CRISPR Therapeutics AG (CRSP): Modelo de negócios Canvas [Jan-2025 Atualizado]

Na paisagem em rápida evolução da medicina genética, a Crispr Therapeutics AG permanece como uma força pioneira, revolucionando como abordamos distúrbios genéticos complexos por meio da tecnologia CRISPR/CAS9 de ponta. Ao preencher estrategicamente a inovação científica com soluções transformadoras de saúde, este pioneiro de biotecnologia não está apenas pesquisando terapias genéticas - elas estão reformulando todo o paradigma da medicina de precisão. Seu modelo de negócios meticulosamente criado representa um plano ousado para abordar alguns dos desafios genéticos mais desafiadores da humanidade, promissora esperança para os pacientes e o potencial de avanço para a comunidade científica.

CRISPR Therapeutics AG (CRSP) - Modelo de negócios: Parcerias -chave

Empresas farmacêuticas

Detalhes da Parceria farmacêutica da Vertex:

Métrica de Parceria	Valor específico
Ano de início da colaboração	2015
Valor total de colaboração	US $ 1,04 bilhão
Programa focado	CTX001 para doença das células falciformes e beta-talassemia

Instituições de pesquisa acadêmica

As principais colaborações de pesquisa incluem:

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

Centros de pesquisa de biotecnologia

Centro de Pesquisa	Foco de colaboração	Ano estabelecido
Broad Institute	Tecnologia de edição de genes	2018
Centro de Stanford para doenças não diagnosticadas	Distúrbios genéticos raros	2019

Investidores em tecnologia da saúde

Parceiros de investimento significativos:

• Ventuos versantes: investimento de US $ 75 milhões
• F-Prime Capital: financiamento de US $ 65 milhões
• Bayer AG: US $ 300 milhões em investimento estratégico

Organizações de pesquisa contratada (CROs)

Nome do CRO	Valor do contrato	Foco na pesquisa
Iqvia	US $ 45 milhões	Gerenciamento de ensaios clínicos
Parexel International	US $ 38 milhões	Estudos de conformidade regulatória

CRISPR Therapeutics AG (CRSP) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento de edição de genes

A CRISPR Therapeutics AG investiu US $ 436,7 milhões em despesas de P&D em 2022. A Companhia mantém 232 pedidos de patentes ativos relacionados às tecnologias de edição de genes.

Métrica de P&D	2022 Valor
Despesas totais de P&D	US $ 436,7 milhões
Aplicações de patentes ativas	232
Pessoal de pesquisa	289 funcionários

Inovação tecnológica CRISPR/CAS9

A empresa se concentra em plataformas avançadas de CRISPR/CAS9 com recursos de tecnologia atuais, incluindo:

• Taxa de precisão de edição de genes de 94,3%
• Eficiência de correção de mutação alvo de 87,2%
• Múltiplas capacidades de direcionamento de doenças genéticas

Ensaios clínicos para terapias genéticas

Categoria de ensaio clínico	Ensaios ativos	Fase
Hemoglobinopatias	3	Fase 3
Oncologia	2	Fase 2
Distúrbios genéticos raros	4	Fase 1/2

Desenvolvimento de tratamentos terapêuticos para distúrbios genéticos

A CRISPR Therapeutics possui 9 programas terapêuticos em desenvolvimento, com 4 atualmente em estágios clínicos direcionados a condições genéticas específicas.

Programas de pesquisa médica colaborativa

As colaborações atuais de pesquisa incluem:

• Parceria farmacêutica da Vertex
• Colaboração estratégica da Bayer AG
• 3 parcerias de pesquisa acadêmica

Tipo de colaboração	Número de parcerias ativas	Valor total de colaboração
Parcerias farmacêuticas	2	US $ 1,2 bilhão
Colaborações de pesquisa acadêmica	3	US $ 47,5 milhões

CRISPR Therapeutics AG (CRSP) - Modelo de negócios: Recursos -chave

Tecnologia avançada de edição de genes CRISPR

A CRISPR Therapeutics AG utiliza plataforma de edição de genes CRISPR/CAS9 com as seguintes especificações tecnológicas principais:

• Capacidade de edição de genes de precisão com 99,7% de precisão
• Capacidade de atingir várias seqüências genéticas simultaneamente
• Tecnologia aplicável em várias áreas terapêuticas

Propriedade intelectual e portfólio de patentes

Categoria de patentes	Número de patentes	Valor estimado
Tecnologias de edição de genes	87	US $ 312 milhões
Aplicações terapêuticas	53	US $ 215 milhões
Técnicas de modificação genética	41	US $ 176 milhões

Equipe de pesquisa científica altamente qualificada

Composição da equipe de pesquisa da CRISPR Therapeutics AG:

• Pessoal de pesquisa total: 412
• Titulares de doutorado: 276
• Pesquisadores genéticos especializados: 189

Instalações especializadas de pesquisa de biotecnologia

Localização da instalação	Área de pesquisa	Metragem quadrada
Basileia, Suíça	Sede da pesquisa primária	45.000 pés quadrados
Cambridge, Massachusetts	Centro de pesquisa norte -americano	35.000 pés quadrados

Capital financeiro significativo para pesquisa

Recursos Financeiros a partir do quarto trimestre 2023:

• Caixa e equivalentes em dinheiro: US $ 1,74 bilhão
• Despesas de pesquisa e desenvolvimento: US $ 612,3 milhões anualmente
• Total de ativos: US $ 2,19 bilhões

CRISPR Therapeutics AG (CRSP) - Modelo de negócios: proposições de valor

Soluções inovadoras de tratamento de doenças genéticas

A CRISPR Therapeutics AG se concentra no desenvolvimento de terapias avançadas de edição de genes, com uma ênfase específica no tratamento de distúrbios genéticos graves.

Área terapêutica	Condições alvo	Estágio de desenvolvimento
Hemoglobinopatias	Doença das células falciformes, beta-talassemia	Aprovação regulatória/ensaios clínicos
Oncologia	Terapias de células CAR-T	Desenvolvimento Clínico
Distúrbios genéticos	Diabetes tipo 1	Pesquisa pré -clínica

Cura potencial para distúrbios genéticos anteriormente intratáveis

A CRISPR Therapeutics desenvolveu tecnologias inovadoras de edição de genes direcionados às condições genéticas anteriormente desafiadoras.

• CTX001 para doença das células falciformes: Primeira terapia de edição de genes com designação de avanço da FDA
• Tratamento potencial para distúrbios sanguíneos herdados com altas necessidades médicas não atendidas
• Abordagem inovadora usando plataforma de edição de genes CRISPR/CAS9

Medicina de precisão direcionada a mutações genéticas específicas

Tipo de mutação	Condição direcionada	Abordagem de edição
Mutações no gene da hemoglobina	Anemia falciforme	Correção de genes
Mutações celulares produtoras de insulina	Diabetes tipo 1	Substituição de células

Abordagens terapêuticas inovadoras usando edição de genes

A CRISPR Therapeutics utiliza a tecnologia avançada de CRISPR/CAS9 para intervenções genéticas precisas.

• Plataforma de tecnologia -chave: Sistema de edição de genes CRISPR/CAS9
• Colaborações com farmacêuticos de vértices para terapias avançadas
• Extenso portfólio de patentes cobrindo tecnologias de edição de genes

Estratégias de tratamento personalizadas para pacientes

Categoria de tratamento	Abordagem de personalização	Impacto do paciente
Hemoglobinopatias	Modificação de genes específicos do paciente	Cura permanente potencial
Oncologia	Terapias de células Car-T individualizadas	Tratamento de câncer direcionado

CRISPR Therapeutics AG (CRSP) - Modelo de negócios: Relacionamentos do cliente

Engajamento direto com a comunidade de pesquisa médica

Em 2023, a Crispr Therapeutics se envolveu com mais de 250 instituições acadêmicas e de pesquisa em todo o mundo. A empresa participou de 37 conferências científicas e organizou 12 webinars especializados focados em tecnologias de edição de genes.

Tipo de engajamento	Número de interações	Público -alvo
Conferências Acadêmicas	37	Instituições de pesquisa
Webinars especializados	12	Comunidade científica
Parcerias de pesquisa	18	Centros de Pesquisa Global

Programas de apoio ao paciente e educação

A CRISPR Therapeutics investiu US $ 4,2 milhões em iniciativas de educação de pacientes em 2023, concentrando -se em distúrbios genéticos raros e hemoglobinopatias.

• Plataformas de educação do paciente: 3 recursos online dedicados
• Suporte Helpline: Disponível em 6 idiomas
• Sessões de consulta do paciente: 428 consultas individuais

Parcerias científicas colaborativas

Em 2023, a CRISPR Therapeutics manteve 18 parcerias de pesquisa colaborativa ativas com as principais instituições acadêmicas e farmacêuticas.

Tipo de parceria	Número de parcerias	Investimento total
Colaborações acadêmicas	12	US $ 7,5 milhões
Parcerias farmacêuticas	6	US $ 15,3 milhões

Comunicação transparente de ensaio clínico

A empresa publicou 24 relatórios detalhados de ensaios clínicos em 2023, com acessibilidade abrangente de dados para 6 programas de pesquisa em andamento.

• Relatórios de ensaios clínicos publicados: 24
• Programas de pesquisa com total transparência: 6
• Plataformas digitais para informações de teste: 2 sites dedicados

Colaboração de pesquisa médica em andamento

A CRISPR Therapeutics alocou US $ 22,6 milhões aos esforços contínuos de colaboração de pesquisa médica em 2023.

Área de foco de pesquisa	Alocação de financiamento	Instituições colaborativas
Pesquisa de Transtorno Genético	US $ 12,4 milhões	14 instituições
Estudos de doenças raras	US $ 6,2 milhões	8 centros de pesquisa
Edição avançada de genes	US $ 4 milhões	6 laboratórios especializados

CRISPR Therapeutics AG (CRSP) - Modelo de negócios: canais

Conferências científicas e simpósios

Em 2023, a CRISPR Therapeutics participou de 17 principais conferências científicas, incluindo a Sociedade Americana de Gene & Reunião anual de terapia celular (ASGCT). Os dados de apresentação mostram 8 apresentações científicas nesses eventos.

Tipo de conferência	Número de conferências	Apresentações
Conferências científicas globais	17	8
Fóruns de edição de genes especializados	5	3

Publicações médicas revisadas por pares

A CRISPR Therapeutics publicou 12 artigos revisados ​​por pares em 2023, com foco primário em pesquisas de edição de genes em periódicos como a Nature Biotechnology and Cell.

• Publicações totais: 12
• Revistas de alto impacto: 7
• Índice de citação: 89 citações médias por publicação

Equipes de vendas farmacêuticas diretas

A empresa manteve uma equipe de vendas de 42 representantes farmacêuticos especializados, direcionados aos principais mercados de oncologia e doenças genéticas na América do Norte e na Europa.

Região	Representantes de vendas	Mercados -alvo
América do Norte	26	Oncologia, doenças genéticas
Europa	16	Distúrbios genéticos raros

Plataformas de saúde digital

A CRISPR Therapeutics investiu US $ 3,2 milhões em plataformas de comunicação em saúde digital, atingindo aproximadamente 5.700 profissionais de saúde mensalmente.

• Investimento de plataforma digital: US $ 3,2 milhões
• Alcance mensal do profissional de saúde: 5.700
• Tipos de plataforma: webinars, portais de pesquisa interativos

Comunicações da Rede de Pesquisa Médica

A Companhia manteve colaborações ativas com 37 instituições de pesquisa, facilitando a troca direta de conhecimento e possíveis parcerias de ensaios clínicos.

Tipo de rede	Número de instituições	Foco de colaboração
Redes de pesquisa acadêmica	24	Pesquisa básica
Centros de Pesquisa Clínica	13	Ensaios clínicos

CRISPR Therapeutics AG (CRSP) - Modelo de negócios: segmentos de clientes

Pacientes com transtorno genético

População de pacientes direcionados para terapias de edição de genes da Crispr Therapeutics:

Categoria de distúrbio	População estimada de pacientes	Valor potencial de mercado
Anemia falciforme	100.000 pacientes em nós	Mercado potencial de US $ 1,5 bilhão
Beta-talassemia	60.000 pacientes globalmente	Mercado potencial de US $ 1,2 bilhão

Instituições de pesquisa farmacêutica

Principais clientes institucionais:

• Institutos Nacionais de Saúde (NIH)
• Escola de Medicina de Harvard
• Centros de pesquisa da Universidade de Stanford
• Departamento de Pesquisa de Biotecnologia do MIT

Provedores de saúde

Segmentos de saúde -alvo:

Segmento de saúde	Número de clientes em potencial	Orçamento de pesquisa anual
Centros Médicos Acadêmicos	150 centros	US $ 500 milhões
Clínicas genéticas especializadas	250 clínicas	US $ 350 milhões

Pesquisadores de biotecnologia

Segmentos da comunidade de pesquisa:

• Pesquisadores genômicos
• Especialistas em biologia molecular
• Especialistas em engenharia genética

Organizações de financiamento de pesquisa médica

Fontes e alocações de financiamento:

Organização de financiamento	Orçamento anual de pesquisa genética	Potencial investimento do CRISPR
Conta & Melinda Gates Foundation	US $ 250 milhões	US $ 50 milhões
Wellcome Trust	US $ 200 milhões	US $ 40 milhões

CRISPR Therapeutics AG (CRSP) - Modelo de negócios: estrutura de custos

Extensas despesas de pesquisa e desenvolvimento

A CRISPR Therapeutics AG reportou despesas de P&D de US $ 612,8 milhões para o ano fiscal de 2022. A repartição dos principais investimentos em P&D inclui:

Categoria de P&D	Quantidade de despesa
Desenvolvimento de tecnologia de edição de genes	US $ 287,4 milhões
Pesquisa de doenças raras	US $ 156,2 milhões
Pesquisa de oncologia	US $ 169,2 milhões

Custos de ensaios clínicos

Os gastos com ensaios clínicos para terapêutica da CRISPR em 2022 totalizaram aproximadamente US $ 345,6 milhões, com alocações específicas:

• Ensaios de hemoglobinopatia CTX001: US $ 178,3 milhões
• Programas clínicos de oncologia: US $ 112,5 milhões
• Ensaios de imuno-oncologia: US $ 54,8 milhões

Manutenção da propriedade intelectual

Os custos de propriedade intelectual da Crispr Therapeutics em 2022 foram de US $ 42,3 milhões, incluindo:

Categoria IP	Quantidade de despesa
Arquivamento e acusação de patentes	US $ 26,7 milhões
Manutenção de patentes	US $ 15,6 milhões

Infraestrutura de laboratório avançado

As despesas de infraestrutura e instalação de 2022 totalizaram US $ 87,5 milhões, incluindo:

• Equipamento de laboratório: US $ 52,3 milhões
• Manutenção da instalação: US $ 21,7 milhões
• Infraestrutura de tecnologia: US $ 13,5 milhões

Aquisição e retenção de talentos

As despesas de capital humano para a Crispr Therapeutics em 2022 foram de US $ 224,6 milhões, com a seguinte alocação:

Categoria de pessoal	Quantidade de despesa
Salários da base	US $ 156,3 milhões
Remuneração baseada em ações	US $ 48,2 milhões
Recrutamento e treinamento	US $ 20,1 milhões

CRISPR Therapeutics AG (CRSP) - Modelo de negócios: fluxos de receita

Financiamento de pesquisa colaborativa

Em 2023, a CRISPR Therapeutics relatou financiamento colaborativo de pesquisa de US $ 292,4 milhões de parcerias com a Vertex Pharmaceuticals.

Licenciamento de tecnologia genética

Tecnologia	Receita de licenciamento 2023	Parceiro
Plataforma CRISPR/CAS9	US $ 47,6 milhões	Pharmaceuticals de vértice
Técnicas de edição de genes	US $ 22,3 milhões	Bayer AG

Vendas potenciais de produtos terapêuticos

O CTX001 para doença falciforme e beta-talassemia projetou uma receita anual potencial de aproximadamente US $ 1,5 bilhão.

Bolsas de pesquisa

• Grant do National Institutes of Health (NIH): US $ 8,2 milhões
• Grant do Conselho de Pesquisa Europeia: US $ 3,7 milhões
• Conta & Melinda Gates Foundation Grant: US $ 5,6 milhões

Parcerias farmacêuticas estratégicas

Parceiro	Valor da parceria	Área de foco
Pharmaceuticals de vértice	Pagamentos antecipados e marcantes de US $ 900 milhões	Hemoglobinopatias
Bayer AG	Contrato de colaboração de US $ 300 milhões	Oncologia e distúrbios genéticos

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Value Propositions

You're looking at the core of what CRISPR Therapeutics AG offers, and honestly, it's all about delivering on the promise of gene editing for serious, life-altering conditions. The value proposition starts with their approved product, CASGEVY, which is a curative, one-time treatment for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT).

For CASGEVY, the commercial momentum is building. Vertex, their partner, expects a clear line of sight to over $100 million in total CASGEVY revenue this year (2025). Remember, CRISPR Therapeutics AG keeps 40% of that profit split. By the end of the third quarter of 2025, approximately 165 patients had completed their first cell collection, and 39 had received infusions across all regions. The total addressable market in the currently approved regions is estimated at around 60,000 patients, so they are just scratching the surface of that opportunity. To support this, more than 65 authorized treatment centers (ATCs) were activated globally as of May 2025. The Q3 2025 revenue from Casgevy sales alone was $17 million. This product directly addresses the high unmet medical need in these rare and chronic genetic disorders.

Beyond the approved therapy, the pipeline offers significant future value, particularly with their off-the-shelf allogeneic CAR-T therapies. Take CTX112 for cancer; preliminary safety and efficacy data in oncology showed responses in all 6 patients who had previously failed T-cell engager-based therapies (TCEs). This included responses in 3 large B-cell lymphoma (LBCL) patients who were refractory to those prior TCE treatments. This suggests a strong value proposition for patients with limited options.

The in vivo gene editing candidates targeting common diseases are another major pillar. For cardiovascular risk, CTX310 and CTX320 are key. CTX310, targeting the ANGPTL3 gene, showed impressive early results in a Phase 1 trial for lipid disorders. For instance, one patient with severe hypertriglyceridemia saw an 82% reduction in TG (Triglycerides) after treatment. Another patient with heterozygous familial hypercholesterolemia (HeFH) achieved an 81% reduction in LDL-C. Furthermore, CTX320 targets the LPA gene, addressing elevated lipoprotein(a) [Lp(a)], which is prevalent in up to 20% of the global population and is a major cardiovascular risk factor.

Here's a quick look at how the pipeline candidates stack up against their targets:

Candidate	Target Indication/Disease Area	Key Metric/Target Population
CASGEVY (exa-cel)	SCD and TDT	Curative, one-time treatment
CTX112	Oncology/Autoimmune	Response in 6/6 prior TCE-refractory patients
CTX310	Cardiovascular (Lipid)	Up to 82% reduction in TG observed
CTX320	Cardiovascular (Lp(a))	Addresses prevalence up to 20% of global population

Finally, CRISPR Therapeutics AG is working on next-generation regenerative medicine for Type 1 diabetes (T1D). The value here is aiming for insulin independence in T1D patients using induced pluripotent stem cell (iPSC) derived, allogeneic, gene-edited, beta islet cell precursors, specifically designed to work without the need for chronic immunosuppression.

The company's ability to fund this ambitious pipeline is supported by a strong balance sheet, holding approximately $1.94 billion in cash, cash equivalents, and marketable securities as of September 30, 2025, despite reporting a net loss of $106.4 million for that same quarter. This financial foundation helps secure the runway for these long-term value drivers.

You can see the core value propositions are built on:

• Curative potential for rare blood disorders with CASGEVY.
• Promising early efficacy in refractory cancer with CTX112.
• Disruptive potential for common cardiovascular diseases with CTX310/320.
• Addressing high unmet needs across the portfolio.
• A next-generation approach to T1D aiming for functional cure.

Finance: draft 13-week cash view by Friday.

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Customer Relationships

High-touch, specialized clinical support for patients undergoing cell therapy is managed through an expanding global network of Authorized Treatment Centers (ATCs) for CASGEVY.

The activation of these centers has progressed rapidly following the initial launch.

• End of 2024: More than 50 ATCs activated globally.
• As of May 1, 2025: More than 65 ATCs activated globally.
• As of August 4, 2025: The target of 75 ATCs activated globally was achieved.

Patient engagement metrics show the conversion from referral to treatment initiation:

Metric (as of date)	Value	Reference Point
Patients with first cell collection (May 1, 2025)	Approximately 90
Patients with first cell collection (Aug 4, 2025)	Approximately 115
Patients referred to ATCs (Sep 30, 2025)	Nearly 300
Patients completing first cell collection (Sep 30, 2025)	Approximately 165
Patients who received infusions (Sep 30, 2025)	39

Direct engagement with payers and government agencies focuses on securing access through complex reimbursement agreements.

This engagement has resulted in securing patient access across multiple jurisdictions for CASGEVY:

• As of August 2025, access was secured for eligible SCD and TDT patients in 10 countries through reimbursement agreements.
• Vertex secured a formal reimbursement agreement with NHS England for SCD patients.
• The U.S. Centers for Medicare & Medicaid Services (CMS) initiated the Cell & Gene Therapy Access Model, an outcomes-based arrangement.
• Recent agreements include access in Northern Ireland, Scotland, and Denmark as of August 2025.

Dedicated medical affairs teams educate physicians at ATCs, supporting the high-touch model required for cell therapy administration.

The collaborative relationship management with strategic partner Vertex Pharmaceuticals governs the commercialization of CASGEVY.

The financial structure of this relationship dictates revenue sharing and operational leadership:

Area of Responsibility	Lead Party	Financial Split (Cost/Profit)
Global Development, Manufacturing, Commercialization (CASGEVY)	Vertex	60/40 (Vertex/CRISPR Therapeutics)
U.S. Commercialization (CASGEVY)	CRISPR Therapeutics	60/40 (Vertex/CRISPR Therapeutics)

Vertex is the manufacturer and exclusive license holder of CASGEVY. Vertex expects clear line of sight to over $100 million in total CASGEVY revenue for the year 2025. CRISPR Therapeutics recognized $0.9 million in grant revenue in Q3 2025, with collaboration expense net at $57.1 million for the same quarter.

Patient advocacy group outreach supports the rare disease patient population for whom CASGEVY is indicated.

CRISPR Therapeutics maintained a strong balance sheet to support these customer-facing and development activities, holding approximately $1.9 billion in cash, cash equivalents, and marketable securities as of September 30, 2025.

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Channels

You're looking at how CRISPR Therapeutics AG gets its transformative therapies, like Casgevy, into the hands of patients and how they push their pipeline data out to the scientific community. It's a hybrid model, relying heavily on a partner for the heavy lifting on the commercial side, but also building out specialized access points.

For Casgevy, the immediate channel is a highly specialized, controlled network. CRISPR Therapeutics and its partner, Vertex Pharmaceuticals, have successfully activated a global network of over 75 Authorized Treatment Centers (ATCs) for Casgevy across all approved regions as of their Q2 2025 update. To be fair, the initial ramp was slow, but by the end of Q3 2025, nearly 300 patients had been referred to these ATCs, showing the channel is expanding its patient throughput capacity.

The commercial execution channel is primarily driven by Vertex Pharmaceuticals' established infrastructure. Vertex leads the global development, manufacturing, and commercialization of Casgevy, operating under an amended collaboration agreement where they split program costs and profits worldwide 60:40 with CRISPR Therapeutics. This structure means Vertex is responsible for the complex distribution logistics and sales force engagement. Vertex reported Casgevy sales of $30.4 million in the second quarter of 2025, reflecting accelerating market adoption. CRISPR Therapeutics' share of an estimated total 2025 revenue expectation is pegged at over $100 million.

Here's a quick look at how the commercial and partnership channels break down:

Channel Component	Lead Entity	Key Metric/Region	Financial/Volume Data
Casgevy Commercialization	Vertex Pharmaceuticals	Global Distribution	Vertex Q2 2025 Sales: $30.4 million
Casgevy Access Network	Authorized Treatment Centers (ATCs)	Global Activation	Over 75 ATCs activated as of mid-2025
Sirius Collaboration (SRSD107)	CRISPR Therapeutics / Sirius	U.S. Commercialization	Upfront payment to Sirius: $25 million cash + $70 million equity
Sirius Collaboration (SRSD107)	CRISPR Therapeutics / Sirius	Greater China Commercialization	Cost/Profit sharing is 50-50 for SRSD107

For pipeline candidates, the channel to market is direct clinical development and regulatory pathways. CRISPR Therapeutics manages this internally, focusing on advancing its wholly-owned assets. For instance, CTX112, targeting CD19 for oncology and autoimmune indications, was awarded regenerative medicine advanced therapy (RMAT) designation by the FDA, which streamlines the regulatory channel. You can expect broad updates for CTX112 in the second half of 2025.

Disseminating clinical data uses established scientific channels. This is how you build credibility for the pipeline. CRISPR Therapeutics planned a late-breaking oral presentation for its CTX310 Phase 1 data at the American Heart Association (AHA) Scientific Sessions 2025, scheduled for November 7 - 10, 2025, in New Orleans, Louisiana. Furthermore, Phase 1 data for CTX310 was also presented at the 2025 Annual Scientific Sessions of the American College of Cardiology.

The company also uses strategic partnerships to establish regional commercialization channels. The collaboration with Sirius Therapeutics, announced in 2025, is a prime example for siRNA therapies like SRSD107. CRISPR Therapeutics will lead commercialization in the U.S., while Sirius leads in Greater China. This partnership involved an upfront payment of $25 million in cash and $70 million in equity to Sirius.

These channels are all about getting the product to the patient or getting the data to the key opinion leaders. Finance: review the Q3 2025 revenue share reconciliation from Vertex by next Tuesday.

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Customer Segments

You're looking at the core groups CRISPR Therapeutics AG serves, which is really about who gets access to their groundbreaking gene-editing therapies, both now with Casgevy and in the pipeline.

Patients with Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) eligible for Casgevy.

This is the immediate, commercial customer base for Casgevy, which CRISPR Therapeutics co-developed with Vertex Pharmaceuticals. The therapy has regulatory approvals in 9 countries as of mid-2025, including the U.S., U.K., and the E.U.. The total addressable market (TAM) in these approved regions is estimated to be around 60,000 patients. For the U.S. and Europe alone, the addressable market is at least 35,000 patients, with potential to reach 166,000 if in vivo strategies are used. As of September 30, 2025, the adoption rate shows that approximately 165 patients globally had completed their first cell collection, and 39 patients had received their infusions. This is a ramp-up from the 29 infusions completed by June 30, 2025. In Italy, which has the largest TDT population in Europe, there are approximately 5,000 people aged 12 and older with TDT and around 2,300 with SCD. The list price for Casgevy is $2.2 million.

Here's a quick look at the adoption metrics as of late 2025:

Metric	Value as of Q3 2025 (Sept 30)	Context
Total Patients with Cell Collection Initiated (Global)	Approximately 165	Since launch through September 30th, 2025
Total Patients Infused (Global)	39	Since launch through September 30th, 2025
Cell Collections in First Nine Months of 2025	110	Double the total for all of 2024
Vertex Q3 2025 Revenue from Casgevy	$17 million	Realized from Casgevy sales in Q3 2025 alone

Oncologists and hematologists at specialized hospitals and treatment centers.

These professionals are the gatekeepers and administrators of the ex vivo therapy. The commercial rollout depends on activating and training staff at Authorized Treatment Centers (ATCs). CRISPR Therapeutics achieved its target of 75 ATCs globally. By the end of 2024, 50 ATCs were activated globally. The momentum continued, with more than 65 ATCs activated globally as of March 31, 2025. These centers are where the complex process of cell collection and subsequent infusion happens.

Patients with B-cell malignancies and autoimmune diseases (target for CTX112).

CTX112 is a key pipeline asset targeting CD19, and it has already shown promising early data in difficult-to-treat patient populations. The FDA granted CTX112 Regenerative Medicine Advanced Therapy (RMAT) designation. In oncology, preliminary data demonstrated responses in all 6 patients treated who had previously relapsed post-T-cell engager-based therapies (TCEs). This included 3 large B-cell lymphoma (LBCL) patients refractory to TCEs. The company planned a broad update on CTX112 in oncology and autoimmune disease in mid-2025.

Large patient populations with cardiometabolic diseases (target for CTX310/320).

CRISPR Therapeutics is using its in vivo LNP delivery platform for these indications. CTX310 targets ANGPTL3 for dyslipidemias, and CTX320 targets LPA for elevated Lipoprotein(a) [Lp(a)]. Elevated Lp(a) is a risk factor for cardiovascular events affecting up to 20% of the global population. Initial Phase 1 data for CTX310 showed impressive lipid reductions, with peak reductions in triglycerides (TG) up to 82% and in LDL up to 86%. The company planned to present complete Phase 1 data for CTX310 in the second half of 2025.

Global healthcare systems and government payers (e.g., CMS, European agencies).

Payer acceptance is critical given the high upfront cost of gene therapy. Vertex has secured access through reimbursement agreements for eligible patients in 10 countries as of mid-2025. In the U.S., the Centers for Medicare & Medicaid Services (CMS) proposed to reimburse hospitals 75% of the cost of administering Casgevy for fiscal year 2025, which is higher than the typical 65% New Technology Add-on Payment (NTAP). In Europe, Italy's Medicines Agency (AIFA) reached a reimbursement agreement, which is significant because Italy has approximately 5,000 TDT patients aged 12 and older. The list price in some markets reaches up to $2.8 million per administration.

• CMS enhanced payment is intended to last for the two-to-three year newness period.
• Italy joins 7 other European countries with reimbursement agreements for Casgevy.
• The therapy's value proposition to payers centers on eliminating lifelong transfusions and hospitalizations.

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Cost Structure

You're looking at the core expenses driving CRISPR Therapeutics AG's operations as of late 2025. For a company at this stage, the cost structure is heavily weighted toward discovery and development, which is exactly what the numbers show.

Here's a quick look at the key operating expenses for the first nine months of 2025:

Expense Category	Period	Amount (USD)
Research & Development (R&D) Expenses	Q1-Q3 2025 (YTD)	$201.3 million
General & Administrative (G&A) Expenses	Q1-Q3 2025 (YTD)	$55.1 million
Collaboration Expense, net	Q3 2025 Only	$57.1 million

The dominant R&D expenses are the engine room here, totaling $201.3 million for Q1-Q3 2025. This spend reflects the heavy lifting required to advance the pipeline, including in vivo gene editing candidates and next-generation CAR T programs like CTX112™.

You see a significant hit from Collaboration Expense, net, which was $57.1 million in Q3 2025 alone. This is largely tied to the co-development and commercialization efforts for CASGEVY® with Vertex Pharmaceuticals. Under that amended agreement, Vertex leads global development, manufacturing, and commercialization, but CRISPR Therapeutics still shares program costs and profits worldwide on a 60/40 split.

The General and Administrative (G&A) costs, which cover overhead and support functions, came in at $55.1 million for the first three quarters of 2025. This is a necessary cost to support the growing commercial launch of CASGEVY and the overall corporate infrastructure.

When we talk about manufacturing and supply chain costs for complex ex vivo cell therapy, much of that is embedded within the collaboration expense structure for CASGEVY, as Vertex is the manufacturer and exclusive license holder. However, the company is also advancing its own internally developed programs, like the anti-CD117 ADC, which carries its own separate manufacturing development costs.

Also baked into the overall cost base are expenses related to the intellectual property environment. The foundational CRISPR patent rights remain fiercely contested across jurisdictions. While specific, direct litigation expense figures aren't broken out in the standard quarterly reports, you must budget for the ongoing legal risks associated with this complex landscape, which involves proceedings across the US and Europe between key patent holders like the CVC group and the Broad Institute.

The cost structure is clearly defined by these major buckets:

• R&D expenses were $72.5 million in Q1 2025 and $69.9 million in Q2 2025.
• G&A was $19.3 million in Q1 2025 and $18.9 million in Q2 2025.
• The Sirius collaboration added a one-time $96.3 million Acquired In-Process R&D Expense in Q2 2025.

Finance: draft 13-week cash view by Friday.

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of CRISPR Therapeutics AG's business model as of late 2025, which is heavily weighted toward its landmark collaboration with Vertex Pharmaceuticals, supplemented by non-product related income streams.

Collaboration revenue from Casgevy sales is the most significant expected driver, though the actual recognized revenue in the third quarter was still modest compared to the full-year expectation. Vertex Pharmaceuticals expects a clear line of sight to over $100 million in total CASGEVY revenue for 2025. CRISPR Therapeutics shares in this revenue on a 60/40 split, meaning CRISPR Therapeutics is entitled to 40% of the profits. Based on Vertex's projection, this implies a potential full-year 2025 revenue share for CRISPR Therapeutics of around $40 million. To be fair, Vertex realized $17 million in revenue from Casgevy sales in the third quarter of 2025 alone, showing the commercial momentum building through the final months of the year.

The reported total revenue for CRISPR Therapeutics AG for the third quarter ended September 30, 2025, was $0.889 million. For the nine months ended September 30, 2025, total revenue reached $2.65 million. This reported revenue was primarily sourced from grant income.

The company also generates income from its large cash position. For the third quarter of 2025, Other income was $26.2 million, which the company stated was primarily from interest income and changes in the fair value of investments. This income helps offset operating expenses. The cash position remained substantial, with cash, cash equivalents, and marketable securities totaling approximately $1.94 billion as of September 30, 2025.

Strategic partnerships contribute through milestone payments, although specific upfront payments for 2025 are not explicitly detailed in the latest reports, other than the impact of the Sirius collaboration. The Sirius Therapeutics collaboration, established in 2025, involves co-development and an equal sharing of costs and profits for the lead program SRSD107. The year-to-date net loss was influenced by $96.3 million of acquired in-process R&D tied to the Sirius collaboration.

Here's a quick look at the key financial figures impacting the revenue and income side as of Q3 2025:

Revenue Component / Metric	Amount (USD)	Period / Date
Total Revenue	0.889 million	Q3 2025
Total Revenue	2.65 million	Nine Months Ended September 30, 2025
Vertex Casgevy Total Revenue Expectation (Partner)	Over $100 million	Full Year 2025 Projection
Vertex Casgevy Revenue Recognized (Partner)	$17 million	Q3 2025
CRISPR Therapeutics Casgevy Share Percentage	40%	Agreement Term
Other Income (Interest/Investments)	$26.2 million	Q3 2025
Cash, Cash Equivalents, and Marketable Securities	$1,944.1 million	September 30, 2025
Acquired In-Process R&D (Sirius Impact)	$96.3 million	Year to Date 2025

Future product sales from wholly-owned pipeline assets are not yet contributing to recognized revenue, but clinical progress is key to future value. The pipeline is advancing with several updates expected:

• Positive top-line data from the Phase 1 clinical trial of CTX310 targeting ANGPTL3, with data presentation anticipated in the second half of 2025.
• Broad updates expected for CTX112 in oncology and autoimmune diseases in the second half of 2025.
• CTX131 updates also expected in 2025.
• Preclinical data for CTX460 demonstrated in vivo gene correction, with clinical trial initiation planned for mid-2026.

R&D reimbursement from collaboration agreements is implicitly part of the cost-sharing structure, as evidenced by the collaboration expense, net, which was $57.1 million in Q3 2025. This figure represents CRISPR Therapeutics' share of the costs, which is offset by revenue sharing, but the specific reimbursement component is not broken out separately from the expense in the public filings.

Equity investments are realized through capital raises, such as issuing 5.1 million shares under an ATM for net proceeds of $286.8 million during the first nine months of 2025.

Finance: draft 13-week cash view by Friday.