Abeona Therapeutics Inc. (ABEO): Business Model Canvas [Jan-2025 Mis à jour]

Abeona Therapeutics Inc. (ABEO) est à l'avant-garde de la thérapie génique révolutionnaire, transformant le paysage des rares traitements de troubles génétiques avec son approche scientifique à la pointe. En tirant parti des plateformes innovantes et des partenariats stratégiques, ce pionnier de la biotechnologie développe des solutions thérapeutiques révolutionnaires qui offrent de l'espoir aux patients pédiatriques et aux familles luttant contre les conditions génétiques complexes. Leur modèle commercial complet représente un plan sophistiqué pour l'innovation médicale, le mélange de recherches scientifiques avancées, de réseaux collaboratifs et d'interventions médicales transformatrices qui pourraient potentiellement changer d'innombrables vies.

Abeona Therapeutics Inc. (ABEO) - Modèle commercial: partenariats clés

Collaborations stratégiques avec les institutions de recherche et les universités

Abeona Therapeutics a établi des partenariats avec les institutions de recherche suivantes:

Institution	Focus de la collaboration	Année établie
Université de Pennsylvanie	Recherche sur la thérapie génique	2017
Hôpital pour enfants à l'échelle nationale	Recherche de troubles génétiques rares	2016

Partenariats avec des sociétés de biotechnologie et de pharmaceutique

Les collaborations pharmaceutiques clés comprennent:

• En partenariat avec Alexion Pharmaceuticals pour la recherche de maladies rares
• Collaboration avec Spark Therapeutics pour le développement de la thérapie génique

Accords de licence pour les technologies de thérapie génique

Technologie	Partenaire de licence	Frais de licence
ABO-102 (thérapie MPS III)	Instituts nationaux de santé	2,5 millions de dollars
EB-101 (épidermolyse Bullosa Therapy)	Université du Massachusetts	1,8 million de dollars

Recherche collaborative avec les sites d'essais cliniques

Réseaux de collaboration des essais cliniques:

• Clinique de Cleveland
• Centre médical de l'Université de Stanford
• Hôpital pour enfants de Philadelphie

Financement et concession des partenariats avec les agences gouvernementales

Agence	Montant d'octroi	Focus de recherche
National Institutes of Health (NIH)	4,2 millions de dollars	Recherche de troubles génétiques rares
Ministère de la Défense	3,7 millions de dollars	Thérapie génique Recherche avancée

Abeona Therapeutics Inc. (ABEO) - Modèle d'entreprise: activités clés

Recherche et développement de la thérapie génique

En 2024, Abeona Therapeutics s'est concentrée sur le développement de thérapies géniques pour les maladies génétiques rares. Le budget de la recherche et du développement de l'entreprise s'est élevé à environ 15,2 millions de dollars au cours de l'exercice le plus récent.

Domaine de recherche	Programmes actifs	Étape de développement
Troubles génétiques rares	3 programmes thérapeutiques primaires	Essais cliniques précliniques à la phase 2

Gestion des essais cliniques

Abeona a géré plusieurs essais cliniques dans différentes zones thérapeutiques.

• Essais cliniques actifs totaux: 4
• Investissement en essai clinique: 8,7 millions de dollars en 2023
• Zones d'intervention principales: troubles du stockage lysosomal

Développement du traitement des maladies génétiques rares

L'entreprise a consacré des ressources importantes au développement de traitements pour des conditions génétiques rares spécifiques.

Cible de la maladie	Type de thérapie	État actuel
Syndrome de Sanfilippo	Thérapie génique	Essais cliniques de phase 2
Épidermolyse bullosa	Thérapie génique	Développement préclinique

Innovations thérapeutiques de scène préclinique et clinique

Abeona maintient un pipeline robuste d'approches thérapeutiques innovantes.

• Total des candidats thérapeutiques: 6
• Personnel de recherche et développement: 42 employés
• Dépenses annuelles de R&D: 22,5 millions de dollars

Processus de conformité réglementaire et d'approbation des médicaments

La société a investi considérablement dans des stratégies réglementaires et une conformité.

Activité réglementaire	Investissements de conformité	Interactions réglementaires
Interactions de la FDA	3,2 millions de dollars	12 communications formelles en 2023
Applications IND	2 soumis	Examen en attente

Abeona Therapeutics Inc. (ABEO) - Modèle d'entreprise: Ressources clés

Plateformes de thérapie génique propriétaire

Abeona Therapeutics maintient des plateformes de thérapie génique spécialisées axées sur les maladies génétiques rares:

• Plateforme EB-101 pour l'épidermolyse Bullosa
• Plateforme ABO-102 pour le syndrome du syndrome de Sanfilippo
• Plateforme ABO-202 pour le syndrome du syndrome de Sanfilippo

Plate-forme	Maladie cible	Étape de développement
EB-101	Épidermolyse bullosa	Étape clinique
ABO-102	Syndrome de Sanfilippo Type A	Essais cliniques de phase 1/2
ABO-202	Syndrome de Sanfilippo Type B	Développement préclinique

Équipe de recherche et développement scientifique

En 2024, Abeona Therapeutics a:

• 18 Personnel de recherche et développement total
• 7 scientifiques de niveau doctoral
• 3 experts en génie génétique spécialisés

Portefeuille de propriété intellectuelle

Mesures clés de la propriété intellectuelle:

Catégorie	Nombre
Familles totales de brevets	12
Brevets actifs	8
Demandes de brevet en instance	4

Installations avancées de laboratoire et de recherche

Détails de l'infrastructure de recherche:

• 2 laboratoires de recherche sur la thérapie génique dédiée
• Espace total des installations de recherche: 12 000 pieds carrés
• Équipement d'édition de gènes avancé: investissement de 3,2 millions de dollars

Capacités spécialisées de génie génétique

Capacités technologiques de génie génétique:

Technologie	Niveau de capacité
Édition du gène CRISPR	Avancé
Production de vecteurs viraux	Grande capacité
Dépistage de la thérapie génique	Complet

Abeona Therapeutics Inc. (ABEO) - Modèle d'entreprise: propositions de valeur

Traitements innovants pour les troubles génétiques rares

Abeona Therapeutics se concentre sur le développement de thérapies géniques pour des troubles génétiques rares avec des zones cibles spécifiques:

Catégorie de troubles	Conditions génétiques spécifiques	Étape de développement
Troubles du stockage lysosomal	Types du syndrome de Sanfilippo A et B	Phase d'essai clinique
Troubles de la peau	Épidermolyse bullosa	Recherche préclinique

Solutions thérapeutiques ciblées pour les patients pédiatriques

Zones de mise au point pédiatrique avec des approches thérapeutiques spécifiques:

• Rare plateformes de traitement de la maladie génétique pédiatrique
• Vecteurs de thérapie génique spécialement conçus pour les enfants
• Stratégies d'intervention génétique personnalisées

Technologies de thérapie génique avancée

Plateformes et capacités technologiques:

Technologie	Application spécifique	Caractéristiques uniques
Thérapie génique AAV	Traitement du syndrome de Sanfilippo	Conception de vecteur viral propriétaire
Techniques d'édition de gènes	Correction de troubles génétiques rares	Approches basées sur CRISPR

Interventions médicales potentielles qui changent la vie

Zones d'intervention clés avec un impact potentiel:

• Thérapies géniques des troubles neurologiques
• Traitements génétiques du trouble de la peau
• Interventions des maladies métaboliques

Approches de gestion des maladies génétiques personnalisées

Stratégies de personnalisation:

Approche de gestion	Cible de la population de patients	Niveau de personnalisation
Thérapie spécifique à la mutation génétique	Patients de troubles génétiques rares	Ciblage de haute précision
Conception de vecteur spécifique au patient	Génétique individuelle Profile	Traitement individualisé

Abeona Therapeutics Inc. (ABEO) - Modèle d'entreprise: relations clients

Engagement direct avec les communautés de patients

En 2024, Abeona Therapeutics maintient l'engagement direct de la communauté des patients:

• Groupes de soutien aux patients atteints de maladies génétiques rares
• Interactions de réseau de patients en ligne
• Programmes de sensibilisation de la communauté des maladies rares ciblées

Métriques d'engagement communautaire des patients	Données annuelles
Interactions du groupe de soutien aux patients	87 interactions documentées
Engagement communautaire en ligne	3 214 membres du réseau de patients enregistrés
Webinaires de l'éducation des patients	12 événements virtuels annuels

Consultation et soutien professionnel en médecine

Abeona fournit un engagement professionnel médical spécialisé à travers:

• Réseaux de consultation spécialisés de troubles génétiques rares
• Plateformes de collaboration de recherche clinique
• Interactions spécialisées du conseil consultatif médical

Engagement professionnel médical	Métriques annuelles
Membres du conseil consultatif médical	24 professionnels spécialisés
Séances de consultation clinique	46 Consultations spécialisées
Réunions de collaboration de recherche	18 Sessions collaboratives annuelles

Programmes d'aide aux patients et d'éducation

Les stratégies complètes de soutien aux patients comprennent:

• Ressources d'information sur les troubles génétiques
• Assistance à la navigation au traitement
• Coordination du programme de soutien financier

Réseaux de collaboration de recherche

Abeona entretient des partenariats de recherche avec:

• Établissements de recherche universitaire
• Centres de recherche sur les troubles génétiques
• Plateformes de collaboration de recherche internationale

Détails de collaboration de recherche	Données annuelles
Partenaires de l'institution de recherche	7 collaborations institutionnelles actives
Projets de recherche collaborative	3 initiatives de recherche en cours
Contributions de financement de la recherche	1,2 million de dollars d'investissement annuel

Communication transparente sur les développements thérapeutiques

Les canaux de communication comprennent:

• Rapports de progression scientifique trimestriels
• Mises à jour annuelles des essais cliniques
• Plateformes de communication des investisseurs et des parties prenantes

Métriques de transparence de la communication	Données annuelles
Communications scientifiques publiques	24 rapports d'étape détaillés
Événements de divulgation des essais cliniques	4 mises à jour annuelles complètes
Points de contact de la communication des investisseurs	8 événements de communication formels

Abeona Therapeutics Inc. (ABEO) - Modèle d'entreprise: canaux

Communications de recherche médicale directes

En 2024, Abeona Therapeutics utilise des canaux de communication directs avec 47 institutions de recherche clés dans le monde. La société entretient 23 accords de collaboration de recherche actifs.

Type de communication	Nombre de contacts	Fréquence d'interaction annuelle
Institutions de recherche	47	128 interactions
Centres de recherche clinique	31	92 interactions

Conférences et présentations scientifiques

Abeona participe à 16 grandes conférences de biotechnologie chaque année, avec une moyenne de 7 présentations directes.

• Conférences totales présentes: 16
• Présentations directes: 7
• Présentations des affiches: 9

Publications de l'industrie de la biotechnologie

La société publie des recherches dans 12 revues à comité de lecture, avec 18 publications en 2023.

Catégorie de publication	Nombre de publications
Journaux évalués par des pairs	18
Résumé de la recherche	24

Plates-formes et sites Web numériques

Abeona maintient 3 canaux de communication numériques principaux:

• Site Web de l'entreprise: www.abeonatherapeutics.com
• Portail des relations avec les investisseurs
• Référentiel de publication de recherche

Partenariats avec les institutions médicales

Le portefeuille de partenariats actuel comprend 31 établissements de recherche médicale dans 12 pays.

Type de partenariat	Nombre de partenariats	Propagation géographique
Collaborations de recherche	31	12 pays
Sites d'essais cliniques	23	8 pays

Abeona Therapeutics Inc. (ABEO) - Modèle d'entreprise: segments de clientèle

Patiens de maladies génétiques rares

Population estimée des patients pour des troubles génétiques rares ciblés:

Trouble	Compte estimé des patients
Syndrome de Sanfilippo Type A	1 naissances vivantes sur 70 000
Syndrome de Sanfilippo Type B	1 sur 160 000 naissances vivantes
EB (épidermolyse bullosa)	Environ 50 000 patients aux États-Unis

Fournisseurs de soins de santé pédiatriques

Segments de soins de santé cibles:

• Spécialistes génétiques pédiatriques: 1 200 à l'échelle nationale
• Hôpitaux pour enfants ayant des programmes de maladies génétiques: 62 aux États-Unis
• Centres de traitement génétique spécialisés: 87 à l'échelle nationale

Institutions de recherche génétique

Métriques d'engagement des établissements de recherche:

Type d'institution	Nombre de collaborateurs potentiels
Centres de recherche universitaires	214
Installations de recherche génétique financée par les NIH	89
Fondations de recherche privée	37

Centres de traitement médical spécialisés

Distribution du centre de traitement:

• Centres spécialisés dans les troubles génétiques rares: 43
• Centres de soins complets avec des programmes génétiques: 126
• Hôpitaux spécialisés pédiatriques avec des unités génétiques: 92

Les familles touchées par les troubles génétiques

Insistance démographique de la famille:

Catégorie	Données statistiques
Total des familles souffrant de troubles génétiques rares	Environ 25 000 à 30 000
Engagement moyen du réseau de soutien familial	72% recherchent activement des options de traitement avancées
Utilisation du counseling génétique	58% des familles touchées

Abeona Therapeutics Inc. (ABEO) - Modèle d'entreprise: Structure des coûts

Frais de recherche et de développement

Pour l'exercice 2023, Abeona Therapeutics a déclaré des dépenses de R&D de 23,4 millions de dollars.

Année	Dépenses de R&D	Pourcentage du total des coûts opérationnels
2023	23,4 millions de dollars	62.3%
2022	31,2 millions de dollars	68.5%

Investissements d'essais cliniques

Les investissements en essais cliniques pour Abeona Therapeutics en 2023 ont totalisé environ 15,7 millions de dollars.

• Essais cliniques de thérapie génique: 9,2 millions de dollars
• Études de troubles génétiques rares: 6,5 millions de dollars

Maintenance de la propriété intellectuelle

Les coûts annuels de maintenance de la propriété intellectuelle étaient de 2,1 millions de dollars en 2023.

Catégorie IP	Coût
Dépôt de brevet	1,3 million de dollars
Renouvellement des brevets	0,8 million de dollars

Coûts de conformité réglementaire

Les dépenses de conformité réglementaire en 2023 s'élevaient à 4,5 millions de dollars.

• Coûts de soumission de la FDA: 2,3 millions de dollars
• Documentation de la conformité: 1,7 million de dollars
• Consultations réglementaires externes: 0,5 million de dollars

Recrutement du personnel et des talents scientifiques

Les dépenses totales du personnel pour 2023 étaient de 18,6 millions de dollars.

Catégorie de personnel	Coût annuel	Nombre d'employés
Chercheur	8,9 millions de dollars	45
Développement clinique	5,7 millions de dollars	30
Personnel administratif	4,0 millions de dollars	25

Abeona Therapeutics Inc. (ABEO) - Modèle d'entreprise: Strots de revenus

Ventes de produits thérapeutiques potentiels

Depuis le quatrième trimestre 2023, Abeona Therapeutics n'a pas de produits approuvés commercialement générant des revenus directs. Le potentiel de revenus de l'entreprise est lié à son rare pipeline thérapeutique de la maladie génétique.

Subventions et financement de recherche

Année	Source d'octroi	Montant
2023	National Institutes of Health (NIH)	1,2 million de dollars
2022	Fondation de recherche sur les maladies rares	$750,000

Accords de licence

Les accords de licence actuels sont axés sur les technologies de thérapie génique, en particulier pour les troubles génétiques rares.

• ABO-102 (MPS IIIA) Potentiel de licence
• ABO-101 (Syndrome du Sanfilippo Type B) Licence de technologie

Partenariats de recherche collaborative

Partenaire	Focus de recherche	Valeur de partenariat estimé
Université de Pennsylvanie	Recherche sur la thérapie génique	3,5 millions de dollars
Hôpital pour enfants de Philadelphie	Thérapies de troubles génétiques rares	2,8 millions de dollars

Paiements de jalons potentiels

Structure potentielle de paiement des étapes pour le développement thérapeutique:

• Étape préclinique jalon: jusqu'à 5 millions de dollars
• Phase I Jalon d'essai clinique: jusqu'à 10 millions de dollars
• Phase II Essai clinique jalon: jusqu'à 25 millions de dollars
• Jalon d'approbation réglementaire: jusqu'à 50 millions de dollars

Revenu total pour Abeona Therapeutics en 2023: 4,6 millions de dollars, principalement à partir de subventions de recherche et de partenariats collaboratifs.

Abeona Therapeutics Inc. (ABEO) - Canvas Business Model: Value Propositions

The core value proposition for Abeona Therapeutics Inc. is simple: delivering the first and only autologous cell-based gene therapy, ZEVASKYN, to address the profound, chronic wounds of Recessive Dystrophic Epidermolysis Bullosa (RDEB). This is a game-changer for a disease with historically no cure, and it creates immediate, tangible value for patients and the healthcare system.

You are buying into a commercial-stage company that has successfully translated a complex gene therapy into an FDA-approved product, plus it has a deep pipeline in other high-unmet-need areas. That's a strong foundation.

Providing the first and only autologous cell-based gene therapy for treating chronic wounds in RDEB patients.

ZEVASKYN (prademagene zamikeracel) is the first and only autologous (using the patient's own cells) cell-based gene therapy approved by the U.S. Food and Drug Administration (FDA) for treating RDEB wounds. This is a crucial distinction because it directly addresses the root cause of the disease-a defect in the COL7A1 gene-by providing functional Type VII collagen to anchor the skin layers.

The clinical data from the pivotal Phase 3 VIITAL™ study is compelling and forms the backbone of this value proposition. It showed a dramatic and clinically meaningful difference from the standard of care, which is essentially just palliative wound management.

Here's the quick math on the clinical impact:

Metric	ZEVASKYN (prademagene zamikeracel)	Standard of Care (Control)	Value Proposition
Wounds with 50% or More Healing at 6 Months	81% of treated wounds	16% of matched wounds	5x greater healing rate
Patient Population Addressed	Adult and pediatric patients with RDEB	N/A	First and only FDA-approved gene therapy for this indication

This level of durable wound healing offers a significant reduction in the constant pain, infection risk, and debilitating nature of RDEB, which can see wounds cover between 30% and 80% of a patient's body surface.

Offering a potentially curative, one-time treatment for ultra-rare, severe genetic disorders.

The value of ZEVASKYN is amplified by its design as a one-time treatment. Unlike daily creams or repeated surgeries, this is a single surgical application of gene-corrected cell sheets. This single-application approach is a major benefit for patients, caregivers, and the healthcare system because it drastically reduces the long-term burden of care.

The treatment is designed to be potentially durable, with clinical benefits having been observed to last up to 8 years post-treatment in trials. This durability is the key to calling it a potentially curative therapy for the treated areas, moving the value proposition far beyond temporary symptom relief.

• Reduces chronic wound burden with a single procedure.
• Offers durable skin adhesion and wound healing.
• Targets the genetic defect, not just the symptoms.

This model is particularly valuable for ultra-rare diseases like RDEB, where the patient population is small and the cost of chronic, lifelong care is exceptionally high.

Addressing high unmet medical needs in rare ophthalmic diseases through the pipeline.

Beyond ZEVASKYN, Abeona Therapeutics' pipeline of adeno-associated virus (AAV)-based gene therapies is a secondary, but still critical, value driver. This portfolio focuses on rare ophthalmic diseases that currently have high unmet medical needs, meaning there are few, if any, effective treatments.

The company is leveraging its next-generation AAV capsids to improve delivery to the eye, targeting diseases that cause severe vision loss or blindness.

• ABO-504: Targets Stargardt disease.
• ABO-503: Targets X-linked retinoschisis.
• ABO-505: Targets autosomal dominant optic atrophy.

While these programs are still in preclinical development, the most advanced program, likely ABO-503 (RS1), is expected to enter human studies in the second half of 2026. This pipeline shows a strategic commitment to translating gene therapy technology into solutions for other devastating rare conditions, providing future revenue streams and a long-term growth story.

Simplifying claims and reimbursement for QTCs via a permanent J-Code for ZEVASKYN.

For a high-cost gene therapy, the value proposition to the healthcare system-hospitals, payers, and Qualified Treatment Centers (QTCs)-is as important as the patient benefit. The Centers for Medicare and Medicaid Services (CMS) has assigned a permanent Healthcare Common Procedure Coding System (HCPCS) J-code J3389 for ZEVASKYN, effective January 1, 2026.

This J-code is defintely a pivotal milestone. It simplifies the complex billing and reimbursement process for the QTCs, making it much easier for hospitals to adopt the therapy, which is critical for a successful commercial launch.

As of late 2025, commercial momentum is building:

• Major commercial health plans have published coverage policies for ZEVASKYN, covering approximately 60% of RDEB patients.
• Abeona has received 12 ZEVASKYN product order forms (ZPOFs) from QTCs.
• Approximately 30 eligible RDEB patients have been identified at the initial treatment centers.

The company is also establishing outcomes-based agreements with major payer contracting organizations, which ties reimbursement to the clinical success of the therapy. This structure mitigates financial risk for payers, which is a strong value proposition in itself.

Abeona Therapeutics Inc. (ABEO) - Canvas Business Model: Customer Relationships

Abeona Therapeutics Inc.'s customer relationship model is a high-touch, hybrid approach, blending personalized patient support with a highly specialized, direct-to-center commercial structure. This is essential because ZEVASKYN (prademagene zamikeracel), an autologous cell-based gene therapy, requires complex logistics and deep patient-level support for a rare disease population (Recessive Dystrophic Epidermolysis Bullosa, or RDEB).

High-touch, direct engagement with Qualified Treatment Centers (QTCs) for complex logistics

The core of the commercial relationship is a direct, high-touch partnership with a select network of Qualified Treatment Centers (QTCs). These centers are chosen for their expertise in cell and gene therapy and specialized care for Epidermolysis Bullosa (EB) patients. The relationship is transactional but requires extensive collaboration due to the autologous nature of the therapy, which involves a complex chain of custody from patient biopsy to drug product manufacturing and final treatment.

As of late 2025, Abeona Therapeutics has activated three QTCs in the U.S. to handle the ZEVASKYN process. This number is a critical bottleneck and a key focus for expansion to meet patient demand.

• Activated QTCs: Ann & Robert H. Lurie Children's Hospital of Chicago, Lucile Packard Children's Hospital Stanford, and Children's Hospital Colorado.
• Logistical Support: Provide comprehensive training, scheduling systems, and coordination for the biopsy-to-treatment process.
• Expansion Goal: Several additional centers across the US are in various stages of site onboarding to expand the QTC network.

Personalized patient services through the Abeona Assist program

To acquire and retain patients in this ultra-rare disease space, the company provides the Abeona Assist Program, a highly customized, personalized support service. This program acts as a concierge service for patients, caregivers, and the QTC staff, removing major logistical and financial hurdles that often plague rare disease treatments. It's a crucial retention and access tool.

The program is a HUB partnership with AscellaHealth, designed to create a seamless, end-to-end patient experience. As of May 2025, approximately 30 patients and caregivers had started registering in the Abeona Assist program, showing strong early interest in the commercial launch. This is the ultimate 'white glove' service model.

Abeona Assist Program Component	Relationship Type	Purpose
Insurance & Benefits Verification	Personal Assistance	Optimize reimbursement and secure broad market access.
Financial Assistance Options	Personal Assistance	Reduce out-of-pocket costs for eligible patients.
Travel & Logistical Assistance	Dedicated Service	Coordinate patient and caregiver travel to the specialized QTCs.
Scheduling & Process Navigation	Dedicated Service	Guide patients through the complex biopsy, manufacturing, and treatment timeline.

Building defintely collaborative relationships with key opinion leaders and patient advocacy groups

Success in a rare disease market is impossible without deep, collaborative ties to the community. Abeona Therapeutics has a strong foundation built on years of clinical development, which translates into a collaborative relationship with the patient community and the medical experts who treat them. This is a vital, non-transactional relationship that drives patient identification and trust.

The partnership with DEBRA of America (Dystrophic Epidermolysis Bullosa Research Association) is central to this strategy, providing patient education and identification. Furthermore, the company maintains a strong Key Opinion Leader (KOL) network, leveraging the credibility of leading trial investigators to support the commercial launch. This collaboration has resulted in growing demand, with approximately 30 eligible patients now identified across the QTC network as of November 2025, more than doubling the initial dozen identified patients.

Direct-to-physician sales and medical affairs support for a specialized product

The company employs a specialized, direct-to-physician sales and medical affairs team, focusing solely on the QTCs and the RDEB physician community. This is a consultative sales model, not a volume-driven one, where the relationship is built on scientific expertise and operational support for a first-in-class gene therapy.

The scaling of this commercial effort is reflected in the company's financials. Selling, General, and Administrative (SG&A) expenses for the three months ended September 30, 2025, rose to $19.3 million, a significant increase from $6.4 million in the same period of 2024, primarily reflecting the increased headcount and professional costs associated with the ZEVASKYN commercial launch. This investment is directly tied to establishing and maintaining the direct-to-physician and QTC relationships. As a result of this direct engagement, the company has received 12 ZEVASKYN product order forms (ZPOFs) from patients at the first two QTCs, with the first commercial patient treatment anticipated in the fourth quarter of 2025.

Abeona Therapeutics Inc. (ABEO) - Canvas Business Model: Channels

You need to understand exactly how Abeona Therapeutics Inc. gets its complex, autologous gene therapy, ZEVASKYN (prademagene zamikeracel), to the patient. It's not a simple pharmacy distribution model; this is a highly specialized, controlled channel that relies on a small, expert network and direct patient engagement. The entire channel strategy is built around minimizing risk and maximizing access for a very rare patient population.

Direct Sales and Medical Teams Targeting the Specialized QTC Network

The primary channel is a direct-to-clinic model, utilizing specialized sales and medical teams focused exclusively on the Qualified Treatment Center (QTC) network. This is crucial because ZEVASKYN is an autologous cell-based gene therapy, meaning it uses the patient's own cells and requires highly specialized administration and surgical expertise. The sales team's job is less about volume and more about deep, technical engagement with these few, high-value centers.

As of the Q3 2025 update, Abeona Therapeutics Inc. has successfully activated 3 QTCs in the U.S.. These centers are the sole points of care for ZEVASKYN treatment. The company is actively working to activate additional sites, but this process is slow because of the complexity of onboarding a new gene therapy. The increase in Selling, General, and Administrative (SG&A) expenses to $19.3 million for the third quarter of 2025, up from $6.4 million in Q3 2024, directly reflects the investment in this commercial infrastructure, including the dedicated sales and medical teams needed to support these QTCs.

Here's the quick math: you are targeting a U.S. RDEB patient population of roughly 750 patients. Focusing on a small, high-throughput network is the only logical path to commercialization.

• Activated QTCs (as of late 2025): 3
• Identified Eligible Patients at QTCs: Approximately 30 (more than doubled from Q2 2025)
• Key QTC Locations: Lurie Children's Hospital of Chicago, Lucile Packard Children's Hospital Stanford, and Children's Hospital Colorado

A Complex Logistics Chain for Autologous Biopsy Collection and Drug Product Delivery

The channel for the drug product itself is a highly complex, closed-loop supply chain (logistics chain) because the therapy is autologous (patient-specific). This isn't a shelf product; it's a living drug. The channel starts with a patient biopsy at a QTC and ends with the surgical application of the final product back at the QTC. This process is the definition of a high-touch, controlled channel.

The central hub for this channel is Abeona Therapeutics Inc.'s fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, Ohio. This facility handles the gene modification and cell sheet production. The logistics challenge is maintaining the integrity and temperature of the patient's cells during transport from the QTC to Cleveland and then the final product back to the QTC. A recent operational risk was a temporary pause on biopsy collection in Q3 2025 due to the need to optimize a product release assay, but biopsy collection resumed in November 2025, with the first commercial treatment now anticipated in the fourth quarter of 2025.

Patient Referral Pathways Driven by the RDEB Community and Specialist Physicians

Patient acquisition relies heavily on a soft channel: the tight-knit Recessive Dystrophic Epidermolysis Bullosa (RDEB) patient and caregiver community, plus the specialist physician network. The company supports this with the Abeona Assist™ patient support program, which acts as a central intake and navigation channel for patients and caregivers.

This program helps patients navigate the complex journey, from initial inquiry and insurance prior authorization to scheduling the biopsy and the final treatment. The strong momentum from the community is clear, with patient demand more than doubling in Q3 2025. This is a classic rare disease model: the product sells itself through patient word-of-mouth and physician conviction, but the company must provide the service infrastructure to make the logistics happen.

Investor and Analyst Outreach via Conferences

A secondary, but critical, channel for capital and credibility is investor relations. For a commercial-stage biotech, maintaining a visible presence with financial stakeholders is essential for valuation and future funding flexibility. The key channel here is direct engagement at major healthcare conferences, which provides a platform for management to communicate commercial progress, like the QTC expansion and patient demand figures, directly to analysts and institutional investors.

The company has been highly active in late 2025, which is defintely a good sign for transparency.

Investor Channel Event	Date (2025)	Purpose
Stifel 2025 Healthcare Conference	November 13	Fireside Chat and Investor Meetings
Third Quarter 2025 Earnings Call	November 12	Discuss Q3 Financial Results and Commercial Progress
H.C. Wainwright 27th Annual Global Investment Conference	September 9	Company Presentation and Investor Meetings
Cantor Global Healthcare Conference 2025	September 4	Fireside Chat and Investor Meetings

This consistent outreach keeps the investment community apprised of the commercial ramp-up for ZEVASKYN, including the robust cash position of $207.5 million as of September 30, 2025, which is expected to fund operations for over two years without accounting for anticipated revenue.

Abeona Therapeutics Inc. (ABEO) - Canvas Business Model: Customer Segments

You're looking at Abeona Therapeutics Inc. as it transitions into a commercial-stage company, and the first thing to grasp is who actually pays for and receives their product, ZEVASKYN (prademagene zamikeracel). It's not a single customer; it's a triad of patients, specialized clinicians, and the payers who hold the purse strings.

The core customer segment is small but highly concentrated, typical of a rare disease (orphan drug) market. Your focus should be on the near-term revenue drivers-the RDEB patients and the payers who have already established coverage for this high-cost gene therapy.

Adult and pediatric patients suffering from recessive dystrophic epidermolysis bullosa (RDEB)

This is the primary, immediate customer segment for ZEVASKYN, an autologous cell-based gene therapy approved in April 2025 for RDEB wounds. This population is defined by a severe genetic disorder causing chronic, painful skin wounds due to a lack of Type VII collagen (C7).

The estimated total addressable market in the U.S. for patients who may benefit from COL7A1-mediated treatments is approximately 3,850 patients. However, the near-term focus is on the most severe cases with chronic wounds eligible for treatment at a Qualified Treatment Center (QTC).

The current commercial momentum is strong, even with the initial treatment start shifting to the fourth quarter of 2025.

• Patients Identified: Approximately 30 eligible patients have been identified across the activated QTCs as of November 2025.
• Product Orders: 12 ZEVASKYN product order forms have been received.
• 2025 Treatment Goal: Management has an initial goal to treat 10-14 patients in 2025.

Here's the quick math: targeting 10-14 patients is a tiny fraction of the total RDEB population, but at an estimated cost of $3.1 million per treatment (for 12 sheets), even a handful of successful treatments will generate significant initial revenue, defintely justifying the commercial infrastructure build.

Specialized physicians and clinicians at Gene Therapy/EB Qualified Treatment Centers

This segment is the key gatekeeper and execution partner. Because ZEVASKYN is a complex, cell-based gene therapy that requires a biopsy, specialized manufacturing, and a surgical application, the product is only available through a limited, highly-vetted network of centers called Qualified Treatment Centers (QTCs).

Abeona Therapeutics Inc. is focused on activating these sites to ensure patient access and proper administration. As of late 2025, there are three activated Qualified Treatment Centers (QTCs) in the network, including Ann & Robert H. Lurie Children's Hospital of Chicago and Lucile Packard Children's Hospital Stanford.

The company is actively engaging these specialized physicians-dermatologists, geneticists, and histopathologists-to drive referrals and patient flow. They are the ones who assess patient eligibility (age $\ge$ 6 years, chronic wounds $\ge$ 6 months, etc.) and perform the surgical application.

Commercial and government payers who determine ZEVASKYN coverage

The payer segment is arguably the most critical for a high-cost gene therapy. Securing broad coverage early de-risks the commercial launch and ensures patient access.

Abeona Therapeutics Inc. has achieved significant early market access momentum. Policies covering ZEVASKYN have been published by all major commercial payers, including United Healthcare, Cigna, Aetna, Anthem, and most Blue Cross Blue Shield plans.

This commercial coverage accounts for approximately 80 percent of lives covered by commercial insurance, which translates to coverage for about 60 percent of all RDEB patients. For government payers, the Centers for Medicare and Medicaid Services (CMS) has established a permanent Healthcare Common Procedure Coding System (HCPCS) J-code, J3389, effective January 1, 2026, which will streamline reimbursement across Medicare and Medicaid programs.

Payer Segment	Coverage Status (Late 2025)	Impact on Access
Major Commercial Payers	Policies published by all major payers (e.g., United Healthcare, Cigna).	Covers 80 percent of commercially-insured lives and 60 percent of all RDEB patients.
Government Payers (CMS)	Permanent HCPCS J-code J3389 established.	Facilitates expedited coverage and reimbursement across all 51 state Medicaid programs starting January 1, 2026.
Prior Authorizations	100% of submitted prior authorization requests have been approved so far.	Indicates strong alignment between the FDA label and payer coverage criteria.

Future patients with rare ophthalmic diseases like X-linked Retinoschisis (XLRS)

This segment represents the future growth opportunity and pipeline value, though it is not a revenue driver in 2025. The target here is the male population affected by X-linked Retinoschisis (XLRS), a disease causing progressive vision loss due to mutations in the RS1 gene.

The lead product for this segment is ABO-503, a gene therapy that is still in the preclinical development stage.

The market scale is significant for a rare disease, with an estimated 35,000 people affected in the United States and Europe. The prevalence is estimated to range from 1 in 5,000 to 1 in 25,000 males worldwide.

The development risk is somewhat mitigated by the program's selection for the FDA's Rare Disease Endpoint Advancement (RDEA) Pilot Program in October 2025, which should accelerate the development and validation of novel efficacy endpoints. The company anticipates completing the Investigational New Drug (IND)-enabling studies in the second half of 2026, so commercialization is still several years away.

Abeona Therapeutics Inc. (ABEO) - Canvas Business Model: Cost Structure

You need a clear picture of where Abeona Therapeutics Inc. (ABEO) is spending its capital, especially now that their lead product, ZEVASKYN (prademagene zamikeracel), is approved. The cost structure has fundamentally shifted from a pure research-and-development (R&D) model to one dominated by commercialization and fixed infrastructure, and that means a major swing in where the money is going.

Here's the quick math: the company is spending far less on core R&D-down to $4.2 million in Q3 2025-but the total burn remains high because commercial launch costs have exploded. This is the classic biopharma transition, but with the added complexity of a high-cost, autologous gene therapy.

Dominance of fixed costs related to R&D and manufacturing infrastructure maintenance

The cost base for Abeona Therapeutics Inc. is now heavily weighted toward fixed costs, which is typical for a commercial-stage gene therapy company. You have to maintain a state-of-the-art facility regardless of patient volume, and that infrastructure is not cheap.

The company operates a fully integrated cell and gene therapy Current Good Manufacturing Practice (cGMP) facility in Cleveland, Ohio. This facility is the sole manufacturing site for commercial ZEVASKYN production. Maintaining this specialized, high-standard infrastructure-including specialized personnel, utilities, and equipment depreciation-represents a substantial, largely fixed, operating expense.

Also, a portion of what used to be R&D spending is now capitalized into inventory or reclassified as Selling, General, and Administrative (SG&A) expenses following the FDA approval of ZEVASKYN, which makes the R&D line item look artificially low at $4.2 million for the three months ended September 30, 2025.

High selling, general, and administrative (SG&A) expenses, driven by the commercial launch

This is where the major cost increase has landed. Selling, General, and Administrative (SG&A) expenses for the third quarter of 2025 surged to $19.3 million, a massive jump from $6.4 million in the same period of 2024. Honestly, this is the number you need to focus on right now; it reflects the real-world cost of launching an ultra-rare disease therapy.

The increase reflects the build-out of the commercial team, professional costs for market access, and the reclassification of certain production costs, like engineering runs, from R&D to SG&A. They are staffing up and spending money to get ZEVASKYN to the small, specific patient population with recessive dystrophic epidermolysis bullosa (RDEB).

Here is a snapshot of the core expense shift for the quarter:

Expense Category	Q3 2025 Amount (3 Months Ended Sept 30, 2025)	Q3 2024 Amount (3 Months Ended Sept 30, 2024)	Change
Selling, General, and Administrative (SG&A)	$19.3 million	$6.4 million	+201.6%
Research and Development (R&D)	$4.2 million	$8.9 million	-52.8%
Net Loss	$(5.2) million	$(30.3) million	+82.8% Improvement

Significant investment in clinical trials for late-stage pipeline candidates

Even with ZEVASKYN approved, the company isn't defintely a one-product shop. They still have an active pipeline, which requires ongoing clinical trial investment. This is the long-term cost of staying relevant in gene therapy.

Their development portfolio includes adeno-associated virus (AAV)-based gene therapies, specifically for ophthalmic diseases. For example, their candidate ABO-503 (for X-linked retinoschisis - XLRS) was selected for the FDA's Rare Disease Endpoint Advancement Pilot Program. This selection signals a continued, albeit lower, commitment to clinical development and the associated costs for late-stage candidates.

The R&D spending of $4.2 million in Q3 2025 covers these ongoing programs, though the primary focus has clearly shifted to commercial operations. What this estimate hides is the potential for R&D costs to spike again if a pipeline candidate moves into a costly Phase 3 trial.

Costs associated with maintaining regulatory compliance and quality control for an autologous product

The cost of compliance and quality control for an autologous (patient-specific) cell-based gene therapy like ZEVASKYN is inherently high. You are manufacturing a unique, living product for each patient, and the FDA demands perfection in the process. This isn't like making pills.

A concrete example of this is the production challenge faced in Q3 2025: Abeona Therapeutics Inc. had to temporarily pause collecting patient biopsies because a full batch of drug product could not be released. The issue was a false positive result from a rapid sterility assay, which is a key release assay mandated by the FDA. The costs here are not just the lost batch, but the time, personnel, and regulatory submission expenses required to investigate, optimize the assay, and resume operations.

Key cost drivers for autologous product compliance include:

• Maintaining the cGMP facility and its specialized clean-room environment.
• Personnel training and quality assurance (QA) overhead.
• Rigorous, often costly, release testing procedures for each patient's batch.
• Regulatory affairs expenses for managing post-approval commitments and process changes.

Abeona Therapeutics Inc. (ABEO) - Canvas Business Model: Revenue Streams

The revenue model for Abeona Therapeutics Inc. is in a critical transition phase, moving from primarily non-recurring, non-dilutive capital to a recurring product sales stream following the late 2025 launch of its first commercial product. The biggest 2025 revenue event was a one-time asset sale, but the future growth is tied entirely to the scaling of ZEVASKYN sales.

Product sales of ZEVASKYN (prademagene zamikeracel), with first commercial treatments expected to start in Q4 2025

The primary, recurring revenue stream is the direct sale of ZEVASKYN (prademagene zamikeracel), an autologous cell-based gene therapy for wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB). While the first patient treatment was initially slated for Q3 2025, it was shifted to Q4 2025 due to the time needed to optimize a rapid sterility release assay mandated by the FDA.

The company is strategically focused on a successful commercial rollout, anticipating profitability by early 2026. Management expects to treat between 10 to 14 patients and recognize revenue from those treatments in the 2025 fiscal year. Demand is strong, with the number of identified eligible patients at Qualified Treatment Centers (QTCs) more than doubling to approximately 30 patients as of the third quarter of 2025. The manufacturing capacity is expected to reach up to six treatments per month by the end of 2025, which will be a key driver for 2026 revenue.

Securing reimbursement is crucial, and the Centers for Medicare & Medicaid Services (CMS) have assigned a permanent J-code for ZEVASKYN, which is scheduled to become effective on January 1, 2026. This simplifies the billing process significantly for the Qualified Treatment Centers.

Non-recurring, non-dilutive capital from the sale of the Rare Pediatric Disease Priority Review Voucher (PRV) for $155 million in Q2 2025

A major, non-recurring revenue event in 2025 was the sale of the Rare Pediatric Disease Priority Review Voucher (PRV), which was awarded upon the FDA approval of ZEVASKYN. This transaction closed in Q2 2025 for gross proceeds of $155 million. This sale was a critical, non-dilutive capital infusion that significantly strengthened the balance sheet, contributing to the $207.5 million in cash, cash equivalents, restricted cash, and short-term investments as of September 30, 2025. This cash runway is expected to fund operations for over two years, not accounting for anticipated ZEVASKYN sales.

Potential future milestone and royalty revenue from out-licensed or partnered assets with companies like Taysha Gene Therapies, Inc.

Abeona Therapeutics maintains potential future revenue streams from out-licensed assets, including:

• Sublicense agreements with Taysha Gene Therapies, Inc. for gene therapies targeting CLN1 disease and Rett syndrome.
• Licensing of its proprietary AIM™ capsids to third parties, such as Beacon Therapeutics for ophthalmology gene therapy.

As of the nine months ended September 30, 2025, the company had not recognized any sales-based or royalty revenue from the Taysha Gene Therapies agreements. However, the company did incur a royalty expense of $0.1 million for the nine months ended September 30, 2025, due to royalties owed to the University of North Carolina at Chapel Hill resulting from a third party's option exercise to license certain AIM™ capsids.

Analyst expectations for the full-year 2025 revenue are low, but the forecast annual revenue growth rate is high at 702.96% as the launch scales

The full-year 2025 revenue picture is dominated by the one-time PRV sale, which is why the analyst-forecasted annual revenue growth rate is so dramatic. The forecast annual revenue growth rate is an impressive 702.96%, reflecting the shift from a pre-commercial stage to a commercial-stage company.

Here is a snapshot of the 2025 revenue components and forecasts:

Revenue Stream Type	Source/Product	2025 Value/Forecast	Notes
Non-Recurring Capital	Rare Pediatric Disease Priority Review Voucher (PRV) Sale	$155 million	Gross proceeds from sale closed in Q2 2025.
Recurring Product Sales (Forecast)	ZEVASKYN (prademagene zamikeracel)	$28.02 million	Analyst consensus revenue forecast for 2025.
Recurring Product Sales (Guidance)	ZEVASKYN (prademagene zamikeracel)	10-14 patient treatments expected	Management guidance for revenue-recognized treatments in 2025.
Licensing/Royalty Revenue	Taysha Gene Therapies, Inc. & other out-licensed assets	$0	No sales-based or royalty revenue recognized through Q3 2025.
Forecast Annual Revenue Growth Rate	Total Revenue	702.96%	Reflects the scale-up from a pre-commercial base.

The quick math here is that the high growth rate is an artifact of the low revenue base-reported revenue for 2025 is around $400,000 before the PRV sale and ZEVASKYN launch. The real story is the transition to a recurring stream, which starts in Q4 2025 and will be the focus for 2026.