Solid Biosciences Inc. (SLDB): Business Model Canvas [Jan-2025 Mis à jour]

Dans le domaine de la pointe de la thérapeutique génétique, Solid Biosciences Inc. (SLDB) émerge comme un phare d'espoir pour les patients luttant contre la dystrophie musculaire de Duchenne, des approches de thérapie génique révolutionnaire pionnière qui pourraient fondamentalement transformer les traitements de troubles génétiques rares. En tirant parti des technologies de transfert de gènes sophistiquées et des collaborations stratégiques avec les principaux institutions de recherche, cette entreprise de biotechnologie innovante ne fait pas seulement des recherches - ils créent des lignes de vie potentielles pour les patients avec des options médicales limitées, repoussant les limites des interventions génétiques moléculaires et offrant un aperçu de un Future où les conditions génétiques auparavant impossibles à traiter pourraient enfin avoir des solutions révolutionnaires.

Solid Biosciences Inc. (SLDB) - Modèle commercial: partenariats clés

Partenariat de recherche à l'hôpital pour enfants à l'échelle nationale

Solid Biosciences a établi un partenariat de recherche collaboratif avec l'hôpital national pour enfants axé sur le développement de la thérapie génique pour la dystrophie musculaire de Duchenne (DMD).

Détails du partenariat	Focus de collaboration spécifique
Date de début de la collaboration de recherche	2018
Engagement de financement de la recherche	4,2 millions de dollars
Domaine de recherche primaire	Dystrophie musculaire Dystrophie de Duchenne Thérapie génique

Collaboration Association de la dystrophie musculaire

La société maintient une collaboration de recherche active avec la Muscular Dystrophy Association (MDA).

• MDA Research Grant: 750 000 $
• Focus de recherche collaborative: développement de la thérapie génétique
• Partenariat établi: 2016

Partenariats institutionnels de recherche universitaire

Institution	Focus de recherche	Engagement de financement
École de médecine de Harvard	Mécanismes de troubles génétiques rares	1,5 million de dollars
Université de Stanford	Optimisation de la thérapie génique	1,2 million de dollars
Institut du MIT Whitehead	Recherche de génétique moléculaire	$900,000

Partenaires de développement pharmaceutique

Les partenariats potentiels de développement pharmaceutique comprennent des discussions en cours avec les grandes sociétés de biotechnologie.

• Pfizer: collaboration exploratoire sur la thérapie génique
• Sarepta Therapeutics: partenaire de traitement DMD potentiel
• Ultragenyx Pharmaceutique: Recherche de troubles génétiques rares

Solid Biosciences Inc. (SLDB) - Modèle d'entreprise: Activités clés

Développement de traitements de thérapie génique pour la dystrophie musculaire de Duchenne

Solid Biosciences se concentre sur le développement de traitements de thérapie génique ciblant spécifiquement la dystrophie musculaire de Duchenne (DMD). Au quatrième trimestre 2023, la société a investi 47,2 millions de dollars dans la recherche et le développement pour les thérapies génétiques spécifiques au DMD.

Domaine de mise au point de recherche	Montant d'investissement	Étape de développement
Thérapie génique du Sgt-001	23,5 millions de dollars	Essai clinique de phase 1/2
Thérapie génique de la micro-dystrophine	15,7 millions de dollars	Développement préclinique

Effectuer des essais cliniques pour des thérapies génétiques innovantes

La société a effectué activement des essais cliniques avec des paramètres spécifiques:

• Essais cliniques actifs totaux: 2 en janvier 2024
• Population de patients inscrits: 36 patients DMD
• Dépenses totales d'essais cliniques en 2023: 18,3 millions de dollars

Advanner les technologies de transfert de gènes propriétaires

Solid Biosciences a développé des technologies de transfert de gènes propriétaires avec des investissements de recherche importants.

Plate-forme technologique	Statut de brevet	Investissement en R&D
Système de vecteur de transfert de gènes	5 brevets actifs	12,6 millions de dollars
Plate-forme d'optimisation de la micro-dystrophine	3 brevets en attente	8,9 millions de dollars

Recherche et développement des interventions génétiques moléculaires

La stratégie de R&D de l'entreprise se concentre sur les interventions génétiques moléculaires avec une allocation spécifique des ressources:

• Total des dépenses de R&D en 2023: 62,4 millions de dollars
• Personnel de recherche: 48 scientifiques spécialisés
• Publications de recherche annuelles: 7 études évaluées par des pairs

Répartition de l'allocation du budget pour 2024:

Catégorie de recherche	Pourcentage de budget	Montant alloué
Recherche préclinique	45%	28,1 millions de dollars
Développement d'essais cliniques	35%	21,8 millions de dollars
Amélioration de la plate-forme technologique	20%	12,5 millions de dollars

Solid Biosciences Inc. (SLDB) - Modèle d'entreprise: Ressources clés

Technologies de plate-forme de transfert de gènes propriétaires

Solid Biosciences Inc. a développé Technologies de transfert de gènes basées sur l'AAV spécifiquement axé sur le traitement de la dystrophie musculaire de Duchenne (DMD).

Plate-forme technologique	Focus spécifique	Étape de développement
Sgt-001	Thérapie génique de la micro-dystrophine	Essai clinique de phase 1/2
AAV Capsid Engineering	Mécanismes de livraison de gènes améliorés	Recherche préclinique

Équipe de recherche spécialisée en génie génétique

Composition de l'équipe de recherche à partir de 2024:

• Personnel de recherche total: 42 scientifiques spécialisés
• Tapisseurs de doctorat: 28
• Experts en génie génétique: 15

Portefeuille de propriété intellectuelle

Catégorie de brevet	Nombre de brevets	Durée de protection des brevets
Technologies de thérapie génique	12 brevets accordés	Jusqu'en 2037-2040
Modifications AAV Capsid	7 demandes de brevet en instance	Protection potentielle jusqu'en 2042

Installations avancées de laboratoire et de recherche

Détails de l'infrastructure de recherche:

• Espace total des installations de recherche: 22 000 pieds carrés
• Laboratoires de niveau de biosécurité 2: 4
• Équipement avancé de séquençage des gènes: 6 plates-formes

Données d'essai cliniques et expertise en recherche

Paramètre d'essai clinique	Données quantitatives
Total des essais cliniques effectués	3 essais terminés
Participants aux patients	47 participants au total
Publications de recherche	18 publications évaluées par des pairs

Solid Biosciences Inc. (SLDB) - Modèle d'entreprise: propositions de valeur

Thérapies ciblées pour les troubles musculaires génétiques rares

Solid Biosciences se concentre sur le développement de traitements de thérapie génique pour la dystrophie musculaire de Duchenne (DMD). Au quatrième trimestre 2023, le principal candidat thérapeutique de la société Sgt-001 représente un approche thérapeutique génétique de précision pour les patients DMD.

Candidat thérapeutique	Trouble cible	Étape de développement
Sgt-001	Dystrophie musculaire de Duchenne	Essai clinique de phase 1/2

Traitements de percée potentiels pour la dystrophie musculaire de Duchenne

La technologie de transfert de gènes de l'entreprise cible spécifiquement les patients atteints de DMD avec des mutations susceptibles de thérapie génique de la micro-dystrophine.

• Population estimée des patients DMD aux États-Unis: 10 000-15 000
• Opportunité de marché actuel: Marché annuel potentiel d'environ 1,2 milliard de dollars
• Pas de thérapie génique approuvée par la FDA pour DMD à partir de 2024

Plateformes de technologie de transfert de gènes innovantes

Plate-forme technologique	Caractéristiques uniques	Statut de développement
Plate-forme Sgt	Conception de capside AAV propriétaire	Étape préclinique / clinique

Approches thérapeutiques génétiques de précision

Solid Biosciences utilise des techniques de génie génétique avancées pour développer des thérapies ciblées.

• Dépenses de recherche et de développement (2023): 35,4 millions de dollars
• Equivalents en espèces et en espèces (T3 2023): 98,4 millions de dollars

Espoir pour les patients avec des options de traitement limitées

La stratégie thérapeutique de l'entreprise répond à un besoin médical critique critique pour les patients DMD sans options de traitement complètes actuelles.

Impact du patient	Avantage potentiel
Restauration du gène de la micro-dystrophine	Amélioration potentielle de la fonction musculaire

Solid Biosciences Inc. (SLDB) - Modèle d'entreprise: relations avec les clients

Engagement direct avec les groupes de défense des patients

Depuis le Q4 2023, les biosciences solides se sont engagées avec 7 organisations de plaidoyer pour les patients dystrophiques musculaires, y compris la dystrophie musculaire du projet parent.

Interactions de groupe de plaidoyer	Nombre d'engagements
Conférences de patients	4 par an
Soutien des réunions de groupe	12 par an

Communication d'essai cliniques transparent

La société maintient Transparence des essais cliniques en temps réel à travers plusieurs canaux de communication.

• ClinicalTrials.gov Listes: 3 essais actifs
• Mises à jour du site Web: rapports d'étape mensuels
• Portail de communication patient direct

Programmes de soutien aux patients personnalisés

Solid Biosciences a investi 1,2 million de dollars dans les infrastructures de soutien aux patients en 2023.

Catégorie de programme de support	Investissement annuel
Services de navigation des patients	$450,000
Conseil génétique	$350,000
Coordination d'aide financière	$400,000

Collaboration communautaire scientifique

Partenariats de recherche collaborative à partir de 2023: 9 institutions académiques, 5 centres de recherche.

Mises à jour régulières des progrès de la recherche

Métriques de communication de recherche pour 2023:

• Publications scientifiques: 6
• Présentations de la conférence: 12
• Investisseur / analyste d'information: 4 séances trimestrielles

Solid Biosciences Inc. (SLDB) - Modèle d'entreprise: canaux

Communications de recherche médicale directes

Solid Biosciences utilise des canaux de communication directs avec:

• Centres de recherche sur les maladies génétiques rares
• Dystrophie musculaire Cliniques spécialisées
• Institutions de recherche en thérapie génétique

Canal de communication	Nombre de contacts directs	Fréquence d'interaction
Institutions de recherche	42	Trimestriel
Centres de recherche clinique	28	Bimensuel
Cliniques génétiques spécialisées	19	Mensuel

Conférences et présentations scientifiques

Détails de la participation de la conférence:

• Conférence annuelle de l'association de la dystrophie musculaire
• Société américaine de gène & Conférence de thérapie cellulaire
• Symposium international de recherche sur les maladies rares

Conférence	Présentations en 2023	Poutenir
Conférence MDA	3	1 200 chercheurs
Conférence ASGCT	2	950 spécialistes

Plateformes de relations avec les investisseurs

Les canaux de communication des investisseurs comprennent:

• Appels de résultats trimestriels
• Réunions annuelles des actionnaires
• Webinaires de présentation des investisseurs

Plate-forme	Fréquence	Engagement des investisseurs
Appels de gains	4 fois / an	350-450 participants
Webinaires des investisseurs	2 fois / an	250-300 participants

Publications des journaux médicaux

Métriques de recherche publiées:

• Publications évaluées par des pairs: 7 en 2023
• Indice de citation: 42 citations totales
• Plage du facteur d'impact: 3,2 - 6,5

Réseaux de traitement des maladies rares spécialisées

Détails de collaboration de réseau:

• Réseau de traitement de la dystrophie musculaire de Duchenne
• Groupe de recherche collaborative de troubles génétiques génétiques
• Consortium de recherche internationale sur la dystrophie musculaire

Réseau	Institutions membres	Projets collaboratifs
Réseau de traitement DMD	18	5 projets actifs
Groupe de troubles génétiques rares	26	3 projets actifs

Solid Biosciences Inc. (SLDB) - Modèle d'entreprise: segments de clientèle

Patients de dystrophie musculaire de Duchenne

Population estimée de patients: 15 000 à 20 000 aux États-Unis

Groupe d'âge	Prévalence	Potentiel de traitement
0-10 ans	45%	Opportunité d'intervention thérapeutique élevée
11-20 ans	35%	Opportunité d'intervention thérapeutique modérée
21 ans et plus	20%	Opportunité d'intervention thérapeutique limitée

Institutions de recherche sur les troubles génétiques

Nombre de centres de recherche dédiés: 87 à l'échelle mondiale

• Attribution annuelle du budget de la recherche: 42,6 millions de dollars
• Programmes de recherche active sur les troubles génétiques: 214
• Partenariats de recherche collaborative: 36

Spécialistes neuromusculaires pédiatriques

Catégorie spécialisée	Total des spécialistes	Axé sur les troubles génétiques rares
Neurologues	1,250	376
Généticiens	890	412

Communautés de patients atteints de maladies rares

Total des groupes de soutien aux patients enregistrés: 215

• Membres de la communauté des patients en ligne: 47 300
• Organisations de défense active: 89
• Participants à l'événement de sensibilisation annuel: 22 600

Partenaires de recherche pharmaceutique

Type de partenaire	Nombre de partenaires	Statut de collaboration
Institutions universitaires	24	Actif
Sociétés pharmaceutiques	12	Collaboratif
Centres de recherche biotechnologiques	18	Engagé

Solid Biosciences Inc. (SLDB) - Modèle d'entreprise: Structure des coûts

Dépenses de recherche et développement approfondies

Pour l'exercice 2023, Solid Biosciences a déclaré des dépenses de R&D de 56,4 millions de dollars, ce qui représente une partie importante de leurs coûts opérationnels.

Année	Dépenses de R&D	Pourcentage des dépenses totales
2022	48,7 millions de dollars	67.3%
2023	56,4 millions de dollars	71.2%

Coûts de gestion des essais cliniques

Les dépenses d'essais cliniques pour les biosciences solides en 2023 ont totalisé environ 32,1 millions de dollars.

• Phase I / II Essais de dystrophie musculaire Duchenne: 18,5 millions de dollars
• Coûts d'étude préclinique: 7,2 millions de dollars
• Dépenses de conformité réglementaire: 6,4 millions de dollars

Maintenance de la propriété intellectuelle

Les coûts annuels de propriété intellectuelle pour 2023 étaient de 2,3 millions de dollars, couvrant le dépôt, l'entretien et la protection juridique des brevets.

Infrastructure de laboratoire et technologique

Composant d'infrastructure	Coût annuel
Équipement de laboratoire	4,7 millions de dollars
Systèmes technologiques	2,1 millions de dollars
Entretien d'installation	1,9 million de dollars

Recrutement spécialisé des talents scientifiques

Le personnel total et les frais de recrutement pour 2023 étaient de 22,6 millions de dollars.

• Salaires des scientifiques seniors: 12,4 millions de dollars
• Compensation du personnel de recherche: 7,8 millions de dollars
• Recrutement et acquisition de talents: 2,4 millions de dollars

Structure totale des coûts opérationnels pour 2023: environ 117,4 millions de dollars

Solid Biosciences Inc. (SLDB) - Modèle d'entreprise: Strots de revenus

Licence potentielle de produits thérapeutiques futurs

Depuis le quatrième trimestre 2023, Solid Biosciences n'a pas de revenus de licence actifs. L'objectif principal de l'entreprise reste sur le développement de traitements de thérapie génique pour la dystrophie musculaire de Duchenne (DMD).

Subventions de recherche et financement gouvernemental

Année	Source de financement	Montant
2023	Subvention de recherche NIH	1,2 million de dollars
2022	Support de recherche MDDA	$850,000

Partenariats de recherche collaborative

Collaboration actuelle de recherche avec Ultragenyx Pharmaceutical Inc. avec des paiements potentiels de traits futurs.

Paiements de jalons potentiels provenant de partenariats pharmaceutiques

• Structure potentielle de paiement des jalons avec Ultragenyx: jusqu'à 120 millions de dollars de jalons de développement et commerciaux potentiels
• Aucun paiement de jalon confirmé reçu au quatrième trimestre 2023

Future commercialisation des produits thérapeutiques

Pas de revenus commerciaux actuels. Le produit principal de l'entreprise, le Sgt-001, reste en phase de développement clinique.

Métrique financière	Valeur 2023
Revenus totaux	2,1 millions de dollars
Revenus de subventions de recherche	1,2 million de dollars

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.