of its gene editing therapies. The company's leadership position in the field of genome editing is driven by its broad and deep gene editing toolbox, which enables it to select the best tools for each therapeutic application.

The biotechnology and pharmaceutical industries are highly competitive, and Intellia faces competition from companies that use genome editing or gene therapy development platforms and from companies focused on more traditional therapeutic modalities such as small molecules and antibodies. Intellia's competitors include Alnylam Pharmaceuticals, AstraZeneca Pharmaceuticals LP, BridgeBio Pharma Inc., Ionis Pharmaceuticals, Inc., Metagenomi Technologies, LLC, Novo Nordisk A/S, and Pfizer, Inc.

Intellia has formed several collaborations and partnerships to accelerate the development and commercialization of CRISPR/Cas9-based products. These collaborations include Regeneron Pharmaceuticals, Inc., AvenCell Therapeutics, Inc., SparingVision SAS, Kyverna Therapeutics, Inc., ONK Therapeutics, Ltd, and Rewrite Therapeutics Inc.

Intellia believes it is well-positioned in terms of its intellectual property due to its broad worldwide portfolio of IP, including patents and patent applications, in areas relevant to the development and commercialization of human therapeutic products using CRISPR/Cas9 technology. The company protects its IP by maintaining trade secrets relating to its proprietary technology innovations and know-how and intends to take additional steps, where appropriate, to further protect its IP rights.

As a biopharmaceutical company, Intellia is subject to extensive legal and regulatory requirements. The company needs approval from regulatory agencies for its clinical studies, development, manufacturing, distribution, exportation and importation, commercialization, marketing, and reimbursement relating to its products and product candidates. Relevant regulatory authorities include the FDA, the EMA, the EC, EU Member State agencies, and other countries' similar agencies.

In vivo gene therapy products, including genetically modified cells, that lead to a durable modification of cells or tissues may meet the definition of a regenerative medicine therapy. Intellia's products are subject to regulation under the Food, Drug and Cosmetic Act and the Public Health Service Act and other federal, state, local, and foreign statutes and regulations.

Intellia's allogeneic platform is designed to overcome one of the key challenges to current allogeneic approaches, which is host rejection of the adoptive cell therapy. In preclinical studies, Intellia's allogeneic technology demonstrated the ability to create T cells with high anti-tumor activity capable of avoiding host T and NK cells, and thereby persisting to maintain durable responses.

In addition to its lead in vivo candidates, Intellia is advancing multiple ex vivo programs, including engineered cell therapies for immuno-oncology and autoimmune diseases. The company is leveraging its proprietary allogeneic cell engineering platform to develop allogeneic cellular therapies that can be used to treat both oncological and immunological diseases.

Intellia's manufacturing strategy involves entering into certain manufacturing and supply arrangements with third-party suppliers to support production of its product candidates and their components. The company plans to continue to rely on qualified third-party organizations and its own capabilities to produce or process bulk compounds, formulated compounds, viral vectors, or engineered cells for IND-supporting activities and to supply materials for clinical trials.