Proqr Therapeutics N.V. (PRQR): Modelo de negócios Canvas [Jan-2025 Atualizado]

A Proqr Therapeutics N.V. (PRQR) surge como uma empresa inovadora de biotecnologia que revoluciona a paisagem do tratamento raro de doenças genéticas por meio de sua inovadora abordagem terapêutica de RNA. Ao alavancar a biologia molecular de ponta e as tecnologias de edição de genes, essa organização pioneira está transformando a maneira como entendemos e potencialmente curamos distúrbios genéticos anteriormente intratáveis. Seu modelo de negócios exclusivo combina excelência científica, parcerias estratégicas e um compromisso focado a laser em desenvolver intervenções genéticas personalizadas que poderiam oferecer esperança a pacientes com condições genéticas raras e complexas.

Proqr Therapeutics N.V. (PRQR) - Modelo de negócios: Parcerias -chave

Instituições de pesquisa acadêmica

A Proqr Therapeutics colabora com as seguintes instituições de pesquisa acadêmica:

Instituição	Foco na pesquisa	Detalhes da colaboração
Centro Médico da Universidade de Leiden	Pesquisa de doenças genéticas	Desenvolvimento terapêutico de RNA
Escola de Medicina de Harvard	Distúrbios genéticos raros	Colaboração de genética molecular

Empresas farmacêuticas

As principais colaborações farmacêuticas incluem:

• Novartis - Parceria para terapias de doenças genéticas raras
• Roche - Colaboração de Desenvolvimento de Medicamentos

Organizações de pesquisa contratada (CROs)

O Proqr trabalha com vários CROs para ensaios clínicos:

Nome do CRO	Fase de ensaios clínicos	Estudos ativos
Iqvia	Fase II/III	3 ensaios de transtorno genético em andamento
Parexel	Fase I/II	2 ensaios terapêuticos de RNA

Laboratórios de testes genéticos

Parcerias com laboratórios de testes genéticos:

• Invitae Corporation - Triagem genética do paciente
• Colaboração genômica de saúde - diagnóstico molecular

Empresas de investimento em biotecnologia

Parceiros de suporte de financiamento:

Empresa de investimentos	Valor do investimento	Ano de investimento
Consultores orbimed	US $ 45 milhões	2022
Ventuos versantes	US $ 30 milhões	2021

Proqr Therapeutics N.V. (PRQR) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento terapêuticos de RNA

A Proqr Therapeutics se concentra no desenvolvimento de terapêutica de RNA para doenças genéticas raras. Em 2023, a empresa investiu US $ 37,4 milhões em despesas de pesquisa e desenvolvimento.

Área de pesquisa	Investimento (2023)	Programas ativos
Desenvolvimento terapêutico de RNA	US $ 37,4 milhões	4 programas primários de doenças genéticas

Edição de genes e inovação em tecnologia de reparo de RNA

A empresa utiliza tecnologias proprietárias de plataforma de edição de RNA com foco atual em:

• Tecnologia de edição de RNA axiômero
• Técnicas de edição de RNA de precisão
• Linc Platform para modificações genéticas

Gerenciamento de ensaios pré -clínicos e clínicos

Fase de teste	Número de ensaios em andamento	Investimento total
Ensaios pré -clínicos	3 programas	US $ 12,6 milhões
Ensaios clínicos	2 ensaios ativos	US $ 22,8 milhões

Desenvolvimento de medicamentos para doenças genéticas raras

O ProQR concentra -se no desenvolvimento da terapêutica para distúrbios genéticos raros específicos, com o direcionamento atual do pipeline:

• Fibrose cística
• Síndrome de Usher
• Distrofia muscular de Duchenne

Proteção à propriedade intelectual e arquivamento de patentes

Categoria de patentes	Número de patentes	Duração da proteção de patentes
Tecnologia de edição de RNA	17 patentes concedidas	20 anos a partir da data de arquivamento
Métodos terapêuticos genéticos	9 pedidos de patente pendente	Proteção potencial de 20 anos

Proqr Therapeutics N.V. (PRQR) - Modelo de negócios: Recursos -chave

Tecnologia de plataforma de edição de RNA proprietária

A Proqr Therapeutics mantém uma plataforma de edição de RNA especializada com as seguintes características -chave:

Atributo de tecnologia	Detalhes específicos
Nome da plataforma	Plataforma de edição de RNA
Aplicações de patentes	17 famílias de patentes a partir de 2023
Foco em tecnologia	Edição e modificação da base de RNA

Experiência especializada em pesquisa genética

Os recursos de pesquisa da Proqr incluem:

• 15 cientistas de pesquisa dedicados
• Experiência média de pesquisa de 12,5 anos
• Experiência em distúrbios genéticos raros

Instalações de Laboratório de Biologia Molecular Avançada

Especificação da instalação	Dados quantitativos
Espaço total de laboratório	2.500 metros quadrados
Investimento avançado de equipamento	US $ 4,3 milhões em 2023
Instrumentos de pesquisa	42 Instrumentos Especializados de Biologia Molecular

Portfólio de propriedade intelectual

Principais detalhes do IP:

• 17 famílias de patentes
• Cobrindo tecnologias de edição de RNA
• Cobertura geográfica em nós, UE e Ásia

Equipe científica e de pesquisa qualificada

Composição da equipe	Dados quantitativos
Pessoal de pesquisa total	45 funcionários
Titulares de doutorado	28 pesquisadores
Investimento anual de treinamento	$620,000

Proqr Therapeutics N.V. (PRQR) - Modelo de negócios: proposições de valor

Terapias de RNA direcionadas para distúrbios genéticos raros

A Proqr Therapeutics se concentra no desenvolvimento de terapias de RNA direcionadas especificamente a distúrbios genéticos raros com opções de tratamento limitadas ou nenhuma existente.

Área terapêutica	Transtorno alvo	Estágio de desenvolvimento	Potencial população de pacientes
Fibrose cística	Mutação f508del	Fase 2/3 Ensaios Clínicos	Aproximadamente 30.000 a 70.000 pacientes globalmente
Síndrome de Usher	Mutação genética tipo 2	Estágio pré -clínico	Estimado 4-17 por 100.000 indivíduos

Possíveis tratamentos inovadores para condições intratáveis

A plataforma de edição de RNA proprietária da ProQR permite intervenções em potencial para condições genéticas atualmente sem soluções terapêuticas.

• Tecnologia exclusiva de reparo de RNA direcionada a mutações genéticas específicas
• Abordagem de medicina de precisão com intervenções genéticas personalizadas
• Mecanismos avançados de correção molecular

Estratégias personalizadas de intervenção em doenças genéticas

A proposta de valor da empresa centra -se no desenvolvimento de terapias de RNA personalizadas adaptadas a mutações genéticas específicas.

Plataforma de tecnologia	Capacidade de personalização	Impacto potencial
Plataforma de edição de RNA	Motivo específico da mutação	Tratamento potencial para condições genéticas anteriormente intratáveis

Tecnologias avançadas de reparo molecular

As capacidades tecnológicas da ProQR incluem plataformas proprietárias de edição de RNA projetadas para lidar com mutações genéticas no nível molecular.

• Investimento tecnológico: US $ 42,3 milhões em despesas de P&D (ano fiscal de 2022)
• Mecanismos patenteados de reparo de RNA
• Estratégias de intervenção genética de precisão

Abordagem inovadora ao gerenciamento de doenças genéticas

A proposta de valor distintiva da ProQR reside em sua abordagem abrangente ao gerenciamento de doenças genéticas por meio de tecnologias avançadas de RNA.

Métrica de inovação	Valor	Vantagem comparativa
Portfólio de patentes	17 patentes concedidas	Proteção tecnológica única
Colaboração de pesquisa	3 parcerias farmacêuticas ativas	Desenvolvimento tecnológico aprimorado

Proqr Therapeutics N.V. (PRQR) - Modelo de negócios: Relacionamentos do cliente

Engajamento direto com comunidades de pacientes com doenças raras

A Proqr Therapeutics se envolve com as comunidades de pacientes por meio de estratégias de divulgação direcionadas:

Grupo Comunitário de Pacientes	Método de engajamento	Frequência de interação anual
Pacientes com fibrose cística	Redes de suporte on -line	12-15 Eventos virtuais
Pacientes de doença da retina	Conselhos de consultoria de pacientes	6-8 reuniões estratégicas
Comunidades de Transtorno Genético	Plataformas de comunicação digital	24 pontos de contato digitais

Apresentações de Conferência Científica e Simpósio Médico

O Proqr participa ativamente de conferências científicas para mostrar o progresso da pesquisa:

• Participação anual: 8-10 Conferências Médicas Internacionais
• Formatos de apresentação: apresentações orais, sessões de pôsteres, painéis especializados
• Conferências -alvo: simpósios de doenças raras, conferências de terapia genética

Parcerias de pesquisa colaborativa

O PROQR mantém colaborações estratégicas de pesquisa:

Tipo de parceiro	Número de parcerias ativas	Investimento anual de pesquisa
Instituições acadêmicas	5-7 parcerias	US $ 3,2 milhões
Hospitais de pesquisa	3-4 projetos colaborativos	US $ 2,1 milhões
Centros de pesquisa farmacêutica	2-3 Iniciativas de pesquisa conjunta	US $ 1,5 milhão

Programas de apoio ao paciente e educação

O Proqr implementa iniciativas abrangentes de apoio ao paciente:

• Recursos educacionais online: 15-20 módulos informativos
• Helpline do paciente: disponibilidade de suporte 24/7
• Serviços de aconselhamento genético: opções de consulta personalizadas

Comunicação transparente sobre o progresso da pesquisa

Estratégias de comunicação para a transparência de pesquisa:

Canal de comunicação	Frequência de atualizações	Alcançar
Site corporativo	Atualizações mensais de pesquisa	Acesso global às partes interessadas
Comunicações de Relações com Investidores	Relatórios trimestrais de progresso	Comunidade de investidores e analistas
Submissões de publicação científica	4-6 publicações revisadas por pares anualmente	Comunidade de pesquisa científica

Proqr Therapeutics N.V. (PRQR) - Modelo de negócios: canais

Publicações científicas diretas

A Proqr Therapeutics publica pesquisas em periódicos revisados ​​por pares, como:

Nome do diário	Frequência de publicação	Fator de impacto
Biotecnologia da natureza	Mensal	41.4
Célula	Quinzenal	38.6
Ciência Medicina Translacional	Semanalmente	16.9

Apresentações da conferência médica

Detalhes anuais da participação na conferência:

Conferência	Localização	Contagem de apresentação
Sociedade Americana de Gene & Terapia celular	Baltimore, MD	4
Sociedade Europeia de Gene & Terapia celular	Barcelona, ​​Espanha	3

Relações com investidores da indústria de biotecnologia

Canais de comunicação de investidores:

• Chamadas de ganhos trimestrais
• Reuniões anuais de acionistas
• Apresentações de investidores
• Sec Comunicação de arquivamento

Plataformas de comunicação científica online

Plataformas de engajamento digital:

Plataforma	Seguidores/membros	Tipo de conteúdo
Pesquisa	12,500	Publicações de pesquisa
LinkedIn	8,700	Atualizações corporativas

Eventos de rede da indústria farmacêutica

Plataformas de rede -chave:

• Organização de Inovação de Biotecnologia (BIO) Convenção Internacional
• Conferência de Saúde JP Morgan
• Conferência de Parceria de Doenças Genéticas

Proqr Therapeutics N.V. (PRQR) - Modelo de negócios: segmentos de clientes

Pacientes de doenças genéticas raras

A Proqr Therapeutics se concentra em pacientes com distúrbios genéticos raros específicos. A partir de 2024, a empresa tem como alvo aproximadamente 2.000 a 5.000 pacientes globalmente para cada condição genética.

Transtorno genético	População estimada de pacientes	Tamanho do mercado -alvo
Fibrose cística (mutação F508del)	30.000-40.000 pacientes	América do Norte e Europa
Síndrome de Usher	4.000-6.000 pacientes	Mundialmente

Instituições de Pesquisa Genética

O ProQR colabora com 15 a 20 instituições de pesquisa líder globalmente, concentrando-se na pesquisa de doenças genéticas.

• Escola de Medicina de Harvard
• Departamento de Genética da Universidade de Stanford
• Universidade da Califórnia, São Francisco

Médicos especializados

O grupo de clientes-alvo inclui aproximadamente 500 a 700 geneticistas e profissionais médicos especializados em todo o mundo.

Especialidade	Número de praticantes	Foco geográfico
Especialistas genéticos	350-450	Estados Unidos
Médicos de doenças raras	150-250	Europa e América do Norte

Departamentos de pesquisa farmacêutica

O PROQR se envolve com 25-30 departamentos de pesquisa farmacêutica para possíveis colaborações e parcerias.

• Unidade de pesquisa de doenças raras da Pfizer
• Terapias genéticas da Novartis
• Diagnóstico molecular de Roche

Investidores de biotecnologia

A partir de 2024, a ProQR atrai investimentos de biotecnologia especializada e grupos de investidores focados em doenças raras.

Tipo de investidor	Número de investidores	Faixa de investimento total
Empresas de capital de risco	8-12	US $ 50-80 milhões
Investidores institucionais	15-20	US $ 100-150 milhões

Proqr Therapeutics N.V. (PRQR) - Modelo de negócios: estrutura de custos

Despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2022, a Proqr Therapeutics registrou despesas totais de P&D de US $ 52,4 milhões. A quebra dessas despesas é a seguinte:

Categoria de P&D	Valor da despesa ($)
Custos de pessoal	22,1 milhões
Custos de pesquisa externos	18,3 milhões
Materiais de laboratório	7,6 milhões
Licenciamento de tecnologia	4,4 milhões

Custos de gerenciamento de ensaios clínicos

As despesas de ensaios clínicos para 2022 totalizaram US $ 34,6 milhões, com a seguinte alocação:

• Fase I/II Despesas de teste: US $ 15,2 milhões
• Fase III Despesas de teste: US $ 19,4 milhões

Manutenção de propriedades patentes e intelectuais

A Proqr investiu US $ 3,2 milhões em proteção de propriedade intelectual e manutenção de patentes durante 2022.

Equipamentos de laboratório e investimentos em tecnologia

Os investimentos em tecnologia e equipamentos para 2022 foram de US $ 8,7 milhões, incluindo:

Categoria de investimento	Valor ($)
Tecnologia de edição de genes	4,3 milhões
Equipamento de sequenciamento avançado	2,9 milhões
Ferramentas de biologia computacional	1,5 milhão

Compensação de pessoal científico

A compensação total do pessoal científica para 2022 foi de US $ 28,5 milhões, estruturada da seguinte maneira:

• Salários base: US $ 18,2 milhões
• Compensação baseada em ações: US $ 6,3 milhões
• Bônus de desempenho: US $ 4 milhões

Estrutura de custo total para 2022: US $ 126,9 milhões

Proqr Therapeutics N.V. (PRQR) - Modelo de negócios: fluxos de receita

Potenciais acordos futuros de licenciamento de medicamentos

A partir do quarto trimestre 2023, a Proqr Therapeutics possui possíveis acordos de licenciamento no desenvolvimento, com foco específico em terapias de doenças genéticas raras.

Área terapêutica	Valor potencial de licenciamento	Status atual
Distúrbios genéticos raros	US $ 12,5 milhões em potencial pagamento inicial	Negociações em andamento
RNA Therapeutics	US $ 25 milhões em potenciais pagamentos marcantes	Discussões em estágio inicial

Bolsas de pesquisa e financiamento do governo

A Proqr garantiu financiamento de pesquisa de várias fontes.

Fonte de financiamento	Valor de concessão	Ano
Programa Europeu Horizon 2020	€ 3,6 milhões	2023
Conselho de Pesquisa Holandesa	€ 1,2 milhão	2023

Contratos de desenvolvimento farmacêutico colaborativo

As colaborações atuais de desenvolvimento farmacêutico incluem:

• Colaboração com a Vertex Pharmaceuticals
• Parceria com o Leiden University Medical Center
• Aliança de pesquisa com institutos de pesquisa de doenças genéticas

Vendas potenciais de produtos terapêuticos

Projeção de receita para possíveis produtos terapêuticos:

Produto terapêutico	Potencial estimado de receita anual	Mercado -alvo
Sepofarsen (distrofia herdada da retina)	US $ 15-20 milhões	Oftalmologia
Candidatos terapêuticos de RNA	US $ 10-15 milhões	Distúrbios genéticos raros

Licenciamento de propriedade intelectual

O portfólio de propriedade intelectual da ProQR gera possíveis fluxos de receita.

Categoria IP	Número de patentes	Potencial receita de licenciamento
Tecnologias de edição de RNA	12 patentes registradas	US $ 5-8 milhões em potencial licenciamento anual
Plataformas de terapia genética	8 patentes registradas	US $ 3-6 milhões em potencial licenciamento anual

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.