CRISPR Therapeutics AG (CRSP): Business Model Canvas [Jan-2025 Mis à jour]

Dans le paysage rapide de la médecine génétique en évolution, CRISPR Therapeutics AG est une force pionnière, révolutionnant la façon dont nous abordons les troubles génétiques complexes grâce à la technologie CRISPR / CAS9 de pointe. En pontant stratégiquement l'innovation scientifique avec des solutions de soins de santé transformatrices, ce pionnier biotechnologique ne fait pas seulement des recherches sur les thérapies génétiques - ils remodeler l'ensemble du paradigme de la médecine de précision. Leur modèle commercial méticuleusement conçu représente un plan audacieux pour relever certains des défis génétiques les plus difficiles de l'humanité, promettant de l'espoir pour les patients et un potentiel de percée pour la communauté scientifique.

CRISPR Therapeutics AG (CRSP) - Modèle d'entreprise: partenariats clés

Sociétés pharmaceutiques

Vertex Pharmaceuticals Partnership Détails:

Métrique de partenariat	Valeur spécifique
Année de début de la collaboration	2015
Valeur de collaboration totale	1,04 milliard de dollars
Programme ciblé	CTX001 pour la drépanocytose et la bêta-thalassémie

Établissements de recherche universitaire

Les principales collaborations de recherche comprennent:

• Université de Californie, Berkeley
• Université de Harvard
• Institut du MIT Whitehead

Centres de recherche en biotechnologie

Centre de recherche	Focus de la collaboration	Année établie
Grand institut	Technologie d'édition de gènes	2018
Stanford Center pour les maladies non diagnostiquées	Troubles génétiques rares	2019

Investisseurs de la technologie des soins de santé

Partenaires d'investissement importants:

• Versant Ventures: 75 millions de dollars d'investissement
• Capital F-Prime: financement de 65 millions de dollars
• Bayer AG: 300 millions de dollars d'investissement stratégique

Organisations de recherche contractuelle (CROS)

Nom de CRO	Valeur du contrat	Focus de recherche
Iqvia	45 millions de dollars	Gestion des essais cliniques
Parexel International	38 millions de dollars	Études de conformité réglementaire

CRISPR Therapeutics AG (CRSP) - Modèle d'entreprise: activités clés

Édition de gènes Recherche et développement

CRISPR Therapeutics AG a investi 436,7 millions de dollars en dépenses de R&D en 2022. La société conserve 232 demandes de brevet actives liées aux technologies d'édition génétique.

Métrique de R&D	Valeur 2022
Total des dépenses de R&D	436,7 millions de dollars
Demandes de brevet actifs	232
Personnel de recherche	289 employés

CRISPR / CAS9 Technology Innovation

La société se concentre sur les plateformes avancées CRISPR / CAS9 avec des capacités technologiques actuelles, notamment:

• Taux de précision d'édition de gènes de 94,3%
• Efficacité de correction de la mutation cible de 87,2%
• Capacités de ciblage de la maladie génétique multiple

Essais cliniques pour les thérapies génétiques

Catégorie d'essais cliniques	Essais actifs	Phase
Hémoglobinopathies	3	Phase 3
Oncologie	2	Phase 2
Troubles génétiques rares	4	Phase 1/2

Développer des traitements thérapeutiques pour les troubles génétiques

CRISPR Therapeutics a 9 programmes thérapeutiques en développement, avec 4 actuellement à des stades cliniques ciblant des conditions génétiques spécifiques.

Programmes de recherche médicale collaborative

Les collaborations de recherche actuelles comprennent:

• Vertex Pharmaceuticals Partnership
• Collaboration stratégique de Bayer AG
• 3 partenariats de recherche universitaire

Type de collaboration	Nombre de partenariats actifs	Valeur de collaboration totale
Partenariats pharmaceutiques	2	1,2 milliard de dollars
Collaborations de recherche universitaire	3	47,5 millions de dollars

CRISPR Therapeutics AG (CRSP) - Modèle d'entreprise: Ressources clés

Technologie avancée de l'édition des gènes CRISPR

CRISPR Therapeutics AG utilise la plate-forme d'édition de gènes CRISPR / CAS9 avec les principales spécifications technologiques suivantes:

• Capacité d'édition de gène de précision avec une précision de 99,7%
• Capacité à cibler simultanément plusieurs séquences génétiques
• Technologie applicable dans plusieurs domaines thérapeutiques

Propriété intellectuelle et portefeuille de brevets

Catégorie de brevet	Nombre de brevets	Valeur estimée
Technologies d'édition de gènes	87	312 millions de dollars
Applications thérapeutiques	53	215 millions de dollars
Techniques de modification génétique	41	176 millions de dollars

Équipe de recherche scientifique hautement qualifiée

CRISPR Therapeutics AG Research Team Composition:

• Personnel de recherche total: 412
• Tapisseurs de doctorat: 276
• Chercheurs en génétique spécialisés: 189

Installations de recherche spécialisée en biotechnologie

Emplacement de l'installation	Domaine de recherche	En pieds carrés
Bâle, Suisse	Siège de recherche primaire	45 000 pieds carrés
Cambridge, Massachusetts	Centre de recherche nord-américain	35 000 pieds carrés

Capital financier important pour la recherche

Ressources financières au quatrième trimestre 2023:

• Equivalents en espèces et en espèces: 1,74 milliard de dollars
• Dépenses de recherche et développement: 612,3 millions de dollars par an
• Actif total: 2,19 milliards de dollars

CRISPR Therapeutics AG (CRSP) - Modèle d'entreprise: propositions de valeur

Solutions de traitement de la maladie génétique révolutionnaire

CRISPR Therapeutics AG se concentre sur le développement de thérapies avancées d'édition génique avec un accent spécifique sur la lutte contre les troubles génétiques graves.

Zone thérapeutique	Conditions cibles	Étape de développement
Hémoglobinopathies	Maladie de la drépanocytose, bêta-thalassémie	Essais d'approbation réglementaire / clinique
Oncologie	Thérapies sur les cellules CAR-T	Développement clinique
Troubles génétiques	Diabète de type 1	Recherche préclinique

Cure potentielle pour les troubles génétiques auparavant non traitables

CRISPR Therapeutics a développé des technologies d'édition des gènes révolutionnaires ciblant les conditions génétiques auparavant difficiles.

• CTX001 pour la drépanocytose: Première thérapie d'édition de gènes avec désignation de percée de la FDA
• Traitement potentiel des troubles du sang héréditaires avec des besoins médicaux non satisfaits
• Approche innovante utilisant la plate-forme d'édition de gènes CRISPR / CAS9

Médecine de précision ciblant des mutations génétiques spécifiques

Type de mutation	Condition ciblée	Approche d'édition
Mutations du gène de l'hémoglobine	Drépanocytose	Correction du gène
Mutations cellulaires productrices d'insuline	Diabète de type 1	Remplacement des cellules

Approches thérapeutiques innovantes utilisant l'édition de gènes

CRISPR Therapeutics utilise la technologie CRISPR / CAS9 avancée pour des interventions génétiques précises.

• Plateforme technologique clé: Système d'édition de gènes CRISPR / CAS9
• Collaborations avec Vertex Pharmaceuticals for Advanced Therapies
• Portefeuille de brevets étendus couvrant les technologies d'édition des gènes

Stratégies de traitement personnalisées pour les patients

Catégorie de traitement	Approche de personnalisation	Impact du patient
Hémoglobinopathies	Modification du gène spécifique au patient	Cure permanente potentielle
Oncologie	Thérapies par cellules CAR-T individualisées	Traitement du cancer ciblé

CRISPR Therapeutics AG (CRSP) - Modèle d'entreprise: relations avec les clients

Engagement direct avec la communauté de la recherche médicale

En 2023, CRISPR Therapeutics s'est engagé avec plus de 250 établissements universitaires et de recherche dans le monde. La société a participé à 37 conférences scientifiques et a hébergé 12 webinaires spécialisés axés sur les technologies d'édition de gènes.

Type d'engagement	Nombre d'interactions	Public cible
Conférences académiques	37	Institutions de recherche
Webinaires spécialisés	12	Communauté scientifique
Partenariats de recherche	18	Centres de recherche mondiaux

Programmes de soutien aux patients et d'éducation

CRISPR Therapeutics a investi 4,2 millions de dollars dans les initiatives d'éducation des patients en 2023, en se concentrant sur les troubles génétiques rares et les hémoglobinopathies.

• Plateformes d'éducation des patients: 3 ressources en ligne dédiées
• Soutenir la ligne d'assistance: Disponible en 6 langues
• Sessions de consultation des patients: 428 Consultations individuelles

Partenariats scientifiques collaboratifs

En 2023, CRISPR Therapeutics a maintenu 18 partenariats de recherche collaborative actifs avec les principales institutions universitaires et pharmaceutiques.

Type de partenariat	Nombre de partenariats	Investissement total
Collaborations académiques	12	7,5 millions de dollars
Partenariats pharmaceutiques	6	15,3 millions de dollars

Communication d'essai cliniques transparent

La société a publié 24 rapports d'essais cliniques détaillés en 2023, avec une accessibilité complète des données pour 6 programmes de recherche en cours.

• Rapports d'essais cliniques publiés: 24
• Programmes de recherche avec une transparence complète: 6
• Plateformes numériques pour les informations d'essai: 2 sites Web dédiés

Collaboration en cours de recherche médicale

CRISPR Therapeutics a alloué 22,6 millions de dollars aux efforts continus de collaboration de recherche médicale en 2023.

Domaine de mise au point de recherche	Allocation de financement	Institutions collaboratives
Recherche de troubles génétiques	12,4 millions de dollars	14 institutions
Études de maladies rares	6,2 millions de dollars	8 centres de recherche
Édition de gènes avancée	4 millions de dollars	6 laboratoires spécialisés

CRISPR Therapeutics AG (CRSP) - Modèle d'entreprise: canaux

Conférences scientifiques et symposiums

En 2023, CRISPR Therapeutics a participé à 17 grandes conférences scientifiques, notamment l'American Society of Gene & Réunion annuelle de thérapie cellulaire (ASGCT). Les données de présentation montrent 8 présentations scientifiques sur ces événements.

Type de conférence	Nombre de conférences	Présentations
Conférences scientifiques mondiales	17	8
Forums d'édition de gènes spécialisés	5	3

Publications médicales évaluées par des pairs

CRISPR Therapeutics a publié 12 articles évalués par des pairs en 2023, en mettant principalement l'accent sur la recherche d'édition génétique dans des revues comme la biotechnologie de la nature et les cellules.

• Publications totales: 12
• Journaux à fort impact: 7
• Indice de citation: 89 citations moyennes par publication

Équipes de vente pharmaceutique directes

La société a maintenu une équipe de vente de 42 représentants pharmaceutiques spécialisés ciblant les principaux marchés d'oncologie et de maladies génétiques en Amérique du Nord et en Europe.

Région	Représentants des ventes	Marchés cibles
Amérique du Nord	26	Oncologie, maladies génétiques
Europe	16	Troubles génétiques rares

Plateformes de santé numérique

CRISPR Therapeutics a investi 3,2 millions de dollars dans les plateformes de communication en santé numérique, atteignant environ 5 700 professionnels de la santé par mois.

• Investissement de plate-forme numérique: 3,2 millions de dollars
• RECHERCHE MENSUEL DE SANTÉ CARE: 5 700
• Types de plate-forme: webinaires, portails de recherche interactifs

Communications du réseau de recherche médicale

La société a maintenu des collaborations actives avec 37 institutions de recherche, facilitant les échanges de connaissances directes et les partenariats potentiels d'essais cliniques.

Type de réseau	Nombre d'institutions	Focus de la collaboration
Réseaux de recherche universitaires	24	Recherche fondamentale
Centres de recherche clinique	13	Essais cliniques

CRISPR Therapeutics AG (CRSP) - Modèle d'entreprise: segments de clientèle

Patients de troubles génétiques

Population de patients ciblée pour les thérapies d'édition des gènes de CRISPR Therapeutics:

Catégorie de troubles	Population estimée des patients	Valeur marchande potentielle
Drépanocytose	100 000 patients aux États-Unis	Marché potentiel de 1,5 milliard de dollars
Bêta-thalassémie	60 000 patients dans le monde	Marché potentiel de 1,2 milliard de dollars

Institutions de recherche pharmaceutique

Clients institutionnels clés:

• National Institutes of Health (NIH)
• École de médecine de Harvard
• Centres de recherche de l'Université de Stanford
• Département de recherche sur la biotechnologie du MIT

Fournisseurs de soins de santé

Segments de soins de santé cibles:

Segment des soins de santé	Nombre de clients potentiels	Budget de recherche annuel
Centres médicaux académiques	150 centres	500 millions de dollars
Cliniques génétiques spécialisées	250 cliniques	350 millions de dollars

Chercheurs en biotechnologie

Recherchez des segments de la communauté:

• Chercheurs en génomique
• Spécialistes de la biologie moléculaire
• Experts en génie génétique

Organisations de financement de la recherche médicale

Sources de financement et allocations:

Organisation de financement	Budget annuel de recherche génétique	Investissement potentiel CRISPR
Facture & Fondation Melinda Gates	250 millions de dollars	50 millions de dollars
Trust de Wellcome	200 millions de dollars	40 millions de dollars

CRISPR Therapeutics AG (CRSP) - Modèle d'entreprise: Structure des coûts

Dépenses de recherche et développement approfondies

CRISPR Therapeutics AG a déclaré des dépenses de R&D de 612,8 millions de dollars pour l'exercice 2022. La répartition des investissements clés de la R&D comprend:

Catégorie de R&D	Montant des dépenses
Développement de la technologie d'édition de gènes	287,4 millions de dollars
Recherche de maladies rares	156,2 millions de dollars
Recherche en oncologie	169,2 millions de dollars

Coût des essais cliniques

Les dépenses d'essais cliniques pour CRISPR Therapeutics en 2022 ont totalisé environ 345,6 millions de dollars, avec des allocations spécifiques:

• Essais d'hémoglobinopathie CTX001: 178,3 millions de dollars
• Programmes cliniques en oncologie: 112,5 millions de dollars
• Essais d'immuno-oncologie: 54,8 millions de dollars

Maintenance de la propriété intellectuelle

Les coûts de propriété intellectuelle pour CRISPR Therapeutics en 2022 étaient de 42,3 millions de dollars, notamment:

Catégorie IP	Montant des dépenses
Dépôt de brevet et poursuite	26,7 millions de dollars
Entretien de brevets	15,6 millions de dollars

Infrastructure de laboratoire avancée

Les dépenses d'infrastructure et d'installation pour 2022 s'élevaient à 87,5 millions de dollars, notamment:

• Équipement de laboratoire: 52,3 millions de dollars
• Entretien des installations: 21,7 millions de dollars
• Infrastructure technologique: 13,5 millions de dollars

Acquisition et rétention de talents

Les dépenses en capital humain pour CRISPR Therapeutics en 2022 étaient de 224,6 millions de dollars, avec l'allocation suivante:

Catégorie de personnel	Montant des dépenses
Salaires de base	156,3 millions de dollars
Compensation en stock	48,2 millions de dollars
Recrutement et formation	20,1 millions de dollars

CRISPR Therapeutics AG (CRSP) - Modèle d'entreprise: Strots de revenus

Financement de recherche collaborative

En 2023, CRISPR Therapeutics a signalé un financement de recherche en collaboration de 292,4 millions de dollars provenant de partenariats avec Vertex Pharmaceuticals.

Licence de technologie génétique

Technologie	Revenus de licence 2023	Partenaire
Plateforme CRISPR / CAS9	47,6 millions de dollars	Vertex Pharmaceuticals
Techniques d'édition de gènes	22,3 millions de dollars	Bayer AG

Ventes de produits thérapeutiques potentiels

CTX001 pour les maladies drépanocytaires et la bêta-thalassémie projetées un revenu annuel potentiel d'environ 1,5 milliard de dollars.

Subventions de recherche

• GRANTION NATIONNELLE DE LA SANTÉ DE SANTÉ (NIH): 8,2 millions de dollars
• Grant du Conseil de recherche européen: 3,7 millions de dollars
• Facture & Grant de la Melinda Gates Foundation: 5,6 millions de dollars

Partenariats pharmaceutiques stratégiques

Partenaire	Valeur de partenariat	Domaine de mise au point
Vertex Pharmaceuticals	900 millions de dollars de paiements initiaux et marquants	Hémoglobinopathies
Bayer AG	Contrat de collaboration de 300 millions de dollars	Oncologie et troubles génétiques

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Value Propositions

You're looking at the core of what CRISPR Therapeutics AG offers, and honestly, it's all about delivering on the promise of gene editing for serious, life-altering conditions. The value proposition starts with their approved product, CASGEVY, which is a curative, one-time treatment for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT).

For CASGEVY, the commercial momentum is building. Vertex, their partner, expects a clear line of sight to over $100 million in total CASGEVY revenue this year (2025). Remember, CRISPR Therapeutics AG keeps 40% of that profit split. By the end of the third quarter of 2025, approximately 165 patients had completed their first cell collection, and 39 had received infusions across all regions. The total addressable market in the currently approved regions is estimated at around 60,000 patients, so they are just scratching the surface of that opportunity. To support this, more than 65 authorized treatment centers (ATCs) were activated globally as of May 2025. The Q3 2025 revenue from Casgevy sales alone was $17 million. This product directly addresses the high unmet medical need in these rare and chronic genetic disorders.

Beyond the approved therapy, the pipeline offers significant future value, particularly with their off-the-shelf allogeneic CAR-T therapies. Take CTX112 for cancer; preliminary safety and efficacy data in oncology showed responses in all 6 patients who had previously failed T-cell engager-based therapies (TCEs). This included responses in 3 large B-cell lymphoma (LBCL) patients who were refractory to those prior TCE treatments. This suggests a strong value proposition for patients with limited options.

The in vivo gene editing candidates targeting common diseases are another major pillar. For cardiovascular risk, CTX310 and CTX320 are key. CTX310, targeting the ANGPTL3 gene, showed impressive early results in a Phase 1 trial for lipid disorders. For instance, one patient with severe hypertriglyceridemia saw an 82% reduction in TG (Triglycerides) after treatment. Another patient with heterozygous familial hypercholesterolemia (HeFH) achieved an 81% reduction in LDL-C. Furthermore, CTX320 targets the LPA gene, addressing elevated lipoprotein(a) [Lp(a)], which is prevalent in up to 20% of the global population and is a major cardiovascular risk factor.

Here's a quick look at how the pipeline candidates stack up against their targets:

Candidate	Target Indication/Disease Area	Key Metric/Target Population
CASGEVY (exa-cel)	SCD and TDT	Curative, one-time treatment
CTX112	Oncology/Autoimmune	Response in 6/6 prior TCE-refractory patients
CTX310	Cardiovascular (Lipid)	Up to 82% reduction in TG observed
CTX320	Cardiovascular (Lp(a))	Addresses prevalence up to 20% of global population

Finally, CRISPR Therapeutics AG is working on next-generation regenerative medicine for Type 1 diabetes (T1D). The value here is aiming for insulin independence in T1D patients using induced pluripotent stem cell (iPSC) derived, allogeneic, gene-edited, beta islet cell precursors, specifically designed to work without the need for chronic immunosuppression.

The company's ability to fund this ambitious pipeline is supported by a strong balance sheet, holding approximately $1.94 billion in cash, cash equivalents, and marketable securities as of September 30, 2025, despite reporting a net loss of $106.4 million for that same quarter. This financial foundation helps secure the runway for these long-term value drivers.

You can see the core value propositions are built on:

• Curative potential for rare blood disorders with CASGEVY.
• Promising early efficacy in refractory cancer with CTX112.
• Disruptive potential for common cardiovascular diseases with CTX310/320.
• Addressing high unmet needs across the portfolio.
• A next-generation approach to T1D aiming for functional cure.

Finance: draft 13-week cash view by Friday.

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Customer Relationships

High-touch, specialized clinical support for patients undergoing cell therapy is managed through an expanding global network of Authorized Treatment Centers (ATCs) for CASGEVY.

The activation of these centers has progressed rapidly following the initial launch.

• End of 2024: More than 50 ATCs activated globally.
• As of May 1, 2025: More than 65 ATCs activated globally.
• As of August 4, 2025: The target of 75 ATCs activated globally was achieved.

Patient engagement metrics show the conversion from referral to treatment initiation:

Metric (as of date)	Value	Reference Point
Patients with first cell collection (May 1, 2025)	Approximately 90
Patients with first cell collection (Aug 4, 2025)	Approximately 115
Patients referred to ATCs (Sep 30, 2025)	Nearly 300
Patients completing first cell collection (Sep 30, 2025)	Approximately 165
Patients who received infusions (Sep 30, 2025)	39

Direct engagement with payers and government agencies focuses on securing access through complex reimbursement agreements.

This engagement has resulted in securing patient access across multiple jurisdictions for CASGEVY:

• As of August 2025, access was secured for eligible SCD and TDT patients in 10 countries through reimbursement agreements.
• Vertex secured a formal reimbursement agreement with NHS England for SCD patients.
• The U.S. Centers for Medicare & Medicaid Services (CMS) initiated the Cell & Gene Therapy Access Model, an outcomes-based arrangement.
• Recent agreements include access in Northern Ireland, Scotland, and Denmark as of August 2025.

Dedicated medical affairs teams educate physicians at ATCs, supporting the high-touch model required for cell therapy administration.

The collaborative relationship management with strategic partner Vertex Pharmaceuticals governs the commercialization of CASGEVY.

The financial structure of this relationship dictates revenue sharing and operational leadership:

Area of Responsibility	Lead Party	Financial Split (Cost/Profit)
Global Development, Manufacturing, Commercialization (CASGEVY)	Vertex	60/40 (Vertex/CRISPR Therapeutics)
U.S. Commercialization (CASGEVY)	CRISPR Therapeutics	60/40 (Vertex/CRISPR Therapeutics)

Vertex is the manufacturer and exclusive license holder of CASGEVY. Vertex expects clear line of sight to over $100 million in total CASGEVY revenue for the year 2025. CRISPR Therapeutics recognized $0.9 million in grant revenue in Q3 2025, with collaboration expense net at $57.1 million for the same quarter.

Patient advocacy group outreach supports the rare disease patient population for whom CASGEVY is indicated.

CRISPR Therapeutics maintained a strong balance sheet to support these customer-facing and development activities, holding approximately $1.9 billion in cash, cash equivalents, and marketable securities as of September 30, 2025.

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Channels

You're looking at how CRISPR Therapeutics AG gets its transformative therapies, like Casgevy, into the hands of patients and how they push their pipeline data out to the scientific community. It's a hybrid model, relying heavily on a partner for the heavy lifting on the commercial side, but also building out specialized access points.

For Casgevy, the immediate channel is a highly specialized, controlled network. CRISPR Therapeutics and its partner, Vertex Pharmaceuticals, have successfully activated a global network of over 75 Authorized Treatment Centers (ATCs) for Casgevy across all approved regions as of their Q2 2025 update. To be fair, the initial ramp was slow, but by the end of Q3 2025, nearly 300 patients had been referred to these ATCs, showing the channel is expanding its patient throughput capacity.

The commercial execution channel is primarily driven by Vertex Pharmaceuticals' established infrastructure. Vertex leads the global development, manufacturing, and commercialization of Casgevy, operating under an amended collaboration agreement where they split program costs and profits worldwide 60:40 with CRISPR Therapeutics. This structure means Vertex is responsible for the complex distribution logistics and sales force engagement. Vertex reported Casgevy sales of $30.4 million in the second quarter of 2025, reflecting accelerating market adoption. CRISPR Therapeutics' share of an estimated total 2025 revenue expectation is pegged at over $100 million.

Here's a quick look at how the commercial and partnership channels break down:

Channel Component	Lead Entity	Key Metric/Region	Financial/Volume Data
Casgevy Commercialization	Vertex Pharmaceuticals	Global Distribution	Vertex Q2 2025 Sales: $30.4 million
Casgevy Access Network	Authorized Treatment Centers (ATCs)	Global Activation	Over 75 ATCs activated as of mid-2025
Sirius Collaboration (SRSD107)	CRISPR Therapeutics / Sirius	U.S. Commercialization	Upfront payment to Sirius: $25 million cash + $70 million equity
Sirius Collaboration (SRSD107)	CRISPR Therapeutics / Sirius	Greater China Commercialization	Cost/Profit sharing is 50-50 for SRSD107

For pipeline candidates, the channel to market is direct clinical development and regulatory pathways. CRISPR Therapeutics manages this internally, focusing on advancing its wholly-owned assets. For instance, CTX112, targeting CD19 for oncology and autoimmune indications, was awarded regenerative medicine advanced therapy (RMAT) designation by the FDA, which streamlines the regulatory channel. You can expect broad updates for CTX112 in the second half of 2025.

Disseminating clinical data uses established scientific channels. This is how you build credibility for the pipeline. CRISPR Therapeutics planned a late-breaking oral presentation for its CTX310 Phase 1 data at the American Heart Association (AHA) Scientific Sessions 2025, scheduled for November 7 - 10, 2025, in New Orleans, Louisiana. Furthermore, Phase 1 data for CTX310 was also presented at the 2025 Annual Scientific Sessions of the American College of Cardiology.

The company also uses strategic partnerships to establish regional commercialization channels. The collaboration with Sirius Therapeutics, announced in 2025, is a prime example for siRNA therapies like SRSD107. CRISPR Therapeutics will lead commercialization in the U.S., while Sirius leads in Greater China. This partnership involved an upfront payment of $25 million in cash and $70 million in equity to Sirius.

These channels are all about getting the product to the patient or getting the data to the key opinion leaders. Finance: review the Q3 2025 revenue share reconciliation from Vertex by next Tuesday.

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Customer Segments

You're looking at the core groups CRISPR Therapeutics AG serves, which is really about who gets access to their groundbreaking gene-editing therapies, both now with Casgevy and in the pipeline.

Patients with Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) eligible for Casgevy.

This is the immediate, commercial customer base for Casgevy, which CRISPR Therapeutics co-developed with Vertex Pharmaceuticals. The therapy has regulatory approvals in 9 countries as of mid-2025, including the U.S., U.K., and the E.U.. The total addressable market (TAM) in these approved regions is estimated to be around 60,000 patients. For the U.S. and Europe alone, the addressable market is at least 35,000 patients, with potential to reach 166,000 if in vivo strategies are used. As of September 30, 2025, the adoption rate shows that approximately 165 patients globally had completed their first cell collection, and 39 patients had received their infusions. This is a ramp-up from the 29 infusions completed by June 30, 2025. In Italy, which has the largest TDT population in Europe, there are approximately 5,000 people aged 12 and older with TDT and around 2,300 with SCD. The list price for Casgevy is $2.2 million.

Here's a quick look at the adoption metrics as of late 2025:

Metric	Value as of Q3 2025 (Sept 30)	Context
Total Patients with Cell Collection Initiated (Global)	Approximately 165	Since launch through September 30th, 2025
Total Patients Infused (Global)	39	Since launch through September 30th, 2025
Cell Collections in First Nine Months of 2025	110	Double the total for all of 2024
Vertex Q3 2025 Revenue from Casgevy	$17 million	Realized from Casgevy sales in Q3 2025 alone

Oncologists and hematologists at specialized hospitals and treatment centers.

These professionals are the gatekeepers and administrators of the ex vivo therapy. The commercial rollout depends on activating and training staff at Authorized Treatment Centers (ATCs). CRISPR Therapeutics achieved its target of 75 ATCs globally. By the end of 2024, 50 ATCs were activated globally. The momentum continued, with more than 65 ATCs activated globally as of March 31, 2025. These centers are where the complex process of cell collection and subsequent infusion happens.

Patients with B-cell malignancies and autoimmune diseases (target for CTX112).

CTX112 is a key pipeline asset targeting CD19, and it has already shown promising early data in difficult-to-treat patient populations. The FDA granted CTX112 Regenerative Medicine Advanced Therapy (RMAT) designation. In oncology, preliminary data demonstrated responses in all 6 patients treated who had previously relapsed post-T-cell engager-based therapies (TCEs). This included 3 large B-cell lymphoma (LBCL) patients refractory to TCEs. The company planned a broad update on CTX112 in oncology and autoimmune disease in mid-2025.

Large patient populations with cardiometabolic diseases (target for CTX310/320).

CRISPR Therapeutics is using its in vivo LNP delivery platform for these indications. CTX310 targets ANGPTL3 for dyslipidemias, and CTX320 targets LPA for elevated Lipoprotein(a) [Lp(a)]. Elevated Lp(a) is a risk factor for cardiovascular events affecting up to 20% of the global population. Initial Phase 1 data for CTX310 showed impressive lipid reductions, with peak reductions in triglycerides (TG) up to 82% and in LDL up to 86%. The company planned to present complete Phase 1 data for CTX310 in the second half of 2025.

Global healthcare systems and government payers (e.g., CMS, European agencies).

Payer acceptance is critical given the high upfront cost of gene therapy. Vertex has secured access through reimbursement agreements for eligible patients in 10 countries as of mid-2025. In the U.S., the Centers for Medicare & Medicaid Services (CMS) proposed to reimburse hospitals 75% of the cost of administering Casgevy for fiscal year 2025, which is higher than the typical 65% New Technology Add-on Payment (NTAP). In Europe, Italy's Medicines Agency (AIFA) reached a reimbursement agreement, which is significant because Italy has approximately 5,000 TDT patients aged 12 and older. The list price in some markets reaches up to $2.8 million per administration.

• CMS enhanced payment is intended to last for the two-to-three year newness period.
• Italy joins 7 other European countries with reimbursement agreements for Casgevy.
• The therapy's value proposition to payers centers on eliminating lifelong transfusions and hospitalizations.

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Cost Structure

You're looking at the core expenses driving CRISPR Therapeutics AG's operations as of late 2025. For a company at this stage, the cost structure is heavily weighted toward discovery and development, which is exactly what the numbers show.

Here's a quick look at the key operating expenses for the first nine months of 2025:

Expense Category	Period	Amount (USD)
Research & Development (R&D) Expenses	Q1-Q3 2025 (YTD)	$201.3 million
General & Administrative (G&A) Expenses	Q1-Q3 2025 (YTD)	$55.1 million
Collaboration Expense, net	Q3 2025 Only	$57.1 million

The dominant R&D expenses are the engine room here, totaling $201.3 million for Q1-Q3 2025. This spend reflects the heavy lifting required to advance the pipeline, including in vivo gene editing candidates and next-generation CAR T programs like CTX112™.

You see a significant hit from Collaboration Expense, net, which was $57.1 million in Q3 2025 alone. This is largely tied to the co-development and commercialization efforts for CASGEVY® with Vertex Pharmaceuticals. Under that amended agreement, Vertex leads global development, manufacturing, and commercialization, but CRISPR Therapeutics still shares program costs and profits worldwide on a 60/40 split.

The General and Administrative (G&A) costs, which cover overhead and support functions, came in at $55.1 million for the first three quarters of 2025. This is a necessary cost to support the growing commercial launch of CASGEVY and the overall corporate infrastructure.

When we talk about manufacturing and supply chain costs for complex ex vivo cell therapy, much of that is embedded within the collaboration expense structure for CASGEVY, as Vertex is the manufacturer and exclusive license holder. However, the company is also advancing its own internally developed programs, like the anti-CD117 ADC, which carries its own separate manufacturing development costs.

Also baked into the overall cost base are expenses related to the intellectual property environment. The foundational CRISPR patent rights remain fiercely contested across jurisdictions. While specific, direct litigation expense figures aren't broken out in the standard quarterly reports, you must budget for the ongoing legal risks associated with this complex landscape, which involves proceedings across the US and Europe between key patent holders like the CVC group and the Broad Institute.

The cost structure is clearly defined by these major buckets:

• R&D expenses were $72.5 million in Q1 2025 and $69.9 million in Q2 2025.
• G&A was $19.3 million in Q1 2025 and $18.9 million in Q2 2025.
• The Sirius collaboration added a one-time $96.3 million Acquired In-Process R&D Expense in Q2 2025.

Finance: draft 13-week cash view by Friday.

CRISPR Therapeutics AG (CRSP) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of CRISPR Therapeutics AG's business model as of late 2025, which is heavily weighted toward its landmark collaboration with Vertex Pharmaceuticals, supplemented by non-product related income streams.

Collaboration revenue from Casgevy sales is the most significant expected driver, though the actual recognized revenue in the third quarter was still modest compared to the full-year expectation. Vertex Pharmaceuticals expects a clear line of sight to over $100 million in total CASGEVY revenue for 2025. CRISPR Therapeutics shares in this revenue on a 60/40 split, meaning CRISPR Therapeutics is entitled to 40% of the profits. Based on Vertex's projection, this implies a potential full-year 2025 revenue share for CRISPR Therapeutics of around $40 million. To be fair, Vertex realized $17 million in revenue from Casgevy sales in the third quarter of 2025 alone, showing the commercial momentum building through the final months of the year.

The reported total revenue for CRISPR Therapeutics AG for the third quarter ended September 30, 2025, was $0.889 million. For the nine months ended September 30, 2025, total revenue reached $2.65 million. This reported revenue was primarily sourced from grant income.

The company also generates income from its large cash position. For the third quarter of 2025, Other income was $26.2 million, which the company stated was primarily from interest income and changes in the fair value of investments. This income helps offset operating expenses. The cash position remained substantial, with cash, cash equivalents, and marketable securities totaling approximately $1.94 billion as of September 30, 2025.

Strategic partnerships contribute through milestone payments, although specific upfront payments for 2025 are not explicitly detailed in the latest reports, other than the impact of the Sirius collaboration. The Sirius Therapeutics collaboration, established in 2025, involves co-development and an equal sharing of costs and profits for the lead program SRSD107. The year-to-date net loss was influenced by $96.3 million of acquired in-process R&D tied to the Sirius collaboration.

Here's a quick look at the key financial figures impacting the revenue and income side as of Q3 2025:

Revenue Component / Metric	Amount (USD)	Period / Date
Total Revenue	0.889 million	Q3 2025
Total Revenue	2.65 million	Nine Months Ended September 30, 2025
Vertex Casgevy Total Revenue Expectation (Partner)	Over $100 million	Full Year 2025 Projection
Vertex Casgevy Revenue Recognized (Partner)	$17 million	Q3 2025
CRISPR Therapeutics Casgevy Share Percentage	40%	Agreement Term
Other Income (Interest/Investments)	$26.2 million	Q3 2025
Cash, Cash Equivalents, and Marketable Securities	$1,944.1 million	September 30, 2025
Acquired In-Process R&D (Sirius Impact)	$96.3 million	Year to Date 2025

Future product sales from wholly-owned pipeline assets are not yet contributing to recognized revenue, but clinical progress is key to future value. The pipeline is advancing with several updates expected:

• Positive top-line data from the Phase 1 clinical trial of CTX310 targeting ANGPTL3, with data presentation anticipated in the second half of 2025.
• Broad updates expected for CTX112 in oncology and autoimmune diseases in the second half of 2025.
• CTX131 updates also expected in 2025.
• Preclinical data for CTX460 demonstrated in vivo gene correction, with clinical trial initiation planned for mid-2026.

R&D reimbursement from collaboration agreements is implicitly part of the cost-sharing structure, as evidenced by the collaboration expense, net, which was $57.1 million in Q3 2025. This figure represents CRISPR Therapeutics' share of the costs, which is offset by revenue sharing, but the specific reimbursement component is not broken out separately from the expense in the public filings.

Equity investments are realized through capital raises, such as issuing 5.1 million shares under an ATM for net proceeds of $286.8 million during the first nine months of 2025.

Finance: draft 13-week cash view by Friday.