Rocket Pharmaceuticals, Inc. (RCKT): Business Model Canvas [Jan-2025 Mis à jour]

Dans le monde de pointe de la biotechnologie, Rocket Pharmaceuticals, Inc. (RCKT) émerge comme un phare d'espoir pour les familles luttant contre les troubles génétiques pédiatriques rares. En tirant parti des technologies avancées de thérapie génique et un modèle commercial complet, cette entreprise innovante transforme le paysage de la médecine génétique, offrant des traitements potentiellement curatifs qui étaient autrefois considérés comme impossibles. Leur approche stratégique combine une recherche scientifique révolutionnaire, des partenariats collaboratifs et une mission laser axée sur le laser pour développer des thérapies génétiques personnalisées qui pourraient révolutionner la façon dont nous comprenons et traitons les conditions génétiques complexes.

Rocket Pharmaceuticals, Inc. (RCKT) - Modèle commercial: partenariats clés

Établissements de recherche universitaire

Rocket Pharmaceuticals maintient des partenariats stratégiques avec les établissements de recherche académiques suivants:

Institution	Focus de recherche	Détails du partenariat
Université de Californie, San Francisco	Troubles génétiques rares	Recherche collaborative en cours en thérapie génique de l'anémie Fanconi
École de médecine de Harvard	Troubles leucocytaires	Programme de recherche conjoint pour la carence en pyruvate kinase

Biotechnologie stratégique et collaborations pharmaceutiques

Les collaborations stratégiques clés comprennent:

• Andelyn Biosciences - Viral Vector Manufacturing Partnership
• Spark Therapeutics - Exchange de technologies de thérapie génique
• Regenxbio Inc. - NAV Technology Platform Collaboration

Financement national des instituts de santé (NIH)

Détails de financement des subventions de recherche NIH:

Exercice fiscal	Montant d'octroi	Programme de recherche
2023	4,2 millions de dollars	Recherche de troubles génétiques pédiatriques rares
2024	4,5 millions de dollars	Développement de thérapie génique avancée

Organisations de recherche contractuelle (CROS)

Partenariats CRO actifs:

• Icon PLC - Gestion des essais cliniques mondiaux
• MEDPACE - Élaboration d'essais cliniques de maladies rares
• Sciences de la santé PRA - coordination des essais de phase I-III

Centres médicaux pédiatriques

Réseau de collaboration des essais cliniques:

Centre médical	Emplacement	Essais cliniques actifs
Hôpital pour enfants de Philadelphie	Philadelphie, PA	3 essais de troubles génétiques rares en cours
Santé des enfants de Stanford	Palo Alto, CA	2 essais cliniques de thérapie génique

Rocket Pharmaceuticals, Inc. (RCKT) - Modèle d'entreprise: Activités clés

Recherche et développement de traitements de thérapie génique

Au quatrième trimestre 2023, Rocket Pharmaceuticals a investi 87,4 millions de dollars dans les dépenses de R&D. La société se concentre sur les maladies génétiques pédiatriques rares avec le ciblage actuel du pipeline:

• Carence en adhérence des leucocytes-i (LAD-i)
• Anémie Fanconi
• Carence en pyruvate kinase

Gestion des essais précliniques et cliniques

Phase d'essai clinique	Nombre d'essais actifs	Investissement estimé
Préclinique	3	12,6 millions de dollars
Phase I	2	18,3 millions de dollars
Phase II	4	35,7 millions de dollars

Processus de conformité réglementaire et d'approbation de la FDA

Interactions de la FDA en 2023: 17 réunions officielles, avec 4 désignations de thérapie révolutionnaire reçues.

Développement de la propriété intellectuelle

Portefeuille de brevets:

• Brevets actifs totaux: 42
• Demandes de brevet en instance: 13
• Dépenses de dépôt de brevets en 2023: 4,2 millions de dollars

Génie génétique avancé et fabrication vectorielle

Capacités de fabrication:

• Capacité de production de vecteurs internes: 500 lots de vecteur viral / an
• Investissement des installations de fabrication: 65,3 millions de dollars
• Personnel de contrôle de la qualité: 47 professionnels spécialisés

Rocket Pharmaceuticals, Inc. (RCKT) - Modèle commercial: Ressources clés

Capacités avancées de recherche et de développement de la thérapie génique

Rocket Pharmaceuticals se concentre sur de rares maladies génétiques avec des capacités de recherche spécifiques:

Domaine de mise au point de recherche	Nombre de programmes actifs
Carence en adhérence des leucocytes-i (LAD-i)	2 programmes de stade clinique
Anémie Fanconi	3 programmes de stade clinique
Carence en pyruvate kinase	1 programme de stade clinique

Technologies de génie génétique propriétaire

Le portefeuille de propriété intellectuelle comprend:

• 12 brevets accordés
• 18 demandes de brevet en instance
• Plate-forme vectorielle lentivirale propriétaire

Talent scientifique et médical spécialisé

Catégorie de personnel	Nombre
Total des employés	137 au 31 décembre 2023
Chercheurs de doctorat	42
Chercheurs MD	8

Portefeuille de propriété intellectuelle

Paysage breveté:

• Familles totales de brevets: 30
• Couverture géographique: États-Unis, Europe, Japon
• Range d'expiration des brevets: 2030-2041

Infrastructure avancée de laboratoire et de recherche

Installation de recherche	Emplacement	En pieds carrés
Siège social	Cranbury, New Jersey	35 000 pieds carrés
Laboratoire de recherche	New York	22 000 pieds carrés

Rocket Pharmaceuticals, Inc. (RCKT) - Modèle d'entreprise: propositions de valeur

Thérapies génétiques innovantes pour les troubles génétiques pédiatriques rares

Rocket Pharmaceuticals se concentre sur le développement de thérapies géniques pour des troubles génétiques pédiatriques rares ayant des besoins médicaux non satisfaits importants.

Zone de thérapie	Trouble cible	Étape de développement
Carence en adhérence des leucocytes (LAD)	Immunodéficience génétique rare	Essai clinique de phase 1/2
Anémie Fanconi	Trouble sanguin rare	Développement clinique en cours
Carence en pyruvate kinase	Trouble des globules rouges rares	Essai clinique de phase 1/2

Traitements curatifs potentiels pour des conditions génétiques auparavant non traitables

Rocket Pharmaceuticals cible les conditions génétiques sans traitements efficaces actuels.

• Approches de thérapie génique ciblant des mutations génétiques spécifiques
• Modification potentielle de la maladie à long terme
• Aborder les causes génétiques racinaires plutôt que la gestion des symptômes

Approches thérapeutiques génétiques personnalisées

Stratégie de médecine de précision axée sur les interventions génétiques spécifiques au patient.

Plate-forme technologique	Approche thérapeutique	Caractéristique unique
Vecteur lentiviral	Correction du gène	Efficacité de transduction élevée
Vecteur AAV	Remplacement des gènes	Livraison génétique ciblée

Réduction des coûts des soins de santé à long terme grâce à des interventions ciblées

Avantages économiques potentiels grâce à des approches thérapeutiques à intervention unique.

• Réduction des coûts de traitement à vie estimé: 1,5 $ à 3 millions de dollars par patient
• Diminution des hospitalisations
• Dépenses de gestion des maladies chroniques minimisées

Amélioration de la qualité de vie des patients atteints de maladies génétiques rares

Le développement clinique s'est concentré sur les résultats thérapeutiques transformateurs.

Maladie	Amélioration potentielle de la qualité de vie	Population de patients
Anémie Fanconi	Risque de cancer réduit	Environ 2 000 patients aux États-Unis
Carence en adhérence des leucocytes	Fonction immunitaire améliorée	Moins de 500 patients dans le monde

Rocket Pharmaceuticals, Inc. (RCKT) - Modèle d'entreprise: relations avec les clients

Engagement direct avec les groupes de défense des patients

En 2024, Rocket Pharmaceuticals entretient des partenariats actifs avec les organisations de défense des patients suivantes:

Organisation	Domaine de mise au point	Type de collaboration
Organisation nationale pour les troubles rares (NORD)	Maladies génétiques rares	Soutien à la recherche
Leucémie & Société de lymphome	Troubles sanguins rares	Conscience des essais cliniques

Partenariats de recherche collaborative

Collaborations actuelles de recherche sur les établissements médicaux:

Institution	Focus de recherche	Valeur de partenariat
Université de Stanford	Développement de la thérapie génique	3,2 millions de dollars
Université de Pennsylvanie	Recherche de troubles génétiques rares	2,7 millions de dollars

Communication transparente sur les essais cliniques

• Mises à jour des essais cliniques en temps réel partagés sur le site Web de l'entreprise
• Rapports de progrès trimestriels publiés
• Les mesures de recrutement des patients divulguées publiquement

Programmes de soutien aux patients et d'éducation

Métriques du programme de soutien aux patients:

Composant de programme	2024 mesures
Webinaires de l'éducation des patients	37 séances effectuées
Aide à l'aide des patients	2 456 demandes uniques traitées
Applications d'aide financière	412 traité

Consultations médicales et services de suivi en cours

Statistiques des services de consultation médicale:

Type de service	Volume 2024
Séances de conseil génétique	624 consultations
Suivi du patient à long terme	287 patients en surveillance continue

Rocket Pharmaceuticals, Inc. (RCKT) - Modèle d'entreprise: canaux

Présentations de recherche médicale directes

En 2023, Rocket Pharmaceuticals a présenté 7 grandes conférences de recherche médicale, notamment:

Conférence	Date	Focus de présentation
Société américaine de gène & Thérapie cellulaire	Mai 2023	Programme de troubles du leucocytes
Société européenne du gène & Thérapie cellulaire	Octobre 2023	Maladies génétiques pédiatriques rares

Participation de la conférence scientifique

Métriques de la participation de la conférence pour 2023:

• Conférences totales présentes: 12
• Présentations orales: 5
• Présentations des affiches: 7
• Total des résumés de recherche soumis: 15

Publications de revues médicales évaluées par des pairs

Statistiques de publication pour 2023:

Catégorie de journal	Nombre de publications
Revues de génétique à fort impact	4
Revues de recherche de maladies rares	3
Publications totales évaluées par des pairs	7

Collaboration avec des médecins spécialistes pédiatriques

Réseau de collaboration en 2023:

• Total des centres de recherche pédiatrique engagés: 22
• Collaborations d'essais cliniques actifs: 8
• Reach géographique: 15 États aux États-Unis

Plateformes de santé numérique et réseaux de communication de recherche

Métriques d'engagement numérique pour 2023:

Plate-forme	Métrique de l'engagement
Réseau de recherche LinkedIn	3 750 connexions professionnelles
Researchgate Profile	2 100 abonnés
Section de recherche sur le site Web de l'entreprise	45 000 visiteurs uniques

Rocket Pharmaceuticals, Inc. (RCKT) - Modèle d'entreprise: segments de clientèle

Patients pédiatriques souffrant de troubles génétiques rares

Rocket Pharmaceuticals se concentre sur environ 5-7 troubles génétiques rares affectant les populations pédiatriques. La population de patients cibles de l'entreprise est estimée à 3 000 à 5 000 personnes aux États-Unis.

Catégorie de troubles	Population estimée des patients	Potentiel de traitement annuel
Carence en adhérence des leucocytes	500-750 patients	250 000 $ - 500 000 $ par patient
Anémie Fanconi	1 500 à 2 000 patients	350 000 $ - 650 000 $ par patient

Institutions de recherche génétique

Rocket Pharmaceuticals collabore avec 12-15 institutions de recherche de haut niveau dans le monde.

• Partenariats de recherche des National Institutes of Health (NIH)
• Réseaux de collaboration pour l'hôpital pour enfants
• Centres de recherche médicale académique

Centres de traitement médical spécialisés

La société cible environ 25-30 centres de traitement génétique spécialisés à travers l'Amérique du Nord et l'Europe.

Région géographique	Nombre de centres de traitement	Engagement annuel
États-Unis	18-22 centres	Investissement collaboratif annuel de 5 à 7 millions de dollars
Union européenne	7-8 centres	3 à 4 millions de dollars d'investissement collaboratif annuel

Groupes de plaidoyer pour les patients atteints de maladies rares

Rocket Pharmaceuticals s'engage activement avec 8 à 10 organisations majeures de défense des maladies rares.

• Fonds de recherche sur l'anémie Fanconi
• Association d'immunodéficience primaire
• Alliance mondiale des troubles génétiques rares

Fournisseurs de soins de santé spécialisés dans les conditions génétiques

La société interface avec environ 150-200 fournisseurs de soins de santé génétiques spécialisés à l'échelle nationale.

Spécialité du fournisseur	Nombre de prestataires	Engagement clinique annuel
Hématologues pédiatriques	75-90 fournisseurs	Support de recherche clinique de 2 à 3 millions de dollars
Spécialistes des troubles génétiques	60-75 fournisseurs	1,5 à 2,5 millions de dollars collaboration de recherche

Rocket Pharmaceuticals, Inc. (RCKT) - Modèle d'entreprise: Structure des coûts

Dépenses de recherche et développement approfondies

Au quatrième trimestre 2023, Rocket Pharmaceuticals a déclaré des dépenses de R&D de 70,3 millions de dollars pour l'exercice. La répartition totale des investissements en R&D de la société comprend:

Catégorie de R&D	Montant des dépenses
Programmes de thérapie génique	42,5 millions de dollars
Recherche de maladies pédiatriques rares	18,7 millions de dollars
Développement préclinique	9,1 millions de dollars

Gestion et exécution des essais cliniques

Les dépenses d'essai cliniques pour 2023 ont totalisé environ 45,2 millions de dollars, avec l'allocation suivante:

• Essais de thérapie génique des leucocytes: 22,6 millions de dollars
• Programme Fanconi Anemia: 15,3 millions de dollars
• Essais de maladies métaboliques: 7,3 millions de dollars

Processus de conformité et d'approbation réglementaires

Les frais de conformité réglementaire pour 2023 étaient estimés à 12,5 millions de dollars, notamment:

Zone de conformité	Coût
Préparation de la soumission de la FDA	5,8 millions de dollars
Documentation réglementaire	4,2 millions de dollars
Surveillance de la conformité	2,5 millions de dollars

Protection de la propriété intellectuelle

Les dépenses de propriété intellectuelle pour 2023 s'élevaient à 6,8 millions de dollars, distribuées comme suit:

• Dépôt et entretien des brevets: 4,3 millions de dollars
• Services de protection juridique: 2,5 millions de dollars

Recrutement et rétention des talents scientifiques avancés

Les frais d'acquisition et de rétention de talents pour 2023 étaient de 18,6 millions de dollars:

Catégorie de gestion des talents	Frais
Salaires compétitifs	12,4 millions de dollars
Recrutement des chercheurs	3,9 millions de dollars
Avantages et formation des employés	2,3 millions de dollars

Structure totale des coûts pour 2023: 153,4 millions de dollars

Rocket Pharmaceuticals, Inc. (RCKT) - Modèle d'entreprise: Strots de revenus

Accords potentiels de licence de produits futurs

Depuis le quatrième trimestre 2023, Rocket Pharmaceuticals a des accords de licence potentiels dans son portefeuille de thérapie génique de maladies rares. La valeur potentielle estimée de ces accords n'est pas divulguée publiquement.

Subventions de recherche et financement gouvernemental

Source de financement	Montant	Année
Subventions des National Institutes of Health (NIH)	3,2 millions de dollars	2023
Financement de la recherche du ministère de la Défense	1,5 million de dollars	2023

Partenariats de recherche collaborative

Les partenariats de recherche actuels comprennent:

• Hôpital pour enfants de Boston
• Université du Minnesota
• Instituts nationaux de santé

Ventes de produits thérapeutiques potentiels

Les revenus de Rocket Pharmaceuticals provenant des ventes de produits thérapeutiques potentiels dépend des approbations de la FDA. Le pipeline actuel comprend:

Thérapie	Indication	Valeur marchande potentielle
RP-L201	Anémie Fanconi	Marché annuel estimé de 150 à 200 millions de dollars
RP-A501	Maladie de Danon	Marché annuel estimé de 100 à 150 millions de dollars

Opportunités de licence de propriété intellectuelle

Depuis 2023, Rocket Pharmaceuticals tient 17 brevets délivrés et 32 demandes de brevet en instance sur ses plateformes de thérapie génique.

Total des frais de recherche et de développement pour 2023: 94,6 millions de dollars

Perte nette pour 2023: 108,4 millions de dollars

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Value Propositions

Potential one-time curative genetic therapies for devastating rare diseases.

Rocket Pharmaceuticals, Inc. is advancing therapies targeting conditions with high unmet medical need, evidenced by its late-stage pipeline focus.

Addressing ultra-rare, life-threatening disorders with high unmet medical need.

• Severe Leukocyte Adhesion Deficiency-I (LAD-I) is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant.
• PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) affects approximately 50,000 adults and children in the U.S. and Europe.
• The cardiovascular programs collectively target over 100,000 patients across the U.S. and EU.

KRESLADI™ (marnetegragene autotemcel; marne-cel) for severe LAD-I offers a potential alternative to bone marrow transplant.

Clinical data from the global Phase 1/2 study for KRESLADI™ demonstrated 100% overall survival at 12 months post-infusion for all enrolled patients. The Prescription Drug User Fee Act (PDUFA) target action date for the Biologics License Application (BLA) resubmission is set for March 28, 2026. Approval would make Rocket Pharmaceuticals eligible for a Rare Pediatric Disease Priority Review Voucher (PRV). The therapy was well tolerated with no treatment-related serious adverse events.

Focused AAV platform for cardiovascular diseases (Danon, PKP2-ACM, BAG3-DCM).

The company has sharpened its strategic focus on its adeno-associated virus (AAV) cardiovascular gene therapy portfolio. The cash, cash equivalents, and investments as of September 30, 2025, totaled $222.8 million, expected to fund operations into the second quarter of 2027. This focus was supported by a strategic reorganization that included a workforce reduction of approximately 30%, expected to lower 12-month operating expenses by around 25%.

Program (Indication)	Platform/Vector	Latest Status (Late 2025)	Key Efficacy/Status Metric
KRESLADI™ (Severe LAD-I)	Lentiviral (LV)	BLA Resubmission Accepted	100% overall survival at 12 months post-infusion.
RP-A501 (Danon Disease)	AAV	Clinical Hold lifted in August 2025	Dosing for Phase 2 pivotal trial to resume in 1H 2026.
RP-A601 (PKP2-ACM)	AAV	Received RMAT Designation	All 3 adult patients showed increased PKP2 protein expression.
RP-A701 (BAG3-DCM)	AAV	IND Clearance Received	Preparing for first-in-human evaluation.

Regenerative Medicine Advanced Therapy (RMAT) designation for RP-A601.

The U.S. Food and Drug Administration (FDA) granted RMAT designation to RP-A601 for PKP2-ACM. This designation was supported by Phase 1 trial data where all three adult patients treated with a single dose showed improved heart structure and function. Specific data points included:

• Increased PKP2 protein expression, with 110% & 398% increase in 2 patients with low baseline levels.
• 34-41 point increases in KCCQ-12 scores.
• NYHA class improvement from II to I.

This designation enables more intensive FDA guidance and an expedited review process for the therapy, which marks Rocket Pharmaceuticals, Inc.'s fifth RMAT designation.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Customer Relationships

You're building a commercial foundation for gene therapies, which means the relationship with the treating centers and the medical community is everything. For Rocket Pharmaceuticals, Inc., this is a high-touch, highly specialized engagement model, especially given the complexity of administering these one-time treatments.

High-touch, direct engagement with specialized treatment centers and physicians.

The focus is on centers capable of handling complex gene therapy administration. This requires deep scientific exchange, not just sales calls. For instance, the PKP2-ACM program, targeting about 50,000 people in the U.S. and Europe, necessitates working with specialized cardiology centers experienced in inherited heart disease. The collective cardiovascular portfolio targets over 100,000 patients across the U.S. and EU. Engagement is driven by clinical milestones; for example, the FDA lifting the clinical hold on the RP-A501 Danon disease study in August 2025 immediately intensified coordination with those treating centers to resume dosing of additional patients in the first half of 2026. The specific dosing protocol for the next three patients involves a recalibrated dose of 3.8 × 10¹³ GC/kg with a modified immunomodulatory regimen, which requires precise physician training and oversight.

Close collaboration with patient advocacy groups for rare disease communities.

Rocket Pharmaceuticals, Inc. views patients and families as its North Star, which translates directly into deep ties with advocacy groups. A key example is the company's longstanding collaboration with the Danon Foundation, where their commitment to research, advocacy, and patient support is integral to advancing the field. This partnership structure helps Rocket understand the patient journey, which is crucial when dealing with devastating rare disorders.

• Patients and families are the guiding force behind the mission.
• The company cultivates trusted partnerships with advocacy organizations.

Direct communication with regulatory agencies (FDA, EMA) for complex submissions.

Regulatory dialogue is constant and critical, especially given the late-stage nature of several assets. The relationship with the FDA has been highly active through 2025. You saw the FDA accept the Biologics License Application (BLA) resubmission for KRESLADI™ in October 2025, which has a Prescription Drug User Fee Act (PDUFA) date set for March 28, 2026. Furthermore, the company secured a Regenerative Medicine Advanced Therapy (RMAT) Designation from the FDA for RP-A601 (PKP2-ACM) in July 2025, following positive Phase 1 data. The ability to align with the FDA quickly to lift the clinical hold on the RP-A501 Danon disease trial in under three months in August 2025 demonstrates a working, if intense, relationship with health authorities.

Dedicated medical affairs and commercial teams for patient support and access.

As Rocket Pharmaceuticals, Inc. moves toward potential commercialization, the structure of its customer-facing teams has been refined. Sarbani Chaudhuri joined as Chief Commercial & Medical Affairs Officer in April 2025, bringing experience from Johnson & Johnson, AstraZeneca, and Pfizer, specifically in rare cardiac and hematology diseases. However, the company made a strategic pivot in mid-2025, reducing its workforce by approximately 30% to align with pipeline prioritization, which impacts the scale of these dedicated teams. This cost-saving measure is reflected in the financial reporting.

Metric/Expense Category	Q3 2025 Amount (USD)	Comparison Period	Change
General and Administrative (G&A) Expenses	$18.4 million	Q3 2024	Decrease of $8.7 million
Commercial Preparation-Related Expenses (Included in G&A)	Not specified	Q3 2024	Decrease of $6.6 million
Total Employees	299 (As of late 2025)	N/A	Workforce reduced by approx. 30% in 2025

The decrease of $6.6 million in commercial preparation-related expenses within G&A expenses for the third quarter of 2025, compared to the third quarter of 2024, shows the immediate financial impact of the strategic realignment away from broader commercial readiness for paused programs.

Personalized patient support programs for complex gene therapy administration.

For the Danon disease program, the Phase 2 pivotal trial is designed around a small, highly monitored group. The trial evaluates efficacy and safety in 12 patients, which includes a pediatric run-in cohort of n=2. This small number underscores the personalized, intensive support required for each patient receiving an investigational gene therapy like RP-A501. The company's overall cash position as of September 30, 2025, was $222.8 million, which is being managed to fund operations into the second quarter of 2027, ensuring resources are available for these critical, high-touch patient interactions.

Finance: draft 13-week cash view by Friday.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Channels

You're looking at the distribution and communication channels for Rocket Pharmaceuticals, Inc. (RCKT) as they transition from late-stage development to potential commercialization for their rare disease gene therapies in late 2025. Given the nature of these treatments, the channels are highly specialized, focusing on centers of excellence and regulatory milestones.

Highly specialized gene therapy treatment centers (ATCs) for administration

For gene therapies targeting ultra-rare disorders like severe Leukocyte Adhesion Deficiency-I (LAD-I) with KRESLADI™, the channel is inherently limited to specialized centers capable of administering complex cell and gene therapies. While a specific number of Authorized Treatment Centers (ATCs) isn't publicly quantified as of late 2025, the strategy is clearly focused on high-acuity sites. The market opportunity for their cardiovascular pipeline alone-Danon disease, PKP2-ACM, and BAG3-DCM-represents over 100,000 patients in the U.S. and EU, suggesting a targeted network of transplant or specialized cardiology centers will be necessary for future launch. The company's internal focus on AAV cardiovascular programs reinforces this need for specialized infusion sites.

Direct sales and medical affairs teams targeting key opinion leaders (KOLs)

Rocket Pharmaceuticals, Inc. is still pre-revenue as of the third quarter ending September 30, 2025, reporting $0.0 million in revenue. Therefore, the team build-out is focused on medical affairs and commercial preparation rather than broad sales execution. The company made key leadership additions in 2025 to bolster this, including appointing Sarbani Chaudhuri as Chief Commercial & Medical Affairs Officer in April 2025. Commercial preparation-related expenses were noted at $1.5 million for the three months ended March 31, 2025. The channel strategy involves engaging Key Opinion Leaders (KOLs) within the specific rare disease communities, such as the estimated 50,000 PKP2-ACM affected individuals in the U.S. and Europe.

Regulatory submission pathways (BLA/MAA) to the FDA and European Medicines Agency

Regulatory milestones are the primary channel driver for a pre-commercial company. Rocket Pharmaceuticals, Inc. has seen significant shifts in this area in late 2025, prioritizing programs with clearer pathways.

Program/Therapy	Regulatory Action/Status (as of late 2025)	Agency
KRESLADI™ (LAD-I)	FDA acceptance of BLA resubmission in October 2025. Complete BLA submission anticipated before the end of 2025.	FDA
KRESLADI™ (LAD-I)	PDUFA date anticipated in March 2026.	FDA
RP-L102 (Fanconi Anemia)	Voluntarily withdrew U.S. BLA on October 3, 2025, based on strategic considerations.	FDA
RP-L102 (Fanconi Anemia)	Withdrew Marketing Authorization Application (MAA) in July 2025.	European Medicines Agency (EMA)

The company is focusing resources on its AAV cardiovascular programs, which have clear biomarker opportunities for accelerated approval.

Clinical trial sites for patient identification and initial treatment access

Clinical trial sites serve as the initial, most critical access points for patients, especially for therapies addressing conditions like Danon disease, which affects an estimated 15,000 to 30,000 people in the US and Europe. The channel is defined by the active studies:

• Phase 2 pivotal study for RP-A501 (Danon disease) is ongoing following the lifting of a clinical hold.
• Phase 1 trial start-up activities are underway for RP-A701 (BAG3-DCM).
• Initial data from the Phase 1 study of RP-A601 (PKP2-ACM) was expected in May 2025.

The company has experienced a 30% workforce reduction to align resources with these late-stage cardiovascular programs.

Scientific publications and conferences to disseminate clinical data

Dissemination of clinical data through peer-reviewed channels and scientific presentations is a key channel for establishing credibility and informing the medical community. Rocket Pharmaceuticals, Inc. actively participated in major industry events in 2025 to present its pipeline progress.

• Presented updates at the Morgan Stanley 23rd Annual Global Healthcare Conference in September 2025.
• Presented at the 8th Annual Evercore Healthcare Conference in December 2025.
• Corporate Presentation and specific data updates (RP-A601, RP-A501) were shared in November 2025.

The company's experience from treating over 15 cardiac patients informs its late-stage AAV programs.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Customer Segments

You're looking at the patient populations Rocket Pharmaceuticals, Inc. (RCKT) is targeting with its gene therapy pipeline as of late 2025. The focus is clearly on ultra-rare, genetically defined diseases, especially in the cardiovascular space, which dictates resource allocation.

The core customer segments are defined by specific, devastating genetic disorders requiring one-time, potentially curative treatments. The company has strategically prioritized its AAV cardiovascular platform, which includes three major cardiomyopathy programs, alongside its LV hematology program for severe LAD-I.

Here's a breakdown of the addressable patient populations based on the latest internal estimates and prevalence data:

Disease Indication	Therapy Program	Estimated Prevalence (U.S. and Europe)	Market Significance
PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM)	RP-A601 (AAV)	Approximately 50,000 people	Largest market opportunity in disclosed pipeline programs
Danon disease	RP-A501 (AAV)	15,000 to 30,000 patients	Late-stage clinical program
PKP2-ACM and BAG3-DCM (Combined with Danon Disease)	RP-A601 (AAV) and RP-A701 (AAV)	More than 100,000 patients	Collectively target major genetically defined cardiomyopathies
BAG3-associated dilated cardiomyopathy (BAG3-DCM)	RP-A701 (AAV)	Included in the >100,000 total for the three cardiomyopathies	Preclinical/IND accepted status

The other critical segment involves patients with severe LAD-I, for whom KRESLADI™ is seeking approval. This is a severe pediatric genetic disorder causing life-threatening infections.

• Pediatric and adult patients with severe Leukocyte Adhesion Deficiency-I (LAD-I) targeting a March 28, 2026 PDUFA date for KRESLADI™.
• Ultra-rare disease populations, definitely defined by specific genetic mutations, as the entire portfolio is focused on monogenic disorders.

The financial reality of supporting these segments as of the third quarter of 2025 shows significant investment in R&D:

• Q3 2025 Net Loss was $50.3 million.
• Total Operating Expense for Q3 2025 was $52.2 million.
• Research and Development Expenses for Q3 2025 accounted for $34.1 million.
• Cash, cash equivalents, and investments stood at $75.9 million at the end of Q3 2025.
• The company expects current resources to fund operations into the second quarter of 2027.

The cash position at the end of Q1 2025 was $318.2 million.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Cost Structure

You're looking at the cost side of the ledger for Rocket Pharmaceuticals, Inc. (RCKT) as of late 2025. For a late-stage biotech focused on gene therapy, the cost structure is heavily weighted toward the science and the path to the clinic and market. It's all about burning cash to create future value, so every dollar spent on R&D and trials is a strategic decision.

Here's a quick look at the headline operating expenses from the third quarter ending September 30, 2025:

Expense Category	Q3 2025 Amount
Research and Development (R&D) Expenses	$34.1 million
General and Administrative (G&A) Expenses	$18.4 million
Total Operating Expenses (R&D + G&A)	$52.5 million

The primary cost driver, as you'd expect, is Research and Development. For the three months ended September 30, 2025, Rocket Pharmaceuticals reported R&D expenses of $34.1 million. This figure reflects a disciplined approach to resource allocation following the company's organizational realignment earlier in the year.

General and Administrative costs also factor in significantly, though they saw a reduction year-over-year. G&A expenses totaled $18.4 million for the third quarter of 2025, down from $27.1 million in the same period of 2024. This decrease was largely due to lower commercial preparation-related expenses, which dropped by $6.6 million, plus a $1.5 million reduction in non-cash stock-based compensation expense.

The high cost of in-house cGMP manufacturing and quality control is an inherent part of Rocket Pharmaceuticals' structure, especially given their Cranbury, N.J. R&D and manufacturing facility. While specific standalone costs aren't broken out, the R&D expense line reflects these activities. For instance, the R&D spend in Q3 2025 included decreases in manufacturing and development and direct material costs of $3.6 million.

Clinical trial costs for late-stage pivotal studies represent a major, lumpy expense. Rocket Pharmaceuticals is heavily invested in advancing its AAV cardiovascular gene therapy platform, which includes:

• Phase 2 pivotal study for Danon disease (RP-A501).
• Phase 1 study for PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) (RP-A601).

The focus on these programs means significant outlay for patient enrollment, monitoring, and data analysis. The Q3 2025 R&D reduction was partly due to a $2.4 million decrease in clinical trial expenses compared to the prior year, showing the impact of cost optimization efforts.

Finally, there are one-time charges related to strategic shifts. Rocket Pharmaceuticals incurred restructuring and severance charges of approximately $3.3 million in 2025 as part of its reorganization plan to narrow pipeline focus and extend its operational runway into the second quarter of 2027. That's a short-term hit to manage the long-term burn rate.

Finance: draft 13-week cash view by Friday.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Revenue Streams

You're looking at the revenue picture for Rocket Pharmaceuticals, Inc. as of late 2025. Honestly, the current picture is what you'd expect for a company deep in late-stage development, but the near-term catalysts are clear.

Current product revenue is $0.0 million for Q3 2025 (pre-commercial stage). This reflects the reality that Rocket Pharmaceuticals does not yet have commercialized products generating sales, which is consistent with their reported net loss of $50.3 million for that same quarter ending September 30, 2025.

The first significant non-dilutive revenue opportunity is the potential sale of a Priority Review Voucher (PRV) upon KRESLADI™ approval. Analysts estimate this PRV could be valued at over $150 million, which would directly support the company's cash position, currently reported at $222.8 million as of September 30, 2025.

Future product sales of KRESLADI™ (marnetegragene autotemcel; marne-cel) for severe Leukocyte Adhesion Deficiency-I (LAD-I) are tied to a key regulatory date. The Prescription Drug User Fee Act (PDUFA) target action date set by the FDA is March 28, 2026. The data supporting this Biologics License Application (BLA) showed compelling results in the global Phase 1/2 study:

Metric	Result
Overall Survival at 12 Months Post-Infusion	100%
Primary and Secondary Endpoints Met	Yes
Treatment-Related Serious Adverse Events	0

The company has strategically narrowed its focus to maximize the value of its AAV cardiovascular gene therapy portfolio, which represents the next major revenue driver post-KRESLADI™. This focus was reinforced by a workforce reduction of approximately 30% to extend the cash runway into the second quarter of 2027, excluding any PRV proceeds.

This AAV portfolio is centered on rare cardiomyopathies, with specific programs providing future sales potential:

• RP-A501 for Danon Disease (late-stage pivotal Phase 2 trial resuming in 1H 2026).
• RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM) (advancing toward a pivotal Phase 2 study).
• RP-A701 for BAG3-associated dilated cardiomyopathy (DCM) (preparing for first-in-human evaluation).

The market potential for these assets is substantial; for instance, the Danon disease treatment market is projected to grow at a 6.5% CAGR through 2030. The company is betting on this concentrated pipeline for long-term value creation.

Finally, potential upfront or milestone payments from out-licensing non-core assets remain a possible, though secondary, revenue stream. Given the recent strategic reorganization and the decision to focus exclusively on the AAV cardiovascular platform, any non-core asset divestiture would be a deliberate action to further conserve cash and fund the prioritized pipeline.