Rocket Pharmaceuticals, Inc. (RCKT): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el mundo de la biotecnología de vanguardia, Rocket Pharmaceuticals, Inc. (RCKT) emerge como un faro de esperanza para familias que luchan contra trastornos genéticos pediátricos raros. Al aprovechar las tecnologías avanzadas de terapia génica y un modelo de negocio integral, esta empresa innovadora está transformando el panorama de la medicina genética, ofreciendo tratamientos potencialmente curativos que alguna vez se consideraron imposibles. Su enfoque estratégico combina investigación científica innovadora, asociaciones colaborativas y una misión centrada en el láser para desarrollar terapias genéticas personalizadas que puedan revolucionar cómo entendemos y tratamos afecciones genéticas complejas.

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negocios: asociaciones clave

Instituciones de investigación académica

Rocket Pharmaceuticals mantiene asociaciones estratégicas con las siguientes instituciones de investigación académica:

Institución	Enfoque de investigación	Detalles de la asociación
Universidad de California, San Francisco	Trastornos genéticos raros	Investigación colaborativa continua en terapia génica de anemia de Fanconi
Escuela de Medicina de Harvard	Trastornos de leucocitos	Programa de investigación conjunta para la deficiencia de piruvato quinasa

Biotecnología estratégica y colaboraciones farmacéuticas

Las colaboraciones estratégicas clave incluyen:

• Andelyn Biosciences - Asociación de fabricación de vectores virales
• Spark Therapeutics - Intercambio de tecnología de terapia génica
• Regenxbio Inc. - Collaboración de la plataforma de tecnología NAV

Financiación de los Institutos Nacionales de Salud (NIH)

Detalles de financiación de la subvención de investigación de NIH:

Año fiscal	Monto de subvención	Programa de investigación
2023	$ 4.2 millones	Investigación de trastorno genético pediátrico raro
2024	$ 4.5 millones	Desarrollo avanzado de terapia génica

Organizaciones de investigación por contrato (CRO)

Asociaciones activas de CRO:

• ICON PLC - Gestión global de ensayos clínicos
• Medpace - Desarrollo de ensayos clínicos de enfermedades raras
• Pra Health Sciences - Fase I -III Coordinación del ensayo

Centros médicos pediátricos

Red de colaboración de ensayos clínicos:

Centro médico	Ubicación	Ensayos clínicos activos
Hospital de Niños de Filadelfia	Filadelfia, PA	3 ensayos de trastorno genético raro en curso
Stanford Health para niños	Palo Alto, CA	2 ensayos clínicos de terapia génica

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negocio: actividades clave

Investigación y desarrollo de tratamientos de terapia génica

A partir del cuarto trimestre de 2023, Rocket Pharmaceuticals invirtió $ 87.4 millones en gastos de I + D. La compañía se centra en enfermedades genéticas pediátricas raras con la orientación de la tubería actual:

• Deficiencia de adhesión de leucocitos-I (LAD-I)
• Anemia fanconi
• Deficiencia de piruvato quinasa

Gestión de ensayos preclínicos y clínicos

Fase de ensayo clínico	Número de pruebas activas	Inversión estimada
Preclínico	3	$ 12.6 millones
Fase I	2	$ 18.3 millones
Fase II	4	$ 35.7 millones

Procesos de cumplimiento regulatorio y de aprobación de la FDA

Interacciones de la FDA en 2023: 17 reuniones formales, con 4 designaciones de terapia de avance recibidas.

Desarrollo de la propiedad intelectual

Cartera de patentes:

• Patentes activas totales: 42
• Aplicaciones de patentes pendientes: 13
• Gastos de presentación de patentes en 2023: $ 4.2 millones

Ingeniería genética avanzada y fabricación de vectores

Capacidades de fabricación:

• Capacidad de producción de vectores internos: 500 lotes de vectores virales/año
• Inversión de instalaciones de fabricación: $ 65.3 millones
• Personal de control de calidad: 47 profesionales especializados

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negocio: recursos clave

Capacidades avanzadas de investigación y desarrollo de terapia génica

Rocket Pharmaceuticals se centra en enfermedades genéticas raras con capacidades de investigación específicas:

Área de enfoque de investigación	Número de programas activos
Deficiencia de adhesión de leucocitos-I (LAD-I)	2 programas de etapa clínica
Anemia fanconi	3 programas de etapa clínica
Deficiencia de piruvato quinasa	1 programa de etapa clínica

Tecnologías de ingeniería genética patentada

La cartera de propiedades intelectuales incluye:

• 12 patentes otorgadas
• 18 Pensas de patentes pendientes
• Plataforma de vector lentiviral patentado

Talento científico y médico especializado

Categoría de personal	Número
Total de empleados	137 al 31 de diciembre de 2023
Investigadores de doctorado	42
Investigadores de MD	8

Cartera de propiedades intelectuales

Paisaje de patentes:

• Familias de patentes totales: 30
• Cobertura geográfica: Estados Unidos, Europa, Japón
• Rango de vencimiento de patentes: 2030-2041

Infraestructura avanzada de laboratorio e investigación

Instalación de investigación	Ubicación	Pies cuadrados
Sede corporativa	Cranbury, Nueva Jersey	35,000 pies cuadrados
Laboratorio de investigación	Nueva York	22,000 pies cuadrados

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negocio: propuestas de valor

Terapias genéticas innovadoras para trastornos genéticos pediátricos raros

Rocket Pharmaceuticals se centra en el desarrollo de terapias genéticas para trastornos genéticos pediátricos raros con importantes necesidades médicas no satisfechas.

Área de terapia	Trastorno objetivo	Etapa de desarrollo
Deficiencia de adhesión de leucocitos (LAD)	Inmunodeficiencia genética rara	Ensayo clínico de fase 1/2
Anemia fanconi	Trastorno sanguíneo raro	Desarrollo clínico en curso
Deficiencia de piruvato quinasa	Trastorno de glóbulos rojos raros	Ensayo clínico de fase 1/2

Tratamientos curativos potenciales para afecciones genéticas previamente no tratables

Los productos farmacéuticos de cohetes se dirigen a condiciones genéticas sin tratamientos efectivos actuales.

• Enfoques de terapia génica dirigida a mutaciones genéticas específicas
• Modificación potencial de la enfermedad a largo plazo
• Abordar las causas genéticas de la raíz en lugar del manejo de los síntomas

Enfoques terapéuticos genéticos personalizados

Estrategia de medicina de precisión centrada en intervenciones genéticas específicas del paciente.

Plataforma tecnológica	Enfoque terapéutico	Característica única
Vector lentiviral	Corrección de genes	Alta eficiencia de transducción
AAV Vector	Reemplazo de genes	Entrega genética dirigida

Costos de atención médica a largo plazo reducidos a través de intervenciones específicas

Beneficios económicos potenciales a través de enfoques terapéuticos de una sola intervención.

• Reducción estimada del costo de tratamiento de por vida: $ 1.5- $ 3 millones por paciente
• Disminución de hospitalizaciones
• Gastos minimizados de manejo de enfermedades crónicas

Mejor calidad de vida para pacientes con enfermedades genéticas raras

El desarrollo clínico se centró en los resultados terapéuticos transformadores.

Enfermedad	Mejora potencial de calidad de vida	Población de pacientes
Anemia fanconi	Reducción de riesgo de cáncer	Aproximadamente 2,000 pacientes en EE. UU.
Deficiencia de adhesión de leucocitos	Función inmune mejorada	Menos de 500 pacientes en todo el mundo

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negocios: relaciones con los clientes

Compromiso directo con grupos de defensa del paciente

A partir de 2024, Rocket Pharmaceuticals mantiene asociaciones activas con las siguientes organizaciones de defensa del paciente:

Organización	Área de enfoque	Tipo de colaboración
Organización Nacional para Trastornos Raros (NORD)	Enfermedades genéticas raras	Soporte de investigación
Leucemia & Sociedad de linfoma	Trastornos de sangre raros	Conciencia del ensayo clínico

Asociaciones de investigación colaborativa

Colaboraciones de investigación de instituciones médicas actuales:

Institución	Enfoque de investigación	Valor de asociación
Universidad de Stanford	Desarrollo de terapia génica	$ 3.2 millones
Universidad de Pensilvania	Investigación de trastorno genético raro	$ 2.7 millones

Comunicación transparente sobre ensayos clínicos

• Actualizaciones de ensayos clínicos en tiempo real compartidas en el sitio web de la compañía
• Informes de progreso trimestral publicados
• Métricas de reclutamiento de pacientes reveladas públicamente

Programas de apoyo y educación del paciente

Métricas del programa de apoyo al paciente:

Componente del programa	2024 métricas
Seminarios web de educación del paciente	37 sesiones realizadas
Línea de ayuda de apoyo al paciente	2,456 consultas únicas manejadas
Solicitudes de asistencia financiera	412 procesado

Servicios continuos de consulta médica y seguimiento

Estadísticas de servicio de consulta médica:

Tipo de servicio	Volumen 2024
Sesiones de asesoramiento genético	624 consultas
Seguimiento de paciente a largo plazo	287 pacientes en monitoreo continuo

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negocio: canales

Presentaciones directas de investigación médica

En 2023, Rocket Pharmaceuticals presentó en 7 principales conferencias de investigación médica, que incluyen:

Conferencia	Fecha	Enfoque de presentación
Sociedad Americana de Gene & Terapia celular	Mayo de 2023	Programa de trastornos de leucocitos
Sociedad Europea de Gene & Terapia celular	Octubre de 2023	Enfermedades genéticas pediátricas raras

Participación de la conferencia científica

Métricas de participación de la conferencia para 2023:

• Conferencias totales a la que asistieron: 12
• Presentaciones orales: 5
• Presentaciones de carteles: 7
• Resúmenes totales de investigación presentados: 15

Publicaciones de revistas médicas revisadas por pares

Estadísticas de publicación para 2023:

Categoría de revista	Número de publicaciones
Revistas de genética de alto impacto	4
Revistas de investigación de enfermedades raras	3
Publicaciones totales revisadas por pares	7

Colaboración con especialistas en médicos pediátricos

Red de colaboración en 2023:

• Centros de investigación pediátricos totales comprometidos: 22
• Colaboraciones de ensayos clínicos activos: 8
• Alcance geográfico: 15 estados en los Estados Unidos

Plataformas de salud digital y redes de comunicación de investigación

Métricas de compromiso digital para 2023:

Plataforma	Métrico de compromiso
Red de investigación de LinkedIn	3.750 conexiones profesionales
Investigador Profile	2,100 seguidores
Sección de investigación del sitio web de la empresa	45,000 visitantes únicos

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

Pacientes pediátricos con trastornos genéticos raros

Rocket Pharmaceuticals se centra en aproximadamente 5-7 trastornos genéticos raros que afectan a las poblaciones pediátricas. La población de pacientes objetivo de la compañía se estima en 3.000-5,000 personas en los Estados Unidos.

Categoría de desorden	Población de pacientes estimada	Potencial de tratamiento anual
Deficiencia de adhesión de leucocitos	500-750 pacientes	$ 250,000- $ 500,000 por paciente
Anemia fanconi	1,500-2,000 pacientes	$ 350,000- $ 650,000 por paciente

Instituciones de investigación genética

Rocket Pharmaceuticals colabora con 12-15 instituciones de investigación de primer nivel a nivel mundial.

• Asociaciones de investigación de los Institutos Nacionales de Salud (NIH)
• Redes de colaboración del Hospital de Niños
• Centros de investigación médica académica

Centros de tratamiento médico especializados

La compañía se dirige a aproximadamente 25-30 centros de tratamiento genético especializados en América del Norte y Europa.

Región geográfica	Número de centros de tratamiento	Compromiso anual
Estados Unidos	18-22 centros	$ 5-7 millones de inversiones colaborativas anuales
unión Europea	7-8 centros	$ 3-4 millones de inversiones colaborativas anuales

Grupos de defensa del paciente de enfermedades raras

Rocket Pharmaceuticals se involucra activamente con 8-10 organizaciones principales de defensa de enfermedades raras.

• Fondo de Investigación de Anemia Fanconi
• Asociación primaria de inmunodeficiencia
• Alianza global de desorden genético raro

Proveedores de atención médica especializados en condiciones genéticas

La compañía interfiere con aproximadamente 150-200 proveedores de atención médica genética especializados en todo el país.

Especialidad de proveedor	Número de proveedores	Compromiso clínico anual
Hematólogos pediátricos	75-90 proveedores	Soporte de investigación clínica de $ 2-3 millones
Especialistas en desorden genético	60-75 proveedores	$ 1.5-2.5 millones de colaboración de investigación

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

A partir del cuarto trimestre de 2023, Rocket Pharmaceuticals reportó gastos de I + D de $ 70.3 millones para el año fiscal. El desglose total de inversión de I + D de la compañía incluye:

Categoría de I + D	Monto del gasto
Programas de terapia génica	$ 42.5 millones
Investigación de enfermedad pediátrica rara	$ 18.7 millones
Desarrollo preclínico	$ 9.1 millones

Gestión y ejecución del ensayo clínico

Los gastos de ensayo clínico para 2023 totalizaron aproximadamente $ 45.2 millones, con la siguiente asignación:

• Ensayos de terapia génica de leucocitos: $ 22.6 millones
• Programa de Anemia Fanconi: $ 15.3 millones
• Ensayos de enfermedad metabólica: $ 7.3 millones

Procesos de cumplimiento y aprobación regulatoria

Los costos de cumplimiento regulatorio para 2023 se estimaron en $ 12.5 millones, incluidos:

Área de cumplimiento	Costo
Preparación de sumisión de la FDA	$ 5.8 millones
Documentación regulatoria	$ 4.2 millones
Monitoreo de cumplimiento	$ 2.5 millones

Protección de propiedad intelectual

Los gastos de propiedad intelectual para 2023 ascendieron a $ 6.8 millones, distribuidos de la siguiente manera:

• Presentación y mantenimiento de patentes: $ 4.3 millones
• Servicios de protección legal: $ 2.5 millones

Reclutamiento y retención de talento científico avanzado

Los costos de adquisición y retención de talentos para 2023 fueron de $ 18.6 millones:

Categoría de gestión del talento	Gastos
Salarios competitivos	$ 12.4 millones
Investigación de reclutamiento de científicos	$ 3.9 millones
Beneficios y capacitación de los empleados	$ 2.3 millones

Estructura de costos totales para 2023: $ 153.4 millones

Rocket Pharmaceuticals, Inc. (RCKT) - Modelo de negocios: flujos de ingresos

Acuerdos potenciales de licencia de productos futuros

A partir del cuarto trimestre de 2023, los productos farmacéuticos de cohetes tienen acuerdos de licencia potenciales en su cartera de terapia génica de enfermedades raras. El valor potencial estimado de estos acuerdos no se revela públicamente.

Subvenciones de investigación y financiación del gobierno

Fuente de financiación	Cantidad	Año
Subvenciones de los Institutos Nacionales de Salud (NIH)	$ 3.2 millones	2023
Financiación del Departamento de Investigación del Departamento de Defensa	$ 1.5 millones	2023

Asociaciones de investigación colaborativa

Las asociaciones de investigación actuales incluyen:

• Hospital de Niños de Boston
• Universidad de Minnesota
• Institutos Nacionales de Salud

Venta de productos terapéuticos potenciales

Los ingresos de Rocket Pharmaceuticals de posibles ventas de productos terapéuticos dependen de las aprobaciones de la FDA. La tubería actual incluye:

Terapia	Indicación	Valor de mercado potencial
RP-L201	Anemia fanconi	Mercado anual estimado de $ 150-200 millones
RP-A501	Enfermedad de Danon	Mercado anual estimado de $ 100-150 millones

Oportunidades de licencia de propiedad intelectual

A partir de 2023, Rocket Pharmaceuticals sostiene 17 patentes emitidas y 32 solicitudes de patentes pendientes a través de sus plataformas de terapia génica.

Gastos totales de investigación y desarrollo para 2023: $ 94.6 millones

Pérdida neta para 2023: $ 108.4 millones

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.