Intellia Therapeutics, Inc. (NTLA): History, Ownership, Mission, How It Works & Makes Money

Intellia Therapeutics, Inc. (NTLA): History, Ownership, Mission, How It Works & Makes Money

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Intellia Therapeutics, Inc. (NTLA) is a critical player in the gene-editing space, but are you clear on its true financial footing given the recent clinical turbulence? This company is pioneering the in vivo (inside the body) application of CRISPR/Cas9 technology, aiming to deliver one-time, curative treatments, yet its business model currently rests on collaboration revenue, which hit $13.8 million in the third quarter of 2025, not product sales. We need to look past the $1.1 billion market capitalization and the promise of its platform to understand the near-term risk-reward, especially after the FDA placed a clinical hold on its flagship nexiguran ziclumeran (nex-z) trials in late October 2025. You need to know how their core science works, how they actually make money now, and what the $669.9 million cash runway means for their path to market with lonvoguran ziclumeran (lonvo-z) in 2027.

Intellia Therapeutics, Inc. (NTLA) History

You're looking at Intellia Therapeutics, Inc. (NTLA) because you want to know if the science is real, and more importantly, if the business model is sustainable. The direct takeaway is this: Intellia is a pioneer in the CRISPR gene editing space, but its history shows a clear, recent pivot from broad research to a laser focus on two late-stage, high-value assets, a move that is defintely a high-stakes gamble.

Given Company's Founding Timeline

Year established

Intellia Therapeutics was founded in 2014, right as the revolutionary potential of CRISPR-Cas9 gene editing technology was becoming globally recognized.

Original location

The company started in Cambridge, Massachusetts, a deliberate choice to be at the epicenter of the US biotech and life sciences hub, ensuring access to top-tier scientific talent and institutional research.

Founding team members

The founding team was a powerful mix of entrepreneurial leadership and academic CRISPR pioneers. Key figures included:

  • Nessan Bermingham (Founding CEO)
  • Jennifer Doudna (Nobel Laureate and CRISPR pioneer)
  • Luciano Marraffini
  • Rodolphe Barrangou
  • Erik Sontheimer
  • Andrew May

Initial capital/funding

Intellia secured its initial runway through venture capital, starting with a Series A round of $15 million in November 2014, led by Atlas Venture and Novartis Institutes for BioMedical Research. Investor confidence quickly grew, so a substantial Series B round followed in September 2015, raising another $70 million. As of September 2025, the company has raised a total of approximately $790 million in funding, demonstrating significant long-term capital backing.

Given Company's Evolution Milestones

The company's evolution is a story of moving from a foundational technology platform to a clinical-stage product company, culminating in a critical 2025 focus on commercial readiness.

Year Key Event Significance
2016 Initial Public Offering (IPO) on Nasdaq Raised approximately $112.1 million, securing capital for R&D and signaling market validation for the CRISPR platform.
2016 Major Collaboration with Regeneron Established a key partnership for in vivo (inside the body) CRISPR therapies, diversifying risk and securing a major funding source.
2021 Landmark Interim Data for NTLA-2001 Demonstrated the first successful systemic in vivo CRISPR gene editing in humans, a pivotal moment for the entire field of genome editing.
January 2025 Dosing in Phase 3 HAELO Trial for lonvo-z Initiated the final-stage clinical trial for lonvo-z (NTLA-2002) for Hereditary Angioedema (HAE), positioning it as a near-term commercial product.
September 2025 Completed Enrollment in Phase 3 HAELO Trial Hit a major clinical execution milestone on schedule, keeping the company on track for a potential US commercial launch in the first half of 2027.

Given Company's Transformative Moments

The most transformative period for Intellia has been the start of the 2025 fiscal year, driven by a need for capital efficiency in a tough biotech market. This was a hard choice, but it was necessary to survive and thrive.

In January 2025, the company announced a significant strategic reorganization to prioritize its two most advanced programs: lonvo-z (NTLA-2002) for HAE and nexiguran ziclumeran (nex-z) for transthyretin (ATTR) amyloidosis. This was a clear signal to the market that the company was transitioning from a research-heavy platform company to a commercial-ready one.

  • Pipeline Focus: Discontinued the development of NTLA-3001 for Alpha-1 Antitrypsin Deficiency and several other research-stage programs.
  • Cost Reduction: The reorganization included a net workforce reduction of approximately 27% in 2025, with an expected charge of around $8 million in the first quarter.
  • Cash Runway Extension: This strategic focus, combined with its cash position of approximately $862 million at the end of 2024, extended the company's cash runway into the first half of 2027.

However, the risks are real. In late 2025, a patient in the MAGNITUDE Phase 3 trial for nex-z passed away following a liver-related adverse event, leading to an awaited FDA clinical hold letter. This event, which caused a stock drop earlier in the year, highlights the inherent volatility and high stakes of developing first-in-class gene therapies. The market is now watching closely for the risk mitigation plan. If you want to dive deeper into the financial implications of these clinical events, you should read Breaking Down Intellia Therapeutics, Inc. (NTLA) Financial Health: Key Insights for Investors.

Here's the quick math on the cash position: Intellia ended the third quarter of 2025 with approximately $670 million in cash, cash equivalents, and marketable securities, which supports operations through the anticipated US launch of lonvo-z. That's a strong buffer, but clinical setbacks can burn cash fast.

Intellia Therapeutics, Inc. (NTLA) Ownership Structure

Intellia Therapeutics, Inc. (NTLA) is a publicly traded, clinical-stage gene editing company, and its ownership structure is heavily weighted toward institutional investors, which is typical for a high-growth biotech firm.

This structure means that major investment funds and asset managers hold the vast majority of the shares, giving them significant influence over corporate governance and strategic direction.

Intellia Therapeutics' Current Status

Intellia Therapeutics is a publicly traded company, listed on the Nasdaq Global Market under the ticker symbol NTLA.

As of November 2025, the company remains a clinical-stage biotechnology firm, focused on advancing its lead programs, including NTLA-2002 for hereditary angioedema (HAE) and nexiguran ziclumeran (nex-z) for transthyretin (ATTR) amyloidosis, toward commercial readiness by the end of 2026.

The company held approximately $862 million in cash and investments as of the end of the fourth quarter of 2024, with a strategic reorganization in 2025 designed to extend its operational runway into the first half of 2027.

For a deeper dive into the core principles driving these decisions, you can review the Mission Statement, Vision, & Core Values of Intellia Therapeutics, Inc. (NTLA).

Intellia Therapeutics' Ownership Breakdown

The company's stock is overwhelmingly owned by institutional investors, demonstrating high conviction from large funds in the long-term potential of its CRISPR-based gene editing platform.

Here's the quick math on who controls the shares outstanding as of November 2025:

Shareholder Type Ownership, % Notes
Institutional Investors 93.33% Includes firms like Vanguard Group Inc, BlackRock, Inc., and ARK Investment Management LLC.
Company Insiders 3.24% Shares held by officers, directors, and key employees, aligning management's interests with shareholders.
Public/Retail Investors 3.43% The remaining float, calculated as 100% minus institutional and insider holdings.

The high institutional ownership-over 93%-means that a small number of large transactions can defintely impact the stock price, so watch the 13F filings closely.

Intellia Therapeutics' Leadership

The executive team is a mix of seasoned biotech veterans and scientific experts, steering the company through late-stage clinical trials and toward its goal of commercialization.

The leadership team, as of November 2025, is focused on executing the strategic priorities laid out for the year, which included completing the buildout of the commercial leadership team by the second half of 2025.

  • John Leonard, M.D.: President and Chief Executive Officer (CEO). He is the central figure driving the company's clinical and strategic vision.
  • Edward Dulac: Executive Vice President, Chief Financial Officer (CFO), and Treasurer. He took on the role in July 2024, bringing over two decades of finance and business development experience to the executive team.
  • Birgit Schultes, Ph.D.: Executive Vice President and Chief Scientific Officer (CSO). Promoted in January 2025, she is responsible for leading the company's research and early development efforts.
  • Jose Rivera: Chief Operating & Legal Officer.
  • Jennifer Smoter: Chief External Affairs & Communications Officer.

Intellia Therapeutics, Inc. (NTLA) Mission and Values

Intellia Therapeutics, Inc. stands for a singular, ambitious goal: developing potentially curative genome editing treatments to transform the lives of people with severe diseases, moving beyond chronic management to one-time, lasting fixes. This commitment is their cultural DNA, driving tough strategic choices like the 2025 reorganization to focus resources on the most promising late-stage programs.

Honestly, every financial decision in biotech, like the strategic net workforce reduction of approximately 27% in 2025, is ultimately a mission-driven one-it's about maximizing the chance to deliver a cure, not just a profit, though the two are defintely linked.

Intellia Therapeutics' Core Purpose

The company's core purpose is to harness the revolutionary potential of CRISPR-Cas9 (a gene editing technology that acts like molecular scissors to precisely modify DNA) to create a new class of medicine. This purpose is supported by a solid cash position of approximately $670 million as of September 30, 2025, which is expected to fund operations into mid-2027, giving them the runway to execute on this mission.

Official Mission Statement

Intellia's mission is clear and patient-centric, focusing on the highest-impact diseases where existing treatments fall short.

  • Transforming lives of people with severe diseases by developing potentially curative genome editing treatments.

This focus meant a strategic pipeline prioritization in early 2025, which included discontinuing select research-stage programs to concentrate on high-value assets like lonvo-z (formerly NTLA-2002) for hereditary angioedema (HAE). Here's the quick math: that prioritization resulted in about $8 million in charges in Q1 2025, but it streamlined their path to commercialization.

Vision Statement

The vision is about a paradigm shift in healthcare, not just incremental improvement.

  • To bring forth a new era in medicine through the revolutionary potential of CRISPR-based therapies.

Achieving this vision requires relentless execution. For example, in September 2025, Intellia completed enrollment in the pivotal Phase 3 HAELO clinical trial for lonvo-z, a critical milestone toward a potential U.S. commercial launch in the first half of 2027. You can see how that focus is paying off on the financial side, too: Breaking Down Intellia Therapeutics, Inc. (NTLA) Financial Health: Key Insights for Investors.

Intellia Therapeutics' Core Values

Intellia distills its cultural DNA into four active principles that guide their daily work, from the lab bench to the boardroom.

  • One: Work as a unified team.
  • Explore: Push scientific boundaries.
  • Disrupt: Challenge the status quo of medicine.
  • Deliver: Bring life-changing medicines to patients.

Intellia Therapeutics Slogan/Tagline

  • Revolutionize the course of medicine.

Intellia Therapeutics, Inc. (NTLA) How It Works

Intellia Therapeutics, Inc. is a clinical-stage biotechnology company pioneering the use of CRISPR-Cas9 technology to develop curative gene-editing therapies, primarily focusing on fixing genetic defects in vivo (inside the body) with a single-dose treatment.

The company's model is built on using a molecular delivery system, often a lipid nanoparticle (LNP), to ferry the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) guide RNA and the Cas9 enzyme-the molecular scissors-directly to the target organ, like the liver, to permanently edit the faulty DNA. This approach aims to offer a functional cure, not just manage symptoms, which is a massive shift in medicine. You can read more about their core philosophy here: Mission Statement, Vision, & Core Values of Intellia Therapeutics, Inc. (NTLA).

Given Company's Product/Service Portfolio

Product/Service Target Market Key Features
Lonvoguran Ziclumeran (lonvo-z) Hereditary Angioedema (HAE) Wholly-owned, single-dose in vivo CRISPR therapy; inactivates the KLKB1 gene in the liver; potential for consistent, lifelong reduction in kallikrein levels; Phase 3 HAELO trial completed enrollment in September 2025.
Nexiguran Ziclumeran (nex-z) Transthyretin (ATTR) Amyloidosis (Cardiomyopathy and Polyneuropathy) In vivo CRISPR therapy partnered with Regeneron Pharmaceuticals, Inc.; designed to reduce the production of the misfolded TTR protein; Phase 3 trials (MAGNITUDE and MAGNITUDE-2) are currently on FDA clinical hold as of October 2025.
Research Programs Immuno-Oncology, Autoimmune Disease, Hemophilia B Leveraging both in vivo and ex vivo (outside the body) platforms for broader applications; Hemophilia B is a partnered program with Regeneron Pharmaceuticals, Inc..

Given Company's Operational Framework

Intellia's operational framework is laser-focused on translating its core CRISPR-Cas9 platform into clinical-stage assets, a process that is capital-intensive and high-risk. Honestly, in this business, you're spending money to prove your science works before you can ever make a dime.

The company's revenue stream in 2025 is almost entirely Collaboration Revenue, which was $13.8 million for the third quarter of 2025, primarily from cost reimbursements related to the Regeneron Pharmaceuticals, Inc. partnership. This revenue offsets some of the massive Research and Development (R&D) expenses, which hit $94.7 million in Q3 2025.

  • Prioritization: A strategic reorganization in early 2025 led to discontinuing select research programs and a net workforce reduction of approximately 27% to concentrate resources on the late-stage lonvo-z and nex-z programs.
  • Manufacturing: Developing proprietary lipid nanoparticle (LNP) delivery systems is a core operational capability, ensuring the gene-editing payload reaches the target cells effectively.
  • Financial Burn: The net loss for Q3 2025 was $101.3 million, which shows the significant investment required to push these therapies through pivotal trials.

Given Company's Strategic Advantages

The strategic edge for Intellia Therapeutics, Inc. isn't just in having the CRISPR tool; it's in how they use it-specifically, their leadership in in vivo applications, which is the holy grail for systemic genetic diseases.

  • In Vivo Leadership: Intellia is a pioneer in delivering CRISPR components directly into the body, particularly the liver, which allows for a single-dose, potentially curative treatment without the complex and costly ex vivo process of removing, editing, and reinfusing a patient's cells.
  • Pipeline Differentiation: Lonvo-z is positioned as a potential single-dose functional cure for HAE, a significant advantage over existing chronic or prophylactic treatments, which is a $3.13 billion addressable market in 2025.
  • Cash Runway Resilience: Despite the recent clinical hold on nex-z, the company's cash, cash equivalents, and marketable securities of approximately $669.9 million as of September 30, 2025, are expected to fund operations into mid-2027. That runway provides crucial time to resolve the regulatory setback.
  • Deep Partnerships: The collaboration with Regeneron Pharmaceuticals, Inc. provides shared development costs, access to a global clinical and commercial infrastructure, and external validation of their platform.

Intellia Therapeutics, Inc. (NTLA) How It Makes Money

Intellia Therapeutics, Inc. is a clinical-stage biotechnology company that currently makes money almost exclusively from strategic collaboration and licensing agreements, not from product sales. The company's financial engine is fueled by upfront payments, research funding, and milestone payments tied to the progress of its gene-editing therapies, primarily through its partnership with Regeneron Pharmaceuticals, Inc. and others.

Intellia Therapeutics, Inc.'s Revenue Breakdown

As a pre-commercial company, Intellia's revenue streams are volatile and dependent on the achievement of contractual milestones, not patient demand. For the third quarter of 2025, the revenue was nearly all from its collaborations.

Revenue Stream % of Total Growth Trend
Collaboration Revenue (e.g., Regeneron) ~100% Increasing
License and Other Revenue <1% Stable/Volatile

In the third quarter of 2025, Intellia reported total revenue of approximately $13.78 million. Nearly all of this came from Collaboration Revenue, which was reported at $13.8 million for the quarter. This stream is growing, showing a 51% jump year-over-year from the $9.1 million reported in the third quarter of 2024, mostly driven by cost reimbursements from its collaboration with Regeneron Pharmaceuticals, Inc. This is a great sign of partnership commitment, but it's defintely not a sustainable commercial model yet.

Business Economics

The core economic reality for Intellia is that it operates on a high-burn, high-reward model typical of clinical-stage biotech. Its current economics are driven by research and development (R&D) investment, not gross margin on sales.

  • Cost Structure: The business is dominated by R&D spending, which was $94.7 million in Q3 2025. This is the cost of building future revenue. General and administrative (G&A) expenses, which cover things like commercial infrastructure build-out, were much lower at $30.5 million in the same quarter.
  • Future Pricing Model: The company's pipeline is centered on one-time, potentially curative treatments, such as lonvoguran ziclumeran (lonvo-z) for hereditary angioedema (HAE). These therapies typically command ultra-premium pricing-think millions of dollars per patient-to reflect the lifetime value of the cure and the massive R&D investment. The HAE global market alone was estimated at $2.86 billion in 2024, showing the size of the opportunity.
  • Cash Runway: Intellia is currently burning cash, but strategic cost-cutting and equity raises have extended its financial breathing room. The company expects its cash, cash equivalents, and marketable securities of approximately $669.9 million as of September 30, 2025, to fund operations into mid-2027. Here's the quick math: management has guided for an average cash use of about $95 million per quarter across 2025 and 2026.

The business model is a simple, high-stakes trade: spend hundreds of millions on R&D now to capture billions in product revenue later. You can learn more about the institutional interest in this model at Exploring Intellia Therapeutics, Inc. (NTLA) Investor Profile: Who's Buying and Why?

Intellia Therapeutics, Inc.'s Financial Performance

Intellia's financial performance as of November 2025 reflects a company deep in the investment phase, focused on clinical execution rather than immediate profitability.

  • Net Loss: The company reported a net loss of $101.3 million for the third quarter of 2025. This is a common and expected metric for a clinical-stage biotech, but it highlights the dependency on capital markets and partnerships.
  • Loss Per Share: The reported Earnings Per Share (EPS) for Q3 2025 was a loss of -$0.99. This figure is a key measure of burn rate for investors.
  • Liquidity: The company's current ratio of 5.19 and a low debt-to-equity ratio of 0.14 indicate a strong liquidity position and low leverage, which is crucial for weathering clinical setbacks like the recent FDA clinical hold on the nexiguran ziclumeran (nex-z) trials.
  • Capital Raising: Intellia raised an additional $114.5 million in net equity proceeds from its At the Market (ATM) program during Q3 2025, demonstrating its ability to tap capital markets to extend its runway.

The whole financial story hinges on the mid-2026 topline data for lonvo-z, which could be the catalyst for the first commercial launch in the first half of 2027. Until then, collaboration revenue and capital raises are the only things keeping the lights on.

Intellia Therapeutics, Inc. (NTLA) Market Position & Future Outlook

Intellia Therapeutics, Inc. (NTLA) is positioned at a high-risk, high-reward inflection point as of late 2025, driven by its lead pipeline asset, lonvo-z (NTLA-2002), and the recent clinical hold on nexiguran ziclumeran (nex-z). The company's future trajectory hinges on the successful commercialization of its in vivo (inside the body) CRISPR-based therapies, especially the one-time treatment for hereditary angioedema (HAE).

The strategic reorganization in early 2025, which included a 27% workforce reduction, focused the company's approximately $670 million in cash, cash equivalents, and marketable securities (as of September 30, 2025) toward these two priority programs, extending the cash runway into mid-2027. Collaboration revenue for the third quarter of 2025 was $13.8 million, reflecting the clinical-stage nature of the business; the core value is entirely in the pipeline. Honestly, the next 18 months will defintely determine Intellia's long-term value.

Competitive Landscape

In the pure-play CRISPR space, Intellia's competitive standing is measured by its market capitalization relative to peers, as commercial market share is still non-existent for most gene-editing companies. Intellia's in vivo platform, which uses a Lipid Nanoparticle (LNP) delivery system to edit genes directly in the liver, is its core competitive advantage over rivals focused on ex vivo (outside the body) editing or other delivery methods.

Company Market Share, % (Market Cap Proxy) Key Advantage
Intellia Therapeutics, Inc. 16% Leading in vivo LNP-delivered CRISPR platform
CRISPR Therapeutics 80% First-to-market CRISPR therapy (ex vivo)
Editas Medicine 4% Pioneering AAV delivery for ocular diseases

Here's the quick math: Intellia's market cap of approximately $954.44 million is significantly smaller than CRISPR Therapeutics' $4.79 billion as of November 2025. This difference reflects CRISPR Therapeutics' lead with the first approved CRISPR product, but Intellia holds a strong position as the leader in the in vivo gene-editing modality, which promises a simpler, one-time treatment for systemic diseases.

Opportunities & Challenges

The company's strategic focus maps near-term opportunities directly to the lonvo-z program, but the recent safety event for nex-z introduces a major challenge to the platform's reputation. You need to weigh the huge potential of a one-time cure against the binary risk of clinical failure.

Opportunities Risks
Lonvo-z (NTLA-2002) for HAE: Potential single-dose cure in a market estimated at $3.13 billion in 2025. Clinical hold on nex-z (ATTR) Phase 3 trial following a patient death in October 2025.
Lonvo-z Phase 1/2 data showing a 98% reduction in monthly HAE attack rates, setting a high bar for competitors. Regulatory risk and potential delays for the LNP delivery platform due to the nex-z safety signal.
Regeneron Pharmaceuticals, Inc. collaboration provides funding, expertise, and validation for the ATTR program. Competition from established, non-CRISPR therapies like Alnylam's Vutrisiran for ATTR amyloidosis.
Completion of Phase 3 HAELO enrollment in September 2025, keeping the potential U.S. launch on track for 1H 2027. High cash burn rate, which, despite the reorganization, remains a concern until commercial revenue starts.

Industry Position

Intellia Therapeutics, Inc. is a foundational pioneer in the gene-editing industry, holding a strong intellectual property position alongside its co-founders. Its industry standing is defined by its commitment to in vivo therapies, which represents the next frontier in genetic medicine.

  • In Vivo Leadership: Intellia is a leader in systemic delivery, having been the first to report clinical data for an in vivo CRISPR gene-editing therapy in humans.
  • Financial Prudence: The 2025 strategic reorganization and 27% workforce reduction demonstrate a clear focus on the most valuable assets, prioritizing capital efficiency to support operations into mid-2027.
  • Pipeline Focus: The company's entire near-term value proposition rests on the success of lonvo-z for HAE, where it holds a clear lead over other gene-editing rivals.
  • Analyst Sentiment: Despite the nex-z setback, the average analyst price target as of November 2025 suggests a significant potential upside, reflecting belief in the lonvo-z program's potential.

To understand the core principles guiding this focus, you can review the Mission Statement, Vision, & Core Values of Intellia Therapeutics, Inc. (NTLA).

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