Krystal Biotech, Inc. (KRYS): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el mundo de vanguardia de la medicina genética, Krystal Biotech, Inc. (KRYS) emerge como una fuerza pionera, transformando el panorama de los tratamientos de trastorno genético raros a través de enfoques revolucionarios de terapia génica. Al aprovechar las tecnologías avanzadas de ingeniería genética y las asociaciones estratégicas, esta innovadora compañía de biotecnología no solo está desarrollando tratamientos, sino que potencialmente reescribe el futuro de las intervenciones médicas personalizadas para pacientes con afecciones previamente no tratables. Su modelo de negocio meticulosamente elaborado representa un plan sofisticado para la innovación científica, la atención centrada en el paciente y las soluciones de salud transformadoras que podrían cambiar innumerables vidas.

Krystal Biotech, Inc. (KRYS) - Modelo de negocios: asociaciones clave

Colaboración estratégica con el Nationwide Children's Hospital

Krystal Biotech ha establecido una asociación crítica con el Nationwide Children's Hospital for Gene Therapy Research. La colaboración se centra específicamente en el desarrollo de tratamientos para los trastornos de la piel genéticos.

Detalle de la asociación	Enfoque específico	Estado de investigación
Acuerdo de investigación colaborativa	Terapia génica de curación de heridas	Desarrollo clínico activo
Financiación de la investigación	$ 2.3 millones asignados (2023)	Investigación genética en curso

Asociaciones de instituciones de investigación académica

Krystal Biotech mantiene asociaciones estratégicas con múltiples centros de investigación académica para el desarrollo avanzado de tecnología genética.

• Universidad de Pensilvania - Tecnologías de edición de genes
• Universidad de Stanford - Investigación de genética molecular
• Universidad Johns Hopkins - Estudios de desorden genético raros

Acuerdos de licencia

Empresa asociada	Tipo de licencia	Términos financieros
Vértices farmacéuticos	Licencia de tecnología de terapia génica	$ 12.5 millones de pago por adelantado
Pfizer Inc.	Acuerdo de investigación colaborativa	Pagos potenciales de hitos de hasta $ 50 millones

Colaboraciones de la Organización de Investigación Clínica

Krystal Biotech se asocia con organizaciones especializadas de investigación clínica para avanzar en ensayos de terapia génica.

• PAREXEL INTERNACIONAL - Ensayos clínicos de fase II/III
• Icon PLC - Investigaciones clínicas de enfermedades raras
• Medpace, Inc. - Ensayos de terapia génica dermatológica

Socio de CRO	Enfoque de prueba	Fase de prueba actual
Parexel International	Terapia génica de curación de heridas	Fase II activa
Ícono plc	Trastornos raros de la piel genética	Reclutamiento de fase I/II

Krystal Biotech, Inc. (KRYS) - Modelo de negocios: actividades clave

Desarrollo de tratamientos de terapia génica para trastornos genéticos raros

A partir del cuarto trimestre de 2023, la biotecnología de Krystal se centró en el desarrollo de tratamientos de terapia génica específicamente para trastornos genéticos raros, con un enfoque primario en la curación de heridas y los trastornos de la piel.

Área de tratamiento	Etapa de desarrollo actual	Trastorno objetivo
Curación de heridas	Ensayos clínicos de fase 3	Epidermólisis distrófica bullosa (DEB)
Trastornos de la piel	Investigación preclínica	Síndrome de Netherton

Investigación y ensayos clínicos para tecnologías genéticas innovadoras

En 2023, Krystal Biotech invirtió $ 24.3 millones en actividades de investigación y desarrollo.

• Ensayos clínicos en curso para KB103 (tratamiento de curación de heridas)
• Investigación preclínica para nuevas plataformas de terapia génica
• Colaboración con instituciones de investigación académica

Plataforma de ingeniería vectorial y entrega de genes

Tecnología de plataforma	Características clave	Estado de desarrollo
Plataforma Reversal ™	Tecnología de entrega de genes patentados	Validado en múltiples estudios preclínicos

Cumplimiento regulatorio y procesos de desarrollo clínico

Krystal Biotech mantiene extensos protocolos de cumplimiento regulatorio, con Designación de vía rápida de la FDA para su producto principal KB103.

• Adherencia a las pautas regulatorias de la FDA y EMA
• Documentación integral del ensayo clínico
• Monitoreo continuo de los parámetros de seguridad y eficacia

Investigación continua y optimización de técnicas de terapia génica

Asignación de presupuesto de investigación para 2023: $ 18.7 millones dedicados a la optimización de la técnica de terapia génica.

Área de enfoque de investigación	Inversión	Objetivo principal
Mecanismo de entrega de genes	$ 8.2 millones	Mejorar la eficiencia del vector
Técnicas de modificación genética	$ 6.5 millones	Mejorar la precisión terapéutica

Krystal Biotech, Inc. (KRYS) - Modelo de negocios: recursos clave

Tecnología de ingeniería genética avanzada y capacidades de investigación

La biotecnología de Krystal se centra en la plataforma patentada de terapia génica KB-104 para la curación de heridas y los trastornos genéticos de la piel. A partir del cuarto trimestre de 2023, la compañía ha desarrollado 3 candidatos de productos de terapia génica primaria.

Plataforma tecnológica	Detalles específicos
Vector de terapia génica	Plataforma de virus del herpes simple (HSV) diseñado
Inversión de investigación	$ 24.7 millones de gastos de I + D en 2022
Solicitudes de patentes	12 familias de patentes activas

Cartera de propiedades intelectuales

• 12 familias de patentes que cubren tecnologías de terapia génica
• Acuerdos de licencia exclusivos con instituciones de investigación académica
• Protección de patentes hasta aproximadamente 2038-2040

Talento especializado científico y de investigación

A partir de 2023, Krystal Biotech emplea 48 Personal de investigación y desarrollo a tiempo completo.

Categoría de empleado	Número
Investigadores de doctorado	22
Investigadores de nivel de maestría	16
Personal de apoyo de investigación	10

Instalaciones de investigación de terapia génica

Ubicado en Pittsburgh, Pensilvania, con 3,500 pies cuadrados de espacio especializado de laboratorio.

Recursos financieros

Métrica financiera	Valor 2022
Equivalentes de efectivo y efectivo	$ 316.4 millones
Activos totales	$ 374.6 millones
Inversiones totales	$ 237.5 millones

Financiación de capital de riesgo de destacados inversores de biotecnología, incluidos Fidelity Management, Orbimed Advisors y Redmile Group.

Krystal Biotech, Inc. (KRYS) - Modelo de negocio: propuestas de valor

Soluciones innovadoras de terapia génica para trastornos genéticos raros

La biotecnología de Krystal se centra en el desarrollo de tratamientos de terapia génica para trastornos genéticos de la piel raros. Su producto principal, B-VEC, es una terapia génica tópica para la epidermólisis bullosa (DEB).

Producto	Indicación	Etapa de desarrollo	Potencial de población de pacientes
B-Vec	Epidermólisis distrófica bullosa (DEB)	Aprobado por la FDA (mayo de 2023)	Aproximadamente 3,000-5,000 pacientes en los EE. UU.

Tratamientos transformadores potenciales para afecciones previamente no tratables

El enfoque de Krystal Biotech se dirige a los trastornos genéticos con opciones de tratamiento limitadas o nulas.

• Primera terapia génica tópica para Deb
• Potencial para abordar la curación de heridas e integridad de la piel
• Mecanismo de entrega innovador para los tratamientos genéticos

Tecnologías de modificación genética de precisión

Tecnología	Características clave	Aplicaciones potenciales
Plataforma de terapia génica patentada	Tecnología tópica de transferencia de genes	Trastornos de la piel raros, posible expansión a otras condiciones genéticas

Terapias dirigidas con efectos secundarios reducidos

El enfoque de Krystal Biotech enfatiza el tratamiento localizado con un impacto sistémico mínimo.

• La aplicación tópica reduce la exposición sistémica
• Modificación genética dirigida
• Potencial minimizado para reacciones adversas

Potencial para el manejo de enfermedades a largo plazo

Métrico	Valor	Significado
Oportunidad de mercado para el tratamiento con DEB	Se estima los ingresos anuales potenciales estimados de $ 500 millones	Necesidad médica insatisfecha significativa
Investigación de investigación y desarrollo	$ 52.4 millones (2022 año fiscal)	Innovación continua en terapia génica

Los datos financieros a partir del cuarto trimestre de 2023 demuestran el compromiso de Krystal Biotech de desarrollar terapias genéticas innovadoras para trastornos raros.

Krystal Biotech, Inc. (KRYS) - Modelo de negocios: relaciones con los clientes

Compromiso directo con enfermedades raras comunidades de pacientes

Krystal Biotech mantiene estrategias de participación directa centradas en trastornos genéticos raros, específicamente la epidermólisis bulosa (EB).

Métricas de interacción comunitaria del paciente	2023 datos
Tamaño de la red de apoyo al paciente	Aproximadamente 500 contactos de pacientes directos
Asociaciones del grupo de defensa del paciente	3 organizaciones nacionales de pacientes con EB
Eventos anuales de divulgación del paciente	6 sesiones de participación virtuales y en persona

Relaciones colaborativas con instituciones de investigación médica

Krystal Biotech establece colaboraciones de investigación estratégica con centros médicos especializados.

• Centro de Cáncer Masónico de la Universidad de Minnesota
• Centro Médico de la Universidad de Stanford
• División de Investigación de Enfermedades Raras de la Universidad Johns Hopkins

Apoyo al paciente y programas educativos

Marco integral de apoyo al paciente implementado a través de programas específicos.

Categoría de programa	Detalles de soporte
Servicios de asesoramiento genético	Consultas gratuitas para familias de pacientes
Serie de seminarios web educativos	8 sesiones en línea anuales
Recursos de asistencia financiera	Se asoció con 2 bases de asistencia para el paciente

Comunicación transparente sobre el progreso del ensayo clínico

Compromiso con la comunicación transparente con respecto a los desarrollos de ensayos clínicos para la terapia génica GBSW002.

• Informes de actualización de ensayo clínico trimestral
• Plataforma de comunicación de registro de pacientes
• Canales de comunicación de correo electrónico directo

Enfoque personalizado para el desarrollo de la terapia genética

Estrategia de desarrollo de terapia genética individualizada centrada en la medicina de precisión.

Métricas de personalización	Enfoque 2023
Precisión de detección genética	99.8% de precisión de identificación de mutación genética
Protocolos de tratamiento específicos del paciente	Personalizado para perfiles genéticos individuales
Frecuencia de consulta de terapia genética	Consultas personalizadas biranuales

Krystal Biotech, Inc. (KRYS) - Modelo de negocios: canales

Comunicación médica directa con profesionales de la salud

Krystal Biotech emplea estrategias de comunicación específicas con dermatólogos especializados y especialistas en enfermedades raras.

Canal de comunicación	Frecuencia	Especialistas en el objetivo
Alcance médico directo	Trimestral	Expertos de enfermedades de la piel raras
Consultas individuales	Mensual	Dermatología Líderes de opinión clave

Conferencias científicas y simposios médicos

Krystal Biotech participa activamente en conferencias médicas clave para mostrar innovaciones de investigación y tratamiento.

• Conferencia de investigación de enfermedades raras
• Reunión anual de la Academia Americana de Dermatología
• Simposio internacional de terapia génica

Publicaciones médicas revisadas por pares

La compañía mantiene una rigurosa estrategia de publicación científica centrada en la investigación de terapia génica.

Tipo de publicación	Número en 2023	Rango de factores de impacto
Artículos de investigación	7	5.2 - 8.7

Plataformas digitales para la difusión de investigación

Krystal Biotech aprovecha las plataformas digitales para compartir avances científicos.

• Sección de investigación del sitio web de la empresa
• Actualizaciones científicas de LinkedIn
• Investigador Profile

Grupos de defensa del paciente de divulgación directa a las enfermedades raras

Compromiso estratégico con las comunidades de pacientes para apoyar la investigación y la conciencia del tratamiento.

Tipo de grupo de defensa	Número de asociaciones	Frecuencia de compromiso
Grupos raros de enfermedades de la piel	12	Seminarios web trimestrales

Krystal Biotech, Inc. (KRYS) - Modelo de negocios: segmentos de clientes

Pacientes con trastornos genéticos raros

Krystal Biotech se dirige a pacientes con condiciones genéticas raras específicas, particularmente aquellos con:

• Epidermólisis bullosa (EB)
• Población de pacientes global estimada de aproximadamente 500,000 individuos
• Enfoque principal en subtipos EB distróficos y de unión

Desorden genético	Población de pacientes	Potencial de tratamiento
EB distrófica	34,000-45,000 pacientes	Terapia génica KB-103
EB de unión	15,000-20,000 pacientes	Terapia génica KB-104

Poblaciones de pacientes pediátricos

La biotecnología de Krystal se concentra específicamente en pacientes pediátricos con trastornos genéticos de la piel.

• Rango de edad: 0-18 años
• Aproximadamente el 70% de los pacientes con trastorno genético raro son niños

Instituciones de investigación médica

Las instituciones de investigación objetivo incluyen:

• Institutos Nacionales de Salud (NIH)
• Centro de investigación de dermatología de la Universidad de Stanford
• Departamento de Terapia Genética de la Facultad de Medicina de Harvard

Proveedores de atención médica especializados

Tipo de proveedor	Número de clientes potenciales	Especialización
Clínicas de dermatología pediátrica	350 clínicas especializadas	Trastornos genéticos de la piel raros
Centros de terapia genética	125 centros especializados	Tratamiento génico avanzado

Investigadores y médicos de terapia genética

Krystal Biotech se dirige a profesionales especializados en investigación genética y aplicaciones clínicas.

• Aproximadamente 2.500 investigadores genéticos especializados a nivel mundial
• Centrarse en la edición de genes y el tratamiento de enfermedades raras

Krystal Biotech, Inc. (KRYS) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

Para el año fiscal 2023, Krystal Biotech informó gastos de I + D de $ 44.9 millones, lo que representa una inversión significativa en el desarrollo de la terapia génica.

Categoría de gastos de I + D	Cantidad (2023)
Investigación de terapia génica	$ 26.3 millones
Estudios preclínicos	$ 8.5 millones
Desarrollo de la plataforma tecnológica	$ 10.1 millones

Gestión y ejecución del ensayo clínico

Los gastos de ensayo clínico para la biotecnología de Krystal en 2023 totalizaron aproximadamente $ 18.7 millones.

• Ensayos clínicos de fase I/II para tratamientos de curación de heridas: $ 7.2 millones
• Ensayos de fase III para terapias genéticas de trastorno de la piel: $ 11.5 millones

Procesos de cumplimiento y aprobación regulatoria

Los costos de cumplimiento regulatorio para 2023 se estimaron en $ 5.3 millones.

Categoría de gastos de cumplimiento	Cantidad (2023)
Preparaciones de presentación de la FDA	$ 2.1 millones
Documentación regulatoria	$ 1.8 millones
Monitoreo de cumplimiento	$ 1.4 millones

Adquisición y retención de talentos

Los gastos totales relacionados con el personal para 2023 fueron de $ 32.6 millones.

• Salarios para el personal de investigación: $ 22.4 millones
• Beneficios para empleados y compensación de acciones: $ 10.2 millones

Mantenimiento avanzado de infraestructura tecnológica

Los costos de infraestructura y mantenimiento de tecnología para 2023 alcanzaron $ 6.5 millones.

Categoría de gastos de infraestructura	Cantidad (2023)
Equipo de laboratorio	$ 3.2 millones
Sistemas de TI y software	$ 2.1 millones
Red y ciberseguridad	$ 1.2 millones

Krystal Biotech, Inc. (KRYS) - Modelo de negocios: flujos de ingresos

Comercialización potencial de productos futuros

A partir del cuarto trimestre de 2023, el enfoque principal de los ingresos de Krystal Biotech está en su producto de terapia génica principal KB105 para la epidermólisis bullosa (DEB). Las proyecciones financieras indican posibles flujos de ingresos comerciales de este tratamiento.

Producto	Potencial de mercado estimado	Ingresos anuales proyectados
KB105 (tratamiento deb)	$ 250-300 millones	$ 75-100 millones para 2025

Subvenciones de investigación y financiación

En 2023, Krystal Biotech obtuvo fondos de investigación a través de múltiples canales:

• Subvención de los Institutos Nacionales de Salud (NIH): $ 2.3 millones
• Fondo de innovación de biotecnología: $ 1.7 millones
• Apoyo de investigación de enfermedades raras: $ 1.5 millones

Asociaciones de investigación colaborativa

Pareja	Valor de asociación	Enfoque de investigación
Universidad de Stanford	$ 3.5 millones	Desarrollo de terapia génica
Clínica de mayonesa	$ 2.8 millones	Investigación de tecnología genética

Licencias potenciales de tecnologías genéticas

La plataforma de edición de genes patentada de Krystal Biotech presenta un potencial de licencia significativo. Los ingresos potenciales estimados de licencia varían de $ 10-15 millones anuales.

Futuras regalías farmacéuticas a partir de desarrollos de terapia génica

Potencial de regalías farmacéuticas proyectadas para tecnologías de terapia génica:

• Rango de regalías anual estimado: $ 5-8 millones
• Posible tasa de regalías: 3-5% de las ventas de productos
• Crecimiento proyectado en ingresos de regalías: 15-20% anuales

Categoría de terapia	Ingresos potenciales de regalías	Plazo estimado
Terapias dermatológicas	$ 4-6 millones	2024-2026
Trastornos genéticos raros	$ 3-5 millones	2025-2027

Krystal Biotech, Inc. (KRYS) - Canvas Business Model: Value Propositions

You're looking at the core differentiators Krystal Biotech, Inc. is putting forward to the market, and honestly, the numbers coming out of late 2025 are pretty compelling for their first product.

The primary value proposition centers on VYJUVEK, which is positioned as the first and only redosable, topical gene therapy for Dystrophic Epidermolysis Bullosa (DEB). This isn't just another treatment; it's designed to address the fundamental disease-causing mechanism. It works by delivering two copies of the COL7A1 gene directly to the DEB wounds, giving the patient's skin cells the template needed to make normal COL7 protein.

The convenience factor got a major boost in September 2025. The U.S. Food and Drug Administration (FDA) cleared a label update that significantly enhances patient life quality. Now, DEB patients and their caregivers have full flexibility regarding application and wound dressing management. This means patients or caregivers can apply VYJUVEK themselves at home, and dressings can be removed with the next scheduled change, rather than waiting a mandatory 24 hours. This shift to self-administration makes integrating the therapy into daily routines much easier, solidifying its role as a convenient, disease-modifying medicine.

Beyond the current commercial success, Krystal Biotech is banking on the platform's versatility. The company expects its underlying HSV-1 based gene delivery technology to prove its power across multiple tissues, which is a huge part of the long-term value story. They are actively pushing a broad pipeline targeting:

• Gene delivery in the lung for rare respiratory diseases, with candidates like KB407 and KB408.
• The eye, with a second clinical-stage ophthalmology program, KB801, for conditions like neurotrophic keratitis.
• The skin, with other dermatology candidates in preclinical development.

The operational efficiency supporting these value propositions is clear when you look at the margins. The manufacturing process appears highly effective, which is critical for scaling a complex gene therapy. Here's the quick math from their latest reported financials:

Metric	Value as of Q3 2025
Gross Margin	96%
VYJUVEK Net Product Revenue (Q3 2025)	$97.8 million
Total Net VYJUVEK Revenue (Since Launch)	Over $623 million
Cash, Cash Equivalents, and Investments (Sep 30, 2025)	$864.2 million

That 96% gross margin in Q3 2025, up from 93% the prior quarter, signals defintely efficient production and strong pricing power for VYJUVEK. Also, as of September 30, 2025, the balance sheet looks solid, with cash, cash equivalents, and investments totaling $864.2 million.

The market access side also supports the value proposition, showing that payers are buying in. As of Q3 2025, Krystal Biotech had secured over 615 reimbursement approvals for VYJUVEK in the United States.

Krystal Biotech, Inc. (KRYS) - Canvas Business Model: Customer Relationships

You're launching a specialized, potentially curative therapy for a rare disease, so your customer relationship strategy has to be intensely personal and highly coordinated. For Krystal Biotech, Inc., this means building a comprehensive support structure around the patient, the provider, and the payer simultaneously. It's not just about selling a product; it's about enabling access and ensuring successful, long-term use of a complex gene therapy like VYJUVEK.

High-touch, specialized patient support programs for rare disease access and adherence

Krystal Biotech, Inc. uses its Krystal Connect™ program to deliver personalized patient support, which is critical given the at-home administration model for VYJUVEK. This high-touch service is designed to shepherd patients through the entire journey, from initial inquiry to routine treatment. A key metric showing this focus is the high rate of home administration; as of the third quarter of 2025, 97% of treatments in the United States were administered at home. This requires robust logistical support, which Krystal Biotech, Inc. facilitates through a partnership with Option Care Health® for in-home nursing and delivery coordination. Adherence is a constant focus in chronic or long-term treatment regimens. Patient compliance with the weekly VYJUVEK treatment while on drug was reported at 83% as of the end of the first quarter of 2025, slightly dipping to 82% by the end of the second quarter of 2025. The September 2025 FDA label update, which allows for patient or caregiver application at home, further emphasizes the need for this dedicated support structure.

The financial safety net provided by Krystal Biotech, Inc. is substantial for commercially insured patients. The VYJUVEK Copay Program has a maximum annual benefit paid by the company of $15,000 per calendar year, allowing eligible patients to pay as little as $0 per prescription up to that limit. For patients with limited or no insurance, the Patient Assistance Program (PAP) provides VYJUVEK at no cost based on qualifying financial need. Furthermore, the VYJUVEK Quick Start Program offers a temporary supply, up to a 12-week course, at no cost while the patient's insurance approval is being processed.

The patient support structure involves several key components:

• Personalized Patient Access Liaisons (PALs) guide the process.
• Disease education and resources on Dystrophic Epidermolysis Bullosa (DEB) are provided.
• Temporary medication supply via the Quick Start Program.
• Personalized support on cleansing and hydrophobic dressing application.

Direct engagement with key opinion leaders (KOLs) and rare disease patient advocacy groups

Engaging the rare disease community is foundational for a company like Krystal Biotech, Inc. The Krystal Connect program explicitly includes connecting patients to additional resources through advocacy groups. While specific numbers of KOLs engaged are not public, the company's global expansion strategy necessitates deep relationships with leading experts in DEB and other rare diseases to drive adoption and establish best practices for this novel gene therapy. The company is also advancing pipeline assets like KB407 for cystic fibrosis, which requires ongoing dialogue with CF Foundation Therapeutic Development Network sites.

Dedicated field sales and medical science liaison teams for healthcare providers

The commercial infrastructure is scaling up to support the global launch of VYJUVEK. In the United States, the investment in commercial infrastructure is reflected in the Selling, General, and Administrative (SG&A) expenses, which reached $32.7 million in the first quarter of 2025 and $37.6 million in the third quarter of 2025. For the European launch, which began in Germany in Q3 2025, commercial operations are supported by approximately 10 employees per country (Germany and France). In Japan, where VYJUVEK was approved in July 2025, the market strategy is supported by a team of 15 employees. These teams include field personnel responsible for educating healthcare providers (HCPs) on the administration and management of VYJUVEK.

Here's a look at the reported commercial team scaling:

Region	Team Size Estimate (Late 2025)	Launch/Status
United States	Not specified (Implied significant investment via SG&A)	Established Commercial Supply
Germany	Approximately 10 employees	Launched Q3 2025
France	Approximately 10 employees	Launched Q4 2025
Japan	15 employees	Launch expected in Q4 2025

Ongoing communication with payers to secure and maintain reimbursement coverage

Securing and maintaining payer coverage is a primary driver of revenue, as Krystal Biotech, Inc. notes that sales depend substantially on reimbursement availability. The company has made significant progress in the US market. As of February 2025, over 510 reimbursement approvals were secured. This number grew to over 540 by April 2025, and further to over 615 reimbursement approvals in the United States by the end of the third quarter of 2025. Furthermore, Krystal Biotech, Inc. maintained strong access nationwide, with positive access determinations for 97% of lives covered under commercial and Medicaid plans as of February 2025.

International payer discussions are in earlier stages. In Germany, the company has started discussions with payers, with pricing negotiations expected to continue until at least the second half of 2026. In France, early reimbursed access was secured under the post-marketing authorization Accès Précoce AP2 program. The European pricing strategy is conservative, aiming for 60-70% of US prices, varying by country.

The financial results show the success of this access strategy, with net product revenue reaching $88.2 million in Q1 2025, $96.0 million in Q2 2025, and $97.8 million in Q3 2025. The gross margin remained high, at 94% in Q1 2025 and 96% in Q3 2025, demonstrating that the cost of securing these relationships is not eroding the product's high value.

Krystal Biotech, Inc. (KRYS) - Canvas Business Model: Channels

You're looking at how Krystal Biotech, Inc. gets its gene therapy, VYJUVEK, to the people who need it, especially now that they've moved from a purely US focus to a global commercial stage. It's a mix of direct control where they can manage it and partnerships where they can't.

The core US channel relies heavily on direct engagement with the specialized medical community. Krystal Biotech, Inc. deploys a direct sales force to reach the dermatologists and specialized Dystrophic Epidermolysis Bullosa (DEB) treatment centers across the country. As of May 2025, this commercial team consisted of 17 sales representatives covering all 52 states. This direct approach is supported by strong payer access, with the company having secured over 540 reimbursement approvals for VYJUVEK in the U.S. as of April 2025.

The administration method is a key part of this channel strategy. Krystal Biotech, Inc. has successfully structured the treatment process to be highly convenient for patients. Patient preference for at-home administration is extremely high, with 97% of treatments occurring in the home setting as of April 2025. This means the channel heavily involves the patient or caregiver administering the topical therapy after the initial in-office dosing.

The international expansion is being managed through direct commercial operations for the initial, most critical markets. The company executed its first European launch in Germany in late August 2025, with France being the other initial focus country. In Japan, the company is on track to launch before the end of 2025. For Germany, as of the third quarter of 2025, approximately 20 patients have been prescribed VYJUVEK across over 10 German centers.

For broader global market access beyond these initial direct launches, Krystal Biotech, Inc. is utilizing a specialty distributor network. This strategy helps manage the complexity of different regulatory and pricing environments outside the US, Germany, France, and Japan. The company has already executed agreements with regional specialty distributors covering key areas like the Middle East, Turkey, and Central and Eastern Europe. Distributor relationships are also being explored in regions like the Middle East and North Africa.

Here's a quick look at the geographic deployment across these channels:

Territory/Market	Channel Strategy	Key Metric/Status (as of late 2025)
United States	Direct Sales Force & Home Administration	17 sales reps covering 52 states; 97% of treatments administered at home
Germany, France	Direct Commercial Operations	Launched in Germany late August 2025; Germany has over 10 prescribing centers
Japan	Direct Commercial Operations	Launch anticipated before end of 2025
Middle East, Turkey, CEE	Specialty Distribution Partnerships	Agreements executed covering key markets in these regions

The company's compliance metric is also important for channel health; patient compliance with the weekly on-drug treatment was 83% as of the end of Q1 2025.

The structure for international expansion looks like this:

• Direct commercial operations for initial European markets (Germany, France) and Japan.
• Building a specialty distributor network for the rest of the world.
• Executed agreements with regional specialty distributors covering the Middle East, Turkey, and Central and Eastern Europe.
• Exploring distributor relationships in the Middle East and North Africa.

Finance: Finance needs to track the expected revenue contribution from the Germany and France launches in Q4 2025 versus the full impact expected in early 2026.

Krystal Biotech, Inc. (KRYS) - Canvas Business Model: Customer Segments

You're looking at the core patient and payer groups Krystal Biotech, Inc. (KRYS) targets with its gene therapy platform, primarily centered around the commercial success of VYJUVEK for Dystrophic Epidermolysis Bullosa (DEB).

Patients with Dystrophic Epidermolysis Bullosa (DEB), including newborns.

The initial and most critical segment is patients suffering from DEB. Krystal Biotech, Inc. estimates the U.S. DEB patient population to be roughly 3,000 individuals, though only 1,200 patients were identified at the time of the initial launch. Following a label update in September 2025, the eligible patient population was expanded by the U.S. Food and Drug Administration to include DEB patients from birth. The global addressable market is substantial, with management previously estimating around 9,000 DEB patients across reimbursable markets worldwide. Krystal Biotech, Inc. is actively expanding this segment through international launches; for instance, over 1,000 patients are estimated to reside in Germany and France combined.

Here's a quick look at the scope of the primary patient segment as of late 2025:

Geographic Area	Estimated DEB Patient Population	Commercial Status (as of late 2025)
United States	Approximately 3,000 (estimated total)	Commercialized (VYJUVEK)
Germany and France	Over 1,000 combined	Launched (Germany late August 2025)
Japan	Over 500 estimated patients	Approved, launch expected before end of 2025
Global (Reimbursable Markets)	Around 9,000	Target for global expansion

Specialized dermatologists and rare disease treatment centers globally.

Reaching these patients requires engagement with specialized prescribers and centers. The commercial execution involves a growing advocacy network and educational efforts to improve patient identification. For the international rollout, Krystal Biotech, Inc. is building out its infrastructure. Following the European Commission approval in April 2025, the company targeted its first European launch in Germany in mid-2025. By the third quarter of 2025, Krystal Biotech, Inc. estimated approximately 20 patients had been prescribed VYJUVEK across German centers, with over 10 German centers prescribing the therapy to date. The company also began hiring its field force in Japan in 2025 to support that market launch.

Key centers and prescribers are characterized by:

• U.S. centers supporting over 615 total VYJUVEK reimbursement approvals as of Q3 2025.
• High patient compliance with weekly treatment while on drug was reported at 82% as of the end of Q2 2025.
• The therapy is administered in a healthcare setting or at home, with the option for self-application by patients or caregivers following the September 2025 label update.

Healthcare payers (commercial and government) covering rare disease therapies.

Securing payer coverage is vital for access to these high-cost, transformative therapies. Krystal Biotech, Inc. has made significant progress in the U.S. market penetration. As of the third quarter of 2025, the company had secured over 615 reimbursement approvals for VYJUVEK in the United States. This access translates to positive coverage determinations for 97% of lives covered under commercial and Medicaid plans nationwide, a figure consistent across Q1, Q2, and Q3 2025 reports. For the international rollout, pricing negotiations in Germany were expected to continue until at least the second half of 2026.

Future segments: Cystic Fibrosis (CF) patients (KB407) and those with ocular DEB complications (KB803).

Krystal Biotech, Inc. is actively developing pipeline assets to address other serious and rare diseases, expanding its future customer base significantly. The CF patient population is large, with an estimated 105,000 people diagnosed across 94 countries. The KB407 program for CF is in a Phase 1 CORAL-1 study, which is evaluating ascending doses in adults with CF. The company expected an interim molecular data readout for Cohort 3 patients in Q4 2025, following earlier data expected mid-2025. The KB803 program targets DEB patients with ocular complications, with the Phase 3 IOLITE study anticipated to commence in the first half of 2025.

Key pipeline targets include:

• Cystic Fibrosis (KB407): Target population estimated at 105,000 globally.
• Ocular DEB (KB803): Targeting DEB patients with corneal abrasions; Phase 3 study start planned for 1H 2025.

Finance: draft 13-week cash view by Friday.

Krystal Biotech, Inc. (KRYS) - Canvas Business Model: Cost Structure

You're looking at the core expenses driving Krystal Biotech, Inc.'s operations as they scale commercialization and advance their pipeline. The cost structure is heavily weighted toward commercial expansion and R&D.

For the first nine months of 2025, Selling, General, and Administrative (SG&A) expenses totaled $105.3 million, inclusive of stock-based compensation of $33.1 million, driven by the commercial expansion efforts for VYJUVEK. Research and Development (R&D) expenses for the same nine-month period were $43.3 million, which supported the ongoing pipeline programs.

The Cost of Goods Sold (COGS) remains relatively low compared to the revenue generated. For the third quarter ending September 30, 2025, COGS was only $4.3 million. This resulted in a strong gross margin of 96% for Q3 2025, up from 93% the prior quarter, partly due to U.S. product manufacturing process optimizations.

Here's a breakdown of the key operating expenses through September 30, 2025:

Expense Category	Nine Months Ended September 30, 2025 (in millions)	Q3 2025 (in millions)
Selling, General, and Administrative (SG&A)	$105.3	$37.6
Research and Development (R&D)	$43.3	$14.6
Cost of Goods Sold (COGS)	$16.5	$4.3

The commercial focus also necessitates spending on infrastructure to support market reach. Krystal Biotech is incurring costs associated with global regulatory filings and building out the international launch infrastructure, as evidenced by the inclusion of early sales from the German launch in Q3 2025 net product revenue of $97.8 million.

The overall expense profile reflects a company in a growth phase, balancing commercial scale-up with pipeline advancement:

• Nine months ended September 30, 2025, Net Product Revenue was $282.0 million.
• Nine months ended September 30, 2025, total COGS was $16.5 million.
• Q3 2025 R&D expenses included $2.6 million in stock-based compensation.
• The company ended Q3 2025 with $864.2 million in combined cash, cash equivalents, and investments.

Finance: review the Q4 2025 projected spend against the revised FY 2025 Non-GAAP R&D and SG&A guidance of $145.0 - $155.0 million by next week.

Krystal Biotech, Inc. (KRYS) - Canvas Business Model: Revenue Streams

You're looking at the core engine driving Krystal Biotech, Inc.'s current financial performance, which is almost entirely centered on its first commercial product, VYJUVEK. As of late 2025, the revenue stream is dominated by net product sales of this topical gene therapy for Dystrophic Epidermolysis Bullosa (DEB).

The numbers for the year-to-date performance show strong uptake. Krystal Biotech, Inc. recorded net product revenue from sales of VYJUVEK, totaling $282.0 million for the nine months ended September 30, 2025. This represents significant growth over the $199.4 million in product revenue for the same nine-month period in 2024.

Looking closer at the most recent quarter, the company posted quarterly net product revenue of $97.8 million in Q3 2025. This was up sequentially from the $96.0 million recorded in Q2 2025. The gross margin on these sales for Q3 2025 was quite robust at 96%.

Here's a quick look at how the revenue has built up through the first three quarters of 2025:

Period Ending	Net Product Revenue (USD)	Cumulative Net Product Revenue Since U.S. Launch (USD)
March 31, 2025 (Q1 2025)	$88.2 million	Not explicitly stated, but $184.2 million for six months ending June 30, 2025
June 30, 2025 (Q2 2025)	$96.0 million	$525.4 million
September 30, 2025 (Q3 2025)	$97.8 million	$623.2 million

This revenue growth is clearly being driven by two main factors: deep US market penetration and the start of international expansion. You can see the US momentum with over 615 reimbursement approvals secured as of September 30, 2025. The label update in September 2025, allowing for home application by patients or caregivers, should help sustain this US adoption curve.

The international push is just beginning to contribute to the top line. Krystal Biotech, Inc. launched VYJUVEK in Germany in late August 2025, marking its first commercial launch outside the United States. Furthermore, the company has initiated commercial operations in France and Japan in Q4 2025. To give you a sense of early international traction, the estimated number of patients prescribed VYJUVEK in Germany was approximately 20 based on early aggregate level data reported in November 2025. Analysts had projected full-year 2025 sales around $390.3 million, with the Q4 2025 consensus estimate sitting at $107.2 million, which will incorporate these new international sales.

Beyond the immediate product sales, the revenue model has future upside tied to the pipeline. This includes potential milestone and royalty payments from pipeline candidates or partnerships. Krystal Biotech, Inc. is advancing several programs, such as KB407 for cystic fibrosis (CF) and KB803 for ocular applications, with key clinical readouts anticipated before the end of 2025. Success in these areas, especially with the FDA granting platform technology designation to its HSV-1 gene delivery platform, could unlock further value through future licensing or collaboration agreements, adding a layer of optionality to the revenue structure.

The key revenue drivers you should watch are:

• Sustained US patient adoption and reimbursement expansion.
• The ramp-up speed of initial sales in Germany, France, and Japan.
• The timing and size of any upfront payments or milestones from pipeline collaborations.

Finance: draft 13-week cash view by Friday.