ProQR Therapeutics N.V. (PRQR): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

ProQR Therapeutics N.V. (PRQR) emerge como una innovadora compañía de biotecnología que revoluciona el panorama del tratamiento de enfermedad genética rara a través de su innovador enfoque terapéutico de ARN. Al aprovechar la biología molecular de vanguardia y las tecnologías de edición de genes, esta organización pionera está transformando la forma en que entendemos y potencialmente cuoramos trastornos genéticos previamente no tratables. Su modelo de negocio único combina excelencia científica, asociaciones estratégicas y un compromiso centrado en el láser para desarrollar intervenciones genéticas personalizadas que podrían ofrecer esperanza a pacientes con afecciones genéticas raras y complejas.

ProQR Therapeutics N.V. (PRQR) - Modelo de negocio: asociaciones clave

Instituciones de investigación académica

PROQR Therapeutics colabora con las siguientes instituciones de investigación académica:

Institución	Enfoque de investigación	Detalles de colaboración
Centro Médico de la Universidad de Leiden	Investigación de enfermedades genéticas	Desarrollo terapéutico de ARN
Escuela de Medicina de Harvard	Trastornos genéticos raros	Colaboración de genética molecular

Compañías farmacéuticas

Las colaboraciones farmacéuticas clave incluyen:

• Novartis - Asociación para terapias de enfermedad genética raras
• Roche - Colaboración del desarrollo de medicamentos

Organizaciones de investigación por contrato (CRO)

PROQR funciona con múltiples CRO para ensayos clínicos:

Nombre de Cro	Fase de ensayo clínico	Estudios activos
IQVIA	Fase II/III	3 ensayos de trastorno genético en curso
Parexel	Fase I/II	2 ensayos terapéuticos de ARN

Laboratorios de pruebas genéticas

Asociaciones con laboratorios de pruebas genéticas:

• Invitae Corporation: detección genética del paciente
• Salud genómica - Colaboración de diagnóstico molecular

Empresas de inversión de biotecnología

Socios de apoyo de financiación:

Firma de inversión	Monto de la inversión	Año de inversión
Orbimed Advisors	$ 45 millones	2022
Versant Ventures	$ 30 millones	2021

ProQR Therapeutics N.V. (PRQR) - Modelo de negocio: actividades clave

Investigación y desarrollo terapéutico de ARN

PROQR Therapeutics se centra en el desarrollo de la terapéutica de ARN para enfermedades genéticas raras. A partir de 2023, la compañía ha invertido $ 37.4 millones en gastos de investigación y desarrollo.

Área de investigación	Inversión (2023)	Programas activos
Desarrollo terapéutico de ARN	$ 37.4 millones	4 programas primarios de enfermedades genéticas

Innovación de tecnología de edición de genes y reparación de ARN

La Compañía utiliza tecnologías de plataforma de edición de ARN propietarias con enfoque actual en:

• Tecnología de edición de AXIOMER ARN
• Técnicas de edición de ARN de precisión
• Plataforma Linc para modificaciones genéticas

Gestión de ensayos preclínicos y clínicos

Fase de prueba	Número de pruebas en curso	Inversión total
Pruebas preclínicas	3 programas	$ 12.6 millones
Ensayos clínicos	2 pruebas activas	$ 22.8 millones

Desarrollo de fármacos de enfermedad genética rara

PROQR se concentra en el desarrollo de la terapéutica para trastornos genéticos raros específicos, con la orientación actual de la tubería:

• Fibrosis quística
• Síndrome de usher
• Distrofia muscular de Duchenne

Protección de propiedad intelectual y presentación de patentes

Categoría de patente	Número de patentes	Duración de protección de patentes
Tecnología de edición de ARN	17 patentes otorgadas	20 años desde la fecha de presentación
Métodos terapéuticos genéticos	9 solicitudes de patentes pendientes	Potencial de protección de 20 años

ProQR Therapeutics N.V. (PRQR) - Modelo de negocio: recursos clave

Tecnología de la plataforma de edición de ARN patentada

ProQR Therapeutics mantiene una plataforma de edición de ARN especializada con las siguientes características clave:

Atributo tecnológico	Detalles específicos
Nombre de la plataforma	Plataforma de edición de ARN
Solicitudes de patentes	17 familias de patentes a partir de 2023
Enfoque tecnológico	Edición y modificación de la base de ARN

Experiencia de investigación genética especializada

Las capacidades de investigación de ProQR incluyen:

• 15 científicos de investigación dedicados
• Experiencia de investigación promedio de 12.5 años
• Experiencia en trastornos genéticos raros

Instalaciones avanzadas de laboratorio de biología molecular

Especificación de la instalación	Datos cuantitativos
Espacio total de laboratorio	2.500 metros cuadrados
Inversión de equipos avanzados	$ 4.3 millones en 2023
Instrumentos de investigación	42 instrumentos especializados de biología molecular

Cartera de propiedades intelectuales

Detalles de IP clave:

• 17 familias de patentes
• Cubriendo tecnologías de edición de ARN
• Cobertura geográfica a través de Estados Unidos, UE y Asia

Equipo científico e de investigación experto

Composición del equipo	Datos cuantitativos
Personal de investigación total	45 empleados
Titulares de doctorado	28 investigadores
Inversión de capacitación anual	$620,000

ProQR Therapeutics N.V. (PRQR) - Modelo de negocio: propuestas de valor

Terapias de ARN dirigidas para trastornos genéticos raros

PROQR Therapeutics se centra en el desarrollo de terapias de ARN específicamente dirigidas a trastornos genéticos raros con opciones de tratamiento limitadas o no existentes.

Área terapéutica	Trastorno objetivo	Etapa de desarrollo	Potencial de población de pacientes
Fibrosis quística	Mutación f508del	Fase 2/3 ensayos clínicos	Aproximadamente 30,000-70,000 pacientes a nivel mundial
Síndrome de usher	Mutación genética tipo 2	Etapa preclínica	Estimado 4-17 por cada 100,000 personas

Posibles tratamientos innovadores para afecciones no tratables

La plataforma de edición de ARN patentada de ProQR permite intervenciones potenciales para las condiciones genéticas que actualmente carecen de soluciones terapéuticas.

• Tecnología de reparación de ARN única dirigida a mutaciones genéticas específicas
• Enfoque de medicina de precisión con intervenciones genéticas personalizadas
• Mecanismos de corrección molecular avanzados

Estrategias de intervención de enfermedades genéticas personalizadas

La propuesta de valor de la Compañía se centra en el desarrollo de terapias de ARN personalizadas adaptadas a mutaciones genéticas específicas.

Plataforma tecnológica	Capacidad de personalización	Impacto potencial
Plataforma de edición de ARN	Orientación específica de mutación	Tratamiento potencial para afecciones genéticas previamente no tratables

Tecnologías avanzadas de reparación molecular

Las capacidades tecnológicas de PROQR incluyen plataformas de edición de ARN patentadas diseñadas para abordar las mutaciones genéticas a nivel molecular.

• Inversión tecnológica: $ 42.3 millones en gastos de I + D (año fiscal 2022)
• Mecanismos de reparación de ARN patentados
• Estrategias de intervención genética de precisión

Enfoque innovador para el manejo de enfermedades genéticas

La propuesta de valor distintivo de ProQR radica en su enfoque integral para el manejo de enfermedades genéticas a través de tecnologías avanzadas de ARN.

Métrica de innovación	Valor	Ventaja comparativa
Cartera de patentes	17 patentes otorgadas	Protección tecnológica única
Colaboración de investigación	3 asociaciones farmacéuticas activas	Desarrollo tecnológico mejorado

ProQR Therapeutics N.V. (PRQR) - Modelo de negocios: relaciones con los clientes

Compromiso directo con enfermedades raras comunidades de pacientes

PROQR Therapeutics se involucra con las comunidades de los pacientes a través de estrategias de divulgación específicas:

Grupo comunitario de pacientes	Método de compromiso	Frecuencia de interacción anual
Pacientes con fibrosis quística	Redes de soporte en línea	12-15 eventos virtuales
Pacientes con enfermedad de la retina	Juntas de asesoramiento de pacientes	6-8 reuniones estratégicas
Comunidades de desorden genético	Plataformas de comunicación digital	24 puntos de contacto digitales

Conferencia científica y presentaciones de simposio médico

ProQR participa activamente en conferencias científicas para mostrar el progreso de la investigación:

• Participación anual: 8-10 conferencias médicas internacionales
• Formatos de presentación: presentaciones orales, sesiones de carteles, paneles expertos
• Conferencias objetivo: simposios de enfermedades raras, conferencias de terapia genética

Asociaciones de investigación colaborativa

ProQR mantiene colaboraciones de investigación estratégica:

Tipo de socio	Número de asociaciones activas	Inversión de investigación anual
Instituciones académicas	5-7 asociaciones	$ 3.2 millones
Investigar hospitales	3-4 proyectos de colaboración	$ 2.1 millones
Centros de investigación farmacéutica	2-3 iniciativas de investigación conjunta	$ 1.5 millones

Programas de apoyo y educación del paciente

PROQR implementa iniciativas integrales de apoyo al paciente:

• Recursos educativos en línea: 15-20 módulos informativos
• Limpieza del paciente: disponibilidad de soporte 24/7
• Servicios de asesoramiento genético: opciones de consulta personalizada

Comunicación transparente sobre el progreso de la investigación

Estrategias de comunicación para la transparencia de la investigación:

Canal de comunicación	Frecuencia de actualizaciones	Alcanzar
Sitio web corporativo	Actualizaciones de investigación mensuales	Acceso global a las partes interesadas
Comunicaciones de relaciones con los inversores	Informes de progreso trimestral	Comunidad de inversores y analistas
Publicaciones de publicación científica	4-6 Publicaciones revisadas por pares anualmente	Comunidad de investigación científica

ProQR Therapeutics N.V. (PRQR) - Modelo de negocio: canales

Publicaciones científicas directas

ProQR Therapeutics publica investigación en revistas revisadas por pares como:

Nombre del diario	Frecuencia de publicación	Factor de impacto
Biotecnología de la naturaleza	Mensual	41.4
Celúla	Quincenal	38.6
Medicina de traducción de la ciencia	Semanalmente	16.9

Presentaciones de conferencia médica

Detalles anuales de participación de la conferencia:

Conferencia	Ubicación	Recuento de presentación
Sociedad Americana de Gene & Terapia celular	Baltimore, MD	4
Sociedad Europea de Gene & Terapia celular	Barcelona, ​​España	3

Relaciones con inversores de la industria biotecnología

Canales de comunicación de inversores:

• Llamadas de ganancias trimestrales
• Reuniones anuales de accionistas
• Presentaciones de inversores
• SEC presentando comunicaciones

Plataformas de comunicación científica en línea

Plataformas de compromiso digital:

Plataforma	Seguidores/miembros	Tipo de contenido
Investigador	12,500	Publicaciones de investigación
LinkedIn	8,700	Actualizaciones corporativas

Eventos de redes de la industria farmacéutica

Plataformas de redes clave:

• Convención Internacional de la Organización de Innovación Biotecnología (BIO)
• Conferencia de atención médica de JP Morgan
• Conferencia de asociación de enfermedades genéticas

ProQR Therapeutics N.V. (PRQR) - Modelo de negocio: segmentos de clientes

Pacientes de enfermedad genética rara

PROQR Therapeutics se centra en pacientes con trastornos genéticos raros específicos. A partir de 2024, la compañía se dirige a aproximadamente 2,000-5,000 pacientes a nivel mundial por cada condición genética.

Desorden genético	Población de pacientes estimada	Tamaño del mercado objetivo
Fibrosis quística (mutación f508del)	30,000-40,000 pacientes	América del Norte y Europa
Síndrome de usher	4,000-6,000 pacientes	Mundial

Instituciones de investigación genética

PROQR colabora con 15-20 instituciones de investigación líderes a nivel mundial, centrándose en la investigación de enfermedades genéticas.

• Escuela de Medicina de Harvard
• Departamento de Genética de la Universidad de Stanford
• Universidad de California, San Francisco

Médicos especializados

El grupo de clientes objetivo incluye aproximadamente 500-700 genetistas y profesionales médicos especializados en todo el mundo.

Especialidad	Número de practicantes	Enfoque geográfico
Especialistas genéticos	350-450	Estados Unidos
Clínicos de enfermedades raras	150-250	Europa y América del Norte

Departamentos de investigación farmacéutica

PROQR se involucra con 25-30 departamentos de investigación farmacéutica para posibles colaboraciones y asociaciones.

• Unidad de investigación de enfermedades raras de Pfizer
• Terapias genéticas de Novartis
• Roche Molecular Diagnostics

Inversores de biotecnología

A partir de 2024, PROQR atrae la inversión de biotecnología especializada y grupos de inversores centrados en las enfermedades raras.

Tipo de inversor	Número de inversores	Rango de inversión total
Empresas de capital de riesgo	8-12	$ 50-80 millones
Inversores institucionales	15-20	$ 100-150 millones

ProQR Therapeutics N.V. (PRQR) - Modelo de negocio: Estructura de costos

Gastos de investigación y desarrollo

Para el año fiscal 2022, PROQR Therapeutics reportó gastos totales de I + D de $ 52.4 millones. El desglose de estos gastos es el siguiente:

Categoría de I + D	Monto de gasto ($)
Costos de personal	22.1 millones
Costos de investigación externos	18.3 millones
Materiales de laboratorio	7.6 millones
Licencias de tecnología	4.4 millones

Costos de gestión de ensayos clínicos

Los gastos de ensayo clínico para 2022 totalizaron $ 34.6 millones, con la siguiente asignación:

• Gastos de prueba de fase I/II: $ 15.2 millones
• Fase III Gastos de prueba: $ 19.4 millones

Mantenimiento de patentes e propiedad intelectual

ProQR invirtió $ 3.2 millones en protección de propiedad intelectual y mantenimiento de patentes durante 2022.

Inversiones de equipos e tecnología de laboratorio

Las inversiones en tecnología y equipos para 2022 fueron de $ 8.7 millones, que incluyen:

Categoría de inversión	Monto ($)
Tecnología de edición de genes	4.3 millones
Equipo de secuenciación avanzado	2.9 millones
Herramientas de biología computacional	1.5 millones

Compensación de personal científico

La compensación total del personal científico para 2022 fue de $ 28.5 millones, estructurada de la siguiente manera:

• Salarios base: $ 18.2 millones
• Compensación basada en acciones: $ 6.3 millones
• Bonos de rendimiento: $ 4 millones

Estructura de costos totales para 2022: $ 126.9 millones

ProQR Therapeutics N.V. (PRQR) - Modelo de negocio: flujos de ingresos

Acuerdos potenciales de licencia de medicamentos futuros

A partir del cuarto trimestre de 2023, PROQR Therapeutics tiene acuerdos de licencia potenciales en el desarrollo, con un enfoque específico en las terapias de enfermedad genética raras.

Área terapéutica	Valor de licencia potencial	Estado actual
Trastornos genéticos raros	$ 12.5 millones de pago inicial potencial	Negociaciones continuas
Terapéutica de ARN	Pagos de hitos potenciales de $ 25 millones	Discusiones en etapa inicial

Subvenciones de investigación y financiación del gobierno

ProQR ha obtenido fondos de investigación de múltiples fuentes.

Fuente de financiación	Monto de subvención	Año
Programa European Horizon 2020	3.6 millones de euros	2023
Consejo de Investigación Holandesa	€ 1.2 millones	2023

Contratos de desarrollo farmacéutico colaborativo

Las colaboraciones actuales de desarrollo farmacéutico incluyen:

• Colaboración con Vertex Pharmaceuticals
• Asociación con el Centro Médico de la Universidad de Leiden
• Alianza de investigación con enfermedades genéticas Institutos de Investigación

Venta de productos terapéuticos potenciales

Proyección de ingresos para posibles productos terapéuticos:

Producto terapéutico	Potencial de ingresos anual estimado	Mercado objetivo
Sepofarsen (distrofia retiniana hereditaria)	$ 15-20 millones	Oftalmología
Candidatos terapéuticos de ARN	$ 10-15 millones	Trastornos genéticos raros

Licencia de propiedad intelectual

La cartera de propiedades intelectuales de ProQR genera posibles flujos de ingresos.

Categoría de IP	Número de patentes	Ingresos potenciales de licencia
Tecnologías de edición de ARN	12 patentes registradas	$ 5-8 millones de posibles licencias anuales
Plataformas de terapia genética	8 patentes registradas	$ 3-6 millones de posibles licencias anuales

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.