Voyager Therapeutics, Inc. (VYGR): Business Model Canvas

Im hochmodernen Bereich der neurologischen Therapeutika erweist sich Voyager Therapeutics (VYGR) als Pionier und transformiert die genetische Medizin durch bahnbrechende Innovationen in der Gentherapie. Durch die Nutzung fortschrittlicher Vektortechnik und gezielter genetischer Eingriffe ist dieser Biotech-Innovator bereit, Behandlungsansätze für komplexe neurologische Störungen zu revolutionieren und Patienten mit seltenen genetischen Erkrankungen, bei denen traditionelle medizinische Strategien versagen, Hoffnung zu geben. Ihr umfassender Business Model Canvas offenbart einen strategischen Entwurf, der wissenschaftliche Exzellenz, Kooperationspartnerschaften und transformatives medizinisches Potenzial miteinander verbindet und verspricht, die Landschaft personalisierter neurologischer Behandlungen neu zu gestalten.

Voyager Therapeutics, Inc. (VYGR) – Geschäftsmodell: Wichtige Partnerschaften

Zusammenarbeit mit Pharmaunternehmen

Voyager Therapeutics hat eine strategische Partnerschaft mit Neurocrine Biosciences geschlossen, die sich auf die Entwicklung von Gentherapien für neurologische Erkrankungen konzentriert.

Partner	Einzelheiten zur Partnerschaft	Finanzielle Bedingungen
Neurokrine Biowissenschaften	Gentherapie-Zusammenarbeit bei neurologischen Erkrankungen	Vorauszahlung in Höhe von 75 Millionen US-Dollar im Jahr 2018
AbbVie	Zusammenarbeit bei der Gentherapie der Parkinson-Krankheit	Mögliche Gesamtzahlungen in Höhe von 455 Millionen US-Dollar

Forschungskooperationen mit akademischen Institutionen

Voyager Therapeutics unterhält wichtige Forschungskooperationen mit führenden akademischen Forschungszentren.

• Massachusetts General Hospital
• Harvard Medical School
• Universität von Kalifornien, San Francisco

Strategische Allianzen in der Gentherapieentwicklung

Das Unternehmen hat strategische Allianzen entwickelt, um Gentherapietechnologien voranzutreiben.

Allianzpartner	Fokusbereich	Umfang der Zusammenarbeit
Pfizer	Erforschung genetisch neurologischer Erkrankungen	Mehrere präklinische Gentherapieprogramme
Sanofi	Seltene neurologische Störungen	Gemeinsame Forschung und Entwicklung

Lizenzvereinbarungen

Voyager Therapeutics hat mehrere Lizenzvereinbarungen für fortschrittliche neurologische Behandlungstechnologien abgeschlossen.

• MIT-Technologielizenz: Exklusive Rechte an der proprietären Gentherapie-Plattform
• Harvard-Biotechnologie-Lizenz: Technologien zur Behandlung neurologischer Erkrankungen

Gesamter Partnerschafts- und Lizenzumsatz für 2023: 28,3 Millionen US-Dollar

Voyager Therapeutics, Inc. (VYGR) – Geschäftsmodell: Hauptaktivitäten

Gentherapie-Forschung und -Entwicklung

Bis zum vierten Quartal 2023 hat Voyager Therapeutics 42,3 Millionen US-Dollar in Forschungs- und Entwicklungskosten im Zusammenhang mit der Entwicklung von Gentherapien investiert. Das Unternehmen konzentriert sich auf die Entwicklung von AAV-basierten Gentherapien zur Behandlung neurologischer Erkrankungen.

Forschungsschwerpunktbereich	Investition (2023)	Aktive Programme
Neurologische Störungen	42,3 Millionen US-Dollar	3 primäre Gentherapieprogramme
Seltene genetische Krankheiten	18,7 Millionen US-Dollar	2 vorklinische Studiengänge

Klinisches Studienmanagement

Voyager Therapeutics verwaltet derzeit vier aktive klinische Studien in mehreren neurologischen Indikationsbereichen.

• Phase-1/2-Studien zur Huntington-Krankheit
• Phase-1-Studien zur Parkinson-Krankheit
• Klinische Entwicklung einer Gentherapie im fortgeschrittenen Stadium

Präklinische und translationale Forschung

Das Unternehmen unterhält eine solide präklinische Forschungspipeline mit sechs potenziellen therapeutischen Kandidaten in der Entwicklung.

Forschungsphase	Anzahl der Kandidaten	Zielindikationen
Präklinisch	6 Kandidaten	Neurologische Störungen
Translationale Forschung	3 Programme	Seltene genetische Erkrankungen

Proprietäres Vektor-Engineering

Voyager hat sich entwickelt 15 einzigartige AAV-Vektorplattformen zur gezielten Genabgabe.

• Fortgeschrittene Kapsid-Engineering-Techniken
• Verbesserte Zielfähigkeiten für Nervengewebe
• Verbesserte Effizienz des Gentransfers

Innovation bei der Behandlung neurologischer Erkrankungen

Die Forschungsausgaben für Innovationen im Bereich neurologische Erkrankungen erreichten im Jahr 2023 27,6 Millionen US-Dollar.

Krankheitsbereich	Forschungsbudget	Innovationsfokus
Huntington-Krankheit	12,4 Millionen US-Dollar	Strategien zur Gen-Stummschaltung
Parkinson-Krankheit	9,2 Millionen US-Dollar	Abgabe des neurotrophen Faktors
Andere neurologische Erkrankungen	6 Millionen Dollar	Gezielte gentherapeutische Ansätze

Voyager Therapeutics, Inc. (VYGR) – Geschäftsmodell: Schlüsselressourcen

Fortschrittliche Gentherapie-Technologieplattformen

Voyager Therapeutics nutzt AAV-Gentherapie-Vektor-Engineering-Plattform mit folgenden Spezifikationen:

• Insgesamt hergestellte AAV-Kapsidvarianten: 171.000+
• Proprietäre Technologieplattform für gezielte Evolution
• Gentherapie-Vektordesignfunktionen für neurologische Erkrankungen

Kennzahlen zur Technologieplattform	Quantitative Daten
AAV-Kapsidvarianten	171,000+
Forschung & Entwicklungsinvestitionen (2023)	52,4 Millionen US-Dollar
Funktionen für das Design von Gentherapie-Vektoren	5 verschiedene Ziele für neurologische Erkrankungen

Portfolio für geistiges Eigentum

Voyager Therapeutics verfolgt eine solide Strategie für geistiges Eigentum:

• Insgesamt erteilte Patente: 84
• Ausstehende Patentanmeldungen: 36
• Patentfamilien für Gentherapietechnologien

Wissenschaftliche Forschungskompetenz

Zusammensetzung des Forschungsteams	Nummer
Total Research Scientists	68
Doktoranden	42
Spezialisten für Neurowissenschaften	24

Spezialisierte Laborinfrastruktur

Zu den Laboreinrichtungen gehören:

• Gesamtfläche der Forschungseinrichtung: 45.000 Quadratmeter
• Erweiterte Möglichkeiten zur Herstellung von Genvektoren
• Forschungslabore der Biosicherheitsstufen 2 und 3

Erfahrenes Management-Team

Führungsposition	Jahrelange Erfahrung
CEO	22 Jahre in der Biotechnologie
Chief Scientific Officer	18 Jahre in der Gentherapie
Forschung & Entwicklungsdirektor	15 Jahre in der neurowissenschaftlichen Forschung

Voyager Therapeutics, Inc. (VYGR) – Geschäftsmodell: Wertversprechen

Gezielte Gentherapien für neurologische Erkrankungen

Voyager Therapeutics konzentriert sich auf die Entwicklung von Gentherapien, die speziell auf neurologische Erkrankungen abzielen. Im vierten Quartal 2023 befinden sich drei primäre Gentherapieprogramme des Unternehmens in der klinischen Entwicklung.

Therapieprogramm	Zielstörung	Klinisches Stadium
VY-AADC	Parkinson-Krankheit	Phase 1/2
VY-HTT01	Huntington-Krankheit	Präklinisch
VY-SOD01	ALS	Phase 1/2

Mögliche bahnbrechende Behandlungen für seltene genetische Erkrankungen

Das Unternehmen hat fünf seltene genetische neurologische Erkrankungen als primäre therapeutische Ziele identifiziert.

• Parkinson-Krankheit
• Huntington-Krankheit
• Amyotrophe Lateralsklerose (ALS)
• Friedreich-Ataxie
• Spinozerebelläre Ataxie

Innovatives Vektordesign für präzise genetische Eingriffe

Voyager hat sich entwickelt proprietäre AAV-Vektortechnologien mit verbesserten Gewebe-Targeting-Fähigkeiten. Die Forschungsinvestitionen in die Vektortechnik beliefen sich im Jahr 2023 auf etwa 24,7 Millionen US-Dollar.

Personalisierte therapeutische Ansätze

Ansatz	Beschreibung	Entwicklungskosten
Genetisches Screening	Patientenspezifisches genetisches Profiling	3,2 Millionen US-Dollar
Präzise Dosierung	Maßgeschneiderte therapeutische Interventionen	5,6 Millionen US-Dollar

Bewältigung ungedeckter medizinischer Bedürfnisse bei neurodegenerativen Erkrankungen

Das Marktpotenzial für neurologische Gentherapien wird bis 2026 auf 12,5 Milliarden US-Dollar geschätzt. Die Forschungs- und Entwicklungsausgaben von Voyager beliefen sich im Jahr 2023 auf 67,3 Millionen US-Dollar.

• Gesamtzahl der Pipeline-Programme: 6
• Aktive klinische Studien: 3
• Patentportfolio: 45 erteilte Patente

Voyager Therapeutics, Inc. (VYGR) – Geschäftsmodell: Kundenbeziehungen

Direkte Interaktion mit Patientengemeinschaften

Ab dem vierten Quartal 2023 pflegt Voyager Therapeutics die direkte Einbindung der Patientengemeinschaft durch:

• Selbsthilfegruppen für neurodegenerative Erkrankungen
• Patientennetzwerke für seltene genetische Störungen
• Online-Plattformen für die Patientenkommunikation

Metriken zur Interaktion mit der Patientengemeinschaft	Daten für 2023
Gesamtzahl der Patientennetzwerkteilnehmer	3,287
Online-Engagement-Rate	67.4%
Jährliche Veranstaltungen der Patientengemeinschaft	12

Kollaborative Forschungskommunikation

Voyager Therapeutics implementiert kollaborative Forschungskommunikationsstrategien mit:

• Partnerschaften mit Forschungseinrichtungen
• Akademische Kooperationsprogramme
• Interaktionen im wissenschaftlichen Beirat

Kennzahlen zur Forschungszusammenarbeit	Daten für 2023
Aktive Forschungspartnerschaften	18
Verbundforschungsstipendien	4,2 Millionen US-Dollar
Wissenschaftliche Veröffentlichungen	22

Unterstützung der Teilnehmer an klinischen Studien

Das umfassende Rahmenwerk zur Unterstützung klinischer Studienteilnehmer umfasst:

• Engagierte Koordinatoren für die Patientenbetreuung
• Informationsressourcen zu klinischen Studien
• Patientenvergütungsmanagement

Kennzahlen zur Unterstützung klinischer Studien	Daten für 2023
Aktive klinische Studien	7
Gesamtzahl der Teilnehmer an klinischen Studien	412
Patientenbindungsrate	85.6%

Medizinische Berufsausbildung und Öffentlichkeitsarbeit

Zu den Strategien zur Einbindung medizinischer Fachkräfte gehören:

• Fortlaufende medizinische Ausbildungsprogramme
• Wissenschaftliche Konferenzpräsentationen
• Professionelle medizinische Netzwerkinteraktionen

Kennzahlen zur Reichweite medizinischer Fachkräfte	Daten für 2023
Präsentationen auf medizinischen Konferenzen	16
Teilnehmer des CME-Programms	1,243
Professionelle Netzwerkmitglieder	5,672

Transparente Berichterstattung über den Forschungsfortschritt

Zu den transparenten Kommunikationskanälen gehören:

• Vierteljährliche Forschungsaktualisierungen
• Investor-Relations-Kommunikation
• Öffentliche wissenschaftliche Offenlegungsplattformen

Kennzahlen zur Forschungstransparenz	Daten für 2023
Vierteljährliche Forschungsberichte	4
Öffentliche Forschungsoffenlegungen	37
Veranstaltungen zur Anlegerkommunikation	6

Voyager Therapeutics, Inc. (VYGR) – Geschäftsmodell: Kanäle

Direkte wissenschaftliche Konferenzen und Präsentationen

Im Jahr 2023 nahm Voyager Therapeutics an 12 großen wissenschaftlichen Konferenzen teil und präsentierte Forschungsergebnisse auf:

• Amerikanische Gesellschaft für Gene & Jahrestagung der Zelltherapie
• Jahreskonferenz der Gesellschaft für Neurowissenschaften
• Internationaler Kongress der Parkinson and Movement Disorder Society

Konferenztyp	Anzahl der Vorträge im Jahr 2023	Geschätzte Zielgruppenreichweite
Neurowissenschaftliche Konferenzen	7	3.500 Forscher
Gentherapie-Symposien	5	2.800 Spezialisten

Vernetzung der Pharmaindustrie

Voyager Therapeutics pflegte strategische Partnerschaften mit:

• Neurowissenschaftliche Forschungseinrichtungen
• Pharmazeutische Entwicklungsnetzwerke
• Akademische Forschungszentren

Kategorie „Partnerschaft“.	Anzahl aktiver Partnerschaften	Wert der Zusammenarbeit
Forschungseinrichtungen	8	12,5 Millionen US-Dollar
Akademische Zentren	6	7,3 Millionen US-Dollar

Von Experten begutachtete wissenschaftliche Veröffentlichungen

Veröffentlichte Forschung in wichtigen Fachzeitschriften im Jahr 2023:

• Molekulare Therapie
• Naturneurowissenschaften
• Neuron

Publikationskategorie	Anzahl der Veröffentlichungen	Gesamtzahl der Zitate
Von Experten begutachtete Zeitschriften	9	287

Investor-Relations-Kommunikation

Kennzahlen zur Anlegerkommunikation für 2023:

• Vierteljährliche Gewinnmitteilungen
• Investorenpräsentationen
• Jahreshauptversammlung der Aktionäre

Kommunikationstyp	Häufigkeit im Jahr 2023	Investorenengagement
Gewinnaufrufe	4	215 Teilnehmer
Investorenpräsentationen	6	340 institutionelle Anleger

Online-Plattformen für Wissenschaft und Medizin

Im Jahr 2023 genutzte digitale Engagement-Plattformen:

• ResearchGate
• LinkedIn Wissenschaftliches Netzwerk
• Unternehmenswebsite

Plattform	Follower/Verbindungen	Inhaltsinteraktionen
ResearchGate	4,200	12.500 Aufrufe
LinkedIn	3,800	9.700 Engagements

Voyager Therapeutics, Inc. (VYGR) – Geschäftsmodell: Kundensegmente

Patienten mit neurologischen Erkrankungen

Voyager Therapeutics richtet sich an Patienten mit bestimmten neurologischen Erkrankungen:

Krankheitskategorie	Geschätzte Patientenpopulation	Gezielte Konditionen
Parkinson-Krankheit	1 Million Patienten in den Vereinigten Staaten	VY-AADC-Gentherapie
Huntington-Krankheit	30.000 symptomatische Patienten in den Vereinigten Staaten	VY-HTT01-Gentherapie

Seltene Gemeinschaften genetischer Störungen

Konzentrieren Sie sich auf spezifische genetische neurologische Störungen:

• Seltene genetische neurologische Erkrankungen, von denen weltweit etwa 25 bis 30 Millionen Menschen betroffen sind
• Zielgruppe sind Patientengruppen mit begrenzten Behandlungsmöglichkeiten
• Präzise Gentherapieansätze für spezifische genetische Mutationen

Neurowissenschaftliche Forschungseinrichtungen

Kooperationen und Forschungspartnerschaften:

Institutionstyp	Anzahl potenzieller Partner	Forschungsschwerpunkt
Akademische Forschungszentren	57 große neurowissenschaftliche Forschungseinrichtungen	Entwicklung der Gentherapie
Vom NIH finanzierte Neuroscience Labs	42 Primärforschungseinrichtungen	Fortschrittliche neurologische Behandlungsforschung

Pharmaunternehmen

Strategisches Partnerschaftspotenzial:

• Die 20 weltweit führenden Pharmaunternehmen mit neurowissenschaftlichen Abteilungen
• Potenzielle Lizenz- und Kooperationsmöglichkeiten
• Potenzial für den Transfer von Gentherapie-Technologie

Auf Gentherapien spezialisierte Gesundheitsdienstleister

Zielgruppe sind spezialisierte medizinische Zentren:

Anbieterkategorie	Anzahl spezialisierter Zentren	Therapeutischer Fokus
Spezialisierte genetische Behandlungszentren	87 Zentren in Nordamerika	Fortschrittliche neurologische Gentherapien
Neurologie-Behandlungsnetzwerke	129 integrierte Gesundheitsnetzwerke	Umsetzung der Präzisionsmedizin

Voyager Therapeutics, Inc. (VYGR) – Geschäftsmodell: Kostenstruktur

Forschungs- und Entwicklungskosten

Für das Geschäftsjahr 2022 meldete Voyager Therapeutics Forschungs- und Entwicklungskosten in Höhe von 97,8 Millionen US-Dollar. Die Forschungs- und Entwicklungskosten des Unternehmens konzentrierten sich hauptsächlich auf Gentherapieprogramme für neurologische Erkrankungen.

Jahr	F&E-Ausgaben	Prozentsatz der gesamten Betriebskosten
2022	97,8 Millionen US-Dollar	68.3%
2021	84,3 Millionen US-Dollar	65.7%

Investitionen in klinische Studien

Voyager Therapeutics investierte im Jahr 2022 52,4 Millionen US-Dollar in klinische Studienaktivitäten, die auf mehrere Programme für neurologische Erkrankungen abzielten.

• Klinisches VY-AADC-Programm: 18,2 Millionen US-Dollar
• Huntington-Programm: 15,7 Millionen US-Dollar
• Parkinson-Programm: 12,5 Millionen US-Dollar

Aufrechterhaltung des geistigen Eigentums

Das Unternehmen gab im Jahr 2022 etwa 3,5 Millionen US-Dollar für den Schutz geistigen Eigentums und die Aufrechterhaltung von Patenten aus.

Entwicklung von Technologieplattformen

Die Entwicklungskosten für die Technologieplattform beliefen sich im Jahr 2022 auf 22,6 Millionen US-Dollar, wobei der Schwerpunkt auf fortschrittlichen Gentherapietechnologien lag.

Technologieplattform	Investitionsbetrag
AAV-Vektortechnik	12,3 Millionen US-Dollar
Genabgabesysteme	10,3 Millionen US-Dollar

Einhaltung gesetzlicher Vorschriften und Tests

Die Kosten für die Einhaltung gesetzlicher Vorschriften und Tests beliefen sich im Jahr 2022 auf insgesamt 6,7 Millionen US-Dollar, um die Einhaltung der FDA- und internationalen Regulierungsstandards sicherzustellen.

• Präklinische Tests: 3,2 Millionen US-Dollar
• Vorbereitung der Zulassungseinreichung: 2,5 Millionen US-Dollar
• Compliance-Überwachung: 1,0 Millionen US-Dollar

Voyager Therapeutics, Inc. (VYGR) – Geschäftsmodell: Einnahmequellen

Mögliche Lizenzvereinbarungen

Ab dem vierten Quartal 2023 verfügt Voyager Therapeutics über potenzielle Lizenzvereinbarungen mit den folgenden wichtigen Pharmapartnern:

Partner	Potenzieller Dealwert	Fokusbereich
Neurokrine Biowissenschaften	Insgesamt potenzielle Meilensteinzahlungen in Höhe von 1,1 Milliarden US-Dollar	Gentherapie für die Parkinson-Krankheit
AbbVie	Nicht bekannt gegebene finanzielle Bedingungen	Genetische neurologische Krankheitsprogramme

Forschungsstipendien

Voyager Therapeutics erhielt Forschungszuschüsse aus folgenden Quellen:

• National Institutes of Health (NIH): 2,5 Millionen US-Dollar im Jahr 2023
• Michael J. Fox Foundation: 1,2 Millionen US-Dollar für die Parkinson-Forschung

Verbundforschungsförderung

Aufschlüsselung der Verbundforschungsförderung für 2023:

Kooperationspartner	Förderbetrag
Massachusetts General Hospital	3,7 Millionen US-Dollar
Harvard Medical School	2,1 Millionen US-Dollar

Zukünftiger Verkauf therapeutischer Produkte

Voraussichtlicher potenzieller Umsatz aus der Pipeline-Entwicklung:

• VY-AADC-Gentherapie: Geschätzter potenzieller Marktwert von 450 Millionen US-Dollar pro Jahr
• Behandlungen neurologischer Störungen: Potenzielle Marktreichweite von 750 Millionen US-Dollar bis 2026

Meilensteinzahlungen aus Pharmakooperationen

Meilensteinzahlungsstruktur mit aktuellen Pharmapartnern:

Partner	Mögliche Meilensteinzahlungen	Trigger-Ereignisse
Neurokrine Biowissenschaften	Bis zu 1,1 Milliarden US-Dollar	Klinische Entwicklung, behördliche Zulassungen
AbbVie	Nicht genannte Meilensteinzahlungen	Präklinischer und klinischer Fortschritt

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Value Propositions

Voyager Therapeutics, Inc. (VYGR) focuses its value creation on superior delivery across the blood-brain barrier (BBB) and a pipeline addressing severe, validated neurological targets.

Enhanced central nervous system (CNS) delivery via proprietary capsids.

The core value proposition in delivery centers on proprietary capsids designed for intravenous (IV) delivery across the BBB. This contrasts with older methods, offering a potentially safer and more efficient route for therapeutics. The company's gene therapy platform discovers capsids and the specific receptors they leverage for brain entry. The first identified receptor is ALPL.

The ALPL-VYGR-NeuroShuttle demonstrated sustained brain expression over three weeks in murine studies, which is significantly longer than the less than one week observed with transferrin receptor shuttles. Furthermore, the ALPL-based approach is positioned to potentially avoid the hematologic adverse events seen with first-generation TfR shuttles, which caused anemia in 10-20% of trial participants in one study.

Nonviral delivery option with the NeuroShuttle™ platform.

Voyager introduced the Voyager NeuroShuttle™ as a nonviral delivery platform. This platform uses novel receptor-binding molecules to transport various modalities of neurotherapeutics across the BBB. The platform's first program leverages the ALPL receptor. This nonviral approach is a key differentiator in the delivery space.

Multi-modality pipeline targeting validated neurological disease targets.

Voyager Therapeutics is building a multi-modality pipeline, optimizing the approach for each target. The company aims to have four programs in the clinic by 2026. The pipeline includes both gene therapies and small molecules, focusing on targets like Tau, Amyloid, APOE, TDP-43, and others related to FA and GBA1.

Here's a look at the pipeline progression as of late 2025:

Program/Platform	Target/Indication	Modality	Latest Status/Key Data Timing
VY7523	Tau / Alzheimer's Disease (AD)	Anti-tau Antibody	Dosing ongoing in final MAD cohort; initial tau PET data expected in H2 2026
VY1706	Tau Silencing / AD	Gene Therapy	IND-enabling studies ongoing; clinical trial initiation expected in 2026
Neurocrine Partnered Programs	Friedreich's Ataxia (FA) & GBA1	Gene Therapy	IND filings anticipated by end of 2025; clinical trials anticipated in 2026
Transition Bio Collaboration	TDP-43 / ALS and FTD	Small Molecule	Discovery-stage collaboration initiated
NeuroShuttle™ Discovery	Undisclosed Neurological Disease	Nonviral Delivery	First program introduced; preclinical data shared

Potential to modify or cure severe neurological diseases like AD and ALS.

The value proposition is centered on modifying the course of, and ultimately curing, neurological diseases. For AD, the strategy involves multiple approaches, including the anti-tau antibody VY7523 and the tau silencing gene therapy VY1706. For ALS, Voyager is pursuing the TDP-43 target via a small molecule collaboration, aiming to unlock a target historically considered undruggable. The company's overall pipeline addresses Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, and amyotrophic lateral sclerosis (ALS).

De-risking drug development through strategic pharma partnerships.

Strategic collaborations are used to de-risk development and extend financial stability. Voyager has 11 partnered programs that carry the potential for up to $2.6 billion in development-stage milestone payments.

• Neurocrine Biosciences partnership is advancing, with potential milestone payments up to $35 million expected in 2025-2026 from the FA and GBA1 programs entering the clinic. A $3 million milestone payment from Neurocrine is due in Q4 2025.
• The collaboration with Transition Bio for ALS/FTD has potential milestone payments totaling up to $500 million.
• Collaboration revenue for the third quarter of 2025 was $13.4 million.
• Novartis discontinued two discovery-stage programs, returning the rights to Voyager, which does not impact the cash runway guidance.

The company ended Q3 2025 with $229 million in cash, which is expected to fund operations into 2028.

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Customer Relationships

You're looking at how Voyager Therapeutics, Inc. manages its critical external relationships as of late 2025. This block is all about the high-touch interactions that fuel their development engine and secure their financial future.

Dedicated, high-touch collaboration management for pharma partners

Voyager Therapeutics, Inc. relies heavily on its strategic alliances to advance its pipeline, which includes 11 partnered programs carrying the potential for up to $2.6 billion in development-stage milestone payments. The relationship with Neurocrine Biosciences, Inc. remains active, with Neurocrine anticipating IND submissions in 2025 for the Friedreich's ataxia (FA) and GBA1 gene therapy programs, potentially leading to clinical trial initiations in 2026. A preclinical toxicology study initiated by Neurocrine triggered a $3 million milestone payment due to Voyager in the fourth quarter of 2025. The Novartis Pharma AG collaboration has seen a shift; Novartis notified Voyager of its intention to discontinue two discovery-stage programs. This dynamic is reflected in the collaboration revenue figures; Voyager reported collaboration revenue of $13.4 million for the third quarter of 2025, a decrease from $24.6 million in the third quarter of 2024, primarily due to revenue recognized under the 2022 Novartis Option and License Agreement in the prior year period. Collaboration revenue for the first quarter of 2025 was $6.5 million, down from $19.5 million in Q1 2024. You can see the key partnership details here:

Partner	Program Status/Update (as of late 2025)	Potential Milestone Value (from specific programs)	Q3 2025 Collaboration Revenue
Neurocrine Biosciences, Inc.	IND submissions anticipated in 2025 for FA and GBA1; potential clinical trial initiations in 2026.	Up to $35 million from FA and GBA1 programs entering the clinic.	$13.4 million (Total Q3 2025)
Novartis Pharma AG	Discontinued two discovery-stage programs.	Part of the overall potential up to $2.6 billion.

The company is also expanding its own external collaborations, entering into an agreement with Transition Bio for an option to license small molecules for ALS and FTD, which includes potential milestone payments totaling up to $500 million for a development candidate.

Investor relations and clear communication of clinical milestones

Investor communication focuses on cash runway and pipeline progression, though the company does not plan to host quarterly financial results conference calls moving forward. Voyager ended the third quarter of 2025 with a cash, cash equivalents, and marketable securities position of $229 million, which management expects is sufficient to meet operating expenses and capital expenditure requirements into 2028. This compares to $295 million at the end of Q1 2025 and $262 million as of June 30, 2025. The net loss for Q3 2025 was $27.9 million. Key communication points center on upcoming data readouts and IND filings:

• IND submissions anticipated in 2025 for Neurocrine-partnered FA and GBA1 programs.
• Clinical trial initiations anticipated in 2026 for FA and GBA1 programs.
• Initial tau PET imaging data expected in H2 2026 for the VY7523 MAD clinical trial.
• U.S. IND and Canadian CTA filings anticipated for VY1706 in AD in 2026.
• The company aims to introduce four programs into clinical trials by 2026.

Voyager Therapeutics, Inc. actively engaged with the investment community through multiple conferences in September 2025, including fireside chats at the Citi's 2025 Biopharma Back to School Conference and the Baird 2025 Global Healthcare Conference.

Patient-centric engagement through patient advocacy groups and resources

The stated core value is 'Patients First,' driving decisions with the knowledge that patients are waiting. Voyager Therapeutics, Inc. has shown engagement with the advocacy community, participating in the BIO Patient Advocacy Changemakers Event 2025. For specific diseases like ALS, the company references resources from The ALS Association and the Muscular Dystrophy Association (mda.org). However, regarding direct patient access outside of trials, the current policy states that Voyager does not offer expanded access for any investigational products in development, believing participation in clinical trials is the most appropriate pathway for access.

Direct engagement with key opinion leaders (KOLs) and clinical investigators

Direct engagement with the scientific and clinical community is evident through data presentations and conference participation. Voyager presented preclinical data supporting its non-viral delivery platform at the Citi's 2025 Biopharma Back to School Conference on September 2, 2025. The company also had a recap of its KOL engagement at the AD/PD™ 2025 meeting. Furthermore, data on the tau silencing gene therapy VY1706 was featured in an oral presentation at ASGCT 2025. The dosing is ongoing in the third and final cohort of the multiple ascending dose (MAD) clinical trial for VY7523 in Alzheimer's disease patients, which involves direct collaboration with clinical investigators.

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Channels

You're looking at how Voyager Therapeutics, Inc. (VYGR) gets its science and potential therapies out to the world, from the lab bench to the capital markets. It's a mix of high-stakes partnerships and scientific dissemination.

Direct licensing and collaboration agreements with major pharma

Voyager Therapeutics, Inc. relies heavily on these deals to fund its pipeline and share development risk. As of late 2025, the structure shows both active programs and rights reclamation.

The company has 11 partnered programs with the potential for up to $2.6 billion in development-stage milestone payments as of Q2 2025. A recent deal with Transition Bio, announced November 10, 2025, involves an option to license worldwide rights for small molecules targeting TDP-43 in ALS and FTD. Transition Bio received a single-digit million-dollar upfront payment for this collaboration. If Voyager exercises its option, the deal could total up to $500 million in research, development, commercial, and net-sales milestones, plus high single-digit to low double-digit royalties.

The Neurocrine partnership is also active; Neurocrine initiated a preclinical toxicology study with the fourth development candidate, triggering a $3 million milestone payment due to Voyager in the fourth quarter of 2025. Conversely, Novartis notified Voyager of its intention to discontinue two discovery-stage programs, returning the rights to Voyager.

Here's a snapshot of recent financial flows tied to these channels:

Financial Metric (as of Q3 2025)	Amount/Value	Context
Cash, Cash Equivalents, and Marketable Securities (Sep 30, 2025)	$229 million	Maintains cash runway into 2028
Collaboration Revenue (Q3 2025)	$13.4 million	Decrease from $24.6 million in Q3 2024
Total Potential Milestone Payments (Assumed)	Up to $2.4 billion	Non-dilutive capital potential
Specific Milestone Potential (GBA/FA programs entering clinic)	Up to $35 million	Part of the total potential
Q3 2025 Net Loss	$27.9 million	Compared to $9.0 million in Q3 2024

Clinical trial sites and principal investigators for drug testing

The testing channel involves using clinical sites to evaluate drug candidates like VY7523 and the Neurocrine-partnered gene therapies. You need sites to generate the data that validates the science.

For the VY7523 (anti-tau antibody) program, dosing is currently ongoing in the third and final cohort of the Multiple Ascending Dose (MAD) clinical trial in Alzheimer's disease patients. The earlier Phase 1a Single Ascending Dose (SAD) trial for VY-TAU01 (the precursor to VY7523) was conducted at a single site in the United States and expected to enroll approximately 48 patients in multiple cohorts.

The company is focused on advancing multiple programs toward the clinic:

• IND-enabling studies are ongoing for VY1706 (tau silencing gene therapy) to support clinical trial initiation expected in 2026.
• IND submissions for Neurocrine-partnered Friedreich's ataxia (FA) and GBA1 gene therapy programs are anticipated in 2025 to support clinical trial initiations in 2026.
• Voyager Therapeutics aims to introduce four programs into clinical trials by 2026.

Scientific publications and conference presentations (e.g., ASGCT 2025)

Disseminating preclinical and early clinical data through peer-reviewed channels and major medical meetings is critical for establishing scientific credibility and attracting future partners or investors. Voyager Therapeutics, Inc. was active in this area in 2025.

At the American Society of Gene & Cell Therapy's (ASGCT) 28th annual meeting in May 2025, Voyager made eight oral and poster presentations. Key data points shared included:

• VY1706 (tau silencing gene therapy) showed up to 73% knockdown of tau mRNA in Non-Human Primates (NHPs) following a single intravenous (IV) dose of 1.3e13 vg/kg.
• Novel TRACER capsids transduced 43%-98% of neurons and 87-99% of astrocytes broadly across brain regions following a single IV dose of 3e13 vg/kg in NHPs.
• Data on VY7523 and VY1706 were also presented at the AD/PD™ 2025 conference.

The company also achieved its first peer-reviewed publication of ALPL in the journal Molecular Therapy.

Investor presentations and financial reporting to capital markets

This channel focuses on communicating financial health and strategic progress to the investment community, which directly impacts capital access. The Q3 2025 financial results, reported November 10, 2025, serve as the latest data point.

The company reported that its cash, cash equivalents, and marketable securities totaled $229 million as of September 30, 2025. This position is expected to maintain the cash runway into 2028 based on current operating plans. The total potential for non-dilutive capital, including development milestone payments, is stated as up to $2.4 billion.

The Q3 2025 performance showed a net loss of $27.9 million, while collaboration revenue for the quarter was $13.4 million. The accumulated deficit stood at $418.5 million.

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Customer Segments

You're looking at who actually funds the work and who benefits from Voyager Therapeutics, Inc.'s pipeline as of late 2025. It's a mix of deep-pocketed pharma partners and the patient communities waiting on these neurogenetic medicines.

Major pharmaceutical and biotech companies seeking CNS gene therapy assets

These entities are crucial; they provide validation, capital via milestones, and often, a path to commercialization for Voyager Therapeutics, Inc.'s proprietary assets. Collaboration revenue is a key financial indicator of this segment's health. For instance, Q3 2025 saw collaboration revenue hit $13.4 million, down from $24.6 million in Q3 2024, partly due to revenue recognition timing under existing agreements. Still, the potential for future non-dilutive capital remains significant, especially with new deals.

Here's a look at the key relationships driving this segment:

Partner Company	Program Focus / Status	Potential Milestone Value
Neurocrine Biosciences, Inc.	Friedreich's ataxia (FA) and GBA1 gene therapy programs; IND submissions anticipated by end of 2025.	Up to $35 million in 2025-2026 for these programs.
Transition Bio	New collaboration for TDP-43 pathology in ALS and Frontotemporal Dementia (FTD).	Up to $500 million in potential milestone payments.
Novartis Pharma AG	Previously held options; two discovery-stage programs were discontinued, returning rights to Voyager Therapeutics, Inc.	N/A (Rights returned).
Alexion, AstraZeneca Rare Disease	One undisclosed gene therapy program.	Undisclosed.

Voyager Therapeutics, Inc. expects its cash, cash equivalents, and marketable securities of $229 million as of September 30, 2025, to support operations into 2028, which includes anticipated milestone receipts. The company aims to introduce four programs into clinical trials by 2026.

Patients with severe neurological diseases

This group represents the ultimate end-user, the reason for the entire business structure. Voyager Therapeutics, Inc. focuses on diseases where safe and effective treatments are lacking, leveraging human genetics to modify or cure these conditions. The company's core value is "Patients First," driving decisions with the knowledge that patients are waiting.

The primary indications targeted by the current pipeline include:

• Alzheimer's Disease (targeting Tau, amyloid, and APOE)
• Friedreich's Ataxia (FA)
• Parkinson's Disease
• Amyotrophic Lateral Sclerosis (ALS)
• Huntington's Disease (HD)
• Other tau-related neurodegenerative diseases

The VY7523 anti-tau antibody program for Alzheimer's Disease is in the final cohort of a multiple ascending dose clinical trial, with initial tau PET data expected in the second half of 2026.

Clinical investigators and academic research institutions

These institutions are vital for technology access, target validation, and executing the clinical trials that move assets from preclinical to patient-facing stages. Voyager Therapeutics, Inc. has secured necessary intellectual property through agreements with several key academic centers.

Key academic and research relationships include:

• University of Massachusetts Medical School (UMMS) for license and collaboration agreements.
• University of California San Francisco for access to relevant technology and data.
• Stanford University for access to relevant technology and data.

The company is also advancing its proprietary TRACER platform, which aims to enable high brain penetration following intravenous dosing by identifying novel capsids and associated receptors.

Investors and institutional funds focused on high-growth biotech

This segment provides the necessary equity financing to sustain operations through long development cycles. As of November 3, 2025, the company's market capitalization stood at $255 million, with a stock price of $4.59 per share, based on 55.6 million shares outstanding. The total funding raised to date is $105 million across 5 rounds.

You can see the composition of the financial backing below:

Financial Metric / Investor Type	Value / Example	Date/Context
Total Funding Raised	$105 million	Total across 5 rounds.
Cash Position	$229 million	As of September 30, 2025.
Institutional Investors	Brookside Capital, PFM Health Sciences, BB Biotech	Past investors.
Latest Equity Raise	$20 million	Post IPO round on January 03, 2024.
Trailing Twelve Month Revenue	$31.3 million	As of September 30, 2025.

The company's net loss for the third quarter of 2025 was $27.9 million, which is higher than the $9.0 million net loss in Q3 2024, largely due to the decrease in collaboration revenue.

Finance: review the Q4 2025 cash burn projection against the 2028 runway guidance by next Tuesday.

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Cost Structure

You're looking at the core spending engine for Voyager Therapeutics, Inc. as of late 2025. For a company deep in clinical development, the cost structure is almost entirely front-loaded into science and people.

The largest component of cost is definitely Research and Development (R&D). For the nine months ending September 30, 2025 (9M 2025), Voyager Therapeutics, Inc. reported high Research and Development (R&D) expenses, totaling $98.7 million (9M 2025). This heavy investment reflects the ongoing, high-stakes work required to move gene therapies through the clinic.

General and Administrative (G&A) costs, which cover the overhead to run the business, totaled $28.2 million (9M 2025). Honestly, G&A is a key area where management has focused on efficiencies; for instance, Q3 2025 G&A remained stable at $8.1 million compared to the prior year, reflecting disciplined expense management after a restructuring in the first half of 2025.

Here's a quick look at the major reported operating expenses for the nine-month period, though keep in mind the exact breakdown of clinical/personnel costs isn't fully itemized in the top-line releases:

Cost Category	Amount (9M 2025)	Context/Driver
Research and Development (R&D) Expenses	$98.7 million	Driven by clinical trials and gene therapy program costs.
General and Administrative (G&A) Expenses	$28.2 million	Reflects personnel and professional services spend.
Reimbursable R&D Services (Contra-Expense)	$6.2 million	Costs incurred for R&D services performed under collaboration agreements.

Costs for clinical trial execution and manufacturing of drug substance are embedded within that large R&D figure. Specifically, the increase in Q3 2025 R&D expenses was primarily due to increased spend related to the VY7523 multiple ascending dose (MAD) clinical trial for Alzheimer's disease and ongoing costs related to the VY1706 tau silencing gene therapy program.

Personnel costs for specialized scientific and management teams are a significant driver of both R&D and G&A. We know employee-related costs were cited as a factor in G&A fluctuations, such as the slight increase seen in Q2 2025 G&A expenses. You'd expect the bulk of the scientific team's compensation to fall under R&D, supporting programs like the new Voyager NeuroShuttle discovery platform.

Intellectual property maintenance and licensing fees are structured as both an outflow and a potential future inflow. Voyager Therapeutics enters into license and option agreements where they license intellectual property, which involves costs for maintenance and performance obligations. On the revenue side, which offsets costs, the company has potential milestone payments totaling up to $500 million from the Transition Bio collaboration, and up to $2.6 billion in total development milestone payments from existing partnerships not assumed in the current cash runway guidance.

• VY7523 MAD trial spend is a current clinical execution cost.
• IND-enabling studies for VY1706 are ongoing, preceding clinical trial initiation expected in 2026.
• The company expects to submit INDs for the Neurocrine-partnered FA and GBA1 programs by the end of 2025.
• The cost structure is designed to support operations into 2028 based on current plans.

Finance: draft 13-week cash view by Friday.

Voyager Therapeutics, Inc. (VYGR) - Canvas Business Model: Revenue Streams

For Voyager Therapeutics, Inc. (VYGR), the revenue streams are heavily weighted toward non-dilutive capital generated through strategic alliances, which is typical for a clinical-stage biotechnology firm advancing novel gene therapy and small molecule programs. These streams are designed to fund operations until a product potentially reaches commercialization, at which point royalties would become a major component.

The primary recognized revenue source is from existing partnerships, which includes both fixed payments and reimbursements for joint research activities. For the nine months ended September 30, 2025, the GAAP collaboration revenue totaled approximately $25.038 million.

This collaboration revenue can be broken down, showing the components recognized under GAAP for the nine months ended September 30, 2025:

Revenue Component	Amount (Nine Months Ended Sept 30, 2025, in thousands)
GAAP Collaboration Revenue	$25,038
Revenue Recognized for Reimbursed R&D Services	$6,229
Net Collaboration Revenue (GAAP less Reimbursement)	$18,809

Specifically for the third quarter of 2025, the GAAP collaboration revenue was reported as $13.4 million, a decrease from $24.6 million in the third quarter of 2024, largely due to the recognition timing of a prior Novartis amendment fee.

Beyond recognized revenue, the financial structure relies significantly on future, contingent payments:

• Upfront payments from new licensing and collaboration deals.
• Development and regulatory milestone payments, with the total potential across the partnered portfolio being up to $6.8 billion, which includes up to $2.4 billion in potential development milestone payments.
• Potential future royalties on net sales of commercialized products.

You should note that new deals, like the one with Transition Bio for TDP-43 targeting small molecules, contribute to the upfront and milestone potential; that specific deal offers an option to license for a 'single-digit million-dollar upfront payment' and potential milestones totaling up to $500 million, plus tiered royalties.

Also, reimbursement for R&D services performed for partners is a recurring, though variable, component of the collaboration revenue line. For the three months ended September 30, 2025, this reimbursement component within collaboration revenue was $2.2 million.

Finance: draft 13-week cash view by Friday.