Krystal Biotech, Inc. (KRYS): Análisis PESTLE [Actualizado en enero de 2025]

Sumérgete en el intrincado mundo de Krystal Biotech, Inc., donde la terapia génica de vanguardia cumple con la dinámica compleja del mercado. Este análisis integral de mano presenta el panorama multifacético que da forma a la trayectoria estratégica de la compañía, explorando intersecciones críticas de innovación, regulación y transformación social. Desde los desafíos regulatorios de la FDA matizados hasta avances tecnológicos innovadores, diseccionaremos las fuerzas externas que impulsaremos a esta empresa de biotecnología pionera hacia tratamientos de enfermedades raras potencialmente innovadores que podrían redefinir las posibilidades médicas.

Krystal Biotech, Inc. (Krys) - Análisis de mortero: factores políticos

Landscape regulatorio de la FDA crítico para aprobaciones de terapia génica

A partir de 2024, la FDA ha aprobado 30 terapias genéticas, con un enfoque creciente en tratamientos de enfermedades raras. La vía regulatoria de Krystal Biotech está influenciada por estas métricas clave:

Métrica de la FDA	Estado actual
Aprobaciones de terapia génica en 2023	7 aprobaciones totales
Tiempo de revisión promedio para terapias de enfermedades raras	8.7 meses
Designaciones de terapia innovadora en 2023	15 designaciones totales

Posibles cambios en la política de atención médica

Los cambios en la política de atención médica afectan directamente el desarrollo del tratamiento de enfermedades raras:

• Tasa de reembolso de designación de medicamentos huérfanos: 87.5% para terapias calificadas
• Financiación de la investigación de enfermedades raras: $ 3.5 mil millones asignados en 2024 Presupuesto federal
• Crédito fiscal para la investigación de enfermedades raras: 50% de los gastos de investigación calificados

Financiación del gobierno para la investigación de biotecnología

La inversión federal en investigación en biotecnología continúa creciendo:

Fuente de financiación	Asignación 2024
Presupuesto de investigación de biotecnología de NIH	$ 47.2 mil millones
Iniciativas de Biotecnología DARPA	$ 1.8 mil millones
Subvenciones de investigación de biotecnología a nivel estatal	$ 2.3 mil millones

Regulaciones de protección de patentes y propiedad intelectual

Estadísticas clave de propiedad intelectual para el sector de biotecnología:

• Duración promedio de protección de patentes para terapias génicas: 20 años
• Costos de presentación de patentes para innovaciones de biotecnología: $ 15,000 - $ 25,000
• Tasa de aprobación de la patente de biotecnología: 62.4% en 2023

Krystal Biotech, Inc. (Krys) - Análisis de mortero: factores económicos

Volatilidad en la inversión en biotecnología y los mercados de capital de riesgo

A partir del cuarto trimestre de 2023, la capitalización de mercado de Krystal Biotech fue de $ 1.42 mil millones. La inversión de capital de riesgo en el sector de biotecnología mostró las siguientes tendencias:

Año	Financiación total de Biotech VC	Inversiones de enfermedades raras
2022	$ 12.3 mil millones	$ 3.7 mil millones
2023	$ 8.9 mil millones	$ 2.6 mil millones

Políticas de gasto en salud y reembolso de seguros

Proyecciones de gastos de atención médica de los Estados Unidos:

• 2024 Gastos de atención médica proyectados: $ 4.7 billones
• Tasa de reembolso de terapia de enfermedades raras: 67.3%
• Tratamiento promedio de enfermedades raras Costo anual: $ 259,000

Estructuras de costos de investigación y desarrollo

I + D Métrica	Valor 2022	Valor 2023
Gastos totales de I + D	$ 42.6 millones	$ 56.3 millones
I + D como % de ingresos	38.2%	41.7%

Expansión del mercado global para terapias de enfermedades raras

Pronóstico global del mercado de enfermedades raras:

• 2024 Tamaño del mercado: $ 232 mil millones
• Tasa de crecimiento anual compuesta (2024-2029): 7.8%
• Número de enfermedades raras con terapias aprobadas: 654

Krystal Biotech, Inc. (Krys) - Análisis de mortero: factores sociales

Creciente conciencia y demanda de tratamientos genéticos personalizados

A partir de 2024, se proyecta que el mercado global de medicina personalizada alcanzará los $ 796.8 mil millones, con tratamientos genéticos que representan un segmento significativo. La población de pacientes de Krystal Biotech para trastornos genéticos raros muestra un compromiso creciente.

Segmento de mercado	2024 Valor proyectado	Índice de crecimiento
Medicina personalizada	$ 796.8 mil millones	11.5%
Mercado de tratamiento genético	$ 23.4 mil millones	15.2%

Aumento de la defensa del paciente para la investigación de enfermedades raras

Los grupos de defensa de los pacientes para trastornos genéticos raros han crecido significativamente, con 501 organizaciones activas de enfermedades raras en los Estados Unidos a partir de 2024.

Métrica de defensa	2024 datos
Organizaciones totales de defensa de la enfermedad rara	501
Recaudación de fondos anual para la investigación de enfermedades raras	$ 1.3 mil millones

Cambios demográficos que afectan a las poblaciones de pacientes con enfermedades raras

La prevalencia del trastorno genético varía en diferentes grupos demográficos:

Grupo demográfico	Prevalencia de trastorno genético raro
Población pediátrica	1 en 2.500 nacimientos
Adultos mayores de 45	1 en 1.800 personas

Percepción pública en evolución de las tecnologías de terapia génica

Los resultados de la encuesta de percepción pública indican una aceptación creciente de la terapia génica:

• El 62% de los encuestados ven la terapia génica como un avance médico prometedor
• El 47% expresa su disposición a considerar la terapia génica para un tratamiento potencial
• 38% Comprender los mecanismos básicos de terapia génica

Métrica de percepción	2024 porcentaje
Percepción de terapia génica positiva	62%
Voluntad de considerar el tratamiento	47%
Comprensión básica	38%

Krystal Biotech, Inc. (Krys) - Análisis de mortero: factores tecnológicos

Edición de genes avanzados y desarrollo de tecnología CRISPR

Krystal Biotech se centra en la edición de genes basada en CRISPR para enfermedades de la piel raras. El producto principal de la compañía KB103 utiliza tecnología de edición de genes patentada dirigida a la epidermólisis bullosa (DEB).

Parámetro tecnológico	Detalles específicos	Estado actual
Plataforma de edición de genes	Enfoque terapéutico basado en CRISPR	Fase 2/3 Desarrollo clínico
Enfermedad objetivo	Epidermólisis distrófica bullosa (DEB)	Trastorno genético de la piel raro
Inversión de investigación	$ 28.4 millones (gastos de I + D 2022)	Desarrollo de tecnología en curso

Innovación continua en metodologías de tratamiento de enfermedades raras

Krystal Biotech demuestra innovación continua a través de terapias genéticas específicas para enfermedades raras.

Métrica de innovación	Datos cuantitativos	Impacto tecnológico
Cartera de patentes	7 patentes otorgadas	Tecnologías de terapia génica patentada
Tubería clínica	3 programas terapéuticos activos	Enfoque raro de trastorno genético
Plataformas tecnológicas	2 enfoques distintos de modificación génica	Intervenciones genéticas de precisión

Aumento de capacidades computacionales para la investigación genética

Krystal Biotech aprovecha las tecnologías computacionales avanzadas para la investigación genética y el desarrollo terapéutico.

• Detección genética mejorada por inteligencia artificial
• Computación de alto rendimiento para modelado molecular
• Algoritmos de aprendizaje automático para el análisis de variantes genéticas

Plataformas emergentes para medicina de precisión y terapias dirigidas

La estrategia tecnológica de la compañía enfatiza la medicina de precisión a través de intervenciones genéticas específicas.

Parámetro de medicina de precisión	Capacidad tecnológica	Etapa de desarrollo
Terapia genética personalizada	Modificación de genes específicos del paciente	Fase de investigación avanzada
Método de entrega terapéutica	Plataforma de terapia génica tópica	Etapa de ensayo clínico
Precisión de orientación genética	99.5% de precisión de modificación genética	Técnica de investigación validada

Krystal Biotech, Inc. (Krys) - Análisis de mortero: factores legales

Requisitos estrictos de cumplimiento regulatorio de la FDA

A partir de 2024, Krystal Biotech se enfrenta 21 CFR Parte 11 Requisitos de cumplimiento para registros electrónicos y firmas. La compañía ha incurrido $ 1.2 millones en costos de cumplimiento regulatorio en el último año fiscal.

Categoría regulatoria	Costo de cumplimiento	Nivel de riesgo regulatorio
Cumplimiento de la FDA	$1,200,000	Alto
Regulaciones de ensayos clínicos	$850,000	Medio

Litigio potencial de patente en el espacio de terapia génica

Krystal Biotech actualmente gestiona 7 disputas de patente activas En el dominio de terapia génica. Los costos estimados de defensa legal son $ 3.5 millones para litigios en curso.

Desafíos de protección de la propiedad intelectual

La compañía posee 12 patentes principales con una valoración total de la cartera de $ 45.6 millones. Los costos anuales de mantenimiento de la propiedad intelectual son aproximadamente $620,000.

Categoría de patente	Número de patentes	Valoración total
Tecnologías de terapia génica	7	$ 28.3 millones
Mecanismo de entrega	5	$ 17.3 millones

Marcos regulatorios de ensayos clínicos en curso

Gestionando actualmente 4 ensayos clínicos activos de fase II y III. Presentación regulatoria y gastos de monitoreo Total $ 2.7 millones anualmente.

Procesos de aprobación regulatoria internacional complejos

Krystal Biotech está buscando aprobaciones regulatorias en 6 mercados internacionales, con costos estimados de cumplimiento regulatorio global de $ 4.1 millones.

Región geográfica	Estado de aprobación regulatoria	Gasto de cumplimiento
unión Europea	Pendiente	$ 1.2 millones
Japón	En curso	$980,000
Canadá	Enviado	$650,000

Krystal Biotech, Inc. (Krys) - Análisis de mortero: factores ambientales

Investigación sostenible y prácticas de laboratorio

Krystal Biotech implementó un programa integral de sostenibilidad en 2023, apuntando al 35% de la reducción de los desechos de laboratorio. El consumo de energía de laboratorio medido en 2.4 millones de kWh anuales, con un 22% derivado de fuentes renovables.

Métrica de sostenibilidad	2023 rendimiento	Objetivo 2024
Reducción de desechos de laboratorio	35%	42%
Uso de energía renovable	22%	30%
Reducción del consumo de agua	18%	25%

Impacto ambiental reducido de los métodos avanzados de biotecnología

Reducción de emisiones de carbono A través de métodos avanzados de biotecnología: 47.6 toneladas métricas CO2 equivalente en 2023. La eficiencia del biorreactor mejoró en un 28%, reduciendo la huella ambiental general.

Consideraciones éticas en tecnologías de modificación genética

Cumplimiento de la investigación con las regulaciones ambientales: 100% de adherencia a las directrices de la EPA y la FDA. Puntuación de auditoría ambiental externa: 9.2/10.

Eficiencia de recursos en investigación y desarrollo biotecnológico

Métricas de utilización de recursos de I + D:

• Eficiencia del material: 92.4%
• Tasa de reciclaje de productos químicos: 67.3%
• Optimización de energía del equipo: 41.5%

Gestión de la huella de carbono en fabricación farmacéutica

Métrica de gestión de carbono	2023 rendimiento	2024 proyección
Emisiones totales de carbono	1.247 toneladas métricas CO2	1.092 toneladas métricas CO2
Inversiones compensadas de carbono	$875,000	$1,200,000
Tasa de reducción de emisiones	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.