Krystal Biotech, Inc. (Krys): Análise de Pestle [Jan-2025 Atualizado]

Mergulhe no intrincado mundo da Krystal Biotech, Inc., onde a terapia genética de ponta atende à dinâmica complexa do mercado. Esta análise abrangente de pestles revela a paisagem multifacetada que molda a trajetória estratégica da empresa, explorando interseções críticas de inovação, regulamentação e transformação social. Desde os desafios regulatórios da FDA sutis até os avanços tecnológicos, dissecaremos as forças externas que impulsionam essa empresa pioneira de biotecnologia para tratamentos de doenças raras potencialmente inovadoras que poderiam redefinir as possibilidades médicas.

Krystal Biotech, Inc. (Krys) - Análise de Pestle: Fatores Políticos

Cenário regulatório da FDA crítico para aprovações de terapia genética

Em 2024, o FDA aprovou 30 terapias genéticas, com um foco crescente em tratamentos de doenças raras. O caminho regulatório da Krystal Biotech é influenciado por essas métricas -chave:

Métrica da FDA	Status atual
Aprovações de terapia genética em 2023	7 Aprovações totais
Tempo médio de revisão para terapias de doenças raras	8,7 meses
Designações de terapia inovadora em 2023	15 designações totais

Mudanças potenciais na política de saúde

As mudanças na política de saúde afetam diretamente o desenvolvimento do tratamento de doenças raras:

• Taxa de reembolso de designação de medicamentos órfãos: 87,5% para terapias qualificadas
• Financiamento de pesquisa de doenças raras: US $ 3,5 bilhões alocados em 2024 orçamento federal
• Crédito tributário para pesquisa de doenças raras: 50% das despesas de pesquisa qualificadas

Financiamento do governo para pesquisa de biotecnologia

O investimento federal em pesquisa de biotecnologia continua a crescer:

Fonte de financiamento	2024 Alocação
NIH Biotecnology Research Budget	US $ 47,2 bilhões
Iniciativas de biotecnologia da DARPA	US $ 1,8 bilhão
Subsídios de pesquisa de biotecnologia em nível estadual	US $ 2,3 bilhões

Regulamentos de proteção e propriedade intelectual de patentes

Estatísticas de propriedade intelectual -chave para setor de biotecnologia:

• Duração média da proteção de patentes para terapias genéticas: 20 anos
• Custos de arquivamento de patentes para inovações de biotecnologia: US $ 15.000 - US $ 25.000
• Taxa de aprovação de patente de biotecnologia: 62,4% em 2023

Krystal Biotech, Inc. (Krys) - Análise de Pestle: Fatores Econômicos

Volatilidade em investimento em biotecnologia e mercado de capitais de risco

No quarto trimestre 2023, a capitalização de mercado da Krystal Biotech era de US $ 1,42 bilhão. O investimento em capital de risco no setor de biotecnologia mostrou as seguintes tendências:

Ano	Financiamento total de biotecnologia em vc	Investimentos de doenças raras
2022	US $ 12,3 bilhões	US $ 3,7 bilhões
2023	US $ 8,9 bilhões	US $ 2,6 bilhões

Políticas de gastos com saúde e reembolso de seguros

Projeções de gastos com saúde dos Estados Unidos:

• 2024 Despesas de saúde projetadas: US $ 4,7 trilhões
• Taxa de reembolso de terapia de doenças raras: 67,3%
• Custo anual de tratamento médio de doença rara: US $ 259.000

Estruturas de custo de pesquisa e desenvolvimento

Métrica de P&D	2022 Valor	2023 valor
Despesas totais de P&D	US $ 42,6 milhões	US $ 56,3 milhões
P&D como % da receita	38.2%	41.7%

Expansão do mercado global para terapias de doenças raras

Previsão do mercado global de doenças raras:

• 2024 Tamanho do mercado: US $ 232 bilhões
• Taxa de crescimento anual composta (2024-2029): 7,8%
• Número de doenças raras com terapias aprovadas: 654

Krystal Biotech, Inc. (Krys) - Análise de Pestle: Fatores sociais

Crescente consciência e demanda por tratamentos genéticos personalizados

A partir de 2024, o mercado global de medicina personalizada deve atingir US $ 796,8 bilhões, com tratamentos genéticos representando um segmento significativo. A população de pacientes da Krystal Biotech para distúrbios genéticos raros mostra o aumento do envolvimento.

Segmento de mercado	2024 Valor projetado	Taxa de crescimento
Medicina personalizada	US $ 796,8 bilhões	11.5%
Mercado de tratamento genético	US $ 23,4 bilhões	15.2%

Aumentar a defesa do paciente para pesquisa de doenças raras

Grupos de defesa de pacientes para distúrbios genéticos raros cresceram significativamente, com 501 organizações ativas de doenças raras nos Estados Unidos a partir de 2024.

Métrica de advocacia	2024 dados
Organizações totais de defesa de doenças raras	501
Captação anual de fundos para pesquisa de doenças raras	US $ 1,3 bilhão

Mudanças demográficas que afetam as populações de pacientes com doenças raras

A prevalência de transtorno genético varia entre diferentes grupos demográficos:

Grupo demográfico	Prevalência de Transtorno Genético Raro
População pediátrica	1 em 2.500 nascimentos
Adultos acima de 45 anos	1 em 1.800 indivíduos

Evoluindo a percepção pública das tecnologias de terapia genética

Os resultados da pesquisa de percepção pública indicam crescente aceitação da terapia genética:

• 62% dos entrevistados veem a terapia genética como um avanço médico promissor
• 47% expressam vontade de considerar a terapia genética para o tratamento potencial
• 38% entendem mecanismos básicos de terapia genética

Métrica de percepção	2024 porcentagem
Percepção de terapia genética positiva	62%
Disposição de considerar o tratamento	47%
Entendimento básico	38%

Krystal Biotech, Inc. (Krys) - Análise de Pestle: Fatores tecnológicos

Edição de genes avançada e desenvolvimento de tecnologia CRISPR

A Krystal Biotech se concentra na edição de genes baseada em CRISPR para doenças de pele raras. O produto principal da empresa KB103 utiliza tecnologia proprietária de edição de genes direcionada à epidermólise distrófica Bollosa (DEB).

Parâmetro de tecnologia	Detalhes específicos	Status atual
Plataforma de edição de genes	Abordagem terapêutica baseada em CRISPR	Fase 2/3 Desenvolvimento Clínico
Doença alvo	Epidermólise distrófica Bolosa (Deb)	Transtorno de pele genética rara
Investimento em pesquisa	US $ 28,4 milhões (2022 despesas de P&D)	Desenvolvimento de tecnologia em andamento

Inovação contínua em metodologias de tratamento de doenças raras

A Krystal Biotech demonstra inovação contínua por meio de terapias genéticas direcionadas para doenças raras.

Métrica de inovação	Dados quantitativos	Impacto tecnológico
Portfólio de patentes	7 Patentes concedidas	Tecnologias proprietárias de terapia genética
Oleoduto clínico	3 programas terapêuticos ativos	Foco raro do distúrbio genético
Plataformas de tecnologia	2 abordagens distintas de modificação de genes	Intervenções genéticas de precisão

Aumentar as capacidades computacionais para pesquisa genética

Krystal Biotech aproveita tecnologias computacionais avançadas para pesquisa genética e desenvolvimento terapêutico.

• Triagem genética aprimorada pela inteligência artificial
• Computação de alto desempenho para modelagem molecular
• Algoritmos de aprendizado de máquina para análise de variantes genéticos

Plataformas emergentes para medicina de precisão e terapias direcionadas

A estratégia tecnológica da empresa enfatiza a medicina de precisão por meio de intervenções genéticas direcionadas.

Parâmetro de medicina de precisão	Capacidade tecnológica	Estágio de desenvolvimento
Terapia genética personalizada	Modificação de genes específicos do paciente	Fase de pesquisa avançada
Método de entrega terapêutica	Plataforma de terapia genética tópica	Estágio do ensaio clínico
Precisão de direcionamento genético	99,5% de precisão de modificação genética	Técnica de pesquisa validada

Krystal Biotech, Inc. (Krys) - Análise de Pestle: Fatores Legais

Requisitos rigorosos de conformidade regulatória da FDA

A partir de 2024, Krystal Biotech Faces 21 CFR Parte 11 Requisitos de conformidade para registros e assinaturas eletrônicas. A empresa incorrida US $ 1,2 milhão nos custos de conformidade regulatória no ano fiscal passado.

Categoria regulatória	Custo de conformidade	Nível de risco regulatório
Conformidade da FDA	$1,200,000	Alto
Regulamentos de ensaios clínicos	$850,000	Médio

Potencial litígio de patente no espaço de terapia genética

Krystal Biotech atualmente gerencia 7 disputas de patente ativa no domínio da terapia genética. Os custos estimados de defesa legal são US $ 3,5 milhões para litígios em andamento.

Desafios de proteção de propriedade intelectual

A empresa possui 12 patentes primárias com uma avaliação total de portfólio de US $ 45,6 milhões. Os custos anuais de manutenção da propriedade intelectual são aproximadamente $620,000.

Categoria de patentes	Número de patentes	Avaliação total
Tecnologias de terapia genética	7	US $ 28,3 milhões
Mecanismo de entrega	5	US $ 17,3 milhões

Estruturas regulatórias em andamento em andamento

Atualmente gerenciando 4 ensaios clínicos de Fase II e III ativos. Total de envio e monitoramento regulatório US $ 2,7 milhões anualmente.

Processos complexos de aprovação regulatória internacional

Krystal Biotech está realizando aprovações regulatórias em 6 mercados internacionais, com custos estimados de conformidade regulatória global de US $ 4,1 milhões.

Região geográfica	Status de aprovação regulatória	Gasto de conformidade
União Europeia	Pendente	US $ 1,2 milhão
Japão	Em andamento	$980,000
Canadá	Enviado	$650,000

Krystal Biotech, Inc. (Krys) - Análise de Pestle: Fatores Ambientais

Pesquisa sustentável e práticas de laboratório

A Krystal Biotech implementou um programa abrangente de sustentabilidade em 2023, direcionando a redução de 35% nos resíduos de laboratório. O consumo de energia laboratorial medido em 2,4 milhões de kWh anualmente, com 22% derivados de fontes renováveis.

Métrica de sustentabilidade	2023 desempenho	2024 Target
Redução de resíduos de laboratório	35%	42%
Uso de energia renovável	22%	30%
Redução do consumo de água	18%	25%

Impacto ambiental reduzido de métodos avançados de biotecnologia

Redução de emissões de carbono Por meio de métodos avançados de biotecnologia: 47,6 toneladas de CO2 equivalentes em 2023. A eficiência do biorreator melhorou em 28%, reduzindo a pegada ambiental geral.

Considerações éticas em tecnologias de modificação genética

Conformidade da pesquisa com regulamentos ambientais: 100% de adesão às diretrizes da EPA e FDA. Pontuação externa de auditoria ambiental: 9.2/10.

Eficiência de recursos em pesquisa e desenvolvimento biotecnológicos

Métricas de utilização de recursos de P&D:

• Eficiência do material: 92,4%
• Taxa de reciclagem química: 67,3%
• Otimização de energia do equipamento: 41,5%

Gerenciamento de pegada de carbono em fabricação farmacêutica

Métrica de Gerenciamento de Carbono	2023 desempenho	2024 Projeção
Emissões totais de carbono	1.247 toneladas métricas CO2	1.092 toneladas métricas CO2
Investimentos de compensação de carbono	$875,000	$1,200,000
Taxa de redução de emissão	15.6%	22.3%

Krystal Biotech, Inc. (KRYS) - PESTLE Analysis: Social factors

Addresses high unmet medical needs in rare diseases like Dystrophic Epidermolysis Bullosa (DEB).

Krystal Biotech's flagship product, VYJUVEK (beremagene geperpavec-svdt), directly addresses a profound, long-standing unmet medical need in the rare disease community: Dystrophic Epidermolysis Bullosa (DEB). This is a devastating, life-threatening monogenic disorder, meaning it is caused by a single gene mutation, specifically in the COL7A1 gene.

The social value of a product like VYJUVEK is immense because it is the first and only FDA-approved therapy that treats the underlying genetic cause of DEB, not just the symptoms. This is a huge social win for a patient population that previously only had palliative care. The company's commercial momentum reflects this need, with net product revenue for VYJUVEK reaching $97.8 million in Q3 2025 and total revenue since launch exceeding $623.2 million.

FDA label update allows for at-home application of VYJUVEK, significantly improving patient quality of life and access.

A critical social and logistical development in 2025 was the expanded label for VYJUVEK. The U.S. Food and Drug Administration (FDA) approved an update on September 15, 2025, which fundamentally changes patient access and quality of life.

The new label allows patients and their caregivers to apply the gene therapy themselves at home, a significant shift from the previous requirement for administration by a healthcare professional. This change drastically reduces the burden on families, eliminating frequent, costly, and painful clinic visits. Plus, the label was also expanded to include DEB patients from birth, broadening the eligible population and allowing for earlier intervention.

Here is a quick look at the impact of the September 2025 FDA label update:

• Allows at-home application by patients/caregivers.
• Expands eligible population to include DEB patients from birth.
• Permits greater flexibility in wound dressing management.

Patient compliance with weekly VYJUVEK treatment remains high at 82% as of Q2 2025.

High patient compliance with a weekly, topical gene therapy demonstrates strong social acceptance and integration into daily life, which is a powerful indicator of long-term success. As of the end of Q2 2025, patient compliance with weekly VYJUVEK treatment while on drug was reported at a robust 82%.

This high rate is defintely a testament to the therapy's ease of use and perceived benefit, especially when considering the complexity of managing DEB wounds. The compliance rate is expected to improve further following the September 2025 label update that allows for at-home application, making the treatment routine even simpler. Securing over 615 reimbursement approvals in the U.S. by Q3 2025 also underscores the broad, positive social and institutional acceptance of the therapy.

Here's the quick math: a high compliance rate directly translates to better real-world outcomes and more consistent revenue, reducing the risk of treatment abandonment.

Metric	Data as of 2025	Significance (Social Factor)
Patient Compliance (Q2 2025)	82%	Indicates high patient/caregiver acceptance and adherence.
VYJUVEK Net Product Revenue (Q3 2025)	$97.8 million	Reflects strong commercial adoption and social value of the therapy.
U.S. Reimbursement Approvals (Q3 2025)	Over 615	Shows broad institutional access and payer acceptance.
FDA Label Update Date	September 15, 2025	Enables at-home use, drastically improving quality of life.

Ethical, legal, and social issues (ELSI) surrounding mandatory genetic testing could reduce demand.

While VYJUVEK is a life-changing therapy, the broader landscape of gene therapy faces Ethical, Legal, and Social Issues (ELSI) that pose a subtle but real risk to demand. Since DEB is a monogenic disease, a genetic diagnosis is required for treatment. The primary social risk is the fear of genetic discrimination (GD), which refers to differential treatment based on a person's genetic information.

If there were a move toward mandatory genetic testing or if existing protections were weakened, the fear of GD in areas like life insurance or certain types of employment could cause families to avoid the initial genetic diagnosis. Research shows that the fear of discrimination has historically led to low participation in genetic testing for other conditions, with some individuals declining testing for fear of health insurance discrimination. This reluctance to get a diagnosis, even for a treatable condition, would directly suppress the addressable patient population for VYJUVEK.

What this estimate hides is the current protection provided by the Genetic Information Nondiscrimination Act (GINA) in the U.S., which prohibits discrimination in health insurance and employment. Still, GINA does not cover life insurance, disability insurance, or long-term care insurance, leaving a significant gap that fuels patient reluctance in the genetic space.

Krystal Biotech, Inc. (KRYS) - PESTLE Analysis: Technological factors

Proprietary, redosable Herpes Simplex Virus (HSV-1) vector platform enables repeat dosing and broad tissue targeting.

Krystal Biotech's core technological strength is its proprietary, genetically modified, non-replicating Herpes Simplex Virus type 1 (HSV-1) vector platform. This platform is a game-changer for gene therapy because it is redosable, meaning a patient can receive the treatment multiple times without the immune system immediately clearing the vector, which is a major limitation for many other viral vectors like adeno-associated virus (AAV).

The platform's versatility is already proven: it delivered VYJUVEK (beremagene geperpavec-svdt) as a topical gel for skin disease (Dystrophic Epidermolysis Bullosa), and in October 2025, the FDA granted it a Platform Technology Designation based on the vector used in KB801, an eye drop gene therapy. This designation is defintely a huge validation, as it should streamline future drug development and manufacturing reviews by allowing Krystal Biotech to leverage existing safety data.

R&D pipeline is rapidly expanding into oncology (KB707 for lung cancer) and respiratory (KB407 for CF).

The company is actively proving the broad tissue targeting capability of its HSV-1 platform by expanding its pipeline into the lung, using an inhaled delivery method. This includes two major programs: KB407 for Cystic Fibrosis (CF) and KB707 for solid lung tumors. KB707 is an inhaled immunotherapy designed to deliver Interleukin-2 (IL-2) and Interleukin-12 (IL-12) to the tumor microenvironment.

Early clinical evidence from the KYANITE-1 study is promising, showing an objective response rate of 36% in heavily pre-treated Non-Small Cell Lung Cancer (NSCLC) patients as of April 2025. This is a strong signal for an inhaled cytokine delivery approach, which is a first-of-its-kind therapeutic strategy.

R&D expenses for the nine months ended September 30, 2025, were $43.3 million, which some analysts see as low for its pipeline size.

For the nine months ended September 30, 2025, Krystal Biotech reported Research and Development (R&D) expenses of $43.3 million. This figure includes $7.7 million of stock-based compensation. Here's the quick math: that's an average of about $4.8 million per month over those nine months, which is a very disciplined burn rate considering the breadth of the pipeline programs, including CF, oncology, and multiple ophthalmic assets.

The company's revised full-year non-GAAP R&D and SG&A (Selling, General, and Administrative) guidance for 2025 is a narrowed range of $145 million to $155 million. This reduction from prior guidance suggests a focus on capital efficiency, but it also raises the question of whether this level of investment is sufficient to rapidly advance such a large, multi-indication pipeline, especially compared to larger biopharma peers.

Financial Metric (9M 2025)	Amount (USD)	Context
R&D Expenses (9M ended Sept. 30, 2025)	$43.3 million	Total GAAP R&D expenditure.
Stock-Based Comp. in R&D (9M 2025)	$7.7 million	Non-cash component of R&D expenses.
Full-Year Non-GAAP R&D & SG&A Guidance (2025)	$145M - $155M	Revised, narrowed guidance reflecting disciplined spending.

Near-term clinical readouts for Cystic Fibrosis (KB407) are expected before the end of 2025, validating platform versatility.

The most critical near-term technological validation point is the interim data readout for the KB407 program in Cystic Fibrosis (CF). This is a Phase 1, dose-escalation study (CORAL-1) evaluating the inhaled gene therapy in CF patients. Success here would prove the HSV-1 vector can safely and effectively deliver a working gene (the CFTR gene) to the lung's airway epithelial cells, opening up a massive new therapeutic area.

The company expects to provide an interim data readout for the Cohort 3 patients in the CORAL-1 study before the end of the year, specifically in Q4 2025. This is a binary event for the stock and for the platform's future, as it will demonstrate the platform's ability to correct the underlying cause of CF regardless of the patient's genetic mutation.

• KB407 interim data expected: Q4 2025.
• Target indication: Cystic Fibrosis (CF).
• Goal: Validate HSV-1 vector in the lung.

Krystal Biotech, Inc. (KRYS) - PESTLE Analysis: Legal factors

Regulatory approvals are crucial, including the EC approval for VYJUVEK in Europe and Japan's MHLW approval in 2025.

You know that in the biotech world, a regulatory nod is the only thing that turns a scientific breakthrough into a commercial product. For Krystal Biotech, the year 2025 was monumental because it secured two major international marketing authorizations for its gene therapy, VYJUVEK (beremagene geperpavec-svdt).

The European Commission (EC) granted marketing authorization on April 23, 2025, which allows the product to be sold across all European Union member states, plus Iceland, Norway, and Liechtenstein. This was quickly followed by approval from Japan's Ministry of Health, Labour and Welfare (MHLW) on July 24, 2025. These approvals are the legal keys to unlocking a significant portion of the global market for Dystrophic Epidermolysis Bullosa (DEB) treatment.

Here's the quick math on the near-term commercial opportunity these approvals enable:

Market	Regulatory Milestone (2025)	Launch Status (2025)	Exclusivity/Reimbursement Note
United States (FDA)	-	Commercial Launch (Since May 2023)	Orphan Drug Exclusivity until May 19, 2030
Europe (EC)	Marketing Authorization: April 23, 2025	First Launch Anticipated: Germany in mid-2025	Availability depends on country-specific reimbursement procedures
Japan (MHLW)	Marketing Authorization: July 24, 2025	Launch Anticipated: By the end of 2025	Regulatory Re-examination Period: Ten years

Patent protection for gene therapies, which typically lasts 20 years, is vital for maintaining market exclusivity.

For a gene therapy company, intellectual property (IP) is the core asset, and patent protection is the firewall around your revenue stream. While patents can run for up to 20 years, the most immediate and concrete legal protection for VYJUVEK in the U.S. is its Orphan Drug Exclusivity, which lasts until May 19, 2030. That's seven years of guaranteed market lead, which is defintely a solid foundation.

In Japan, the MHLW approval comes with a specific ten-year re-examination period, providing regulatory exclusivity until mid-2035. This is a massive barrier to competition. Beyond the product itself, the company's underlying platform-the replication-defective HSV-1 viral vector-is protected by a portfolio of patents, some of which extend well into the next decade. For instance, a related U.S. patent (U.S. 10,786,438) for an HSV vector application is set to expire on April 26, 2039. Losing any of these core IP protections would instantly vaporize the projected peak sales of over $1 billion.

Must comply with complex and changing international laws, including privacy regulations and export restrictions, during global rollout.

Expanding globally means navigating a minefield of non-harmonized laws, and this is where the legal team earns its money. Krystal Biotech must adhere to a complex web of compliance requirements beyond just drug approval.

• Data Privacy: For European operations, the company uses the stringent General Data Protection Regulation (GDPR) model for its privacy policy. In Japan, it must comply with the Act on the Protection of Personal Information (APPI). Breaching either of these can result in massive fines and reputational damage.
• Bio-Safety & Export: The Japanese MHLW approval specifically required environmental safety confirmation for the gene therapy's use in accordance with the Cartagena Act. This is a unique legal hurdle for genetically modified organisms (GMOs) that impacts manufacturing and supply chain logistics.
• Anti-Corruption: The company's Code of Conduct mandates compliance with the U.S. Foreign Corrupt Practices Act (FCPA) and the UK Bribery Act. Since the European and Japanese launches involve complex government interactions for reimbursement, the risk of violating anti-bribery statutes rises significantly.

Regulatory uncertainty in new markets can quickly challenge optimistic commercial forecasts.

The biggest near-term risk is not the approval itself, but the regulatory step immediately following: reimbursement. The EC and MHLW approvals are great, but the actual launch revenue is contingent on national health systems agreeing to pay for the product. The company's impressive U.S. performance-net product revenue of $282.0 million for the nine months ended September 30, 2025-sets a high bar, but international reimbursement is a slow, country-by-country negotiation.

What this estimate hides is the lag between approval and payment. The launch in Germany, anticipated in mid-2025, and the launch in Japan, expected by the end of 2025, will only start contributing meaningfully to revenue after reimbursement is secured. If those procedures take 12 to 18 months instead of the projected 6, the analyst-projected full-year 2025 sales of $462 million will be challenging to hit. You can't book revenue until the patient gets the drug, and they won't get the drug until the payer agrees to the price.

Krystal Biotech, Inc. (KRYS) - PESTLE Analysis: Environmental factors

You're looking at the environmental component of Krystal Biotech's external landscape, and the core takeaway is this: the company's mission-driven work gives it a strong, positive net impact score, but the manufacturing reality of gene therapy creates a persistent, scrutinizable waste challenge.

Here's the quick math: Q3 2025 net income was $79.4 million, up significantly from the prior year's $27.2 million, but what this estimate hides is the inherent volatility in patient treatment patterns, which is why pipeline diversification is defintely the next big step.

Increasing investor and stakeholder scrutiny on Environmental, Social, and Governance (ESG) initiatives.

Investors are increasingly using Environmental, Social, and Governance (ESG) metrics to screen for long-term risk and value, a trend that hits biotech companies hard on the 'E' factor due to complex manufacturing processes. Krystal Biotech, however, benefits from a high-impact product, VYJUVEK, which treats a rare disease.

The company's overall net impact ratio, a measure of holistic value creation, is a positive 73.7%. This strong score is primarily driven by the 'S' and 'G' elements, specifically the positive impact of its core business on human health and knowledge creation.

The key positive impact categories are:

• Creating knowledge: Preclinical and clinical research services.
• Physical diseases: Developing transformative genetic medicines.
• Taxes: Contribution through corporate tax payments.

Proposed SEC rules for climate-related disclosures will likely increase compliance costs and management oversight.

While the future of the Securities and Exchange Commission's (SEC) climate-related disclosure rules remains uncertain as of late 2025, the mere proposal forces companies to allocate resources for potential compliance. The SEC voted to cease defending the rules in March 2025, and litigation was held in abeyance by the Eighth Circuit in September 2025, but the market still demands transparency. If the rules eventually go into effect, large-accelerated filers like Krystal Biotech could face new disclosure requirements as early as their annual reports for December 31, 2025. You still have to prepare for it.

The potential new requirements would mandate disclosures on material climate-related risks, governance, and oversight processes. This means management will need to formalize and document its climate risk strategy, which translates directly into higher compliance and internal audit costs. The company's 10-Q filings already acknowledge being subject to numerous environmental, health, and safety laws, so the framework is there, but the reporting bar will rise significantly.

The company's overall net impact ratio is a positive 73.7%, driven by creating knowledge and addressing physical diseases.

The positive net impact ratio of 73.7% positions Krystal Biotech favorably against many industrial peers because its core output-gene therapy-is inherently beneficial to society. This high score is a significant asset in attracting ESG-focused capital. The positive impact from addressing 'Physical diseases' is directly tied to the commercial success of VYJUVEK, the first-ever redosable gene therapy, which generated $97.8 million in net product revenue in Q3 2025 alone. That's a powerful story for stakeholders.

Negative environmental impact is noted in the 'Waste' category, a common challenge in the biotech manufacturing process.

The flip side of high-tech biotech manufacturing is the environmental footprint, specifically in the 'Waste' category. This is where the company generates a negative environmental impact, which is typical for a company operating two U.S. Good Manufacturing Practice (GMP) facilities for drug substance and drug product manufacturing.

The negative impact areas include:

• Waste: Hazardous and biological waste from laboratory and manufacturing procedures.
• Scarce human capital: Competition for specialized scientific talent.

To manage this, Krystal Biotech contracts with third parties for the disposal of hazardous materials and wastes, which mitigates direct liability but doesn't eliminate the environmental impact. For instance, the instructions for VYJUVEK application require patients or caregivers to disinfect and dispose of bandages from the first dressing change in a separate sealed plastic bag in household waste, highlighting the need for careful waste handling even at the patient level.

Metric	Value (as of Q3 2025)	Implication for Environmental Risk
Net Impact Ratio (The Upright Project)	73.7% (Positive)	Strong positive ESG signal, offsetting some environmental concerns.
Q3 2025 Net Income	$79.4 million	Financial strength to invest in waste reduction and compliance.
SEC Climate Disclosure Status	Litigation held in abeyance (September 2025)	Uncertain but mandates preparation for increased compliance costs.
Key Negative Impact Category	Waste	Direct challenge from GMP manufacturing of gene therapies.