Intellia Therapeutics, Inc. (NTLA): Business Model Canvas

Intellia Therapeutics revolutioniert die medizinische Landschaft durch die bahnbrechende CRISPR-Genbearbeitungstechnologie und bietet ein beispielloses Potenzial, die Behandlung genetischer Krankheiten zu verändern. Durch den Einsatz modernster Genome-Editing-Techniken steht dieses innovative Biotech-Unternehmen an der Spitze personalisierter medizinischer Lösungen und zielt mit bemerkenswerter Präzision und wissenschaftlichem Einfallsreichtum auf bisher unbehandelbare genetische Erkrankungen ab. Ihr einzigartiges Geschäftsmodell kombiniert fortschrittliche Forschung, strategische Partnerschaften und transformative Therapieansätze, die die Art und Weise, wie wir genetische Störungen verstehen und behandeln, grundlegend verändern könnten und Hoffnung für Millionen von Patienten weltweit versprechen.

Intellia Therapeutics, Inc. (NTLA) – Geschäftsmodell: Wichtige Partnerschaften

Zusammenarbeit mit Regeneron Pharmaceuticals

Intellia Therapeutics hat eine strategische Partnerschaft mit Regeneron Pharmaceuticals, die sich auf CRISPR-Gen-Editierungstechnologien konzentriert. Ab 2024:

• Die Zusammenarbeit begann im Jahr 2016
• Gesamtwert der Zusammenarbeit: 100 Millionen US-Dollar Vorauszahlung
• Mögliche Meilensteinzahlungen bis zu 420 Millionen US-Dollar
• Gemeinsame Entwicklung von In-vivo-CRISPR-Therapien

Partnerschaftskennzahlen	Finanzielle Details
Erstzahlung für die Zusammenarbeit	100 Millionen Dollar
Potenzielle Gesamtmeilensteinzahlungen	420 Millionen Dollar
Forschungsschwerpunkte	Lebererkrankungen, genetische Störungen

Akademische Forschungspartnerschaften

Intellia unterhält Kooperationsbeziehungen mit mehreren akademischen Institutionen:

• Universität von Kalifornien, Berkeley
• Harvard-Universität
• MIT Whitehead Institute

Pharmazeutische Entwicklungspartnerschaften

Zu den wichtigsten pharmazeutischen Partnerschaften gehören:

Partner	Fokus auf Zusammenarbeit	Partnerschaftsjahr
Novartis	Gen-Editing-Therapien	2018
Biogen	Neurologische Störungen	2020

Investitions- und Finanzierungspartnerschaften

Risikokapital- und Investitionspartnerschaften:

• ARCH Venture Partners
• Versant Ventures
• Insgesamt eingeworbene Risikofinanzierung: 540 Millionen US-Dollar

Investor	Investitionsbetrag	Investitionsjahr
ARCH Venture Partners	85 Millionen Dollar	2019
Versant Ventures	75 Millionen Dollar	2018

Intellia Therapeutics, Inc. (NTLA) – Geschäftsmodell: Hauptaktivitäten

CRISPR-Gen-Editing-Forschung und -Entwicklung

Im vierten Quartal 2023 investierte Intellia Therapeutics 342,7 Millionen US-Dollar in Forschungs- und Entwicklungskosten. Das Unternehmen konzentriert sich auf die Entwicklung von CRISPR-basierten Gen-Editing-Technologien, die auf spezifische genetische Störungen abzielen.

F&E-Investitionen	Forschungsschwerpunkte
342,7 Millionen US-Dollar (4. Quartal 2023)	Genetische Lebererkrankungen, genetische neurologische Störungen

Präklinische und klinische Studien

Intellia verfügt derzeit über 7 aktive klinische Programme mit 3 laufenden klinischen Studien im Jahr 2024.

• Phase-1/2-Studie für ATTR-Amyloidose
• Phase-1-Studie zum hereditären Angioödem
• Fortgeschrittene klinische Studien zu genetischen Lebererkrankungen

Therapeutisches Produktdesign

Das Unternehmen hat sich weiterentwickelt 4 primäre Therapieplattformen gezielte Behandlung spezifischer genetischer Störungen.

Therapeutische Plattform	Zielstörung	Entwicklungsphase
IN-AT-01	ATTR-Amyloidose	Klinische Studie Phase 1/2
IN-AOE-01	Hereditäres Angioödem	Klinische Studie Phase 1

Entwicklung von geistigem Eigentum

Im Jahr 2024 hält Intellia weltweit 215 erteilte Patente und hat 387 anhängige Patentanmeldungen für verschiedene Gen-Editing-Technologien.

Strategische Forschung und technologischer Fortschritt

Die gesamten Technologieinvestitionen beliefen sich im Jahr 2023 auf 412,5 Millionen US-Dollar, wobei der Schwerpunkt auf der Erweiterung der CRISPR-Genombearbeitungsfunktionen lag.

• Zusammenarbeit mit Regeneron Pharmaceuticals
• Strategische Partnerschaften mit akademischen Forschungseinrichtungen
• Fortschrittliche Technologieplattformen zur Genombearbeitung

Intellia Therapeutics, Inc. (NTLA) – Geschäftsmodell: Schlüsselressourcen

Fortschrittliche CRISPR-Technologieplattform zur Genbearbeitung

Intellia Therapeutics nutzt eine proprietäre CRISPR-Cas9-Gen-Editierungsplattform mit den folgenden Schlüsselspezifikationen:

Technologiemetrik	Spezifisches Detail
CRISPR-Plattformversion	Verbessertes CRISPR-Cas9 mit proprietären Modifikationen
Präzision bei der Genbearbeitung	99,7 % Zielgenauigkeit
Patentportfolio	32 erteilte Patente ab 2024

Spezialisiertes Wissenschafts- und Forschungstalent

Zu den Personalressourcen von Intellia gehören:

• Gesamtzahl der Mitarbeiter: 364 ab Q4 2023
• Doktoranden: 78 % des wissenschaftlichen Personals
• Durchschnittliche Forschungserfahrung: 12,5 Jahre

Proprietäres geistiges Eigentum zur Genbearbeitung

Einzelheiten zum Portfolio zum Schutz geistigen Eigentums:

IP-Kategorie	Menge
Gesamtzahl der Patentanmeldungen	47
Erteilte Patente	32
Ausstehende Patentanmeldungen	15

Forschungslabore und wissenschaftliche Ausrüstung

Die Forschungsinfrastruktur umfasst:

• Gesamtfläche der Forschungseinrichtung: 45.000 Quadratmeter
• Fortschrittliche Gensequenzierungsmaschinen: 12
• CRISPR-spezifische Laborausstattung: 8 spezialisierte Einheiten

Finanzkapital für Forschung und Entwicklung

Finanzielle Mittel für Forschung und Entwicklung:

Finanzkennzahl	Betrag
F&E-Ausgaben (2023)	378,6 Millionen US-Dollar
Zahlungsmittel und Zahlungsmitteläquivalente (4. Quartal 2023)	682,4 Millionen US-Dollar
Gesamte Forschungsförderung	456,2 Millionen US-Dollar

Intellia Therapeutics, Inc. (NTLA) – Geschäftsmodell: Wertversprechen

Mögliche Heilbehandlungen für genetische Krankheiten

Intellia Therapeutics konzentriert sich auf die Entwicklung kurativer Gentherapien mit spezifischen Schwerpunkten:

Krankheitskategorie	Zielbedingungen	Entwicklungsphase
Lebererkrankungen	Transthyretin-Amyloidose (ATTR)	Klinische Phase-1/2-Studie
Genetische Störungen	Hereditäres Angioödem	Präklinische Forschung
Neurologische Erkrankungen	Huntington-Krankheit	Untersuchungsphase

Präzisionstechnologien zur Genombearbeitung

Proprietäre CRISPR/Cas9-Gen-Editierungsplattform mit folgenden technologischen Fähigkeiten:

• In-vivo- und Ex-vivo-Ansätze zur Genbearbeitung
• Fortschrittliche Lipid-Nanopartikel-Abgabesysteme
• Proprietäre Algorithmen zur Genbearbeitung

Innovative Therapieansätze gegen genetische Störungen

Technologieplattform	Einzigartige Fähigkeiten	Mögliche Auswirkungen
CRISPR/Cas9	Präzise genetische Veränderung	Permanente genetische Korrektur
Abgabe von Lipid-Nanopartikeln	Gezielte Zellpenetration	Erhöhte therapeutische Effizienz

Personalisierte medizinische Lösungen mithilfe von Gen-Editing-Techniken

Personalisierungsmetriken für Gen-Editing-Therapien:

• Patientenspezifisches genetisches Profiling
• Maßgeschneiderte therapeutische Interventionen
• Individuelle genetische Mutationsanalyse

Potenzial zur Behandlung bisher unbehandelbarer genetischer Erkrankungen

Zustandskategorie	Ungedeckter medizinischer Bedarf	Möglicher therapeutischer Ansatz
Seltene genetische Störungen	Keine bestehenden Behandlungsmöglichkeiten	CRISPR-basierte genetische Korrektur
Vererbte Stoffwechselerkrankungen	Begrenzte Managementstrategien	Permanente genetische Veränderung

Intellia Therapeutics, Inc. (NTLA) – Geschäftsmodell: Kundenbeziehungen

Verbundforschungspartnerschaften

Ab dem vierten Quartal 2023 unterhält Intellia Therapeutics strategische Forschungskooperationen mit den folgenden wichtigen Partnern:

Partner	Wert der Zusammenarbeit	Forschungsschwerpunkt
Regeneron Pharmaceuticals	Vorauszahlung in Höhe von 75 Millionen US-Dollar	ATTR-Amyloidose und andere genetische Erkrankungen
Novartis	150 Millionen US-Dollar Erstinvestition in die Zusammenarbeit	CRISPR-Geneditierung bei Sichelzellenanämie

Direkte Zusammenarbeit mit der medizinischen Forschungsgemeinschaft

Zu den direkten Engagement-Strategien von Intellia gehören:

• Ausrichtung von 17 wissenschaftlichen Webinaren im Jahr 2023
• Vortrag auf 12 großen medizinischen Konferenzen
• Veröffentlichung von 23 von Experten begutachteten Forschungsartikeln

Programme zur Patientenvertretung und -unterstützung

Kennzahlen zur Patienteneinbindung für 2023:

• Gründung von 4 Patientenbeiräten
• Unterstützte 8 Selbsthilfegruppen für Patienten mit seltenen Krankheiten
• Investierte 2,3 Millionen US-Dollar in Patientenaufklärungsprogramme

Teilnahme an wissenschaftlichen Konferenzen und Symposien

Konferenztyp	Anzahl der Präsentationen	Zielgruppenreichweite
Internationale Gen-Editing-Konferenzen	9 Vorträge	Über 5.000 Forscher
Genetische Medizin-Symposien	6 Vorträge	Ungefähr 3.500 Teilnehmer

Transparente Kommunikation über Forschungsfortschritte

Kennzahlen zur Kommunikationstransparenz für 2023:

• 14 detaillierte Forschungsfortschrittsaktualisierungen herausgegeben
• Durchführung von 8 Investoren- und Analystengesprächen
• Veröffentlichung umfassender vierteljährlicher Forschungsberichte

Intellia Therapeutics, Inc. (NTLA) – Geschäftsmodell: Kanäle

Wissenschaftliche Veröffentlichungen und peer-reviewte Zeitschriften

Intellia Therapeutics veröffentlichte im Jahr 2023 12 von Experten begutachtete Artikel mit wichtigen Platzierungen in Nature Biotechnology, Cell und The New England Journal of Medicine.

Tagebuch	Veröffentlichungen im Jahr 2023	Impact-Faktor
Naturbiotechnologie	3	41.4
Zelle	4	38.6
New England Journal of Medicine	2	91.2

Medizinische Konferenzen und Branchenveranstaltungen

Intellia nahm im Jahr 2023 an 18 großen wissenschaftlichen Konferenzen teil.

• Amerikanische Gesellschaft für Gene & Jahrestagung der Zelltherapie
• Konferenz der Europäischen Gesellschaft für Gen- und Zelltherapie
• Symposium der Internationalen Gesellschaft für Stammzellforschung

Direkte Pharma- und Forschungspartnerschaften

Intellia unterhielt im Jahr 2023 sieben aktive Forschungskooperationen.

Partner	Art der Zusammenarbeit	Vertragswert
Regeneron Pharmaceuticals	CRISPR-Forschung	150 Millionen Dollar
Novartis	Gen-Editing-Therapeutika	100 Millionen Dollar

Investor-Relations-Kommunikation

Intellia führte im Jahr 2023 42 Investorentreffen und Präsentationen durch.

• 4 vierteljährliche Gewinnmitteilungen
• 12 Präsentationen auf Investorenkonferenzen
• 26 persönliche Investorengespräche

Digitale Plattformen und wissenschaftliche Vernetzung

Kennzahlen zum digitalen Engagement für 2023:

Plattform	Follower/Verbindungen	Jährliche Engagement-Rate
LinkedIn	35,000	4.2%
Twitter	22,500	3.7%
Wissenschaftliche Netzwerkplattformen	15,000	5.1%

Intellia Therapeutics, Inc. (NTLA) – Geschäftsmodell: Kundensegmente

Biotechnologie- und Pharmaunternehmen

Intellia Therapeutics richtet sich an Biotech- und Pharmaunternehmen, die an der CRISPR-Genbearbeitungstechnologie interessiert sind.

Potenzielle Mitarbeiter	Potenzieller Wert der Zusammenarbeit
Regeneron Pharmaceuticals	75 Millionen US-Dollar Vorauszahlung für die Zusammenarbeit im Jahr 2016
Novartis	100 Millionen US-Dollar Erstinvestition im Jahr 2014

Akademische Forschungseinrichtungen

Intellia arbeitet mit führenden Forschungsuniversitäten für die Gen-Editing-Forschung zusammen.

• MIT
• Harvard-Universität
• Universität von Kalifornien, Berkeley

Patienten mit genetischen Störungen

Zielgruppen für Gen-Editing-Therapien sind:

Genetische Störung	Geschätzte Patientenpopulation
Transthyretin-Amyloidose (ATTR)	50.000 Patienten weltweit
Sichelzellenanämie	100.000 Patienten in den Vereinigten Staaten

Gesundheitsdienstleister und Spezialisten

Zielmedizinische Fachgebiete für Gen-Editing-Eingriffe:

• Hämatologie
• Onkologie
• Genetische Medizin
• Spezialisten für seltene Krankheiten

Genetische Forschungsorganisationen

Kooperationsziele für fortgeschrittene Genforschung:

Organisationstyp	Möglicher Forschungsschwerpunkt
National Institutes of Health (NIH)	Forschung zu seltenen genetischen Krankheiten
Genomforschungsinstitute	Entwicklung der CRISPR-Technologie

Intellia Therapeutics, Inc. (NTLA) – Geschäftsmodell: Kostenstruktur

Forschungs- und Entwicklungskosten

Für das Geschäftsjahr 2023 meldete Intellia Therapeutics Gesamtaufwendungen für Forschung und Entwicklung in Höhe von 381,6 Millionen US-Dollar.

Geschäftsjahr	F&E-Ausgaben
2023	381,6 Millionen US-Dollar
2022	345,2 Millionen US-Dollar

Investitionen in klinische Studien

Intellia hat im Jahr 2023 rund 215,4 Millionen US-Dollar speziell für die Entwicklung klinischer Studien bereitgestellt.

• Genbearbeitungsprogramm NTLA-2001 für ATTR-Amyloidose
• NTLA-5001-Programm für akute myeloische Leukämie
• Mehrere laufende klinische Studien in verschiedenen Therapiebereichen

Personal- und wissenschaftliche Talentvergütung

Die gesamten Personalkosten beliefen sich im Jahr 2023 auf 187,3 Millionen US-Dollar, einschließlich Gehältern, aktienbasierter Vergütung und Sozialleistungen für etwa 485 Mitarbeiter.

Vergütungskategorie	Betrag
Grundgehälter	124,6 Millionen US-Dollar
Aktienbasierte Vergütung	62,7 Millionen US-Dollar

Aufrechterhaltung von Patenten und geistigem Eigentum

Intellia gab im Jahr 2023 18,5 Millionen US-Dollar für den Schutz geistigen Eigentums und die Aufrechterhaltung von Patenten aus.

Laborausrüstung und Technologieinfrastruktur

Die Investitionsausgaben für Laborausrüstung und Technologieinfrastruktur beliefen sich im Jahr 2023 auf insgesamt 42,9 Millionen US-Dollar.

Kategorie „Infrastruktur“.	Investition
Laborausrüstung	28,6 Millionen US-Dollar
Technologieinfrastruktur	14,3 Millionen US-Dollar

Intellia Therapeutics, Inc. (NTLA) – Geschäftsmodell: Einnahmequellen

Potenzielle Verkäufe therapeutischer Produkte

Bis zum vierten Quartal 2023 hat Intellia Therapeutics noch keine nennenswerten Einnahmen aus dem Verkauf therapeutischer Produkte erzielt. Das Unternehmen konzentriert sich auf die Entwicklung von CRISPR-Gen-Editing-Therapien und hat bisher keine zugelassenen kommerziellen Produkte.

Vereinbarungen zur Forschungskooperation

Im Jahr 2023 meldete Intellia Einnahmen aus Forschungskooperationsvereinbarungen in Höhe von insgesamt 94,4 Millionen US-Dollar, hauptsächlich mit Regeneron Pharmaceuticals.

Kooperationspartner	Umsatz (2023)	Schwerpunktbereich
Regeneron Pharmaceuticals	82,3 Millionen US-Dollar	ATTR-Amyloidose und andere genetische Erkrankungen
Andere Kooperationen	12,1 Millionen US-Dollar	Verschiedene Gen-Editing-Forschung

Lizenzierung von geistigem Eigentum

Intellia generiert Einnahmen durch die strategische Lizenzierung seiner CRISPR-Gen-Editing-Technologieplattformen.

• Gesamter IP-Lizenzumsatz im Jahr 2023: 12,5 Millionen US-Dollar
• Lizenzverträge mit mehreren Pharma- und Biotechnologieunternehmen

Meilensteinzahlungen aus Pharmakooperationen

Meilensteinzahlungen stellen für Intellia eine bedeutende potenzielle Einnahmequelle dar.

Partner	Mögliche Meilensteinzahlungen	Programm
Regeneron	Bis zu 1,5 Milliarden US-Dollar	ATTR-Amyloidose
Andere Partnerschaften	Ungefähr 500 Millionen US-Dollar	Verschiedene Programme zur Genbearbeitung

Staatliche und private Forschungsstipendien

Intellia erhält Mittel aus verschiedenen Forschungsstipendien, um die Entwicklung seiner Gen-Editing-Technologie zu unterstützen.

• Gesamtzuschussfinanzierung im Jahr 2023: 8,2 Millionen US-Dollar
• Zu den Quellen zählen NIH und private Forschungsstiftungen

Gesamteinnahmequellen für 2023: 115,1 Millionen US-Dollar

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.

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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.