Regenxbio Inc. (RGNX): Canvas de modèle d'entreprise [Jan-2025 MISE À JOUR]

Dans le monde de pointe de la thérapie génique, Regenxbio Inc. (RGNX) émerge comme une force pionnière, exerçant sa plate-forme de technologie révolutionnaire NAV pour transformer la façon dont nous abordons les troubles génétiques rares. Avec un modèle commercial innovant qui prie la percée scientifique et la stratégie commerciale, ce pionnier de la biotechnologie est sur le point de redéfinir la médecine génétique personnalisée, offrant de l'espoir aux patients confrontés à des défis neurologiques et rétiniens complexes grâce à des technologies thérapeutiques révolutionnaires.

Regenxbio Inc. (RGNX) - Modèle d'entreprise: partenariats clés

Établissements de recherche universitaire

RegenxBio a établi des partenariats avec les institutions de recherche universitaires suivantes:

Institution	Focus de la collaboration	Année établie
Université de Pennsylvanie	Recherche de thérapie génique pour les troubles neurologiques	2017
École de médecine de Harvard	Développement de la plate-forme technologique NAV	2015

Collaborations de l'entreprise pharmaceutique

Les partenariats stratégiques avec les sociétés pharmaceutiques comprennent:

• AbbVie Inc.: Valeur de collaboration de 200 millions de dollars de paiement initial
• Novartis Gene Therapies: Contrat de licence pour 60 millions de dollars de paiement initial

Funding National Institutes of Health

Détails du support de financement du NIH:

Type de subvention	Financement total	Domaine de recherche
Subventions SBIR / STTR	3,5 millions de dollars	Recherche de maladies génétiques rares
Subvention de recherche R01	2,1 millions de dollars	Développement de la thérapie génique

Organisations de recherche contractuelle

Regenxbio fonctionne avec les CRO suivants:

• Icon PLC: Gestion des essais cliniques
• Parexel International: coordination des essais de phase II
• Iqvia: soutien mondial du développement clinique

Partenariats d'investissement en biotechnologie

Partenariats d'investissement clés:

Entreprise d'investissement	Montant d'investissement	Année
Conseillers perceptifs	85 millions de dollars	2022
Farallon Capital Management	65 millions de dollars	2021

Regenxbio Inc. (RGNX) - Modèle d'entreprise: Activités clés

Recherche et développement de la thérapie génique

Au quatrième trimestre 2023, Regenxbio a investi 148,3 millions de dollars en dépenses de R&D. L'entreprise s'est concentrée sur le développement de thérapies géniques dans plusieurs domaines thérapeutiques.

Zone de focus R&D	Nombre de programmes actifs
Maladies neurologiques	4 programmes
Maladies rétiniennes	3 programmes
Maladies génétiques rares	2 programmes

Avancement de la plate-forme technologique NAV

La plate-forme de technologie de navigation propriétaire de Regenxbio comprend 10 sérotypes vectoriels AAV exclusifs.

• La plate-forme a généré 6 vecteurs hors licence
• Soutient le développement de thérapies géniques à travers de multiples indications
• Investissements d'optimisation des plateformes en cours

Gestion des essais précliniques et cliniques

Étape de l'essai	Essais actifs
Préclinique	5 programmes
Phase 1	3 programmes
Phase 2	2 programmes
Phase 3	1 programme

Soumission et conformité réglementaires

En 2023, Regenxbio a soumis 2 demandes d'enquête sur les nouveaux médicaments (IND) à la FDA.

Protection et licence de propriété intellectuelle

En décembre 2023, Regenxbio a tenu 285 brevets délivrés et en attente à l'échelle mondiale.

Catégorie de brevet	Nombre de brevets
NAV Technology Platform	127 brevets
Compositions de thérapie génique	98 brevets
Processus de fabrication	60 brevets

Regenxbio Inc. (RGNX) - Modèle d'entreprise: Ressources clés

Technologie vectorielle virale de Nav propriétaire

La plate-forme de technologie NAV de RegenxBio comprend 10 vecteurs de virus adéno-associés distincts (AAV). Depuis 2024, la société détient 100+ licences Pour cette technologie propriétaire à travers diverses applications de thérapie génique.

Type vecteur	Caractéristiques uniques	Applications potentielles
NAV AAV9	Pénétration améliorée du SNC	Troubles neurologiques
NAV AAV8	Tropisme hépatique élevé	Maladies métaboliques

Installations de recherche de thérapie génique avancée

Regenxbio fonctionne 2 centres de recherche primaires Situé à Rockville, Maryland, avec un espace de recherche total d'environ 75 000 pieds carrés.

Talent scientifique et médical spécialisé

Au quatrième trimestre 2023, Regenxbio utilise:

• Total des employés: 327
• Chercheurs au niveau du doctorat: 68
• Personnel de recherche et développement: 212

Portfolio de brevets fort

Paysage des brevets à partir de 2024:

Catégorie de brevet	Nombre de brevets
Nous a délivré des brevets	87
Demandes de brevet en instance	53
Déposages de brevets internationaux	42

Capital financier important pour la R&D

Ressources financières pour la recherche et le développement:

• Equivalents en espèces et en espèces (Q4 2023): 536,2 millions de dollars
• Dépenses de R&D (2023 Exercice): 274,3 millions de dollars
• Engagements totaux de financement de la recherche: 612,5 millions de dollars

Regenxbio Inc. (RGNX) - Modèle d'entreprise: propositions de valeur

Solutions innovantes de thérapie génique pour les maladies génétiques rares

Regenxbio se concentre sur le développement de traitements de thérapie génique ciblant les maladies génétiques rares avec des besoins médicaux non satisfaits importants. Depuis le quatrième trimestre 2023, l'entreprise a 6 programmes de thérapie génique à stade clinique en cours.

Programme	Maladie cible	Étape de développement
RGX-314	AMD humide	Phase 2/3
RGX-121	MPS II	Phase 1/2
RGX-111	MPS I	Phase 1/2

Traitements potentiels pour les troubles neurologiques et rétiniens

La plate-forme de thérapie génique de l'entreprise cible des conditions neurologiques et rétiniennes spécifiques avec des besoins médicaux non satisfaits.

• Marché des maladies rétiniennes estimées à 12,3 milliards de dollars d'ici 2026
• Troubles neurologiques Marché de la thérapie génique prévue pour atteindre 8,5 milliards de dollars d'ici 2027

Approches de médecine génétique personnalisée

Regenxbio utilise la plate-forme de technologie NAV pour les interventions génétiques ciblées. En 2023, la société détient 129 brevets émis dans le monde.

Plate-forme technologique	Nombre de licences	Flux de redevances potentiels
Technologie de navigation	9 accords de licence	Jusqu'à 1,4 milliard de dollars de paiement potentiels

Technologies thérapeutiques transformatrices

La performance financière reflète l'investissement continu dans la recherche avancée en thérapie génique.

Métrique financière	Valeur 2023
Dépenses de R&D	252,4 millions de dollars
Espèce et investissements	623,5 millions de dollars

Interventions génétiques ciblées avec des avantages potentiels à long terme

L'approche de thérapie génique de Regenxbio cible les troubles génétiques à leur racine moléculaire.

• Potentiel à fournir des traitements pondérés et potentiellement curatifs
• Concentrez-vous sur les maladies génétiques rares avec des options de traitement existantes limitées
• Approche de la médecine de précision ciblant des mutations génétiques spécifiques

Regenxbio Inc. (RGNX) - Modèle d'entreprise: relations avec les clients

Engagement direct avec les groupes de défense des patients

Depuis le quatrième trimestre 2023, Regenxbio a établi des relations avec 7 organisations de défense des patients atteints de maladies génétiques rares. La stratégie d'engagement directe de l'entreprise implique:

• Sessions de partage d'informations trimestrielles
• Soutien financier aux programmes d'éducation des patients totalisant 325 000 $ par an
• Plateformes de communication de recherche collaborative

Interactions du groupe de défense des patients	Fréquence	Investissement annuel
Réseaux de maladies génétiques rares	4 réunions trimestrielles	$325,000
Programmes de formation des patients	6 événements annuels	$175,000

Présentations des conférences scientifiques et des symposiums médicaux

En 2023, Regenxbio a participé à 12 conférences médicales internationales, avec des frais de présentation estimés à 450 000 $.

Type de conférence	Nombre de conférences	Frais de présentation
Symposiums de thérapie génique	5	$225,000
Conférences de maladies rares	7	$225,000

Partenariats de recherche collaborative

Regenxbio maintient 9 collaborations de recherche active avec des établissements universitaires et médicaux, avec des investissements en partenariat totaux de 3,2 millions de dollars en 2023.

Communication transparente sur les progrès des essais cliniques

La société a publié 24 mises à jour des essais cliniques Sur divers canaux de communication en 2023, avec un budget de relations avec les investisseurs dédié de 275 000 $.

Aide à la consultation médicale personnalisée

Regenxbio fournit des services de consultation spécialisés avec:

• 3 équipes de conseil génétique dédiées
• Budget de soutien à la consultation de 425 000 $ par an
• Moyenne de 87 consultations personnalisées par trimestre

Service de consultation	Taille de l'équipe	Budget annuel	Consultations trimestrielles
Conseil génétique	3 équipes	$425,000	87 Consultations

Regenxbio Inc. (RGNX) - Modèle d'entreprise: canaux

Communications de recherche médicale directes

Regenxbio utilise des stratégies de communication ciblées avec des institutions de recherche clés et des centres médicaux. Au quatrième trimestre 2023, la société a maintenu des partenariats de recherche actifs avec 12 centres de recherche universitaires.

Canal de communication de recherche	Nombre de partenariats actifs	Engagement annuel
Institutions universitaires	12	45 projets de recherche collaborative
Centres de recherche médicale	8	37 études cliniques en cours

Publications scientifiques et revues à comité de lecture

Regenxbio publié 23 articles évalués par des pairs En 2023, en se concentrant sur la recherche sur la thérapie génique et les développements de la plate-forme de technologie NAV.

• Revues publiées dans: Nature Biotechnology
• Médecine translationnelle scientifique
• Thérapie moléculaire

Biotechnologie et conférences médicales

La participation de la conférence en 2023 comprenait 18 événements internationaux de biotechnologie.

Type de conférence	Nombre de conférences	Focus de présentation
Conférences internationales de biotechnologie	18	Innovations de thérapie génique
Symposiums de recherche médicale	12	NAV Technology Platform

Plateformes numériques et site Web d'entreprise

Métriques d'engagement numérique pour 2023:

• Site Web d'entreprise Visiteurs uniques: 124 567
• LinkedIn adepte: 45 230
• Twitter abonnés: 22 145

Communications des relations avec les investisseurs

Canaux de communication des investisseurs en 2023:

Canal de communication	Fréquence	Atteindre
Appels de résultats trimestriels	4 fois par an	Plus de 200 investisseurs institutionnels
Réunion des actionnaires annuelle	1 fois par an	Environ 500 participants
Présentations des investisseurs	12 événements	Plus de 1 500 participants au total

Regenxbio Inc. (RGNX) - Modèle d'entreprise: segments de clientèle

Patients souffrant de troubles génétiques rares

En 2024, Regenxbio cible environ 7 000 troubles génétiques rares connus. Les populations clés des patients comprennent:

Catégorie de troubles	Population estimée des patients	Zones de traitement potentiels
Troubles génétiques neurologiques	Environ 500 000 patients	Maladie de Huntington, Parkinson
Troubles génétiques ophtalmologiques	Environ 250 000 patients	Maladies rétiniennes héritées

Chercheurs de maladie neurologique

Regenxbio collabore avec les institutions de recherche dans le monde.

• Plus de 120 partenariats de recherche actifs
• 42,3 millions de dollars investis dans des collaborations de recherche en 2023
• Engagement avec 37 centres de recherche universitaires

Spécialistes en ophtalmologie

Le marché cible comprend des praticiens spécialisés en ophtalmologie axés sur les thérapies génétiques.

Catégorie spécialisée	Nombre de spécialistes potentiels	Portée géographique
Spécialistes génétiques en ophtalmologie	Environ 2 500 dans le monde	Amérique du Nord, Europe, Asie

Praticiens de la médecine génétique

Regenxbio cible les professionnels de la médecine génétique dans diverses spécialités.

• Environ 5 600 spécialistes de la médecine génétique du monde entier
• Axé sur l'avancement de la thérapie génique
• Valeur marchande potentielle estimée à 1,2 milliard de dollars

Sociétés pharmaceutiques et biotechnologiques

Partenariats stratégiques avec des entités biotechnologiques et pharmaceutiques.

Type de partenariat	Nombre de partenariats actifs	Valeur de collaboration estimée
Accords de licence	12 partenariats actifs	78,5 millions de dollars en paiements de jalons potentiels
Collaborations de recherche	8 partenaires pharmaceutiques majeurs	63,2 millions de dollars en financement de recherche collaborative

Regenxbio Inc. (RGNX) - Modèle d'entreprise: Structure des coûts

Dépenses de recherche et développement approfondies

Pour l'exercice 2022, Regenxbio a déclaré des dépenses totales de R&D de 237,3 millions de dollars. Les coûts de R&D de l'entreprise ont toujours été une partie importante de ses dépenses opérationnelles.

Exercice fiscal	Dépenses de R&D	Pourcentage des dépenses totales
2022	237,3 millions de dollars	68.4%
2021	212,5 millions de dollars	65.7%

Coûts de gestion des essais cliniques

Les dépenses des essais cliniques pour Regenxbio en 2022 étaient d'environ 145,6 millions de dollars, couvrant plusieurs programmes de thérapie génique à tous les étapes de développement.

• Essais cliniques de phase 1: 42,3 millions de dollars
• Essais cliniques de phase 2: 68,9 millions de dollars
• Essais cliniques de phase 3: 34,4 millions de dollars

Maintenance de la propriété intellectuelle

La société a investi 18,2 millions de dollars dans la protection de la propriété intellectuelle et l'entretien des brevets au cours de l'exercice 2022.

Catégorie IP	Coût annuel
Dépôt de brevet	8,7 millions de dollars
Entretien de brevets	9,5 millions de dollars

Acquisition et rétention de talents

Regenxbio a dépensé 52,4 millions de dollars pour la rémunération et le recrutement des employés en 2022.

• Compensation totale des employés: 45,6 millions de dollars
• Recrutement et formation: 6,8 millions de dollars

Investissements de développement de la plate-forme technologique

Les coûts de développement de la plate-forme technologique pour 2022 étaient de 36,7 millions de dollars, en se concentrant sur les améliorations de la plate-forme technologique NAV.

Zone d'investissement technologique	Montant d'investissement
Infrastructure de plate-forme	22,3 millions de dollars
Développement des logiciels et des outils	14,4 millions de dollars

Regenxbio Inc. (RGNX) - Modèle d'entreprise: Strots de revenus

Licence potentielle de la plateforme de technologie NAV

Au quatrième trimestre 2023, Regenxbio a déclaré des revenus de licence potentiels de sa plate-forme de technologie NAV. Les accords de licence comprennent:

Partenaire	Type de licence	Revenus potentiels
Abbvie	Licence exclusive	Paiement initial de 370 millions de dollars
Ultragenyx	Licence non exclusive	85 millions de dollars de paiement initial

Accords de recherche collaborative

Regenxbio a établi des accords de recherche collaboratifs avec plusieurs sociétés pharmaceutiques.

• Revenus de recherche collaborative totale en 2023: 42,3 millions de dollars
• Partenariats de recherche actifs avec 5 sociétés pharmaceutiques
• Durée de l'accord moyen: 3-5 ans

Future commercialisation des produits thérapeutiques

Strots de revenus potentiels des produits thérapeutiques en développement:

Zone thérapeutique	Produit candidat	Valeur marchande potentielle estimée
Maladies rétiniennes héritées	RGX-314	500 millions de dollars - 1 milliard de dollars par an
Troubles neurologiques	RGX-121	250 millions de dollars - 450 millions de dollars par an

Subventions de recherche gouvernementales et privées

Regenxbio reçoit un financement de recherche de diverses sources:

• Financement total des subventions en 2023: 15,7 millions de dollars
• Les sources incluent les fondations de la recherche des NIH et privées
• Subventions axées sur la recherche sur la thérapie génique

Payments de jalons potentiels à partir de partenariats

Structure potentielle de paiement des étapes:

Partenaire	Paiements de jalons potentiels	Conditions
Abbvie	Jusqu'à 1,2 milliard de dollars	Jalons réglementaires et commerciaux
Ultragenyx	Jusqu'à 350 millions de dollars	Jalons de développement et de commercialisation

REGENXBIO Inc. (RGNX) - Canvas Business Model: Value Propositions

You're looking at the core promises REGENXBIO Inc. is making to patients, prescribers, and partners as of late 2025. These value propositions are grounded in their late-stage pipeline and their underlying AAV technology platform.

Potential one-time, curative treatments for severe genetic diseases

The primary value is offering single-dose treatments designed to alter the course of devastating genetic conditions. For Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, clemidsogene lanparvovec (RGX-121) is positioned as the first and only potential one-time, commercially-available therapy designed to directly address the underlying genetic cause, if approved. The Biologics License Application (BLA) for RGX-121 has a Prescription Drug User Fee Act (PDUFA) date set for February 8, 2026.

Addressing high unmet medical needs like Duchenne muscular dystrophy (DMD)

REGENXBIO Inc. is targeting diseases where current standards of care are inadequate or burdensome. For DMD, their investigational gene therapy, RGX-202, is advancing rapidly. The pivotal trial enrollment for RGX-202 was completed in October, with topline results anticipated in early Q2 2026. The company is also focused on securing a commissioner's voucher for this DMD program. For MPS II, the potential approval of RGX-121 would offer a significant alternative, as 80% of patients in the pivotal study no longer required enzyme replacement therapy by the last assessment.

Durable, long-term therapeutic effect from a single administration

The platform is designed to provide sustained benefit from one injection, which is a major shift from chronic dosing regimens. The data for RGX-121 supports this, showing a more than 80% reduction in CSF levels of HS D2S6, a key biomarker of MPS II brain disease, sustained through 1 year. For retinal disease, surabgene lomparvovec (sura-vec, ABBV-RGX-314) is being developed for chronic conditions like wet Age-related Macular Degeneration (wet AMD) and diabetic retinopathy (DR).

Proprietary AAV vector design for enhanced safety and efficacy

The foundation of these treatments is REGENXBIO Inc.'s proprietary AAV (Adeno-Associated Virus) gene therapy platform. Generally, AAV vectors are known to exhibit a favorable safety profile with low immunogenicity, which reduces the risk of adverse immune responses. Specific program data supports this: for RGX-202 in DMD, no serious adverse events or adverse events of special interest were observed in the Phase I/II study as of May 7, 2025, with no patients developing signs of liver injury. Furthermore, FDA pre-license and bioresearch monitoring inspections for RGX-121 found no adverse findings.

Potential for non-invasive, in-office delivery (suprachoroidal for retinal diseases)

REGENXBIO Inc. is advancing sura-vec for diabetic retinopathy (DR) using a suprachoroidal delivery method. This approach is supported by positive 2-year Phase II trial data from the ALTITUDE trial, which demonstrated a durable safety and efficacy profile through two years with a single, in-office injection for patients with non-proliferative DR. Enrollment has been completed in pivotal trials for wet AMD using subretinal delivery, with topline data expected in Q4 2026.

Here's a quick look at the financial strength supporting these value propositions as of the third quarter of 2025:

Metric	Value / Date	Context
Cash, Cash Equivalents & Marketable Securities	$302.0 million (as of Sep 30, 2025)	Funded operations into early 2027
Q3 2025 Revenue	$29.7 million	Beat estimate of $24.61 million by 20.58%
Q3 2025 Net Loss per Share (Non-GAAP)	$1.20 loss	Narrower than forecasted loss of $1.33
RGX-121 PDUFA Date	February 8, 2026	Original date was November 9, 2025, extended for longer-term data
RGX-202 DMD Topline Data Expected	Early Q2 2026	Pivotal trial enrollment completed in October

The company's operational progress is also reflected in key financial events that bolstered the balance sheet:

• Received $110.0 million upfront payment under the Nippon Shinyaku partnership in March 2025.
• Received $144.5 million in net proceeds from the royalty monetization with HCRx in May 2025.
• Q3 2025 revenue included $5.9 million of development service revenue under the Nippon Shinyaku partnership.

Finance: draft 13-week cash view by Friday.

REGENXBIO Inc. (RGNX) - Canvas Business Model: Customer Relationships

Close, high-touch relationships with rare disease patient advocacy groups.

• Patient Advocacy team contact email: patientadvocacy@regenxbio.com.
• REGENXBIO participated in the BIO Patient Advocacy Changemakers Event in October 2025.
• The company is focused on clinical trials for broad availability and is not accepting applications for expanded access at this time.
• Personal stories of patients and families guide the work to improve treatment options for serious diseases.

Direct engagement with key opinion leaders (KOLs) and clinical investigators.

• The AFFINITY DUCHENNE® pivotal trial for RGX-202 is ongoing, expecting enrollment completion of approximately 30 patients aged 1+ in the U.S. and Canada by October 2025.
• A separate AEV8 antibody assessment study involves a central primary investigator, Dr. Han Fan, at the Rare Disease Research Institute at UH in Atlanta, Georgia.
• REGENXBIO engaged with financial KOLs by participating in investor conferences in September 2025, including the Wells Fargo, Morgan Stanley, Baird, and H.C. Wainwright conferences.

Strategic management of co-development and commercialization partnerships.

These relationships are quantified by upfront payments, potential future value, and service revenue generated in 2025.

Partner/Agreement	Product(s) Covered	Upfront/Initial Payment Received (2025)	Total Potential Future Value (Milestones)	2025 Service Revenue Reported
Nippon Shinyaku	RGX-121 (MPS II), RGX-111 (MPS I) in US/Asia	$110 million (Closed March 2025)	Up to an additional $700 million	$2.7 million (Q2 2025), $5.9 million (Q3 2025)
AbbVie	Surabgene lomparvovec (sura-vec, ABBV-RGX-314) for Retinal Disease	$150 million at closing (prior to Aug 2025 amendment)	$200 million in potential milestones from DR program amendment ($100 million per Phase III trial)	Not explicitly detailed as separate service revenue in Q2/Q3 2025 reports
HCRx	Royalty Monetization	$144.5 million net proceeds (May 2025)	N/A	N/A

Regulatory dialogue with the FDA and other global health authorities.

• The Prescription Drug User Fee Act (PDUFA) goal date for clemidsogene lanparvovec (RGX-121) was extended to February 8, 2026, from the original November 9, 2025 date.
• The extension followed the submission of additional 12-month clinical data for all 13 patients in the pivotal study.
• The initial BLA review was accepted in May 2025.
• FDA pre-license inspection and bioresearch monitoring inspections concluded with no observations, and no safety concerns were raised during the review.
• RGX-121 has received Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations from the FDA.
• Data for RGX-121 showed a median D2S6 level reduction of 86%, with 80% of patients discontinuing enzyme replacement therapy by the last assessment.

REGENXBIO Inc. (RGNX) - Canvas Business Model: Channels

You're looking at how REGENXBIO Inc. gets its gene therapy innovations to patients and partners as of late 2025. It's a mix of leveraging established giants and preparing for self-sufficiency.

Pharmaceutical partners (AbbVie, Nippon Shinyaku) for commercial reach

REGENXBIO Inc. uses strategic partnerships to ensure broad commercial reach for its late-stage assets, especially outside its immediate operational focus.

The collaboration with Nippon Shinyaku covers the development and commercialization of clemidsogene lanparvovec (RGX-121) for MPS II and RGX-111 for MPS I in the United States and Asia, which is the Licensed Territory. REGENXBIO received an upfront payment of $110 million in March 2025 under this agreement. Revenue from this partnership included $5.9 million in development service revenue for the three months ended September 30, 2025. REGENXBIO is eligible for up to an additional $700 million in milestones, split between development/regulatory milestones of $40 million and sales milestones of $660 million. Furthermore, REGENXBIO retains the right to all proceeds from the sale of the Priority Review Voucher (PRV) for RGX-121, which has a potential FDA approval expected in late 2025 (PDUFA date moved to February 8, 2026). Nippon Shinyaku leads commercialization in the Licensed Territory, while REGENXBIO retains all rights outside it and receives meaningful double-digit royalties on net sales in the U.S. and Asia.

For the retinal program, surabgene lomparvovec (ABBV-RGX-314) for wet AMD, the collaboration with AbbVie involves a 50/50 profit share. An amendment in August 2025 established new milestone payments from AbbVie totaling $200 million, contingent on dosing subjects in subsequent clinical trials for the diabetic retinopathy program.

Here's a quick look at the financial structure of the Nippon Shinyaku deal:

Payment Type	Amount	Status/Target
Upfront Payment Received	$110 million	Received March 2025
Total Potential Milestones	Up to $700 million	Development, Regulatory, and Sales
Potential Sales Milestones	$660 million	Part of total potential milestones
Q3 2025 Partnership Revenue	$5.9 million	Development service revenue

Specialized distribution networks for ultra-rare disease therapies

For the ultra-rare disease therapies partnered with Nippon Shinyaku (RGX-121 and RGX-111), the distribution channel is primarily managed by the partner, leveraging their established rare disease expertise in the U.S. and Asia. REGENXBIO Inc. itself leads the manufacturing of both products for clinical and commercial supply within that Licensed Territory.

• Nippon Shinyaku leads commercialization in the U.S. and Asia.
• REGENXBIO Inc. leads manufacturing for clinical and commercial supply in the Licensed Territory.

Clinical trial sites for patient access to investigational therapies

Patient access to investigational therapies is channeled directly through a broad network of clinical trial sites globally.

The ATMOSPHERE and ASCENT pivotal trials for surabgene lomparvovec (sura-vec) in wet AMD completed enrollment with over 1,200 participants across more than 200 sites globally, representing the largest gene therapy program reported for an ocular indication.

For RGX-202 in Duchenne muscular dystrophy, the AFFINITY DUCHENNE pivotal trial completed enrollment of approximately 30 patients aged 1+ in the U.S. and Canada by October 2025.

Direct sales force (future build-out) for wholly-owned commercial products

REGENXBIO Inc. is positioning itself to transition to a commercial company, with plans for self-commercialization outside of specific licensed territories. The company reported initiating commercial supply manufacturing in Q3 2025 to support expected launches.

• Expected to become a commercial company early next year (early 2026).
• Commercial-ready manufacturing is in-house at the Rockville, MD headquarters.
• Manufacturing of clinical and confirmatory trial supply for RGX-202 is complete.

REGENXBIO Inc. (RGNX) - Canvas Business Model: Customer Segments

You're looking at the core groups REGENXBIO Inc. (RGNX) serves, which are primarily patients with severe, often rare, conditions and the large biopharma entities that partner to bring these gene therapies to market. This is where the revenue potential and the clinical focus truly meet.

The patient segments are defined by the late-stage pipeline assets as of late 2025. For Duchenne muscular dystrophy, the pivotal AFFINITY DUCHENNE trial for RGX-202 completed enrollment of 30 participants aged 1+ in the U.S. and Canada in October 2025. The company has manufactured initial batches intended for commercial supply, targeting a launch in 2027, with an in-house manufacturing capacity of 2,500 doses of RGX-202 per year.

For the rare lysosomal storage disorders, clemidsogene lanparvovec (RGX-121) for MPS II (Hunter syndrome) has a PDUFA date set for February 8, 2026. This program is a partnership with Nippon Shinyaku, which provided REGENXBIO Inc. with an upfront payment of $110 million in the first quarter of 2025. RGX-111 targets MPS I (Hurler syndrome) under the same partnership structure.

The chronic retinal disease segment involves surabgene lomparvovec (ABBV-RGX-314), developed with AbbVie. Enrollment for the wet AMD pivotal trials is complete. Under an August 2025 amendment for the diabetic retinopathy (DR) program, AbbVie is set to pay REGENXBIO Inc. $100 million upon first subject dosed in the Phase IIb/III trial, plus another $100 million upon first subject dosed in a second Phase III clinical trial.

The biopharma segment is crucial, as evidenced by the financials. REGENXBIO Inc.'s third-quarter 2025 revenue was $29.7 million, with development service revenue under the Nippon Shinyaku partnership contributing $5.9 million in that quarter alone. The company exited Q3 2025 with $302.0 million in cash, cash equivalents, and marketable securities, which management expects will fund operations into early 2027.

Here's a quick look at the key relationships and financial anchors for these customer groups as of the third quarter of 2025:

Customer Segment	Key Program/Asset	Relevant Metric/Value (Late 2025)
Patients with MPS II	Clemidsogene lanparvovec (RGX-121)	PDUFA date: February 8, 2026
Patients with MPS I	RGX-111	Partnership with Nippon Shinyaku for US and Asia
Patients with Duchenne	RGX-202	Pivotal enrollment completed: 30 participants
Patients with Retinal Diseases	Surabgene lomparvovec (ABBV-RGX-314)	Potential milestone payments from AbbVie: $200 million total for DR program
Biopharma Companies	Nippon Shinyaku Partnership	Upfront payment received: $110 million (Q1 2025)
Biopharma Companies	Overall Financial Health	Cash/Securities as of September 30, 2025: $302.0 million

You can see the direct financial impact from the partnerships. For instance, the nine months ended September 30, 2025, revenue reached $140.1 million, a significant jump from $62.11 million the prior year, largely due to these collaborations. Also, the company received $145 million in net proceeds from a royalty monetization with HCRx in Q2 2025, which supports the runway for these customer-facing development efforts.

The customer segments for the NAV Technology Platform are the partners themselves, who pay for development services and milestones. The platform has supported thousands of patients treated, including those receiving Novartis' ZOLGENSMA®.

• Patients with rare genetic diseases (MPS II/Hunter syndrome, MPS I/Hurler syndrome).
• Patients with neuromuscular disorders (Duchenne muscular dystrophy).
• Patients with chronic retinal diseases (wet AMD, diabetic retinopathy).
• Biopharma companies licensing the NAV Technology Platform.

Finance: review the Q4 2025 revenue projections against the $83.04 million sales estimate for that quarter by next Tuesday.

REGENXBIO Inc. (RGNX) - Canvas Business Model: Cost Structure

You're looking at the expense side of REGENXBIO Inc.'s operations as of late 2025, and honestly, it looks exactly like what you'd expect from a late-stage gene therapy company pushing toward commercialization. The costs are heavily weighted toward getting those late-stage assets across the finish line.

The Research and Development (R&D) spend is the clear dominant cost driver. For the three months ended September 30, 2025, R&D expenses totaled $56.1 million. This figure is up from $54.4 million in the same period last year, showing the ramp-up as programs mature.

To be fair, this R&D spend isn't just bench science; it's the cost of running pivotal trials and building the infrastructure to sell the product. The costs for clinical trial execution and patient enrollment are baked into that R&D number, especially with programs like RGX-202 for Duchenne muscular dystrophy advancing rapidly, having completed pivotal trial enrollment in October 2025.

Also, you see a significant investment in in-house manufacturing and commercial readiness. REGENXBIO highlighted initiating commercial supply manufacturing in Q3 2025 during its Q1 update, and management pointed to its 'commercial-ready manufacturing with capacity to seize blockbuster opportunities' in the Q3 release. These activities drive up both R&D (clinical supply costs) and G&A (overhead for scaling operations).

The supporting overhead, General and Administrative (G&A) expenses, came in at $20.3 million for Q3 2025. This was an increase from $19.4 million year-over-year, and the primary drivers here were professional services, consulting, and other corporate advisory services.

Here's a quick look at the key operating expenses for the quarter:

Expense Category	Q3 2025 Amount (Millions USD)	Q3 2024 Amount (Millions USD)
Research and Development (R&D)	$56.1	$54.4
General and Administrative (G&A)	$20.3	$19.4

The investment focus driving these costs includes:

• High costs for clinical trial execution and patient enrollment for late-stage assets like RGX-202 and sura-vec.
• Significant investment in in-house manufacturing and commercial readiness, with commercial supply manufacturing initiated in Q3 2025.
• Costs associated with professional services, consulting, and corporate advisory services contributing to G&A growth.
• Manufacturing-related expenses and other clinical supply costs for pivotal trials driving R&D increases.

REGENXBIO exited the quarter with $302.0 million in cash, cash equivalents, and marketable securities, which management stated should fund operations into early 2027. That cash runway is defintely critical given these high burn rates.

Finance: draft 13-week cash view by Friday.

REGENXBIO Inc. (RGNX) - Canvas Business Model: Revenue Streams

You're looking at how REGENXBIO Inc. brings in cash right now, late in 2025. It's heavily weighted toward partnerships, which is typical for a company deep in the gene therapy development cycle. The revenue streams are clearly segmented between upfront payments for IP access, ongoing service fees for manufacturing and development work, and the promise of future royalties and milestones.

For the third quarter ending September 30, 2025, total revenues hit \$29.7 million. This was a step up from the \$24.2 million seen in the same period in 2024. Honestly, this revenue profile shows the immediate impact of their major 2025 deals.

The core of the current recognized revenue comes from the development and licensing activities. Specifically, the Nippon Shinyaku partnership contributed \$5.9 million in development service revenue during Q3 2025. To give you a sense of the quarterly flow, Q2 2025 saw \$2.7 million in development service revenue from that same partnership, and Q1 2025 was quite strong, recognizing \$71.8 million in combined license and service revenue from Nippon Shinyaku following the deal closing in March 2025.

The upfront cash infusion from strategic partnerships is a significant component. The January 2025 deal with Nippon Shinyaku for RGX-121 and RGX-111 provided a substantial immediate boost. REGENXBIO Inc. received \$110 million upfront when that transaction closed in March 2025. This upfront fee is recognized as license and royalty revenue upon delivery of the intellectual property licenses.

Here's the quick math on the Nippon Shinyaku deal structure, which dictates future potential revenue:

Financial Component	Amount/Terms	Notes
Upfront Payment Received	\$110 million	Received in March 2025.
Total Potential Milestones	Up to \$700 million	Includes development, regulatory, and sales milestones.
Development/Regulatory Milestones	Up to \$40 million	Part of the total potential milestones.
Sales Milestones	Up to \$660 million	Part of the total potential milestones.
Royalties on Net Sales (U.S. & Asia)	Meaningful double-digit royalties	Applies to RGX-121 and RGX-111.

Royalties on net sales represent the long-term, passive income stream. This includes royalties from existing licensed products like Zolgensma, though those specific royalties decreased in Q2 2025 compared to the prior year. More immediately relevant are the meaningful double-digit royalties REGENXBIO Inc. is entitled to on future net sales of RGX-121 and RGX-111 in the Licensed Territory (U.S. and Asia) under the Nippon Shinyaku agreement. Also, don't forget the royalty monetization with HCRx in May 2025, where REGENXBIO Inc. received \$144.5 million in net proceeds in exchange for select anticipated royalties, including those from ZOLGENSMA and the MPS programs.

The potential monetization of a Priority Review Voucher (PRV) is a major contingent revenue event. This voucher is tied to the potential approval of RGX-121 for Hunter syndrome. The FDA granted the Biologics License Application (BLA) Priority Review, setting a Prescription Drug User Fee Act (PDUFA) target action date of November 9, 2025. If approved, REGENXBIO Inc. retains all rights and 100 percent of any proceeds related to the potential sale of this PRV. What this estimate hides is that this potential PRV cash, along with future milestones, is explicitly excluded from the current cash runway guidance extending into early 2027.

The structure of these revenue streams can be summarized by what REGENXBIO Inc. keeps versus what is shared:

• Retained Rights: REGENXBIO Inc. keeps 100% of proceeds from the potential sale of the RGX-121 Priority Review Voucher.
• Retained Upside: The company retains future potential non-dilutive funding opportunities, including milestones from AbbVie.
• Shared Revenue: Double-digit royalties and up to \$700 million in milestones are shared with Nippon Shinyaku.
• Monetized Royalties: A portion of anticipated royalties (including ZOLGENSMA) was monetized for \$144.5 million in May 2025.

Finance: draft 13-week cash view by Friday.