Krystal Biotech, Inc. (Krys): Modelo de negócios Canvas [Jan-2025 Atualizado]

No mundo de ponta da medicina genética, a Krystal Biotech, Inc. (KRYS) surge como uma força pioneira, transformando a paisagem dos raros tratamentos de transtorno genético por meio de abordagens revolucionárias de terapia genética. Ao alavancar tecnologias avançadas de engenharia genética e parcerias estratégicas, esta empresa inovadora de biotecnologia não está apenas desenvolvendo tratamentos, mas potencialmente reescrevendo o futuro de intervenções médicas personalizadas para pacientes com condições anteriormente incomparáveis. Seu modelo de negócios meticulosamente criado representa um projeto sofisticado de inovação científica, atendimento centrado no paciente e soluções transformadoras de saúde que podem mudar inúmeras vidas.

Krystal Biotech, Inc. (Krys) - Modelo de negócios: Parcerias -chave

Colaboração estratégica com o Nationwide Children's Hospital

A Krystal Biotech estabeleceu uma parceria crítica com o Hospital Infantil em todo o país para pesquisa de terapia genética. A colaboração se concentra especificamente no desenvolvimento de tratamentos para distúrbios genéticos da pele.

Detalhes da parceria	Foco específico	Status de pesquisa
Acordo de Pesquisa Colaborativa	Terapia genética de cicatrização de feridas	Desenvolvimento clínico ativo
Pesquisa financiamento	US $ 2,3 milhões alocados (2023)	Pesquisa genética em andamento

Parcerias das Instituições de Pesquisa Acadêmica

A Krystal Biotech mantém parcerias estratégicas com vários centros de pesquisa acadêmica para desenvolvimento avançado de tecnologia genética.

• Universidade da Pensilvânia - Tecnologias de edição de genes
• Universidade de Stanford - pesquisa de genética molecular
• Universidade Johns Hopkins - Estudos raros de transtorno genético

Acordos de licenciamento

Empresa parceira	Tipo de licença	Termos financeiros
Pharmaceuticals de vértice	Licença de tecnologia de terapia genética	Pagamento inicial de US $ 12,5 milhões
Pfizer Inc.	Acordo de Pesquisa Colaborativa	Potenciais pagamentos em até US $ 50 milhões

Colaborações da Organização de Pesquisa Clínica

A Krystal Biotech faz parceria com organizações especializadas de pesquisa clínica para promover os ensaios de terapia genética.

• Parexel International - Fase II/III ensaios clínicos
• ICON PLC - Investigações clínicas de doenças raras
• Medpace, Inc. - Ensaios de terapia genética dermatológica

CRO Parceiro	Foco no teste	Fase de teste atual
Parexel International	Terapia genética de cicatrização de feridas	Fase II ativa
Icon plc	Distúrbios genéticos raros	Fase I/II Recrutamento

Krystal Biotech, Inc. (Krys) - Modelo de negócios: Atividades -chave

Desenvolvimento de tratamentos de terapia genética para distúrbios genéticos raros

No quarto trimestre 2023, a Krystal Biotech se concentrou no desenvolvimento de tratamentos de terapia genética especificamente para distúrbios genéticos raros, com foco primário na cicatrização de feridas e distúrbios da pele.

Área de tratamento	Estágio de desenvolvimento atual	Transtorno direcionado
Cicatrização de feridas	Ensaios clínicos de fase 3	Epidermólise distrófica Bolosa (Deb)
Distúrbios da pele	Pesquisa pré -clínica	Síndrome de Netherton

Pesquisa e ensaios clínicos para tecnologias genéticas inovadoras

Em 2023, a Krystal Biotech investiu US $ 24,3 milhões em atividades de pesquisa e desenvolvimento.

• Ensaios clínicos em andamento para KB103 (tratamento de cicatrização de feridas)
• Pesquisa pré -clínica para novas plataformas de terapia genética
• Colaboração com instituições de pesquisa acadêmica

Plataforma de engenharia de vetores e genes proprietários

Tecnologia da plataforma	Principais características	Status de desenvolvimento
Plataforma Reversal ™	Tecnologia proprietária de entrega de genes	Validado em vários estudos pré -clínicos

Processos de conformidade regulatória e desenvolvimento clínico

Krystal Biotech mantém extensos protocolos de conformidade regulatória, com Designação de pista rápida da FDA para seu produto principal KB103.

• Aderência às diretrizes regulatórias da FDA e EMA
• Documentação abrangente do ensaio clínico
• Monitoramento contínuo dos parâmetros de segurança e eficácia

Pesquisa contínua e otimização de técnicas de terapia genética

Alocação de orçamento de pesquisa para 2023: US $ 18,7 milhões dedicados à otimização da técnica de terapia genética.

Área de foco de pesquisa	Investimento	Objetivo primário
Mecanismo de entrega de genes	US $ 8,2 milhões	Melhorar a eficiência do vetor
Técnicas de modificação genética	US $ 6,5 milhões	Aumente a precisão terapêutica

Krystal Biotech, Inc. (Krys) - Modelo de negócios: Recursos -chave

Tecnologia avançada de engenharia genética e recursos de pesquisa

A Krystal Biotech se concentra na plataforma de terapia genética proprietária KB-104 para cicatrização de feridas e distúrbios genéticos da pele. A partir do quarto trimestre 2023, a empresa desenvolveu 3 candidatos a terapia genética primária.

Plataforma de tecnologia	Detalhes específicos
Vetor de terapia genética	Plataforma de vírus Herpes simplex (HSV) projetada de herpes (HSV)
Investimento em pesquisa	US $ 24,7 milhões de despesas de P&D em 2022
Aplicações de patentes	12 famílias de patentes ativas

Portfólio de propriedade intelectual

• 12 famílias de patentes cobrindo tecnologias de terapia genética
• Acordos de licenciamento exclusivos com instituições de pesquisa acadêmica
• Proteção de patentes até aproximadamente 2038-2040

Talento científico e de pesquisa especializado

A partir de 2023, a Krystal Biotech emprega 48 pessoal de pesquisa e desenvolvimento em tempo integral.

Categoria de funcionários	Número
Pesquisadores de doutorado	22
Pesquisadores de nível de mestrado	16
Pessoal de apoio à pesquisa	10

Instalações de pesquisa de terapia genética

Localizado em Pittsburgh, Pensilvânia, com 3.500 pés quadrados de espaço de laboratório especializado.

Recursos financeiros

Métrica financeira	2022 Valor
Caixa e equivalentes de dinheiro	US $ 316,4 milhões
Total de ativos	US $ 374,6 milhões
Total de investimentos	US $ 237,5 milhões

Financiamento de capital de risco de investidores proeminentes de biotecnologia, incluindo gerenciamento de fidelidade, consultores orbimed e grupo Redmile.

Krystal Biotech, Inc. (Krys) - Modelo de negócios: proposições de valor

Soluções inovadoras de terapia genética para distúrbios genéticos raros

Krystal Biotech se concentra no desenvolvimento de tratamentos de terapia genética para distúrbios genéticos raros da pele. Seu produto principal, B-VEC, é uma terapia genética tópica para a epidermólise distrófica Bolosa (Deb).

Produto	Indicação	Estágio de desenvolvimento	Potencial população de pacientes
B-VEC	Epidermólise distrófica Bolosa (Deb)	FDA aprovado (maio de 2023)	Aproximadamente 3.000-5.000 pacientes nos EUA

Tratamentos transformadores em potencial para condições anteriormente intratáveis

A abordagem da Krystal Biotech tem como alvo distúrbios genéticos com opções de tratamento limitadas ou nenhuma existente.

• Primeira terapia genética tópica para Deb
• Potencial para abordar a cicatrização de feridas e a integridade da pele
• Mecanismo de entrega inovador para tratamentos genéticos

Tecnologias de modificação genética de precisão

Tecnologia	Principais características	Aplicações em potencial
Plataforma de terapia genética proprietária	Tecnologia de transferência de genes tópicos	Distúrbios raros da pele, expansão potencial para outras condições genéticas

Terapias direcionadas com efeitos colaterais reduzidos

A abordagem da Krystal Biotech enfatiza o tratamento localizado com impacto sistêmico mínimo.

• A aplicação tópica reduz a exposição sistêmica
• Modificação genética direcionada
• Potencial minimizado para reações adversas

Potencial para gerenciamento de doenças a longo prazo

Métrica	Valor	Significado
Oportunidade de mercado para tratamento Deb	Receita anual potencial estimada em US $ 500 milhões	Necessidade médica não atendida significativa
Investimento de pesquisa e desenvolvimento	US $ 52,4 milhões (2022 ano fiscal)	Inovação contínua na terapia genética

Os dados financeiros do quarto trimestre 2023 demonstram o compromisso da Krystal Biotech em desenvolver terapias genéticas inovadoras para distúrbios raros.

Krystal Biotech, Inc. (Krys) - Modelo de negócios: Relacionamentos do cliente

Engajamento direto com comunidades de pacientes com doenças raras

A Krystal Biotech mantém estratégias de engajamento direto focadas em distúrbios genéticos raros, especificamente epidermólise Bollosa (EB).

Métricas de interação da comunidade de pacientes	2023 dados
Tamanho da rede de suporte ao paciente	Aproximadamente 500 contatos diretos do paciente
Parcerias do Grupo de Advocacia dos Pacientes	3 organizações nacionais de pacientes com EB
Eventos anuais de divulgação de pacientes	6 sessões de engajamento virtual e pessoal

Relações colaborativas com instituições de pesquisa médica

A Krystal Biotech estabelece colaborações estratégicas de pesquisa com centros médicos especializados.

• Centro de Câncer Maçônico da Universidade de Minnesota
• Centro Médico da Universidade de Stanford
• Divisão de Pesquisa de Doenças Raras da Universidade Johns Hopkins

Apoio ao paciente e programas educacionais

Estrutura abrangente de suporte ao paciente implementada por meio de programas direcionados.

Categoria de programa	Detalhes de suporte
Serviços de aconselhamento genético	Consultas gratuitas para famílias de pacientes
Série de webinars educacionais	8 sessões online anuais
Recursos de assistência financeira	Fundamentos em parceria com 2 fundações de assistência ao paciente

Comunicação transparente sobre o progresso do ensaio clínico

Compromisso com a comunicação transparente em relação ao desenvolvimento de ensaios clínicos para a terapia genética GBSW002.

• Relatórios trimestrais de atualização de ensaios clínicos
• Plataforma de comunicação de registro de pacientes
• Canais de comunicação de email direto

Abordagem personalizada para o desenvolvimento da terapia genética

Estratégia de desenvolvimento de terapia genética individualizada focada na medicina de precisão.

Métricas de personalização	2023 abordagem
Precisão de triagem genética	99,8% de precisão de identificação de mutação genética
Protocolos de tratamento específicos para pacientes	Personalizado para perfis genéticos individuais
Frequência de consulta de terapia genética	Consultas personalizadas bi-anuais

Krystal Biotech, Inc. (Krys) - Modelo de Negócios: Canais

Comunicação médica direta com profissionais de saúde

A Krystal Biotech emprega estratégias de comunicação direcionadas com dermatologistas especializados e especialistas em doenças raras.

Canal de comunicação	Freqüência	Especialistas -alvo
Extensão médica direta	Trimestral	Especialistas em doenças de pele raras
Consultas individuais	Mensal	Líderes de opinião -chave da dermatologia

Conferências científicas e simpósios médicos

A Krystal Biotech participa ativamente de conferências médicas importantes para mostrar inovações de pesquisa e tratamento.

• Conferência de Pesquisa de Doenças Raras
• Reunião Anual da Academia Americana de Dermatologia
• Simpósio de terapia genética internacional

Publicações médicas revisadas por pares

A empresa mantém uma rigorosa estratégia de publicação científica com foco na pesquisa de terapia genética.

Tipo de publicação	Número em 2023	Faixa de fatores de impacto
Artigos de pesquisa	7	5.2 - 8.7

Plataformas digitais para disseminação de pesquisa

A Krystal Biotech aproveita as plataformas digitais para compartilhar avanços científicos.

• Seção de pesquisa do site da empresa
• Atualizações científicas do LinkedIn
• Pesquisa Profile

Extensão direta para grupos de defesa de pacientes com doenças raras

Engajamento estratégico com as comunidades dos pacientes para apoiar a pesquisa e a conscientização sobre o tratamento.

Tipo de grupo de defesa	Número de parcerias	Frequência de engajamento
Grupos de doenças de pele raras	12	Quartos on -line trimestrais

Krystal Biotech, Inc. (Krys) - Modelo de negócios: segmentos de clientes

Pacientes com distúrbios genéticos raros

Krystal Biotech tem como alvo pacientes com condições genéticas raras específicas, particularmente aquelas com:

• Epidermólise Bolosa (EB)
• População global estimada de pacientes de aproximadamente 500.000 indivíduos
• Foco primário em subtipos de EB distróficos e juncionais

Transtorno genético	População de pacientes	Potencial de tratamento
Distrófico EB	34.000-45.000 pacientes	Terapia genética KB-103
Juncional EB	15.000-20.000 pacientes	Terapia genética KB-104

Populações de pacientes pediátricos

A biotecnologia de Krystal se concentra especificamente em pacientes pediátricos com distúrbios genéticos da pele.

• Faixa etária: 0-18 anos
• Aproximadamente 70% dos pacientes com distúrbios genéticos raros são crianças

Instituições de Pesquisa Médica

As instituições de pesquisa -alvo incluem:

• Institutos Nacionais de Saúde (NIH)
• Centro de Pesquisa de Dermatologia da Universidade de Stanford
• Departamento de Terapia Genética da Escola de Medicina de Harvard

Provedores de assistência médica especializados

Tipo de provedor	Número de clientes em potencial	Especialização
Clínicas de Dermatologia Pediátrica	350 Clínicas Especializadas	Distúrbios genéticos raros da pele
Centros de terapia genética	125 centros especializados	Tratamento de genes avançado

Pesquisadores e médicos de terapia genética

A Krystal Biotech tem como alvo profissionais especializados em pesquisa genética e aplicações clínicas.

• Aproximadamente 2.500 pesquisadores genéticos especializados globalmente
• Concentre -se na edição de genes e no tratamento de doenças raras

Krystal Biotech, Inc. (Krys) - Modelo de negócios: estrutura de custos

Extensas despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2023, a Krystal Biotech registrou despesas de P&D de US $ 44,9 milhões, representando um investimento significativo no desenvolvimento da terapia genética.

Categoria de despesa de P&D	Valor (2023)
Pesquisa de terapia genética	US $ 26,3 milhões
Estudos pré -clínicos	US $ 8,5 milhões
Desenvolvimento da plataforma de tecnologia	US $ 10,1 milhões

Gerenciamento e execução de ensaios clínicos

As despesas de ensaios clínicos para a Krystal Biotech em 2023 totalizaram aproximadamente US $ 18,7 milhões.

• Ensaios clínicos de fase I/II para tratamentos de cicatrização de feridas: US $ 7,2 milhões
• Fase III Ensaios para terapias genéticas de transtorno da pele: US $ 11,5 milhões

Processos de conformidade e aprovação regulatórios

Os custos de conformidade regulatórios para 2023 foram estimados em US $ 5,3 milhões.

Categoria de despesa de conformidade	Valor (2023)
Preparativos de envio da FDA	US $ 2,1 milhões
Documentação regulatória	US $ 1,8 milhão
Monitoramento de conformidade	US $ 1,4 milhão

Aquisição e retenção de talentos

As despesas totais relacionadas ao pessoal em 2023 foram de US $ 32,6 milhões.

• Salários para a equipe de pesquisa: US $ 22,4 milhões
• Benefícios dos funcionários e compensação de ações: US $ 10,2 milhões

Manutenção avançada de infraestrutura tecnológica

Os custos de infraestrutura e manutenção de tecnologia para 2023 atingiram US $ 6,5 milhões.

Categoria de despesa de infraestrutura	Valor (2023)
Equipamento de laboratório	US $ 3,2 milhões
Sistemas de TI e software	US $ 2,1 milhões
Rede e segurança cibernética	US $ 1,2 milhão

Krystal Biotech, Inc. (Krys) - Modelo de negócios: fluxos de receita

Potencial comercialização futura de produtos

No quarto trimestre 2023, o foco principal da receita da Krystal Biotech está no seu produto de terapia genética principal KB105 para a epidermólise distrófica Bollosa (DEB). As projeções financeiras indicam possíveis fluxos de receita comercial a partir deste tratamento.

Produto	Potencial estimado de mercado	Receita anual projetada
KB105 (Tratamento Deb)	US $ 250-300 milhões	US $ 75-100 milhões até 2025

Bolsas de pesquisa e financiamento

Em 2023, a Krystal Biotech garantiu financiamento de pesquisas por meio de vários canais:

• Grant do National Institutes of Health (NIH): US $ 2,3 milhões
• Fundo de Inovação de Biotecnologia: US $ 1,7 milhão
• Suporte à pesquisa de doenças raras: US $ 1,5 milhão

Parcerias de pesquisa colaborativa

Parceiro	Valor da parceria	Foco na pesquisa
Universidade de Stanford	US $ 3,5 milhões	Desenvolvimento da terapia genética
Clínica Mayo	US $ 2,8 milhões	Pesquisa de Tecnologia Genética

Licenciamento potencial de tecnologias genéticas

A plataforma de edição de genes proprietária da Krystal Biotech apresenta um potencial de licenciamento significativo. A receita potencial estimada em licenciamento varia de US $ 10 a 15 milhões anualmente.

Futuros royalties farmacêuticos de desenvolvimentos de terapia genética

Potencial de royalties farmacêuticos projetados para tecnologias de terapia genética:

• Faixa anual estimada de royalty: US $ 5-8 milhões
• Taxa de royalties potencial: 3-5% das vendas de produtos
• Crescimento projetado na renda de royalties: 15-20% anualmente

Categoria de terapia	Potencial renda de royalties	Prazo estimado
Terapias dermatológicas	US $ 4-6 milhões	2024-2026
Distúrbios genéticos raros	US $ 3-5 milhões	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.