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Intellia Therapeutics, Inc. (NTLA): Business Model Canvas [Dec-2025 Updated] |
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Intellia Therapeutics, Inc. (NTLA) Bundle
You're looking at a classic clinical-stage biotech model here, heavy on R&D and partnerships, but with the potential for massive, one-time curative value. The recent FDA hold on nex-z is a near-term risk you need to factor in, but the $670 million cash runway into mid-2027 gives Intellia Therapeutics, Inc. the financial fortitude to push lonvo-z forward. Honestly, they are burning cash-think $94.7 million on R&D in Q3 2025-to fuel that proprietary CRISPR/Cas9 platform, though collaboration revenue, like the $13.8 million from Regeneron in that same quarter, helps cover some of the $30.5 million in G&A. It's a fascinating structure balancing massive future value against immediate execution risk. Let's defintely break down the nine blocks that make this engine run below.
Intellia Therapeutics, Inc. (NTLA) - Canvas Business Model: Key Partnerships
You're looking at the structure Intellia Therapeutics, Inc. uses to advance its pipeline, and the partnerships are where a lot of the development muscle and financial risk-sharing comes from. It's not all on the balance sheet; a good chunk is shared.
Regeneron Pharmaceuticals, Inc. for Co-development and Cost-Sharing of nex-z
The collaboration with Regeneron Pharmaceuticals, Inc. on nexiguran ziclumeran (nex-z) is a major component of the business model for that asset. Under the agreement, Regeneron Pharmaceuticals, Inc. shares 25% of the development costs and commercial profits for nex-z. This sharing mechanism is reflected in Intellia Therapeutics, Inc.'s reported revenue; for the second quarter of 2025, Collaboration Revenue was $14.2 million, an increase of $7.3 million over the second quarter of 2024, driven by cost reimbursements from Regeneron Pharmaceuticals, Inc.. Similarly, third quarter 2025 Collaboration Revenue hit $13.8 million, up $4.7 million year-over-year due to these reimbursements.
The Phase 3 trials for nex-z faced a significant event on October 29, 2025, when the U.S. Food and Drug Administration (FDA) placed a clinical hold on both the MAGNITUDE and MAGNITUDE-2 trials. Before this, enrollment in the MAGNITUDE trial (ATTR-CM) was tracking to enroll at least 650 patients cumulatively by year-end 2025, with an amendment to expand total enrollment to approximately 1,200 patients from an initial 765 patients. Historically, the initial 2016 deal included a $75 million upfront payment to Intellia Therapeutics, Inc., plus a $50 million investment in the IPO, with milestones up to $320 million.
| Metric | Value / Term | Program |
|---|---|---|
| Cost/Profit Share (Regeneron) | 25% | nex-z (ATTR Amyloidosis) |
| Q2 2025 Collaboration Revenue | $14.2 million | Total (Regeneron component significant) |
| Q3 2025 Collaboration Revenue | $13.8 million | Total (Regeneron component significant) |
| MAGNITUDE Enrollment Target (Pre-Hold Amendment) | 1,200 patients (Amended from 765) | nex-z (ATTR-CM) |
| Historical Upfront Payment (2016) | $75 million | CRISPR Collaboration |
Academic Institutions for Foundational CRISPR/Cas9 Intellectual Property Licensing
Intellia Therapeutics, Inc. secures its foundational technology access through licenses from third parties. The company accesses patent estates covering foundational filings on CRISPR/Cas9 systems, LNP delivery technologies, and cell expansion technology via licenses from Caribou Biosciences and Novartis. Intellia Therapeutics, Inc. previously sublicensed its technology from Caribou Biosciences. The company is actively managing the risk associated with foundational IP, noting that licensed patent portfolios are subject to ongoing interference proceedings between the Broad Institute and UC Berkeley.
- Access to IP via licenses from Caribou Biosciences.
- Access to IP via licenses from Novartis.
- Risk exposure related to Broad Institute/UC Berkeley patent interference proceedings.
Contract Research Organizations (CROs) for Global Phase 3 Clinical Trial Execution
Execution of the late-stage trials relies on CROs to manage global site operations. Enrollment for the wholly owned lonvoguran ziclumeran (lonvo-z) Phase 3 HAELO study for Hereditary Angioedema (HAE) was completed in September 2025. Patients in the HAELO trial are receiving a 50 milligram (mg) dose of lonvo-z. For nex-z, the MAGNITUDE-2 study (ATTRv-PN) expects enrollment completion in the first half of 2026. Research and development expenses, which include contracted services like CRO work, were $94.7 million for the third quarter of 2025.
Contract Manufacturing Organizations (CMOs) for LNP and Drug Substance Production
Intellia Therapeutics, Inc. is pioneering proprietary platform technologies, including extrahepatic lipid nanoparticle (LNP) delivery technologies, to create product candidates. The company's strategy involves leveraging existing third-party expertise for manufacturing. Should a CMO fail its obligations, Intellia Therapeutics, Inc. notes the potential for significant delays in clinical trial supply as it would need to establish alternative sources, for which it may lack the capabilities or resources to self-manufacture.
The company ended the third quarter of 2025 with $669.9 million in cash, cash equivalents and marketable securities, expected to fund operations into mid-2027.
Intellia Therapeutics, Inc. (NTLA) - Canvas Business Model: Key Activities
You're looking at the core engine of Intellia Therapeutics, Inc. right now-the day-to-day, high-stakes work that determines if their CRISPR platform moves from the lab bench to the patient bedside. As of late 2025, the Key Activities are laser-focused on late-stage execution and preparing for a commercial future, even while navigating a significant regulatory hurdle.
Executing pivotal Phase 3 clinical trials for lonvo-z (HAE) and nex-z (ATTR)
The primary activity is driving two distinct, high-value, in vivo CRISPR programs through their final clinical stages. For lonvo-z, treating hereditary angioedema (HAE), Intellia Therapeutics hit a major enrollment milestone. They completed patient enrollment in the global Phase 3 HAELO study in September 2025, which was less than nine months after dosing the first patient in January 2025. This rapid enrollment is a key operational success. The next critical step here is the topline data readout, which is expected by mid-2026, putting them on track for a Biologics License Application (BLA) submission in the second half of 2026 and an anticipated U.S. launch in the first half of 2027.
For nex-z, targeting transthyretin amyloidosis (ATTR), the activity is focused on patient accrual across two trials, though currently complicated by a regulatory hold. The MAGNITUDE trial (ATTR-CM) is progressing well, tracking to enroll at least 650 patients cumulatively by the end of 2025, with plans to expand total enrollment to approximately 1,200 patients, pending health authority review. The MAGNITUDE-2 trial (ATTRv-PN) expects to complete enrollment in the first half of 2026.
Here's a quick look at the trial status as of the third quarter of 2025:
| Program | Indication | Phase 3 Trial Name | Key 2025 Milestone Achieved | Next Major Data Expectation |
| lonvo-z | HAE | HAELO | Enrollment completed in September 2025 | Topline data by mid-2026 |
| nex-z | ATTR-CM | MAGNITUDE | Tracking to enroll at least 650 patients cumulatively | Longer-term Phase 1 data presented in November 2025 |
| nex-z | ATTRv-PN | MAGNITUDE-2 | First patient dosed in Q1 2025 | Enrollment completion by H1 2026 |
Research and Development (R&D) of next-gen CRISPR and delivery platforms
Intellia Therapeutics is actively managing its R&D spend to support these late-stage assets while pruning earlier-stage work. The company made a strategic pivot in early 2025, discontinuing development of NTLA-3001 for alpha-1 antitrypsin deficiency-associated lung disease and cutting select research-stage programs. This was part of a reorganization designed to focus resources on NTLA-2002 and nex-z.
The financial commitment to R&D reflects this prioritization. For instance, Research and Development expenses were $94.7 million in the third quarter of 2025, a decrease from $123.4 million in the third quarter of 2024. Still, the core technology-the CRISPR/Cas9 platform and its delivery methods-remains the foundation of all future value.
- R&D Expense (Q3 2025): $94.7 million
- R&D Expense (Q2 2025): $97.0 million
- R&D Expense (Q1 2025): $108.4 million
- Programs discontinued in 2025 reorganization: NTLA-3001 and select research-stage assets.
Managing regulatory filings and addressing the FDA clinical hold on nex-z
This is arguably the most critical, near-term activity following adverse events in late 2025. The U.S. Food and Drug Administration (FDA) formally placed a clinical hold on both Phase 3 trials for nex-z on October 29, 2025. This followed Intellia Therapeutics' voluntary pause after a patient hospitalized with Grade 4 liver transaminases and increased total bilirubin following dosing on September 30, 2025, tragically passed away.
The immediate activity involves intense engagement with regulators. Intellia Therapeutics is working with clinical investigators and external experts to understand the liver-related events and develop a risk mitigation plan while awaiting the formal clinical hold letter, which was expected within 30 days of the verbal notification. This regulatory uncertainty has forced the company to suspend its milestone guidance for nex-z. To be fair, prior to this event, nex-z for ATTR-CM had secured the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation.
Building out a specialized commercial and medical affairs infrastructure
To prepare for the potential first half of 2027 launch of lonvo-z, Intellia Therapeutics is actively shifting its focus from purely clinical development to commercial readiness. A key operational goal for 2025 was the 'complete buildout of the commercial leadership team by second half of 2025.' Furthermore, the company is already hiring its sales and operations team in anticipation of this launch.
This preparation includes significant medical affairs work:
- Expand medical education activities in both HAE and ATTR amyloidosis.
- Initiate pre-approval information exchange with payers for coverage planning.
- Focus resources on the two late-stage programs to ensure efficient execution toward launch.
Financially, the company ended the third quarter of 2025 with $669.9 million in cash, cash equivalents, and marketable securities, which they expect funds operations into mid-2027, providing a runway to navigate the nex-z hold and reach the lonvo-z launch. This cash position was bolstered by raising $114.5 million in net equity proceeds via their ATM program during Q3 2025. Finance: draft 13-week cash view by Friday.
Intellia Therapeutics, Inc. (NTLA) - Canvas Business Model: Key Resources
You're looking at the core assets Intellia Therapeutics, Inc. relies on to execute its strategy as of late 2025. These aren't just line items; they are the engines driving the next phase of their gene-editing pipeline.
Proprietary CRISPR/Cas9 gene-editing platform technology.
Intellia Therapeutics, Inc. fundamentally relies on its proprietary CRISPR/Cas9 platform. This technology is the foundation for its in vivo (inside the body) and ex vivo (outside the body) therapeutic candidates. The company has specifically prioritized resources toward its late-stage programs, nexiguran ziclumeran (nex-z) for ATTR amyloidosis and lonvoguran ziclumeran (lonvo-z) for Hereditary Angioedema (HAE).
Cash, cash equivalents, and marketable securities of approximately $670 million (Q3 2025).
The balance sheet provides the immediate operational runway. Here's the quick math on the liquidity position reported at the end of the third quarter of 2025:
| Metric | Amount/Period |
| Cash, Cash Equivalents, and Marketable Securities (as of September 30, 2025) | $669.9 million |
| Cash, Cash Equivalents, and Marketable Securities (as of December 31, 2024) | $861.7 million |
| Net Equity Proceeds Raised in Q3 2025 (via ATM) | $114.5 million |
| Projected Cash Runway | Into mid-2027 |
This cash position is critical, as it is expected to fund operations through lonvo-z's anticipated U.S. commercial launch for HAE.
Specialized scientific talent and clinical development expertise.
The human capital is centered on transitioning from late-stage development to commercial readiness. This required strategic shifts in personnel focus throughout 2025. The company completed the buildout of its commercial leadership team by the second half of 2025. However, this focus came with internal restructuring, including a net workforce reduction of approximately 27% over the course of 2025. Furthermore, key leadership transitions were underway, with the Chief Scientific Officer announcing retirement effective December 31, 2025, transitioning to a Senior Scientific Advisor role for the remainder of the year.
The expertise is currently concentrated on advancing these specific programs:
- Advancing the Phase 3 HAELO clinical trial for lonvo-z (HAE) with enrollment completed in Q3 2025.
- Managing the FDA clinical hold on the MAGNITUDE and MAGNITUDE-2 trials for nex-z (ATTR amyloidosis).
- Preparing for topline data from the HAELO trial expected by mid-2026.
Intellectual Property (IP) portfolio covering in vivo and ex vivo applications.
The value of Intellia Therapeutics, Inc. is heavily tied to its IP estate covering its gene-editing technology across both major application types. Protecting this portfolio is a stated risk, as adverse legal rulings could result in the loss of key intellectual property and financial rights, requiring substantial legal expense and diverting employee resources. The company's collaborations, such as the one with Regeneron Pharmaceuticals, Inc., also involve shared IP considerations and revenue streams.
Key IP-related assets and focus areas include:
- Patents covering the use of CRISPR/Cas9 for in vivo editing, particularly in the liver for ATTR amyloidosis.
- IP surrounding the NTLA-2002 (lonvo-z) construct for HAE.
- The ability to protect its platform against competitors developing therapies for ATTR amyloidosis.
Intellia Therapeutics, Inc. (NTLA) - Canvas Business Model: Value Propositions
Intellia Therapeutics, Inc.'s value proposition centers on delivering potentially transformative, single-dose treatments for severe genetic diseases using its in vivo (inside the body) CRISPR-based gene editing platform.
Potential for one-time, curative treatment for severe genetic diseases.
The core offering is the potential for a single administration to provide a deep, consistent, and potentially lifelong therapeutic effect for debilitating conditions. This is exemplified by nexiguran ziclumeran (nex-z) for Transthyretin Amyloidosis (ATTR), which aims to drive a deep, consistent, and potentially lifelong reduction in TTR protein after a single dose. The company ended the third quarter of 2025 with approximately \$669.9 million in cash, cash equivalents and marketable securities, expected to fund operations into mid-2027, supporting the long development cycle required for curative therapies.
Highly effective, single-dose therapy for lifelong control of HAE attacks (lonvo-z).
For Hereditary Angioedema (HAE), lonvoguran ziclumeran (lonvo-z) is positioned as a single-dose therapy designed to inactivate the KLKB1 gene in the liver, aiming for lifelong control of HAE attacks by driving consistent, deep, and potentially lifelong reduction in kallikrein levels. The HAE total addressable market in 2025 was estimated at \$3.13 billion. Enrollment in the Phase 3 HAELO clinical trial for lonvo-z was completed in September 2025. Topline data is anticipated by mid-2026, with a potential U.S. commercial launch targeted for the first half of 2027.
In vivo (inside the body) gene editing, simplifying treatment delivery.
Intellia Therapeutics, Inc. focuses its entire pipeline on two single-dose in vivo gene editing product candidates, lonvo-z and nex-z. This approach avoids the complexity of ex vivo (outside the body) editing, where cells must be removed, modified, and reinfused. The company's Q3 2025 Research and Development (R&D) expenses were \$94.7 million, reflecting the investment in advancing this platform.
Precision medicine targeting the genetic root cause of disease.
The therapies offer precision by targeting the specific genetic error causing the disease. Nex-z is designed to inactivate the TTR gene in the liver for ATTR amyloidosis, while lonvo-z targets the KLKB1 gene for HAE. The company is advancing nex-z in two late-stage studies, MAGNITUDE and MAGNITUDE-2, with more than 650 patients enrolled in the ATTR-CM study (MAGNITUDE) to date.
Here is a snapshot of the key late-stage pipeline progress as of late 2025:
| Program | Indication | Target Gene | Phase 3 Trial Status (as of late 2025) | Key Data/Filing Timeline |
| lonvo-z (NTLA-2002) | Hereditary Angioedema (HAE) | KLKB1 | Enrollment completed in September 2025 in HAELO study. | Topline data by mid-2026; BLA submission in H2 2026. |
| nex-z (NTLA-2001) | ATTR Amyloidosis with Cardiomyopathy (ATTR-CM) | TTR | Ongoing enrollment in MAGNITUDE; over 650 patients enrolled. | Enrollment in MAGNITUDE-2 expected completion by H1 2026. Commercial stage projected by 2028. |
The company's Q3 2025 net loss was \$101.3 million, which narrowed from previous periods partly due to a reduction in R&D spending to \$94.7 million for the quarter. Collaboration revenue for Q3 2025 was \$13.8 million.
The value proposition is further detailed by the planned next steps for the pipeline:
- Anticipated presentation of longer-term Phase 1 clinical data for nex-z for ATTR-CM on November 10, 2025, at AHA 2025.
- Intellia remains on track to submit a potential Biologics License Application (BLA) for lonvo-z in HAE in the second half of 2026.
- The company's cash position of \$669.9 million as of September 30, 2025, is expected to fund operations through mid-2027.
Intellia Therapeutics, Inc. (NTLA) - Canvas Business Model: Customer Relationships
You're building a commercial-ready organization, and for a company like Intellia Therapeutics, Inc., the relationships with the clinical community and partners are the absolute core of the business right now. These aren't just nice-to-haves; they directly translate into trial success and future revenue streams.
High-touch, collaborative relationships with key opinion leaders (KOLs) and clinical investigators
The relationship with the clinical investigators running your pivotal trials is intensely collaborative, especially given the novel nature of CRISPR-based therapies. You need deep trust to manage complex protocols and safety monitoring. The success in trial enrollment speaks volumes about the KOL engagement you've fostered.
For the ATTR amyloidosis with cardiomyopathy (ATTR-CM) program, the MAGNITUDE Phase 3 trial has enrolled more than 650 patients as of November 6, 2025, which was tracking ahead of internal projections. Furthermore, Intellia Therapeutics is amending the study to expand enrollment to approximately 1,200 patients from the initial 765, pending health authority review. For the hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) arm, MAGNITUDE-2 had 47 patients enrolled as of that same date.
The engagement is also demonstrated by the scientific exchange, where longer-term Phase 1 clinical data for nex-z in ATTR-CM was presented on November 10, 2025, at the American Heart Association (AHA) Scientific Sessions. For the hereditary angioedema (HAE) program, the HAELO Phase 3 trial completed enrollment in September 2025, less than nine months after dosing the first patient.
Here's a snapshot of the clinical execution metrics:
| Trial/Program | Key Metric | Value as of Late 2025 |
| MAGNITUDE (ATTR-CM) | Patients Enrolled (as of Nov 6, 2025) | More than 650 |
| MAGNITUDE (ATTR-CM) | Targeted Expansion Enrollment | Approximately 1,200 |
| MAGNITUDE-2 (ATTRv-PN) | Patients Enrolled (as of Nov 6, 2025) | 47 |
| HAELO (NTLA-2002) | Enrollment Status | Completed in September 2025 |
Close engagement with patient advocacy groups for trial recruitment and support
Patient advocacy groups are critical for awareness and driving the necessary patient flow into these specialized trials. Intellia Therapeutics explicitly made this a strategic focus for 2025.
- Strategic priority for 2025 included expanding medical education in HAE and ATTR amyloidosis in partnership with key medical societies and patient organizations.
- The company is evolving into a commercial-ready organization by the end of 2026, which requires building strong patient trust now.
Strategic, long-term partnership management with pharmaceutical collaborators
The collaboration with Regeneron Pharmaceuticals, Inc. is a cornerstone of the financial structure for the ATTR program. This relationship provides significant cost-sharing and future commercial upside, but it also ties a portion of your revenue stream to partner performance.
For the first nine months of 2025, collaboration revenue totaled $45 million, the same as the prior year period, driven by cost reimbursements related to the Regeneron collaboration. In the third quarter of 2025 alone, collaboration revenue was $13.8 million. Regeneron shares approximately 25% of worldwide development costs and commercial profits for the ATTR program. Plus, Regeneron holds an option to enter into a co-promotion agreement for the U.S. commercialization of nex-z.
Here's how the financial relationship looks for the near term:
| Metric | Period Ending Q3 2025 | Prior Year Q3 2024 |
| Collaboration Revenue | $13.8 million | $9.1 million |
| Collaboration Revenue (9 Months) | $45 million | $45 million |
Future direct-to-specialist medical education and support
As Intellia Therapeutics moves toward its anticipated U.S. commercial launch for lonvo-z in the first half of 2027, the focus shifts to preparing the specialist community. The company completed the buildout of its commercial leadership team by the second half of 2025. This infrastructure is what will support the future direct-to-specialist engagement required for adoption once a Biologics License Application (BLA) is submitted in the second half of 2026. The goal is to ensure specialists are educated on the one-time nature of these therapies well ahead of launch.
The company also planned to 'Initiate pre-approval information exchange to allow payers to begin planning for coverage and formulary decisions' in 2025. That's a key relationship management task for the future commercial success of NTLA-2002 and nex-z.
Finance: draft 13-week cash view by Friday.
Intellia Therapeutics, Inc. (NTLA) - Canvas Business Model: Channels
You're hiring before product-market fit, so how you get your science in front of doctors and investors is everything. Here's how Intellia Therapeutics, Inc. is using its channels as of late 2025.
Global network of specialized clinical trial sites and hospitals
The clinical trial network is the primary channel for generating the necessary human data to support future commercialization. Enrollment momentum is a key metric here.
- Phase 3 HAELO study for lonvo-z in Hereditary Angioedema (HAE) completed enrollment in September 2025.
- MAGNITUDE trial for nex-z in ATTR with cardiomyopathy (ATTR-CM) is tracking to enroll at least 650 patients cumulatively by year-end 2025.
- Intellia is seeking health authority review to expand the MAGNITUDE trial size to approximately 1,200 patients.
- The MAGNITUDE-2 trial for ATTR with polyneuropathy (ATTRv-PN) expects enrollment completion in the first half of 2026.
The company is actively preparing for a potential U.S. commercial launch of lonvo-z in the first half of 2027, which dictates the urgency of these site operations.
Direct-to-specialist sales force for future commercial launch
Building the commercial engine is happening concurrently with late-stage trials. This channel is about positioning for market entry.
- Intellia Therapeutics, Inc. is planning a complete buildout of the commercial leadership team by the second half of 2025.
- The company welcomed Jim McNinch, Vice President, U.S. Head of Sales, and Ben Newman, Vice President, Commercial Operations, since the beginning of 2025.
- The strategic goal is to transition to a commercial-ready organization by the end of 2026.
This buildout is focused on the specialists treating Hereditary Angioedema (HAE) and ATTR amyloidosis.
Scientific publications and presentations at major medical congresses
These channels are crucial for establishing scientific credibility and educating key opinion leaders (KOLs) on the in vivo CRISPR data.
Here's a look at key 2025 data dissemination events:
| Program | Event/Publication | Date/Period | Data Type |
| nex-z (ATTR-CM) | 5th International ATTR Amyloidosis Meeting for Patients and Doctors | September 2025 | Longer-term Phase 1 follow-up data |
| nex-z (ATTR-CM) | American Heart Association (AHA) Scientific Sessions | November 10, 2025 | Longer-term Phase 1 data |
| lonvo-z (HAE) | ACAAI 2025 | November 8, 2025 | Longer-term Phase 1/2 data |
| lonvo-z (HAE) | European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025 | June 15, 2025 | Three-year follow-up data from Phase 1/2 |
| nex-z (ATTRv-PN) | 2025 Peripheral Nerve Society (PNS) Annual Meeting | May 2025 | Positive two-year follow-up Phase 1 data |
Also, longer-term Phase 1 data for nex-z for ATTRv-PN was simultaneously published in the New England Journal of Medicine in September 2025.
Investor Relations for capital markets communication
The Investor Relations function channels financial health and strategic milestones to stockholders and analysts, directly impacting capital access.
Key financial metrics as of the third quarter of 2025:
- Cash, cash equivalents and marketable securities: $669.9 million as of September 30, 2025.
- Net equity proceeds raised via ATM program in Q3 2025: $114.5 million.
- Projected cash runway extends into mid-2027.
- Trailing twelve-month revenue as of September 30, 2025: $57.5M.
- Q3 2025 Net Loss: $101.3 million.
Finance: draft 13-week cash view by Friday.
Intellia Therapeutics, Inc. (NTLA) - Canvas Business Model: Customer Segments
You're looking at the core groups Intellia Therapeutics, Inc. serves, which are defined by the rare, severe diseases they target with CRISPR technology.
The most direct customers are the patients themselves, but the commercial pathway involves several other critical entities that influence access and adoption.
| Customer Sub-Segment | Target Disease/Program | Key Statistical/Trial Data (as of late 2025) |
| Patients with ATTR Amyloidosis | nexiguran ziclumeran (nex-z) for ATTR-CM | MAGNITUDE trial tracking to enroll at least 650 patients cumulatively by year-end 2025; expansion to approximately 1,200 patients from 765 is being pursued. |
| Patients with ATTR Amyloidosis | nexiguran ziclumeran (nex-z) for ATTRv-PN | 47 patients enrolled in the MAGNITUDE-2 Phase 3 study as of Q3 2025. Enrollment completion expected by first half of 2026. |
| Patients with Hereditary Angioedema (HAE) | lonvoguran ziclumeran (lonvo-z) | Enrollment in the Phase 3 HAELO study was completed in September 2025. Randomization expected to complete in Q3 2025. |
| Patients with ATTR Amyloidosis (Total Addressable Market Estimate) | ATTR Amyloidosis | Estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis. |
Specialist physicians are the gatekeepers for prescribing these novel therapies, and their adoption hinges on clinical data and ease of administration.
- Specialist physicians treating HAE are showing demand, reflected in the rapid enrollment of the HAELO trial.
- Cardiologists and neurologists treating ATTR-CM and ATTRv-PN are key prescribers, with the MAGNITUDE trial showing patient stabilization or improvement out to 24 months in a majority of participants.
Pharmaceutical companies represent a crucial segment for Intellia Therapeutics, Inc. through strategic alliances that fund development and provide commercial reach.
- The primary partner is Regeneron Pharmaceuticals, Inc. for the nex-z program.
- Regeneron shares 25% of the development costs and commercial profits for nex-z.
- Collaboration revenue, primarily cost reimbursements from Regeneron, was $13.8 million for Q3 2025 and $14.2 million for Q2 2025.
Regulatory bodies are essential stakeholders whose decisions dictate market entry and timing, effectively controlling access to the patient segment.
- The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to nex-z for ATTR-CM in March 2025.
- The FDA placed a clinical hold on the MAGNITUDE and MAGNITUDE-2 Phase 3 trials for nex-z in October 2025.
- Anticipated U.S. commercial launch for lonvo-z (HAE) is targeted for the first half of 2027.
- BLA submission for lonvo-z (HAE) is targeted for the second half of 2026.
Intellia Therapeutics, Inc. (NTLA) - Canvas Business Model: Cost Structure
You're looking at the expense side of Intellia Therapeutics, Inc.'s operations as they push toward commercial readiness. The cost structure is heavily weighted toward the science and the necessary buildout for future product launches. Here's the quick math on the most recent reported figures from late 2025.
Research and Development (R&D) expenses represent the largest operational outlay, reflecting the commitment to advancing the late-stage pipeline programs, lonvo-z and nex-z. For the third quarter of 2025, Intellia Therapeutics, Inc. reported R&D expenses of $94.7 million. This was a year-over-year decrease from the $123.4 million reported in Q3 2024. However, this reduction was partially offset by an increase in clinical trial expenses specifically related to lonvo-z.
General and Administrative (G&A) costs are climbing as the company prepares for potential commercialization. G&A expenses for the third quarter of 2025 were reported at $30.5 million, matching the figure from the third quarter of 2024. This category includes the costs associated with the commercial buildout, such as implementing core commercialization and medical capabilities and completing the buildout of the commercial leadership team by the second half of 2025.
The company also absorbed significant one-time costs related to strategic realignment early in the year. Specifically, Intellia Therapeutics, Inc. expected to incur restructuring charges of approximately $8 million associated with the January 2025 workforce reduction, which were anticipated to be recorded in the first quarter of 2025. To be fair, these restructuring costs were part of larger non-recurring cash payments totaling approximately $51 million in the first quarter of 2025, which also covered portfolio prioritization and real estate consolidation.
Clinical trial costs are embedded within the R&D spend but are a critical driver of the burn rate, especially with pivotal Phase 3 studies ongoing. The focus on clinical execution for lonvo-z (HAELO trial) and nex-z (MAGNITUDE and MAGNITUDE-2 trials) dictates a significant portion of the R&D budget. The costs cover essential activities like:
- Patient enrollment for ongoing trials.
- Site management and monitoring.
- Advancement of lead programs, despite regulatory setbacks.
Here is a snapshot comparing the most recent reported operating expenses:
| Expense Category | Q3 2025 Amount (Millions USD) | Q3 2024 Amount (Millions USD) |
| Research and Development (R&D) | $94.7 | $123.4 |
| General and Administrative (G&A) | $30.5 | $30.5 |
Also, remember that stock-based compensation is a non-cash component within these figures. For Q3 2025, stock-based compensation included in R&D was $12.2 million, and in G&A was $7.4 million. The overall net loss for Q3 2025 was $101.3 million.
Finance: draft 13-week cash view by Friday.
Intellia Therapeutics, Inc. (NTLA) - Canvas Business Model: Revenue Streams
You're looking at the core ways Intellia Therapeutics, Inc. brings in cash right now, which is heavily weighted toward its development partnerships rather than product sales, given its clinical stage. Honestly, the numbers reflect a company still deep in the R&D phase, relying on partners to share the load.
The most concrete, recurring revenue stream as of late 2025 is the Collaboration Revenue, which is largely cost reimbursements from partners like Regeneron Pharmaceuticals, Inc. For the third quarter of 2025, Intellia Therapeutics, Inc. reported $13.8 million in collaboration revenue. Looking at the bigger picture, for the first nine months of 2025, this collaboration revenue totaled $45 million. This revenue helps offset the significant Research and Development expenses, which were $94.7 million in Q3 2025.
Here's a quick look at how the current and near-term revenue sources are structured:
| Revenue Component | Latest Reported Figure | Period/Context | Associated Program/Partner |
|---|---|---|---|
| Collaboration Revenue (Cost Reimbursements) | $13.8 million | Q3 2025 | Regeneron Pharmaceuticals, Inc. (Primary Driver) |
| Collaboration Revenue (Year-to-Date) | $45 million | First Nine Months of 2025 | Regeneron (ATTR Program) |
| Potential Future Milestone Payments | Not specified for Q3 2025 | Future Clinical/Regulatory Goals | Nexiguran Ziclumeran (nex-z) |
| Anticipated U.S. Commercial Launch | 1H27 | Future Product Sales | Lonvoguran Ziclumeran (lonvo-z) for HAE |
You've got to keep an eye on Potential future milestone payments, as these are crucial non-dilutive capital injections tied to clinical or regulatory success. Right now, the path for these payments is clouded because the FDA placed a clinical hold on the MAGNITUDE and MAGNITUDE-2 Phase 3 trials for nex-z due to safety concerns. This regulatory uncertainty is the primary near-term threat to the timeline for achieving and recognizing those milestone payments.
The big potential shift in the revenue profile comes with Future product sales from approved therapies, which Intellia Therapeutics, Inc. is actively preparing for. Specifically, for lonvo-z (NTLA-2002) for Hereditary Angioedema (HAE), topline data is expected by mid-2026, with the company planning a U.S. commercial launch in the first half of 2027. The company has expanded its commercial and medical affairs teams to build a strong foundation for this potential revenue stream.
Finally, the structure of the existing partnerships dictates future income from Licensing fees and royalties from partnered programs. For the nex-z program with Regeneron, Regeneron shares approximately 25% of worldwide development costs and commercial profits. Furthermore, the company has recently streamlined its external collaborations; Intellia Therapeutics, Inc. reported the termination of the ReCode agreement in September 2025 and the termination of the SparingVision collaboration in October 2025.
You should track the cash runway, which as of September 30, 2025, stood at approximately $670 million, expected to fund operations into mid-2027. Finance: draft 13-week cash view by Friday.
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