Solid Biosciences Inc. (SLDB): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el reino de vanguardia de la terapéutica genética, Solid Biosciences Inc. (SLDB) emerge como un faro de esperanza para pacientes que luchan contra la distrofia muscular de Duchenne, pionero en enfoques de terapia génica revolucionaria que podrían transformar fundamentalmente los tratamientos de trastornos genéticos raros. Al aprovechar las tecnologías sofisticadas de transferencia de genes y las colaboraciones estratégicas con las instituciones de investigación líderes, esta innovadora compañía de biotecnología no solo está realizando investigaciones, sino que elaboran posibles líneas de vida para pacientes con opciones médicas limitadas, empujando los límites de las intervenciones genéticas moleculares y ofrecen un glimpse en un un glimpse en un futuro donde las condiciones genéticas previamente no tratables finalmente podrían tener soluciones innovadoras.

Solid Biosciences Inc. (SLDB) - Modelo de negocios: asociaciones clave

Asociación de Investigación del Hospital Nacional de Niños

Solid Biosciences ha establecido una asociación de investigación colaborativa con el Nationwide Children's Hospital centrado en el desarrollo de la terapia génica para la distrofia muscular de Duchenne (DMD).

Detalles de la asociación	Enfoque de colaboración específico
Fecha de inicio de colaboración de investigación	2018
Compromiso de financiación de la investigación	$ 4.2 millones
Área de investigación principal	Terapia génica de distrofia muscular de Duchenne

Colaboración de la asociación de distrofia muscular

La compañía mantiene una colaboración de investigación activa con la Asociación de Distrofia Muscular (MDA).

• Subvención de investigación de MDA: $ 750,000
• Enfoque de investigación colaborativa: desarrollo de terapia genética
• Asociación establecida: 2016

Asociaciones institucionales de investigación académica

Institución	Enfoque de investigación	Compromiso de financiación
Escuela de Medicina de Harvard	Mecanismos de trastorno genético raros	$ 1.5 millones
Universidad de Stanford	Optimización de terapia génica	$ 1.2 millones
Instituto MIT Whitehead	Investigación de genética molecular	$900,000

Socios de desarrollo farmacéutico

Las posibles asociaciones de desarrollo farmacéutico incluyen discusiones en curso con principales empresas de biotecnología.

• Pfizer: colaboración de terapia génica exploratoria
• Terapéutica de Sarepta: asociación potencial de tratamiento con DMD
• Farmacéutico Ultragenyx: investigación de trastorno genético raro

Solid Biosciences Inc. (SLDB) - Modelo de negocio: actividades clave

Desarrollo de tratamientos de terapia génica para la distrofia muscular de Duchenne

Las biosciencias sólidas se centran en el desarrollo de tratamientos de terapia génica específicamente dirigida a la distrofia muscular de Duchenne (DMD). A partir del cuarto trimestre de 2023, la compañía ha invertido $ 47.2 millones en investigación y desarrollo para terapias genéticas específicas de DMD.

Área de enfoque de investigación	Monto de la inversión	Etapa de desarrollo
Terapia génica Sgt-001	$ 23.5 millones	Ensayo clínico de fase 1/2
Terapia génica de micro-discyfina	$ 15.7 millones	Desarrollo preclínico

Realización de ensayos clínicos para terapias genéticas innovadoras

La Compañía ha estado realizando activamente ensayos clínicos con parámetros específicos:

• Ensayos clínicos activos totales: 2 a partir de enero de 2024
• Población de pacientes matriculada: 36 pacientes con DMD
• Gastos totales de ensayos clínicos en 2023: $ 18.3 millones

Avance de tecnologías de transferencia de genes patentados

Solid Biosciences ha desarrollado tecnologías de transferencia de genes patentadas con una inversión de investigación significativa.

Plataforma tecnológica	Estado de patente	Inversión de I + D
Sistema de vectores de transferencia de genes	5 patentes activas	$ 12.6 millones
Plataforma de optimización de micro-discófina	3 patentes pendientes	$ 8.9 millones

Investigación y desarrollo de intervenciones genéticas moleculares

La estrategia de I + D de la compañía se centra en las intervenciones genéticas moleculares con asignación específica de recursos:

• Gastos totales de I + D en 2023: $ 62.4 millones
• Personal de investigación: 48 científicos especializados
• Publicaciones de investigación anuales: 7 estudios revisados ​​por pares

Desglose de asignación de presupuesto de investigación para 2024:

Categoría de investigación	Porcentaje de presupuesto	Cantidad asignada
Investigación preclínica	45%	$ 28.1 millones
Desarrollo de ensayos clínicos	35%	$ 21.8 millones
Mejora de la plataforma de tecnología	20%	$ 12.5 millones

Solid Biosciences Inc. (SLDB) - Modelo de negocio: recursos clave

Tecnologías de plataforma de transferencia de genes patentados

Solid Biosciences Inc. se ha desarrollado Tecnologías de transferencia de genes basadas en AAV específicamente enfocado en el tratamiento con distrofia muscular de Duchenne (DMD).

Plataforma tecnológica	Enfoque específico	Etapa de desarrollo
Sargento 001	Terapia génica de micro-discyfina	Ensayo clínico de fase 1/2
Ingeniería de la cápside AAV	Mecanismos mejorados de suministro de genes	Investigación preclínica

Equipo de investigación de ingeniería genética especializada

Composición del equipo de investigación a partir de 2024:

• Personal de investigación total: 42 científicos especializados
• Titulares de doctorado: 28
• Expertos de ingeniería genética: 15

Cartera de propiedades intelectuales

Categoría de patente	Número de patentes	Duración de protección de patentes
Tecnologías de terapia génica	12 patentes otorgadas	Hasta 2037-2040
Modificaciones de la cápside AAV	7 solicitudes de patentes pendientes	Protección potencial hasta 2042

Instalaciones avanzadas de laboratorio e investigación

Detalles de la infraestructura de investigación:

• Espacio total de la instalación de investigación: 22,000 pies cuadrados
• Laboratorios de bioseguridad de nivel 2: 4
• Equipo avanzado de secuenciación de genes: 6 plataformas

Datos de ensayos clínicos y experiencia en investigación

Parámetro de ensayo clínico	Datos cuantitativos
Ensayos clínicos totales realizados	3 pruebas completadas
Participantes de los pacientes	47 participantes totales
Publicaciones de investigación	18 publicaciones revisadas por pares

Solid Biosciences Inc. (SLDB) - Modelo de negocio: propuestas de valor

Terapias dirigidas para trastornos musculares genéticos raros

Las biosciencias sólidas se centran en el desarrollo de tratamientos de terapia génica para la distrofia muscular de Duchenne (DMD). A partir del cuarto trimestre de 2023, el candidato terapéutico principal de la compañía SGT-001 representa un enfoque terapéutico genético de precisión para pacientes con DMD.

Candidato terapéutico	Trastorno objetivo	Etapa de desarrollo
Sargento 001	Distrofia muscular de Duchenne	Ensayo clínico de fase 1/2

Posibles tratamientos innovadores para la distrofia muscular de Duchenne

La tecnología de transferencia de genes de la compañía se dirige específicamente a pacientes con DMD con mutaciones susceptibles a la terapia génica de microdistrofina.

• Población de pacientes con DMD estimada en Estados Unidos: 10,000-15,000
• Oportunidad de mercado actual: aproximadamente $ 1.2 mil millones del mercado anual potencial
• No hay terapia génica aprobada por la FDA para DMD a partir de 2024

Plataformas de tecnología de transferencia de genes innovadoras

Plataforma tecnológica	Características únicas	Estado de desarrollo
Plataforma de sargento	Diseño patentado de la cápside AAV	Estadio preclínico/clínico

Enfoques terapéuticos genéticos de precisión

Solid Biosciences utiliza técnicas avanzadas de ingeniería genética para desarrollar terapias dirigidas.

• Gastos de investigación y desarrollo (2023): $ 35.4 millones
• Equivalentes en efectivo y efectivo (tercer trimestre de 2023): $ 98.4 millones

Esperanza para pacientes con opciones de tratamiento limitadas

La estrategia terapéutica de la compañía aborda una necesidad médica crítica insatisfecha de pacientes con DMD sin opciones de tratamiento integrales actuales.

Impacto del paciente	Beneficio potencial
Restauración del gen de micro-distrofina	Mejora de la función muscular potencial

Solid Biosciences Inc. (SLDB) - Modelo de negocios: relaciones con los clientes

Compromiso directo con grupos de defensa del paciente

A partir del cuarto trimestre de 2023, las sólidas biociencias participadas en 7 organizaciones de defensa de pacientes de distrofia muscular, incluida la distrofia muscular del proyecto principal.

Interacciones del grupo de defensa	Número de compromisos
Conferencias de pacientes	4 por año
Reuniones del grupo de apoyo	12 por año

Comunicación de ensayos clínicos transparentes

La empresa mantiene Transparencia del ensayo clínico en tiempo real a través de múltiples canales de comunicación.

• Clinicaltrials.gov listados: 3 ensayos activos
• Actualizaciones del sitio web: informes mensuales de progreso
• Portal directo de comunicación del paciente

Programas personalizados de apoyo al paciente

Las sólidas Biosciences invirtieron $ 1.2 millones en infraestructura de apoyo al paciente en 2023.

Categoría del programa de apoyo	Inversión anual
Servicios de navegación del paciente	$450,000
Asesoramiento genético	$350,000
Coordinación de asistencia financiera	$400,000

Colaboración de la comunidad científica

Asociaciones de investigación colaborativa a partir de 2023: 9 instituciones académicas, 5 centros de investigación.

Actualizaciones periódicas sobre el progreso de la investigación

Métricas de comunicación de investigación para 2023:

• Publicaciones científicas: 6
• Presentaciones de conferencia: 12
• Informes de inversores/analistas: 4 sesiones trimestrales

Solid Biosciences Inc. (SLDB) - Modelo de negocios: canales

Comunicaciones directas de investigación médica

Solid BioSciences utiliza canales de comunicación directa con:

• Centros de investigación de enfermedades genéticas raras
• Clínicas especializadas de distrofia muscular
• Instituciones de investigación de terapia genética

Canal de comunicación	Número de contactos directos	Frecuencia de interacción
Instituciones de investigación	42	Trimestral
Centros de investigación clínica	28	Bimensual
Clínicas genéticas especializadas	19	Mensual

Conferencias y presentaciones científicas

Detalles de participación de la conferencia:

• Conferencia anual de la Asociación de Distrofia Muscular
• Sociedad Americana de Gene & Conferencia de terapia celular
• Simposio internacional de investigación de enfermedades raras

Conferencia	Presentaciones en 2023	Alcance de la audiencia
Conferencia MDA	3	1.200 investigadores
Conferencia ASGCT	2	950 especialistas

Plataformas de relaciones con los inversores

Los canales de comunicación de los inversores incluyen:

• Llamadas de ganancias trimestrales
• Reuniones anuales de accionistas
• Sebinarios web de presentación de inversores

Plataforma	Frecuencia	Compromiso de los inversores
Llamadas de ganancias	4 veces/año	350-450 participantes
Seminarios web de inversores	2 veces/año	250-300 participantes

Publicaciones de la revista médica

Métricas de investigación publicadas:

• Publicaciones revisadas por pares: 7 en 2023
• Índice de citas: 42 citas totales
• Rango de factor de impacto: 3.2 - 6.5

Redes especializadas de tratamiento de enfermedades raras

Detalles de colaboración de red:

• Duchenne Red de tratamiento de distrofia muscular
• Grupo de investigación colaborativa de trastornos genéticos raros
• Consorcio internacional de investigación de distrofia muscular

Red	Instituciones miembros	Proyectos de colaboración
Red de tratamiento con DMD	18	5 proyectos activos
Grupo de trastornos genéticos raros	26	3 proyectos activos

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

Pacientes de distrofia muscular de Duchenne

Población de pacientes estimada: 15,000-20,000 en los Estados Unidos

Grupo de edad	Predominio	Potencial de tratamiento
0-10 años	45%	Alta oportunidad de intervención terapéutica
11-20 años	35%	Oportunidad de intervención terapéutica moderada
Más de 21 años	20%	Oportunidad de intervención terapéutica limitada

Instituciones de investigación de desorden genético

Número de centros de investigación dedicados: 87 a nivel mundial

• Asignación anual de presupuesto de investigación: $ 42.6 millones
• Programas de investigación de trastorno genético activo: 214
• Asociaciones de investigación colaborativa: 36

Especialistas neuromusculares pediátricos

Categoría especialista	Total de especialistas	Centrado en trastornos genéticos raros
Neurólogos	1,250	376
Genetistas	890	412

Enfermedades raras comunidades de pacientes

Grupos de apoyo de pacientes registrados totales: 215

• Miembros de la comunidad de pacientes en línea: 47,300
• Organizaciones de defensa activa: 89
• Participantes del evento anual de concientización: 22,600

Socios de investigación farmacéutica

Tipo de socio	Número de socios	Estado de colaboración
Instituciones académicas	24	Activo
Compañías farmacéuticas	12	Colaborativo
Centros de investigación de biotecnología	18	Comprometido

Solid Biosciences Inc. (SLDB) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

Para el año fiscal 2023, Solid BioSciences informó gastos de I + D de $ 56.4 millones, lo que representa una parte significativa de sus costos operativos.

Año	Gastos de I + D	Porcentaje de gastos totales
2022	$ 48.7 millones	67.3%
2023	$ 56.4 millones	71.2%

Costos de gestión de ensayos clínicos

Los gastos de ensayos clínicos para biosciencias sólidas en 2023 totalizaron aproximadamente $ 32.1 millones.

• Ensayos de distrofia muscular de Fase I/II Duchenne: $ 18.5 millones
• Costos de estudio preclínico: $ 7.2 millones
• Gastos de cumplimiento regulatorio: $ 6.4 millones

Mantenimiento de la propiedad intelectual

Los costos anuales de propiedad intelectual para 2023 fueron de $ 2.3 millones, que cubren la presentación de patentes, el mantenimiento y la protección legal.

Infraestructura de laboratorio y tecnología

Componente de infraestructura	Costo anual
Equipo de laboratorio	$ 4.7 millones
Sistemas tecnológicos	$ 2.1 millones
Mantenimiento de la instalación	$ 1.9 millones

Reclutamiento de talento científico especializado

El personal total y los gastos de reclutamiento para 2023 fueron de $ 22.6 millones.

• Salarios científicos senior: $ 12.4 millones
• Compensación del personal de investigación: $ 7.8 millones
• Reclutamiento y adquisición de talento: $ 2.4 millones

Estructura total de costos operativos para 2023: aproximadamente $ 117.4 millones

Solid Biosciences Inc. (SLDB) - Modelo de negocios: flujos de ingresos

Licencias potenciales de productos terapéuticos futuros

A partir del cuarto trimestre de 2023, Solid Biosciences no tiene ingresos activos de licencia. El enfoque principal de la compañía sigue siendo el desarrollo de tratamientos de terapia génica para la distrofia muscular de Duchenne (DMD).

Subvenciones de investigación y financiación del gobierno

Año	Fuente de financiación	Cantidad
2023	Subvención de investigación de NIH	$ 1.2 millones
2022	Apoyo de investigación de MDDA	$850,000

Asociaciones de investigación colaborativa

Colaboración actual de investigación con Ultragenyx Pharmaceutical Inc. con posibles pagos de hitos futuros.

Pagos potenciales de hitos de asociaciones farmacéuticas

• Estructura de pago de hito potencial con ultragenyx: hasta $ 120 millones en hitos comerciales y de desarrollo potenciales
• No hay pagos de hito confirmados recibidos a partir del cuarto trimestre de 2023

Comercialización de productos terapéuticos futuros

No hay ingresos comerciales actuales. El producto principal de la empresa SGT-001 permanece en la fase de desarrollo clínico.

Métrica financiera	Valor 2023
Ingresos totales	$ 2.1 millones
Ingresos de subvención de investigación	$ 1.2 millones

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.