Solid Biosciences Inc. (SLDB): Business Model Canvas

Im hochmodernen Bereich der Gentherapie erweist sich Solid Biosciences Inc. (SLDB) als Hoffnungsträger für Patienten mit Duchenne-Muskeldystrophie und leistet Pionierarbeit bei revolutionären Gentherapieansätzen, die die Behandlung seltener genetischer Störungen grundlegend verändern könnten. Durch den Einsatz hochentwickelter Gentransfertechnologien und strategischer Kooperationen mit führenden Forschungseinrichtungen betreibt dieses innovative Biotech-Unternehmen nicht nur Forschung – es schafft potenzielle Lebensadern für Patienten mit begrenzten medizinischen Möglichkeiten, verschiebt die Grenzen molekulargenetischer Eingriffe und bietet einen Blick in eine Zukunft, in der es für bisher unbehandelbare genetische Erkrankungen endlich bahnbrechende Lösungen geben könnte.

Solid Biosciences Inc. (SLDB) – Geschäftsmodell: Wichtige Partnerschaften

Bundesweite Forschungspartnerschaft für Kinderkrankenhäuser

Solid Biosciences hat eine kooperative Forschungspartnerschaft mit dem Nationwide Children's Hospital gegründet, die sich auf die Entwicklung von Gentherapien für Duchenne-Muskeldystrophie (DMD) konzentriert.

Einzelheiten zur Partnerschaft	Spezifischer Fokus auf Zusammenarbeit
Startdatum der Forschungskooperation	2018
Zusage zur Forschungsförderung	4,2 Millionen US-Dollar
Primärer Forschungsbereich	Gentherapie bei Duchenne-Muskeldystrophie

Zusammenarbeit der Muskeldystrophie-Vereinigung

Das Unternehmen pflegt eine aktive Forschungskooperation mit der Muscular Dystrophy Association (MDA).

• MDA-Forschungsstipendium: 750.000 US-Dollar
• Verbundforschungsschwerpunkt: Gentherapieentwicklung
• Partnerschaft gegründet: 2016

Institutionelle Partnerschaften für akademische Forschung

Institution	Forschungsschwerpunkt	Finanzierungszusage
Harvard Medical School	Seltene genetische Störungsmechanismen	1,5 Millionen Dollar
Stanford-Universität	Optimierung der Gentherapie	1,2 Millionen US-Dollar
MIT Whitehead Institute	Molekulargenetische Forschung	$900,000

Pharmazeutische Entwicklungspartner

Zu möglichen pharmazeutischen Entwicklungspartnerschaften gehören laufende Gespräche mit großen Biotechnologieunternehmen.

• Pfizer: Explorative Gentherapie-Zusammenarbeit
• Sarepta Therapeutics: Potenzielle DMD-Behandlungspartnerschaft
• Ultragenyx Pharmaceutical: Forschung zu seltenen genetischen Störungen

Solid Biosciences Inc. (SLDB) – Geschäftsmodell: Hauptaktivitäten

Entwicklung gentherapeutischer Behandlungen für Duchenne-Muskeldystrophie

Solid Biosciences konzentriert sich auf die Entwicklung gentherapeutischer Behandlungen, die speziell auf die Duchenne-Muskeldystrophie (DMD) abzielen. Bis zum vierten Quartal 2023 hat das Unternehmen 47,2 Millionen US-Dollar in Forschung und Entwicklung für DMD-spezifische Gentherapien investiert.

Forschungsschwerpunktbereich	Investitionsbetrag	Entwicklungsphase
SGT-001 Gentherapie	23,5 Millionen US-Dollar	Klinische Phase-1/2-Studie
Mikrodystrophin-Gentherapie	15,7 Millionen US-Dollar	Präklinische Entwicklung

Durchführung klinischer Studien für innovative Gentherapien

Das Unternehmen führt aktiv klinische Studien mit bestimmten Parametern durch:

• Gesamtzahl aktiver klinischer Studien: 2, Stand Januar 2024
• Eingeschriebene Patientenpopulation: 36 DMD-Patienten
• Gesamtausgaben für klinische Studien im Jahr 2023: 18,3 Millionen US-Dollar

Weiterentwicklung proprietärer Gentransfertechnologien

Solid Biosciences hat mit erheblichen Forschungsinvestitionen proprietäre Gentransfertechnologien entwickelt.

Technologieplattform	Patentstatus	F&E-Investitionen
Gentransfer-Vektorsystem	5 aktive Patente	12,6 Millionen US-Dollar
Mikro-Dystrophin-Optimierungsplattform	3 angemeldete Patente	8,9 Millionen US-Dollar

Forschung und Entwicklung molekulargenetischer Interventionen

Die F&E-Strategie des Unternehmens konzentriert sich auf molekulargenetische Interventionen mit gezielter Ressourcenallokation:

• Gesamtausgaben für Forschung und Entwicklung im Jahr 2023: 62,4 Millionen US-Dollar
• Forschungspersonal: 48 spezialisierte Wissenschaftler
• Jährliche Forschungspublikationen: 7 peer-reviewte Studien

Aufschlüsselung der Forschungsbudgetzuteilung für 2024:

Forschungskategorie	Prozentsatz des Budgets	Zugeteilter Betrag
Präklinische Forschung	45%	28,1 Millionen US-Dollar
Entwicklung klinischer Studien	35%	21,8 Millionen US-Dollar
Verbesserung der Technologieplattform	20%	12,5 Millionen US-Dollar

Solid Biosciences Inc. (SLDB) – Geschäftsmodell: Schlüsselressourcen

Proprietäre Plattformtechnologien für den Gentransfer

Solid Biosciences Inc. hat sich entwickelt AAV-basierte Gentransfertechnologien Der Schwerpunkt liegt insbesondere auf der Behandlung der Duchenne-Muskeldystrophie (DMD).

Technologieplattform	Spezifischer Fokus	Entwicklungsphase
SGT-001	Mikro-Dystrophin-Gentherapie	Klinische Phase-1/2-Studie
AAV-Kapsid-Engineering	Verbesserte Mechanismen zur Genabgabe	Präklinische Forschung

Spezialisiertes Gentechnik-Forschungsteam

Zusammensetzung des Forschungsteams ab 2024:

• Gesamtes Forschungspersonal: 42 spezialisierte Wissenschaftler
• Doktoranden: 28
• Gentechnikexperten: 15

Portfolio für geistiges Eigentum

Patentkategorie	Anzahl der Patente	Patentschutzdauer
Gentherapie-Technologien	12 erteilte Patente	Bis 2037-2040
AAV-Kapsidmodifikationen	7 anhängige Patentanmeldungen	Möglicher Schutz bis 2042

Fortschrittliche Labor- und Forschungseinrichtungen

Details zur Forschungsinfrastruktur:

• Gesamtfläche der Forschungseinrichtung: 22.000 Quadratmeter
• Labore der Biosicherheitsstufe 2: 4
• Fortschrittliche Gensequenzierungsausrüstung: 6 Plattformen

Klinische Studiendaten und Forschungsexpertise

Parameter für klinische Studien	Quantitative Daten
Gesamtzahl der durchgeführten klinischen Studien	3 abgeschlossene Versuche
Patiententeilnehmer	Insgesamt 47 Teilnehmer
Forschungspublikationen	18 peer-reviewte Veröffentlichungen

Solid Biosciences Inc. (SLDB) – Geschäftsmodell: Wertversprechen

Gezielte Therapien für seltene genetische Muskelerkrankungen

Solid Biosciences konzentriert sich auf die Entwicklung gentherapeutischer Behandlungen für Duchenne-Muskeldystrophie (DMD). Ab dem vierten Quartal 2023 stellt der führende therapeutische Kandidat des Unternehmens, SGT-001, eine präziser gentherapeutischer Ansatz für DMD-Patienten.

Therapeutischer Kandidat	Zielstörung	Entwicklungsphase
SGT-001	Duchenne-Muskeldystrophie	Klinische Phase-1/2-Studie

Mögliche bahnbrechende Behandlungen für Duchenne-Muskeldystrophie

Die Gentransfertechnologie des Unternehmens richtet sich speziell an DMD-Patienten mit Mutationen, die für eine Mikro-Dystrophin-Gentherapie geeignet sind.

• Geschätzte DMD-Patientenpopulation in den Vereinigten Staaten: 10.000–15.000
• Aktuelle Marktchance: Ungefähr 1,2 Milliarden US-Dollar potenzieller Jahresmarkt
• Ab 2024 gibt es keine von der FDA zugelassene Gentherapie für DMD

Innovative Gentransfer-Technologieplattformen

Technologieplattform	Einzigartige Eigenschaften	Entwicklungsstand
SGT-Plattform	Proprietäres AAV-Kapsid-Design	Präklinisches/klinisches Stadium

Präzise genetische Therapieansätze

Solid Biosciences nutzt fortschrittliche gentechnische Techniken, um gezielte Therapien zu entwickeln.

• Forschungs- und Entwicklungskosten (2023): 35,4 Millionen US-Dollar
• Zahlungsmittel und Zahlungsmitteläquivalente (3. Quartal 2023): 98,4 Millionen US-Dollar

Hoffnung für Patienten mit begrenzten Behandlungsmöglichkeiten

Die Therapiestrategie des Unternehmens adressiert einen kritischen ungedeckten medizinischen Bedarf für DMD-Patienten, für den es derzeit keine umfassenden Behandlungsmöglichkeiten gibt.

Auswirkungen auf den Patienten	Potenzieller Nutzen
Wiederherstellung des Mikrodystrophin-Gens	Mögliche Verbesserung der Muskelfunktion

Solid Biosciences Inc. (SLDB) – Geschäftsmodell: Kundenbeziehungen

Direkte Zusammenarbeit mit Patienteninteressengruppen

Seit dem vierten Quartal 2023 arbeitete Solid Biosciences mit sieben Organisationen zusammen, die sich für Patienten mit Muskeldystrophie einsetzen, darunter auch mit dem Parent Project Muscular Dystrophy.

Interaktionen mit Interessengruppen	Anzahl der Engagements
Patientenkonferenzen	4 pro Jahr
Selbsthilfegruppentreffen	12 pro Jahr

Transparente Kommunikation über klinische Studien

Das Unternehmen unterhält Transparenz klinischer Studien in Echtzeit über mehrere Kommunikationskanäle.

• ClinicalTrials.gov-Einträge: 3 aktive Studien
• Website-Updates: Monatliche Fortschrittsberichte
• Direktes Patientenkommunikationsportal

Personalisierte Patientenunterstützungsprogramme

Solid Biosciences investierte im Jahr 2023 1,2 Millionen US-Dollar in die Infrastruktur zur Patientenunterstützung.

Kategorie „Unterstützungsprogramm“.	Jährliche Investition
Patientennavigationsdienste	$450,000
Genetische Beratung	$350,000
Koordinierung der finanziellen Unterstützung	$400,000

Zusammenarbeit der wissenschaftlichen Gemeinschaft

Verbundforschungspartnerschaften ab 2023: 9 akademische Einrichtungen, 5 Forschungszentren.

Regelmäßige Updates zum Forschungsfortschritt

Forschungskommunikationskennzahlen für 2023:

• Wissenschaftliche Veröffentlichungen: 6
• Konferenzvorträge: 12
• Investoren-/Analystenbriefings: 4 vierteljährliche Sitzungen

Solid Biosciences Inc. (SLDB) – Geschäftsmodell: Kanäle

Direkte medizinische Forschungskommunikation

Solid Biosciences nutzt direkte Kommunikationskanäle mit:

• Forschungszentren für seltene genetische Krankheiten
• Spezialkliniken für Muskeldystrophie
• Forschungseinrichtungen für Gentherapie

Kommunikationskanal	Anzahl der direkten Kontakte	Häufigkeit der Interaktion
Forschungseinrichtungen	42	Vierteljährlich
Klinische Forschungszentren	28	Zweimonatlich
Spezialisierte genetische Kliniken	19	Monatlich

Wissenschaftliche Konferenzen und Präsentationen

Details zur Konferenzteilnahme:

• Jährliche Konferenz der Muscular Dystrophy Association
• Amerikanische Gesellschaft für Gene & Zelltherapie-Konferenz
• Internationales Forschungssymposium für seltene Krankheiten

Konferenz	Präsentationen im Jahr 2023	Zielgruppenreichweite
MDA-Konferenz	3	1.200 Forscher
ASGCT-Konferenz	2	950 Spezialisten

Investor-Relations-Plattformen

Zu den Kommunikationskanälen für Investoren gehören:

• Vierteljährliche Gewinnmitteilungen
• Jährliche Aktionärsversammlungen
• Webinare zur Investorenpräsentation

Plattform	Häufigkeit	Investorenengagement
Gewinnaufrufe	4 Mal/Jahr	350-450 Teilnehmer
Investoren-Webinare	2 Mal/Jahr	250-300 Teilnehmer

Veröffentlichungen in medizinischen Fachzeitschriften

Veröffentlichte Forschungsmetriken:

• Von Experten begutachtete Veröffentlichungen: 7 im Jahr 2023
• Zitationsindex: 42 Gesamtzitate
• Schlagfaktorbereich: 3,2 – 6,5

Spezialisierte Netzwerke zur Behandlung seltener Krankheiten

Details zur Netzwerkzusammenarbeit:

• Netzwerk zur Behandlung von Duchenne-Muskeldystrophie
• Kollaborative Forschungsgruppe für seltene genetische Störungen
• Internationales Forschungskonsortium für Muskeldystrophie

Netzwerk	Mitgliedsinstitutionen	Gemeinschaftsprojekte
DMD-Behandlungsnetzwerk	18	5 aktive Projekte
Gruppe für seltene genetische Störungen	26	3 aktive Projekte

Solid Biosciences Inc. (SLDB) – Geschäftsmodell: Kundensegmente

Patienten mit Duchenne-Muskeldystrophie

Geschätzte Patientenpopulation: 15.000–20.000 in den Vereinigten Staaten

Altersgruppe	Prävalenz	Behandlungspotenzial
0-10 Jahre	45%	Hohe therapeutische Interventionsmöglichkeiten
11-20 Jahre	35%	Moderate therapeutische Interventionsmöglichkeit
21+ Jahre	20%	Begrenzte therapeutische Interventionsmöglichkeiten

Forschungseinrichtungen für genetische Störungen

Anzahl dedizierter Forschungszentren: 87 weltweit

• Jährliche Zuweisung des Forschungsbudgets: 42,6 Millionen US-Dollar
• Aktive Forschungsprogramme für genetische Störungen: 214
• Forschungskooperationen: 36

Pädiatrische neuromuskuläre Spezialisten

Fachkategorie	Totale Spezialisten	Konzentriert sich auf seltene genetische Störungen
Neurologen	1,250	376
Genetiker	890	412

Patientengemeinschaften für seltene Krankheiten

Insgesamt registrierte Patientenselbsthilfegruppen: 215

• Mitglieder der Online-Patientengemeinschaft: 47.300
• Aktive Interessenvertretungen: 89
• Jährliche Teilnehmer der Sensibilisierungsveranstaltung: 22.600

Pharmazeutische Forschungspartner

Partnertyp	Anzahl der Partner	Kooperationsstatus
Akademische Institutionen	24	Aktiv
Pharmaunternehmen	12	Kollaborativ
Biotech-Forschungszentren	18	Verlobt

Solid Biosciences Inc. (SLDB) – Geschäftsmodell: Kostenstruktur

Umfangreiche Forschungs- und Entwicklungskosten

Für das Geschäftsjahr 2023 meldete Solid Biosciences Forschungs- und Entwicklungskosten in Höhe von 56,4 Millionen US-Dollar, was einen erheblichen Teil seiner Betriebskosten darstellt.

Jahr	F&E-Ausgaben	Prozentsatz der Gesamtausgaben
2022	48,7 Millionen US-Dollar	67.3%
2023	56,4 Millionen US-Dollar	71.2%

Kosten für das Management klinischer Studien

Die Ausgaben für klinische Studien für Solid Biosciences beliefen sich im Jahr 2023 auf insgesamt etwa 32,1 Millionen US-Dollar.

• Phase-I/II-Studien zur Duchenne-Muskeldystrophie: 18,5 Millionen US-Dollar
• Kosten der präklinischen Studie: 7,2 Millionen US-Dollar
• Kosten für die Einhaltung gesetzlicher Vorschriften: 6,4 Millionen US-Dollar

Aufrechterhaltung des geistigen Eigentums

Die jährlichen Kosten für geistiges Eigentum beliefen sich im Jahr 2023 auf 2,3 Millionen US-Dollar und deckten die Patentanmeldung, die Wartung und den Rechtsschutz ab.

Labor- und Technologieinfrastruktur

Infrastrukturkomponente	Jährliche Kosten
Laborausrüstung	4,7 Millionen US-Dollar
Technologiesysteme	2,1 Millionen US-Dollar
Anlagenwartung	1,9 Millionen US-Dollar

Spezialisierte wissenschaftliche Talentrekrutierung

Die gesamten Personal- und Rekrutierungskosten beliefen sich im Jahr 2023 auf 22,6 Millionen US-Dollar.

• Gehälter für leitende Wissenschaftler: 12,4 Millionen US-Dollar
• Vergütung des Forschungspersonals: 7,8 Millionen US-Dollar
• Rekrutierung und Talentakquise: 2,4 Millionen US-Dollar

Gesamtbetriebskostenstruktur für 2023: Ungefähr 117,4 Millionen US-Dollar

Solid Biosciences Inc. (SLDB) – Geschäftsmodell: Einnahmequellen

Mögliche zukünftige Lizenzierung therapeutischer Produkte

Im vierten Quartal 2023 verfügt Solid Biosciences über keine aktiven Lizenzeinnahmen. Der Hauptfokus des Unternehmens liegt weiterhin auf der Entwicklung gentherapeutischer Behandlungen für Duchenne-Muskeldystrophie (DMD).

Forschungsstipendien und staatliche Förderung

Jahr	Finanzierungsquelle	Betrag
2023	NIH-Forschungsstipendium	1,2 Millionen US-Dollar
2022	MDDA-Forschungsunterstützung	$850,000

Verbundforschungspartnerschaften

Aktuelle Forschungskooperation mit Ultragenyx Pharmaceutical Inc. mit möglichen zukünftigen Meilensteinzahlungen.

Potenzielle Meilensteinzahlungen aus Pharmakooperationen

• Mögliche Meilensteinzahlungsstruktur mit Ultragenyx: Bis zu 120 Millionen US-Dollar an potenziellen Entwicklungs- und kommerziellen Meilensteinen
• Bis zum vierten Quartal 2023 sind keine bestätigten Meilensteinzahlungen eingegangen

Zukünftige Kommerzialisierung therapeutischer Produkte

Keine aktuellen kommerziellen Einnahmen. Das Hauptprodukt des Unternehmens, SGT-001, befindet sich weiterhin in der klinischen Entwicklungsphase.

Finanzkennzahl	Wert 2023
Gesamtumsatz	2,1 Millionen US-Dollar
Einnahmen aus Forschungsstipendien	1,2 Millionen US-Dollar

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.