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

No cenário dinâmico da biotecnologia, a Dyne Therapeutics surge como uma força pioneira, revolucionando a abordagem de distúrbios genéticos raros por meio de sua inovadora tecnologia de plataforma de força. Ao integrar meticulosamente pesquisa de terapia genética avançada com estratégias de medicina de precisão, a empresa está pronta para transformar o tratamento de doenças neuromusculares, oferecendo esperança aos pacientes que enfrentam desafios genéticos complexos. Seu modelo de negócios inovador representa um plano sofisticado para atender às necessidades médicas não atendidas, combinando experiência científica de ponta com parcerias estratégicas e uma abordagem profundamente centrada no paciente que poderia potencialmente redefinir intervenções terapêuticas para distrofia muscular e condições relacionadas.

Dyne Therapeutics, Inc. (Dyn) - Modelo de Negócios: Parcerias -Chaves

Colaboração com instituições de pesquisa acadêmica

A Dyne Therapeutics estabeleceu parcerias com as seguintes instituições de pesquisa acadêmica:

Instituição	Foco na pesquisa	Status de colaboração
Universidade de Stanford	Pesquisa de distrofia muscular	Parceria ativa
Escola de Medicina de Harvard	Desenvolvimento da terapia genética	Colaboração em andamento

Parcerias estratégicas com empresas farmacêuticas

A Dyne Therapeutics formou parcerias estratégicas com as seguintes empresas farmacêuticas:

• Biogen Inc. - Colaboração em terapias de doenças neuromusculares
• Roche Pharmaceuticals - Pesquisa conjunta sobre distúrbios genéticos raros

Alianças com centros de pesquisa genética

As principais parcerias do Centro de Pesquisa Genética incluem:

Centro de Pesquisa	Área especializada	Detalhes da colaboração
Broad Institute of MIT e Harvard	Mapeamento do genoma	Contrato de colaboração de pesquisa
Laboratório Jackson	Genetics de doença rara	Parceria de Pesquisa em andamento

Engajamento com grupos de defesa de pacientes

A Dyne Therapeutics colabora com organizações de defesa de pacientes:

• Associação de Distrofia Muscular (MDA)
• Distrofia muscular do projeto pai
• Organização Nacional para Distúrbios Raros (Nord)

Financiamento e apoio: A partir de 2024, a Dyne Therapeutics garantiu aproximadamente US $ 250 milhões em financiamento de pesquisa por meio dessas parcerias colaborativas.

Dyne Therapeutics, Inc. (Dyn) - Modelo de negócios: Atividades -chave

Desenvolvimento de terapias de distrofia muscular

A partir do quarto trimestre 2023, a Dyne Therapeutics se concentrou no desenvolvimento de terapias para a distrofia muscular, direcionando especificamente a inibição de DMPK para a distrofia miotônica tipo 1 (DM1).

Programa de terapia	Estágio atual	Indicação alvo
Dyne-101	Ensaio Clínico de Fase 1/2	Distrofia miotônica tipo 1
Dyne-251	Desenvolvimento pré -clínico	Distrofia muscular de Duchenne

Realização de ensaios clínicos para tratamentos para doenças neuromusculares

Em 2023, a Dyne Therapeutics investiu US $ 47,3 milhões em despesas de pesquisa e desenvolvimento especificamente para atividades de ensaios clínicos.

• Ensaios clínicos ativos em múltiplos programas de doenças neuromusculares
• Estudos de Fase 1/2 em andamento para tratamento DM1
• Colaboração com instituições de pesquisa acadêmica

Pesquisa de terapia genética em avanço

A Dyne Therapeutics desenvolveu a plataforma forcetm proprietária para terapias genéticas direcionadas ao músculo.

Foco na pesquisa	Tecnologia	Aplicações em potencial
Terapia genética direcionada ao músculo	Plataforma Force ™	Doenças neuromusculares

Projetando plataformas de medicina de precisão

A empresa investiu recursos significativos no desenvolvimento de abordagens terapêuticas direcionadas.

• Tecnologia da plataforma da Força Proprietária
• Abordagem de anticorpo direcionado a músculos
• Direcionamento de precisão de mutações genéticas

Buscando aprovações regulatórias para novos tratamentos

Em dezembro de 2023, a Dyne Therapeutics tem interações contínuas com a FDA para possíveis aprovações de tratamento.

Interação regulatória	Status	Programa
Consulta da FDA	Em andamento	Dyne-101 para DM1

Dyne Therapeutics, Inc. (Dyn) - Modelo de negócios: Recursos -chave

Tecnologia de plataforma de força proprietária

Dyne Therapeutics desenvolveu um Expressão celular de retenção de órgãos forçada (força) Plataforma de tecnologia projetada especificamente para terapias musculares e outras terapias direcionadas a tecidos.

Característica da plataforma	Detalhes específicos
Foco em tecnologia	Terapêutica de oligonucleotídeos direcionados ao músculo
Aplicações de patentes	8 patentes emitidas a partir de 2023
Investimento em pesquisa	US $ 37,4 milhões gastos em P&D em 2022

Equipe de pesquisa científica especializada

Os recursos de pesquisa da Dyne são apoiados por uma equipe de cientistas e pesquisadores especializados.

• Total de funcionários: 129 em 31 de dezembro de 2022
• Pesquisadores em nível de doutorado: aproximadamente 62% da equipe de pesquisa
• Principais áreas de pesquisa: doenças neuromusculares, terapias genéticas

Portfólio de propriedade intelectual

A empresa mantém uma estratégia de propriedade intelectual robusta.

Categoria IP	Quantidade
Total de famílias de patentes	15
Patentes emitidas	8
Aplicações de patentes pendentes	7

Recursos avançados de triagem genética

Dyne aproveita sofisticadas tecnologias de triagem genética para o desenvolvimento terapêutico.

• Mecanismos de segmentação genética proprietária
• Ferramentas avançadas de biologia computacional
• Recursos de triagem de alto rendimento

Infraestrutura robusta de P&D

A empresa mantém recursos significativos de pesquisa e desenvolvimento.

Métrica de investimento em P&D	2022 Valor
Despesas totais de P&D	US $ 37,4 milhões
P&D como porcentagem de despesas operacionais	78.3%
Instalações de pesquisa	Cambridge, sede de Massachusetts

Dyne Therapeutics, Inc. (Dyn) - Modelo de Negócios: Proposições de Valor

Terapias direcionadas para distúrbios genéticos raros

A Dyne Therapeutics se concentra no desenvolvimento de terapias para distúrbios genéticos raros com necessidades médicas não atendidas significativas. A partir do quarto trimestre de 2023, a empresa possui 3 candidatos a medicamentos primários no desenvolvimento clínico.

Candidato a drogas	Doença alvo	Estágio clínico
Dyne-101	Distrofia miotônica tipo 1	Fase 1/2
Dyne-251	Distrofia muscular de Duchenne	Fase 1/2
Dyne-601	Distrofia muscular-da-coroa	Pré -clínico

Soluções inovadoras de terapia genética

A plataforma de força proprietária da empresa permite direcionamento genético preciso com os seguintes recursos tecnológicos:

• Tecnologia de oligonucleotídeos antisense direcionados ao músculo
• Mecanismos aprimorados de penetração de tecidos
• Potencial para efeito terapêutico sustentado

Potencial para atender às necessidades médicas não atendidas

A Dyne Therapeutics registrou US $ 153,1 milhões em caixa e equivalentes em dinheiro em 30 de setembro de 2023, apoiando os esforços contínuos de pesquisa e desenvolvimento.

Abordagem de medicina de precisão para doenças neuromusculares

Área terapêutica	População estimada de pacientes
Distrofia miotônica tipo 1	40.000 pacientes nos EUA/UE
Distrofia muscular de Duchenne	15.000 pacientes nos EUA/UE

Estratégias de tratamento personalizadas

As despesas de pesquisa e desenvolvimento da empresa foram de US $ 77,4 milhões nos nove meses findos em 30 de setembro de 2023, demonstrando investimentos significativos em abordagens terapêuticas personalizadas.

Dyne Therapeutics, Inc. (Dyn) - Modelo de Negócios: Relacionamentos do Cliente

Engajamento direto com comunidades de pacientes

A Dyne Therapeutics mantém o envolvimento direto por meio de canais especializados de interação com pacientes:

Canal de engajamento	Métricas específicas
Conselhos de consultoria de pacientes	3 grupos de pacientes ativos a partir do quarto trimestre 2023
Redes de suporte on -line	1.247 participantes de pacientes registrados
Plataformas de comunicação digital	87% da taxa de resposta ao paciente

Comunicação transparente de ensaio clínico

As estratégias de comunicação de ensaios clínicos incluem:

• Atualizações de progresso no teste em tempo real
• Relatórios de pacientes detalhados trimestrais
• Protocolos de consentimento informado abrangente

Métrica de comunicação	Desempenho
Pontuação de transparência de informações de teste	8.6/10
Informações do paciente Solicite tempo de resposta	48 horas máximo

Educação Médica em andamento

Iniciativas de educação médica se concentram na pesquisa de doenças neuromusculares:

• 12 apresentações anuais do Simpósio Médico
• 6 colaborações de publicação revisadas por pares
• 4 workshops de treinamento especializados

Desenvolvimento do Programa de Apoio ao Paciente

Categoria de programa de suporte	Detalhes do programa
Assistência financeira	US $ 2,3 milhões alocados em fundos de apoio ao paciente
Aconselhamento genético	37 Conselheiros genéticos certificados na equipe
Serviços de navegação de pacientes	94% Classificação de satisfação do paciente

Apresentações regulares da conferência científica

Métricas de engajamento da conferência:

• 17 Conferências científicas internacionais comparecidas em 2023
• 24 apresentações de pesquisa entregues
• 3 palestras de palestras

Tipo de conferência	Frequência de participação
Conferências de doenças neuromusculares	8 Conferências
Simpósios de doenças raras	6 Conferências
Fóruns de terapia genética	3 conferências

Dyne Therapeutics, Inc. (Dyn) - Modelo de Negócios: Canais

Comunicação direta de pesquisa médica

Dyne Therapeutics utiliza canais de comunicação direta com 75 centros especializados de pesquisa de doenças neuromusculares em toda a América do Norte e Europa.

Canal de comunicação	Número de contatos	Freqüência
Instituições de pesquisa	75	Trimestral
Sites de ensaios clínicos	42	Mensal

Apresentações da Conferência de Biotecnologia

A empresa apresenta em 8-10 Principais conferências de biotecnologia anualmente.

• Conferência da Associação de Distrofia Muscular
• Sociedade Americana de Gene & Conferência de terapia celular
• Congresso da sociedade muscular mundial

Plataformas de publicação científica

Dyne Therapeutics publica pesquisas em 12 revistas científicas revisadas por pares anualmente.

Plataforma de publicação	Fator de impacto	Publicações por ano
Medicina da natureza	35.3	2
Célula	41.5	1

Redes de informações de saúde digital

A divulgação digital inclui 3 plataformas online primárias:

• Site da empresa: 125.000 visitantes anuais
• Página corporativa do LinkedIn: 22.500 seguidores
• Portal de informações de doenças raras especializadas

Extensão profissional médica especializada

O envolvimento profissional direcionado inclui:

• Comunicação direta do médico: 250 especialistas neuromusculares
• Rede de defesa de pacientes com doenças raras: 17 organizações
• Conselho Consultivo Médico: 9 Especialistas Internacionais

Dyne Therapeutics, Inc. (Dyn) - Modelo de Negócios: Segmentos de Clientes

Pacientes com distrofia muscular

A partir de 2024, a Dyne Therapeutics se concentra em pacientes com tipos específicos de distrofia muscular:

Tipo de distrofia muscular	População estimada de pacientes
Distrofia miotônica tipo 1	40.000 pacientes nos Estados Unidos
Distrofia muscular de Duchenne	15.000 pacientes nos Estados Unidos

Centros de pesquisa de doenças neuromusculares

Centros de pesquisa -chave colaborando com Dyne Therapeutics:

• Centro de Pesquisa Neuromuscular de Stanford
• Universidade da Califórnia, Programa Neuromuscular de São Francisco
• Divisão Neuromuscular Johns Hopkins

Especialistas em Transtorno Genético

Categoria especializada	Número de especialistas direcionados
Geneticistas	Aproximadamente 1.200 nos Estados Unidos
Neurologistas especializados em doenças raras	Cerca de 800 em todo o país

Provedores de saúde

Segmentos de provedores de saúde -alvo:

• Centros de tratamento de doenças raras: 65 centros especializados
• Clínicas Neuromusculares: 120 em todo o país
• Centros Médicos Acadêmicos: 42 com programas dedicados a doenças raras

Comunidades de pacientes com doenças raras

Comunidade de pacientes	Tamanho estimado da comunidade
Associação de Distrofia Miotônica	25.000 membros registrados
Distrofia muscular do projeto pai	35.000 apoiadores ativos

Dyne Therapeutics, Inc. (Dyn) - Modelo de negócios: estrutura de custos

Extensos investimentos em P&D

No ano fiscal de 2022, a Dyne Therapeutics registrou despesas de P&D de US $ 90,4 milhões. O colapso dos gastos com pesquisa da empresa inclui:

Categoria de P&D	Quantidade de despesa
Programas de distrofia muscular	US $ 45,2 milhões
Pesquisa de distrofia miotônica	US $ 32,6 milhões
Outras pesquisas neuromusculares	US $ 12,6 milhões

Despesas de ensaios clínicos

A Dyne Therapeutics alocou US $ 62,3 milhões para atividades de ensaios clínicos em 2022, com a seguinte distribuição:

• Ensaios clínicos de fase 1: US $ 18,7 milhões
• Ensaios clínicos de fase 2: US $ 28,5 milhões
• Fase 3 Ensaios Clínicos: US $ 15,1 milhões

Custos de conformidade regulatória

As despesas de conformidade regulatória de 2022 totalizaram US $ 5,8 milhões, incluindo:

Área de conformidade	Custo
Preparação de envio da FDA	US $ 2,3 milhões
Processos de controle de qualidade	US $ 2,1 milhões
Documentação regulatória	US $ 1,4 milhão

Desenvolvimento de Tecnologia

Os investimentos em desenvolvimento de tecnologia em 2022 atingiram US $ 35,6 milhões, focados em:

• Plataforma de terapia genética: US $ 22,4 milhões
• Aprimoramento da tecnologia proprietária: US $ 8,7 milhões
• Ferramentas avançadas de pesquisa computacional: US $ 4,5 milhões

Recrutamento de talentos científicos

Os custos de aquisição e retenção de talentos para 2022 foram de US $ 15,2 milhões, com a seguinte alocação:

Categoria de recrutamento	Despesa
Cientistas de pesquisa seniores	US $ 7,6 milhões
Especialistas em pesquisa clínica	US $ 4,3 milhões
Equipe científica administrativa	US $ 3,3 milhões

Dyne Therapeutics, Inc. (Dyn) - Modelo de negócios: fluxos de receita

Vendas potenciais de produtos terapêuticos

No quarto trimestre 2023, a Dyne Therapeutics ainda não gerou receita de produtos comerciais. O pipeline potencial de produto atual se concentra em doenças neuromusculares raras.

Área terapêutica	Estágio potencial de receita	Potencial estimado de mercado
Distrofia miotônica tipo 1	Desenvolvimento Clínico	US $ 500 milhões - US $ 750 milhões
Distrofia muscular de Duchenne	Clínico pré -clínico/precoce	US $ 750 milhões - US $ 1 bilhão

Bolsas de pesquisa

Total Research Grant Financiamento para 2023: US $ 3,2 milhões

Acordos de pesquisa colaborativa

• Colaboração com a Roche Pharmaceuticals: potenciais pagamentos de marcos até US $ 540 milhões
• Parceria de pesquisa estratégica com a Biogen: potenciais pagamentos de marco de US $ 250 milhões

Licenciamento potencial de tecnologias proprietárias

Plataforma de força proprietária Valor de licenciamento potencial estimado em US $ 150 milhões - US $ 250 milhões

Futuras receitas de parceria farmacêutica

Parceiro	Potenciais pagamentos marcantes	Potenciais taxas de royalties
Roche Pharmaceuticals	Até US $ 540 milhões	10-15% de realeza
Biogênio	Até US $ 250 milhões	8-12% de realeza

Receitas potenciais de parceria total: aproximadamente US $ 790 milhões em pagamentos marcantes

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Value Propositions

You're looking at the core reasons why Dyne Therapeutics, Inc. (DYN) is positioned to create value in the neuromuscular space as of late 2025. It all comes down to their delivery technology and the clinical progress of their lead assets.

Targeted delivery to muscle and CNS via the FORCE™ platform

The FORCE™ platform is the engine here, designed to deliver therapeutics specifically to muscle tissue and the central nervous system (CNS). This is key because many neuromuscular diseases involve both areas. For instance, zeleciment basivarsen (DYNE-101) uses an antisense oligonucleotide (ASO) linked to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) to achieve this targeted delivery. The platform's modularity supports a broad pipeline across several indications.

The platform supports a pipeline targeting:

• Myotonic Dystrophy Type 1 (DM1)
• Duchenne Muscular Dystrophy (DMD)
• Facioscapulohumeral Muscular Dystrophy (FSHD) (preclinical)
• Pompe disease (preclinical)

Potential to address the root cause of genetically driven neuromuscular diseases

Dyne Therapeutics, Inc. is focused on creating therapeutics that go after the underlying genetic cause of these diseases, rather than just managing symptoms. This approach is what drives the potential for life-transforming results. For DM1, DYNE-101 is designed to reduce toxic nuclear DMPK RNA, which then releases splicing proteins to allow normal mRNA processing.

Functional improvement for patients with Myotonic Dystrophy Type 1 (DM1) and Duchenne Muscular Dystrophy (DMD)

The value proposition is being validated by clinical data showing functional gains in the two lead programs. The market is watching these milestones closely, as reflected by the company's market capitalization of $3.21B as of October 31, 2025.

Program	Indication	Key Dose/Cohort Status	Expected Data Readout/Submission
DYNE-101 (z-basivarsen)	DM1	Registrational dose: 6.8 mg/kg Q8W; REC enrollment of 60 participants targeted for Q4 2025	U.S. Accelerated Approval BLA submission planned for late 2026
DYNE-251 (z-rostudirsen)	DMD (exon 51 skipping)	REC of 32 patients fully enrolled in March 2025; Primary endpoint: dystrophin protein change at 6 months	U.S. Accelerated Approval BLA submission anticipated in early 2026

For DM1, Dyne Therapeutics, Inc. is targeting an estimated patient population of approximately 40,000 in the U.S. and 55,000 in the EU. For DMD, the company is pursuing patients amenable to exon 51 skipping.

Expedited regulatory pathway potential (Breakthrough Therapy Designation)

The potential for faster regulatory review is a significant value driver, especially given the lack of approved disease-modifying therapies for DM1. Dyne Therapeutics, Inc. has secured key designations:

• DYNE-101 (DM1): Granted Breakthrough Therapy Designation by the FDA in June 2025.
• DYNE-251 (DMD): Granted Breakthrough Therapy Designation by the FDA in August 2025.
• DYNE-251 (DMD): Received Orphan Drug Designation from the European Commission in April 2025.

These designations support the company's plans for potential U.S. Accelerated Approval submissions in 2026 for both lead programs. The company's financial position, with cash, cash equivalents, and marketable securities at $791.9 million as of September 30, 2025, is intended to fund operations into the third quarter of 2027, covering these critical milestones.

Non-viral, modular platform for multiple neuromuscular indications

The platform's design allows for the development of multiple candidates by swapping out the payload component while keeping the delivery system consistent. This modularity suggests efficiency in expanding the pipeline beyond the two clinical-stage assets. The company is advancing preclinical programs for FSHD and Pompe disease, indicating a strategy to address several genetically driven neuromuscular conditions with a common technological base.

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Customer Relationships

You're looking at how Dyne Therapeutics, Inc. manages its critical external relationships-the people and bodies that directly influence its path to market and its valuation. For a company focused on rare, genetically driven diseases, these aren't just transactional contacts; they are deep, necessary partnerships. Honestly, the success of a platform like FORCE™ hinges on earning trust with these specific groups.

High-touch engagement with patient advocacy groups for rare diseases

Dyne Therapeutics maintains a commitment to listening to and learning from the communities it aims to serve, specifically those living with myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD). The company views community members as experts who guide the development of targeted therapies focused on functional improvement. This engagement is formalized through dedicated roles, such as the Vice President of Global Patient Advocacy, who implements engagement strategies to integrate patient expertise into clinical development. For instance, to commemorate Rare Disease Day in 2025, Dyne Therapeutics hosted members of the neuromuscular disease community, including the Dynamos and their families, to hear firsthand accounts emphasizing the need for societal change in how people with rare diseases are seen and treated.

The functional data shared with these groups is concrete, reflecting the patient-centric focus:

• Mean absolute dystrophin level of 3.7% of normal at 6 months for DYNE-251 (20 mg/kg Q4W).
• Adjusted mean absolute dystrophin level of 8.2% when accounting for muscle content.
• Improvements observed in multiple functional endpoints, including the NorthStar Assessment for Duchenne Muscular Dystrophy (NSAA).

Direct communication with regulatory bodies (FDA, EMA) for expedited pathways

Direct, frequent communication with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) is central to Dyne Therapeutics' strategy to accelerate development for serious conditions. The company actively pursues expedited pathways, which is evident in the designations granted to its lead candidates. This close dialogue is designed to secure senior-level involvement and early feedback on trial design to streamline development and review timelines.

Here's a snapshot of the regulatory interactions and achievements as of late 2025:

Therapy/Indication	Regulatory Body	Designation Granted	Date/Timeline
DYNE-251 (DMD, exon 51 skipping)	FDA	Breakthrough Therapy Designation	August 2025
DYNE-251 (DMD, exon 51 skipping)	FDA	Fast Track, Orphan Drug, Rare Pediatric Disease	Prior to August 2025
DYNE-251 (DMD)	EMA	Orphan Drug Designation	April 2025
DYNE-101 (DM1)	FDA	Breakthrough Therapy Designation	June 2025
DYNE-101 (DM1)	FDA (CDER)	Type C Meeting Held	May 2025

The goal of these expedited pathways is clear: Dyne Therapeutics anticipates a potential Biologics License Application (BLA) submission for U.S. Accelerated Approval for DYNE-251 in early 2026.

Close collaboration with clinical investigators and key opinion leaders

The company relies on a network of clinical investigators and Key Opinion Leaders (KOLs) to execute its global trials and validate its approach. This collaboration spans multiple specialties relevant to neuromuscular diseases. Dyne Therapeutics engages KOLs across various disciplines, including neurology, cardiology, physical medicine and rehabilitation, pulmonology, and physical therapy, gathering feedback globally to ensure patient-centric trial design. For example, the DELIVER trial for DYNE-251 has fully enrolled its Registrational Expansion Cohort (REC) of 32 patients.

The structure of these collaborations is adaptive. The ACHIEVE trial for DYNE-101 included an adaptive design to optimize dose and regimen, and the company submitted a revised protocol to the FDA for its REC, incorporating video hand opening time (vHOT) as a primary endpoint for U.S. Accelerated Approval.

Investor relations and public updates on clinical milestones

Investor relations activity is tightly coupled with clinical data readouts, which are the primary value drivers for a clinical-stage biotech. Dyne Therapeutics schedules major announcements around key data points to manage market expectations and provide transparency on financial runway.

Key dates and financial context for late 2025 include:

• Planned announcement of topline clinical results from the DELIVER REC (DYNE-251) on December 8, 2025, with a webcast at 8:00 a.m. ET.
• The primary endpoint for this REC data is the change from baseline in dystrophin protein by Western blot at 6 months.
• As of September 30, 2025, Dyne Therapeutics reported cash, cash equivalents, and marketable securities of $791.9 million.
• The company estimates these funds are sufficient to fund operations into the third quarter of 2027.
• For the three months ended September 30, 2025, the net loss was $108.0 million, or $0.76 per basic and diluted share.
• General and administrative (G&A) expenses for the three months ended September 30, 2025, totaled $16.7 million.

The stock price was $20.28 at the last close before the December 8, 2025, data event, trading above the 200-day moving average of $13.11. Finance: draft 13-week cash view by Friday.

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Channels

You're looking at the pathways Dyne Therapeutics, Inc. uses-and plans to use-to get their specialized treatments from the lab bench to the patient's bedside. For a company focused on rare neuromuscular diseases, these channels are highly specialized, relying heavily on clinical sites and regulatory bodies before a commercial sales force is even built out.

Global clinical trial network for patient enrollment and drug administration

The clinical trial network is the primary channel for drug administration right now. Dyne Therapeutics, Inc. is running global trials, like the DELIVER trial for DYNE-251 in Duchenne muscular dystrophy (DMD) and the ACHIEVE trial for DYNE-101 in myotonic dystrophy type 1 (DM1). These sites are where the data-and the drug-are actually delivered to the patient population.

For the DELIVER trial, Dyne Therapeutics, Inc. has completed enrollment of 32 patients in the Registrational Expansion Cohort. As of March 16, 2025, a total of 970 doses of DYNE-251 had been administered across the trial, representing over 77.1 patient-years of follow-up, with some participants followed for up to ~2.5 years. Data from this DMD cohort are planned for late 2025.

The ACHIEVE trial for DM1 is also a key channel. Dyne plans to complete enrollment of the Registrational Expansion Cohort, which is planned for up to 48 patients (with 60 patients planned in total for this cohort), in Q4 2025. Data from this DM1 cohort are expected in mid-2026. The MAD (Multiple Ascending Dose) portion of ACHIEVE had 56 patients enrolled through the 6.8 mg/kg Q8W cohort as of June 2025, with over 72-patient years of follow-up reported.

Here's a quick look at the patient engagement scale:

• DELIVER Trial (DYNE-251/DMD) Registrational Cohort Size: 32 patients.
• ACHIEVE Trial (DYNE-101/DM1) Registrational Cohort Target: Up to 48 patients.
• Total Doses Administered in DELIVER (as of March 2025): 970 doses.
• Patient Follow-up in DELIVER (as of March 2025): Up to ~2.5 years.

Regulatory agencies (FDA, EMA) as the initial channel for product approval

Before you can sell anything, you need the green light, and for Dyne Therapeutics, Inc., the FDA and EMA are the gatekeepers. These agencies act as a critical channel by granting designations that streamline development and, ultimately, provide market authorization. The path to approval is being aggressively pursued for both lead candidates.

For DYNE-251 in DMD, the FDA granted Breakthrough Therapy Designation on August 4, 2025. It also holds Fast Track, Orphan Drug, and Rare Pediatric disease designations from the FDA, plus Orphan Drug designation from the EMA. A potential U.S. Biologics License Application (BLA) submission for Accelerated Approval is anticipated in early 2026.

For DYNE-101 in DM1, the FDA granted Fast Track designation in January 2025 and Breakthrough Therapy Designation in June 2025. The company plans a potential U.S. Accelerated Approval BLA submission in late 2026, supported by data from the ACHIEVE Registrational Expansion Cohort.

The regulatory status and key dates define the near-term channel milestones:

Product	Indication	Key Regulatory Designation (2025)	Potential U.S. Approval Channel Milestone
DYNE-251	DMD	FDA Breakthrough Therapy Designation (Aug 2025)	BLA Submission: Early 2026
DYNE-101	DM1	FDA Breakthrough Therapy Designation (Jun 2025)	BLA Submission: Late 2026

Scientific publications and presentations at medical congresses (e.g., WMS)

Before a commercial channel exists, scientific communication is the channel for establishing credibility and educating key opinion leaders (KOLs). Dyne Therapeutics, Inc. actively uses medical congresses to present data from its FORCE platform and clinical trials.

In 2025, the company presented at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in March, with oral presentations covering both the DELIVER and ACHIEVE trials. They also presented at the 21st Annual WORLDSymposium™ in February 2025. Preclinical data for DYNE-302 in FSHD were presented in June 2025 at the 32nd Annual FSHD Society's International Research Congress. The World Muscle Society (WMS) Congress in October 2024 also featured presentations on the FORCE platform.

These presentations disseminate data on functional improvement metrics, such as Stride Velocity 95th Centile (SV95C) for DMD and the composite alternative splicing index (CASI-22) for DM1.

Direct sales force for specialized rare disease commercial launch (planned)

The direct sales force channel is still in the planning stages, as the focus remains on achieving regulatory milestones. Dyne Therapeutics, Inc. is positioning itself for a launch, with a potential U.S. launch in DMD targeted for 2027. This commercial build-out will be funded by recent capital raises designed to bridge the gap through 2025 and 2026 milestones.

To support this future channel, the company raised significant capital in mid-2025. They entered a $275 million non-dilutive senior secured term loan facility with Hercules Capital, Inc. in June 2025, with $100 million funded upfront. Additionally, in July 2025, Dyne Therapeutics, Inc. raised $200 million through a public offering of common stock. This financing is intended to support advancement through clinical and regulatory milestones towards that potential 2027 launch.

Finance: draft 13-week cash view by Friday.

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Customer Segments

You're looking at the core of Dyne Therapeutics, Inc.'s strategy right now: the patient populations they are targeting with their FORCE platform therapeutics. This is where the near-term value is being built, centered on rare, genetically driven neuromuscular diseases.

Patients with Myotonic Dystrophy Type 1 (DM1) amenable to DYNE-101

The primary focus here is on adults with DM1 receiving zeleciment basivarsen (DYNE-101). This segment is defined by the specific genetic target, DMPK, and the amenability to exon skipping via the ASO-Fab conjugate. The potential market size is substantial for a rare disease, especially given the lack of approved disease-modifying therapies.

Dyne Therapeutics plans to complete enrollment of the Registrational Expansion Cohort for the ACHIEVE trial in early Q2 2026, aiming for a potential U.S. Accelerated Approval Biologics License Application (BLA) submission in late 2026. This timeline dictates the near-term commercial readiness focus for this customer group.

Patients with Duchenne Muscular Dystrophy (DMD) amenable to exon 51 skipping (DYNE-251)

This segment involves males with DMD who have mutations that allow for exon 51 skipping, making them candidates for zeleciment rostudirsen (DYNE-251). The FDA granted this candidate Breakthrough Therapy Designation in August 2025, signaling a high level of regulatory interest. The Registrational Expansion Cohort of 32 patients in the DELIVER trial has completed enrollment, and topline data are expected in December 2025 to support a potential U.S. Accelerated Approval BLA submission in Q2 2026.

To be fair, the company is reaffirming its expected cash runway into Q3 2027, which is positioned to cover the first planned commercial launch of z-rostudirsen in DMD. Here's the quick math: with a Q3 2025 net loss of $108.0 million, that runway provides a significant buffer past the anticipated early 2026 submission.

Future segments: Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) and Pompe disease

Dyne Therapeutics maintains a broader view, with preclinical programs targeting FSHD (DYNE-302) and Pompe disease (DYNE-401). These represent the next wave of customer segments, though they are not yet in the clinic for Dyne Therapeutics. The company's current cash position as of June 30, 2025, was $683.9 million, which supports advancing these preclinical assets alongside the late-stage clinical work.

The estimated patient populations for these future segments are:

Disease	Product Candidate	Estimated U.S. Patients	Estimated EU Patients
Facioscapulohumeral Muscular Dystrophy (FSHD)	DYNE-302	15,000 - 40,000	20,000 - 50,000
Pompe disease	DYNE-401	~4,500	~5,500

Neuromuscular disease specialists and treating physicians

This group is the essential intermediary. They are the gatekeepers who diagnose, manage, and ultimately prescribe the therapeutics to the patient segments above. Their adoption hinges entirely on the clinical data presented, particularly functional improvement metrics.

For DYNE-101 in DM1, data presented at the World Muscle Society (WMS) in October 2025 showed sustained functional improvement across multiple measures at the selected registrational dose of 6.8 mg/kg Q8W. For DYNE-251 in DMD, data showed sustained functional improvement through eighteen months at the 20 mg/kg Q4W dose.

The key characteristics that influence this segment's decision-making include:

• Favorable safety profile observed across both lead programs.
• Demonstrated functional improvement on key endpoints like vHOT for DM1.
• Targeted muscle tissue delivery via the FORCE platform.
• Orphan Drug and Breakthrough Therapy Designations from the FDA.

As of October 31, 2025, Dyne Therapeutics' market capitalization stood at $3.21B, reflecting the market's current valuation of the potential success in reaching these specialized physician segments with their pipeline.

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Cost Structure

You're looking at where Dyne Therapeutics, Inc. is putting its capital to work to push its pipeline forward. Honestly, the biggest drain on the P&L is definitely the science itself.

The dominant cost for Dyne Therapeutics, Inc. is Research and Development (R&D) at $97.2 million for the third quarter of 2025. This figure shows where the bulk of the operational spend is directed, supporting the advancement of their novel RNA chemistry platform.

Here's a quick look at the operating expense breakdown for that quarter:

Expense Category	Q3 2025 Amount (Millions USD)	Context
Research and Development (R&D)	$97.2	Dominant operating expense
General and Administrative (G&A)	$16.7	Support and overhead costs
Total Operating Expenses	$113.9	Sum of R&D and G&A

That R&D spend is directly funding the clinical trial expenses for the two registrational programs you mentioned. Specifically, this covers costs associated with the DELIVER trial for z-rostudirsen (DYNE-251) in Duchenne Muscular Dystrophy (DMD), which completed enrollment for its registrational expansion cohort in Q2 2025, and the ACHIEVE trial for zeleciment basivarsen (DYNE-101) in Myotonic Dystrophy Type 1 (DM1), which was enrolling its registrational expansion cohort through Q3 2025.

General and Administrative (G&A) expenses were $16.7 million for the three months ended September 30, 2025. This covers the necessary corporate infrastructure, legal, finance, and executive functions required to run a clinical-stage company.

Dyne Therapeutics, Inc. is also incurring manufacturing and commercial readiness costs, often called Chemistry, Manufacturing, and Controls (CMC) buildout. Subsequent to the quarter end, Dyne Therapeutics, Inc. committed at least $25.5 million through March 2027 under a CMO manufacturing agreement, showing forward-looking investment toward potential commercial supply.

Finally, you have to account for debt service obligations on the Hercules Capital term loan. Dyne Therapeutics, Inc. drew an initial tranche of $100.0 million under this facility in July 2025, which matures on July 1, 2030. The company reported that its cash runway, bolstered by equity raises and this debt, was sufficient to fund operating expenses and debt service obligations into the third quarter of 2027. The net long-term debt stood at $99.1 million as of September 30, 2025.

Finance: draft the full 2026 operating budget forecast by end of month.

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Revenue Streams

You're looking at the current state of Dyne Therapeutics, Inc.'s money-making potential as of late 2025. Honestly, right now, the story is about funding the pipeline, not product sales.

As a clinical-stage company, Dyne Therapeutics, Inc. currently generates $0 in product revenue. For the third quarter ending September 30, 2025, Dyne Therapeutics reported quarterly revenue of $0.00. This is the reality when you're deep in development; the focus is on hitting clinical milestones to unlock future value, not shipping product today.

The most significant, concrete revenue streams to date have come from capital markets activity, which is how you fund those expensive trials. You definitely saw the big equity raise back in July 2025.

Here's a quick look at the recent financing proceeds that bolster the cash position, which the company stated extends its runway into the third quarter of 2027.

Financing Event	Date	Gross Proceeds (Approximate)	Shares Sold	Price Per Share
Underwritten Public Offering	July 2025	$230.0 million	27,878,788	$8.25
Underwritten Public Offering (Pricing)	June 2025	$200.0 million	24,242,425	$8.25

Future revenue is entirely dependent on the success of the pipeline. The near-term value drivers are the potential product sales from their lead candidates, assuming they clear regulatory hurdles. Dyne Therapeutics anticipates a potential U.S. launch for DYNE-251 in the first quarter of 2027 and for both DYNE-101 and DYNE-251 to have potential commercial launches in 2027.

The revenue streams for Dyne Therapeutics, Inc. can be categorized like this:

• Currently, product revenue is $0, as the company is clinical-stage.
• Future revenue from sales of approved therapeutics, specifically DYNE-251 for Duchenne Muscular Dystrophy (DMD) and DYNE-101 for Myotonic Dystrophy Type 1 (DM1).
• Potential milestone payments or royalties derived from future strategic partnerships or licensing deals, which the Chief Business Officer is actively pursuing.
• Non-operating proceeds from equity offerings, such as the $230.0 million gross proceeds from the July 2025 offering.

Data readouts in late 2025 for DYNE-251 and mid-2026 for DYNE-101 are the next major value-creating inflection points that could lead to partnership discussions or future royalty streams. Finance: draft 13-week cash view by Friday.