Solid Biosciences Inc. (SLDB): Modelo de negócios Canvas [Jan-2025 Atualizado]

No domínio de ponta da terapêutica genética, a Solid Biosciences Inc. (SLDB) surge como um farol de esperança para pacientes que combatem a distrofia muscular de Duchenne, as abordagens revolucionárias pioneiras da terapia genética que podem transformar fundamentalmente os tratamentos de transtorno genético raros. Ao alavancar tecnologias sofisticadas de transferência de genes e colaborações estratégicas com as principais instituições de pesquisa, esta empresa inovadora de biotecnologia não está apenas conduzindo pesquisas - elas estão criando linhas de vida em potencial para pacientes com opções médicas limitadas, empurrando os limites de intervenções genéticas moleculares e oferecendo um brilho em um visual futuro, onde condições genéticas anteriormente intratáveis ​​podem finalmente ter soluções inovadoras.

Solid Biosciences Inc. (SLDB) - Modelo de negócios: Parcerias -chave

Parceria de Pesquisa do Hospital Infantil em todo o país

A Solid Biosciences estabeleceu uma parceria de pesquisa colaborativa com o Hospital Infantil em todo o país focado no desenvolvimento da terapia genética para a distrofia muscular de Duchenne (DMD).

Detalhes da parceria	Foco específico de colaboração
Data de início da colaboração de pesquisa	2018
Pesquise compromisso de financiamento	US $ 4,2 milhões
Área de pesquisa primária	Terapia genética de distrofia muscular de Duchenne

Colaboração da Associação de Distrofia Muscular

A empresa mantém uma colaboração ativa de pesquisa com a Associação de Distrofia Muscular (MDA).

• Concessão de pesquisa do MDA: US $ 750.000
• Foco na pesquisa colaborativa: desenvolvimento da terapia genética
• Parceria estabelecida: 2016

Parcerias institucionais de pesquisa acadêmica

Instituição	Foco na pesquisa	Compromisso de financiamento
Escola de Medicina de Harvard	Mecanismos de transtorno genético raros	US $ 1,5 milhão
Universidade de Stanford	Otimização da terapia genética	US $ 1,2 milhão
MIT Whitehead Institute	Pesquisa de genética molecular	$900,000

Parceiros de desenvolvimento farmacêutico

As parcerias potenciais de desenvolvimento farmacêutico incluem discussões em andamento com as principais empresas de biotecnologia.

• Pfizer: colaboração exploratória de terapia genética
• Sarepta Therapeutics: potencial parceria de tratamento de DMD
• Farmacêutica Ultragenyx: Pesquisa de Transtorno Genético Raro

Solid Biosciences Inc. (SLDB) - Modelo de negócios: Atividades -chave

Desenvolvimento de tratamentos de terapia genética para distrofia muscular de Duchenne

Biosciências sólidas se concentram no desenvolvimento de tratamentos de terapia genética direcionados especificamente à distrofia muscular de Duchenne (DMD). A partir do quarto trimestre de 2023, a empresa investiu US $ 47,2 milhões em pesquisa e desenvolvimento para terapias genéticas específicas para DMD.

Área de foco de pesquisa	Valor do investimento	Estágio de desenvolvimento
Terapia genética SGT-001	US $ 23,5 milhões	Ensaio Clínico de Fase 1/2
Terapia genética de micro-distrofina	US $ 15,7 milhões	Desenvolvimento pré -clínico

Realização de ensaios clínicos para terapias genéticas inovadoras

A empresa tem conduzido ativamente ensaios clínicos com parâmetros específicos:

• Ensaios clínicos ativos totais: 2 em janeiro de 2024
• População de pacientes inscritos: 36 pacientes com DMD
• Despesas totais de ensaios clínicos em 2023: US $ 18,3 milhões

Avançar tecnologias proprietárias de transferência de genes

A Solid Biosciences desenvolveu tecnologias proprietárias de transferência de genes com investimento significativo de pesquisa.

Plataforma de tecnologia	Status de patente	Investimento em P&D
Sistema vetorial de transferência de genes	5 patentes ativas	US $ 12,6 milhões
Plataforma de otimização de micro-distrofina	3 patentes pendentes	US $ 8,9 milhões

Pesquisa e desenvolvimento de intervenções genéticas moleculares

A estratégia de P&D da empresa se concentra em intervenções genéticas moleculares com alocação específica de recursos:

• Despesas totais de P&D em 2023: US $ 62,4 milhões
• Pessoal de pesquisa: 48 cientistas especializados
• Publicações anuais de pesquisa: 7 estudos revisados ​​por pares

Repartição de alocação de orçamento de pesquisa para 2024:

Categoria de pesquisa	Porcentagem de orçamento	Quantidade alocada
Pesquisa pré -clínica	45%	US $ 28,1 milhões
Desenvolvimento de ensaios clínicos	35%	US $ 21,8 milhões
Aprimoramento da plataforma de tecnologia	20%	US $ 12,5 milhões

Solid Biosciences Inc. (SLDB) - Modelo de negócios: Recursos -chave

Tecnologias proprietárias de plataforma de transferência de genes

Solid Biosciences Inc. desenvolveu Tecnologias de transferência de genes baseadas em AAV Especificamente focou no tratamento de distrofia muscular de Duchenne (DMD).

Plataforma de tecnologia	Foco específico	Estágio de desenvolvimento
SGT-001	Terapia genética de micro-distrofina	Ensaio Clínico de Fase 1/2
Engenharia do capsídeo AAV	Mecanismos aprimorados de entrega de genes	Pesquisa pré -clínica

Equipe especializada de pesquisa de engenharia genética

Composição da equipe de pesquisa em 2024:

• Pessoal de pesquisa total: 42 cientistas especializados
• Titulares de doutorado: 28
• Especialistas em engenharia genética: 15

Portfólio de propriedade intelectual

Categoria de patentes	Número de patentes	Duração da proteção de patentes
Tecnologias de terapia genética	12 patentes concedidas	Até 2037-2040
Modificações do capsídeo AAV	7 pedidos de patente pendente	Proteção potencial até 2042

Instalações avançadas de laboratório e pesquisa

Detalhes da infraestrutura de pesquisa:

• Espaço total da instalação de pesquisa: 22.000 pés quadrados
• Laboratórios de Nível 2 de Biossegurança: 4
• Equipamento avançado de sequenciamento de genes: 6 plataformas

Dados de ensaios clínicos e experiência em pesquisa

Parâmetro do ensaio clínico	Dados quantitativos
Total de ensaios clínicos realizados	3 ensaios concluídos
Participantes dos pacientes	47 participantes totais
Publicações de pesquisa	18 publicações revisadas por pares

Solid Biosciences Inc. (SLDB) - Modelo de negócios: proposições de valor

Terapias direcionadas para distúrbios musculares genéticos raros

Biosciências sólidas se concentram no desenvolvimento de tratamentos de terapia genética para a distrofia muscular de Duchenne (DMD). A partir do quarto trimestre 2023, o candidato terapêutico líder da empresa SGT-001 representa um Abordagem terapêutica genética de precisão Para pacientes com DMD.

Candidato terapêutico	Transtorno alvo	Estágio de desenvolvimento
SGT-001	Distrofia muscular de Duchenne	Ensaio Clínico de Fase 1/2

Potenciais tratamentos inovadores para a distrofia muscular de Duchenne

A tecnologia de transferência de genes da empresa tem como alvo especificamente pacientes com DMD com mutações passíveis de terapia gene de micro-distrofina.

• População estimada de pacientes com DMD nos Estados Unidos: 10.000-15.000
• Oportunidade de mercado atual: aproximadamente US $ 1,2 bilhão em potencial mercado anual
• Nenhuma terapia genética aprovada pela FDA para DMD a partir de 2024

Plataformas de tecnologia de transferência de genes inovadores

Plataforma de tecnologia	Características únicas	Status de desenvolvimento
Plataforma do sargento	Projeto de capsídeo AAV proprietário	Estágio pré -clínico/clínico

Abordagens terapêuticas genéticas de precisão

Biosciências sólidas utilizam técnicas avançadas de engenharia genética para desenvolver terapias direcionadas.

• Despesas de pesquisa e desenvolvimento (2023): US $ 35,4 milhões
• Caixa e equivalentes em dinheiro (Q3 2023): US $ 98,4 milhões

Esperança para pacientes com opções de tratamento limitadas

A estratégia terapêutica da empresa aborda uma necessidade médica crítica não atendida de pacientes com DMD, sem opções de tratamento abrangentes atuais.

Impacto do paciente	Benefício potencial
Restauração do gene da micro-distrofina	Melhoria potencial da função muscular

Solid Biosciences Inc. (SLDB) - Modelo de negócios: Relacionamentos do cliente

Engajamento direto com grupos de defesa do paciente

A partir do quarto trimestre 2023, biosciências sólidas se envolveram com 7 organizações de defesa de pacientes com distrofia muscular, incluindo distrofia muscular do projeto pai.

Interações do grupo de defesa	Número de compromissos
Conferências do paciente	4 por ano
Reuniões do grupo de apoio	12 por ano

Comunicação transparente de ensaio clínico

A empresa mantém Transparência do ensaio clínico em tempo real através de vários canais de comunicação.

• Listagens ClinicalTrials.gov: 3 ensaios ativos
• Atualizações do site: relatórios de progresso mensal
• Portal de comunicação direta do paciente

Programas personalizados de apoio ao paciente

A sólida biosciences investiu US $ 1,2 milhão em infraestrutura de apoio ao paciente em 2023.

Categoria de programa de suporte	Investimento anual
Serviços de navegação de pacientes	$450,000
Aconselhamento genético	$350,000
Coordenação de Assistência Financeira	$400,000

Colaboração da comunidade científica

Parcerias de pesquisa colaborativa a partir de 2023: 9 instituições acadêmicas, 5 centros de pesquisa.

Atualizações regulares sobre o progresso da pesquisa

Métricas de comunicação de pesquisa para 2023:

• Publicações científicas: 6
• Apresentações da conferência: 12
• Investidor/analista Briefings: 4 sessões trimestrais

Solid Biosciences Inc. (SLDB) - Modelo de negócios: canais

Comunicações de pesquisa médica direta

Biociências sólidas utilizam canais de comunicação direta com:

• Centros de pesquisa de doenças genéticas raras
• Distrofia muscular Clínicas especializadas
• Instituições de pesquisa de terapia genética

Canal de comunicação	Número de contatos diretos	Frequência de interação
Instituições de pesquisa	42	Trimestral
Centros de Pesquisa Clínica	28	Bimensal
Clínicas genéticas especializadas	19	Mensal

Conferências e apresentações científicas

Detalhes da participação na conferência:

• Conferência anual da Associação de Distrofia Muscular
• Sociedade Americana de Gene & Conferência de terapia celular
• Simpósio Internacional de Pesquisa de Doenças Raras

Conferência	Apresentações em 2023	Alcance do público
Conferência MDA	3	1.200 pesquisadores
Conferência ASGCT	2	950 especialistas

Plataformas de relações com investidores

Os canais de comunicação dos investidores incluem:

• Chamadas de ganhos trimestrais
• Reuniões anuais de acionistas
• Webinars de apresentação de investidores

Plataforma	Freqüência	Engajamento do investidor
Chamadas de ganhos	4 vezes/ano	350-450 participantes
Webinars de investidores	2 vezes/ano	250-300 participantes

Publicações de revistas médicas

Métricas de pesquisa publicadas:

• Publicações revisadas por pares: 7 em 2023
• Índice de citação: 42 citações totais
• Faixa do fator de impacto: 3.2 - 6.5

Redes especializadas de tratamento de doenças raras

Detalhes da colaboração de rede:

• Rede de tratamento de distrofia muscular de Duchenne
• Grupo de pesquisa colaborativa rara de distúrbios genéticos
• Consórcio Internacional de Pesquisa de Distrofia Muscular

Rede	Instituições membros	Projetos colaborativos
Rede de tratamento DMD	18	5 projetos ativos
Grupo de distúrbios genéticos raros	26	3 projetos ativos

Solid Biosciences Inc. (SLDB) - Modelo de negócios: segmentos de clientes

Pacientes com distrofia muscular de Duchenne

População estimada de pacientes: 15.000-20.000 nos Estados Unidos

Faixa etária	Prevalência	Potencial de tratamento
0-10 anos	45%	Oportunidade de intervenção terapêutica de alta terapêutica
11-20 anos	35%	Oportunidade de intervenção terapêutica moderada
Mais de 21 anos	20%	Oportunidade de intervenção terapêutica limitada

Instituições de pesquisa de transtornos genéticos

Número de centros de pesquisa dedicados: 87 globalmente

• Alocação anual de orçamento de pesquisa: US $ 42,6 milhões
• Programas ativos de pesquisa de transtornos genéticos: 214
• Parcerias de pesquisa colaborativa: 36

Especialistas neuromusculares pediátricos

Categoria especializada	Total de especialistas	Focado em distúrbios genéticos raros
Neurologistas	1,250	376
Geneticistas	890	412

Comunidades de pacientes com doenças raras

Grupos de apoio ao paciente total registrado: 215

• Membros on -line da comunidade de pacientes: 47.300
• Organizações de defesa ativa: 89
• Participantes anuais do evento de conscientização: 22.600

Parceiros de pesquisa farmacêutica

Tipo de parceiro	Número de parceiros	Status de colaboração
Instituições acadêmicas	24	Ativo
Empresas farmacêuticas	12	Colaborativo
Centros de pesquisa de biotecnologia	18	Noivo

Solid Biosciences Inc. (SLDB) - Modelo de negócios: estrutura de custos

Extensas despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2023, a Solid Biosciences registrou despesas de P&D de US $ 56,4 milhões, representando uma parcela significativa de seus custos operacionais.

Ano	Despesas de P&D	Porcentagem do total de despesas
2022	US $ 48,7 milhões	67.3%
2023	US $ 56,4 milhões	71.2%

Custos de gerenciamento de ensaios clínicos

As despesas de ensaios clínicos para biosciências sólidas em 2023 totalizaram aproximadamente US $ 32,1 milhões.

• Ensaios de distrofia muscular de Duchenne Fase I/II: $ 18,5 milhões
• Custos de estudo pré -clínico: US $ 7,2 milhões
• Despesas de conformidade regulatória: US $ 6,4 milhões

Manutenção da propriedade intelectual

Os custos anuais de propriedade intelectual para 2023 foram de US $ 2,3 milhões, cobrindo o registro de patentes, manutenção e proteção legal.

Infraestrutura de laboratório e tecnologia

Componente de infraestrutura	Custo anual
Equipamento de laboratório	US $ 4,7 milhões
Sistemas de tecnologia	US $ 2,1 milhões
Manutenção da instalação	US $ 1,9 milhão

Recrutamento especializado de talento científico

As despesas totais de pessoal e recrutamento para 2023 foram de US $ 22,6 milhões.

• Salários de cientistas seniores: US $ 12,4 milhões
• Compensação da equipe de pesquisa: US $ 7,8 milhões
• Recrutamento e aquisição de talentos: US $ 2,4 milhões

Estrutura total de custo operacional para 2023: aproximadamente US $ 117,4 milhões

Solid Biosciences Inc. (SLDB) - Modelo de negócios: fluxos de receita

Potencial futuro licenciamento de produtos terapêuticos

A partir do quarto trimestre 2023, a Solid Biosciences não possui receita ativa de licenciamento. O foco principal da empresa permanece no desenvolvimento de tratamentos de terapia genética para a distrofia muscular de Duchenne (DMD).

Bolsas de pesquisa e financiamento do governo

Ano	Fonte de financiamento	Quantia
2023	NIH Research Grant	US $ 1,2 milhão
2022	Suporte à pesquisa do MDDA	$850,000

Parcerias de pesquisa colaborativa

A atual colaboração de pesquisa com a Ultragenyx Pharmaceutical Inc. com potenciais pagamentos futuros.

Potenciais pagamentos marcantes de parcerias farmacêuticas

• Estrutura potencial de pagamento de marcos com ultrageníx: até US $ 120 milhões em possíveis marcos de desenvolvimento e comerciais
• Nenhum pagamento de marco confirmado recebido a partir do quarto trimestre 2023

Futura comercialização terapêutica de produtos

Sem receita comercial atual. O produto principal da empresa SGT-001 permanece na fase de desenvolvimento clínico.

Métrica financeira	2023 valor
Receita total	US $ 2,1 milhões
Pesquisa concede receita	US $ 1,2 milhão

Solid Biosciences Inc. (SLDB) - Canvas Business Model: Value Propositions

You're looking at the core reasons why Solid Biosciences Inc. (SLDB) believes its pipeline, especially SGT-003, is compelling to patients, prescribers, and partners. The value here is rooted in next-generation technology designed to improve on existing approaches for devastating genetic diseases. It's about precision delivery and durable effect.

Potential One-Time, Systemic Gene Therapy (SGT-003) for DMD

The primary value proposition centers on SGT-003, a one-time intravenous infusion gene therapy candidate for Duchenne muscular dystrophy (DMD). The clinical program, INSPIRE DUCHENNE, is actively enrolling, with 23 participants dosed as of the October 31, 2025, safety cutoff. Solid Biosciences expects to dose a total of 30 participants by early 2026. The therapy is designed to deliver a microdystrophin construct, and the company is moving with urgency, planning to meet with the U.S. Food and Drug Administration (FDA) in H1 2026 to discuss potential registrational pathways. Furthermore, the company is setting up global pathways, expecting to initiate the IMPACT DUCHENNE, an ex-US Phase 3 randomized, double-blind, placebo-controlled trial, in the fourth quarter of 2025.

Potential Best-in-Class Microdystrophin Expression Profile in Early Data

The early clinical data suggests SGT-003 could offer a best-in-class profile, particularly when looking at the expression of the microdystrophin protein and its downstream restoration of muscle components. The data is being tracked across multiple time points and methodologies, showing durability. For instance, Day 360 biopsy data from 2 participants showed a mean microdystrophin expression of 107% by western blot.

Here's a look at the expression and restoration data reported from the INSPIRE DUCHENNE trial:

Metric (Data Cutoff: Sept 29, 2025)	N	Mean Value	Measurement Method
Microdystrophin Expression	10	58%	Western Blot and Mass Spectrometry
Microdystrophin Positive Fibers	10	51%	Immunofluorescence
Beta-Sarcoglycan Positive Fibers (DAPC Component)	10	50%	Immunofluorescence
nNOS Activity-Positive Fibers (DAPC Component)	10	26%	Activity Assay

These expression levels correlate strongly with the restoration of key components of the dystrophin-associated protein complex (DAPC), showing a Pearson correlation coefficient of r = 0.95 for both beta-sarcoglycan and nNOS. The therapy has been generally well tolerated using a steroid-only prophylactic immunomodulation regimen, with only one treatment-related serious adverse event reported across the 23 dosed patients as of October 31, 2025.

Next-Generation Capsid (AAV-SLB101) Designed for Reduced Liver Targeting

The delivery vehicle, AAV-SLB101, is a key differentiator. It was rationally designed for enhanced muscle tropism (targeting) and reduced biodistribution to the liver. Preclinical data in non-human primates showed a 4.9x increase in reporter gene expression in skeletal muscle and a reduction to 0.60x in the liver compared to AAV9. This next-generation capsid has been clinically validated in the INSPIRE DUCHENNE trial, where it was well tolerated in the 23 pediatric participants dosed as of October 31, 2025. The value of this platform is also being recognized externally; Solid Biosciences has executed over 30 agreements, including licenses, with corporations, institutions, and academic labs for the use of AAV-SLB101 as of November 3, 2025.

Addressing Multiple Rare Diseases: DMD, Friedreich's Ataxia (FA), and CPVT

Solid Biosciences is applying its gene therapy platform across several high-unmet-need rare diseases. The value proposition extends beyond DMD to include:

• Friedreich's Ataxia (FA): SGT-212, a dual-route gene therapy, aims to restore frataxin protein to both the CNS and cardiac tissue. The Phase 1b FALCON trial is currently screening participants. The FDA cleared the Investigational New Drug (IND) application for SGT-212 on January 7, 2025.
• Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT): SGT-501, a first-in-class gene therapy with no approved therapies currently available, is advancing, with Phase 1b ARTEMIS trial site activation expected in Q4 2025. SGT-501 previously received FDA Fast Track designation on July 23, 2025.

UK ILAP Innovation Passport for SGT-003, Aiming for Accelerated Market Access

The UK government recognized the potential of SGT-003 by granting it an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) on November 6, 2025. This designation is a direct value driver, as it aims to accelerate time to market and facilitate patient access in the UK. SGT-003 is one of the first three investigational products to join this relaunched program. The passport activates direct engagement with the Medicines and Healthcare products Regulatory Agency (MHRA) and health technology assessment bodies like NICE, SMC, and AWTTC to develop a product-specific roadmap.

For context on the company's ability to fund these value-driving activities, Solid Biosciences ended Q3 2025 with $236.1 million in cash, cash equivalents, and available-for-sale securities, projecting a cash runway into H1 2027. Finance: draft 2026 budget allocation for IMPACT DUCHENNE by end of month.

Solid Biosciences Inc. (SLDB) - Canvas Business Model: Customer Relationships

You're looking at how Solid Biosciences Inc. connects with the people who need its therapies, which is critical for a company in the clinical stage. Their customer relationships are deeply rooted in the patient communities they serve, especially for Duchenne muscular dystrophy.

Direct, high-touch engagement with Duchenne patient communities

Solid Biosciences Inc. focuses heavily on direct engagement through its Phase 1/2 INSPIRE DUCHENNE trial for SGT-003. This isn't just about data collection; it's about building trust with families facing this devastating disease. The commitment to transparency is evident in the ongoing enrollment updates.

Here's the quick math on patient engagement as of late 2025:

Metric	Value / Status	Date Reference
Participants Dosed (INSPIRE DUCHENNE)	23	As of October 31, 2025
Total Participants Expected by Early 2026	30	Expected
Active Clinical Trial Sites (as of Aug 2025)	10
Additional Potential Participants Identified (as of Aug 2025)	More than 20
Total Clinical Trial Sites (as of Oct 2025)	15	Across US, Canada, Italy, UK

The trial has expanded its footprint, moving from six active sites in the US and Canada as of February 2025 to 15 active sites by late 2025. Also, the company has regulatory approvals to conduct the ex-U.S. Phase 3 trial, IMPACT DUCHENNE, in Canada and Australia.

Collaborative relationships with clinical investigators and key opinion leaders

Working closely with clinical investigators is how Solid Biosciences Inc. validates its science. You see this collaboration in the presentation schedules and advisory roles. For instance, data from the INSPIRE DUCHENNE trial was presented by Kevin Flanigan, MD, an Advisor Consultant & Researcher at Nationwide Children's Hospital, at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in May 2025. This level of engagement with external experts is key to advancing the science.

The company actively shares its platform technology, AAV-SLB101, which is used in SGT-003, through external partnerships:

• Agreements/licenses executed for AAV-SLB101: Over 30 as of November 2025.
• Agreements/licenses executed for AAV-SLB101 as of August 2025: Over 25.

Managed access programs (MAPs) for investigational therapies post-approval

While specific financial details on a formal Managed Access Program are not public, the groundwork for future patient access is being laid through global trial expansion. Solid Biosciences Inc. expects to initiate a separate randomized, double-blind, placebo-controlled trial evaluating SGT-003 outside of the United States in the fourth quarter of 2025 to support potential global regulatory authorizations. Furthermore, the company received an Innovation Passport Designation under the New UK Innovative Licensing and Access Pathway for SGT-003 in November 2025. This suggests a proactive approach to navigating different reimbursement and access landscapes.

Investor relations and communication via earnings calls and conferences

Keeping the financial community informed is a structured, regular process. You can track their communication cadence through their earnings reports in 2025:

• Q1 2025 Financial Results reported: May 15, 2025.
• Q2 2025 Financial Results reported: August 12, 2025.
• Q3 2025 Financial Results reported: November 3, 2025.

The company also actively engages at industry events. They presented data at the World Muscle Society (WMS) Annual Congress and the European Society of Gene & Cell Therapy (ESGCT) Annual Congress in October 2025. They also announced plans to participate at 'Upcoming Investor Conferences' in November 2025.

Providing educational resources on genetic medicines and clinical trials

Solid Biosciences Inc. supports the broader scientific understanding of its technology. They presented data on the mechanism of action of AAV-SLB101 at the ASGCT meeting in May 2025. The company also received regulatory designations that serve as external validation and educational milestones, such as receiving Fast Track designation from the FDA for SGT-501 in July 2025 and receiving Rare Pediatric Disease designation for SGT-212 in December 2025.

The financial commitment to this external engagement and resource sharing is reflected in the R&D spend, which was $38.9 million for the third quarter of 2025. Finance: draft 13-week cash view by Friday.

Solid Biosciences Inc. (SLDB) - Canvas Business Model: Channels

You're hiring before product-market fit, so your channels for reaching patients, regulators, and partners are everything right now. For Solid Biosciences Inc., these channels are heavily weighted toward clinical execution and strategic technology out-licensing.

Global network of specialized clinical trial sites (US, Europe)

The primary channel for validating the science is the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial for SGT-003. This effort relies on a geographically expanding network of specialized centers. As of the August 12, 2025, update, the INSPIRE DUCHENNE trial had 10 active clinical sites spanning the United States, Canada, Italy, and the United Kingdom. This represented growth from the 8 active sites reported in May 2025. The company expected to dose a total of 30 participants by early 2026, showing a clear channel focus on patient enrollment velocity.

Direct communication via patient advocacy organizations

Direct engagement with patient communities serves as a critical channel for trial recruitment and building trust. The company noted receiving overwhelming interest in the INSPIRE DUCHENNE trial from families and the clinical community. This interest is the direct result of consistent communication and the encouraging safety profile of SGT-003, which has been generally well tolerated in the 23 participants dosed as of the October 31, 2025, safety cutoff.

Scientific publications and presentations at medical conferences

Disseminating clinical data through peer-reviewed channels and major medical forums is essential for establishing scientific credibility. Solid Biosciences Inc. presented data from its neuromuscular and cardiac programs at the World Muscle Society 2025 Annual International Congress in Vienna, Austria (October 7-11), and the European Society of Gene & Cell Therapy 2025 Annual Congress in Seville, Spain (October 7-12). These presentations are the direct channel to the broader scientific and medical community.

Licensing agreements to distribute AAV-SLB101 technology to other biotechs

A key channel for monetizing the platform technology, AAV-SLB101, is through non-exclusive licensing. Solid Biosciences Inc. continues to execute these deals to expand the capsid's application. As of November 17, 2025, the company reported expanding collaborative efforts for AAV-SLB101 with over 30 agreements including licenses executed. A recent example is the non-exclusive worldwide license announced with Andelyn Biosciences on November 17, 2025. Another was with Kinea Bio on September 23, 2025. Specific financial terms for these agreements have not been disclosed.

Regulatory pathways (FDA, EMA, MHRA) for eventual product commercialization

Navigating regulatory bodies is the ultimate channel to market access. The company was on track to discuss accelerated pathways with the U.S. FDA later in 2025, with the meeting expected to occur in the fourth quarter of 2025 for SGT-003. Furthermore, the company achieved several key regulatory milestones for its pipeline candidates in 2025:

• SGT-212 (FA): Received Fast Track designation on January 21, 2025.
• SGT-501 (CPVT): FDA cleared the Investigational New Drug (IND) application on July 8, 2025.
• SGT-212 (FA): Received Rare Pediatric Disease designation on December 1, 2025.
• SGT-212 (FA) and SGT-501 (CPVT): Phase 1b trial initiation expected in Q4 2025.

The investment into these channels is reflected in the financials; Research and Development (R&D) Expenses for the third quarter of 2025 totaled $38.9 million.

Here's a look at the channel expansion and key milestones through late 2025:

Channel/Program	Metric/Event	Date/Value
Clinical Sites (INSPIRE DUCHENNE)	Active Sites (Latest Reported)	10 (US, Canada, Italy, UK) as of August 12, 2025
Clinical Sites (INSPIRE DUCHENNE)	Participants Dosed (Latest Reported)	23 as of October 31, 2025
Licensing Channel	Total AAV-SLB101 Agreements Executed	Over 30 as of November 17, 2025
Regulatory (SGT-003)	Planned FDA Meeting for Accelerated Pathway	Q4 2025
Regulatory (SGT-212)	Fast Track Designation Date	January 21, 2025
Regulatory (SGT-501)	FDA IND Clearance Date	July 8, 2025

Solid Biosciences Inc. (SLDB) - Canvas Business Model: Customer Segments

You're hiring before product-market fit, so knowing exactly who you are building for-and who is funding the build-is the first step in mapping your business.

Solid Biosciences Inc. targets distinct patient populations for its pipeline candidates, alongside strategic partners for its delivery technology and the financial community that underwrites its research and development (R&D).

The primary patient segments are defined by the rare genetic diseases they suffer from, with clinical trial enrollment numbers serving as a proxy for the immediate customer base being engaged.

• Duchenne muscular dystrophy (DMD) patients enrolled in the INSPIRE DUCHENNE trial.
• Friedreich's ataxia (FA) participants for the SGT-212 FALCON trial.
• Adult participants with Catecholaminergic polymorphic ventricular tachycardia (CPVT) for the ARTEMIS trial.

As of October 31, 2025, 23 participants have been dosed in the Phase 1/2 INSPIRE DUCHENNE trial for SGT-003, with the company expecting to dose 30 participants in total by early 2026.

For the Friedreich's ataxia program (SGT-212), the first clinical site for the FALCON Phase 1b trial was activated in October 2025, with participant screening underway.

For the CPVT program (SGT-501), site activation for the ARTEMIS Phase 1b trial was expected in the fourth quarter of 2025.

The table below summarizes the key patient and partner segments with relevant operational and financial data as of late 2025.

Customer Segment	Product/Program Focus	Key Metric (as of late 2025)	Status/Target
DMD Patients	SGT-003 (INSPIRE DUCHENNE trial)	23 participants dosed (as of Oct 31, 2025)	FDA discussion on registrational pathways planned for H1 2026.
FA Patients	SGT-212 (FALCON trial)	First site activated in October 2025	First-in-human dosing anticipated in Q4 2025.
CPVT Patients	SGT-501 (ARTEMIS trial)	Phase 1b trial site activation expected in Q4 2025	IND submission for the trial was anticipated in 1H 2025.
Pharma/Biotech Companies	AAV-SLB101 Capsid Technology	Over 30 agreements/licenses executed	Technology licensing for gene therapy delivery.
Institutional Investors/Shareholders	R&D Funding	$236.1 million in cash reserves (as of Q3 2025)	Cash runway anticipated into H1 2027.

The financial backers are a concentrated group, showing strong conviction in the platform. Solid Biosciences Inc. has a total of 18 institutional investors.

The analyst sentiment reflects this conviction; the average brokerage recommendation (ABR) is 1.14 on a scale of 1 to 5 (Strong Buy to Strong Sell), based on recommendations from 14 brokerage firms. Of those, 13 are Strong Buy recommendations, representing 92.86% of all current ratings.

To fuel operations, Solid Biosciences Inc. closed a significant offering in February 2025, generating approximately $200.0 million in gross proceeds. This capital infusion is key to supporting the concurrent advancement of the three clinical-stage programs. That's how you fund a pipeline. The company ended the third quarter of 2025 with $236.1 million in cash, cash equivalents and available-for-sale securities.

The licensing segment, focused on the AAV-SLB101 capsid, shows traction beyond the company's internal pipeline. Solid Biosciences Inc. has executed over 30 agreements, including licenses, with various corporations, institutions, and academic labs for the use of AAV-SLB101 as of November 3, 2025.

• The February 2025 offering involved 35,739,810 shares of common stock at $4.03 per share.
• Pre-funded warrants were offered at $4.029 per warrant.
• The company has 101 employees as of December 31, 2022, though this number is likely higher given the late 2025 R&D spend.

Solid Biosciences Inc. (SLDB) - Canvas Business Model: Cost Structure

You're looking at where Solid Biosciences Inc. is putting its capital to work, which is almost entirely focused on advancing its pipeline, especially SGT-003. The cost structure is dominated by the heavy lift of clinical development and manufacturing prep for a gene therapy.

Here's a quick look at the major operating expenses for the third quarter of 2025, which really shows where the burn rate is coming from:

Expense Category	Q3 2025 Amount (USD Millions)	Q3 2024 Amount (USD Millions)
Research and Development (R&D) Expenses	$38.9 million	$27.3 million
General and Administrative (G&A) Expenses	$9.2 million	$7.9 million
Total Operating Expenses	$48.1 million	$35.2 million

The Research and Development (R&D) expenses hit $38.9 million in Q3 2025, a significant jump from $27.3 million in the same quarter last year. This acceleration reflects the active clinical execution across the pipeline.

The primary cost drivers within R&D are tied directly to your lead assets. You see the costs associated with the INSPIRE DUCHENNE trial for SGT-003 ramping up as enrollment continues. For instance, in Q2 2025, the R&D increase was largely due to a $9.9 million rise in SGT-003 costs related to manufacturing, regulatory, and clinical activities.

Manufacturing and process development costs are a major component, particularly for SGT-003. While the FDA meeting was pushed to H1 2026, the company is still pushing forward with commercial-readiness CMC activities, with the expectation that the Process Performance Qualification (PPQ) manufacturing batches for SGT-003 will be completed in 2026. This prep work is expensive, even before commercial launch.

General and Administrative (G&A) expenses were approximately $9.2 million in Q3 2025, up from $7.9 million the prior year. This increase is often tied to personnel costs needed to support the growing clinical and regulatory complexity of advancing multiple programs simultaneously.

When you look at other specific costs, you can see the ebb and flow of program-specific spending. For example, in Q3 2025, costs for SGT-212 saw a decrease of $3.3 million, partially due to lower license and milestone related costs. Still, intellectual property maintenance and licensing fees are an ongoing, necessary cost of doing business in this space, as evidenced by prior periods showing increases in license fees for development programs.

The major buckets of spending look like this:

• Costs for SGT-003: Primarily manufacturing, regulatory, and clinical costs.
• Costs for SGT-601: Including manufacturing costs.
• Costs for SGT-212: Including clinical and research costs, and license/milestone payments.
• Personnel related expenses across R&D and G&A.

Finance: draft 13-week cash view by Friday.

Solid Biosciences Inc. (SLDB) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of Solid Biosciences Inc. (SLDB) as of late 2025, and honestly, it looks exactly like what you'd expect from a company deep in clinical development. The revenue streams aren't from selling a drug yet; they are about future potential and non-product income sources that keep the lights on while the science matures.

Currently $0.0 in product revenue (pre-revenue clinical-stage company)

For the trailing twelve months ending September 30, 2025, Solid Biosciences revenue was reported as $0.00. This is the reality for a clinical-stage company focused on bringing a novel gene therapy to market. The Q3 2025 reported revenue was also $0.0.

Potential future revenue from sales of approved gene therapies (e.g., SGT-003)

The primary long-term revenue driver is the potential commercialization of SGT-003 for Duchenne muscular dystrophy. As of the October 31, 2025, safety cutoff, 23 participants had been dosed in the Phase 1/2 INSPIRE DUCHENNE trial. Solid Biosciences expects to dose 30 participants in total by early 2026 and plans to meet with the U.S. Food and Drug Administration (FDA) in the first half of 2026 to discuss potential registrational pathways. This is the inflection point that unlocks potential blockbuster sales, but until then, it's zero product revenue.

Upfront payments and milestones from AAV-SLB101 capsid licensing agreements

This is a key near-term, non-product revenue stream. Solid Biosciences is actively licensing its proprietary AAV-SLB101 capsid technology broadly. As of November 17, 2025, Solid Biosciences had executed over 30 agreements, including licenses, with corporations, institutions, and academic labs. For instance, the agreement with Kinea Bio confirms Solid receives an upfront fee and is eligible for development/sales milestones and tiered royalties, though the specific upfront amount wasn't disclosed. Similarly, financial terms for the non-exclusive license with Andelyn Biosciences were not disclosed.

Potential non-dilutive funding from government grants for rare disease research

While Solid Biosciences is pursuing multiple rare disease indications (Friedreich's ataxia with SGT-212, CPVT with SGT-501), specific dollar amounts received from government grants as of late 2025 aren't detailed in the latest public updates. This funding source is a crucial, non-dilutive component that supports the high Research and Development (R&D) spend.

Equity financing and public offerings to fund operations

Since product sales are absent, financing is the lifeblood. The company ended Q3 2025 with $236.1 million in cash, cash equivalents, and available-for-sale securities. Management projects this cash position will fund the operational runway into the first half of 2027. This is a critical metric to watch, especially given R&D expenses for Q3 2025 were $38.9 million. The company previously announced a Series C Financing of up to $50 Million in 2017, which, along with subsequent financing, built this current war chest.

Here's a quick snapshot of the key financial and operational data points underpinning these revenue expectations:

Metric	Value as of Late 2025	Context/Date
Trailing 12-Month Product Revenue	$0.00	TTM ending September 30, 2025
Cash Position	$236.1 million	As of September 30, 2025
Projected Cash Runway	Into the first half of 2027	Based on September 30, 2025 cash
SGT-003 Dosed Participants	23	As of October 31, 2025
AAV-SLB101 Licensing Agreements Executed	Over 30	As of November 17, 2025
Q3 2025 R&D Expense	$38.9 million	For the quarter ended September 30, 2025

The revenue model right now is entirely dependent on successfully executing these non-dilutive licensing deals and managing the cash burn until SGT-003 can potentially generate sales, which hinges on the planned H1 2026 FDA discussions.

You should keep an eye on the pace of new AAV-SLB101 deals, as these upfront payments directly offset the operating loss. The current non-product revenue streams are structured around:

• Upfront fees from new capsid licenses.
• Future milestone payments tied to partners advancing their AAV-SLB101 programs.
• Tiered royalties on net sales from commercialized partner products.
• Non-dilutive grant funding for pipeline programs.

The company's liquidity, sitting at $236.1 million as of the end of Q3 2025, is the buffer that allows them to pursue these revenue-generating milestones without immediate reliance on public offerings, though that remains a fallback option.