Krystal Biotech, Inc. (KRYS): Analyse de Pestle [Jan-2025 Mise à jour]

Plongez dans le monde complexe de Krystal Biotech, Inc., où la thérapie génique de pointe rencontre une dynamique de marché complexe. Cette analyse complète du pilon dévoile le paysage multiforme qui façonne la trajectoire stratégique de l'entreprise, explorant les intersections critiques de l'innovation, de la réglementation et de la transformation sociétale. Des défis réglementaires de la FDA nuancés aux progrès technologiques révolutionnaires, nous disséquerons les forces externes qui propulsent cette entreprise de biotechnologie pionnière vers des traitements de maladies rares potentiellement révolutionnaires qui pourraient redéfinir les possibilités médicales.

Krystal Biotech, Inc. (KRYS) - Analyse du pilon: facteurs politiques

Paysage régulatrice de la FDA critique pour les approbations de la thérapie génique

En 2024, la FDA a approuvé 30 thérapies géniques, en mettant de plus en plus l'accent sur les traitements de maladies rares. La voie réglementaire de Krystal Biotech est influencée par ces mesures clés:

Métrique de la FDA	État actuel
Approbations de la thérapie génique en 2023	7 approbations totales
Temps de revue moyen pour les thérapies par maladies rares	8,7 mois
Désignations de thérapie révolutionnaire en 2023	15 désignations totales

Changements potentiels dans la politique des soins de santé

Les changements de politique de santé ont un impact direct sur le développement du traitement des maladies rares:

• Taux de remboursement de la désignation des médicaments orphelins: 87,5% pour les thérapies qualifiées
• Financement de la recherche sur les maladies rares: 3,5 milliards de dollars alloués au budget fédéral 2024
• Crédit d'impôt pour la recherche sur les maladies rares: 50% des frais de recherche qualifiés

Financement gouvernemental pour la recherche sur la biotechnologie

L'investissement fédéral dans la recherche en biotechnologie continue de croître:

Source de financement	2024 allocation
Budget de recherche en biotechnologie du NIH	47,2 milliards de dollars
Initiatives de biotechnologie DARPA	1,8 milliard de dollars
Subventions de recherche de biotechnologie au niveau de l'État	2,3 milliards de dollars

Règlement sur la protection des brevets et la propriété intellectuelle

Statistiques clés de la propriété intellectuelle pour le secteur de la biotechnologie:

• Durée moyenne de protection des brevets pour les thérapies géniques: 20 ans
• Coûts de dépôt de brevets pour les innovations de biotechnologie: 15 000 $ - 25 000 $
• Taux d'approbation des brevets en biotechnologie: 62,4% en 2023

Krystal Biotech, Inc. (KRYS) - Analyse du pilon: facteurs économiques

Volatilité des marchés d'investissement et de capital-risque biotechnologiques

Au quatrième trimestre 2023, la capitalisation boursière de Krystal Biotech était de 1,42 milliard de dollars. L'investissement en capital-risque dans le secteur de la biotechnologie a montré les tendances suivantes:

Année	Financement total de VC biotechnologique	Investissements de maladies rares
2022	12,3 milliards de dollars	3,7 milliards de dollars
2023	8,9 milliards de dollars	2,6 milliards de dollars

Polices de remboursement des dépenses de santé et d'assurance

Projections de dépenses de santé des États-Unis:

• 2024 dépenses de santé projetées: 4,7 billions de dollars
• Taux de remboursement de la thérapie par maladie rare: 67,3%
• Traitement des maladies rares moyens Coût annuel: 259 000 $

Structures de coûts de recherche et de développement

Métrique de R&D	Valeur 2022	Valeur 2023
Total des dépenses de R&D	42,6 millions de dollars	56,3 millions de dollars
R&D en% des revenus	38.2%	41.7%

Expansion du marché mondial pour les thérapies contre les maladies rares

Prévisions du marché mondial des maladies rares:

• 2024 Taille du marché: 232 milliards de dollars
• Taux de croissance annuel composé (2024-2029): 7,8%
• Nombre de maladies rares avec des thérapies approuvées: 654

Krystal Biotech, Inc. (KRYS) - Analyse du pilon: facteurs sociaux

Conscience et demande croissantes de traitements génétiques personnalisés

En 2024, le marché mondial de la médecine personnalisée devrait atteindre 796,8 milliards de dollars, les traitements génétiques représentant un segment important. La population de patients de Krystal Biotech pour les troubles génétiques rares montre une augmentation de l'engagement.

Segment de marché	2024 Valeur projetée	Taux de croissance
Médecine personnalisée	796,8 milliards de dollars	11.5%
Marché du traitement génétique	23,4 milliards de dollars	15.2%

Augmentation du plaidoyer des patients pour la recherche sur les maladies rares

Les groupes de plaidoyer pour les patients pour les troubles génétiques rares ont augmenté de manière significative, avec 501 organisations de maladies rares actives aux États-Unis en 2024.

Métrique de plaidoyer	2024 données
Organisation totale de défense des maladies rares	501
Collecte de fonds annuelle pour la recherche sur les maladies rares	1,3 milliard de dollars

Chart démographique affectant les populations de patients atteints de maladies rares

La prévalence des troubles génétiques varie selon différents groupes démographiques:

Groupe démographique	Prévalence des troubles génétiques rares
Population pédiatrique	1 naissance sur 2 500
Adultes de plus de 45 ans	1 individus sur 1 800

Évolution de la perception du public des technologies de thérapie génique

Les résultats de l'enquête sur la perception du public indiquent une acceptation croissante de la thérapie génique:

• 62% des répondants considèrent la thérapie génique comme un progrès médical prometteur
• 47% expriment la volonté de considérer la thérapie génique pour un traitement potentiel
• 38% comprennent les mécanismes de base de la thérapie génique de base

Métrique de la perception	2024 pourcentage
Perception positive de la thérapie génique	62%
Volonté de considérer le traitement	47%
Compréhension de base	38%

Krystal Biotech, Inc. (KRYS) - Analyse du pilon: facteurs technologiques

Édition de gènes avancée et développement technologique CRISPR

Krystal Biotech se concentre sur l'édition de gènes basée sur CRISPR pour les maladies cutanées rares. Le produit principal de la société KB103 utilise la technologie de montage de gènes propriétaire ciblant l'épidermolyse dystrophique Bullosa (DEB).

Paramètre technologique	Détails spécifiques	État actuel
Plate-forme d'édition de gènes	Approche thérapeutique basée sur CRISPR	Phase 2/3 Développement clinique
Maladie cible	Épidermolyse dystrophique Bullosa (DEB)	Trouble de la peau génétique rare
Investissement en recherche	28,4 millions de dollars (2022 dépenses de R&D)	Développement technologique continu

Innovation continue dans les méthodologies de traitement des maladies rares

Krystal Biotech démontre l'innovation continue à travers des thérapies génétiques ciblées pour les maladies rares.

Métrique d'innovation	Données quantitatives	Impact technologique
Portefeuille de brevets	7 brevets accordés	Technologies de thérapie génique propriétaire
Pipeline clinique	3 programmes thérapeutiques actifs	Focus de troubles génétiques rares
Plates-formes technologiques	2 approches de modification des gènes distinctes	Interventions génétiques de précision

Augmentation des capacités de calcul pour la recherche génétique

Krystal Biotech exploite les technologies de calcul avancées pour la recherche génétique et le développement thérapeutique.

• Dépistage génétique amélioré de l'intelligence artificielle
• Calcul haute performance pour la modélisation moléculaire
• Algorithmes d'apprentissage automatique pour l'analyse des variantes génétiques

Plates-formes émergentes pour la médecine de précision et les thérapies ciblées

La stratégie technologique de l'entreprise met l'accent sur la médecine de précision grâce à des interventions génétiques ciblées.

Paramètre de médecine de précision	Capacité technologique	Étape de développement
Thérapie génétique personnalisée	Modification du gène spécifique au patient	Phase de recherche avancée
Méthode de livraison thérapeutique	Plateforme de thérapie génique topique	Étape d'essai clinique
Précision de ciblage génétique	Précision de la modification génétique à 99,5%	Technique de recherche validée

Krystal Biotech, Inc. (KRYS) - Analyse du pilon: facteurs juridiques

Exigences strictes de conformité réglementaire de la FDA

En 2024, les visages de biotechnologie de Krystal 21 CFR partie 11 Exigences de conformité pour les dossiers et signatures électroniques. L'entreprise a engagé 1,2 million de dollars dans les frais de conformité réglementaire au cours de l'exercice précédent.

Catégorie de réglementation	Coût de conformité	Niveau de risque réglementaire
Conformité de la FDA	$1,200,000	Haut
Règlement sur les essais cliniques	$850,000	Moyen

Litige potentiel en matière de brevets dans l'espace de thérapie génique

Krystal Biotech gère actuellement 7 différends de brevet actifs Dans le domaine de la thérapie génique. Les frais de défense juridique estimés sont 3,5 millions de dollars pour les litiges en cours.

Défis de protection de la propriété intellectuelle

L'entreprise détient 12 brevets primaires avec une évaluation totale du portefeuille de 45,6 millions de dollars. Les coûts annuels de maintenance de la propriété intellectuelle sont approximativement $620,000.

Catégorie de brevet	Nombre de brevets	Évaluation totale
Technologies de thérapie génique	7	28,3 millions de dollars
Mécanisme de livraison	5	17,3 millions de dollars

Cadres de réglementation des essais cliniques en cours

Gestion actuellement 4 essais cliniques de phase II et III actifs. Total des dépenses de soumission et de surveillance réglementaires 2,7 millions de dollars annuellement.

Processus d'approbation réglementaire internationaux complexes

Krystal Biotech poursuit les approbations réglementaires 6 marchés internationaux, avec les frais de conformité réglementaire mondiaux estimés de 4,1 millions de dollars.

Région géographique	Statut d'approbation réglementaire	Dépenses de conformité
Union européenne	En attente	1,2 million de dollars
Japon	En cours	$980,000
Canada	Soumis	$650,000

Krystal Biotech, Inc. (KRYS) - Analyse du pilon: facteurs environnementaux

Recherche durable et pratiques de laboratoire

Krystal Biotech a mis en œuvre un programme complet de durabilité en 2023, ciblant une réduction de 35% des déchets de laboratoire. La consommation d'énergie de laboratoire mesurée à 2,4 millions de kWh par an, avec 22% dérivée de sources renouvelables.

Métrique de la durabilité	Performance de 2023	Cible 2024
Réduction des déchets de laboratoire	35%	42%
Consommation d'énergie renouvelable	22%	30%
Réduction de la consommation d'eau	18%	25%

Impact environnemental réduit des méthodes de biotechnologie avancées

Réduction des émissions de carbone Grâce à des méthodes avancées de biotechnologie: 47,6 tonnes métriques CO2 équivalent en 2023. L'efficacité du bioréacteur s'est améliorée de 28%, ce qui réduit l'empreinte environnementale globale.

Considérations éthiques dans les technologies de modification génétique

Conformité à la recherche avec les réglementations environnementales: 100% d'adhésion aux directives de l'EPA et de la FDA. Score d'audit environnemental externe: 9.2 / 10.

Efficacité des ressources dans la recherche et le développement biotechnologiques

Métriques d'utilisation des ressources R&D:

• Efficacité des matériaux: 92,4%
• Taux de recyclage chimique: 67,3%
• Optimisation d'énergie de l'équipement: 41,5%

Gestion de l'empreinte carbone dans la fabrication pharmaceutique

Métrique de gestion du carbone	Performance de 2023	2024 projection
Émissions totales de carbone	1 247 tonnes métriques CO2	1 092 tonnes métriques CO2
Investissements de compensation de carbone	$875,000	$1,200,000
Taux de réduction des émissions	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.