AI Analysis | Feedback

Here are 1-3 brief analogies to describe Intellia Therapeutics (NTLA):

• The Genentech of the gene editing era.
• The Tesla of genetic medicine.
• A biotech like an early Moderna, but focused on permanently editing human DNA.

AI Analysis | Feedback

• NTLA-2001: An investigational *in vivo* CRISPR gene editing therapy being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis.
• NTLA-2002: An investigational *in vivo* CRISPR gene editing therapy being developed as a single-dose treatment for hereditary angioedema (HAE).

AI Analysis | Feedback

Intellia Therapeutics (NASDAQ: NTLA) is a clinical-stage biotechnology company focused on developing CRISPR-based gene editing therapies. As such, it sells primarily to other companies through strategic collaborations, licensing agreements, and co-development partnerships, rather than directly to individuals or patients.

Its major customer companies and collaborators include:

• Regeneron Pharmaceuticals (NASDAQ: REGN): This is a foundational and broad collaboration focusing on *in vivo* CRISPR-based therapies, including the lead program for transthyretin (ATTR) amyloidosis (NTLA-2001).
• SparingVision: A private company based in France, collaborating with Intellia on *ex vivo* CRISPR gene editing for ocular diseases.
• Aardvark Therapeutics: A private company collaborating with Intellia on *ex vivo* CRISPR gene editing to develop therapies for autoimmune diseases.

AI Analysis | Feedback

AI Analysis | Feedback

John Leonard, President and Chief Executive Officer

John Leonard, M.D., became Intellia's President and Chief Executive Officer in January 2018, having joined the company in 2014 to direct research and development. Before Intellia, he had a 30-year career in pharmaceutical R&D, retiring in 2013 as chief scientific officer and senior vice president of R&D at AbbVie. At AbbVie and Abbott Laboratories, he oversaw the development of numerous therapeutics, including the HIV protease inhibitors Norvir® and Kaletra®, and Humira®. His teams were twice recognized with the Prix Galien Award for excellence in pharmaceutical research. Leonard's entrepreneurial ventures include co-founding Virtue Cider, a craft cider company that was later acquired by Anheuser-Busch, and a working ranch in Bolivia. He also serves on the board of directors for IQVIA, IFM Therapeutics, and 3T Biosciences.

Glenn Goddard, Executive Vice President, Chief Financial and Accounting Officer

Glenn Goddard joined Intellia Therapeutics in November 2018 as Executive Vice President, Chief Financial and Accounting Officer. He brings nearly two decades of experience in financial and business support operations for biotechnology companies. Prior to Intellia, he was the Chief Financial Officer at Generation Bio Company, where he closed a $100 million private financing. Before that, he served as Senior Vice President of Finance and Principal Financial Officer at Agios Pharmaceuticals, where he played a key role in transitioning the company to a commercial-ready organization and led strategic financing efforts that raised over $700 million in equity financings, including an initial public offering. His past experience also includes senior-level financial management roles at Archemix, a privately held biopharmaceutical company, and ImmunoGen, a publicly traded oncology-focused biopharmaceutical company. Earlier in his career, he worked as an audit supervisor at Ernst & Young LLP and a business advisory manager at Feeley & Driscoll, P.C. He is a Certified Public Accountant.

Laura Sepp-Lorenzino, Executive Vice President, Chief Scientific Officer

Laura Sepp-Lorenzino, Ph.D., serves as Intellia Therapeutics' Executive Vice President, Chief Scientific Officer, overseeing drug research across in vivo and engineered cell therapy areas. Before joining Intellia, she was Vice President, Head of Nucleic Acid Therapies, and a member of the External Innovation team at Vertex Pharmaceuticals Incorporated. She also served as Vice President and Entrepreneur-in-Residence at Alnylam Pharmaceuticals, Inc., where she developed and implemented the hepatic infectious disease strategy and championed extra-hepatic oligonucleotide delivery. Dr. Sepp-Lorenzino spent 14 years at Merck & Co., Inc., where she was an industry pioneer in advancing RNAi as a novel therapeutic modality and led the Cancer Research Department. She began her career in academia as an Assistant Attending Molecular Biologist at Memorial Sloan Kettering Cancer Center.

Andrew Schiermeier, Former Chief Operating Officer (now CEO of AvenCell Therapeutics)

Andrew Schiermeier, Ph.D., spent five years in successive leadership roles at Intellia Therapeutics, Inc., most recently as Chief Operating Officer. In this role, he was responsible for establishing and overseeing Intellia's strategic direction, portfolio management, drug development, manufacturing, and business development. Prior to Intellia, he was Senior Vice President and Global Head of Merck KGaA's Oncology Business, where he rebuilt the oncology pipeline and oversaw the development and launch of a commercialized blood-based biopsy. He also served as General Manager of the Merck-Pfizer Immune-Oncology Alliance. His executive experience includes serving as Chief Operating Officer of Aura Biosciences, Inc. and Chief Executive Officer of Medicine in Need Corporation (MEND) and Lantibio, Inc. Schiermeier is currently the Chief Executive Officer of AvenCell Therapeutics, a company launched by Blackstone Life Sciences, Cellex Cell Professionals, and Intellia Therapeutics.

AI Analysis | Feedback

The key risks to Intellia Therapeutics (NTLA) are primarily centered on clinical trial outcomes, intellectual property disputes, and ongoing financial challenges inherent to a clinical-stage biotechnology company.

1. Clinical Trial Holds and Safety Concerns: Intellia Therapeutics is facing significant setbacks due to a formal clinical hold imposed by the U.S. FDA on its Phase 3 trials for nexiguran ziclumeran (nex-z). This decision followed reports of a severe liver-related adverse event in a trial participant, including a patient fatality, and grade 4 liver enzyme elevations. This "worst-case outcome" in biotech clinical trials has led to a halt in dosing and enrollment for its MAGNITUDE and MAGNITUDE-2 trials, which were evaluating nex-z for transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN). The clinical hold has resulted in a substantial decline in the company's stock price and analyst downgrades. The company is now working with the FDA to address the hold and develop a strategy to resume enrollment.

2. Intellectual Property Disputes: Intellia's business relies heavily on its CRISPR/Cas9 gene-editing technology, but it has been embroiled in ongoing intellectual property (IP) disputes. A significant ruling by the U.S. Patent and Trademark Office in 2022 determined that key patents for the basic technology belonged to the Broad Institute of MIT and Harvard, rather than the University of California, from which Intellia holds a license. This judgment creates uncertainty regarding the ownership of foundational CRISPR technology and may necessitate Intellia to negotiate new legal agreements with the Broad Institute and its licensee, Editas Medicine, to secure rights for the development and commercialization of its drugs.

3. Financial Health and Funding Challenges: As a pre-commercial, clinical-stage biotechnology company, Intellia Therapeutics consistently faces significant financial challenges. The company is not yet profitable, reporting substantial operational inefficiencies, negative margins, and declining revenue growth. It has a high cash burn rate and relies on collaboration revenue, equity or debt financings, or other sources to fund its operations. While Intellia had approximately $669.9 million in cash, cash equivalents, and marketable securities as of September 30, 2025, providing a cash runway into mid-2027, the recent clinical hold on nex-z could significantly impact future collaboration revenue and increase its net cash burn rate. The company has already undertaken cost-cutting measures, including workforce reductions and the discontinuation of certain pipeline assets, to conserve cash and focus resources on its most promising programs.

AI Analysis | Feedback

The emergence of advanced gene-editing technologies, specifically base editing and prime editing, represents a clear emerging threat to Intellia Therapeutics. While Intellia focuses on CRISPR/Cas9 technology, companies such as Beam Therapeutics (BEAM) and Verve Therapeutics (VERV) are developing and advancing base editing and prime editing platforms. These next-generation technologies offer the potential for more precise genetic modifications with potentially fewer off-target effects and reduced risk of double-strand DNA breaks, which are inherent to traditional CRISPR/Cas9. If these alternative gene-editing modalities prove to be superior in terms of safety, efficacy, or broader applicability in clinical trials, they could significantly displace or limit the market for CRISPR/Cas9-based therapies, impacting Intellia's pipeline and long-term competitive position.

AI Analysis | Feedback

Intellia Therapeutics (symbol: NTLA) is a clinical-stage gene editing company with two main product candidates in late-stage clinical trials: Nexiguran Ziclumeran (also known as nex-z or NTLA-2001) for Transthyretin (ATTR) Amyloidosis and Lonvoguran Ziclumeran (also known as lonvo-z or NTLA-2002) for Hereditary Angioedema (HAE).

Nexiguran Ziclumeran (nex-z or NTLA-2001) for Transthyretin (ATTR) Amyloidosis

The global addressable market for Transthyretin Amyloidosis is projected to reach approximately $12 billion by 2028. Intellia Therapeutics estimates that approximately 50,000 people worldwide live with hereditary ATTR (ATTRv) amyloidosis, and between 200,000 and 500,000 people have the wild-type form of the disease.

Lonvoguran Ziclumeran (lonvo-z or NTLA-2002) for Hereditary Angioedema (HAE)

The global hereditary angioedema therapeutics market size was valued at USD 4.1 billion in 2023 and is projected to grow at a Compound Annual Growth Rate (CAGR) of 9.2% between 2024 and 2032. Other estimates indicate the global market size was USD 3.3 billion in 2022 and is expected to reach USD 5.8 billion by 2030, growing at a CAGR of 7.4% from 2023 to 2030. Another source valued the global hereditary angioedema therapeutics market at USD 4.36 billion in 2024 and anticipates it will reach USD 7.28 billion by 2030, with a CAGR of 8.89%. North America was identified as the dominant region in the global hereditary angioedema therapeutics market in 2024.

AI Analysis | Feedback

Intellia Therapeutics (NTLA) anticipates several key drivers for future revenue growth over the next two to three years, primarily stemming from the progression and potential commercialization of its lead gene-editing therapies, alongside ongoing collaboration agreements.

1. Commercialization of Lonvo-z (NTLA-2002) for Hereditary Angioedema (HAE): Intellia's wholly-owned investigational therapy, lonvo-z, is a significant expected driver. The company completed enrollment in its pivotal Phase 3 HAELO study for HAE in September 2025. Intellia expects to report topline data by mid-2026 and plans to submit a Biologics License Application (BLA) in the second half of 2026, with a potential U.S. commercial launch anticipated in the first half of 2027. This represents the most immediate opportunity for product-based revenue.
2. Collaboration Revenue from Regeneron Partnership: Intellia currently generates revenue primarily through its collaboration agreements, notably with Regeneron Pharmaceuticals. This partnership has been a consistent source of revenue, driven by cost reimbursements related to the co-development of programs like those for ATTR amyloidosis. This ongoing collaboration is expected to continue contributing to Intellia's revenue stream in the near term.
3. Potential Future Commercialization of Nexiguran Ziclumeran (nex-z) for ATTR Amyloidosis: While subject to recent developments, nexiguran ziclumeran (nex-z), developed in collaboration with Regeneron for transthyretin (ATTR) amyloidosis, holds significant long-term revenue potential. However, a clinical hold on its Phase 3 MAGNITUDE and MAGNITUDE-2 trials, following observations of Grade 4 liver transaminase elevations in a patient, has temporarily suspended its milestone guidance. Intellia is working with regulators to address the issue and finalize a path forward. A successful resolution and eventual commercial launch would provide a substantial revenue driver, though the timing within the next 2-3 years is now uncertain.

AI Analysis | Feedback

Share Issuance

• Intellia Therapeutics raised $114.5 million of net equity proceeds from its "At the Market" (ATM) program during the third quarter of 2025.
• An additional 4,081,183 shares of common stock became available for issuance under the Amended and Restated 2015 Stock Option and Incentive Plan, effective January 1, 2025.
• During the six months ended June 30, 2024, the company generated $96.4 million in net proceeds from at-the-market offerings, $3.1 million from the exercise of stock options, and $1.7 million from the employee stock purchase plan.

Inbound Investments

• Cathie Wood's ARK Invest recently invested approximately $4 million in Intellia Therapeutics, acquiring 349,930 shares.
• Intellia's financial health is supported by strategic partnerships, including a significant collaboration with Regeneron Pharmaceuticals, Inc.

Outbound Investments

• In February 2022, Intellia Therapeutics acquired Rewrite Therapeutics, Inc., which subsequently merged into Intellia in September 2022.
• The company holds an investment in Kyverna Therapeutics, Inc., a publicly traded company.

Capital Expenditures

• Intellia's capital expenditures are part of its ongoing operating expenses, with cash, cash equivalents, and marketable securities expected to fund these requirements into mid-2027.
• The primary focus of capital expenditures is likely on advancing its CRISPR-based therapeutic programs through clinical trials and expanding its gene editing platform capabilities.