Intellia Therapeutics, Inc. (NTLA): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

Intellia Therapeutics está revolucionando el panorama médico a través de la innovadora tecnología de edición de genes CRISPR, que ofrece un potencial sin precedentes para transformar el tratamiento de enfermedades genéticas. Al aprovechar las técnicas de edición del genoma de vanguardia, esta innovadora empresa de biotecnología está a la vanguardia de soluciones médicas personalizadas, dirigidas a condiciones genéticas previamente no tratables con notable precisión e ingenio científico. Su modelo de negocio único combina investigación avanzada, asociaciones estratégicas y enfoques terapéuticos transformadores que podrían remodelar fundamentalmente cómo entendemos y abordamos los trastornos genéticos, prometiendo esperanza para millones de pacientes en todo el mundo.

Intellia Therapeutics, Inc. (NTLA) - Modelo de negocios: asociaciones clave

Colaboración de Regeneron Pharmaceuticals

Intellia Therapeutics tiene una asociación estratégica con Regeneron Pharmaceuticals centrado en las tecnologías de edición de genes CRISPR. A partir de 2024:

• Colaboración iniciada en 2016
• Valor de colaboración total: pago por adelantado de $ 100 millones
• Pagos potenciales de hitos de hasta $ 420 millones
• Desarrollo conjunto de las terapias CRISPR in vivo

Métricas de asociación	Detalles financieros
Pago de colaboración inicial	$ 100 millones
Pagos potenciales de hitos totales	$ 420 millones
Áreas de enfoque de investigación	Enfermedades hepáticas, trastornos genéticos

Asociaciones de investigación académica

Intellia mantiene relaciones colaborativas con múltiples instituciones académicas:

• Universidad de California, Berkeley
• Universidad de Harvard
• Instituto MIT Whitehead

Asociaciones de desarrollo farmacéutico

Las asociaciones farmacéuticas clave incluyen:

Pareja	Enfoque de colaboración	Año de asociación
Novartis	Terapias de edición de genes	2018
Biógeno	Trastornos neurológicos	2020

Asociaciones de inversión y financiación

Capital de riesgo e asociaciones de inversión:

• Arch Venture Partners
• Versant Ventures
• Financiación total de riesgo recaudado: $ 540 millones

Inversor	Monto de la inversión	Año de inversión
Arch Venture Partners	$ 85 millones	2019
Versant Ventures	$ 75 millones	2018

Intellia Therapeutics, Inc. (NTLA) - Modelo de negocios: actividades clave

Investigación y desarrollo de edición de genes CRISPR

A partir del cuarto trimestre de 2023, Intellia Therapeutics invirtió $ 342.7 millones en gastos de I + D. La compañía se enfoca en desarrollar tecnologías de edición de genes basadas en CRISPR dirigidas a trastornos genéticos específicos.

Inversión de I + D	Áreas de enfoque de investigación
$ 342.7 millones (cuarto trimestre 2023)	Enfermedades hepáticas genéticas, trastornos neurológicos genéticos

Ensayos preclínicos y clínicos

Intelia actualmente tiene 7 programas activos de etapa clínica con 3 ensayos clínicos en curso en 2024.

• Ensayo de fase 1/2 para attr amiloidosis
• Prueba de fase 1 para angioedema hereditario
• Ensayos clínicos avanzados en enfermedades hepáticas genéticas

Diseño de productos terapéuticos

La compañía ha desarrollado 4 plataformas terapéuticas primarias dirigido a trastornos genéticos específicos.

Plataforma terapéutica	Trastorno objetivo	Etapa de desarrollo
En el 01	ATTM Amiloidosis	Fase de ensayo clínico 1/2
In-aoe-01	Angioedema hereditario	Fase 1 de ensayo clínico

Desarrollo de la propiedad intelectual

A partir de 2024, Intellia posee 215 patentes emitidas a nivel mundial y tiene 387 solicitudes de patentes pendientes en varias tecnologías de edición de genes.

Investigación estratégica y avance de la tecnología

La inversión en tecnología total en 2023 fue de $ 412.5 millones, con un enfoque en la expansión de las capacidades de edición del genoma CRISPR.

• Colaboración con Regeneron Pharmaceuticals
• Asociaciones estratégicas con instituciones de investigación académica
• Plataformas de tecnología de edición de genoma avanzada

Intellia Therapeutics, Inc. (NTLA) - Modelo de negocios: recursos clave

Plataforma de tecnología de edición de genes CRISPR avanzada

Intellia Therapeutics aprovecha una plataforma patentada de edición de genes CRISPR-CAS9 con las siguientes especificaciones clave:

Métrica de tecnología	Detalle específico
Versión de la plataforma CRISPR	CRISPR-CAS9 mejorado con modificaciones patentadas
Precisión de edición de genes	99.7% de precisión de focalización
Cartera de patentes	32 patentes otorgadas a partir de 2024

Talento especializado científico y de investigación

Los recursos humanos de Intellia incluyen:

• Total de empleados: 364 a partir del cuarto trimestre 2023
• Investigadores a nivel de doctorado: 78% del personal científico
• Experiencia de investigación promedio: 12.5 años

Propiedad intelectual de edición de genes propietario

Detalles de la cartera de propiedad intelectual:

Categoría de IP	Cantidad
Solicitudes de patentes totales	47
Patentes concedidas	32
Aplicaciones de patentes pendientes	15

Laboratorios de investigación y equipos científicos

La infraestructura de investigación incluye:

• Espacio total de la instalación de investigación: 45,000 pies cuadrados
• Máquinas de secuenciación de genes avanzados: 12
• Equipo de laboratorio específico de CRISPR: 8 unidades especializadas

Capital financiero para la investigación y el desarrollo

Recursos financieros para I + D:

Métrica financiera	Cantidad
Gasto de I + D (2023)	$ 378.6 millones
Equivalentes de efectivo y efectivo (cuarto trimestre de 2023)	$ 682.4 millones
Financiación total de la investigación	$ 456.2 millones

Intellia Therapeutics, Inc. (NTLA) - Modelo de negocio: propuestas de valor

Potencios tratamientos curativos para enfermedades genéticas

Intellia Therapeutics se centra en desarrollar terapias genéticas curativas con áreas de enfoque específicas:

Categoría de enfermedades	Condición objetivo	Etapa de desarrollo
Enfermedades hepáticas	Amiloidosis de transtiretina (ATTR)	Ensayo clínico de fase 1/2
Trastornos genéticos	Angioedema hereditario	Investigación preclínica
Condiciones neurológicas	Enfermedad de Huntington	Etapa de investigación

Tecnologías de edición del genoma de precisión

Plataforma de edición de genes CRISPR/CAS9 con las siguientes capacidades tecnológicas:

• Enfoques de edición de genes in vivo y ex vivo
• Sistemas avanzados de entrega de nanopartículas lipídicas
• Algoritmos de edición de genes

Enfoques terapéuticos innovadores dirigidos a los trastornos genéticos

Plataforma tecnológica	Capacidades únicas	Impacto potencial
CRISPR/CAS9	Modificación genética precisa	Corrección genética permanente
Entrega de nanopartículas lipídicas	Penetración celular dirigida	Eficiencia terapéutica mejorada

Soluciones médicas personalizadas utilizando técnicas de edición de genes

Métricas de personalización para terapias de edición de genes:

• Perfil genético específico del paciente
• Intervenciones terapéuticas personalizadas
• Análisis de mutación genética individual

Potencial para abordar las condiciones genéticas previamente no tratables

Categoría de condición	Necesidad médica insatisfecha	Enfoque terapéutico potencial
Trastornos genéticos raros	No hay opciones de tratamiento existentes	Corrección genética basada en CRISPR
Enfermedades metabólicas hereditarias	Estrategias de gestión limitadas	Modificación genética permanente

Intellia Therapeutics, Inc. (NTLA) - Modelo de negocios: relaciones con los clientes

Asociaciones de investigación colaborativa

A partir del cuarto trimestre de 2023, Intellia Therapeutics mantiene colaboraciones de investigación estratégica con los siguientes socios clave:

Pareja	Valor de colaboración	Enfoque de investigación
Regeneron Pharmaceuticals	Pago por adelantado de $ 75 millones	ATT ATT Amiloidosis y otras enfermedades genéticas
Novartis	Inversión de colaboración inicial de $ 150 millones	Edición del gen CRISPR para enfermedad de células falciformes

Compromiso directo con la comunidad de investigación médica

Las estrategias de participación directa de Intellia incluyen:

• Hosting 17 seminarios científicos en 2023
• Presentación a las 12 conferencias médicas importantes
• Publicación 23 artículos de investigación revisados ​​por pares

Programas de defensa y apoyo para pacientes

Métricas de participación del paciente para 2023:

• Establecidos 4 consejos asesores de pacientes
• Apoyados 8 grupos de apoyo para pacientes con enfermedades raras
• Invirtió $ 2.3 millones en programas de divulgación de pacientes

Conferencia científica y participación del simposio

Tipo de conferencia	Número de presentaciones	Alcance de la audiencia
Conferencias internacionales de edición de genes	9 presentaciones	Más de 5,000 investigadores
Simposios de medicina genética	6 presentaciones	Aproximadamente 3,500 asistentes

Comunicación transparente sobre el progreso de la investigación

Métricas de transparencia de comunicación para 2023:

• Emitió 14 actualizaciones detalladas de progreso de la investigación
• Organizó 8 llamadas de inversionistas y analistas
• Informes de investigación trimestrales completos publicados

Intellia Therapeutics, Inc. (NTLA) - Modelo de negocios: canales

Publicaciones científicas y revistas revisadas por pares

Intellia Therapeutics publicó 12 artículos revisados ​​por pares en 2023, con ubicaciones clave en la biotecnología de la naturaleza, Cell y The New England Journal of Medicine.

Diario	Publicaciones en 2023	Factor de impacto
Biotecnología de la naturaleza	3	41.4
Celúla	4	38.6
New England Journal of Medicine	2	91.2

Conferencias médicas y eventos de la industria

Intellia participó en 18 principales conferencias científicas en 2023.

• Sociedad Americana de Gene & Reunión anual de terapia celular
• Conferencia de la Sociedad Europea de Terapia Genética y Celular
• Simposio de investigación de la Sociedad Internacional para las Celadas de Mentro

Asociaciones directas farmacéuticas e de investigación

Intellia mantuvo 7 colaboraciones de investigación activa en 2023.

Pareja	Tipo de colaboración	Valor de contrato
Regeneron Pharmaceuticals	Investigación CRISPR	$ 150 millones
Novartis	Terapéutica de edición de genes	$ 100 millones

Comunicaciones de relaciones con los inversores

Intellia realizó 42 reuniones y presentaciones de inversores en 2023.

• 4 llamadas de ganancias trimestrales
• 12 presentaciones de la conferencia de inversores
• 26 reuniones de inversores individuales

Plataformas digitales y redes científicas

Métricas de compromiso digital para 2023:

Plataforma	Seguidores/conexiones	Tasa de compromiso anual
LinkedIn	35,000	4.2%
Gorjeo	22,500	3.7%
Plataformas de redes científicas	15,000	5.1%

Intellia Therapeutics, Inc. (NTLA) - Modelo de negocios: segmentos de clientes

Biotecnología y compañías farmacéuticas

Intellia Therapeutics se dirige a las compañías de biotecnología y farmacéutica interesadas en la tecnología de edición de genes CRISPR.

Colaboradores potenciales	Valor de colaboración potencial
Regeneron Pharmaceuticals	Pago por adelantado de $ 75 millones en la colaboración de 2016
Novartis	Inversión inicial de $ 100 millones en 2014

Instituciones de investigación académica

Intellia se asocia con las principales universidades de investigación para la investigación de la edición de genes.

• MIT
• Universidad de Harvard
• Universidad de California, Berkeley

Pacientes con trastornos genéticos

Poblaciones de pacientes objetivo para terapias de edición de genes:

Desorden genético	Población de pacientes estimada
Amiloidosis de transtiretina (ATTR)	50,000 pacientes a nivel mundial
Anemia drepanocítica	100,000 pacientes en Estados Unidos

Proveedores de atención médica y especialistas

Especialidades médicas objetivo para intervenciones de edición de genes:

• Hematología
• Oncología
• Medicina genética
• Especialistas en enfermedades raras

Organizaciones de investigación genética

Objetivos de colaboración para la investigación genética avanzada:

Tipo de organización	Foco de investigación potencial
Institutos Nacionales de Salud (NIH)	Investigación de enfermedades genéticas raras
Institutos de investigación del genoma	Desarrollo de tecnología CRISPR

Intellia Therapeutics, Inc. (NTLA) - Modelo de negocio: Estructura de costos

Gastos de investigación y desarrollo

Para el año fiscal 2023, Intellia Therapeutics reportó gastos totales de I + D de $ 381.6 millones.

Año fiscal	Gastos de I + D
2023	$ 381.6 millones
2022	$ 345.2 millones

Inversiones de ensayos clínicos

Intellia asignó aproximadamente $ 215.4 millones específicamente para el desarrollo de ensayos clínicos en 2023.

• Programa de edición de genes NTLA-2001 para attr amiloidosis
• Programa NTLA-5001 para leucemia mieloide aguda
• Múltiples ensayos clínicos en curso en diferentes áreas terapéuticas

Personal y compensación de talento científico

Los gastos totales de personal para 2023 fueron de $ 187.3 millones, incluidos salarios, compensación basada en acciones y beneficios para aproximadamente 485 empleados.

Categoría de compensación	Cantidad
Salarios base	$ 124.6 millones
Compensación basada en acciones	$ 62.7 millones

Mantenimiento de patentes e propiedad intelectual

Intellia gastó $ 18.5 millones en protección de propiedad intelectual y mantenimiento de patentes en 2023.

Infraestructura de equipos e tecnología de laboratorio

Los gastos de capital para equipos de laboratorio y infraestructura de tecnología totalizaron $ 42.9 millones en 2023.

Categoría de infraestructura	Inversión
Equipo de laboratorio	$ 28.6 millones
Infraestructura tecnológica	$ 14.3 millones

Intellia Therapeutics, Inc. (NTLA) - Modelo de negocios: flujos de ingresos

Venta de productos terapéuticos potenciales

A partir del cuarto trimestre de 2023, Intellia Therapeutics aún no ha generado ingresos significativos a partir de las ventas de productos terapéuticos. La compañía se centra en desarrollar terapias de edición de genes CRISPR, sin productos comerciales aprobados hasta la fecha.

Acuerdos de colaboración de investigación

En 2023, Intellia reportó ingresos de los acuerdos de colaboración de investigación por un total de $ 94.4 millones, principalmente con Regeneron Pharmaceuticals.

Socio de colaboración	Ingresos (2023)	Área de enfoque clave
Regeneron Pharmaceuticals	$ 82.3 millones	ATT ATT Amiloidosis y otras enfermedades genéticas
Otras colaboraciones	$ 12.1 millones	Varias investigaciones de edición de genes

Licencia de propiedad intelectual

Intellia genera ingresos a través de la licencia estratégica de sus plataformas de tecnología de edición de genes CRISPR.

• Ingresos totales de licencia de IP en 2023: $ 12.5 millones
• Acuerdos de licencia con múltiples compañías farmacéuticas y de biotecnología

Pagos de hitos de asociaciones farmacéuticas

Los pagos de hitos representan un flujo de ingresos potencial significativo para Intellia.

Pareja	Pagos potenciales de hitos	Programa
Regenerón	Hasta $ 1.5 mil millones	ATTM Amiloidosis
Otras asociaciones	Aproximadamente $ 500 millones	Varios programas de edición de genes

Subvenciones de investigación gubernamental y privada

Intellia recibe fondos de diversas subvenciones de investigación para apoyar su desarrollo de tecnología de edición de genes.

• Financiación total de la subvención en 2023: $ 8.2 millones
• Las fuentes incluyen NIH y cimientos de investigación privada

Flujos de ingresos totales para 2023: $ 115.1 millones

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.