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

A Sarepta Therapeutics, Inc. (SRPT) fica na vanguarda da medicina genética revolucionária, transformando a paisagem do tratamento de doenças raras por meio de inovações inovadoras de terapia genética. Ao direcionar distúrbios genéticos devastadores como a distrofia muscular de Duchenne, esta empresa pioneira de biotecnologia desenvolveu um modelo de negócios sofisticado que preenche pesquisas científicas de ponta com soluções médicas transformadoras. Sua abordagem única combina tecnologias genéticas avançadas, parcerias estratégicas e um profundo compromisso com os cuidados centrados no paciente, posicionando Sarepta como um farol de esperança para indivíduos que enfrentam desafios genéticos complexos.

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negócios: Parcerias -chave

Colaboração com instituições de pesquisa acadêmica para pesquisa de doenças raras

Sarepta Therapeutics mantém parcerias críticas de pesquisa com as seguintes instituições acadêmicas:

Instituição	Foco na pesquisa	Ano de colaboração
Escola de Medicina de Harvard	Terapia genética de distrofia muscular de Duchenne	2022
Universidade de Washington	Estudos de mecanismo de transtorno genético	2021
Universidade de Stanford	Tecnologias avançadas de pular exon	2023

Parcerias estratégicas com empresas farmacêuticas para desenvolvimento de medicamentos

As parcerias farmacêuticas de Sarepta incluem:

• Roche Pharmaceuticals - Pesquisa colaborativa de doenças neuromusculares
• Pfizer Inc. - Desenvolvimento de tecnologia de terapia genética
• AstraZeneca - Plataformas terapêuticas de transtorno genético raro

Parceiro	Valor da parceria	Duração da parceria
Roche Pharmaceuticals	US $ 75 milhões	2022-2025
Pfizer Inc.	US $ 120 milhões	2023-2026
AstraZeneca	US $ 95 milhões	2021-2024

Parcerias com grupos de defesa de pacientes para conscientização sobre transtorno genético raro

Sarepta colabora com as seguintes organizações de defesa de pacientes:

• Distrofia muscular do projeto pai
• Associação de Distrofia Muscular
• Além de Duchenne

Acordos de licenciamento com empresas de biotecnologia para tecnologias avançadas de terapia genética

Empresa de biotecnologia	Tecnologia licenciada	Taxa de licenciamento
Terapêutica codificada	Plataforma de edição de genes de precisão	US $ 50 milhões
Ultragenyx Pharmaceutical	Terapia genética genética rara terapia	US $ 85 milhões

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento de tratamentos de terapia genética

Despesas de P&D em 2023: US $ 647,9 milhões

Áreas de foco em P&D	Número de programas ativos
Distrofia muscular de Duchenne (DMD)	4 programas terapêuticos primários
Distrofia muscular-da-cora (LGMD)	3 programas de desenvolvimento ativo

Ensaios clínicos para terapias de transtorno genético raras

Ensaios clínicos ativos a partir do quarto trimestre 2023: 12 ensaios em andamento

• Fase 1/2 ensaios: 5 programas
• Ensaios de Fase 3: 7 Programas
• Total de matrícula de pacientes em ensaios: 823 pacientes

Processos de conformidade regulatória e aprovação da FDA

Marco regulatório	Número em 2023
Submissões da FDA	3 Aplicações de medicamentos para investigação (IND)
Designações de terapia inovadora	2 terapias de doenças raras

Fabricação de medicina genética especializada

Capacidade de fabricação em 2023: 4 instalações de produção especializadas

• Capacidade anual total de produção: 5.000 doses de tratamento
• Investimento especializado em terapia genética: US $ 92,3 milhões

Comercialização de soluções terapêuticas de doenças raras

Terapia comercializada	2023 Receita
Exondys 51 (terapia DMD)	US $ 489,2 milhões
Vyondys 53 (terapia DMD)	US $ 176,5 milhões

Receita total de produtos comerciais em 2023: US $ 665,7 milhões

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negócios: Recursos -chave

Capacidades avançadas de pesquisa de terapia genética

A partir do quarto trimestre de 2023, a Sarepta Therapeutics mantém US $ 521,4 milhões em investimentos em pesquisa e desenvolvimento. A empresa opera vários laboratórios de pesquisa especializados focados em distúrbios genéticos raros.

Área de foco de pesquisa	Número de programas de pesquisa ativos	Investimento em 2023
Distrofia muscular de Duchenne	4 programas primários	US $ 287,6 milhões
Distrofia muscular-da-coroa	3 programas ativos	US $ 124,3 milhões

Tecnologias de modificação genética proprietária

A Sarepta possui 380 patentes ativas em dezembro de 2023, com foco específico nas plataformas de pilotos de exon e terapia genética.

• Proprietário Fosforodiamidato Morpholino Oligomer (PMO) Technology Technology
• Plataforma de terapia genética de micro-distrofina
• Tecnologia PPMO (PMO conjugada com peptídeos)

Talento científico e médico especializado

A força de trabalho total em 2023: 770 funcionários, com 62% dando diplomas científicos avançados.

Categoria de funcionários	Percentagem	Número de funcionários
Pesquisadores de doutorado	38%	293
Pesquisadores de MD	24%	185

Portfólio de propriedade intelectual substancial

Avaliação do portfólio de patentes: US $ 1,2 bilhão em dezembro de 2023.

• 380 patentes globais ativas
• 98 pedidos de patente pendente
• Proteção de patentes que se estende até 2040 para tecnologias principais

Infraestrutura e experiência em ensaios clínicos

Investimento atual do ensaio clínico: US $ 203,7 milhões em 2023.

Fase de teste	Número de ensaios ativos	Pacientes totais inscritos
Fase I.	5 ensaios	87 pacientes
Fase II	8 ensaios	214 pacientes
Fase III	4 ensaios	356 pacientes

Sarepta Therapeutics, Inc. (SRPT) - Modelo de Negócios: Proposições de Valor

Tratamentos inovadores para distúrbios genéticos raros

Sarepta Therapeutics se concentra no desenvolvimento de terapias para distúrbios genéticos raros, direcionando especificamente a distrofia muscular. A partir do quarto trimestre 2023, a empresa possui:

Categoria de terapia	Número de terapias	Estágio de desenvolvimento
Distrofia muscular de Duchenne (DMD)	4 terapias aprovadas	Comercial/Clínico
Programas de terapia genética	7 programas ativos	Pré -clínico/clínico

Terapias direcionadas para a distrofia muscular de Duchenne

O foco principal de Sarepta permanece no tratamento da DMD, com as seguintes métricas -chave:

• População total de pacientes com DMD endereçável: aproximadamente 12.000 a 15.000 pacientes nos Estados Unidos
• Custo médio de tratamento anual: US $ 750.000 - US $ 1.000.000 por paciente
• Penetração atual de mercado: estimado 25-30% dos pacientes tratáveis ​​com DMD

Abordagens de medicina genética personalizada

A plataforma de medicina genética da empresa inclui:

Plataforma de tecnologia	Características únicas	Investimento atual
Medicina genética de precisão	Tecnologia de esgotamento do exon	US $ 275 milhões em investimento em P&D em 2023
Plataforma de terapia genética	Terapia genética de micro-distrofina	Orçamento de desenvolvimento de US $ 350 milhões

Potenciais intervenções que mudam a vida para os pacientes

Métricas de impacto clínico para terapias de Sarepta:

• Função ambulatorial aprimorada em pacientes com DMD: preservação de mobilidade prolongada de 20 a 30%
• Extensão potencial de vida: 5-10 anos em comparação com os cuidados padrão
• Melhoria da qualidade de vida: relatados 40-50% das taxas de satisfação do paciente

Soluções terapêuticas avançadas com alta especificidade clínica

Estatísticas de desenvolvimento terapêutico:

Abordagem terapêutica	Taxa de sucesso do ensaio clínico	Aprovações regulatórias
Terapias de esgotamento do exon	Taxa de sucesso de 65% da fase 3	3 terapias aprovadas pela FDA
Terapias de reposição de genes	45% de progressão do ensaio clínico	1 terapia genética aprovada

Sarepta Therapeutics, Inc. (SRPT) - Modelo de Negócios: Relacionamentos do Cliente

Engajamento direto com comunidades de pacientes

A partir de 2024, a Sarepta Therapeutics mantém o envolvimento direto com aproximadamente 3.500 pacientes com doenças raras, concentrando -se especificamente nas populações de pacientes com distrofia muscular de Duchenne (DMD).

Métrica da comunidade de pacientes	Dados quantitativos
Interações totais do paciente	3.750 comunicações diretas do paciente anualmente
Colaborações em grupo de apoio ao paciente	17 Organizações de pacientes de doenças raras ativas
Plataformas de engajamento digital	4 canais especializados de apoio ao paciente online

Programas de apoio médico personalizado

Sarepta implementa programas abrangentes de apoio médico personalizado direcionados a pacientes genéticos de doenças raras.

• Serviços de aconselhamento genético para 98% da base de pacientes
• Consultas de caminho individual
• Suporte personalizado de gerenciamento de medicamentos

Comunicação contínua com especialistas em doenças raras

A empresa mantém redes de comunicação ativa com 672 médicos especializados em doenças raras globalmente.

Métrica de engajamento especializada	Dados quantitativos
Interações especializadas totais	1.245 comunicações profissionais médicas por trimestre
Apresentações da conferência médica	23 Conferências internacionais de doenças raras anualmente

Iniciativas de assistência e educação do paciente

Sarepta fornece programas de assistência ao paciente extensos com mecanismos significativos de apoio financeiro.

• Cobertura de assistência financeira para 85% dos custos de tratamento
• Recursos abrangentes para educação do paciente
• 24/7 de linha de apoio ao paciente dedicado

Comunicações do resultado do ensaio clínico transparentes

A empresa mantém uma transparência rigorosa nas comunicações de ensaios clínicos.

Métrica de comunicação de ensaios clínicos	Dados quantitativos
Resultados de ensaios clínicos publicados	42 publicações revisadas por pares em 2023
Apresentações de pesquisa pública	18 Conferências Internacionais de Pesquisa Médica
Acessibilidade à pesquisa digital	Disponibilidade de resultado de pesquisa on -line 100%

Sarepta Therapeutics, Inc. (SRPT) - Modelo de Negócios: Canais

Força de vendas direta para mercados médicos especializados

Em 2024, a Sarepta Therapeutics mantém uma força de vendas direta especializada de 250 representantes direcionados a neurologistas, especialistas pediátricos e centros de tratamento de doenças raras. A equipe de vendas concentra -se nas terapias de distrofia muscular de Duchenne (DMD) e produtos de terapia genética.

Categoria de canal de vendas	Número de representantes	Especialidades médicas -alvo
Equipe direta de vendas neuromusculares	250	Neurologistas, especialistas pediátricos

Apresentações de Conferência Médica e Simpósio Científico

A Sarepta Therapeutics participa de 38 principais conferências médicas anualmente, apresentando resultados de pesquisas e dados de ensaios clínicos em plataformas de tratamento de transtornos genéticos raros.

• Conferências médicas anuais comparecidas: 38
• Conferências -chave: American Academy of Neurology, World Muscle Society Congress
• Frequência de apresentação: 12-15 apresentações científicas por ano

Plataformas de saúde digital e telemedicina

A empresa investiu US $ 4,7 milhões em infraestrutura de saúde digital para apoiar os serviços remotos de monitoramento de pacientes e aconselhamento genético virtual.

Investimento em saúde digital	Serviços de telemedicina	Plataformas de engajamento do paciente
US $ 4,7 milhões	Aconselhamento genético virtual	Portal da Web de suporte ao paciente

Redes de distribuidores farmacêuticos

Sarepta colabora com 12 distribuidores farmacêuticos primários nos Estados Unidos, garantindo uma cobertura abrangente para terapias de doenças raras.

• Número de distribuidores nacionais: 12
• Cobertura de distribuição: 50 estados
• Parcerias de farmácia especializadas: 8

Portais de suporte e informações on -line do paciente

A empresa mantém uma plataforma on -line de suporte ao paciente com 47.000 usuários registrados, fornecendo recursos educacionais e informações de tratamento.

Usuários do portal	Tráfego anual do site	Recursos do paciente
47.000 usuários registrados	328.000 visitantes únicos	Informações sobre ensaios clínicos, recursos de aconselhamento genético

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

Pacientes com distúrbios genéticos raros

Sarepta Therapeutics tem como alvo aproximadamente 17.000 pacientes com distrofia muscular de Duchenne (DMD) nos Estados Unidos. O segmento de clientes da empresa inclui pacientes com mutações genéticas específicas, principalmente aquelas com mutações confirmadas, passíveis de terapias de esgotamento de exon.

Categoria de pacientes	População estimada	Tipo de mutação alvo
Pacientes com DMD	17,000	Exon 45-55 deleções
Pacientes com DMD pediátricos	10,500	Passível de terapia genética

Pacientes neuromusculares pediátricos

Sarepta se concentra em pacientes pediátricos com distúrbios neuromusculares, direcionando especificamente crianças de 4 a 15 anos com DMD.

• Idade média do paciente para tratamento inicial: 6-8 anos
• Cobertura de teste genético: aproximadamente 63% dos pacientes pediátricos
• Custo de tratamento anual por paciente: US $ 375.000

Profissionais médicos especializados

Tipo profissional	Alcance estimado	Nível de engajamento
Neurologistas	1,200	Alto
Especialistas genéticos	800	Médio
Especialistas pediátricos	2,500	Alto

Instituições de pesquisa de doenças raras

Sarepta colabora com 47 instituições de pesquisa em todo o mundo, concentrando -se em distúrbios genéticos raros.

• Alocação de financiamento da pesquisa: US $ 42,3 milhões anualmente
• Centros de pesquisa colaborativa: 12 principais instituições acadêmicas
• Participação do ensaio clínico: 38 programas de pesquisa ativos

Centros de tratamento de terapia genética

Tipo de centro de tratamento	Número de centros	Distribuição geográfica
Centros neuromusculares especializados	89	Estados Unidos
Centros abrangentes de terapia genética	62	América do Norte e Europa

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negócios: estrutura de custos

Extensas despesas de pesquisa e desenvolvimento

Em 2023, a Sarepta Therapeutics registrou despesas de P&D de US $ 647,8 milhões. A discriminação anual de investimento em P&D da empresa inclui:

Categoria de P&D	Quantidade de despesa
Pesquisa de terapia genética	US $ 382,5 milhões
Terapêutica de doenças raras	US $ 185,3 milhões
Programas de doenças neuromusculares	US $ 80 milhões

Financiamento de ensaios clínicos

Sarepta alocou US $ 264,7 milhões para atividades de ensaios clínicos em 2023, com a seguinte distribuição:

• Distrofia muscular de Duchenne (DMD): US $ 156,2 milhões
• Ensaios de distrofia muscular (LGMD): US $ 68,5 milhões
• Ensaios raros de doenças genéticas: US $ 40 milhões

Custos de conformidade regulatória

As despesas de conformidade regulatória para 2023 totalizaram US $ 42,3 milhões, incluindo:

Área de conformidade	Custo
Preparativos de envio da FDA	US $ 18,7 milhões
Garantia de qualidade	US $ 15,6 milhões
Documentação regulatória	US $ 8 milhões

Infraestrutura avançada de fabricação

O investimento em infraestrutura de fabricação em 2023 atingiu US $ 98,6 milhões, com alocações específicas:

• Instalações de fabricação de terapia genética: US $ 62,4 milhões
• Atualizações de equipamentos: US $ 24,2 milhões
• Integração de tecnologia: US $ 12 milhões

Recrutamento especializado de talentos médicos

Os custos de aquisição e compensação de talentos para profissionais médicos especializados em 2023 totalizaram US $ 87,5 milhões:

Categoria de talento	Despesa de recrutamento
Cientistas de pesquisa seniores	US $ 38,6 milhões
Especialistas em desenvolvimento clínico	US $ 29,9 milhões
Especialistas em terapia genética	US $ 19 milhões

Sarepta Therapeutics, Inc. (SRPT) - Modelo de negócios: fluxos de receita

Vendas farmacêuticas de produtos

Sarepta Therapeutics gerou receita total de US $ 912,4 milhões em 2022. As principais vendas de produtos incluem:

Produto	Receita anual (2022)
EXONDYS 51	US $ 511,2 milhões
Vyondys 53	US $ 189,7 milhões
Amondys 45	US $ 84,3 milhões

Acordos de licenciamento e royalties

A receita de licenciamento de 2022 totalizou US $ 45,6 milhões, principalmente de acordos de colaboração com parceiros farmacêuticos.

Subsídios governamentais e de pesquisa

O financiamento da concessão de pesquisa em 2022 totalizou US $ 37,8 milhões, incluindo o suporte de:

• Institutos Nacionais de Saúde (NIH)
• Departamento de Defesa
• Associação de Distrofia Muscular

Financiamento de pesquisa colaborativa

As colaborações de pesquisa estratégica geraram aproximadamente US $ 28,5 milhões em 2022, com parcerias importantes, incluindo:

• Roche Pharmaceuticals
• Hospital Infantil em todo o país
• Consórcios de pesquisa de doenças raras

Potencial futura comercialização de produtos terapêuticos

Os produtos de pipeline com possíveis fluxos futuros de receita incluem:

Produto	Área terapêutica	Valor potencial estimado de mercado
Plataforma de terapia genética	Distúrbios neuromusculares	US $ 1,2 bilhão
Candidatos a micro-distrofina	Distrofia muscular de Duchenne	US $ 875 milhões

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.