Intellia Therapeutics, Inc. (NTLA): Business Model Canvas [Jan-2025 Mis à jour]

Intellia Therapeutics révolutionne le paysage médical grâce à une technologie d'édition de gènes CRISPR révolutionnaire, offrant un potentiel sans précédent pour transformer le traitement des maladies génétiques. En tirant parti des techniques de montage du génome de pointe, cette entreprise de biotechnologie innovante est à l'avant-garde des solutions médicales personnalisées, ciblant des conditions génétiques auparavant non traitables avec une précision remarquable et une ingéniosité scientifique. Leur modèle commercial unique combine des recherches avancées, des partenariats stratégiques et des approches thérapeutiques transformatrices qui pourraient fondamentalement remodeler la façon dont nous comprenons et abordons les troubles génétiques, promettant de l'espoir de millions de patients dans le monde.

Intellia Therapeutics, Inc. (NTLA) - Modèle commercial: partenariats clés

Regeneron Pharmaceuticals Collaboration

Intellia Therapeutics a un partenariat stratégique avec Regeneron Pharmaceuticals axé sur les technologies d'édition des gènes CRISPR. En 2024:

• Collaboration initiée en 2016
• Valeur de collaboration totale: 100 millions de dollars de paiement initial
• Paiements de jalons potentiels jusqu'à 420 millions de dollars
• Développement conjoint des thérapies CRISPR in vivo

Métriques de partenariat	Détails financiers
Paiement de collaboration initiale	100 millions de dollars
Paiements potentiels de jalon total	420 millions de dollars
Les domaines de recherche sur la recherche	Maladies hépatiques, troubles génétiques

Partenariats de recherche universitaire

Intellia entretient des relations collaboratives avec plusieurs établissements universitaires:

• Université de Californie, Berkeley
• Université de Harvard
• Institut du MIT Whitehead

Partenariats de développement pharmaceutique

Les partenariats pharmaceutiques clés comprennent:

Partenaire	Focus de la collaboration	Année de partenariat
Novartis	Thérapies de montage de gènes	2018
Biogène	Troubles neurologiques	2020

Partenariats d'investissement et de financement

Capital de capital-risque et partenariats d'investissement:

• Arch Venture Partners
• Versant Ventures
• Financement total de capital-risque levé: 540 millions de dollars

Investisseur	Montant d'investissement	Année d'investissement
Arch Venture Partners	85 millions de dollars	2019
Versant Ventures	75 millions de dollars	2018

Intellia Therapeutics, Inc. (NTLA) - Modèle d'entreprise: Activités clés

CRISPR Gene-iting Research and Development

Au quatrième trimestre 2023, Intellia Therapeutics a investi 342,7 millions de dollars dans les dépenses de R&D. L'entreprise se concentre sur le développement de technologies d'édition génétique basées sur CRISPR ciblant des troubles génétiques spécifiques.

Investissement en R&D	Les domaines de recherche sur la recherche
342,7 millions de dollars (Q4 2023)	Maladies hépatiques génétiques, troubles neurologiques génétiques

Essais précliniques et cliniques

Intellia possède actuellement 7 programmes de stade clinique actifs avec 3 essais cliniques en cours en 2024.

• Essai de phase 1/2 pour l'amylose de l'atte
• Essai de phase 1 pour œdème héréditaire de l'angio
• Essais cliniques avancés dans les maladies du foie génétiques

Conception de produits thérapeutiques

L'entreprise a développé 4 plates-formes thérapeutiques primaires ciblant des troubles génétiques spécifiques.

Plate-forme thérapeutique	Trouble cible	Étape de développement
IN-AT-01	Attirner l'amylose	Essai clinique Phase 1/2
IN-AOE-01	Œdème héréditaire de l'angio	Essai clinique phase 1

Développement de la propriété intellectuelle

En 2024, Intellia détient 215 brevets émis à l'échelle mondiale et possède 387 demandes de brevet en attente dans diverses technologies d'édition génique.

Recherche stratégique et progrès technologique

L'investissement total technologique en 2023 était de 412,5 millions de dollars, en mettant l'accent sur l'élargissement des capacités d'édition du génome CRISPR.

• Collaboration avec Regeneron Pharmaceuticals
• Partenariats stratégiques avec les établissements de recherche universitaires
• Plateformes de technologie de modification du génome avancé

Intellia Therapeutics, Inc. (NTLA) - Modèle commercial: Ressources clés

Plateforme de technologie d'édition de gènes CRISPR avancée

Intellia Therapeutics exploite une plate-forme de réédition de gènes CRISPR-CAS9 propriétaire avec les spécifications clés suivantes:

Métrique technologique	Détail spécifique
Version de la plate-forme CRISPR	CRISPR-CAS9 amélioré avec des modifications propriétaires
Précision d'édition de gènes	99,7% de précision de ciblage
Portefeuille de brevets	32 Brevets accordés à partir de 2024

Talent scientifique et de recherche spécialisés

Les ressources humaines d'Intellia comprennent:

• Total des employés: 364 au quatrième trimestre 2023
• Rechercheurs de niveau doctoral: 78% du personnel scientifique
• Expérience de recherche moyenne: 12,5 ans

Propriété intellectuelle de l'édition génétique propriétaire

Détails du portefeuille de propriété intellectuelle:

Catégorie IP	Quantité
Demandes totales de brevets	47
Brevets accordés	32
Demandes de brevet en instance	15

Laboratoires de recherche et équipement scientifique

L'infrastructure de recherche comprend:

• Espace total des installations de recherche: 45 000 pieds carrés
• Machines de séquençage des gènes avancés: 12
• Équipement de laboratoire spécifique à CRISPR: 8 unités spécialisées

Capital financier pour la recherche et le développement

Ressources financières pour la R&D:

Métrique financière	Montant
Dépenses de R&D (2023)	378,6 millions de dollars
Cash and Cash équivalents (T4 2023)	682,4 millions de dollars
Financement total de la recherche	456,2 millions de dollars

Intellia Therapeutics, Inc. (NTLA) - Modèle d'entreprise: propositions de valeur

Traitements curatifs potentiels pour les maladies génétiques

Intellia Therapeutics se concentre sur le développement de thérapies génétiques curatives avec des domaines d'intervention spécifiques:

Catégorie de maladie	Conditions cibles	Étape de développement
Maladies du foie	Amylose de la transthyrétine (ATR)	Essai clinique de phase 1/2
Troubles génétiques	Œdème héréditaire de l'angio	Recherche préclinique
Conditions neurologiques	La maladie de Huntington	Étape d'enquête

Technologies d'édition de génome de précision

Plateforme d'édition de gènes CRISPR / CAS9 propriétaire avec des capacités technologiques suivantes:

• Approches d'édition de gènes in vivo et ex vivo
• Systèmes de livraison avancés de nanoparticules lipidiques
• Algorithmes de rédaction de gènes propriétaires

Approches thérapeutiques innovantes ciblant les troubles génétiques

Plate-forme technologique	Capacités uniques	Impact potentiel
CRISPR / CAS9	Modification génétique précise	Correction génétique permanente
Livraison des nanoparticules lipidiques	Pénétration cellulaire ciblée	Efficacité thérapeutique améliorée

Solutions médicales personnalisées utilisant des techniques d'édition de gènes

Métriques de personnalisation pour les thérapies d'édition génétique:

• Profilage génétique spécifique au patient
• Interventions thérapeutiques personnalisées
• Analyse de mutation génétique individuelle

Potentiel pour résoudre les conditions génétiques auparavant inextraitables

Catégorie d'état	Besoin médical non satisfait	Approche thérapeutique potentielle
Troubles génétiques rares	Aucune option de traitement existante	Correction génétique basée sur CRISPR
Maladies métaboliques héritées	Stratégies de gestion limitées	Modification génétique permanente

Intellia Therapeutics, Inc. (NTLA) - Modèle d'entreprise: relations clients

Partenariats de recherche collaborative

Depuis le quatrième trimestre 2023, Intellia Therapeutics maintient des collaborations de recherche stratégique avec les partenaires clés suivants:

Partenaire	Valeur de collaboration	Focus de recherche
Regeneron Pharmaceuticals	75 millions de dollars de paiement initial	Attirne amylose et autres maladies génétiques
Novartis	150 millions de dollars d'investissement de collaboration initiale	Édition du gène CRISPR pour la drépanocytose

Engagement direct avec la communauté de la recherche médicale

Les stratégies d'engagement directes d'Intellia comprennent:

• Hébergeant 17 webinaires scientifiques en 2023
• Présentant 12 conférences médicales majeures
• Publication de 23 articles de recherche évalués par des pairs

Programmes de plaidoyer et de soutien aux patients

Métriques d'engagement des patients pour 2023:

• Établi 4 conseils consultatifs pour les patients
• Soutenu 8 groupes de soutien aux patients atteints de maladies rares
• Investi 2,3 millions de dollars dans des programmes de sensibilisation des patients

Conférence scientifique et participation au symposium

Type de conférence	Nombre de présentations	Poutenir
Conférences internationales d'édition de gènes	9 présentations	Plus de 5 000 chercheurs
Symposiums de médecine génétique	6 présentations	Environ 3 500 participants

Communication transparente sur les progrès de la recherche

Mesures de transparence de la communication pour 2023:

• Émis 14 mises à jour de progrès de recherche détaillées
• Hébergé 8 appels d'investisseur et analyste
• Publié des rapports de recherche trimestriels complets

Intellia Therapeutics, Inc. (NTLA) - Modèle d'entreprise: canaux

Publications scientifiques et revues à comité de lecture

Intellia Therapeutics a publié 12 articles évalués par des pairs en 2023, avec des placements clés dans la biotechnologie de la nature, la cellule et le New England Journal of Medicine.

Journal	Publications en 2023	Facteur d'impact
Biotechnologie de la nature	3	41.4
Cellule	4	38.6
Journal de médecine de la Nouvelle-Angleterre	2	91.2

Conférences médicales et événements de l'industrie

Intellia a participé à 18 conférences scientifiques majeures en 2023.

• Société américaine de gène & Réunion annuelle de thérapie cellulaire
• Conférence de la Société européenne de la thérapie génétique et cellulaire
• Symposium de recherche sur la Société internationale pour les cellules souches

Partenariats pharmaceutiques et de recherche directs

Intellia a maintenu 7 collaborations de recherche active en 2023.

Partenaire	Type de collaboration	Valeur du contrat
Regeneron Pharmaceuticals	CRISPR Research	150 millions de dollars
Novartis	Gene Édition de thérapies	100 millions de dollars

Communications des relations avec les investisseurs

Intellia a organisé 42 réunions et présentations d'investisseurs en 2023.

• 4 appels de résultats trimestriels
• 12 présentations de la conférence des investisseurs
• 26 réunions d'investisseurs individuels

Plates-formes numériques et réseautage scientifique

Métriques d'engagement numérique pour 2023:

Plate-forme	Abonnés / connexions	Taux d'engagement annuel
Liendin	35,000	4.2%
Gazouillement	22,500	3.7%
Plateformes de réseautage scientifique	15,000	5.1%

Intellia Therapeutics, Inc. (NTLA) - Modèle d'entreprise: segments de clientèle

Biotechnology et sociétés pharmaceutiques

Intellia Therapeutics cible les sociétés de biotechnologie et pharmaceutiques intéressées par la technologie d'édition de gènes CRISPR.

Collaborateurs potentiels	Valeur de collaboration potentielle
Regeneron Pharmaceuticals	75 millions de dollars de paiement initial en 2016 Collaboration
Novartis	100 millions de dollars d'investissement initial en 2014

Établissements de recherche universitaire

Intellia s'associe aux principales universités de recherche pour la recherche sur l'édition de gènes.

• Mit
• Université de Harvard
• Université de Californie, Berkeley

Patients souffrant de troubles génétiques

Populations de patients cibles pour les thérapies d'édition de gènes:

Trouble génétique	Population estimée des patients
Amylose de la transthyrétine (ATR)	50 000 patients dans le monde
Drépanocytose	100 000 patients aux États-Unis

Fournisseurs de soins de santé et spécialistes

Cible des spécialités médicales pour les interventions d'édition de gènes:

• Hématologie
• Oncologie
• Médecine génétique
• Spécialistes de maladies rares

Organisations de recherche génétique

Cibles de collaboration pour la recherche génétique avancée:

Type d'organisation	Focus de recherche potentielle
National Institutes of Health (NIH)	Recherche de maladies génétiques rares
Instituts de recherche du génome	Développement de la technologie CRISPR

Intellia Therapeutics, Inc. (NTLA) - Modèle d'entreprise: Structure des coûts

Frais de recherche et de développement

Pour l'exercice 2023, Intellia Therapeutics a déclaré des dépenses totales de R&D de 381,6 millions de dollars.

Exercice fiscal	Dépenses de R&D
2023	381,6 millions de dollars
2022	345,2 millions de dollars

Investissements d'essais cliniques

Intellia a alloué environ 215,4 millions de dollars spécifiquement pour le développement d'essais cliniques en 2023.

• Édition de gènes Programme NTLA-2001 pour l'amylose de l'atte
• Programme NTLA-5001 pour la leucémie myéloïde aiguë
• Plusieurs essais cliniques en cours dans différentes zones thérapeutiques

Personnel et compensation des talents scientifiques

Les dépenses totales du personnel pour 2023 étaient de 187,3 millions de dollars, y compris des salaires, une rémunération en actions et des avantages sociaux pour environ 485 employés.

Catégorie de compensation	Montant
Salaires de base	124,6 millions de dollars
Compensation en stock	62,7 millions de dollars

Entretien de la propriété brevet et intellectuelle

Intellia a dépensé 18,5 millions de dollars pour la protection de la propriété intellectuelle et l'entretien des brevets en 2023.

Équipement de laboratoire et infrastructure technologique

Les dépenses en capital pour les équipements de laboratoire et les infrastructures technologiques ont totalisé 42,9 millions de dollars en 2023.

Catégorie d'infrastructure	Investissement
Équipement de laboratoire	28,6 millions de dollars
Infrastructure technologique	14,3 millions de dollars

Intellia Therapeutics, Inc. (NTLA) - Modèle commercial: Strots de revenus

Ventes de produits thérapeutiques potentiels

Depuis le quatrième trimestre 2023, Intellia Therapeutics n'a pas encore généré des revenus importants à partir des ventes de produits thérapeutiques. L'entreprise se concentre sur le développement de thérapies d'édition des gènes CRISPR, sans produits commerciaux approuvés à ce jour.

Accords de collaboration de recherche

En 2023, Intellia a déclaré des revenus des accords de collaboration de recherche totalisant 94,4 millions de dollars, principalement avec Regeneron Pharmaceuticals.

Partenaire de collaboration	Revenus (2023)	Domaine d'intervention clé
Regeneron Pharmaceuticals	82,3 millions de dollars	Attirne amylose et autres maladies génétiques
Autres collaborations	12,1 millions de dollars	Diverses recherches d'édition de gènes

Licence de propriété intellectuelle

Intellia génère des revenus grâce à des licences stratégiques de ses plateformes de technologie CRISPR Gene-Modise.

• Revenu total des licences IP en 2023: 12,5 millions de dollars
• Accords de licence avec plusieurs sociétés pharmaceutiques et biotechnologiques

Payments d'étape provenant des partenariats pharmaceutiques

Les paiements d'étape représentent une source de revenus potentiel importante pour Intellia.

Partenaire	Paiements de jalons potentiels	Programme
Regeneron	Jusqu'à 1,5 milliard de dollars	Attirner l'amylose
Autres partenariats	Environ 500 millions de dollars	Divers programmes d'édition de gènes

Subventions de recherche gouvernementales et privées

Intellia reçoit des financements de diverses subventions de recherche pour soutenir son développement de technologies d'édition génétique.

• Financement total des subventions en 2023: 8,2 millions de dollars
• Les sources incluent les fondations de la recherche des NIH et privées

Total des sources de revenus pour 2023: 115,1 millions de dollars

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.