Proqr Therapeutics N.V. (PRQR): Business Model Canvas [Jan-2025 Mis à jour]

Proqr Therapeutics N.V. (PRQR) émerge comme une entreprise de biotechnologie révolutionnaire révolutionnant le paysage du traitement des maladies génétiques rares grâce à son approche thérapeutique innovante de l'ARN. En tirant parti de la biologie moléculaire de pointe et des technologies d'édition génétique, cette organisation pionnière transforme notre façon de comprendre et potentiellement guérir les troubles génétiques auparavant inexploités. Leur modèle commercial unique combine l'excellence scientifique, les partenariats stratégiques et un engagement axé sur le laser à développer des interventions génétiques personnalisées qui pourraient offrir de l'espoir aux patients souffrant de conditions génétiques rares et complexes.

Proqr Therapeutics N.V. (PRQR) - Modèle d'entreprise: partenariats clés

Établissements de recherche universitaire

Proqr Therapeutics collabore avec les établissements de recherche académiques suivants:

Institution	Focus de recherche	Détails de collaboration
Centre médical de l'Université de Leiden	Recherche de maladies génétiques	Développement thérapeutique de l'ARN
École de médecine de Harvard	Troubles génétiques rares	Collaboration de génétique moléculaire

Sociétés pharmaceutiques

Les collaborations pharmaceutiques clés comprennent:

• Novartis - Partenariat pour les thérapies par maladies génétiques rares
• Roche - Collaboration de développement de médicaments

Organisations de recherche contractuelle (CROS)

ProQR travaille avec plusieurs CRO pour les essais cliniques:

Nom de CRO	Phase d'essai clinique	Études actives
Iqvia	Phase II / III	3 essais de troubles génétiques en cours
Parxel	Phase I / II	2 essais thérapeutiques de l'ARN

Laboratoires de tests génétiques

Partenariats avec les laboratoires de tests génétiques:

• Invitae Corporation - Dépistage génétique des patients
• Santé génomique - collaboration de diagnostic moléculaire

Biotechnology Investment Installations

Partners de soutien au financement:

Entreprise d'investissement	Montant d'investissement	Année d'investissement
Conseillers orbimés	45 millions de dollars	2022
Versant Ventures	30 millions de dollars	2021

Proqr Therapeutics N.V. (PRQR) - Modèle d'entreprise: Activités clés

Recherche et développement thérapeutiques de l'ARN

ProQR Therapeutics se concentre sur le développement de la thérapeutique à l'ARN pour les maladies génétiques rares. En 2023, la société a investi 37,4 millions de dollars dans les frais de recherche et de développement.

Domaine de recherche	Investissement (2023)	Programmes actifs
Développement thérapeutique de l'ARN	37,4 millions de dollars	4 programmes primaires de maladies génétiques

Édition de gènes et technologie de réparation de l'ARN innovation

L'entreprise utilise des technologies de plate-forme de montage d'ARN propriétaire en mettant l'accent sur:

• Technologie d'édition ARNA axiomère
• Techniques d'édition de l'ARN de précision
• Plateforme Linc pour les modifications génétiques

Gestion des essais précliniques et cliniques

Phase de procès	Nombre d'essais en cours	Investissement total
Essais précliniques	3 programmes	12,6 millions de dollars
Essais cliniques	2 essais actifs	22,8 millions de dollars

Développement de médicaments génétiques rares

ProQR se concentre sur le développement de la thérapeutique pour des troubles génétiques rares spécifiques, avec un ciblage actuel du pipeline:

• Fibrose kystique
• Syndrome d'Usher
• Dystrophie musculaire de Duchenne

Protection de la propriété intellectuelle et dépôt de brevets

Catégorie de brevet	Nombre de brevets	Durée de protection des brevets
Technologie d'édition d'ARN	17 brevets accordés	20 ans à compter de la date de dépôt
Méthodes thérapeutiques génétiques	9 demandes de brevet en instance	Protection potentielle de 20 ans

Proqr Therapeutics N.V. (PRQR) - Modèle d'entreprise: Ressources clés

Technologie de plate-forme de montage d'ARN propriétaire

Proqr Therapeutics maintient une plate-forme d'édition d'ARN spécialisée avec les caractéristiques clés suivantes:

Attribut technologique	Détails spécifiques
Nom de la plate-forme	Plate-forme d'édition d'ARN
Demandes de brevet	17 familles de brevets à partir de 2023
Focus technologique	Édition et modification de base d'ARN

Expertise spécialisée de la recherche génétique

Les capacités de recherche de Proqr comprennent:

• 15 chercheurs dévoués
• Expérience de recherche moyenne de 12,5 ans
• Expertise dans les troubles génétiques rares

Installations avancées de laboratoire de biologie moléculaire

Spécification de l'installation	Données quantitatives
Espace de laboratoire total	2 500 mètres carrés
Investissement d'équipement avancé	4,3 millions de dollars en 2023
Instruments de recherche	42 Instruments de biologie moléculaire spécialisés

Portefeuille de propriété intellectuelle

Détails IP clés:

• 17 familles de brevets
• Couvrant les technologies d'édition d'ARN
• Couverture géographique à travers nous, UE et Asie

Équipe scientifique et de recherche qualifiée

Composition de l'équipe	Données quantitatives
Personnel de recherche total	45 employés
Titulaires de doctorat	28 chercheurs
Investissement de formation annuelle	$620,000

Proqr Therapeutics N.V. (PRQR) - Modèle d'entreprise: propositions de valeur

Thérapies d'ARN ciblées pour des troubles génétiques rares

ProQR Therapeutics se concentre sur le développement de thérapies d'ARN ciblant spécifiquement des troubles génétiques rares avec des options de traitement limitées ou non existantes.

Zone thérapeutique	Trouble cible	Étape de développement	Population potentielle de patients
Fibrose kystique	Mutation f508del	Phase 2/3 essais cliniques	Environ 30 000 à 70 000 patients dans le monde
Syndrome d'Usher	Mutation génétique de type 2	Étape préclinique	Estimé 4-17 pour 100 000 individus

Traitements de percée potentielles pour des conditions non traitables

La plate-forme d'édition de l'ARN propriétaire de Proqr permet des interventions potentielles pour les conditions génétiques qui manquent actuellement de solutions thérapeutiques.

• Technologie de réparation d'ARN unique ciblant des mutations génétiques spécifiques
• Approche de la médecine de précision avec des interventions génétiques personnalisées
• Mécanismes de correction moléculaire avancés

Stratégies d'intervention des maladies génétiques personnalisées

La proposition de valeur de l'entreprise se concentre sur le développement de thérapies ARN personnalisées adaptées à des mutations génétiques spécifiques.

Plate-forme technologique	Capacité de personnalisation	Impact potentiel
Plate-forme d'édition d'ARN	Ciblage spécifique à la mutation	Traitement potentiel pour des conditions génétiques auparavant non traitables

Technologies de réparation moléculaire avancée

Les capacités technologiques de ProQR comprennent les plates-formes d'édition d'ARN propriétaires conçues pour traiter les mutations génétiques au niveau moléculaire.

• Investissement technologique: 42,3 millions de dollars en dépenses de R&D (2022 Exercice)
• Mécanismes de réparation de l'ARN breveté
• Stratégies d'intervention génétique de précision

Approche innovante de la gestion des maladies génétiques

La proposition de valeur distinctive de Proqr réside dans son approche complète de la gestion des maladies génétiques grâce à des technologies ARN avancées.

Métrique d'innovation	Valeur	Avantage comparatif
Portefeuille de brevets	17 brevets accordés	Protection technologique unique
Collaboration de recherche	3 partenariats pharmaceutiques actifs	Développement technologique amélioré

Proqr Therapeutics N.V. (PRQR) - Modèle d'entreprise: relations avec les clients

Engagement direct avec les communautés de patients atteints de maladies rares

Proqr Therapeutics s'engage avec les communautés de patients grâce à des stratégies de sensibilisation ciblées:

Groupe communautaire patient	Méthode d'engagement	Fréquence d'interaction annuelle
Patients atteints de fibrose kystique	Réseaux de support en ligne	12-15 événements virtuels
Patients atteints de maladie rétinienne	Boards consultatifs des patients	6-8 réunions stratégiques
Communautés de troubles génétiques	Plateformes de communication numérique	24 points de contact numériques

Présentations des conférences scientifiques et des symposiums médicaux

Proqr participe activement à des conférences scientifiques pour présenter les progrès de la recherche:

• Participation annuelle: 8-10 Conférences médicales internationales
• Formats de présentation: présentations orales, séances d'affiches, panneaux d'experts
• Conférences cibles: symposiums de maladies rares, conférences de thérapie génétique

Partenariats de recherche collaborative

Proqr maintient des collaborations de recherche stratégique:

Type de partenaire	Nombre de partenariats actifs	Investissement de recherche annuel
Institutions universitaires	5-7 partenariats	3,2 millions de dollars
Hôpitaux de recherche	3-4 projets collaboratifs	2,1 millions de dollars
Centres de recherche pharmaceutique	2-3 initiatives de recherche conjointes	1,5 million de dollars

Programmes de soutien aux patients et d'éducation

ProQR met en œuvre des initiatives complètes de soutien aux patients:

• Ressources éducatives en ligne: 15-20 modules d'information
• Aide à la patiente: Disponibilité du soutien 24/7
• Services de conseil génétique: options de consultation personnalisées

Communication transparente sur les progrès de la recherche

Stratégies de communication pour la transparence de la recherche:

Canal de communication	Fréquence des mises à jour	Atteindre
Site Web de l'entreprise	Mises à jour de la recherche mensuelles	Accès mondial des parties prenantes
Communications des relations avec les investisseurs	Rapports de progression trimestriels	Communauté des investisseurs et analystes
Souvances de publication scientifique	4-6 publications évaluées par les pairs chaque année	Communauté de recherche scientifique

Proqr Therapeutics N.V. (PRQR) - Modèle d'entreprise: canaux

Publications scientifiques directes

Proqr Therapeutics publie des recherches dans des revues à comité de lecture telles que:

Nom de journal	Fréquence de publication	Facteur d'impact
Biotechnologie de la nature	Mensuel	41.4
Cellule	Bihebdomadaire	38.6
Médecine translationnelle scientifique	Hebdomadaire	16.9

Présentations de la conférence médicale

Détails de la participation annuelle de la conférence:

Conférence	Emplacement	Déteau de présentation
Société américaine de gène & Thérapie cellulaire	Baltimore, MD	4
Société européenne du gène & Thérapie cellulaire	Barcelone, Espagne	3

Biotechnology Industry Investor Relations

Canaux de communication des investisseurs:

• Appels de résultats trimestriels
• Réunions annuelles des actionnaires
• Présentations des investisseurs
• Communications de classement SEC

Plateformes de communication scientifique en ligne

Plates-formes de fiançailles numériques:

Plate-forme	Abonnés / membres	Type de contenu
Researchgate	12,500	Publications de recherche
Liendin	8,700	Mises à jour de l'entreprise

Événements de réseautage de l'industrie pharmaceutique

Plates-formes de réseautage clés:

• Biotechnology Innovation Organisation (BIO) Convention internationale
• Conférence JP Morgan Healthcare
• Conférence de partenariat génétique

Proqr Therapeutics N.V. (PRQR) - Modèle d'entreprise: segments de clientèle

Patiens de maladies génétiques rares

ProQR Therapeutics se concentre sur les patients souffrant de troubles génétiques rares spécifiques. En 2024, la société cible environ 2 000 à 5 000 patients dans le monde pour chaque condition génétique.

Trouble génétique	Population estimée des patients	Taille du marché cible
Fibrose kystique (mutation f508del)	30 000 à 40 000 patients	Amérique du Nord et Europe
Syndrome d'Usher	4 000 à 6 000 patients	Mondial

Institutions de recherche génétique

ProQR collabore avec 15-20 institutions de recherche les plus importantes dans le monde, en se concentrant sur la recherche sur les maladies génétiques.

• École de médecine de Harvard
• Département de génétique de l'Université de Stanford
• Université de Californie, San Francisco

Médecins spécialisés

Le groupe de clients cible comprend environ 500 à 700 généticiens et des professionnels de la santé spécialisés dans le monde entier.

Spécialité	Nombre de pratiquants	Focus géographique
Spécialistes génétiques	350-450	États-Unis
Cliniciens de maladies rares	150-250	Europe et Amérique du Nord

Départements de recherche pharmaceutique

ProQR s'engage avec 25-30 départements de recherche pharmaceutique pour des collaborations et des partenariats potentiels.

• Unité de recherche de maladies rares Pfizer
• Thérapies génétiques Novartis
• Diagnostic moléculaire de Roche

Investisseurs en biotechnologie

En 2024, ProQR attire les investissements de la biotechnologie spécialisée et des groupes d'investisseurs axés sur les maladies rares.

Type d'investisseur	Nombre d'investisseurs	Plage d'investissement total
Sociétés de capital-risque	8-12	50-80 millions de dollars
Investisseurs institutionnels	15-20	100 à 150 millions de dollars

Proqr Therapeutics N.V. (PRQR) - Modèle d'entreprise: Structure des coûts

Frais de recherche et de développement

Pour l'exercice 2022, Proqr Therapeutics a déclaré des dépenses totales de R&D de 52,4 millions de dollars. La répartition de ces dépenses est la suivante:

Catégorie de R&D	Montant de dépenses ($)
Frais de personnel	22,1 millions
Coûts de recherche externes	18,3 millions
Matériaux de laboratoire	7,6 millions
Licence de technologie	4,4 millions

Coûts de gestion des essais cliniques

Les dépenses d'essai cliniques pour 2022 ont totalisé 34,6 millions de dollars, l'allocation suivante:

• Frais d'essai de phase I / II: 15,2 millions de dollars
• Frais d'essai de phase III: 19,4 millions de dollars

Entretien de la propriété brevet et intellectuelle

Proqr a investi 3,2 millions de dollars dans la protection de la propriété intellectuelle et l'entretien des brevets en 2022.

Équipements de laboratoire et investissements technologiques

Les investissements technologiques et d'équipement pour 2022 étaient de 8,7 millions de dollars, notamment:

Catégorie d'investissement	Montant ($)
Technologie d'édition de gènes	4,3 millions
Équipement de séquençage avancé	2,9 millions
Outils de biologie informatique	1,5 million

Compensation du personnel scientifique

La rémunération totale du personnel scientifique pour 2022 était de 28,5 millions de dollars, structurée comme suit:

• Salaires de base: 18,2 millions de dollars
• Compensation à base d'actions: 6,3 millions de dollars
• Bonus de performance: 4 millions de dollars

Structure totale des coûts pour 2022: 126,9 millions de dollars

Proqr Therapeutics N.V. (PRQR) - Modèle d'entreprise: Strots de revenus

Accords potentiels de licence de médicament potentiel

Depuis le quatrième trimestre 2023, ProQR Therapeutics a des accords de licence potentiels dans le développement, avec un accent spécifique sur les thérapies par la maladie génétique rares.

Zone thérapeutique	Valeur de licence potentielle	État actuel
Troubles génétiques rares	12,5 millions de dollars de paiement initial potentiel	Négociations en cours
Thérapeutique à l'ARN	25 millions de dollars de paiement potentiel	Discussions à un stade précoce

Subventions de recherche et financement gouvernemental

ProQR a obtenu un financement de recherche à partir de plusieurs sources.

Source de financement	Montant d'octroi	Année
Programme européen Horizon 2020	3,6 millions d'euros	2023
Conseil de recherche néerlandais	1,2 million d'euros	2023

Contrats de développement pharmaceutique collaboratif

Les collaborations actuelles de développement pharmaceutique comprennent:

• Collaboration avec Vertex Pharmaceuticals
• Partenariat avec Leiden University Medical Center
• Alliance de recherche avec les instituts de recherche sur les maladies génétiques

Ventes de produits thérapeutiques potentiels

Projection de revenus pour les produits thérapeutiques potentiels:

Produit thérapeutique	Potentiel des revenus annuels estimés	Marché cible
Sépofarsen (dystrophie rétinienne héréditaire)	15-20 millions de dollars	Ophtalmologie
Candidats thérapeutiques à l'ARN	10-15 millions de dollars	Troubles génétiques rares

Licence de propriété intellectuelle

Le portefeuille de propriété intellectuelle de Proqr génère des sources de revenus potentiels.

Catégorie IP	Nombre de brevets	Revenus de licence potentielle
Technologies d'édition d'ARN	12 brevets enregistrés	5 à 8 millions de dollars de licence annuelle potentielle
Plateformes de thérapie génétique	8 brevets enregistrés	3 à 6 millions de dollars de licence annuelle potentielle

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Value Propositions

You're looking at the core of what ProQR Therapeutics N.V. is offering the market-it's a fundamental shift in how we treat genetic diseases. The value proposition centers on their proprietary Axiomer™ RNA editing technology platform, which is designed to be a next-generation approach compared to traditional gene therapies.

Pioneering next-generation RNA base editing for genetic diseases

ProQR Therapeutics N.V. is pioneering this next-generation RNA base editing technology called Axiomer™. This technology uses the cell's own editing machinery, specifically ADAR (Adenosine Deaminase Acting on RNA), to make precise, single nucleotide edits directly in the RNA molecule. This DNA-free, reversible editing approach is a key differentiator in the field. The company expects up to four clinical data readouts across its pipeline in 2025 and 2026, signaling an aggressive push to validate the platform.

Correcting disease-causing mutations directly at the RNA level

The core mechanism is the ability to make specific single nucleotide edits in RNA to either reverse a mutation or modulate protein expression. This capability falls under three key applications for the Axiomer pipeline: modulate, correct, and protect. The platform's clinical entry is marked by its lead program, AX-0810, which targets the NTCP protein. The Clinical Trial Application (CTA) for AX-0810 was submitted in Q2 2025, received authorization in October 2025, and initial safety, tolerability, and PK (pharmacokinetics) data from Cohort 1 of the Phase 1 study were expected toward the end of 2025.

Targeting high unmet need diseases like cholestatic diseases (AX-0810)

The immediate focus is on high unmet need areas. AX-0810 is specifically targeting NTCP for cholestatic liver diseases. This investigational RNA editing oligonucleotide is designed to selectively modulate NTCP function to reduce toxic bile acid accumulation in the liver, potentially stopping inflammation, fibrosis, and progression to liver failure. The strategy is grounded in human genetics, as naturally occurring variants like NTCP Q68R are associated with no clinical symptoms related to bile acid levels. The financial backing to pursue this is substantial; as of September 30, 2025, ProQR Therapeutics N.V. held € 106.9 million in cash and cash equivalents, providing a financial runway into mid-2027. This operational spending included Research and development (R&D) costs of € 34.8 million for the nine-month period ending September 30, 2025.

Potential to treat both rare and prevalent diseases with one platform

The platform's value extends beyond liver disease, positioning ProQR Therapeutics N.V. to address a broad spectrum of conditions. The Axiomer technology is intended to yield a new class of medicines for diverse types of diseases, covering both rare and prevalent conditions. The pipeline reflects this diversity, moving beyond the lead candidate. Here's a quick look at the pipeline programs that support this broad potential:

• The lead program, AX-0810, targets NTCP for cholestatic diseases.
• AX-2402 targets MECP2 (R270X) for Rett Syndrome, a rare CNS disorder, supported by a partnership that secured up to $9.2 million from the Rett Syndrome Research Trust.
• AX-2911 is advancing toward clinical candidate selection, targeting MASH (a prevalent liver condition).
• AX-1412 targets B4GALT1 for cardiovascular diseases.

The company's financial health is also supported by external validation, including a collaboration with Eli Lilly, which generated $2.0 million in milestone payments during the first nine months of 2025. The overall net loss for the nine-month period ending September 30, 2025, was € 33.3 million.

Program	Target Indication	Technology Application	Key 2025/2026 Milestone
AX-0810	Cholestatic Liver Diseases (NTCP)	Modulate	Initial Safety/PK Data by year-end 2025
AX-2402	Rett Syndrome (MECP2)	Correct/Modulate	Phase 1/2 Trial planned for 2026
AX-2911	MASH	Modulate	Advancing toward clinical candidate selection in 2025
AX-1412	Cardiovascular Diseases (B4GALT1)	Modulate	Preclinical/Translational updates expected

The platform's ability to address both rare and prevalent diseases using a single, validated RNA editing approach is the central value proposition. If you're looking at the near-term catalysts, the first human data for AX-0810 in Q4 2025 is the key event that will validate the entire Axiomer technology in a clinical setting.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Customer Relationships

ProQR Therapeutics N.V. structures its external relationships around strategic alliances, patient-centric advocacy, and transparent communication with the financial community.

High-touch, collaborative relationships with major pharmaceutical partners

The relationship with Eli Lilly and Company (Lilly) is a cornerstone, representing a significant, long-term collaboration focused on the discovery, development, and commercialization of RNA editing medicines for genetic disorders. This partnership, which began in 2021 and was expanded in 2022, utilizes ProQR Therapeutics N.V.'s proprietary Axiomer™ RNA editing platform. The total potential value of this strategic collaboration is up to $3.9 billion. ProQR Therapeutics N.V. retains an option to expand the collaboration to a total of 15 targets, which would trigger a $50 million opt-in payment to ProQR Therapeutics N.V. The financial flow from this partnership directly supports operations, as evidenced by the $2.0 million (~€ 1.8 million) in milestone income earned by ProQR Therapeutics N.V. during the first nine months of 2025. Lilly also participated in the October 2024 financing, contributing to the $82.1 million in gross proceeds raised then.

Here's a snapshot of the financial interaction with the key pharmaceutical partner:

Metric	Value/Detail
Collaboration Start Year	2021
Total Potential Collaboration Value	$3.9 billion
Potential Opt-in Payment (Additional 5 Targets)	$50 million
Milestone Income (First Nine Months of 2025)	$2.0 million
Cash Reserves from Oct 2024 Financing (Lilly Participation)	$82.1 million (Gross Proceeds)

Close engagement with patient advocacy groups like RSRT

Engagement with patient advocacy groups is critical, particularly for rare diseases like Rett Syndrome. ProQR Therapeutics N.V. has an active, expanded collaboration with the Rett Syndrome Research Trust (RSRT) to advance AX-2402, targeting the MECP2 R270X mutation. This relationship is structured as a co-investment model, where RSRT funding de-risks the program to incentivize full internal development by ProQR Therapeutics N.V. The total funding commitment from RSRT to date is $9.1 million. This total includes an initial $1 million research grant announced in January 2024, followed by an expansion in December 2024 with an additional $8.1 million in funding. ProQR Therapeutics N.V. is matching RSRT's investment in this program.

The RSRT partnership drives specific program milestones:

• Total RSRT funding commitment: $9.1 million
• Initial grant amount (Jan 2024): $1 million
• Additional funding (Dec 2024): $8.1 million
• Program focus: AX-2402 for Rett Syndrome

Investor relations and analyst events for transparency on data readouts

ProQR Therapeutics N.V. maintains active communication with investors and analysts to provide transparency, especially around clinical data milestones. The company hosted a virtual Analyst and Investor Event in November 2025, which detailed the Phase 1 trial design for AX-0810 and outlined expectations for 2025 data. Furthermore, management actively participated in several key industry and investor conferences in late 2025.

Investor engagement events in late 2025 included:

• H.C. Wainwright Genetic Medicines Virtual Conference: October 14-15, 2025 (Presentation available on demand Oct 14 at 7:00 am ET)
• H.C. Wainwright Liver Disease Virtual Conference: October 21-22, 2025 (Presentation available on demand Oct 21 at 7:00 am ET)
• Chardan's 9th Annual Genetic Medicines Conference: October 21, 2025 (Included an RNA Editing panel at 1:00 p.m. ET)
• Virtual Analyst and Investor Event (focused on AX-0810): November 2025
• Evercore Healthcare Conference: December 01, 2025

Archived webcasts from these presentations are typically available for approximately 30 days following the presentation date on the ProQR Therapeutics N.V. website.

Specialized clinical support for trial participants

The relationship with trial participants is managed through rigorous, protocol-driven engagement, particularly as the lead program enters first-in-human testing. ProQR Therapeutics N.V. received Clinical Trial Application (CTA) authorization for AX-0810 and initiated the Phase 1 study in healthy volunteers. The trial is designed to assess specific parameters directly related to the therapeutic approach.

Key elements of the initial clinical engagement for AX-0810 include:

• Trial Status (as of Q3 2025): Initiating Phase 1 study in healthy volunteers.
• Assessment Focus: Safety, tolerability, pharmacokinetics (PK), and target engagement.
• Data Timeline: Initial safety and PK data from Cohort 1 expected by year-end 2025.
• Target Engagement Data: Expected in the first half of 2026 from all cohorts.

The company's cash position as of September 30, 2025, was € 106.9 million, providing a runway into mid-2027, which supports the ongoing operational commitment to these clinical programs.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Channels

You're looking at how ProQR Therapeutics N.V. gets its science and potential therapies in front of partners, regulators, and investors as of late 2025. It's a mix of formal agreements, clinical execution, and scientific dissemination. Honestly, the channels reflect a company deep in the clinical transition phase.

Direct licensing and collaboration agreements with pharmaceutical companies

The core of ProQR Therapeutics N.V.'s external commercial and development channel is through strategic alliances. The most significant is the $3.9 billion strategic collaboration with Eli Lilly and Company ("Lilly"), which started back in 2021 and saw expansion in 2022. This partnership is key for developing their Axiomer® RNA editing platform across multiple targets. ProQR Therapeutics N.V. is still executing on this, having recognized milestone income of $2.0 million through the first nine months of 2025 from Lilly.

Furthermore, the Lilly agreement includes an option for ProQR Therapeutics N.V. to expand to a total of 15 targets, which would trigger a substantial $50 million opt-in payment. On a smaller scale, the collaboration with the Rett Syndrome Research Trust ("RSRT") was expanded in December 2024, securing an additional funding for a total commitment of $9.2 million to support the advancement of AX-2402.

You should also note the historical licensing activity, such as the agreement signed in October 2018 with Ionis Pharmaceuticals for QR-1123, which involved upfront payments totaling $6,001,000 (made in shares) and includes future milestone payments plus royalties of 20% on net sales. To be fair, one older CNS license agreement was terminated as of July 2023.

Clinical trial sites in Europe and North America for drug testing

The primary channel for testing drug candidates like AX-0810 is through formal clinical trials. ProQR Therapeutics N.V. advanced its lead program by submitting a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) in June 2025. Pending regulatory clearance, the first-in-human Phase 1 study for AX-0810 is expected to commence at a single site in the Netherlands. This is the first clinical development milestone for their Axiomer™ platform.

While the initial trial is in Europe, the target patient populations for their lead programs are significant across both regions. For instance, Primary Sclerosing Cholangitis (PSC) and Biliary Atresia (BA) are estimated to affect 80,000 and 20,000 individuals in North America and Europe, respectively. The company maintains a U.S. office in Cambridge, MA, which supports North American operations and engagement.

Here's a quick look at the financial context surrounding these clinical advancements as of late 2025:

Financial Metric (as of September 30, 2025)	Amount (in millions)	Context
Cash and Cash Equivalents	€ 106.9 million	Provides runway into mid-2027.
Net Cash Used in Operating Activities (9 months 2025)	€ 39.4 million	Reflects ongoing R&D and clinical spend.
Research & Development (R&D) Costs (9 months 2025)	€ 34.8 million	Significant investment into pipeline advancement.

Scientific publications and conferences to showcase platform data

Disseminating platform data is a crucial channel for building scientific credibility and attracting future partners or investment. ProQR Therapeutics N.V. actively uses scientific venues to present its Axiomer technology. You can see evidence of this through their participation in key 2025 events, including the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting and the Oligonucleotide and Peptide Therapeutics Conference (TIDES USA) in May, and the RNA Editing Summit in July.

The company also uses dedicated investor events to detail trial designs and data expectations. They hosted a virtual Analyst and Investor Event in the fall of 2025, which focused on the AX-0810 Phase 1 trial design. The company is positioned to deliver initial safety and PK data from the first cohort of the AX-0810 trial by the end of 2025, with target engagement data expected in the first half of 2026. They anticipate up to four clinical data readouts across 2025 and 2026.

• Presentations included data on the Axiomer ADAR-mediated RNA editing platform.
• Specific abstracts presented at ASGCT 2025 related to NTCP and PNPLA3.
• The company plans to provide an update on AX-1412 in mid-2025.

Investor communications for capital market access

Access to capital markets is a direct channel for funding operations, especially given the net loss reported. ProQR Therapeutics N.V. ended the third quarter of 2025 with € 106.9 million in cash and cash equivalents, which supports a financial runway extending well into mid-2027. This follows a successful underwritten public offering in October 2024 that generated gross proceeds of $82.1 million.

The company's financial reporting itself serves as a primary communication channel. They reported a net loss of € 33.3 million for the nine-month period ending September 30, 2025. For ongoing capital market access, ProQR Therapeutics N.V. has several effective registration statements on Form F-3 on file with the SEC, including File No. 333-282419. The company also uses press releases to announce key operational and financial milestones, such as the Q3 2025 results on November 6, 2025.

The key financial metrics related to investor confidence and capital structure as of late 2025 are summarized below:

Financial Event/Data Point	Date/Period	Value
Gross Proceeds from Oct 2024 Offering	October 2024	$82.1 million
Cash & Equivalents	September 30, 2025	€ 106.9 million
Net Loss	Nine Months Ended Sept 30, 2025	€ 33.3 million
Lilly Collaboration Milestone Income	First 9 Months of 2025	$2.0 million

Finance: draft 13-week cash view by Friday.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Customer Segments

You're looking at the core groups ProQR Therapeutics N.V. is targeting with its Axiomer RNA editing platform as of late 2025. These segments are defined by the specific, high unmet medical need diseases their pipeline programs address, and the strategic partners needed to fund and commercialize the technology.

Large pharmaceutical companies seeking novel RNA editing technology represent a major segment, primarily through strategic alliances that provide non-dilutive funding and future commercial reach. The collaboration with Eli Lilly is a prime example of this segment engagement.

The company's financial health, which supports engaging these segments, showed cash and cash equivalents of approximately €106.9 million at September 30, 2025, providing runway into mid-2027.

Partner/Segment Type	Program Focus	Financial Metric/Value	Data Point (as of late 2025)
Large Pharmaceutical Partner (Eli Lilly)	Axiomer Platform Technology	Total Collaboration Value	$3.9 billion (Source 11)
Large Pharmaceutical Partner (Eli Lilly)	Milestone Achievement	Income YTD Q3 2025	$2.0 million (~€ 1.8 million) (Source 1, 6)
Large Pharmaceutical Partner (Eli Lilly)	Potential Future Value	Opt-in Payment for 5 Additional Targets	$50 million (Source 2)

Patients with severe cholestatic liver diseases (NTCP target) are targeted by the lead program, AX-0810. This segment is critical as it represents the first clinical validation of the Axiomer platform.

• Program: AX-0810, modulating NTCP to reduce bile acid accumulation.
• Clinical Status: CTA authorization received in October 2025 (Source 1, 6).
• Trial Location: Phase 1 study initiating at a single site in the Netherlands (Source 8).
• Near-Term Readout: Initial safety, tolerability, and PK data for Cohort 1 expected by year-end 2025 (Source 1, 6).
• Follow-up Readout: Target engagement data across cohorts expected in H1 2026 (Source 1, 6).

Patients with rare neurodevelopmental disorders like Rett Syndrome (MECP2 target) are addressed by the CNS program, AX-2402. This segment is characterized by a severe, rare genetic condition with high unmet need.

• Program: AX-2402, targeting the R270X mutation in the MECP2 gene.
• Patient Population Size: Rett Syndrome affects approximately 350,000 people worldwide (Source 9).
• Targeted Subset: Mutations addressable by this RNA editing approach account for 40% of all Rett cases (Source 10).
• Development Status: Advancing toward clinical candidate selection as of Q2 2025 (Source 8).

Patient advocacy and research foundations funding drug development are essential early-stage customers/supporters, particularly for rare disease programs where patient advocacy drives research funding.

The Rett Syndrome Research Trust (RSRT) is a key example of this segment providing direct, non-dilutive funding to advance the AX-2402 program.

• Foundation Partner: Rett Syndrome Research Trust (RSRT).
• Total Funding Secured: Up to $9.2 million to support advancement into clinical trials (Source 2).
• Funding Context: This funding supports IND-enabling studies, with ProQR Therapeutics N.V. matching the investment (Source 10).

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Cost Structure

You're looking at the core expenditures driving ProQR Therapeutics N.V.'s operations as of late 2025. For a development-stage biotech, the cost structure is almost entirely weighted toward getting the science through the clinic and protecting the underlying technology.

Dominant Research and Development (R&D) expenses are the clear cost leader. For the nine-month period ended September 30, 2025, R&D costs hit € 34.8 million. This is a significant jump from the € 25.7 million reported for the same period in 2024, showing the increased investment as programs mature. Net cash used in operating activities for those nine months was € 39.4 million, meaning R&D accounts for the vast majority of cash burn.

The primary driver for this elevated R&D spend is the Clinical trial costs for AX-0810 and preclinical advancement. ProQR Therapeutics N.V. achieved a major milestone with the CTA authorization and is initiating the first-in-human Phase 1 study for AX-0810, which targets NTCP for cholestatic diseases.

Here's a quick look at how the main operating costs stacked up for the first nine months of 2025:

Cost Category	Amount (9M 2025)	Comparison Point
Research and Development (R&D)	€ 34.8 million	Up from € 25.7 million (9M 2024)
General and Administrative (G&A)	€ 11.2 million	Up from € 9.7 million (9M 2024)
Total Operating Costs (Implied from Net Loss & Revenue)	Approx. € 45.6 million	Net Loss of € 33.3M + Revenue of € 11.2M (9M 2025)

The R&D expenditure covers several critical, high-cost activities:

• Costs associated with the Phase 1 trial initiation for AX-0810.
• Manufacturing and supply chain setup for clinical material.
• Ongoing work supporting preclinical advancement of other pipeline assets, like AX-2402 for Rett Syndrome.
• Platform costs related to the Axiomer RNA editing technology.

General and Administrative (G&A) costs were € 11.2 million for the nine-month period ending September 30, 2025. This reflects the necessary overhead to run a public company advancing clinical trials. This category includes things like executive salaries, finance, and legal functions.

A key, though often bundled, component of the G&A and R&D spend is Intellectual property maintenance and patent prosecution fees. You have to spend money to keep the Axiomer platform protected globally. This is a non-negotiable cost of doing business in this space, ensuring exclusivity for their novel RNA editing approach.

To be fair, some of the cost pressure is offset by partnership income. During the first nine months of 2025, ProQR Therapeutics N.V. achieved milestones from its collaboration agreement with Eli Lilly amounting to $2.0 million, which is roughly € 1.8 million. That income helps temper the overall cash burn, but the underlying operational costs remain high as they push AX-0810 toward initial data readouts by year-end 2025.

Finance: draft 13-week cash view by Friday.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Revenue Streams

You're looking at the core ways ProQR Therapeutics N.V. brings in cash right now, which is heavily weighted toward partnerships and non-dilutive funding as they advance their Axiomer RNA editing platform.

The most immediate, concrete revenue source comes from the strategic collaboration with Eli Lilly and Company (Lilly). During the nine-month period ended September 30, 2025, ProQR Therapeutics N.V. achieved milestones under this agreement amounting to exactly $2.0 million. This is a key near-term income driver while the pipeline matures.

Another vital component of ProQR Therapeutics N.V.'s funding structure involves non-dilutive capital from non-profit organizations. Specifically, the collaboration with the Rett Syndrome Research Trust (RSRT) has been expanded. The total funding secured from RSRT to support the advancement of AX-2402 is $9.1 million, which includes an initial grant of $1.0 million and an additional $8.1 million announced in late 2024.

The potential for significant future revenue is tied directly to the success of these partnerships, particularly the Lilly agreement. This collaboration includes an option for Lilly to expand to a total of 15 targets, which would trigger a substantial future opt-in payment to ProQR Therapeutics N.V. of $50 million.

The long-term revenue vision for ProQR Therapeutics N.V. rests on successful commercialization, which translates into future royalties and product sales once any of their pipeline candidates, like AX-0810, achieve regulatory approval and market entry. Still, as of late 2025, the company's reported revenue for the nine-month period ended September 30, 2025, was €11.22 million.

Here's a quick look at the reported revenue and cash position as of the end of Q3 2025:

Financial Metric	Amount (9M 2025)	Amount (Q3 2025)
Total Sales/Revenue	€11.22 million	EUR 2.88 million
Lilly Milestone Payments	$2.0 million	Not Specified
Cash & Cash Equivalents (as of Sep 30)	€106.9 million	€106.9 million
Net Cash Used in Operating Activities	€39.4 million	Not Specified

The revenue streams can be categorized by their source and expected timing:

• Milestone Payments from Eli Lilly and Company
• Research Grants from Non-Profit Entities (e.g., RSRT)
• Future Opt-In Payments (e.g., $50 million from Lilly expansion)
• Long-Term Royalties and Product Sales

You can see the RSRT funding is a significant non-dilutive source supporting specific program advancement:

• RSRT Total Funding Secured: $9.1 million
• RSRT Additional Funding (Dec 2024): $8.1 million
• Initial RSRT Grant (Jan 2024): $1.0 million

Finance: review the Q4 2025 cash burn rate against the mid-2027 runway projection by next Tuesday.