Rocket Pharmaceuticals, Inc. (RCKT): Modelo de negócios Canvas [Jan-2025 Atualizado]

No mundo da biotecnologia de ponta, a Rocket Pharmaceuticals, Inc. (RCKT) surge como um farol de esperança para famílias que lutam contra distúrbios genéticos pediátricos raros. Ao alavancar tecnologias avançadas de terapia genética e um modelo de negócios abrangente, esta empresa inovadora está transformando o cenário da medicina genética, oferecendo tratamentos potencialmente curativos que antes eram considerados impossíveis. Sua abordagem estratégica combina pesquisas científicas inovadoras, parcerias colaborativas e uma missão focada em laser para desenvolver terapias genéticas personalizadas que podem revolucionar como entendemos e tratamos condições genéticas complexas.

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negócios: Parcerias -chave

Instituições de pesquisa acadêmica

A Rocket Pharmaceuticals mantém parcerias estratégicas com as seguintes instituições de pesquisa acadêmica:

Instituição	Foco na pesquisa	Detalhes da parceria
Universidade da Califórnia, São Francisco	Distúrbios genéticos raros	Pesquisa colaborativa em andamento em Fanconi Anemia Gene Therapy
Escola de Medicina de Harvard	Distúrbios de leucócitos	Programa de pesquisa conjunta para deficiência de piruvato quinase

Biotecnologia estratégica e colaborações farmacêuticas

As principais colaborações estratégicas incluem:

• Andleyn Biosciences - Parceria de fabricação de vetores virais
• Spark Therapeutics - Gene Therapy Technology Exchange
• Regenxbio Inc. - Colaboração da plataforma de tecnologia NAV

Financiamento dos Institutos Nacionais de Saúde (NIH)

Detalhes de financiamento do NIH Research Grant:

Ano fiscal	Valor de concessão	Programa de Pesquisa
2023	US $ 4,2 milhões	Pesquisa rara de transtorno genético pediátrico
2024	US $ 4,5 milhões	Desenvolvimento avançado de terapia genética

Organizações de pesquisa contratada (CROs)

Parcerias CRO ativas:

• ICON PLC - Gerenciamento global de ensaios clínicos
• Medpace - Desenvolvimento de ensaios clínicos de doenças raras
• PRA Health Sciences - Fase I -III Coordenação do Trial

Centros médicos pediátricos

Rede de colaboração de ensaios clínicos:

Centro Médico	Localização	Ensaios clínicos ativos
Hospital Infantil da Filadélfia	Philadelphia, PA	3 ensaios raros de transtorno genético em andamento
Stanford Children's Health	Palo Alto, CA.	2 ensaios clínicos de terapia genética

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento de tratamentos de terapia genética

A partir do quarto trimestre de 2023, a Rocket Pharmaceuticals investiu US $ 87,4 milhões em despesas de P&D. A empresa se concentra em doenças genéticas pediátricas raras com a segmentação atual do pipeline:

• Deficiência de adesão de leucócitos-i (lad-i)
• Anemia de Fanconi
• Deficiência de piruvato quinase

Gerenciamento de ensaios pré -clínicos e clínicos

Fase de ensaios clínicos	Número de ensaios ativos	Investimento estimado
Pré -clínico	3	US $ 12,6 milhões
Fase I.	2	US $ 18,3 milhões
Fase II	4	US $ 35,7 milhões

Processos de conformidade regulatória e aprovação da FDA

Interações FDA em 2023: 17 reuniões formais, com 4 designações de terapia inovadora recebidas.

Desenvolvimento da Propriedade Intelectual

Portfólio de patentes:

• Total de patentes ativas: 42
• Aplicações de patentes pendentes: 13
• Despesas de arquivamento de patentes em 2023: US $ 4,2 milhões

Engenharia genética avançada e fabricação de vetores

Recursos de fabricação:

• Capacidade de produção de vetores internos: 500 lotes de vetores virais/ano
• Investimento de instalação de fabricação: US $ 65,3 milhões
• Equipe de controle de qualidade: 47 profissionais especializados

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negócios: Recursos -chave

Capacidades avançadas de pesquisa e desenvolvimento de terapia genética

Rocket Pharmaceuticals se concentra em doenças genéticas raras com recursos de pesquisa específicos:

Área de foco de pesquisa	Número de programas ativos
Deficiência de adesão de leucócitos-i (lad-i)	2 programas de estágio clínico
Anemia de Fanconi	3 programas de estágio clínico
Deficiência de piruvato quinase	1 Programa de estágio clínico

Tecnologias de engenharia genética proprietária

O portfólio de propriedade intelectual inclui:

• 12 patentes concedidas
• 18 pedidos de patente pendente
• Plataforma de vetor lentiviral proprietário

Talento científico e médico especializado

Categoria de pessoal	Número
Total de funcionários	137 em 31 de dezembro de 2023
Pesquisadores de doutorado	42
Pesquisadores de MD	8

Portfólio de propriedade intelectual

Paisagem de patentes:

• Total de Famílias de Patentes: 30
• Cobertura geográfica: Estados Unidos, Europa, Japão
• Faixa de expiração de patentes: 2030-2041

Infraestrutura de laboratório e pesquisa avançada

Instalação de pesquisa	Localização	Metragem quadrada
Sede corporativa	Cranbury, Nova Jersey	35.000 pés quadrados
Laboratório de Pesquisa	Nova Iorque	22.000 pés quadrados

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negócios: proposições de valor

Terapias genéticas inovadoras para distúrbios genéticos pediátricos raros

A Rocket Pharmaceuticals se concentra no desenvolvimento de terapias genéticas para distúrbios genéticos pediátricos raros com necessidades médicas não atendidas significativas.

Área de terapia	Transtorno alvo	Estágio de desenvolvimento
Deficiência de adesão de leucócitos (LAD)	Imunodeficiência genética rara	Ensaio Clínico de Fase 1/2
Anemia de Fanconi	Transtorno do sangue raro	Desenvolvimento clínico em andamento
Deficiência de piruvato quinase	Transtorno raro de glóbulos vermelhos	Ensaio Clínico de Fase 1/2

Tratamentos curativos em potencial para condições genéticas anteriormente intratáveis

O Rocket Pharmaceuticals tem como alvo as condições genéticas sem tratamentos eficazes atuais.

• As abordagens de terapia genética direcionam a mutações genéticas específicas
• Potencial modificação de doenças a longo prazo
• Abordar causas genéticas radiculares em vez de gerenciamento de sintomas

Abordagens terapêuticas genéticas personalizadas

Estratégia de medicina de precisão focada em intervenções genéticas específicas do paciente.

Plataforma de tecnologia	Abordagem terapêutica	Característica única
Vetor lentiviral	Correção de genes	Alta eficiência de transdução
Vetor AAV	Substituição de genes	Entrega genética direcionada

Custos de saúde reduzidos de longo prazo através de intervenções direcionadas

Potenciais benefícios econômicos por meio de abordagens terapêuticas de intervenção única.

• Redução estimada de custo de tratamento vitalício: US $ 1,5 a US $ 3 milhões por paciente
• Diminuição das hospitalizações
• Despesas de gerenciamento de doenças crônicas minimizadas

Qualidade de vida melhorada para pacientes com doenças genéticas raras

O desenvolvimento clínico focou em resultados terapêuticos transformadores.

Doença	Melhoria da qualidade de vida potencial	População de pacientes
Anemia de Fanconi	Risco reduzido do câncer	Aproximadamente 2.000 pacientes nos EUA
Deficiência de adesão de leucócitos	Função imunológica aprimorada	Menos de 500 pacientes em todo o mundo

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negócios: Relacionamentos do cliente

Engajamento direto com grupos de defesa do paciente

A partir de 2024, a Rocket Pharmaceuticals mantém parcerias ativas com as seguintes organizações de defesa de pacientes:

Organização	Área de foco	Tipo de colaboração
Organização Nacional para Distúrbios Raros (Nord)	Doenças genéticas raras	Suporte de pesquisa
Leucemia & Sociedade de Linfoma	Distúrbios do sangue raros	Consciência do ensaio clínico

Parcerias de pesquisa colaborativa

Colaborações atuais de pesquisa da Instituição Médica:

Instituição	Foco na pesquisa	Valor da parceria
Universidade de Stanford	Desenvolvimento da terapia genética	US $ 3,2 milhões
Universidade da Pensilvânia	Pesquisa de transtorno genético raro	US $ 2,7 milhões

Comunicação transparente sobre ensaios clínicos

• Atualizações de ensaios clínicos em tempo real compartilhados no site da empresa
• Relatórios trimestrais de progresso publicados
• Métricas de recrutamento de pacientes divulgou publicamente

Programas de apoio ao paciente e educação

Métricas do Programa de Apoio ao Paciente:

Componente do programa	2024 métricas
Webinars de educação do paciente	37 sessões realizadas
Helpline de apoio ao paciente	2.456 consultas únicas tratadas
Aplicações de assistência financeira	412 processado

Serviços de consulta médica e acompanhamento em andamento

Estatísticas do Serviço de Consulta Médica:

Tipo de serviço	2024 Volume
Sessões de aconselhamento genético	624 consultas
Acompanhamento de pacientes a longo prazo	287 pacientes em monitoramento em andamento

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negócios: canais

Apresentações de pesquisa médica direta

Em 2023, Rocket Pharmaceuticals apresentados em 7 principais conferências de pesquisa médica, incluindo:

Conferência	Data	Foco de apresentação
Sociedade Americana de Gene & Terapia celular	Maio de 2023	Programa de Distúrbios de Leucócitos
Sociedade Europeia de Gene & Terapia celular	Outubro de 2023	Doenças genéticas pediátricas raras

Participação da conferência científica

Métricas de participação na conferência para 2023:

• Total de conferências participadas: 12
• Apresentações orais: 5
• Apresentações de pôsteres: 7
• Resumos totais de pesquisa enviados: 15

Publicações de revistas médicas revisadas por pares

Estatísticas de publicação para 2023:

Categoria de diário	Número de publicações
Jornais de genética de alto impacto	4
Revistas de pesquisa de doenças raras	3
Total de publicações revisadas por pares	7

Colaboração com especialistas médicos pediátricos

Rede de colaboração em 2023:

• Centros totais de pesquisa pediátrica envolvidos: 22
• Colaborações de ensaios clínicos ativos: 8
• Alcance geográfico: 15 estados nos Estados Unidos

Plataformas de saúde digital e redes de comunicação de pesquisa

Métricas de engajamento digital para 2023:

Plataforma	Métrica de engajamento
Rede de pesquisa do LinkedIn	3.750 conexões profissionais
Pesquisa Profile	2.100 seguidores
Seção de pesquisa do site da empresa	45.000 visitantes únicos

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negócios: segmentos de clientes

Pacientes pediátricos com distúrbios genéticos raros

O Rocket Pharmaceuticals se concentra em aproximadamente 5-7 distúrbios genéticos raros que afetam as populações pediátricas. A população-alvo da empresa é estimada em 3.000 a 5.000 indivíduos nos Estados Unidos.

Categoria de distúrbio	População estimada de pacientes	Potencial de tratamento anual
Deficiência de adesão de leucócitos	500-750 pacientes	US $ 250.000 a US $ 500.000 por paciente
Anemia de Fanconi	1.500-2.000 pacientes	US $ 350.000 a US $ 650.000 por paciente

Instituições de Pesquisa Genética

A Rocket Pharmaceuticals colabora com 12 a 15 instituições de pesquisa de primeira linha em todo o mundo.

• Parcerias de pesquisa do National Institutes of Health (NIH)
• Redes de colaboração do Hospital Infantil
• Centros de Pesquisa Médica Acadêmica

Centros de tratamento médico especializados

A empresa tem como alvo aproximadamente 25 a 30 centros de tratamento genético especializados na América do Norte e na Europa.

Região geográfica	Número de centros de tratamento	Engajamento anual
Estados Unidos	18-22 centros	Investimento colaborativo anual de US $ 5-7 milhões
União Europeia	7-8 centros	Investimento colaborativo anual de US $ 3-4 milhões

Grupos de defesa de pacientes com doenças raras

A Rocket Pharmaceuticals se envolve ativamente com 8 a 10 grandes organizações de defesa de doenças raras.

• Fundo de Pesquisa de Anemia Fanconi
• Associação primária de imunodeficiência
• Aliança de Transtorno Genético Raro Global

Provedores de saúde especializados em condições genéticas

A empresa interfina com aproximadamente 150-200 prestadores de serviços de saúde genéticos especializados em todo o país.

Especialidade do provedor	Número de provedores	Engajamento clínico anual
Hematologistas pediátricos	75-90 provedores	Suporte de pesquisa clínica de US $ 2-3 milhões
Especialistas em Transtorno Genético	60-75 provedores	US $ 1,5-2,5 milhão de colaboração de pesquisa

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negócios: estrutura de custos

Extensas despesas de pesquisa e desenvolvimento

A partir do quarto trimestre de 2023, a Rocket Pharmaceuticals registrou despesas de P&D de US $ 70,3 milhões no ano fiscal. A repartição total do investimento em P&D da empresa inclui:

Categoria de P&D	Quantidade de despesa
Programas de terapia genética	US $ 42,5 milhões
Pesquisa de doenças pediátricas raras	US $ 18,7 milhões
Desenvolvimento pré -clínico	US $ 9,1 milhões

Gerenciamento e execução de ensaios clínicos

As despesas de ensaios clínicos para 2023 totalizaram aproximadamente US $ 45,2 milhões, com a seguinte alocação:

• Ensaios de terapia de genes de leucócitos: US $ 22,6 milhões
• Programa de Anemia Fanconi: US $ 15,3 milhões
• Ensaios de doenças metabólicas: US $ 7,3 milhões

Processos de conformidade e aprovação regulatórios

Os custos de conformidade regulatórios para 2023 foram estimados em US $ 12,5 milhões, incluindo:

Área de conformidade	Custo
Preparação de envio da FDA	US $ 5,8 milhões
Documentação regulatória	US $ 4,2 milhões
Monitoramento de conformidade	US $ 2,5 milhões

Proteção à propriedade intelectual

As despesas de propriedade intelectual de 2023 totalizaram US $ 6,8 milhões, distribuídas da seguinte forma:

• Arquivamento e manutenção de patentes: US $ 4,3 milhões
• Serviços de proteção legal: US $ 2,5 milhões

Recrutamento e retenção de talentos científicos avançados

Os custos de aquisição e retenção de talentos para 2023 foram de US $ 18,6 milhões:

Categoria de gerenciamento de talentos	Despesa
Salários competitivos	US $ 12,4 milhões
Recrutamento de cientistas de pesquisa	US $ 3,9 milhões
Benefícios e treinamento dos funcionários	US $ 2,3 milhões

Estrutura de custo total para 2023: US $ 153,4 milhões

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negócios: fluxos de receita

Potenciais acordos futuros de licenciamento de produtos

A partir do quarto trimestre 2023, a Rocket Pharmaceuticals possui possíveis acordos de licenciamento em seu portfólio de terapia de genes de doenças raras. O valor potencial estimado desses acordos não é divulgado publicamente.

Bolsas de pesquisa e financiamento do governo

Fonte de financiamento	Quantia	Ano
Graças do National Institutes of Health (NIH)	US $ 3,2 milhões	2023
Financiamento do Departamento de Pesquisa de Defesa	US $ 1,5 milhão	2023

Parcerias de pesquisa colaborativa

As parcerias de pesquisa atuais incluem:

• Hospital Infantil de Boston
• Universidade de Minnesota
• Institutos Nacionais de Saúde

Vendas potenciais de produtos terapêuticos

A receita da Rocket Pharmaceuticals de possíveis vendas de produtos terapêuticos depende das aprovações da FDA. O pipeline atual inclui:

Terapia	Indicação	Valor potencial de mercado
RP-L201	Anemia de Fanconi	Estimação de US $ 150-200 milhões no mercado anual
RP-A501	Doença de Danon	Mercado anual estimado de US $ 100-150 milhões

Oportunidades de licenciamento de propriedade intelectual

A partir de 2023, o Rocket Pharmaceuticals possui 17 patentes emitidas e 32 pedidos de patente pendente em suas plataformas de terapia genética.

Despesas totais de pesquisa e desenvolvimento para 2023: US $ 94,6 milhões

Perda líquida para 2023: US $ 108,4 milhões

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Value Propositions

Potential one-time curative genetic therapies for devastating rare diseases.

Rocket Pharmaceuticals, Inc. is advancing therapies targeting conditions with high unmet medical need, evidenced by its late-stage pipeline focus.

Addressing ultra-rare, life-threatening disorders with high unmet medical need.

• Severe Leukocyte Adhesion Deficiency-I (LAD-I) is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant.
• PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) affects approximately 50,000 adults and children in the U.S. and Europe.
• The cardiovascular programs collectively target over 100,000 patients across the U.S. and EU.

KRESLADI™ (marnetegragene autotemcel; marne-cel) for severe LAD-I offers a potential alternative to bone marrow transplant.

Clinical data from the global Phase 1/2 study for KRESLADI™ demonstrated 100% overall survival at 12 months post-infusion for all enrolled patients. The Prescription Drug User Fee Act (PDUFA) target action date for the Biologics License Application (BLA) resubmission is set for March 28, 2026. Approval would make Rocket Pharmaceuticals eligible for a Rare Pediatric Disease Priority Review Voucher (PRV). The therapy was well tolerated with no treatment-related serious adverse events.

Focused AAV platform for cardiovascular diseases (Danon, PKP2-ACM, BAG3-DCM).

The company has sharpened its strategic focus on its adeno-associated virus (AAV) cardiovascular gene therapy portfolio. The cash, cash equivalents, and investments as of September 30, 2025, totaled $222.8 million, expected to fund operations into the second quarter of 2027. This focus was supported by a strategic reorganization that included a workforce reduction of approximately 30%, expected to lower 12-month operating expenses by around 25%.

Program (Indication)	Platform/Vector	Latest Status (Late 2025)	Key Efficacy/Status Metric
KRESLADI™ (Severe LAD-I)	Lentiviral (LV)	BLA Resubmission Accepted	100% overall survival at 12 months post-infusion.
RP-A501 (Danon Disease)	AAV	Clinical Hold lifted in August 2025	Dosing for Phase 2 pivotal trial to resume in 1H 2026.
RP-A601 (PKP2-ACM)	AAV	Received RMAT Designation	All 3 adult patients showed increased PKP2 protein expression.
RP-A701 (BAG3-DCM)	AAV	IND Clearance Received	Preparing for first-in-human evaluation.

Regenerative Medicine Advanced Therapy (RMAT) designation for RP-A601.

The U.S. Food and Drug Administration (FDA) granted RMAT designation to RP-A601 for PKP2-ACM. This designation was supported by Phase 1 trial data where all three adult patients treated with a single dose showed improved heart structure and function. Specific data points included:

• Increased PKP2 protein expression, with 110% & 398% increase in 2 patients with low baseline levels.
• 34-41 point increases in KCCQ-12 scores.
• NYHA class improvement from II to I.

This designation enables more intensive FDA guidance and an expedited review process for the therapy, which marks Rocket Pharmaceuticals, Inc.'s fifth RMAT designation.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Customer Relationships

You're building a commercial foundation for gene therapies, which means the relationship with the treating centers and the medical community is everything. For Rocket Pharmaceuticals, Inc., this is a high-touch, highly specialized engagement model, especially given the complexity of administering these one-time treatments.

High-touch, direct engagement with specialized treatment centers and physicians.

The focus is on centers capable of handling complex gene therapy administration. This requires deep scientific exchange, not just sales calls. For instance, the PKP2-ACM program, targeting about 50,000 people in the U.S. and Europe, necessitates working with specialized cardiology centers experienced in inherited heart disease. The collective cardiovascular portfolio targets over 100,000 patients across the U.S. and EU. Engagement is driven by clinical milestones; for example, the FDA lifting the clinical hold on the RP-A501 Danon disease study in August 2025 immediately intensified coordination with those treating centers to resume dosing of additional patients in the first half of 2026. The specific dosing protocol for the next three patients involves a recalibrated dose of 3.8 × 10¹³ GC/kg with a modified immunomodulatory regimen, which requires precise physician training and oversight.

Close collaboration with patient advocacy groups for rare disease communities.

Rocket Pharmaceuticals, Inc. views patients and families as its North Star, which translates directly into deep ties with advocacy groups. A key example is the company's longstanding collaboration with the Danon Foundation, where their commitment to research, advocacy, and patient support is integral to advancing the field. This partnership structure helps Rocket understand the patient journey, which is crucial when dealing with devastating rare disorders.

• Patients and families are the guiding force behind the mission.
• The company cultivates trusted partnerships with advocacy organizations.

Direct communication with regulatory agencies (FDA, EMA) for complex submissions.

Regulatory dialogue is constant and critical, especially given the late-stage nature of several assets. The relationship with the FDA has been highly active through 2025. You saw the FDA accept the Biologics License Application (BLA) resubmission for KRESLADI™ in October 2025, which has a Prescription Drug User Fee Act (PDUFA) date set for March 28, 2026. Furthermore, the company secured a Regenerative Medicine Advanced Therapy (RMAT) Designation from the FDA for RP-A601 (PKP2-ACM) in July 2025, following positive Phase 1 data. The ability to align with the FDA quickly to lift the clinical hold on the RP-A501 Danon disease trial in under three months in August 2025 demonstrates a working, if intense, relationship with health authorities.

Dedicated medical affairs and commercial teams for patient support and access.

As Rocket Pharmaceuticals, Inc. moves toward potential commercialization, the structure of its customer-facing teams has been refined. Sarbani Chaudhuri joined as Chief Commercial & Medical Affairs Officer in April 2025, bringing experience from Johnson & Johnson, AstraZeneca, and Pfizer, specifically in rare cardiac and hematology diseases. However, the company made a strategic pivot in mid-2025, reducing its workforce by approximately 30% to align with pipeline prioritization, which impacts the scale of these dedicated teams. This cost-saving measure is reflected in the financial reporting.

Metric/Expense Category	Q3 2025 Amount (USD)	Comparison Period	Change
General and Administrative (G&A) Expenses	$18.4 million	Q3 2024	Decrease of $8.7 million
Commercial Preparation-Related Expenses (Included in G&A)	Not specified	Q3 2024	Decrease of $6.6 million
Total Employees	299 (As of late 2025)	N/A	Workforce reduced by approx. 30% in 2025

The decrease of $6.6 million in commercial preparation-related expenses within G&A expenses for the third quarter of 2025, compared to the third quarter of 2024, shows the immediate financial impact of the strategic realignment away from broader commercial readiness for paused programs.

Personalized patient support programs for complex gene therapy administration.

For the Danon disease program, the Phase 2 pivotal trial is designed around a small, highly monitored group. The trial evaluates efficacy and safety in 12 patients, which includes a pediatric run-in cohort of n=2. This small number underscores the personalized, intensive support required for each patient receiving an investigational gene therapy like RP-A501. The company's overall cash position as of September 30, 2025, was $222.8 million, which is being managed to fund operations into the second quarter of 2027, ensuring resources are available for these critical, high-touch patient interactions.

Finance: draft 13-week cash view by Friday.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Channels

You're looking at the distribution and communication channels for Rocket Pharmaceuticals, Inc. (RCKT) as they transition from late-stage development to potential commercialization for their rare disease gene therapies in late 2025. Given the nature of these treatments, the channels are highly specialized, focusing on centers of excellence and regulatory milestones.

Highly specialized gene therapy treatment centers (ATCs) for administration

For gene therapies targeting ultra-rare disorders like severe Leukocyte Adhesion Deficiency-I (LAD-I) with KRESLADI™, the channel is inherently limited to specialized centers capable of administering complex cell and gene therapies. While a specific number of Authorized Treatment Centers (ATCs) isn't publicly quantified as of late 2025, the strategy is clearly focused on high-acuity sites. The market opportunity for their cardiovascular pipeline alone-Danon disease, PKP2-ACM, and BAG3-DCM-represents over 100,000 patients in the U.S. and EU, suggesting a targeted network of transplant or specialized cardiology centers will be necessary for future launch. The company's internal focus on AAV cardiovascular programs reinforces this need for specialized infusion sites.

Direct sales and medical affairs teams targeting key opinion leaders (KOLs)

Rocket Pharmaceuticals, Inc. is still pre-revenue as of the third quarter ending September 30, 2025, reporting $0.0 million in revenue. Therefore, the team build-out is focused on medical affairs and commercial preparation rather than broad sales execution. The company made key leadership additions in 2025 to bolster this, including appointing Sarbani Chaudhuri as Chief Commercial & Medical Affairs Officer in April 2025. Commercial preparation-related expenses were noted at $1.5 million for the three months ended March 31, 2025. The channel strategy involves engaging Key Opinion Leaders (KOLs) within the specific rare disease communities, such as the estimated 50,000 PKP2-ACM affected individuals in the U.S. and Europe.

Regulatory submission pathways (BLA/MAA) to the FDA and European Medicines Agency

Regulatory milestones are the primary channel driver for a pre-commercial company. Rocket Pharmaceuticals, Inc. has seen significant shifts in this area in late 2025, prioritizing programs with clearer pathways.

Program/Therapy	Regulatory Action/Status (as of late 2025)	Agency
KRESLADI™ (LAD-I)	FDA acceptance of BLA resubmission in October 2025. Complete BLA submission anticipated before the end of 2025.	FDA
KRESLADI™ (LAD-I)	PDUFA date anticipated in March 2026.	FDA
RP-L102 (Fanconi Anemia)	Voluntarily withdrew U.S. BLA on October 3, 2025, based on strategic considerations.	FDA
RP-L102 (Fanconi Anemia)	Withdrew Marketing Authorization Application (MAA) in July 2025.	European Medicines Agency (EMA)

The company is focusing resources on its AAV cardiovascular programs, which have clear biomarker opportunities for accelerated approval.

Clinical trial sites for patient identification and initial treatment access

Clinical trial sites serve as the initial, most critical access points for patients, especially for therapies addressing conditions like Danon disease, which affects an estimated 15,000 to 30,000 people in the US and Europe. The channel is defined by the active studies:

• Phase 2 pivotal study for RP-A501 (Danon disease) is ongoing following the lifting of a clinical hold.
• Phase 1 trial start-up activities are underway for RP-A701 (BAG3-DCM).
• Initial data from the Phase 1 study of RP-A601 (PKP2-ACM) was expected in May 2025.

The company has experienced a 30% workforce reduction to align resources with these late-stage cardiovascular programs.

Scientific publications and conferences to disseminate clinical data

Dissemination of clinical data through peer-reviewed channels and scientific presentations is a key channel for establishing credibility and informing the medical community. Rocket Pharmaceuticals, Inc. actively participated in major industry events in 2025 to present its pipeline progress.

• Presented updates at the Morgan Stanley 23rd Annual Global Healthcare Conference in September 2025.
• Presented at the 8th Annual Evercore Healthcare Conference in December 2025.
• Corporate Presentation and specific data updates (RP-A601, RP-A501) were shared in November 2025.

The company's experience from treating over 15 cardiac patients informs its late-stage AAV programs.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Customer Segments

You're looking at the patient populations Rocket Pharmaceuticals, Inc. (RCKT) is targeting with its gene therapy pipeline as of late 2025. The focus is clearly on ultra-rare, genetically defined diseases, especially in the cardiovascular space, which dictates resource allocation.

The core customer segments are defined by specific, devastating genetic disorders requiring one-time, potentially curative treatments. The company has strategically prioritized its AAV cardiovascular platform, which includes three major cardiomyopathy programs, alongside its LV hematology program for severe LAD-I.

Here's a breakdown of the addressable patient populations based on the latest internal estimates and prevalence data:

Disease Indication	Therapy Program	Estimated Prevalence (U.S. and Europe)	Market Significance
PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM)	RP-A601 (AAV)	Approximately 50,000 people	Largest market opportunity in disclosed pipeline programs
Danon disease	RP-A501 (AAV)	15,000 to 30,000 patients	Late-stage clinical program
PKP2-ACM and BAG3-DCM (Combined with Danon Disease)	RP-A601 (AAV) and RP-A701 (AAV)	More than 100,000 patients	Collectively target major genetically defined cardiomyopathies
BAG3-associated dilated cardiomyopathy (BAG3-DCM)	RP-A701 (AAV)	Included in the >100,000 total for the three cardiomyopathies	Preclinical/IND accepted status

The other critical segment involves patients with severe LAD-I, for whom KRESLADI™ is seeking approval. This is a severe pediatric genetic disorder causing life-threatening infections.

• Pediatric and adult patients with severe Leukocyte Adhesion Deficiency-I (LAD-I) targeting a March 28, 2026 PDUFA date for KRESLADI™.
• Ultra-rare disease populations, definitely defined by specific genetic mutations, as the entire portfolio is focused on monogenic disorders.

The financial reality of supporting these segments as of the third quarter of 2025 shows significant investment in R&D:

• Q3 2025 Net Loss was $50.3 million.
• Total Operating Expense for Q3 2025 was $52.2 million.
• Research and Development Expenses for Q3 2025 accounted for $34.1 million.
• Cash, cash equivalents, and investments stood at $75.9 million at the end of Q3 2025.
• The company expects current resources to fund operations into the second quarter of 2027.

The cash position at the end of Q1 2025 was $318.2 million.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Cost Structure

You're looking at the cost side of the ledger for Rocket Pharmaceuticals, Inc. (RCKT) as of late 2025. For a late-stage biotech focused on gene therapy, the cost structure is heavily weighted toward the science and the path to the clinic and market. It's all about burning cash to create future value, so every dollar spent on R&D and trials is a strategic decision.

Here's a quick look at the headline operating expenses from the third quarter ending September 30, 2025:

Expense Category	Q3 2025 Amount
Research and Development (R&D) Expenses	$34.1 million
General and Administrative (G&A) Expenses	$18.4 million
Total Operating Expenses (R&D + G&A)	$52.5 million

The primary cost driver, as you'd expect, is Research and Development. For the three months ended September 30, 2025, Rocket Pharmaceuticals reported R&D expenses of $34.1 million. This figure reflects a disciplined approach to resource allocation following the company's organizational realignment earlier in the year.

General and Administrative costs also factor in significantly, though they saw a reduction year-over-year. G&A expenses totaled $18.4 million for the third quarter of 2025, down from $27.1 million in the same period of 2024. This decrease was largely due to lower commercial preparation-related expenses, which dropped by $6.6 million, plus a $1.5 million reduction in non-cash stock-based compensation expense.

The high cost of in-house cGMP manufacturing and quality control is an inherent part of Rocket Pharmaceuticals' structure, especially given their Cranbury, N.J. R&D and manufacturing facility. While specific standalone costs aren't broken out, the R&D expense line reflects these activities. For instance, the R&D spend in Q3 2025 included decreases in manufacturing and development and direct material costs of $3.6 million.

Clinical trial costs for late-stage pivotal studies represent a major, lumpy expense. Rocket Pharmaceuticals is heavily invested in advancing its AAV cardiovascular gene therapy platform, which includes:

• Phase 2 pivotal study for Danon disease (RP-A501).
• Phase 1 study for PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) (RP-A601).

The focus on these programs means significant outlay for patient enrollment, monitoring, and data analysis. The Q3 2025 R&D reduction was partly due to a $2.4 million decrease in clinical trial expenses compared to the prior year, showing the impact of cost optimization efforts.

Finally, there are one-time charges related to strategic shifts. Rocket Pharmaceuticals incurred restructuring and severance charges of approximately $3.3 million in 2025 as part of its reorganization plan to narrow pipeline focus and extend its operational runway into the second quarter of 2027. That's a short-term hit to manage the long-term burn rate.

Finance: draft 13-week cash view by Friday.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Revenue Streams

You're looking at the revenue picture for Rocket Pharmaceuticals, Inc. as of late 2025. Honestly, the current picture is what you'd expect for a company deep in late-stage development, but the near-term catalysts are clear.

Current product revenue is $0.0 million for Q3 2025 (pre-commercial stage). This reflects the reality that Rocket Pharmaceuticals does not yet have commercialized products generating sales, which is consistent with their reported net loss of $50.3 million for that same quarter ending September 30, 2025.

The first significant non-dilutive revenue opportunity is the potential sale of a Priority Review Voucher (PRV) upon KRESLADI™ approval. Analysts estimate this PRV could be valued at over $150 million, which would directly support the company's cash position, currently reported at $222.8 million as of September 30, 2025.

Future product sales of KRESLADI™ (marnetegragene autotemcel; marne-cel) for severe Leukocyte Adhesion Deficiency-I (LAD-I) are tied to a key regulatory date. The Prescription Drug User Fee Act (PDUFA) target action date set by the FDA is March 28, 2026. The data supporting this Biologics License Application (BLA) showed compelling results in the global Phase 1/2 study:

Metric	Result
Overall Survival at 12 Months Post-Infusion	100%
Primary and Secondary Endpoints Met	Yes
Treatment-Related Serious Adverse Events	0

The company has strategically narrowed its focus to maximize the value of its AAV cardiovascular gene therapy portfolio, which represents the next major revenue driver post-KRESLADI™. This focus was reinforced by a workforce reduction of approximately 30% to extend the cash runway into the second quarter of 2027, excluding any PRV proceeds.

This AAV portfolio is centered on rare cardiomyopathies, with specific programs providing future sales potential:

• RP-A501 for Danon Disease (late-stage pivotal Phase 2 trial resuming in 1H 2026).
• RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM) (advancing toward a pivotal Phase 2 study).
• RP-A701 for BAG3-associated dilated cardiomyopathy (DCM) (preparing for first-in-human evaluation).

The market potential for these assets is substantial; for instance, the Danon disease treatment market is projected to grow at a 6.5% CAGR through 2030. The company is betting on this concentrated pipeline for long-term value creation.

Finally, potential upfront or milestone payments from out-licensing non-core assets remain a possible, though secondary, revenue stream. Given the recent strategic reorganization and the decision to focus exclusively on the AAV cardiovascular platform, any non-core asset divestiture would be a deliberate action to further conserve cash and fund the prioritized pipeline.