Voyager Therapeutics, Inc. (VYGR): Canvas del Modelo de Negocio [Actualizado en Ene-2025]

En el reino de vanguardia de la terapéutica neurológica, Voyager Therapeutics (VYGR) emerge como una fuerza pionera, transformando la medicina genética a través de innovaciones innovadoras de terapia génica. Al aprovechar la ingeniería vectorial avanzada y las intervenciones genéticas dirigidas, este innovador de biotecnología está listo para revolucionar los enfoques de tratamiento para trastornos neurológicos complejos, ofreciendo esperanza a pacientes con afecciones genéticas raras donde las estrategias médicas tradicionales se han quedado cortas. Su lienzo de modelo de negocio integral revela un plan estratégico que entrelaza la excelencia científica, las asociaciones colaborativas y el potencial médico transformador, prometiendo remodelar el panorama de los tratamientos neurológicos personalizados.

Voyager Therapeutics, Inc. (VYGR) - Modelo de negocios: asociaciones clave

Colaboración con compañías farmacéuticas

Voyager Therapeutics ha establecido una asociación estratégica con las biosciencias neurocrinas, centrándose en el desarrollo de la terapia génica para los trastornos neurológicos.

Pareja	Detalles de la asociación	Términos financieros
Biosciencias neurocrinas	Colaboración de terapia génica para afecciones neurológicas	Pago por adelantado de $ 75 millones en 2018
Abad	Terapia génica de la enfermedad de Parkinson	$ 455 millones pagos potenciales totales

Asociaciones de investigación con instituciones académicas

Voyager Therapeutics mantiene colaboraciones críticas de investigación con centros de investigación académicos líderes.

• Hospital General de Massachusetts
• Escuela de Medicina de Harvard
• Universidad de California, San Francisco

Alianzas estratégicas en el desarrollo de la terapia génica

La compañía ha desarrollado alianzas estratégicas para avanzar en las tecnologías de terapia génica.

Socio de la alianza	Área de enfoque	Alcance de colaboración
Pfizer	Investigación de enfermedades neurológicas genéticas	Múltiples programas de terapia génica preclínica
Sanofi	Trastornos neurológicos raros	Investigación y desarrollo colaborativo

Acuerdos de licencia

Voyager Therapeutics ha asegurado múltiples acuerdos de licencia para tecnologías avanzadas de tratamiento neurológico.

• Licencia de tecnología del MIT: Derechos exclusivos para la plataforma de terapia génica patentada
• Licencia de biotecnología de Harvard: Tecnologías de tratamiento de enfermedades neurológicas

Ingresos totales de asociación e licencia para 2023: $ 28.3 millones

Voyager Therapeutics, Inc. (VYGR) - Modelo de negocio: actividades clave

Investigación y desarrollo de terapia génica

A partir del cuarto trimestre de 2023, Voyager Therapeutics ha invertido $ 42.3 millones en gastos de I + D relacionados con el desarrollo de la terapia génica. La compañía se enfoca en desarrollar terapias genéticas basadas en AAV dirigidas a los trastornos neurológicos.

Área de enfoque de investigación	Inversión (2023)	Programas activos
Trastornos neurológicos	$ 42.3 millones	3 programas de terapia génica primaria
Enfermedades genéticas raras	$ 18.7 millones	2 programas de etapa preclínica

Gestión de ensayos clínicos

Voyager Therapeutics actualmente administra 4 ensayos clínicos activos en múltiples áreas de indicación neurológica.

• Ensayos de fase 1/2 para la enfermedad de Huntington
• Ensayos de fase 1 para la enfermedad de Parkinson
• Desarrollo clínico de terapia génica de estadio avanzado

Investigación preclínica y traslacional

La compañía mantiene una sólida canal de investigación preclínica con 6 candidatos terapéuticos potenciales en el desarrollo.

Etapa de investigación	Número de candidatos	Indicaciones objetivo
Preclínico	6 candidatos	Trastornos neurológicos
Investigación traslacional	3 programas	Condiciones genéticas raras

Ingeniería vectorial patentada

Voyager se ha desarrollado 15 plataformas Vector AAV únicas Para la entrega de genes dirigidos.

• Técnicas avanzadas de ingeniería de cápsulas
• Capacidades de orientación de tejido neural mejorado
• Eficiencia mejorada de transferencia de genes

Innovación del tratamiento de enfermedades neurológicas

El gasto de investigación para las innovaciones de enfermedades neurológicas alcanzó los $ 27.6 millones en 2023.

Área de enfermedades	Presupuesto de investigación	Enfoque de innovación
Enfermedad de Huntington	$ 12.4 millones	Estrategias de silenciamiento de genes
Enfermedad de Parkinson	$ 9.2 millones	Entrega de factores neurotróficos
Otras condiciones neurológicas	$ 6 millones	Enfoques de terapia génica dirigidos

Voyager Therapeutics, Inc. (VYGR) - Modelo de negocio: recursos clave

Plataformas de tecnología de terapia génica avanzada

Voyager Therapeutics utiliza Plataforma de ingeniería vectorial de terapia génica AAV Con las siguientes especificaciones:

• Variantes de la cápside AAV totalmente diseñada: 171,000+
• Plataforma de tecnología de evolución dirigida propietaria
• Capacidades de diseño de vectores de terapia génica dirigida a los trastornos neurológicos

Métricas de plataforma tecnológica	Datos cuantitativos
Variantes de cápside AAV	171,000+
Investigación & Inversión en desarrollo (2023)	$ 52.4 millones
Capacidades de diseño de vectores de terapia génica	5 objetivos de enfermedad neurológica distintas

Cartera de propiedades intelectuales

Voyager Therapeutics mantiene una sólida estrategia de propiedad intelectual:

• Patentes totales otorgadas: 84
• Aplicaciones de patentes pendientes: 36
• Familias de patentes que cubren tecnologías de terapia génica

Experiencia de investigación científica

Composición del equipo de investigación	Número
Total de la investigación científicos	68
Investigadores de doctorado	42
Especialistas en neurociencia	24

Infraestructura de laboratorio especializada

Las instalaciones de laboratorio incluyen:

• Espacio total de la instalación de investigación: 45,000 pies cuadrados
• Capacidades de fabricación de vectores de genes avanzados
• Laboratorios de investigación de nivel 2 y nivel de bioseguridad

Equipo de gestión experimentado

Posición de liderazgo	Años de experiencia
CEO	22 años en biotecnología
Oficial científico	18 años en terapia génica
Investigación & Director de desarrollo	15 años en investigación de neurociencia

Voyager Therapeutics, Inc. (VYGR) - Modelo de negocio: propuestas de valor

Terapias génicas dirigidas para trastornos neurológicos

Voyager Therapeutics se centra en el desarrollo de terapias genéticas específicamente dirigidas a los trastornos neurológicos. A partir del cuarto trimestre de 2023, la compañía tiene 3 programas primarios de terapia génica en desarrollo clínico.

Programa de terapia	Trastorno objetivo	Estadio clínico
Vy-Aadc	Enfermedad de Parkinson	Fase 1/2
VY-HTT01	Enfermedad de Huntington	Preclínico
VY-SOD01	Algancios	Fase 1/2

Posibles tratamientos innovadores para afecciones genéticas raras

La compañía ha identificado 5 condiciones neurológicas genéticas raras como objetivos terapéuticos primarios.

• Enfermedad de Parkinson
• Enfermedad de Huntington
• Esclerosis lateral amiotrófica (ELA)
• Ataxia de Friedreich
• Ataxia espinocerebelosa

Diseño vectorial innovador para intervenciones genéticas precisas

Voyager se ha desarrollado tecnologías de vector AAV patentadas con capacidades mejoradas de orientación de tejidos. Las inversiones de investigación en ingeniería vectorial fueron de aproximadamente $ 24.7 millones en 2023.

Enfoques terapéuticos personalizados

Acercarse	Descripción	Costo de desarrollo
Detección genética	Perfil genético específico del paciente	$ 3.2 millones
Dosificación de precisión	Intervenciones terapéuticas personalizadas	$ 5.6 millones

Abordar las necesidades médicas no satisfechas en enfermedades neurodegenerativas

El potencial de mercado para las terapias de genes neurológicos estimados en $ 12.5 mil millones para 2026. El gasto de investigación y desarrollo de Voyager en 2023 fue de $ 67.3 millones.

• Programas de tuberías totales: 6
• Ensayos clínicos activos: 3
• Portafolio de patentes: 45 patentes otorgadas

Voyager Therapeutics, Inc. (VYGR) - Modelo de negocios: relaciones con los clientes

Compromiso directo con las comunidades de pacientes

A partir del cuarto trimestre de 2023, Voyager Therapeutics mantiene la participación directa de la comunidad del paciente a través de:

• Grupos de apoyo a enfermedades neurodegenerativas
• Redes de pacientes de desorden genético raros
• Plataformas de comunicación de pacientes en línea

Métricas de interacción comunitaria del paciente	2023 datos
Participantes de la red total de pacientes	3,287
Tasa de compromiso en línea	67.4%
Eventos anuales de la comunidad de pacientes	12

Comunicación de investigación colaborativa

Voyager Therapeutics implementa estrategias de comunicación de investigación colaborativa con:

• Asociaciones de la institución de investigación
• Programas de colaboración académica
• Interacciones de la Junta Asesora Científica

Investigación de métricas de colaboración	2023 datos
Asociaciones de investigación activa	18
Subvenciones de investigación colaborativa	$ 4.2 millones
Publicaciones científicas	22

Apoyo de los participantes del ensayo clínico

El marco integral de apoyo de los participantes clínicos incluye:

• Coordinadores dedicados de apoyo al paciente
• Recursos de información sobre el ensayo clínico
• Manejo de compensación de pacientes

Métricas de soporte de ensayos clínicos	2023 datos
Ensayos clínicos activos	7
Participantes totales de ensayos clínicos	412
Tasa de retención del paciente	85.6%

Educación y divulgación profesional médico

Las estrategias de participación profesional médica abarcan:

• Programas de educación médica continua
• Presentaciones de conferencias científicas
• Interacciones profesionales de red médica

Métricas de divulgación médica profesional	2023 datos
Presentaciones de conferencia médica	16
Participantes del programa CME	1,243
Miembros de la red profesional	5,672

Informes de progreso de investigación transparente

Los canales de comunicación transparentes incluyen:

• Actualizaciones de investigación trimestrales
• Comunicaciones de relaciones con los inversores
• Plataformas de divulgación científica pública

Investigación de métricas de transparencia	2023 datos
Informes de investigación trimestrales	4
Divulgaciones de investigación pública	37
Eventos de comunicación de inversores	6

Voyager Therapeutics, Inc. (VYGR) - Modelo de negocios: canales

Conferencias y presentaciones científicas directas

En 2023, Voyager Therapeutics participó en 12 principales conferencias científicas, presentando investigaciones en:

• Sociedad Americana de Gene & Reunión anual de terapia celular
• Conferencia anual de la Sociedad de Neurociencia
• Congreso internacional de trastorno de parkinson y movimiento del movimiento

Tipo de conferencia	Número de presentaciones en 2023	Alcance de audiencia estimado
Conferencias de neurociencia	7	3.500 investigadores
Simposios de terapia génica	5	2.800 especialistas

Redes de la industria farmacéutica

Voyager Therapeutics mantuvo asociaciones estratégicas con:

• Instituciones de investigación de neurociencia
• Redes de desarrollo farmacéutico
• Centros de investigación académicos

Categoría de asociación	Número de asociaciones activas	Valor de colaboración
Instituciones de investigación	8	$ 12.5 millones
Centros académicos	6	$ 7.3 millones

Publicaciones científicas revisadas por pares

Investigación publicada en revistas clave durante 2023:

• Terapia molecular
• Neurociencia de la naturaleza
• Neurona

Categoría de publicación	Número de publicaciones	Citas totales
Revistas revisadas por pares	9	287

Comunicaciones de relaciones con los inversores

Métricas de comunicación de inversores para 2023:

• Llamadas de ganancias trimestrales
• Presentaciones de inversores
• Reunión anual de accionistas

Tipo de comunicación	Frecuencia en 2023	Compromiso de los inversores
Llamadas de ganancias	4	215 participantes
Presentaciones de inversores	6	340 inversores institucionales

Plataformas científicas y médicas en línea

Plataformas de participación digital utilizadas en 2023:

• Investigador
• Red científica de LinkedIn
• Sitio web de la empresa

Plataforma	Seguidores/conexiones	Interacciones de contenido
Investigador	4,200	12,500 vistas
LinkedIn	3,800	9,700 compromisos

Voyager Therapeutics, Inc. (VYGR) - Modelo de negocios: segmentos de clientes

Pacientes con enfermedad neurológica

Voyager Therapeutics se dirige a pacientes con afecciones neurológicas específicas:

Categoría de enfermedades	Población de pacientes estimada	Condiciones dirigidas
Enfermedad de Parkinson	1 millón de pacientes en los Estados Unidos	Terapia génica VY-AADC
Enfermedad de Huntington	30,000 pacientes sintomáticos en los Estados Unidos	Terapia génica VY-HTT01

Comunidades de desorden genético raros

Centrarse en trastornos neurológicos genéticos específicos:

• Condiciones neurológicas genéticas raras que afectan a aproximadamente 25-30 millones de personas a nivel mundial
• Dirigirse a las poblaciones de pacientes con opciones de tratamiento limitadas
• Enfoques de terapia génica de precisión para mutaciones genéticas específicas

Instituciones de investigación de neurociencia

Asociaciones de colaboración e investigación:

Tipo de institución	Número de socios potenciales	Enfoque de investigación
Centros de investigación académicos	57 Instituciones de investigación de neurociencia importantes	Desarrollo de terapia génica
Laboratorios de neurociencia financiados por NIH	42 Instalaciones de investigación primarias	Investigación avanzada del tratamiento neurológico

Compañías farmacéuticas

Potencial de asociación estratégica:

• Las 20 principales compañías farmacéuticas globales con divisiones de neurociencia
• Oportunidades potenciales de licencias y colaboración
• Potencial de transferencia de tecnología de terapia génica

Proveedores de atención médica especializados en terapias genéticas

Target Centros médicos especializados:

Categoría de proveedor	Número de centros especializados	Enfoque terapéutico
Centros de tratamiento genético especializados	87 centros en América del Norte	Terapias de genes neurológicos avanzados
Redes de tratamiento de neurología	129 redes de atención médica integradas	Implementación de medicina de precisión

Voyager Therapeutics, Inc. (VYGR) - Modelo de negocio: Estructura de costos

Gastos de investigación y desarrollo

Para el año fiscal 2022, Voyager Therapeutics reportó gastos de I + D de $ 97.8 millones. Los costos de I + D de la compañía se centraron principalmente en los programas de terapia génica para trastornos neurológicos.

Año	Gastos de I + D	Porcentaje de gastos operativos totales
2022	$ 97.8 millones	68.3%
2021	$ 84.3 millones	65.7%

Inversiones de ensayos clínicos

Voyager Therapeutics invirtió $ 52.4 millones en actividades de ensayos clínicos durante 2022, dirigidos a múltiples programas de enfermedades neurológicas.

• Programa clínico VY-AADC: $ 18.2 millones
• Programa de enfermedad de Huntington: $ 15.7 millones
• Programa de enfermedades de Parkinson: $ 12.5 millones

Mantenimiento de la propiedad intelectual

La compañía gastó aproximadamente $ 3.5 millones en protección de la propiedad intelectual y mantenimiento de patentes en 2022.

Desarrollo de la plataforma tecnológica

Los costos de desarrollo de la plataforma de tecnología para 2022 fueron de $ 22.6 millones, centrándose en tecnologías avanzadas de terapia génica.

Plataforma tecnológica	Monto de la inversión
AAV Vector Ingeniería	$ 12.3 millones
Sistemas de entrega de genes	$ 10.3 millones

Cumplimiento y pruebas regulatorias

El cumplimiento regulatorio y los gastos de prueba totalizaron $ 6.7 millones en 2022, asegurando el cumplimiento de la FDA y los estándares regulatorios internacionales.

• Pruebas preclínicas: $ 3.2 millones
• Preparación de presentación regulatoria: $ 2.5 millones
• Monitoreo de cumplimiento: $ 1.0 millones

Voyager Therapeutics, Inc. (VYGR) - Modelo de negocios: flujos de ingresos

Posibles acuerdos de licencia

A partir del cuarto trimestre de 2023, Voyager Therapeutics tiene posibles acuerdos de licencia con los siguientes socios farmacéuticos clave:

Pareja	Valor potencial de acuerdo	Área de enfoque
Biosciencias neurocrinas	Pagos potenciales de hito potencial de $ 1.1 mil millones	Terapia génica para la enfermedad de Parkinson
Abad	Términos financieros no revelados	Programas de enfermedades neurológicas genéticas

Subvenciones de investigación

Voyager Therapeutics recibió subvenciones de investigación de las siguientes fuentes:

• Institutos Nacionales de Salud (NIH): $ 2.5 millones en 2023
• Fundación Michael J. Fox: $ 1.2 millones para la investigación de la enfermedad de Parkinson

Financiación de la investigación colaborativa

Desglose de financiación de la investigación colaborativa para 2023:

Socio de colaboración	Monto de financiación
Hospital General de Massachusetts	$ 3.7 millones
Escuela de Medicina de Harvard	$ 2.1 millones

Venta de productos terapéuticos futuros

Ingresos potenciales proyectados del desarrollo de la tubería:

• Terapia génica VY-AADC: Valor de mercado potencial estimado de $ 450 millones anuales
• Tratamientos de trastorno neurológico: alcance potencial del mercado de $ 750 millones para 2026

Pagos de hitos de asociaciones farmacéuticas

Estructura de pago de hitos con socios farmacéuticos actuales:

Pareja	Pagos potenciales de hitos	Eventos de activación
Biosciencias neurocrinas	Hasta $ 1.1 mil millones	Desarrollo clínico, aprobaciones regulatorias
Abad	Pagos de hitos no revelados	Avance preclínico y clínico

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Value Propositions

Voyager Therapeutics, Inc. (VYGR) focuses its value creation on superior delivery across the blood-brain barrier (BBB) and a pipeline addressing severe, validated neurological targets.

Enhanced central nervous system (CNS) delivery via proprietary capsids.

The core value proposition in delivery centers on proprietary capsids designed for intravenous (IV) delivery across the BBB. This contrasts with older methods, offering a potentially safer and more efficient route for therapeutics. The company's gene therapy platform discovers capsids and the specific receptors they leverage for brain entry. The first identified receptor is ALPL.

The ALPL-VYGR-NeuroShuttle demonstrated sustained brain expression over three weeks in murine studies, which is significantly longer than the less than one week observed with transferrin receptor shuttles. Furthermore, the ALPL-based approach is positioned to potentially avoid the hematologic adverse events seen with first-generation TfR shuttles, which caused anemia in 10-20% of trial participants in one study.

Nonviral delivery option with the NeuroShuttle™ platform.

Voyager introduced the Voyager NeuroShuttle™ as a nonviral delivery platform. This platform uses novel receptor-binding molecules to transport various modalities of neurotherapeutics across the BBB. The platform's first program leverages the ALPL receptor. This nonviral approach is a key differentiator in the delivery space.

Multi-modality pipeline targeting validated neurological disease targets.

Voyager Therapeutics is building a multi-modality pipeline, optimizing the approach for each target. The company aims to have four programs in the clinic by 2026. The pipeline includes both gene therapies and small molecules, focusing on targets like Tau, Amyloid, APOE, TDP-43, and others related to FA and GBA1.

Here's a look at the pipeline progression as of late 2025:

Program/Platform	Target/Indication	Modality	Latest Status/Key Data Timing
VY7523	Tau / Alzheimer's Disease (AD)	Anti-tau Antibody	Dosing ongoing in final MAD cohort; initial tau PET data expected in H2 2026
VY1706	Tau Silencing / AD	Gene Therapy	IND-enabling studies ongoing; clinical trial initiation expected in 2026
Neurocrine Partnered Programs	Friedreich's Ataxia (FA) & GBA1	Gene Therapy	IND filings anticipated by end of 2025; clinical trials anticipated in 2026
Transition Bio Collaboration	TDP-43 / ALS and FTD	Small Molecule	Discovery-stage collaboration initiated
NeuroShuttle™ Discovery	Undisclosed Neurological Disease	Nonviral Delivery	First program introduced; preclinical data shared

Potential to modify or cure severe neurological diseases like AD and ALS.

The value proposition is centered on modifying the course of, and ultimately curing, neurological diseases. For AD, the strategy involves multiple approaches, including the anti-tau antibody VY7523 and the tau silencing gene therapy VY1706. For ALS, Voyager is pursuing the TDP-43 target via a small molecule collaboration, aiming to unlock a target historically considered undruggable. The company's overall pipeline addresses Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, and amyotrophic lateral sclerosis (ALS).

De-risking drug development through strategic pharma partnerships.

Strategic collaborations are used to de-risk development and extend financial stability. Voyager has 11 partnered programs that carry the potential for up to $2.6 billion in development-stage milestone payments.

• Neurocrine Biosciences partnership is advancing, with potential milestone payments up to $35 million expected in 2025-2026 from the FA and GBA1 programs entering the clinic. A $3 million milestone payment from Neurocrine is due in Q4 2025.
• The collaboration with Transition Bio for ALS/FTD has potential milestone payments totaling up to $500 million.
• Collaboration revenue for the third quarter of 2025 was $13.4 million.
• Novartis discontinued two discovery-stage programs, returning the rights to Voyager, which does not impact the cash runway guidance.

The company ended Q3 2025 with $229 million in cash, which is expected to fund operations into 2028.

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Customer Relationships

You're looking at how Voyager Therapeutics, Inc. manages its critical external relationships as of late 2025. This block is all about the high-touch interactions that fuel their development engine and secure their financial future.

Dedicated, high-touch collaboration management for pharma partners

Voyager Therapeutics, Inc. relies heavily on its strategic alliances to advance its pipeline, which includes 11 partnered programs carrying the potential for up to $2.6 billion in development-stage milestone payments. The relationship with Neurocrine Biosciences, Inc. remains active, with Neurocrine anticipating IND submissions in 2025 for the Friedreich's ataxia (FA) and GBA1 gene therapy programs, potentially leading to clinical trial initiations in 2026. A preclinical toxicology study initiated by Neurocrine triggered a $3 million milestone payment due to Voyager in the fourth quarter of 2025. The Novartis Pharma AG collaboration has seen a shift; Novartis notified Voyager of its intention to discontinue two discovery-stage programs. This dynamic is reflected in the collaboration revenue figures; Voyager reported collaboration revenue of $13.4 million for the third quarter of 2025, a decrease from $24.6 million in the third quarter of 2024, primarily due to revenue recognized under the 2022 Novartis Option and License Agreement in the prior year period. Collaboration revenue for the first quarter of 2025 was $6.5 million, down from $19.5 million in Q1 2024. You can see the key partnership details here:

Partner	Program Status/Update (as of late 2025)	Potential Milestone Value (from specific programs)	Q3 2025 Collaboration Revenue
Neurocrine Biosciences, Inc.	IND submissions anticipated in 2025 for FA and GBA1; potential clinical trial initiations in 2026.	Up to $35 million from FA and GBA1 programs entering the clinic.	$13.4 million (Total Q3 2025)
Novartis Pharma AG	Discontinued two discovery-stage programs.	Part of the overall potential up to $2.6 billion.

The company is also expanding its own external collaborations, entering into an agreement with Transition Bio for an option to license small molecules for ALS and FTD, which includes potential milestone payments totaling up to $500 million for a development candidate.

Investor relations and clear communication of clinical milestones

Investor communication focuses on cash runway and pipeline progression, though the company does not plan to host quarterly financial results conference calls moving forward. Voyager ended the third quarter of 2025 with a cash, cash equivalents, and marketable securities position of $229 million, which management expects is sufficient to meet operating expenses and capital expenditure requirements into 2028. This compares to $295 million at the end of Q1 2025 and $262 million as of June 30, 2025. The net loss for Q3 2025 was $27.9 million. Key communication points center on upcoming data readouts and IND filings:

• IND submissions anticipated in 2025 for Neurocrine-partnered FA and GBA1 programs.
• Clinical trial initiations anticipated in 2026 for FA and GBA1 programs.
• Initial tau PET imaging data expected in H2 2026 for the VY7523 MAD clinical trial.
• U.S. IND and Canadian CTA filings anticipated for VY1706 in AD in 2026.
• The company aims to introduce four programs into clinical trials by 2026.

Voyager Therapeutics, Inc. actively engaged with the investment community through multiple conferences in September 2025, including fireside chats at the Citi's 2025 Biopharma Back to School Conference and the Baird 2025 Global Healthcare Conference.

Patient-centric engagement through patient advocacy groups and resources

The stated core value is 'Patients First,' driving decisions with the knowledge that patients are waiting. Voyager Therapeutics, Inc. has shown engagement with the advocacy community, participating in the BIO Patient Advocacy Changemakers Event 2025. For specific diseases like ALS, the company references resources from The ALS Association and the Muscular Dystrophy Association (mda.org). However, regarding direct patient access outside of trials, the current policy states that Voyager does not offer expanded access for any investigational products in development, believing participation in clinical trials is the most appropriate pathway for access.

Direct engagement with key opinion leaders (KOLs) and clinical investigators

Direct engagement with the scientific and clinical community is evident through data presentations and conference participation. Voyager presented preclinical data supporting its non-viral delivery platform at the Citi's 2025 Biopharma Back to School Conference on September 2, 2025. The company also had a recap of its KOL engagement at the AD/PD™ 2025 meeting. Furthermore, data on the tau silencing gene therapy VY1706 was featured in an oral presentation at ASGCT 2025. The dosing is ongoing in the third and final cohort of the multiple ascending dose (MAD) clinical trial for VY7523 in Alzheimer's disease patients, which involves direct collaboration with clinical investigators.

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Channels

You're looking at how Voyager Therapeutics, Inc. (VYGR) gets its science and potential therapies out to the world, from the lab bench to the capital markets. It's a mix of high-stakes partnerships and scientific dissemination.

Direct licensing and collaboration agreements with major pharma

Voyager Therapeutics, Inc. relies heavily on these deals to fund its pipeline and share development risk. As of late 2025, the structure shows both active programs and rights reclamation.

The company has 11 partnered programs with the potential for up to $2.6 billion in development-stage milestone payments as of Q2 2025. A recent deal with Transition Bio, announced November 10, 2025, involves an option to license worldwide rights for small molecules targeting TDP-43 in ALS and FTD. Transition Bio received a single-digit million-dollar upfront payment for this collaboration. If Voyager exercises its option, the deal could total up to $500 million in research, development, commercial, and net-sales milestones, plus high single-digit to low double-digit royalties.

The Neurocrine partnership is also active; Neurocrine initiated a preclinical toxicology study with the fourth development candidate, triggering a $3 million milestone payment due to Voyager in the fourth quarter of 2025. Conversely, Novartis notified Voyager of its intention to discontinue two discovery-stage programs, returning the rights to Voyager.

Here's a snapshot of recent financial flows tied to these channels:

Financial Metric (as of Q3 2025)	Amount/Value	Context
Cash, Cash Equivalents, and Marketable Securities (Sep 30, 2025)	$229 million	Maintains cash runway into 2028
Collaboration Revenue (Q3 2025)	$13.4 million	Decrease from $24.6 million in Q3 2024
Total Potential Milestone Payments (Assumed)	Up to $2.4 billion	Non-dilutive capital potential
Specific Milestone Potential (GBA/FA programs entering clinic)	Up to $35 million	Part of the total potential
Q3 2025 Net Loss	$27.9 million	Compared to $9.0 million in Q3 2024

Clinical trial sites and principal investigators for drug testing

The testing channel involves using clinical sites to evaluate drug candidates like VY7523 and the Neurocrine-partnered gene therapies. You need sites to generate the data that validates the science.

For the VY7523 (anti-tau antibody) program, dosing is currently ongoing in the third and final cohort of the Multiple Ascending Dose (MAD) clinical trial in Alzheimer's disease patients. The earlier Phase 1a Single Ascending Dose (SAD) trial for VY-TAU01 (the precursor to VY7523) was conducted at a single site in the United States and expected to enroll approximately 48 patients in multiple cohorts.

The company is focused on advancing multiple programs toward the clinic:

• IND-enabling studies are ongoing for VY1706 (tau silencing gene therapy) to support clinical trial initiation expected in 2026.
• IND submissions for Neurocrine-partnered Friedreich's ataxia (FA) and GBA1 gene therapy programs are anticipated in 2025 to support clinical trial initiations in 2026.
• Voyager Therapeutics aims to introduce four programs into clinical trials by 2026.

Scientific publications and conference presentations (e.g., ASGCT 2025)

Disseminating preclinical and early clinical data through peer-reviewed channels and major medical meetings is critical for establishing scientific credibility and attracting future partners or investors. Voyager Therapeutics, Inc. was active in this area in 2025.

At the American Society of Gene & Cell Therapy's (ASGCT) 28th annual meeting in May 2025, Voyager made eight oral and poster presentations. Key data points shared included:

• VY1706 (tau silencing gene therapy) showed up to 73% knockdown of tau mRNA in Non-Human Primates (NHPs) following a single intravenous (IV) dose of 1.3e13 vg/kg.
• Novel TRACER capsids transduced 43%-98% of neurons and 87-99% of astrocytes broadly across brain regions following a single IV dose of 3e13 vg/kg in NHPs.
• Data on VY7523 and VY1706 were also presented at the AD/PD™ 2025 conference.

The company also achieved its first peer-reviewed publication of ALPL in the journal Molecular Therapy.

Investor presentations and financial reporting to capital markets

This channel focuses on communicating financial health and strategic progress to the investment community, which directly impacts capital access. The Q3 2025 financial results, reported November 10, 2025, serve as the latest data point.

The company reported that its cash, cash equivalents, and marketable securities totaled $229 million as of September 30, 2025. This position is expected to maintain the cash runway into 2028 based on current operating plans. The total potential for non-dilutive capital, including development milestone payments, is stated as up to $2.4 billion.

The Q3 2025 performance showed a net loss of $27.9 million, while collaboration revenue for the quarter was $13.4 million. The accumulated deficit stood at $418.5 million.

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Customer Segments

You're looking at who actually funds the work and who benefits from Voyager Therapeutics, Inc.'s pipeline as of late 2025. It's a mix of deep-pocketed pharma partners and the patient communities waiting on these neurogenetic medicines.

Major pharmaceutical and biotech companies seeking CNS gene therapy assets

These entities are crucial; they provide validation, capital via milestones, and often, a path to commercialization for Voyager Therapeutics, Inc.'s proprietary assets. Collaboration revenue is a key financial indicator of this segment's health. For instance, Q3 2025 saw collaboration revenue hit $13.4 million, down from $24.6 million in Q3 2024, partly due to revenue recognition timing under existing agreements. Still, the potential for future non-dilutive capital remains significant, especially with new deals.

Here's a look at the key relationships driving this segment:

Partner Company	Program Focus / Status	Potential Milestone Value
Neurocrine Biosciences, Inc.	Friedreich's ataxia (FA) and GBA1 gene therapy programs; IND submissions anticipated by end of 2025.	Up to $35 million in 2025-2026 for these programs.
Transition Bio	New collaboration for TDP-43 pathology in ALS and Frontotemporal Dementia (FTD).	Up to $500 million in potential milestone payments.
Novartis Pharma AG	Previously held options; two discovery-stage programs were discontinued, returning rights to Voyager Therapeutics, Inc.	N/A (Rights returned).
Alexion, AstraZeneca Rare Disease	One undisclosed gene therapy program.	Undisclosed.

Voyager Therapeutics, Inc. expects its cash, cash equivalents, and marketable securities of $229 million as of September 30, 2025, to support operations into 2028, which includes anticipated milestone receipts. The company aims to introduce four programs into clinical trials by 2026.

Patients with severe neurological diseases

This group represents the ultimate end-user, the reason for the entire business structure. Voyager Therapeutics, Inc. focuses on diseases where safe and effective treatments are lacking, leveraging human genetics to modify or cure these conditions. The company's core value is "Patients First," driving decisions with the knowledge that patients are waiting.

The primary indications targeted by the current pipeline include:

• Alzheimer's Disease (targeting Tau, amyloid, and APOE)
• Friedreich's Ataxia (FA)
• Parkinson's Disease
• Amyotrophic Lateral Sclerosis (ALS)
• Huntington's Disease (HD)
• Other tau-related neurodegenerative diseases

The VY7523 anti-tau antibody program for Alzheimer's Disease is in the final cohort of a multiple ascending dose clinical trial, with initial tau PET data expected in the second half of 2026.

Clinical investigators and academic research institutions

These institutions are vital for technology access, target validation, and executing the clinical trials that move assets from preclinical to patient-facing stages. Voyager Therapeutics, Inc. has secured necessary intellectual property through agreements with several key academic centers.

Key academic and research relationships include:

• University of Massachusetts Medical School (UMMS) for license and collaboration agreements.
• University of California San Francisco for access to relevant technology and data.
• Stanford University for access to relevant technology and data.

The company is also advancing its proprietary TRACER platform, which aims to enable high brain penetration following intravenous dosing by identifying novel capsids and associated receptors.

Investors and institutional funds focused on high-growth biotech

This segment provides the necessary equity financing to sustain operations through long development cycles. As of November 3, 2025, the company's market capitalization stood at $255 million, with a stock price of $4.59 per share, based on 55.6 million shares outstanding. The total funding raised to date is $105 million across 5 rounds.

You can see the composition of the financial backing below:

Financial Metric / Investor Type	Value / Example	Date/Context
Total Funding Raised	$105 million	Total across 5 rounds.
Cash Position	$229 million	As of September 30, 2025.
Institutional Investors	Brookside Capital, PFM Health Sciences, BB Biotech	Past investors.
Latest Equity Raise	$20 million	Post IPO round on January 03, 2024.
Trailing Twelve Month Revenue	$31.3 million	As of September 30, 2025.

The company's net loss for the third quarter of 2025 was $27.9 million, which is higher than the $9.0 million net loss in Q3 2024, largely due to the decrease in collaboration revenue.

Finance: review the Q4 2025 cash burn projection against the 2028 runway guidance by next Tuesday.

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Cost Structure

You're looking at the core spending engine for Voyager Therapeutics, Inc. as of late 2025. For a company deep in clinical development, the cost structure is almost entirely front-loaded into science and people.

The largest component of cost is definitely Research and Development (R&D). For the nine months ending September 30, 2025 (9M 2025), Voyager Therapeutics, Inc. reported high Research and Development (R&D) expenses, totaling $98.7 million (9M 2025). This heavy investment reflects the ongoing, high-stakes work required to move gene therapies through the clinic.

General and Administrative (G&A) costs, which cover the overhead to run the business, totaled $28.2 million (9M 2025). Honestly, G&A is a key area where management has focused on efficiencies; for instance, Q3 2025 G&A remained stable at $8.1 million compared to the prior year, reflecting disciplined expense management after a restructuring in the first half of 2025.

Here's a quick look at the major reported operating expenses for the nine-month period, though keep in mind the exact breakdown of clinical/personnel costs isn't fully itemized in the top-line releases:

Cost Category	Amount (9M 2025)	Context/Driver
Research and Development (R&D) Expenses	$98.7 million	Driven by clinical trials and gene therapy program costs.
General and Administrative (G&A) Expenses	$28.2 million	Reflects personnel and professional services spend.
Reimbursable R&D Services (Contra-Expense)	$6.2 million	Costs incurred for R&D services performed under collaboration agreements.

Costs for clinical trial execution and manufacturing of drug substance are embedded within that large R&D figure. Specifically, the increase in Q3 2025 R&D expenses was primarily due to increased spend related to the VY7523 multiple ascending dose (MAD) clinical trial for Alzheimer's disease and ongoing costs related to the VY1706 tau silencing gene therapy program.

Personnel costs for specialized scientific and management teams are a significant driver of both R&D and G&A. We know employee-related costs were cited as a factor in G&A fluctuations, such as the slight increase seen in Q2 2025 G&A expenses. You'd expect the bulk of the scientific team's compensation to fall under R&D, supporting programs like the new Voyager NeuroShuttle discovery platform.

Intellectual property maintenance and licensing fees are structured as both an outflow and a potential future inflow. Voyager Therapeutics enters into license and option agreements where they license intellectual property, which involves costs for maintenance and performance obligations. On the revenue side, which offsets costs, the company has potential milestone payments totaling up to $500 million from the Transition Bio collaboration, and up to $2.6 billion in total development milestone payments from existing partnerships not assumed in the current cash runway guidance.

• VY7523 MAD trial spend is a current clinical execution cost.
• IND-enabling studies for VY1706 are ongoing, preceding clinical trial initiation expected in 2026.
• The company expects to submit INDs for the Neurocrine-partnered FA and GBA1 programs by the end of 2025.
• The cost structure is designed to support operations into 2028 based on current plans.

Finance: draft 13-week cash view by Friday.

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Revenue Streams

For Voyager Therapeutics, Inc. (VYGR), the revenue streams are heavily weighted toward non-dilutive capital generated through strategic alliances, which is typical for a clinical-stage biotechnology firm advancing novel gene therapy and small molecule programs. These streams are designed to fund operations until a product potentially reaches commercialization, at which point royalties would become a major component.

The primary recognized revenue source is from existing partnerships, which includes both fixed payments and reimbursements for joint research activities. For the nine months ended September 30, 2025, the GAAP collaboration revenue totaled approximately $25.038 million.

This collaboration revenue can be broken down, showing the components recognized under GAAP for the nine months ended September 30, 2025:

Revenue Component	Amount (Nine Months Ended Sept 30, 2025, in thousands)
GAAP Collaboration Revenue	$25,038
Revenue Recognized for Reimbursed R&D Services	$6,229
Net Collaboration Revenue (GAAP less Reimbursement)	$18,809

Specifically for the third quarter of 2025, the GAAP collaboration revenue was reported as $13.4 million, a decrease from $24.6 million in the third quarter of 2024, largely due to the recognition timing of a prior Novartis amendment fee.

Beyond recognized revenue, the financial structure relies significantly on future, contingent payments:

• Upfront payments from new licensing and collaboration deals.
• Development and regulatory milestone payments, with the total potential across the partnered portfolio being up to $6.8 billion, which includes up to $2.4 billion in potential development milestone payments.
• Potential future royalties on net sales of commercialized products.

You should note that new deals, like the one with Transition Bio for TDP-43 targeting small molecules, contribute to the upfront and milestone potential; that specific deal offers an option to license for a 'single-digit million-dollar upfront payment' and potential milestones totaling up to $500 million, plus tiered royalties.

Also, reimbursement for R&D services performed for partners is a recurring, though variable, component of the collaboration revenue line. For the three months ended September 30, 2025, this reimbursement component within collaboration revenue was $2.2 million.

Finance: draft 13-week cash view by Friday.