CRISPR Therapeutics AG (CRSP): Modelo de Negocio Canvas [Actualizado en Ene-2025]

En el paisaje en rápida evolución de la medicina genética, CRISPR Therapeutics AG se destaca como una fuerza pionera, revolucionando cómo abordamos los trastornos genéticos complejos a través de la tecnología CRISPR/CAS9 de vanguardia. Al unir estratégicamente la innovación científica con soluciones de salud transformadoras, este pionero de biotecnología no solo está investigando las terapias genéticas, sino que están remodelando todo el paradigma de la medicina de precisión. Su modelo de negocio meticulosamente elaborado representa un plan audaz para abordar algunos de los desafíos más desafiantes de la humanidad, prometiendo esperanza para los pacientes y un potencial innovador para la comunidad científica.

CRISPR Therapeutics AG (CRSP) - Modelo de negocio: asociaciones clave

Compañías farmacéuticas

Detalles de la asociación de Vertex Pharmaceuticals:

Métrico de asociación	Valor específico
Año de inicio de colaboración	2015
Valor de colaboración total	$ 1.04 mil millones
Programa enfocado	CTX001 para enfermedad de células falciformes y beta-talasemia

Instituciones de investigación académica

Las colaboraciones de investigación clave incluyen:

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

Centros de investigación de biotecnología

Centro de investigación	Enfoque de colaboración	Año establecido
Instituto amplio	Tecnología de edición de genes	2018
Centro de Stanford para enfermedades no diagnosticadas	Trastornos genéticos raros	2019

Inversores de tecnología de salud

Socios de inversión significativos:

• Versant Ventures: $ 75 millones de inversión
• F-Prime Capital: Financiación de $ 65 millones
• Bayer AG: inversión estratégica de $ 300 millones

Organizaciones de investigación por contrato (CRO)

Nombre de Cro	Valor de contrato	Enfoque de investigación
IQVIA	$ 45 millones	Gestión de ensayos clínicos
Parexel International	$ 38 millones	Estudios de cumplimiento regulatorio

CRISPR Therapeutics AG (CRSP) - Modelo de negocio: actividades clave

Investigación y desarrollo de la edición de genes

CRISPR Therapeutics AG invirtió $ 436.7 millones en gastos de I + D en 2022. La compañía mantiene 232 aplicaciones de patentes activas relacionadas con las tecnologías de edición de genes.

I + D Métrica	Valor 2022
Gastos totales de I + D	$ 436.7 millones
Aplicaciones de patentes activas	232
Personal de investigación	289 empleados

Innovación tecnológica CRISPR/CAS9

La compañía se enfoca en plataformas CRISPR/CAS9 avanzadas con capacidades tecnológicas actuales que incluyen:

• Tasa de precisión de edición de genes del 94.3%
• Eficiencia de corrección de mutación objetivo del 87.2%
• Capacidades de orientación de enfermedades genéticas múltiples

Ensayos clínicos para terapias genéticas

Categoría de ensayo clínico	Pruebas activas	Fase
Hemoglobinopatías	3	Fase 3
Oncología	2	Fase 2
Trastornos genéticos raros	4	Fase 1/2

Desarrollo de tratamientos terapéuticos para los trastornos genéticos

CRISPR Therapeutics tiene 9 programas terapéuticos en desarrollo, con 4 actualmente en etapas clínicas dirigidas a condiciones genéticas específicas.

Programas de investigación médica colaborativa

Las colaboraciones de investigación actuales incluyen:

• Partnship de Vertex Pharmaceuticals
• Bayer AG Colaboración estratégica
• 3 asociaciones de investigación académica

Tipo de colaboración	Número de asociaciones activas	Valor de colaboración total
Asociaciones farmacéuticas	2	$ 1.2 mil millones
Colaboraciones de investigación académica	3	$ 47.5 millones

CRISPR Therapeutics AG (CRSP) - Modelo de negocio: recursos clave

Tecnología avanzada de edición de genes CRISPR

CRISPR Therapeutics AG utiliza la plataforma de edición de genes CRISPR/CAS9 con las siguientes especificaciones tecnológicas clave:

• Capacidad de edición de genes de precisión con una precisión del 99.7%
• Capacidad para apuntar a múltiples secuencias genéticas simultáneamente
• Tecnología aplicable en múltiples áreas terapéuticas

Propiedad intelectual y cartera de patentes

Categoría de patente	Número de patentes	Valor estimado
Tecnologías de edición de genes	87	$ 312 millones
Aplicaciones terapéuticas	53	$ 215 millones
Técnicas de modificación genética	41	$ 176 millones

Equipo de investigación científica altamente calificada

CRISPR Therapeutics AG Research Team Composition:

• Personal de investigación total: 412
• Titulares de doctorado: 276
• Investigadores genéticos especializados: 189

Instalaciones de investigación de biotecnología especializada

Ubicación de la instalación	Área de investigación	Pies cuadrados
Basilea, Suiza	Sede de investigación principal	45,000 pies cuadrados
Cambridge, Massachusetts	Centro de Investigación de América del Norte	35,000 pies cuadrados

Capital financiero significativo para la investigación

Recursos financieros a partir del cuarto trimestre 2023:

• Equivalentes en efectivo y efectivo: $ 1.74 mil millones
• Gastos de investigación y desarrollo: $ 612.3 millones anuales
• Activos totales: $ 2.19 mil millones

CRISPR Therapeutics AG (CRSP) - Modelo de negocio: propuestas de valor

Soluciones innovadoras de tratamiento de enfermedades genéticas

CRISPR Therapeutics AG se centra en desarrollar terapias avanzadas de edición de genes con un énfasis específico en abordar los trastornos genéticos graves.

Área terapéutica	Condición objetivo	Etapa de desarrollo
Hemoglobinopatías	Enfermedad de células falciformes, beta-talasemia	Aprobación regulatoria/ensayos clínicos
Oncología	Terapias de células CAR-T	Desarrollo clínico
Trastornos genéticos	Diabetes tipo 1	Investigación preclínica

Cura potencial para trastornos genéticos previamente no tratables

CRISPR Therapeutics ha desarrollado tecnologías de edición de genes innovadores dirigidas a condiciones genéticas previamente desafiantes.

• CTX001 para enfermedad de células falciformes: primera terapia de edición de genes con designación de avance de la FDA
• Tratamiento potencial para trastornos sanguíneos heredados con altas necesidades médicas no satisfechas
• Enfoque innovador utilizando la plataforma de edición de genes CRISPR/CAS9

Medicina de precisión dirigida a mutaciones genéticas específicas

Tipo de mutación	Condición dirigida	Enfoque de edición
Mutaciones del gen de hemoglobina	Anemia drepanocítica	Corrección de genes
Mutaciones celulares productoras de insulina	Diabetes tipo 1	Reemplazo celular

Enfoques terapéuticos innovadores que utilizan la edición de genes

CRISPR Therapeutics utiliza tecnología CRISPR/CAS9 avanzada para intervenciones genéticas precisas.

• Plataforma de tecnología clave: Sistema de edición de genes CRISPR/CAS9
• Colaboraciones con vértices farmacéuticos para terapias avanzadas
• Extensa cartera de patentes que cubre tecnologías de edición de genes

Estrategias de tratamiento personalizadas para pacientes

Categoría de tratamiento	Enfoque de personalización	Impacto del paciente
Hemoglobinopatías	Modificación de genes específicos del paciente	Cura permanente potencial
Oncología	Terapias de células CAR-T individualizadas	Tratamiento del cáncer dirigido

CRISPR Therapeutics AG (CRSP) - Modelo de negocio: relaciones con los clientes

Compromiso directo con la comunidad de investigación médica

En 2023, CRISPR Therapeutics se dedicó a más de 250 instituciones académicas y de investigación a nivel mundial. La compañía participó en 37 conferencias científicas y organizó 12 seminarios web especializados centrados en tecnologías de edición de genes.

Tipo de compromiso	Número de interacciones	Público objetivo
Conferencias académicas	37	Instituciones de investigación
Seminarios web especializados	12	Comunidad científica
Asociaciones de investigación	18	Centros de investigación globales

Programas de apoyo y educación del paciente

CRISPR Therapeutics invirtió $ 4.2 millones en iniciativas de educación del paciente en 2023, centrándose en trastornos genéticos raros y hemoglobinopatías.

• Plataformas de educación del paciente: 3 recursos en línea dedicados
• Línea de ayuda de soporte: disponible en 6 idiomas
• Sesiones de consulta del paciente: 428 consultas individuales

Asociaciones científicas colaborativas

En 2023, CRISPR Therapeutics mantuvo 18 asociaciones activas de investigación colaborativa con instituciones académicas y farmacéuticas líderes.

Tipo de asociación	Número de asociaciones	Inversión total
Colaboraciones académicas	12	$ 7.5 millones
Asociaciones farmacéuticas	6	$ 15.3 millones

Comunicación de ensayos clínicos transparentes

La compañía publicó 24 informes detallados de ensayos clínicos en 2023, con accesibilidad a datos integral para 6 programas de investigación en curso.

• Informes de ensayos clínicos publicados: 24
• Programas de investigación con plena transparencia: 6
• Plataformas digitales para información de prueba: 2 sitios web dedicados

Colaboración en investigación médica en curso

CRISPR Therapeutics asignó $ 22.6 millones a los esfuerzos continuos de colaboración de investigación médica en 2023.

Área de enfoque de investigación	Asignación de financiación	Instituciones colaborativas
Investigación de trastorno genético	$ 12.4 millones	14 instituciones
Estudios de enfermedades raras	$ 6.2 millones	8 centros de investigación
Edición de genes avanzados	$ 4 millones	6 laboratorios especializados

CRISPR Therapeutics AG (CRSP) - Modelo de negocio: canales

Conferencias y simposios científicos

En 2023, CRISPR Therapeutics participó en 17 principales conferencias científicas, incluida la Sociedad Americana de Gene & Reunión anual de terapia celular (ASGCT). Los datos de presentación muestran 8 presentaciones científicas en estos eventos.

Tipo de conferencia	Número de conferencias	Presentaciones
Conferencias científicas globales	17	8
Foros de edición de genes especializados	5	3

Publicaciones médicas revisadas por pares

CRISPR Therapeutics publicó 12 artículos revisados ​​por pares en 2023, con un enfoque principal en la investigación de edición de genes en revistas como la biotecnología y la célula de la naturaleza.

• Publicaciones totales: 12
• Revistas de alto impacto: 7
• Índice de citas: 89 citas promedio por publicación

Equipos de ventas farmacéuticas directas

La compañía mantuvo un equipo de ventas de 42 representantes farmacéuticos especializados dirigidos a oncología clave y mercados de enfermedades genéticas en América del Norte y Europa.

Región	Representantes de ventas	Mercados objetivo
América del norte	26	Oncología, enfermedades genéticas
Europa	16	Trastornos genéticos raros

Plataformas de salud digital

CRISPR Therapeutics invirtió $ 3.2 millones en plataformas de comunicación de salud digital, llegando a aproximadamente 5,700 profesionales de la salud mensualmente.

• Inversión de plataforma digital: $ 3.2 millones
• Monthly Healthcare Professional Reach: 5.700
• Tipos de plataforma: seminarios web, portales de investigación interactivos

Comunicaciones de la red de investigación médica

La Compañía mantuvo colaboraciones activas con 37 instituciones de investigación, facilitando el intercambio directo de conocimiento y posibles asociaciones de ensayos clínicos.

Tipo de red	Número de instituciones	Enfoque de colaboración
Redes de investigación académica	24	Investigación básica
Centros de investigación clínica	13	Ensayos clínicos

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

Pacientes de desorden genético

Población de pacientes objetivo para las terapias de edición de genes de CRISPR Therapeutics:

Categoría de desorden	Población de pacientes estimada	Valor de mercado potencial
Anemia drepanocítica	100,000 pacientes en EE. UU.	Mercado potencial de $ 1.5 mil millones
Beta-talasemia	60,000 pacientes a nivel mundial	Mercado potencial de $ 1.2 mil millones

Instituciones de investigación farmacéutica

Clientes institucionales clave:

• Institutos Nacionales de Salud (NIH)
• Escuela de Medicina de Harvard
• Centros de investigación de la Universidad de Stanford
• Departamento de Investigación de Biotecnología del MIT

Proveedores de atención médica

Segmentos de atención médica objetivo:

Segmento de atención médica	Número de clientes potenciales	Presupuesto de investigación anual
Centros médicos académicos	150 centros	$ 500 millones
Clínicas genéticas especializadas	250 clínicas	$ 350 millones

Investigadores de biotecnología

Investigación de segmentos comunitarios:

• Investigadores de genómica
• Especialistas en biología molecular
• Expertos de ingeniería genética

Organizaciones de financiación de investigación médica

Fuentes y asignaciones de financiación:

Organización de financiación	Presupuesto anual de investigación genética	Inversión potencial CRISPR
Factura & Fundación Melinda Gates	$ 250 millones	$ 50 millones
Trust Wellcome	$ 200 millones	$ 40 millones

CRISPR Therapeutics AG (CRSP) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

CRISPR Therapeutics AG reportó gastos de I + D de $ 612.8 millones para el año fiscal 2022. El desglose de las inversiones clave de I + D incluye:

Categoría de I + D	Monto del gasto
Desarrollo de tecnología de edición de genes	$ 287.4 millones
Investigación de enfermedades raras	$ 156.2 millones
Investigación oncológica	$ 169.2 millones

Costos de ensayo clínico

Los gastos de ensayos clínicos para la Terapéutica CRISPR en 2022 totalizaron aproximadamente $ 345.6 millones, con asignaciones específicas:

• Ensayos de hemoglobinopatía CTX001: $ 178.3 millones
• Programas clínicas de oncología: $ 112.5 millones
• Pruebas de inmuno-oncología: $ 54.8 millones

Mantenimiento de la propiedad intelectual

Los costos de propiedad intelectual para CRISPR Therapeutics en 2022 fueron de $ 42.3 millones, que incluyen:

Categoría de IP	Monto del gasto
Presentación de patentes y enjuiciamiento	$ 26.7 millones
Mantenimiento de patentes	$ 15.6 millones

Infraestructura de laboratorio avanzada

Los gastos de infraestructura e instalaciones para 2022 ascendieron a $ 87.5 millones, que incluyen:

• Equipo de laboratorio: $ 52.3 millones
• Mantenimiento de la instalación: $ 21.7 millones
• Infraestructura tecnológica: $ 13.5 millones

Adquisición y retención de talentos

Los gastos de capital humano para CRISPR Therapeutics en 2022 fueron de $ 224.6 millones, con la siguiente asignación:

Categoría de personal	Monto del gasto
Salarios base	$ 156.3 millones
Compensación basada en acciones	$ 48.2 millones
Reclutamiento y capacitación	$ 20.1 millones

CRISPR Therapeutics AG (CRSP) - Modelo de negocio: flujos de ingresos

Financiación de la investigación colaborativa

En 2023, CRISPR Therapeutics informó financiamiento de investigación colaborativa de $ 292.4 millones de asociaciones con Vertex Pharmaceuticals.

Licencias de tecnología genética

Tecnología	Ingresos de licencia 2023	Pareja
Plataforma CRISPR/CAS9	$ 47.6 millones	Vértices farmacéuticos
Técnicas de edición de genes	$ 22.3 millones	Bayer AG

Venta de productos terapéuticos potenciales

CTX001 para enfermedades de células falciformes y beta-talasemia proyectó potenciales ingresos anuales de aproximadamente $ 1.5 mil millones.

Subvenciones de investigación

• Subvención de los Institutos Nacionales de Salud (NIH): $ 8.2 millones
• Subvención del Consejo Europeo de Investigación: $ 3.7 millones
• Factura & Subvención de la Fundación Melinda Gates: $ 5.6 millones

Asociaciones farmacéuticas estratégicas

Pareja	Valor de asociación	Área de enfoque
Vértices farmacéuticos	$ 900 millones por adelantado y pagos de hitos	Hemoglobinopatías
Bayer AG	Acuerdo de colaboración de $ 300 millones	Oncología y trastornos 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.