Intellia Therapeutics, Inc. (NTLA): Modelo de Negócios Canvas [Jan-2025 Atualizado]

A Intellia Therapeutics está revolucionando o cenário médico por meio da inovadora tecnologia de edição de genes da CRISPR, oferecendo potencial sem precedentes para transformar o tratamento de doenças genéticas. Ao alavancar as técnicas de edição de genoma de ponta, esta empresa inovadora de biotecnologia está na vanguarda de soluções médicas personalizadas, visando condições genéticas anteriormente desprezíveis com precisão notável e ingenuidade científica. Seu modelo de negócios exclusivo combina pesquisas avançadas, parcerias estratégicas e abordagens terapêuticas transformadoras que podem remodelar fundamentalmente como entendemos e abordamos distúrbios genéticos, promissores de esperança para milhões de pacientes em todo o mundo.

Intellia Therapeutics, Inc. (NTLA) - Modelo de negócios: Parcerias -chave

Regeneron Pharmaceuticals Collaboration

A Intellia Therapeutics tem uma parceria estratégica com a Regeneron Pharmaceuticals focada nas tecnologias de edição de genes do CRISPR. A partir de 2024:

• Colaboração iniciada em 2016
• Valor total de colaboração: pagamento inicial de US $ 100 milhões
• Potenciais pagamentos marcantes de até US $ 420 milhões
• Desenvolvimento conjunto de terapias CRISPR in vivo

Métricas de parceria	Detalhes financeiros
Pagamento inicial de colaboração	US $ 100 milhões
Pagamentos totais potenciais de marco total	US $ 420 milhões
Áreas de foco de pesquisa	Doenças hepáticas, distúrbios genéticos

Parcerias de pesquisa acadêmica

Intellia mantém relações colaborativas com várias instituições acadêmicas:

• Universidade da Califórnia, Berkeley
• Universidade de Harvard
• MIT Whitehead Institute

Parcerias de desenvolvimento farmacêutico

As principais parcerias farmacêuticas incluem:

Parceiro	Foco de colaboração	Ano de parceria
Novartis	Terapias de edição de genes	2018
Biogênio	Distúrbios neurológicos	2020

Parcerias de investimento e financiamento

Capital de risco e parcerias de investimento:

• Arch Venture Partners
• Ventuos versantes
• Financiamento total do empreendimento arrecadado: US $ 540 milhões

Investidor	Valor do investimento	Ano de investimento
Arch Venture Partners	US $ 85 milhões	2019
Ventuos versantes	US $ 75 milhões	2018

Intellia Therapeutics, Inc. (NTLA) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento de edição de genes CRISPR

A partir do quarto trimestre de 2023, a Intellia Therapeutics investiu US $ 342,7 milhões em despesas de P&D. A empresa se concentra no desenvolvimento de tecnologias de edição de genes baseados em CRISPR direcionados a distúrbios genéticos específicos.

Investimento em P&D	Áreas de foco de pesquisa
US $ 342,7 milhões (quarto de 2023)	Doenças hepáticas genéticas, distúrbios neurológicos genéticos

Ensaios pré -clínicos e clínicos

Atualmente, a Intellia possui 7 programas ativos de estágio clínico com três ensaios clínicos em andamento em 2024.

• Estudo de fase 1/2 para attr amiloidose
• Estudo de fase 1 para angioedema hereditário
• Ensaios clínicos avançados em doenças genéticas de fígado

Design terapêutico do produto

A empresa desenvolveu 4 plataformas terapêuticas primárias direcionando distúrbios genéticos específicos.

Plataforma terapêutica	Transtorno alvo	Estágio de desenvolvimento
AT-01	ATRMILOSIDOS ATTR	Ensaio Clínico Fase 1/2
In-aoe-01	Angioedema hereditário	Fase 1 do ensaio clínico 1

Desenvolvimento da Propriedade Intelectual

A partir de 2024, a Intellia possui 215 patentes emitidas globalmente e possui 387 pedidos de patentes pendentes em várias tecnologias de edição de genes.

Pesquisa estratégica e avanço tecnológico

O investimento total em tecnologia em 2023 foi de US $ 412,5 milhões, com foco na expansão dos recursos de edição do genoma do CRISPR.

• Colaboração com Regeneron Pharmaceuticals
• Parcerias estratégicas com instituições de pesquisa acadêmica
• Plataformas de tecnologia de edição de genoma avançado

Intellia Therapeutics, Inc. (NTLA) - Modelo de negócios: Recursos -chave

Plataforma de tecnologia avançada de edição de genes CRISPR

A Intellia Therapeutics aproveita uma plataforma proprietária de edição de genes CRISPR-CAS9 com as seguintes especificações-chave:

Métrica de tecnologia	Detalhes específicos
Versão da plataforma CRISPR	CRISPR-CAS9 aprimorado com modificações proprietárias
Precisão de edição de genes	99,7% de precisão de direcionamento
Portfólio de patentes	32 Patentes concedidas a partir de 2024

Talento científico e de pesquisa especializado

Os recursos humanos da Intellia incluem:

• Total de funcionários: 364 A partir do quarto trimestre 2023
• Pesquisadores em nível de doutorado: 78% da equipe científica
• Experiência média de pesquisa: 12,5 anos

Propriedade intelectual de edição de genes proprietária

Detalhes da carteira de propriedade intelectual:

Categoria IP	Quantidade
Total de pedidos de patente	47
Patentes concedidas	32
Aplicações de patentes pendentes	15

Laboratórios de pesquisa e equipamentos científicos

A infraestrutura de pesquisa inclui:

• Espaço total da instalação de pesquisa: 45.000 pés quadrados
• Máquinas de sequenciamento de genes avançados: 12
• Equipamento de laboratório específico para CRISPR: 8 unidades especializadas

Capital financeiro para pesquisa e desenvolvimento

Recursos financeiros para P&D:

Métrica financeira	Quantia
Despesas de P&D (2023)	US $ 378,6 milhões
Caixa e equivalentes em dinheiro (Q4 2023)	US $ 682,4 milhões
Financiamento total da pesquisa	US $ 456,2 milhões

Intellia Therapeutics, Inc. (NTLA) - Modelo de negócios: proposições de valor

Tratamentos curativos em potencial para doenças genéticas

A Intellia Therapeutics se concentra no desenvolvimento de terapias genéticas curativas com áreas de foco específicas:

Categoria de doença	Condições alvo	Estágio de desenvolvimento
Doenças hepáticas	Amiloidose transtiretina (att)	Ensaio Clínico de Fase 1/2
Distúrbios genéticos	Angioedema hereditário	Pesquisa pré -clínica
Condições neurológicas	Doença de Huntington	Estágio de investigação

Tecnologias de edição de genoma de precisão

Plataforma proprietária de edição de genes CRISPR/CAS9 com os seguintes recursos tecnológicos:

• Abordagens de edição de genes in vivo e ex vivo
• Sistemas avançados de entrega de nanopartículas lipídicas
• Algoritmos proprietários de edição de genes

Abordagens terapêuticas inovadoras visando distúrbios genéticos

Plataforma de tecnologia	Recursos exclusivos	Impacto potencial
CRISPR/CAS9	Modificação genética precisa	Correção genética permanente
Entrega de nanopartículas lipídicas	Penetração celular direcionada	Eficiência terapêutica aprimorada

Soluções médicas personalizadas usando técnicas de edição de genes

Métricas de personalização para terapias de edição de genes:

• Perfil genético específico do paciente
• Intervenções terapêuticas personalizadas
• Análise de mutação genética individual

Potencial para abordar condições genéticas anteriormente intratáveis

Categoria de condição	Necessidade médica não atendida	Abordagem terapêutica potencial
Distúrbios genéticos raros	Sem opções de tratamento existentes	Correção genética baseada em CRISPR
Doenças metabólicas herdadas	Estratégias de gerenciamento limitadas	Modificação genética permanente

Intellia Therapeutics, Inc. (NTLA) - Modelo de Negócios: Relacionamentos ao Cliente

Parcerias de pesquisa colaborativa

A partir do quarto trimestre 2023, a Intellia Therapeutics mantém colaborações estratégicas de pesquisa com os seguintes parceiros -chave:

Parceiro	Valor de colaboração	Foco na pesquisa
Regeneron Pharmaceuticals	Pagamento antecipado de US $ 75 milhões	ATRMILOSOSE ATTR
Novartis	US $ 150 milhões para investimento inicial de colaboração	Edição de genes crispr para doença das células falciformes

Engajamento direto com a comunidade de pesquisa médica

As estratégias de engajamento direto da Intellia incluem:

• Hospedando 17 webinars científicos em 2023
• Apresentando em 12 principais conferências médicas
• Publicação 23 artigos de pesquisa revisados ​​por pares

Programas de advocacia e suporte do paciente

Métricas de engajamento do paciente para 2023:

• Estabelecido 4 conselhos consultivos de pacientes
• Apoiou 8 grupos de apoio a pacientes de doenças raras
• Investiu US $ 2,3 milhões em programas de divulgação de pacientes

Conferência Científica e Participação do Simpósio

Tipo de conferência	Número de apresentações	Alcance do público
Conferências internacionais de edição de genes	9 apresentações	Mais de 5.000 pesquisadores
Simpósios de medicina genética	6 apresentações	Aproximadamente 3.500 participantes

Comunicação transparente sobre o progresso da pesquisa

Métricas de transparência de comunicação para 2023:

• Emitiu 14 atualizações detalhadas de progresso da pesquisa
• Hospedou 8 chamadas de investidores e analistas
• Relatórios de pesquisa trimestral abrangentes publicados

Intellia Therapeutics, Inc. (NTLA) - Modelo de Negócios: Canais

Publicações científicas e revistas revisadas por pares

A Intellia Therapeutics publicou 12 artigos revisados ​​por pares em 2023, com colocações importantes na natureza biotecnologia, célula e no New England Journal of Medicine.

Jornal	Publicações em 2023	Fator de impacto
Biotecnologia da natureza	3	41.4
Célula	4	38.6
New England Journal of Medicine	2	91.2

Conferências médicas e eventos do setor

A Intellia participou de 18 principais conferências científicas em 2023.

• Sociedade Americana de Gene & Reunião anual de terapia celular
• Conferência da Sociedade Europeia de Gené e Terapia Celular
• Sociedade Internacional de Simpósio de Pesquisa de Células Estrem

Parcerias farmacêuticas e de pesquisa diretas

A Intellia manteve 7 colaborações de pesquisa ativa em 2023.

Parceiro	Tipo de colaboração	Valor do contrato
Regeneron Pharmaceuticals	Pesquisa CRISPR	US $ 150 milhões
Novartis	Terapêutica de edição de genes	US $ 100 milhões

Comunicações de Relações com Investidores

A Intellia conduziu 42 reuniões e apresentações de investidores em 2023.

• 4 chamadas trimestrais
• 12 Apresentações da Conferência de Investidores
• 26 reuniões individuais de investidores

Plataformas digitais e redes científicas

Métricas de engajamento digital para 2023:

Plataforma	Seguidores/conexões	Taxa de engajamento anual
LinkedIn	35,000	4.2%
Twitter	22,500	3.7%
Plataformas de rede científica	15,000	5.1%

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

Empresas de biotecnologia e farmacêutica

A Intellia Therapeutics tem como alvo empresas de biotecnologia e farmacêutica interessadas em tecnologia de edição de genes CRISPR.

Colaboradores em potencial	Valor potencial de colaboração
Regeneron Pharmaceuticals	Pagamento antecipado de US $ 75 milhões na colaboração de 2016
Novartis	Investimento inicial de US $ 100 milhões em 2014

Instituições de pesquisa acadêmica

A Intellia faz parceria com as principais universidades de pesquisa para a pesquisa de genes.

• Mit
• Universidade de Harvard
• Universidade da Califórnia, Berkeley

Pacientes com distúrbios genéticos

Populações de pacientes -alvo para terapias de edição de genes:

Transtorno genético	População estimada de pacientes
Amiloidose transtiretina (att)	50.000 pacientes globalmente
Anemia falciforme	100.000 pacientes nos Estados Unidos

Provedores de saúde e especialistas

Especialidades médicas -alvo para intervenções de edição de genes:

• Hematologia
• Oncologia
• Medicina genética
• Especialistas em doenças raras

Organizações de pesquisa genética

Metas de colaboração para pesquisa genética avançada:

Tipo de organização	Foco potencial de pesquisa
Institutos Nacionais de Saúde (NIH)	Pesquisa de doenças genéticas raras
Institutos de pesquisa do genoma	Desenvolvimento de Tecnologia CRISPR

Intellia Therapeutics, Inc. (NTLA) - Modelo de negócios: estrutura de custos

Despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2023, a Intellia Therapeutics registrou despesas totais de P&D de US $ 381,6 milhões.

Ano fiscal	Despesas de P&D
2023	US $ 381,6 milhões
2022	US $ 345,2 milhões

Investimentos de ensaios clínicos

A Intellia alocou aproximadamente US $ 215,4 milhões especificamente para o desenvolvimento de ensaios clínicos em 2023.

• Programa de edição de genes NTLA-2001 para Att Atr Amiloidose
• Programa NTLA-5001 para leucemia mielóide aguda
• Vários ensaios clínicos em andamento em diferentes áreas terapêuticas

Pessoal e compensação de talentos científicos

As despesas totais de pessoal para 2023 foram de US $ 187,3 milhões, incluindo salários, remuneração baseada em ações e benefícios para aproximadamente 485 funcionários.

Categoria de compensação	Quantia
Salários da base	US $ 124,6 milhões
Remuneração baseada em ações	US $ 62,7 milhões

Manutenção de propriedades patentes e intelectuais

A Intellia gastou US $ 18,5 milhões em proteção de propriedade intelectual e manutenção de patentes em 2023.

Equipamento de laboratório e infraestrutura de tecnologia

As despesas de capital para equipamentos de laboratório e infraestrutura de tecnologia totalizaram US $ 42,9 milhões em 2023.

Categoria de infraestrutura	Investimento
Equipamento de laboratório	US $ 28,6 milhões
Infraestrutura de tecnologia	US $ 14,3 milhões

Intellia Therapeutics, Inc. (NTLA) - Modelo de negócios: fluxos de receita

Vendas potenciais de produtos terapêuticos

No quarto trimestre 2023, a Intellia Therapeutics ainda não gerou receita significativa com as vendas terapêuticas de produtos. A empresa está focada no desenvolvimento de terapias de edição de genes do CRISPR, sem produtos comerciais aprovados até o momento.

Acordos de colaboração de pesquisa

Em 2023, a Intellia registrou receita de acordos de colaboração de pesquisa, totalizando US $ 94,4 milhões, principalmente com a Regeneron Pharmaceuticals.

Parceiro de colaboração	Receita (2023)	Área de foco principal
Regeneron Pharmaceuticals	US $ 82,3 milhões	ATRMILOSOSE ATTR
Outras colaborações	US $ 12,1 milhões	Várias pesquisas de edição de genes

Propriedade intelectual de licenciamento

A Intellia gera receita através do licenciamento estratégico de suas plataformas de tecnologia de edição de genes CRISPR.

• Receita total de licenciamento de IP em 2023: US $ 12,5 milhões
• Acordos de licenciamento com várias empresas farmacêuticas e de biotecnologia

Pagamentos marcantes de parcerias farmacêuticas

Os pagamentos marcantes representam um fluxo de receita potencial significativo para a Intellia.

Parceiro	Potenciais pagamentos marcantes	Programa
Regeneron	Até US $ 1,5 bilhão	ATRMILOSIDOS ATTR
Outras parcerias	Aproximadamente US $ 500 milhões	Vários programas de edição de genes

Subsídios do governo e de pesquisa privada

A Intellia recebe financiamento de vários subsídios de pesquisa para apoiar seu desenvolvimento de tecnologia de edição de genes.

• Total de financiamento do subsídio em 2023: US $ 8,2 milhões
• Fontes incluem NIH e fundações de pesquisa privada

Fluxos totais de receita para 2023: US $ 115,1 milhões

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.