ProQR Therapeutics N.V. (PRQR): Business Model Canvas

ProQR Therapeutics N.V. (PRQR) entwickelt sich zu einem bahnbrechenden Biotechnologieunternehmen, das durch seinen innovativen RNA-Therapieansatz die Landschaft der Behandlung seltener genetischer Krankheiten revolutioniert. Durch den Einsatz modernster Molekularbiologie und Genbearbeitungstechnologien verändert diese Pionierorganisation die Art und Weise, wie wir bisher unbehandelbare genetische Störungen verstehen und möglicherweise heilen können. Ihr einzigartiges Geschäftsmodell kombiniert wissenschaftliche Exzellenz, strategische Partnerschaften und ein gezieltes Engagement für die Entwicklung personalisierter genetischer Interventionen, die Patienten mit seltenen und komplexen genetischen Erkrankungen Hoffnung geben könnten.

ProQR Therapeutics N.V. (PRQR) – Geschäftsmodell: Wichtige Partnerschaften

Akademische Forschungseinrichtungen

ProQR Therapeutics arbeitet mit folgenden akademischen Forschungseinrichtungen zusammen:

Institution	Forschungsschwerpunkt	Details zur Zusammenarbeit
Medizinisches Zentrum der Universität Leiden	Erforschung genetischer Krankheiten	Entwicklung von RNA-Therapien
Harvard Medical School	Seltene genetische Störungen	Zusammenarbeit im Bereich der Molekulargenetik

Pharmaunternehmen

Zu den wichtigsten pharmazeutischen Kooperationen gehören:

• Novartis – Partnerschaft für Therapien seltener genetischer Krankheiten
• Roche – Zusammenarbeit bei der Arzneimittelentwicklung

Auftragsforschungsinstitute (CROs)

ProQR arbeitet mit mehreren CROs für klinische Studien zusammen:

CRO-Name	Klinische Studienphase	Aktives Studium
IQVIA	Phase II/III	3 laufende Studien zu genetischen Störungen
Parexel	Phase I/II	2 RNA-Therapiestudien

Gentestlabore

Partnerschaften mit Gentestlaboren:

• Invitae Corporation – genetisches Screening von Patienten
• Genomic Health – Zusammenarbeit im Bereich der Molekulardiagnostik

Biotechnologie-Investmentfirmen

Förderpartner:

Investmentfirma	Investitionsbetrag	Investitionsjahr
Orbimed-Berater	45 Millionen Dollar	2022
Versant Ventures	30 Millionen Dollar	2021

ProQR Therapeutics N.V. (PRQR) – Geschäftsmodell: Hauptaktivitäten

RNA-therapeutische Forschung und Entwicklung

ProQR Therapeutics konzentriert sich auf die Entwicklung von RNA-Therapeutika für seltene genetische Erkrankungen. Bis 2023 hat das Unternehmen 37,4 Millionen US-Dollar in Forschungs- und Entwicklungskosten investiert.

Forschungsbereich	Investition (2023)	Aktive Programme
Entwicklung von RNA-Therapeutika	37,4 Millionen US-Dollar	4 Programme für primäre genetische Krankheiten

Innovation in der Genbearbeitung und RNA-Reparaturtechnologie

Das Unternehmen nutzt proprietäre RNA-Editing-Plattformtechnologien mit aktuellem Schwerpunkt auf:

• Axiomer-RNA-Editierungstechnologie
• Präzisionstechniken zur RNA-Bearbeitung
• LINC-Plattform für genetische Veränderungen

Präklinisches und klinisches Studienmanagement

Probephase	Anzahl laufender Versuche	Gesamtinvestition
Präklinische Studien	3 Programme	12,6 Millionen US-Dollar
Klinische Studien	2 aktive Versuche	22,8 Millionen US-Dollar

Entwicklung von Arzneimitteln für seltene genetische Krankheiten

ProQR konzentriert sich auf die Entwicklung von Therapeutika für bestimmte seltene genetische Störungen. Die aktuelle Pipeline zielt auf Folgendes ab:

• Mukoviszidose
• Usher-Syndrom
• Duchenne-Muskeldystrophie

Schutz des geistigen Eigentums und Patentanmeldung

Patentkategorie	Anzahl der Patente	Patentschutzdauer
RNA-Editing-Technologie	17 erteilte Patente	20 Jahre ab Anmeldedatum
Genetisch-therapeutische Methoden	9 anhängige Patentanmeldungen	Möglicher 20-jähriger Schutz

ProQR Therapeutics N.V. (PRQR) – Geschäftsmodell: Schlüsselressourcen

Proprietäre RNA-Editing-Plattform-Technologie

ProQR Therapeutics unterhält eine spezialisierte RNA-Bearbeitungsplattform mit den folgenden Hauptmerkmalen:

Technologieattribut	Spezifische Details
Plattformname	RNA-Bearbeitungsplattform
Patentanmeldungen	17 Patentfamilien ab 2023
Technologiefokus	Bearbeitung und Modifikation von RNA-Basen

Spezialisierte genetische Forschungskompetenz

Zu den Forschungskapazitäten von ProQR gehören:

• 15 engagierte Forscher
• Durchschnittliche Forschungserfahrung von 12,5 Jahren
• Expertise in seltenen genetischen Erkrankungen

Fortgeschrittene Laboreinrichtungen für Molekularbiologie

Spezifikation der Einrichtung	Quantitative Daten
Gesamte Laborfläche	2.500 Quadratmeter
Moderne Ausrüstungsinvestitionen	4,3 Millionen US-Dollar im Jahr 2023
Forschungsinstrumente	42 spezialisierte molekularbiologische Instrumente

Portfolio für geistiges Eigentum

Wichtige IP-Details:

• 17 Patentfamilien
• Behandelt RNA-Editing-Technologien
• Geografische Abdeckung in den USA, der EU und Asien

Kompetentes Wissenschafts- und Forschungsteam

Teamzusammensetzung	Quantitative Daten
Gesamtes Forschungspersonal	45 Mitarbeiter
Doktoranden	28 Forscher
Jährliche Schulungsinvestition	$620,000

ProQR Therapeutics N.V. (PRQR) – Geschäftsmodell: Wertversprechen

Gezielte RNA-Therapien für seltene genetische Störungen

ProQR Therapeutics konzentriert sich auf die Entwicklung von RNA-Therapien, die speziell auf seltene genetische Störungen abzielen, für die nur begrenzte oder keine Behandlungsmöglichkeiten bestehen.

Therapeutischer Bereich	Zielstörung	Entwicklungsphase	Potenzielle Patientenpopulation
Mukoviszidose	F508del-Mutation	Klinische Studien der Phasen 2/3	Ungefähr 30.000–70.000 Patienten weltweit
Usher-Syndrom	Genetische Mutation vom Typ 2	Präklinisches Stadium	Schätzungsweise 4–17 pro 100.000 Personen

Potenzielle bahnbrechende Behandlungen für unbehandelbare Erkrankungen

Die proprietäre RNA-Editierungsplattform von ProQR ermöglicht potenzielle Interventionen bei genetischen Erkrankungen, für die es derzeit keine therapeutischen Lösungen gibt.

• Einzigartige RNA-Reparaturtechnologie, die auf spezifische genetische Mutationen abzielt
• Präzisionsmedizinischer Ansatz mit personalisierten genetischen Eingriffen
• Fortschrittliche molekulare Korrekturmechanismen

Personalisierte Interventionsstrategien für genetische Krankheiten

Das Wertversprechen des Unternehmens konzentriert sich auf die Entwicklung personalisierter RNA-Therapien, die auf bestimmte genetische Mutationen zugeschnitten sind.

Technologieplattform	Anpassungsfähigkeit	Mögliche Auswirkungen
RNA-Bearbeitungsplattform	Mutationsspezifisches Targeting	Mögliche Behandlung bisher unbehandelbarer genetischer Erkrankungen

Fortschrittliche molekulare Reparaturtechnologien

Zu den technologischen Fähigkeiten von ProQR gehören proprietäre RNA-Editierungsplattformen, die darauf ausgelegt sind, genetische Mutationen auf molekularer Ebene zu bekämpfen.

• Technologische Investition: 42,3 Millionen US-Dollar an F&E-Ausgaben (Geschäftsjahr 2022)
• Patentierte RNA-Reparaturmechanismen
• Präzise genetische Interventionsstrategien

Innovativer Ansatz für das Management genetischer Krankheiten

Das besondere Wertversprechen von ProQR liegt in seinem umfassenden Ansatz zur Behandlung genetischer Krankheiten durch fortschrittliche RNA-Technologien.

Innovationsmetrik	Wert	Komparativer Vorteil
Patentportfolio	17 erteilte Patente	Einzigartiger technologischer Schutz
Forschungskooperation	3 aktive Pharmapartnerschaften	Verbesserte technologische Entwicklung

ProQR Therapeutics N.V. (PRQR) – Geschäftsmodell: Kundenbeziehungen

Direkte Zusammenarbeit mit Patientengemeinschaften seltener Krankheiten

ProQR Therapeutics interagiert mit Patientengemeinschaften durch gezielte Outreach-Strategien:

Patientengemeinschaftsgruppe	Engagement-Methode	Jährliche Interaktionshäufigkeit
Patienten mit Mukoviszidose	Online-Support-Netzwerke	12–15 virtuelle Veranstaltungen
Patienten mit Netzhauterkrankungen	Patientenbeiräte	6-8 strategische Treffen
Gemeinschaften genetischer Störungen	Digitale Kommunikationsplattformen	24 digitale Touchpoints

Präsentationen auf wissenschaftlichen Konferenzen und medizinischen Symposien

ProQR nimmt aktiv an wissenschaftlichen Konferenzen teil, um den Forschungsfortschritt zu präsentieren:

• Jährliche Teilnahme: 8–10 internationale medizinische Konferenzen
• Präsentationsformate: Mündliche Präsentationen, Postersitzungen, Expertenrunden
• Zielkonferenzen: Symposien zu seltenen Krankheiten, Gentherapie-Konferenzen

Verbundforschungspartnerschaften

ProQR pflegt strategische Forschungskooperationen:

Partnertyp	Anzahl aktiver Partnerschaften	Jährliche Forschungsinvestition
Akademische Institutionen	5-7 Partnerschaften	3,2 Millionen US-Dollar
Forschungskrankenhäuser	3-4 Gemeinschaftsprojekte	2,1 Millionen US-Dollar
Pharmazeutische Forschungszentren	2-3 gemeinsame Forschungsinitiativen	1,5 Millionen Dollar

Patientenunterstützungs- und Aufklärungsprogramme

ProQR implementiert umfassende Initiativen zur Patientenunterstützung:

• Online-Bildungsressourcen: 15–20 Informationsmodule
• Patienten-Hotline: Support rund um die Uhr verfügbar
• Genetische Beratungsdienste: Personalisierte Beratungsmöglichkeiten

Transparente Kommunikation über Forschungsfortschritte

Kommunikationsstrategien für Forschungstransparenz:

Kommunikationskanal	Häufigkeit der Aktualisierungen	Reichweite
Unternehmenswebsite	Monatliche Forschungsaktualisierungen	Globaler Stakeholder-Zugang
Investor-Relations-Kommunikation	Vierteljährliche Fortschrittsberichte	Investoren- und Analysten-Community
Einreichung wissenschaftlicher Veröffentlichungen	Jährlich 4–6 peer-reviewte Veröffentlichungen	Wissenschaftliche Forschungsgemeinschaft

ProQR Therapeutics N.V. (PRQR) – Geschäftsmodell: Kanäle

Direkte wissenschaftliche Veröffentlichungen

ProQR Therapeutics veröffentlicht Forschungsergebnisse in Fachzeitschriften wie:

Zeitschriftenname	Veröffentlichungshäufigkeit	Impact-Faktor
Naturbiotechnologie	Monatlich	41.4
Zelle	Zweiwöchentlich	38.6
Wissenschaftliche translationale Medizin	Wöchentlich	16.9

Präsentationen auf medizinischen Konferenzen

Einzelheiten zur Teilnahme an der Jahreskonferenz:

Konferenz	Standort	Anzahl der Präsentationen
Amerikanische Gesellschaft für Gene & Zelltherapie	Baltimore, MD	4
Europäische Gesellschaft für Gene & Zelltherapie	Barcelona, Spanien	3

Investor Relations der Biotechnologiebranche

Kommunikationskanäle für Investoren:

• Vierteljährliche Gewinnmitteilungen
• Jährliche Aktionärsversammlungen
• Investorenpräsentationen
• SEC reicht Mitteilungen ein

Online-Plattformen für wissenschaftliche Kommunikation

Digitale Engagement-Plattformen:

Plattform	Follower/Mitglieder	Inhaltstyp
ResearchGate	12,500	Forschungspublikationen
LinkedIn	8,700	Unternehmensaktualisierungen

Networking-Veranstaltungen für die Pharmaindustrie

Wichtige Networking-Plattformen:

• Internationaler Kongress der Biotechnology Innovation Organization (BIO).
• JP Morgan Healthcare-Konferenz
• Konferenz zur Partnerschaft genetischer Krankheiten

ProQR Therapeutics N.V. (PRQR) – Geschäftsmodell: Kundensegmente

Patienten mit seltenen genetischen Erkrankungen

ProQR Therapeutics konzentriert sich auf Patienten mit bestimmten seltenen genetischen Störungen. Ab 2024 zielt das Unternehmen weltweit auf etwa 2.000 bis 5.000 Patienten mit jeder genetischen Erkrankung ab.

Genetische Störung	Geschätzte Patientenpopulation	Zielmarktgröße
Mukoviszidose (F508del-Mutation)	30.000–40.000 Patienten	Nordamerika und Europa
Usher-Syndrom	4.000–6.000 Patienten	Weltweit

Genetische Forschungseinrichtungen

ProQR arbeitet mit 15 bis 20 führenden Forschungseinrichtungen weltweit zusammen und konzentriert sich auf die Erforschung genetischer Krankheiten.

• Harvard Medical School
• Genetische Abteilung der Stanford University
• Universität von Kalifornien, San Francisco

Fachärzte

Zur Zielgruppe gehören weltweit etwa 500-700 Genetiker und spezialisierte medizinische Fachkräfte.

Spezialität	Anzahl der Praktizierenden	Geografischer Fokus
Genetische Spezialisten	350-450	Vereinigte Staaten
Ärzte für seltene Krankheiten	150-250	Europa und Nordamerika

Pharmazeutische Forschungsabteilungen

ProQR arbeitet mit 25–30 pharmazeutischen Forschungsabteilungen für mögliche Kooperationen und Partnerschaften zusammen.

• Pfizer-Forschungseinheit für seltene Krankheiten
• Gentherapien von Novartis
• Roche Molekulardiagnostik

Biotechnologie-Investoren

Ab 2024 zieht ProQR Investitionen von spezialisierten Investorengruppen an, die sich auf Biotechnologie und seltene Krankheiten konzentrieren.

Anlegertyp	Anzahl der Investoren	Gesamtinvestitionsbereich
Risikokapitalfirmen	8-12	50-80 Millionen Dollar
Institutionelle Anleger	15-20	100-150 Millionen Dollar

ProQR Therapeutics N.V. (PRQR) – Geschäftsmodell: Kostenstruktur

Forschungs- und Entwicklungskosten

Für das Geschäftsjahr 2022 meldete ProQR Therapeutics Gesamtaufwendungen für Forschung und Entwicklung in Höhe von 52,4 Millionen US-Dollar. Die Aufteilung dieser Ausgaben ist wie folgt:

F&E-Kategorie	Ausgabenbetrag ($)
Personalkosten	22,1 Millionen
Externe Forschungskosten	18,3 Millionen
Labormaterialien	7,6 Millionen
Technologielizenzierung	4,4 Millionen

Kosten für das Management klinischer Studien

Die Ausgaben für klinische Studien beliefen sich im Jahr 2022 auf insgesamt 34,6 Millionen US-Dollar, mit folgender Aufteilung:

• Kosten für Phase-I/II-Studien: 15,2 Millionen US-Dollar
• Kosten der Phase-III-Studie: 19,4 Millionen US-Dollar

Aufrechterhaltung von Patenten und geistigem Eigentum

ProQR investierte im Jahr 2022 3,2 Millionen US-Dollar in den Schutz geistigen Eigentums und die Aufrechterhaltung von Patenten.

Investitionen in Laborausrüstung und Technologie

Die Investitionen in Technologie und Ausrüstung beliefen sich im Jahr 2022 auf 8,7 Millionen US-Dollar, darunter:

Anlagekategorie	Betrag ($)
Gen-Editing-Technologie	4,3 Millionen
Fortschrittliche Sequenzierungsausrüstung	2,9 Millionen
Computational Biology Tools	1,5 Millionen

Vergütung des wissenschaftlichen Personals

Die Gesamtvergütung des wissenschaftlichen Personals für 2022 belief sich auf 28,5 Millionen US-Dollar und war wie folgt strukturiert:

• Grundgehälter: 18,2 Millionen US-Dollar
• Aktienbasierte Vergütung: 6,3 Millionen US-Dollar
• Leistungsprämien: 4 Millionen US-Dollar

Gesamtkostenstruktur für 2022: 126,9 Millionen US-Dollar

ProQR Therapeutics N.V. (PRQR) – Geschäftsmodell: Einnahmequellen

Mögliche zukünftige Arzneimittellizenzvereinbarungen

Ab dem vierten Quartal 2023 befinden sich bei ProQR Therapeutics potenzielle Lizenzvereinbarungen in der Entwicklung, mit besonderem Schwerpunkt auf Therapien für seltene genetische Krankheiten.

Therapeutischer Bereich	Potenzieller Lizenzwert	Aktueller Status
Seltene genetische Störungen	Mögliche Vorauszahlung in Höhe von 12,5 Millionen US-Dollar	Laufende Verhandlungen
RNA-Therapeutika	Mögliche Meilensteinzahlungen in Höhe von 25 Millionen US-Dollar	Diskussionen im Frühstadium

Forschungsstipendien und staatliche Förderung

ProQR hat sich Forschungsgelder aus mehreren Quellen gesichert.

Finanzierungsquelle	Zuschussbetrag	Jahr
Europäisches Programm Horizont 2020	3,6 Millionen Euro	2023
Niederländischer Forschungsrat	1,2 Millionen Euro	2023

Kollaborative pharmazeutische Entwicklungsverträge

Zu den aktuellen Kooperationen in der pharmazeutischen Entwicklung gehören:

• Zusammenarbeit mit Vertex Pharmaceuticals
• Partnerschaft mit dem Leiden University Medical Center
• Forschungsallianz mit Forschungsinstituten für genetische Krankheiten

Potenzielle Verkäufe therapeutischer Produkte

Umsatzprognose für potenzielle therapeutische Produkte:

Therapeutisches Produkt	Geschätztes jährliches Umsatzpotenzial	Zielmarkt
Sepofarsen (erbliche Netzhautdystrophie)	15-20 Millionen Dollar	Augenheilkunde
Kandidaten für RNA-Therapeutika	10-15 Millionen Dollar	Seltene genetische Störungen

Lizenzierung von geistigem Eigentum

Das Portfolio an geistigem Eigentum von ProQR generiert potenzielle Einnahmequellen.

IP-Kategorie	Anzahl der Patente	Potenzielle Lizenzeinnahmen
RNA-Bearbeitungstechnologien	12 angemeldete Patente	Potenzielle jährliche Lizenzierung im Wert von 5 bis 8 Millionen US-Dollar
Gentherapie-Plattformen	8 angemeldete Patente	Potenzielle jährliche Lizenzierung im Wert von 3 bis 6 Millionen US-Dollar

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Value Propositions

You're looking at the core of what ProQR Therapeutics N.V. is offering the market-it's a fundamental shift in how we treat genetic diseases. The value proposition centers on their proprietary Axiomer™ RNA editing technology platform, which is designed to be a next-generation approach compared to traditional gene therapies.

Pioneering next-generation RNA base editing for genetic diseases

ProQR Therapeutics N.V. is pioneering this next-generation RNA base editing technology called Axiomer™. This technology uses the cell's own editing machinery, specifically ADAR (Adenosine Deaminase Acting on RNA), to make precise, single nucleotide edits directly in the RNA molecule. This DNA-free, reversible editing approach is a key differentiator in the field. The company expects up to four clinical data readouts across its pipeline in 2025 and 2026, signaling an aggressive push to validate the platform.

Correcting disease-causing mutations directly at the RNA level

The core mechanism is the ability to make specific single nucleotide edits in RNA to either reverse a mutation or modulate protein expression. This capability falls under three key applications for the Axiomer pipeline: modulate, correct, and protect. The platform's clinical entry is marked by its lead program, AX-0810, which targets the NTCP protein. The Clinical Trial Application (CTA) for AX-0810 was submitted in Q2 2025, received authorization in October 2025, and initial safety, tolerability, and PK (pharmacokinetics) data from Cohort 1 of the Phase 1 study were expected toward the end of 2025.

Targeting high unmet need diseases like cholestatic diseases (AX-0810)

The immediate focus is on high unmet need areas. AX-0810 is specifically targeting NTCP for cholestatic liver diseases. This investigational RNA editing oligonucleotide is designed to selectively modulate NTCP function to reduce toxic bile acid accumulation in the liver, potentially stopping inflammation, fibrosis, and progression to liver failure. The strategy is grounded in human genetics, as naturally occurring variants like NTCP Q68R are associated with no clinical symptoms related to bile acid levels. The financial backing to pursue this is substantial; as of September 30, 2025, ProQR Therapeutics N.V. held € 106.9 million in cash and cash equivalents, providing a financial runway into mid-2027. This operational spending included Research and development (R&D) costs of € 34.8 million for the nine-month period ending September 30, 2025.

Potential to treat both rare and prevalent diseases with one platform

The platform's value extends beyond liver disease, positioning ProQR Therapeutics N.V. to address a broad spectrum of conditions. The Axiomer technology is intended to yield a new class of medicines for diverse types of diseases, covering both rare and prevalent conditions. The pipeline reflects this diversity, moving beyond the lead candidate. Here's a quick look at the pipeline programs that support this broad potential:

• The lead program, AX-0810, targets NTCP for cholestatic diseases.
• AX-2402 targets MECP2 (R270X) for Rett Syndrome, a rare CNS disorder, supported by a partnership that secured up to $9.2 million from the Rett Syndrome Research Trust.
• AX-2911 is advancing toward clinical candidate selection, targeting MASH (a prevalent liver condition).
• AX-1412 targets B4GALT1 for cardiovascular diseases.

The company's financial health is also supported by external validation, including a collaboration with Eli Lilly, which generated $2.0 million in milestone payments during the first nine months of 2025. The overall net loss for the nine-month period ending September 30, 2025, was € 33.3 million.

Program	Target Indication	Technology Application	Key 2025/2026 Milestone
AX-0810	Cholestatic Liver Diseases (NTCP)	Modulate	Initial Safety/PK Data by year-end 2025
AX-2402	Rett Syndrome (MECP2)	Correct/Modulate	Phase 1/2 Trial planned for 2026
AX-2911	MASH	Modulate	Advancing toward clinical candidate selection in 2025
AX-1412	Cardiovascular Diseases (B4GALT1)	Modulate	Preclinical/Translational updates expected

The platform's ability to address both rare and prevalent diseases using a single, validated RNA editing approach is the central value proposition. If you're looking at the near-term catalysts, the first human data for AX-0810 in Q4 2025 is the key event that will validate the entire Axiomer technology in a clinical setting.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Customer Relationships

ProQR Therapeutics N.V. structures its external relationships around strategic alliances, patient-centric advocacy, and transparent communication with the financial community.

High-touch, collaborative relationships with major pharmaceutical partners

The relationship with Eli Lilly and Company (Lilly) is a cornerstone, representing a significant, long-term collaboration focused on the discovery, development, and commercialization of RNA editing medicines for genetic disorders. This partnership, which began in 2021 and was expanded in 2022, utilizes ProQR Therapeutics N.V.'s proprietary Axiomer™ RNA editing platform. The total potential value of this strategic collaboration is up to $3.9 billion. ProQR Therapeutics N.V. retains an option to expand the collaboration to a total of 15 targets, which would trigger a $50 million opt-in payment to ProQR Therapeutics N.V. The financial flow from this partnership directly supports operations, as evidenced by the $2.0 million (~€ 1.8 million) in milestone income earned by ProQR Therapeutics N.V. during the first nine months of 2025. Lilly also participated in the October 2024 financing, contributing to the $82.1 million in gross proceeds raised then.

Here's a snapshot of the financial interaction with the key pharmaceutical partner:

Metric	Value/Detail
Collaboration Start Year	2021
Total Potential Collaboration Value	$3.9 billion
Potential Opt-in Payment (Additional 5 Targets)	$50 million
Milestone Income (First Nine Months of 2025)	$2.0 million
Cash Reserves from Oct 2024 Financing (Lilly Participation)	$82.1 million (Gross Proceeds)

Close engagement with patient advocacy groups like RSRT

Engagement with patient advocacy groups is critical, particularly for rare diseases like Rett Syndrome. ProQR Therapeutics N.V. has an active, expanded collaboration with the Rett Syndrome Research Trust (RSRT) to advance AX-2402, targeting the MECP2 R270X mutation. This relationship is structured as a co-investment model, where RSRT funding de-risks the program to incentivize full internal development by ProQR Therapeutics N.V. The total funding commitment from RSRT to date is $9.1 million. This total includes an initial $1 million research grant announced in January 2024, followed by an expansion in December 2024 with an additional $8.1 million in funding. ProQR Therapeutics N.V. is matching RSRT's investment in this program.

The RSRT partnership drives specific program milestones:

• Total RSRT funding commitment: $9.1 million
• Initial grant amount (Jan 2024): $1 million
• Additional funding (Dec 2024): $8.1 million
• Program focus: AX-2402 for Rett Syndrome

Investor relations and analyst events for transparency on data readouts

ProQR Therapeutics N.V. maintains active communication with investors and analysts to provide transparency, especially around clinical data milestones. The company hosted a virtual Analyst and Investor Event in November 2025, which detailed the Phase 1 trial design for AX-0810 and outlined expectations for 2025 data. Furthermore, management actively participated in several key industry and investor conferences in late 2025.

Investor engagement events in late 2025 included:

• H.C. Wainwright Genetic Medicines Virtual Conference: October 14-15, 2025 (Presentation available on demand Oct 14 at 7:00 am ET)
• H.C. Wainwright Liver Disease Virtual Conference: October 21-22, 2025 (Presentation available on demand Oct 21 at 7:00 am ET)
• Chardan's 9th Annual Genetic Medicines Conference: October 21, 2025 (Included an RNA Editing panel at 1:00 p.m. ET)
• Virtual Analyst and Investor Event (focused on AX-0810): November 2025
• Evercore Healthcare Conference: December 01, 2025

Archived webcasts from these presentations are typically available for approximately 30 days following the presentation date on the ProQR Therapeutics N.V. website.

Specialized clinical support for trial participants

The relationship with trial participants is managed through rigorous, protocol-driven engagement, particularly as the lead program enters first-in-human testing. ProQR Therapeutics N.V. received Clinical Trial Application (CTA) authorization for AX-0810 and initiated the Phase 1 study in healthy volunteers. The trial is designed to assess specific parameters directly related to the therapeutic approach.

Key elements of the initial clinical engagement for AX-0810 include:

• Trial Status (as of Q3 2025): Initiating Phase 1 study in healthy volunteers.
• Assessment Focus: Safety, tolerability, pharmacokinetics (PK), and target engagement.
• Data Timeline: Initial safety and PK data from Cohort 1 expected by year-end 2025.
• Target Engagement Data: Expected in the first half of 2026 from all cohorts.

The company's cash position as of September 30, 2025, was € 106.9 million, providing a runway into mid-2027, which supports the ongoing operational commitment to these clinical programs.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Channels

You're looking at how ProQR Therapeutics N.V. gets its science and potential therapies in front of partners, regulators, and investors as of late 2025. It's a mix of formal agreements, clinical execution, and scientific dissemination. Honestly, the channels reflect a company deep in the clinical transition phase.

Direct licensing and collaboration agreements with pharmaceutical companies

The core of ProQR Therapeutics N.V.'s external commercial and development channel is through strategic alliances. The most significant is the $3.9 billion strategic collaboration with Eli Lilly and Company ("Lilly"), which started back in 2021 and saw expansion in 2022. This partnership is key for developing their Axiomer® RNA editing platform across multiple targets. ProQR Therapeutics N.V. is still executing on this, having recognized milestone income of $2.0 million through the first nine months of 2025 from Lilly.

Furthermore, the Lilly agreement includes an option for ProQR Therapeutics N.V. to expand to a total of 15 targets, which would trigger a substantial $50 million opt-in payment. On a smaller scale, the collaboration with the Rett Syndrome Research Trust ("RSRT") was expanded in December 2024, securing an additional funding for a total commitment of $9.2 million to support the advancement of AX-2402.

You should also note the historical licensing activity, such as the agreement signed in October 2018 with Ionis Pharmaceuticals for QR-1123, which involved upfront payments totaling $6,001,000 (made in shares) and includes future milestone payments plus royalties of 20% on net sales. To be fair, one older CNS license agreement was terminated as of July 2023.

Clinical trial sites in Europe and North America for drug testing

The primary channel for testing drug candidates like AX-0810 is through formal clinical trials. ProQR Therapeutics N.V. advanced its lead program by submitting a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) in June 2025. Pending regulatory clearance, the first-in-human Phase 1 study for AX-0810 is expected to commence at a single site in the Netherlands. This is the first clinical development milestone for their Axiomer™ platform.

While the initial trial is in Europe, the target patient populations for their lead programs are significant across both regions. For instance, Primary Sclerosing Cholangitis (PSC) and Biliary Atresia (BA) are estimated to affect 80,000 and 20,000 individuals in North America and Europe, respectively. The company maintains a U.S. office in Cambridge, MA, which supports North American operations and engagement.

Here's a quick look at the financial context surrounding these clinical advancements as of late 2025:

Financial Metric (as of September 30, 2025)	Amount (in millions)	Context
Cash and Cash Equivalents	€ 106.9 million	Provides runway into mid-2027.
Net Cash Used in Operating Activities (9 months 2025)	€ 39.4 million	Reflects ongoing R&D and clinical spend.
Research & Development (R&D) Costs (9 months 2025)	€ 34.8 million	Significant investment into pipeline advancement.

Scientific publications and conferences to showcase platform data

Disseminating platform data is a crucial channel for building scientific credibility and attracting future partners or investment. ProQR Therapeutics N.V. actively uses scientific venues to present its Axiomer technology. You can see evidence of this through their participation in key 2025 events, including the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting and the Oligonucleotide and Peptide Therapeutics Conference (TIDES USA) in May, and the RNA Editing Summit in July.

The company also uses dedicated investor events to detail trial designs and data expectations. They hosted a virtual Analyst and Investor Event in the fall of 2025, which focused on the AX-0810 Phase 1 trial design. The company is positioned to deliver initial safety and PK data from the first cohort of the AX-0810 trial by the end of 2025, with target engagement data expected in the first half of 2026. They anticipate up to four clinical data readouts across 2025 and 2026.

• Presentations included data on the Axiomer ADAR-mediated RNA editing platform.
• Specific abstracts presented at ASGCT 2025 related to NTCP and PNPLA3.
• The company plans to provide an update on AX-1412 in mid-2025.

Investor communications for capital market access

Access to capital markets is a direct channel for funding operations, especially given the net loss reported. ProQR Therapeutics N.V. ended the third quarter of 2025 with € 106.9 million in cash and cash equivalents, which supports a financial runway extending well into mid-2027. This follows a successful underwritten public offering in October 2024 that generated gross proceeds of $82.1 million.

The company's financial reporting itself serves as a primary communication channel. They reported a net loss of € 33.3 million for the nine-month period ending September 30, 2025. For ongoing capital market access, ProQR Therapeutics N.V. has several effective registration statements on Form F-3 on file with the SEC, including File No. 333-282419. The company also uses press releases to announce key operational and financial milestones, such as the Q3 2025 results on November 6, 2025.

The key financial metrics related to investor confidence and capital structure as of late 2025 are summarized below:

Financial Event/Data Point	Date/Period	Value
Gross Proceeds from Oct 2024 Offering	October 2024	$82.1 million
Cash & Equivalents	September 30, 2025	€ 106.9 million
Net Loss	Nine Months Ended Sept 30, 2025	€ 33.3 million
Lilly Collaboration Milestone Income	First 9 Months of 2025	$2.0 million

Finance: draft 13-week cash view by Friday.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Customer Segments

You're looking at the core groups ProQR Therapeutics N.V. is targeting with its Axiomer RNA editing platform as of late 2025. These segments are defined by the specific, high unmet medical need diseases their pipeline programs address, and the strategic partners needed to fund and commercialize the technology.

Large pharmaceutical companies seeking novel RNA editing technology represent a major segment, primarily through strategic alliances that provide non-dilutive funding and future commercial reach. The collaboration with Eli Lilly is a prime example of this segment engagement.

The company's financial health, which supports engaging these segments, showed cash and cash equivalents of approximately €106.9 million at September 30, 2025, providing runway into mid-2027.

Partner/Segment Type	Program Focus	Financial Metric/Value	Data Point (as of late 2025)
Large Pharmaceutical Partner (Eli Lilly)	Axiomer Platform Technology	Total Collaboration Value	$3.9 billion (Source 11)
Large Pharmaceutical Partner (Eli Lilly)	Milestone Achievement	Income YTD Q3 2025	$2.0 million (~€ 1.8 million) (Source 1, 6)
Large Pharmaceutical Partner (Eli Lilly)	Potential Future Value	Opt-in Payment for 5 Additional Targets	$50 million (Source 2)

Patients with severe cholestatic liver diseases (NTCP target) are targeted by the lead program, AX-0810. This segment is critical as it represents the first clinical validation of the Axiomer platform.

• Program: AX-0810, modulating NTCP to reduce bile acid accumulation.
• Clinical Status: CTA authorization received in October 2025 (Source 1, 6).
• Trial Location: Phase 1 study initiating at a single site in the Netherlands (Source 8).
• Near-Term Readout: Initial safety, tolerability, and PK data for Cohort 1 expected by year-end 2025 (Source 1, 6).
• Follow-up Readout: Target engagement data across cohorts expected in H1 2026 (Source 1, 6).

Patients with rare neurodevelopmental disorders like Rett Syndrome (MECP2 target) are addressed by the CNS program, AX-2402. This segment is characterized by a severe, rare genetic condition with high unmet need.

• Program: AX-2402, targeting the R270X mutation in the MECP2 gene.
• Patient Population Size: Rett Syndrome affects approximately 350,000 people worldwide (Source 9).
• Targeted Subset: Mutations addressable by this RNA editing approach account for 40% of all Rett cases (Source 10).
• Development Status: Advancing toward clinical candidate selection as of Q2 2025 (Source 8).

Patient advocacy and research foundations funding drug development are essential early-stage customers/supporters, particularly for rare disease programs where patient advocacy drives research funding.

The Rett Syndrome Research Trust (RSRT) is a key example of this segment providing direct, non-dilutive funding to advance the AX-2402 program.

• Foundation Partner: Rett Syndrome Research Trust (RSRT).
• Total Funding Secured: Up to $9.2 million to support advancement into clinical trials (Source 2).
• Funding Context: This funding supports IND-enabling studies, with ProQR Therapeutics N.V. matching the investment (Source 10).

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Cost Structure

You're looking at the core expenditures driving ProQR Therapeutics N.V.'s operations as of late 2025. For a development-stage biotech, the cost structure is almost entirely weighted toward getting the science through the clinic and protecting the underlying technology.

Dominant Research and Development (R&D) expenses are the clear cost leader. For the nine-month period ended September 30, 2025, R&D costs hit € 34.8 million. This is a significant jump from the € 25.7 million reported for the same period in 2024, showing the increased investment as programs mature. Net cash used in operating activities for those nine months was € 39.4 million, meaning R&D accounts for the vast majority of cash burn.

The primary driver for this elevated R&D spend is the Clinical trial costs for AX-0810 and preclinical advancement. ProQR Therapeutics N.V. achieved a major milestone with the CTA authorization and is initiating the first-in-human Phase 1 study for AX-0810, which targets NTCP for cholestatic diseases.

Here's a quick look at how the main operating costs stacked up for the first nine months of 2025:

Cost Category	Amount (9M 2025)	Comparison Point
Research and Development (R&D)	€ 34.8 million	Up from € 25.7 million (9M 2024)
General and Administrative (G&A)	€ 11.2 million	Up from € 9.7 million (9M 2024)
Total Operating Costs (Implied from Net Loss & Revenue)	Approx. € 45.6 million	Net Loss of € 33.3M + Revenue of € 11.2M (9M 2025)

The R&D expenditure covers several critical, high-cost activities:

• Costs associated with the Phase 1 trial initiation for AX-0810.
• Manufacturing and supply chain setup for clinical material.
• Ongoing work supporting preclinical advancement of other pipeline assets, like AX-2402 for Rett Syndrome.
• Platform costs related to the Axiomer RNA editing technology.

General and Administrative (G&A) costs were € 11.2 million for the nine-month period ending September 30, 2025. This reflects the necessary overhead to run a public company advancing clinical trials. This category includes things like executive salaries, finance, and legal functions.

A key, though often bundled, component of the G&A and R&D spend is Intellectual property maintenance and patent prosecution fees. You have to spend money to keep the Axiomer platform protected globally. This is a non-negotiable cost of doing business in this space, ensuring exclusivity for their novel RNA editing approach.

To be fair, some of the cost pressure is offset by partnership income. During the first nine months of 2025, ProQR Therapeutics N.V. achieved milestones from its collaboration agreement with Eli Lilly amounting to $2.0 million, which is roughly € 1.8 million. That income helps temper the overall cash burn, but the underlying operational costs remain high as they push AX-0810 toward initial data readouts by year-end 2025.

Finance: draft 13-week cash view by Friday.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Revenue Streams

You're looking at the core ways ProQR Therapeutics N.V. brings in cash right now, which is heavily weighted toward partnerships and non-dilutive funding as they advance their Axiomer RNA editing platform.

The most immediate, concrete revenue source comes from the strategic collaboration with Eli Lilly and Company (Lilly). During the nine-month period ended September 30, 2025, ProQR Therapeutics N.V. achieved milestones under this agreement amounting to exactly $2.0 million. This is a key near-term income driver while the pipeline matures.

Another vital component of ProQR Therapeutics N.V.'s funding structure involves non-dilutive capital from non-profit organizations. Specifically, the collaboration with the Rett Syndrome Research Trust (RSRT) has been expanded. The total funding secured from RSRT to support the advancement of AX-2402 is $9.1 million, which includes an initial grant of $1.0 million and an additional $8.1 million announced in late 2024.

The potential for significant future revenue is tied directly to the success of these partnerships, particularly the Lilly agreement. This collaboration includes an option for Lilly to expand to a total of 15 targets, which would trigger a substantial future opt-in payment to ProQR Therapeutics N.V. of $50 million.

The long-term revenue vision for ProQR Therapeutics N.V. rests on successful commercialization, which translates into future royalties and product sales once any of their pipeline candidates, like AX-0810, achieve regulatory approval and market entry. Still, as of late 2025, the company's reported revenue for the nine-month period ended September 30, 2025, was €11.22 million.

Here's a quick look at the reported revenue and cash position as of the end of Q3 2025:

Financial Metric	Amount (9M 2025)	Amount (Q3 2025)
Total Sales/Revenue	€11.22 million	EUR 2.88 million
Lilly Milestone Payments	$2.0 million	Not Specified
Cash & Cash Equivalents (as of Sep 30)	€106.9 million	€106.9 million
Net Cash Used in Operating Activities	€39.4 million	Not Specified

The revenue streams can be categorized by their source and expected timing:

• Milestone Payments from Eli Lilly and Company
• Research Grants from Non-Profit Entities (e.g., RSRT)
• Future Opt-In Payments (e.g., $50 million from Lilly expansion)
• Long-Term Royalties and Product Sales

You can see the RSRT funding is a significant non-dilutive source supporting specific program advancement:

• RSRT Total Funding Secured: $9.1 million
• RSRT Additional Funding (Dec 2024): $8.1 million
• Initial RSRT Grant (Jan 2024): $1.0 million

Finance: review the Q4 2025 cash burn rate against the mid-2027 runway projection by next Tuesday.