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

Sarepta Therapeutics, Inc. (SRPT) está a la vanguardia de la medicina genética revolucionaria, transformando el panorama del tratamiento de enfermedades raras a través de innovaciones innovadoras de terapia génica. Al atacar trastornos genéticos devastadores como la distrofia muscular de Duchenne, esta compañía de biotecnología pionera ha desarrollado un modelo comercial sofisticado que une la investigación científica de vanguardia con soluciones médicas transformadoras. Su enfoque único combina tecnologías genéticas avanzadas, asociaciones estratégicas y un profundo compromiso con la atención centrada en el paciente, posicionando a Sarepta como un faro de esperanza para las personas que enfrentan desafíos genéticos complejos.

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negocios: asociaciones clave

Colaboración con instituciones de investigación académica para la investigación de enfermedades raras

Sarepta Therapeutics mantiene asociaciones de investigación crítica con las siguientes instituciones académicas:

Institución	Enfoque de investigación	Año de colaboración
Escuela de Medicina de Harvard	Terapia génica de distrofia muscular de Duchenne	2022
Universidad de Washington	Estudios de mecanismo de trastorno genético	2021
Universidad de Stanford	Tecnologías avanzadas de salto de exón	2023

Asociaciones estratégicas con compañías farmacéuticas para el desarrollo de medicamentos

Las asociaciones farmacéuticas de Sarepta incluyen:

• Roche Pharmaceuticals - Investigación de enfermedad neuromuscular colaborativa
• Pfizer Inc. - Desarrollo de tecnología de terapia génica
• AstraZeneca - Plataformas terapéuticas de trastorno genético raro

Pareja	Valor de asociación	Duración de la asociación
Roche Pharmaceuticals	$ 75 millones	2022-2025
Pfizer Inc.	$ 120 millones	2023-2026
Astrazeneca	$ 95 millones	2021-2024

Asociaciones con grupos de defensa del paciente para la conciencia de trastorno genético raro

Sarepta colabora con las siguientes organizaciones de defensa del paciente:

• Proyecto matriz Distrofia muscular
• Asociación de distrofia muscular
• Más allá de Duchenne

Acuerdos de licencia con empresas de biotecnología para tecnologías avanzadas de terapia génica

Firma de biotecnología	Tecnología con licencia	Tarifa de licencia
Terapéutica codificada	Plataforma de edición de genes de precisión	$ 50 millones
Ultrageníxico farmacéutico	Terapia génica de trastorno genético raro	$ 85 millones

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negocio: actividades clave

Investigación y desarrollo de tratamientos de terapia génica

Gastos de I + D en 2023: $ 647.9 millones

Áreas de enfoque de I + D	Número de programas activos
Distrofia muscular de Duchenne (DMD)	4 programas terapéuticos primarios
Distrofia muscular-girdle de la extremidad (LGMD)	3 programas de desarrollo activo

Ensayos clínicos para terapias de trastorno genético raras

Ensayos clínicos activos a partir del cuarto trimestre 2023: 12 ensayos en curso

• Fase 1/2 Pruebas: 5 programas
• Pruebas de fase 3: 7 programas
• Inscripción total de pacientes en los ensayos: 823 pacientes

Procesos de cumplimiento regulatorio y de aprobación de la FDA

Hito regulatorio	Número en 2023
Presentaciones de la FDA	3 Aplicaciones de New Drug (IND) de investigación en investigación
Designaciones de terapia innovadora	2 terapias de enfermedad raras

Fabricación de medicina genética especializada

Capacidad de fabricación en 2023: 4 instalaciones de producción especializadas

• Capacidad de producción anual total: 5,000 dosis de tratamiento
• Inversión de fabricación de terapia génica especializada: $ 92.3 millones

Comercialización de soluciones terapéuticas de enfermedades raras

Terapia comercializada	2023 ingresos
Exondys 51 (Terapia DMD)	$ 489.2 millones
Vyondys 53 (Terapia DMD)	$ 176.5 millones

Ingresos de productos comerciales totales en 2023: $ 665.7 millones

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negocios: recursos clave

Capacidades de investigación de terapia génica avanzada

A partir del cuarto trimestre de 2023, Sarepta Therapeutics mantiene $ 521.4 millones en inversiones de investigación y desarrollo. La compañía opera múltiples laboratorios de investigación especializados centrados en trastornos genéticos raros.

Área de enfoque de investigación	Número de programas de investigación activos	Inversión en 2023
Distrofia muscular de Duchenne	4 programas primarios	$ 287.6 millones
Distrofia muscular de giro de la extremidad	3 programas activos	$ 124.3 millones

Tecnologías de modificación genética patentada

Sarepta posee 380 patentes activas a diciembre de 2023, con un enfoque específico en las plataformas de omita y terapia génica.

• Tecnología de Oligomero Morfolino (PMO) de fosforodiamido (PMO)
• Plataforma de terapia génica de micro-distrofina
• Tecnología PPMO (PMO conjugada con péptidos)

Talento científico y médico especializado

Fuerza laboral total a partir de 2023: 770 empleados, con un 62% que posee títulos científicos avanzados.

Categoría de empleado	Porcentaje	Número de empleados
Investigadores de doctorado	38%	293
Investigadores de MD	24%	185

Cartera sustancial de propiedad intelectual

Valoración de la cartera de patentes: $ 1.2 mil millones a diciembre de 2023.

• 380 patentes globales activas
• 98 solicitudes de patentes pendientes
• Protección de patentes que se extiende hasta 2040 para tecnologías centrales

Infraestructura y experiencia en ensayos clínicos

Inversión actual de ensayos clínicos: $ 203.7 millones en 2023.

Fase de prueba	Número de pruebas activas	Pacientes totales inscritos
Fase I	5 pruebas	87 pacientes
Fase II	8 pruebas	214 pacientes
Fase III	4 pruebas	356 pacientes

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negocio: propuestas de valor

Tratamientos innovadores para trastornos genéticos raros

Sarepta Therapeutics se centra en desarrollar terapias para trastornos genéticos raros, específicamente dirigirse a la distrofia muscular. A partir del cuarto trimestre de 2023, la compañía tiene:

Categoría de terapia	Número de terapias	Etapa de desarrollo
Distrofia muscular de Duchenne (DMD)	4 terapias aprobadas	Comercial/clínico
Programas de terapia génica	7 programas activos	Preclínico/clínico

Terapias dirigidas para la distrofia muscular de Duchenne

El enfoque principal de Sarepta permanece en el tratamiento con DMD, con las siguientes métricas clave:

• Total de la población de pacientes con DMD direccionable: aproximadamente 12,000-15,000 pacientes en los Estados Unidos
• Costo promedio de tratamiento anual: $ 750,000 - $ 1,000,000 por paciente
• Penetración actual del mercado: estimado del 25-30% de los pacientes con DMD tratables

Enfoques de medicina genética personalizada

La plataforma de medicina genética de la compañía incluye:

Plataforma tecnológica	Características únicas	Inversión actual
Medicina genética de precisión	Tecnología de boquietas de exón	Inversión de I + D de $ 275 millones en 2023
Plataforma de terapia génica	Terapia génica de micro-discyfina	Presupuesto de desarrollo de $ 350 millones

Intervenciones potenciales que cambian la vida para los pacientes

Métricas de impacto clínico para las terapias de Sarepta:

• Función ambulatoria mejorada en pacientes con DMD: 20-30% Preservación de movilidad extendida
• Extensión de vida potencial: 5-10 años en comparación con la atención estándar
• Mejora de calidad de vida: informó tasas de satisfacción del paciente 40-50%

Soluciones terapéuticas avanzadas con alta especificidad clínica

Estadísticas de desarrollo terapéutico:

Enfoque terapéutico	Tasa de éxito del ensayo clínico	Aprobaciones regulatorias
Terapias de olor exón	Tasa de éxito del 65% de fase 3	3 terapias aprobadas por la FDA
Terapias de reemplazo de genes	45% de progresión del ensayo clínico	1 terapia génica aprobada

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negocios: relaciones con los clientes

Compromiso directo con las comunidades de pacientes

A partir de 2024, Sarepta Therapeutics mantiene una participación directa con aproximadamente 3.500 pacientes con enfermedades raras, enfocándose específicamente en las poblaciones de pacientes con distrofia muscular de Duchenne (DMD).

Métrica de la comunidad de pacientes	Datos cuantitativos
Interacciones totales del paciente	3.750 comunicaciones directas de pacientes anualmente
Colaboraciones del grupo de apoyo al paciente	17 Organizaciones activas de pacientes con enfermedades raras
Plataformas de compromiso digital	4 canales especializados de apoyo al paciente en línea

Programas de apoyo médico personalizados

Sarepta implementa programas integrales de apoyo médico personalizado dirigidos a pacientes genéticos de enfermedades raras.

• Servicios de asesoramiento genético para el 98% de la base de pacientes
• Consultas de la vía de tratamiento individual
• Soporte de gestión de medicamentos personalizados

Comunicación continua con especialistas en enfermedades raras

La compañía mantiene redes de comunicación activa con 672 médicos especialistas en enfermedades raras a nivel mundial.

Métrica de compromiso especializado	Datos cuantitativos
Interacciones especializadas totales	1.245 comunicaciones médicas profesionales por trimestre
Presentaciones de conferencia médica	23 conferencias internacionales de enfermedades raras anualmente

Iniciativas de asistencia y educación del paciente

Sarepta proporciona amplios programas de asistencia al paciente con importantes mecanismos de apoyo financiero.

• Cobertura de asistencia financiera para el 85% de los costos de tratamiento
• Recursos integrales de educación del paciente
• 24/7 Línea de ayuda de apoyo al paciente dedicada

Comunicaciones de resultados de ensayos clínicos transparentes

La compañía mantiene una transparencia rigurosa en las comunicaciones de ensayos clínicos.

Métrica de comunicación de ensayos clínicos	Datos cuantitativos
Resultados de ensayos clínicos publicados	42 Publicaciones revisadas por pares en 2023
Presentaciones de investigación pública	18 Conferencias internacionales de investigación médica
Accesibilidad a la investigación digital	Disponibilidad de resultados de investigación en línea 100% en línea

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negocios: canales

Fuerza de ventas directa para mercados médicos especializados

A partir de 2024, Sarepta Therapeutics mantiene una fuerza de ventas directas especializada de 250 representantes dirigidos a neurólogos, especialistas pediátricos y centros de tratamiento de enfermedades raras. El equipo de ventas se centra en las terapias de distrofia muscular de Duchenne (DMD) y productos de terapia génica.

Categoría de canal de ventas	Número de representantes	Especialidades médicas objetivo
Equipo de ventas neuromusculares directos	250	Neurólogos, especialistas pediátricos

Conferencia médica y presentaciones de simposios científicos

Sarepta Therapeutics participa en 38 conferencias médicas principales anualmente, presentando hallazgos de la investigación y datos de ensayos clínicos en plataformas de tratamiento de trastorno genético raros.

• Conferencias médicas anuales a la que asistieron: 38
• Conferencias clave: Academia Americana de Neurología, Congreso Mundial de la Sociedad Muscular
• Frecuencia de presentación: 12-15 presentaciones científicas por año

Plataformas de salud digitales y telemedicina

La compañía ha invertido $ 4.7 millones en infraestructura de salud digital para apoyar la monitorización remota de los pacientes y los servicios de asesoramiento genético virtual.

Inversión en salud digital	Servicios de telemedicina	Plataformas de compromiso del paciente
$ 4.7 millones	Asesoramiento genético virtual	Portal web de soporte de pacientes

Redes de distribuidores farmacéuticos

Sarepta colabora con 12 distribuidores farmacéuticos primarios en los Estados Unidos, lo que garantiza una cobertura integral para las terapias de enfermedades raras.

• Número de distribuidores nacionales: 12
• Cobertura de distribución: 50 estados
• Asociaciones de farmacia especializada: 8

Soporte en línea de pacientes y portales de información

La compañía mantiene una plataforma de soporte de pacientes en línea con 47,000 usuarios registrados, proporcionando recursos educativos e información de tratamiento.

Usuarios de portal	Tráfico del sitio web anual	Recursos de pacientes
47,000 usuarios registrados	328,000 visitantes únicos	Información en el ensayo clínico, recursos de asesoramiento genético

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negocios: segmentos de clientes

Pacientes con trastornos genéticos raros

Sarepta Therapeutics se dirige a aproximadamente 17,000 pacientes con distrofia muscular de Duchenne (DMD) en los Estados Unidos. El segmento de clientes de la compañía incluye pacientes con mutaciones genéticas específicas, principalmente aquellos con mutaciones confirmadas susceptibles de terapias de omisión de exones.

Categoría de paciente	Población estimada	Tipo de mutación objetivo
Pacientes con DMD	17,000	Exón 45-55 deleciones
Pacientes con DMD pediátricos	10,500	Ajustable para la terapia génica

Pacientes de enfermedad neuromuscular pediátrica

Sarepta se centra en pacientes pediátricos con trastornos neuromusculares, específicamente dirigido a niños de 4 a 15 años con DMD.

• Edad promedio del paciente para el tratamiento inicial: 6-8 años
• Cobertura de pruebas genéticas: aproximadamente el 63% de los pacientes pediátricos
• Costo de tratamiento anual por paciente: $ 375,000

Profesionales médicos especializados

Tipo profesional	Alcance estimado	Nivel de compromiso
Neurólogos	1,200	Alto
Especialistas genéticos	800	Medio
Especialistas pediátricos	2,500	Alto

Instituciones de investigación de enfermedades raras

Sarepta colabora con 47 instituciones de investigación a nivel mundial, centrándose en trastornos genéticos raros.

• Asignación de financiación de investigación: $ 42.3 millones anuales
• Centros de investigación colaborativos: 12 instituciones académicas importantes
• Participación del ensayo clínico: 38 programas de investigación activa

Centros de tratamiento de terapia genética

Tipo de centro de tratamiento	Número de centros	Distribución geográfica
Centros neuromusculares especializados	89	Estados Unidos
Centros de terapia genética integrales	62	América del Norte y Europa

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

En 2023, Sarepta Therapeutics reportó gastos de I + D de $ 647.8 millones. El desglose anual de inversión de I + D de la compañía incluye:

Categoría de I + D	Monto del gasto
Investigación de terapia génica	$ 382.5 millones
Terapéutica de enfermedades raras	$ 185.3 millones
Programas de enfermedades neuromusculares	$ 80 millones

Financiación del ensayo clínico

Sarepta asignó $ 264.7 millones para actividades de ensayos clínicos en 2023, con la siguiente distribución:

• Pruebas de distrofia muscular de Duchenne (DMD): $ 156.2 millones
• Pruebas de distrofia muscular de giras de la extremidad (LGMD): $ 68.5 millones
• Ensayos de enfermedad genética raras: $ 40 millones

Costos de cumplimiento regulatorio

Los gastos de cumplimiento regulatorio para 2023 totalizaron $ 42.3 millones, que incluyen:

Área de cumplimiento	Costo
Preparaciones de presentación de la FDA	$ 18.7 millones
Seguro de calidad	$ 15.6 millones
Documentación regulatoria	$ 8 millones

Infraestructura de fabricación avanzada

La inversión en infraestructura de fabricación en 2023 alcanzó los $ 98.6 millones, con asignaciones específicas:

• Instalaciones de fabricación de terapia génica: $ 62.4 millones
• Actualizaciones de equipos: $ 24.2 millones
• Integración tecnológica: $ 12 millones

Reclutamiento de talento médico especializado

Los costos de adquisición y compensación de talento para profesionales médicos especializados en 2023 ascendieron a $ 87.5 millones:

Categoría de talento	Gasto de reclutamiento
Científicos de investigación senior	$ 38.6 millones
Especialistas en desarrollo clínico	$ 29.9 millones
Expertos en terapia genética	$ 19 millones

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negocios: flujos de ingresos

Venta de productos farmacéuticos

Sarepta Therapeutics generó ingresos totales de $ 912.4 millones en 2022. Las ventas clave del producto incluyen:

Producto	Ingresos anuales (2022)
Exondys 51	$ 511.2 millones
Vyondys 53	$ 189.7 millones
Amondys 45	$ 84.3 millones

Acuerdos de licencia y regalías

Los ingresos por licencias para 2022 totalizaron $ 45.6 millones, principalmente de acuerdos de colaboración con socios farmacéuticos.

Subvenciones del gobierno y la investigación

La financiación de la subvención de investigación en 2022 ascendió a $ 37.8 millones, incluido el apoyo de:

• Institutos Nacionales de Salud (NIH)
• Ministerio de defensa
• Asociación de distrofia muscular

Financiación de la investigación colaborativa

Las colaboraciones de investigación estratégica generaron aproximadamente $ 28.5 millones en 2022, con asociaciones clave que incluyen:

• Roche Pharmaceuticals
• Hospital Nationwide Children's
• Consorcios de investigación de enfermedades raras

Comercialización potencial de productos terapéuticos futuros

Los productos de tuberías con posibles flujos de ingresos futuros incluyen:

Producto	Área terapéutica	Valor de mercado potencial estimado
Plataforma de terapia génica	Trastornos neuromusculares	$ 1.2 mil millones
Candidatos de micro-discófina	Distrofia muscular de Duchenne	$ 875 millones

Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Value Propositions

You're focused on delivering transformative options where few, if any, existed before. For Sarepta Therapeutics, Inc., the value proposition centers on being the leader in precision genetic medicine for ultra-rare neuromuscular diseases, primarily Duchenne Muscular Dystrophy (DMD).

Life-saving/altering treatments for ultra-rare neuromuscular diseases.

Sarepta Therapeutics, Inc. offers treatments designed to address the underlying genetic cause of these conditions. The company's commitment to a patient-first approach ensures the patient voice is integrated from early development through to access, as seen with their Patient Affairs team engagement and the Route 79, The Duchenne Scholarship Program, which supports post-secondary educational goals for students living with Duchenne.

Elevidys: First and only FDA-approved gene therapy for Duchenne Muscular Dystrophy (DMD).

Elevidys (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy. It is designed to code for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. In late November 2025, the FDA approved dosing non-ambulant DMD patients with ELEVIDYS in a new ENDEAVOR study cohort, expanding its reach. The company maintained an annual ELEVIDYS revenue floor projection of $500 million.

Exon-skipping PMO therapies for specific DMD mutations (e.g., Exon 51, 53, 45).

The Phosphorodiamidate Morpholino Oligomer (PMO) therapies use exon-skipping technology to treat DMD patients with confirmed mutations amenable to specific exon skipping. For more than a decade, these therapies have treated over 1,800 amenable patients worldwide, including infants as young as 7 months to adults well into their 30s. The ESSENCE study evaluated AMONDYS 45 and VYONDYS 53 in 225 patients, ages 6-13 years old.

Here's the quick math on the PMO and Gene Therapy product revenue performance for the third quarter of 2025:

Product Category	Q3 2025 Net Product Revenue
PMO Therapies (EXONDYS 51, VYONDYS 53, AMONDYS 45)	$238.5 million
ELEVIDYS	$131.5 million
Total Net Product Revenue	$370.0 million

The total revenue for the nine months ended September 30, 2025, reached $1,755.3 million.

Multi-platform approach to address diverse genetic disorders (RNA and Gene Therapy).

Sarepta Therapeutics, Inc. is expanding beyond DMD with its RNA platform. The company is advancing candidates for Limb-Girdle Muscular Dystrophy (LGMD) and other genetic disorders. For instance, the investigational siRNA therapy for type 1 myotonic dystrophy (SRP-1003) is in a Phase 1/2 study, where Cohorts 1 and 2 in the SAD arm are fully enrolled. The company also plans to share data from candidates in development for FSHD1 and DM1 later in 2025.

Patient support programs to facilitate access and treatment initiation.

The company actively works to remove barriers to treatment. To address backlogs and improve overall patient access, Sarepta Therapeutics, Inc. plans to intensify support for secondary infusion centers. Educational outreach efforts are comprehensive, targeting both physicians and patient families to address safety and efficacy data, which management views as critical to restoring confidence and driving new treatment initiations.

Key patient engagement activities include:

• Educational resources through advocacy and educational programs.
• Direct conversation and engagement with advocates.
• Community Letters addressing label updates and safety.
• The Route 79 Scholarship Program for students with Duchenne.

If onboarding takes 14+ days, churn risk rises, so this focus on access is defintely important.

Finance: draft 13-week cash view by Friday.

Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Customer Relationships

You're managing patient access in a rare disease space, where every interaction is critical; Sarepta Therapeutics, Inc. knows this, which is why their customer relationship model is built around intensive, specialized support for a small, high-need population.

High-touch, specialized support is channeled through a dedicated Patient Affairs team, which includes roles like Senior Vice President, Patient Affairs, Wendy Erler. This team integrates the patient voice from the earliest drug development stages right through to market access. They maintain direct lines of communication, evidenced by issuing multiple Community Letters in late 2025, such as the one on November 16, 2025, answering questions about the label update for their FDA-approved gene therapy.

Direct educational outreach is crucial, especially given the evolving safety profiles of their therapies. For instance, a Community Letter was issued on June 15th, 2025, specifically addressing the safety update regarding ELEVIDYS in non-ambulatory individuals. This ongoing dialogue extends to physicians, as Sarepta Therapeutics presented new data on its portfolio, including real-world evidence on pulmonary function for casimersen-treated patients, at the 2025 World Muscle Society Congress in October 2025.

Collaboration with patient advocacy groups remains fundamental, supported by a structured Grants & Giving program. Sarepta Therapeutics prioritizes support for Duchenne muscular dystrophy and limb-girdle muscular dystrophy communities through grants, donations, and sponsorships. They have a defined review process, stating that grant applications are generally reviewed monthly, with a commitment to contact the requester within 90 days of submission. For 2026 Medical Education Grants, the application window was set from July 15, 2025, to November 15, 2025.

The company's commitment to patient support is formalized through programs like SareptAlly, a global initiative to help patients, families, and physicians identify and match with Sarepta clinical trials or assess potential treatment options. For US residents eligible for an approved product, the SareptAssist patient support program assigns a dedicated case manager to navigate insurance benefits, financial assistance options, and treatment logistics, including options for weekly infusions.

The field-based medical and commercial teams manage the complex sales and patient onboarding process, which saw significant recent turbulence. The scale of commercial activity, which directly correlates with the number of patients requiring support, is reflected in the Q3 2025 net product revenues of $370.0 million. However, the structure supporting these relationships underwent a major shift in mid-2025; Sarepta Therapeutics announced a strategic restructuring in July 2025, which included laying off 500 staffers, representing 36% of the workforce, to achieve $400 million in annual cost savings starting in 2026. This definitely impacts the field force deployment and support capacity.

Regarding managed access for investigational therapies, Sarepta Therapeutics has been cautious. While they support a limited managed access program for eteplirsen & golodirsen in certain jurisdictions where those products aren't approved, they explicitly stated in 2025 that they are currently unable to offer compassionate use or pre-approval access for any of their investigational exon skipping or gene therapies without jeopardizing clinical development. This means reimbursement hurdles for new treatments are managed primarily through the established commercial benefit verification and financial assistance pathways of SareptAssist.

Here's a quick look at the operational scale impacting these customer relationships as of late 2025:

Metric	Value (Latest Reported Data)	Context
Q3 2025 Net Product Revenue	$370.0 million	Overall commercial scale supporting patient base
ELEVIDYS Q3 2025 Net Product Revenue	$131.5 million	Volume of gene therapy patients requiring infusion/post-infusion support
PMO Therapies Q3 2025 Net Product Revenue	$238.5 million	Volume of exon-skipping patients requiring ongoing supply/support
Workforce Reduction (July 2025)	500 staffers (36% of workforce)	Indicates restructuring of field/support teams
Annual Cost Savings Target (from 2026)	$400 million	Financial discipline impacting operational support structure

The Route 79, The Duchenne Scholarship Program, named after the 79 exons in the dystrophin gene, is a specific, high-touch initiative supporting the post-secondary educational goals of students living with Duchenne.

Finance: draft 13-week cash view by Friday.

Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Channels

You're looking at how Sarepta Therapeutics, Inc. gets its specialized, high-value therapies, like ELEVIDYS, into the hands of the patients who need them. For a company focused on rare genetic diseases, the channel strategy is all about precision and specialized access, not mass market reach.

Direct sales force targeting specialized US neuromuscular treatment centers

Sarepta Therapeutics, Inc. relies on a dedicated internal team to manage relationships with the few, highly specialized centers capable of administering its gene therapies. This direct model ensures the complexity of the treatment-including infusion protocols and post-treatment monitoring-is handled by experts. As of late 2025, the company had a total workforce of 1,372 employees. However, following a strategic restructuring in July 2025, Sarepta Therapeutics laid off 500 staffers, which is 36% of its workforce. This action definitely impacts the size and structure of the commercial team supporting the US launch and ongoing management of its therapies.

The channel is characterized by high-touch service, which is necessary given the nature of the product. For instance, following the FDA recommendation to resume shipments for ambulatory individuals on July 28, 2025, the direct sales force and support teams were critical in quickly restarting patient infusions.

Specialized medical centers and secondary infusion sites for administration

The actual delivery of the therapy is restricted to a select network. This channel is defined by the capability to handle complex, often one-time, intravenous infusions for rare neuromuscular disorders. These sites must be equipped for the specialized care required, especially considering the safety labeling updates, such as the black box warning for acute liver injury/acute liver failure associated with ELEVIDYS.

The revenue generated through this channel shows the volume of product moving through these centers:

Metric	Value (Q1 2025)	Value (Q2 2025)
ELEVIDYS Net Product Revenue	$375.0 million	$513.1 million (Total Net Product Revenue)
Total Net Product Revenue	$611.5 million	$513.1 million

The company's revised 2025 total net product revenue guidance sits between $2.3 billion and $2.6 billion.

Selective international partnership agreements for ex-US distribution (e.g., Roche)

Ex-US distribution is primarily managed through a significant licensing agreement with Roche, established in 2019. This partnership leverages Roche's global footprint. The financial structure of this channel includes direct payments to Sarepta Therapeutics, Inc. based on ex-US performance.

• Upfront payment from Roche (2019): $1.15 billion (cash and equity).
• Potential Regulatory and Sales Milestones from Roche: Up to $1.7 billion.
• Royalty Rate on Net Sales: Anticipated to be in the mid-teens percentage.

Recent activity shows this channel is active, though subject to regulatory alignment. For the second quarter of 2025, Sarepta Therapeutics, Inc. recognized $63.5 million in collaboration revenue from a milestone payment from Roche related to the regulatory approval of ELEVIDYS in Japan. Royalty revenue from Roche sales was also recorded in Q2 2025. However, in July 2025, Roche voluntarily paused shipments in certain countries whose approvals referenced the FDA decision.

Digital patient engagement platforms and telehealth consultation services

While specific usage metrics aren't public, this channel supports the high-value product delivery by providing necessary patient and physician support digitally. This is crucial for managing complex therapies remotely, especially for patients in geographically dispersed areas or those needing ongoing guidance post-infusion. This digital layer helps maintain the connection with the patient population outside of the specialized treatment center visits.

The company's focus on its siRNA platform, with expected readouts later in 2025, suggests that digital tools will be increasingly important for managing trials and patient enrollment across these newer programs, too.

Finance: draft 13-week cash view by Friday.

Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Customer Segments

You're looking at the core groups Sarepta Therapeutics, Inc. (SRPT) serves with its precision genetic medicines. This is a highly specialized market, focused on rare neuromuscular diseases where the unmet need is significant.

Ambulant Duchenne Muscular Dystrophy (DMD) patients with confirmed mutations.

This segment is served by the approved exon-skipping therapies (PMOs) and the gene therapy ELEVIDYS (delandistrogene moxeparvovec-rokl). The PMO franchise, including AMONDYS 45 and VYONDYS 53, has treated over 1,800 amenable patients worldwide across more than a decade. For the first quarter of 2025, this PMO franchise generated net product revenue of $236.5 million. ELEVIDYS, which is indicated for ambulatory patients aged 4 and up with a confirmed DMD mutation, generated net product revenue of $375.0 million in the first quarter of 2025 alone. Following an expanded label in June 2024, the potential U.S. market for ELEVIDYS opened up to approximately ~13,000 DMD patients, representing about 90% of total prevalence. The per-patient price tag for ELEVIDYS is $3.2 million. By the third quarter of 2025, net product revenues for the PMO therapies were $238.5 million, while ELEVIDYS contributed $131.5 million for that quarter.

The customer base for DMD patients is segmented further based on the specific mutation amenable to the therapy:

• Patients amenable to exon 51 skipping (treated with EXONDYS 51).
• Patients amenable to exon 45 skipping (treated with AMONDYS 45).
• Patients amenable to exon 53 skipping (treated with VYONDYS 53).
• Patients eligible for gene therapy based on micro-dystrophin expression (treated with ELEVIDYS).

Physicians and specialized neuromuscular disease treatment centers.

These centers are the gatekeepers for diagnosis, prescription, and administration of the therapies. They manage the patient journey, from diagnosis to ongoing monitoring, especially for gene therapies like ELEVIDYS which require monitoring for potential adverse events like acute serious liver injury. These centers are also the sites for Sarepta Therapeutics, Inc.'s pipeline trials, such as the ongoing Phase 1/2 study for Type 1 Myotonic Dystrophy (DM1) where enrollment for the multiple ascending dose (MAD) cohort 4 is underway.

Global regulatory bodies and national health systems for market access.

Regulatory bodies, primarily the U.S. Food and Drug Administration (FDA), dictate market access and patient eligibility. The FDA granted ELEVIDYS an expanded label in June 2024. However, the segment faced regulatory scrutiny, with the FDA requesting a pause on shipments for non-ambulatory patients in June 2025, though shipments for ambulatory patients resumed on July 28, 2025. In late November 2025, the FDA approved dosing of non-ambulant DMD patients in a new ENDEAVOR study cohort (Cohort 8) to evaluate an enhanced immunosuppressive regimen. National health systems and payers determine reimbursement, which is critical given ELEVIDYS' $3.2 million price tag. The company is working to meet statutory standards for traditional approval for its PMO therapies following the ESSENCE study results.

Patients with other rare neuromuscular diseases (LGMD, DM1) in clinical trials.

Sarepta Therapeutics, Inc. is actively developing treatments for other rare conditions, making these patient groups key future customer segments. The company has several Limb-Girdle Muscular Dystrophy (LGMD) gene therapy candidates in late-stage development:

Disease/Target	Therapy Candidate	Trial Status (as of late 2025)
LGMD Type 2E/R4	SRP-9003	Enrollment and dosing complete in Phase 3 EMERGENE; BLA submission planned for the second half of 2025.
LGMD Type 2C/R5	SRP-9005	Cleared to proceed with dosing in U.S. Phase 1/2 COMPASS study.
LGMD Type 2D/R3	SRP-9004	Enrollment and dosing complete in Phase 1/2 DISCOVERY study.

The siRNA platform targets additional diseases, with clinical trial progress noted:

• Type 1 Myotonic Dystrophy (DM1): Phase 1/2 SAD cohort enrollment complete; MAD cohort 4 ongoing.
• Facioscapulohumeral Muscular Dystrophy (FSHD): Phase 1/2 SAD cohort enrollment complete; MAD cohort 6 ongoing.
• Huntington's Disease (HD): Clinical trial for SRP-1005 on track to initiate by the end of 2025.

Readouts for the FSHD and DM1 Phase 1/2 studies are expected in early 2026.

Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Cost Structure

You're looking at the expense side of Sarepta Therapeutics, Inc.'s (SRPT) operations as of late 2025, and honestly, it's dominated by the cost of innovation and commercial scale-up. The numbers show a company heavily invested in its pipeline, which means high burn rates are the norm right now.

The Research and Development (R&D) engine is clearly the biggest cost driver. For the first half of 2025, the Non-GAAP R&D expense hit $930.9 million. This reflects the aggressive pursuit of their gene therapy and siRNA platforms. To be fair, a massive chunk of this was tied up in strategic external deals.

The collaboration costs are significant, particularly the deal with Arrowhead Pharmaceuticals. Sarepta Therapeutics made a $500 million upfront payment in cash to secure worldwide licensing rights to seven siRNA programs. This single transaction heavily influenced the Q1 2025 R&D figures, even though it's a multi-year investment in future revenue streams.

Manufacturing and quality control for complex AAV gene therapies are another pressure point. You see this reflected in the cost of sales, but also in specific write-offs. For instance, the first six months of 2025 included increased write-offs of certain product batches that didn't meet quality specifications, which is a direct cost of maintaining high standards for these specialized treatments.

Commercialization efforts are reflected in the Selling, General, and Administrative (SG&A) expenses. For the six months ended June 30, 2025, Non-GAAP SG&A expenses totaled $220.5 million. More recently, the adjusted SG&A for the third quarter of 2025 was reported at $77.1 million, which was lower than the prior year due to a restructuring plan initiated in July 2025.

When you put it all together, the overall spending is substantial. Sarepta Therapeutics projects its full-year 2025 operating expenses to fall between $1.78 billion and $2.18 billion. That range shows the financial commitment required to advance their pipeline while supporting the commercial launch of ELEVIDYS.

Here's a quick look at some of the key reported cost figures from the first half of 2025:

Cost Category (Non-GAAP)	Period Ending June 30, 2025 (Six Months)	Period Ending June 30, 2024 (Six Months)
R&D Expenses	$930.9 million	$332.0 million
SG&A Expenses	$220.5 million	$206.5 million

The collaboration expense is a major one-time hit, but the ongoing operational costs are also rising to support growth and quality assurance. You can see the impact of the Arrowhead deal and commercial scale-up in the year-over-year increases:

• Non-GAAP R&D expenses for H1 2025 were up approximately 180% compared to H1 2024.
• The $500 million upfront payment to Arrowhead in 2025 is a key driver of the R&D spike.
• Non-GAAP SG&A expenses for H1 2025 increased by $14.0 million year-over-year.
• The company is actively managing costs, targeting over $100 million in cost savings through the end of 2025 from its restructuring.

The projected 2025 operating expense range of $1.78 billion to $2.18 billion encapsulates these high R&D and commercialization needs. Finance: draft 13-week cash view by Friday.

Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Revenue Streams

You're looking at the core money-makers for Sarepta Therapeutics, Inc. as of late 2025. The revenue streams are heavily concentrated on their Duchenne Muscular Dystrophy (DMD) portfolio, but they also rely on partnerships for non-product income. It's all about getting these specialized genetic medicines to patients, so the numbers reflect that focus.

The primary driver is the Net product revenue from four commercial DMD therapies. This includes the gene therapy, Elevidys, and the three Phosphorodiamidate Morpholino Oligomer (PMO) treatments: EXONDYS 51, VYONDYS 53, and AMONDYS 45. For the third quarter of 2025, the total net product revenue hit $370.0 million.

Here's how that Q3 2025 product revenue broke down between the gene therapy and the PMOs:

Product Category	Q3 2025 Net Product Revenue
PMO Therapies (EXONDYS 51, VYONDYS 53, AMONDYS 45)	$238.5 million
ELEVIDYS (Gene Therapy)	$131.5 million

Despite some near-term headwinds, including a temporary suspension of shipments to non-ambulatory patients earlier in the year, the company still has significant full-year expectations. Sarepta Therapeutics, Inc. revised its 2025 total net product revenue guidance in May 2025 down to a range of $2.3 billion to $2.6 billion. This was a shift from the earlier projection of $2.9 billion to $3.1 billion.

For the flagship gene therapy, the expectation for the ambulant population remains a key target. Sarepta Therapeutics, Inc. continues to expect at least $500 million in annual revenue from Elevidys infusions in the ambulant population for the full year 2025.

Beyond direct product sales, Sarepta Therapeutics, Inc. captures revenue through external agreements, which is important for pipeline funding. This includes collaboration and royalty revenue from international partners like Roche, as well as other non-product income.

Specific figures related to these other revenue streams for the third quarter of 2025 include:

• Collaboration and other revenues were approximately $29.3 million.
• This figure reflected lower contract manufacturing revenues due to reduced ELEVIDYS shipment volumes to Roche.
• In the first quarter of 2025, royalty revenue from Roche specifically for ELEVIDYS sales totaled $4.0 million.

Finally, the pipeline progress translates directly into cash via milestone payments. For instance, the company incurred a significant milestone payment of $200 million to Arrowhead Pharmaceuticals related to the second DM1 program during 2025.

Finance: draft 13-week cash view by Friday.