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CRISPR Therapeutics AG (CRSP) Bundle
In der sich schnell entwickelnden Landschaft der genetischen Medizin gilt die CRISPR Therapeutics AG als Pionierkraft und revolutioniert die Art und Weise, wie wir komplexe genetische Störungen durch modernste CRISPR/Cas9-Technologie angehen. Durch die strategische Verknüpfung wissenschaftlicher Innovationen mit transformativen Gesundheitslösungen erforscht dieser Biotech-Vorreiter nicht nur Gentherapien, sondern gestaltet das gesamte Paradigma der Präzisionsmedizin neu. Ihr sorgfältig ausgearbeitetes Geschäftsmodell stellt einen mutigen Plan für die Bewältigung einiger der größten genetischen Herausforderungen der Menschheit dar und verspricht Hoffnung für Patienten und Durchbruchpotenzial für die wissenschaftliche Gemeinschaft.
CRISPR Therapeutics AG (CRSP) – Geschäftsmodell: Wichtige Partnerschaften
Pharmaunternehmen
Einzelheiten zur Partnerschaft von Vertex Pharmaceuticals:
| Partnerschaftsmetrik | Spezifischer Wert |
|---|---|
| Startjahr der Zusammenarbeit | 2015 |
| Gesamtwert der Zusammenarbeit | 1,04 Milliarden US-Dollar |
| Fokussiertes Programm | CTX001 für Sichelzellenanämie und Beta-Thalassämie |
Akademische Forschungseinrichtungen
Zu den wichtigsten Forschungskooperationen gehören:
- Universität von Kalifornien, Berkeley
- Harvard-Universität
- MIT Whitehead Institute
Forschungszentren für Biotechnologie
| Forschungszentrum | Fokus auf Zusammenarbeit | Gründungsjahr |
|---|---|---|
| Breites Institut | Gen-Editing-Technologie | 2018 |
| Stanford Center für nicht diagnostizierte Krankheiten | Seltene genetische Störungen | 2019 |
Investoren in Gesundheitstechnologie
Bedeutende Investitionspartner:
- Versant Ventures: 75-Millionen-Dollar-Investition
- F-Prime Capital: Finanzierung in Höhe von 65 Millionen US-Dollar
- Bayer AG: 300 Millionen US-Dollar strategische Investition
Auftragsforschungsinstitute (CROs)
| CRO-Name | Vertragswert | Forschungsschwerpunkt |
|---|---|---|
| IQVIA | 45 Millionen Dollar | Klinisches Studienmanagement |
| Parexel International | 38 Millionen Dollar | Studien zur Einhaltung gesetzlicher Vorschriften |
CRISPR Therapeutics AG (CRSP) – Geschäftsmodell: Hauptaktivitäten
Forschung und Entwicklung zur Genbearbeitung
Die CRISPR Therapeutics AG investierte im Jahr 2022 436,7 Millionen US-Dollar in Forschungs- und Entwicklungskosten. Das Unternehmen unterhält 232 aktive Patentanmeldungen im Zusammenhang mit Gen-Editing-Technologien.
| F&E-Metrik | Wert 2022 |
|---|---|
| Gesamte F&E-Ausgaben | 436,7 Millionen US-Dollar |
| Aktive Patentanmeldungen | 232 |
| Forschungspersonal | 289 Mitarbeiter |
CRISPR/Cas9-Technologieinnovation
Das Unternehmen konzentriert sich auf fortschrittliche CRISPR/Cas9-Plattformen mit aktuellen Technologiefunktionen, darunter:
- Gen-Editing-Präzisionsrate von 94,3 %
- Angestrebte Mutationskorrektureffizienz von 87,2 %
- Mehrere Möglichkeiten zur gezielten Bekämpfung genetischer Krankheiten
Klinische Studien für Gentherapien
| Kategorie „Klinische Studie“. | Aktive Versuche | Phase |
|---|---|---|
| Hämoglobinopathien | 3 | Phase 3 |
| Onkologie | 2 | Phase 2 |
| Seltene genetische Störungen | 4 | Phase 1/2 |
Entwicklung therapeutischer Behandlungen für genetische Störungen
CRISPR Therapeutics hat 9 Therapieprogramme in der Entwicklung, von denen sich derzeit 4 in der klinischen Phase befinden und auf bestimmte genetische Erkrankungen abzielen.
Kollaborative medizinische Forschungsprogramme
Zu den aktuellen Forschungskooperationen gehören:
- Partnerschaft mit Vertex Pharmaceuticals
- Strategische Zusammenarbeit der Bayer AG
- 3 akademische Forschungspartnerschaften
| Art der Zusammenarbeit | Anzahl aktiver Partnerschaften | Gesamtwert der Zusammenarbeit |
|---|---|---|
| Pharmazeutische Partnerschaften | 2 | 1,2 Milliarden US-Dollar |
| Akademische Forschungskooperationen | 3 | 47,5 Millionen US-Dollar |
CRISPR Therapeutics AG (CRSP) – Geschäftsmodell: Schlüsselressourcen
Fortschrittliche CRISPR-Genbearbeitungstechnologie
Die CRISPR Therapeutics AG nutzt die Gen-Editing-Plattform CRISPR/Cas9 mit den folgenden wichtigen technologischen Spezifikationen:
- Präzise Genbearbeitungsfunktion mit einer Genauigkeit von 99,7 %
- Fähigkeit, mehrere genetische Sequenzen gleichzeitig anzugreifen
- Technologie, die in mehreren Therapiebereichen anwendbar ist
Geistiges Eigentum und Patentportfolio
| Patentkategorie | Anzahl der Patente | Geschätzter Wert |
|---|---|---|
| Gen-Editing-Technologien | 87 | 312 Millionen Dollar |
| Therapeutische Anwendungen | 53 | 215 Millionen Dollar |
| Genetische Modifikationstechniken | 41 | 176 Millionen Dollar |
Hochqualifiziertes wissenschaftliches Forschungsteam
Zusammensetzung des Forschungsteams der CRISPR Therapeutics AG:
- Gesamtzahl der Forschungsmitarbeiter: 412
- Doktoranden: 276
- Spezialisierte Genforscher: 189
Spezialisierte biotechnologische Forschungseinrichtungen
| Standort der Einrichtung | Forschungsbereich | Quadratmeterzahl |
|---|---|---|
| Basel, Schweiz | Hauptquartier der Primärforschung | 45.000 Quadratfuß |
| Cambridge, Massachusetts | Nordamerikanisches Forschungszentrum | 35.000 Quadratfuß |
Erhebliches Finanzkapital für die Forschung
Finanzielle Ausstattung ab Q4 2023:
- Zahlungsmittel und Zahlungsmitteläquivalente: 1,74 Milliarden US-Dollar
- Forschungs- und Entwicklungsausgaben: 612,3 Millionen US-Dollar pro Jahr
- Gesamtvermögen: 2,19 Milliarden US-Dollar
CRISPR Therapeutics AG (CRSP) – Geschäftsmodell: Wertversprechen
Bahnbrechende Lösungen zur Behandlung genetischer Krankheiten
Die CRISPR Therapeutics AG konzentriert sich auf die Entwicklung fortschrittlicher Gen-Editing-Therapien mit besonderem Schwerpunkt auf der Behandlung schwerer genetischer Störungen.
| Therapeutischer Bereich | Zielbedingungen | Entwicklungsphase |
|---|---|---|
| Hämoglobinopathien | Sichelzellenanämie, Beta-Thalassämie | Behördliche Zulassung/klinische Studien |
| Onkologie | CAR-T-Zelltherapien | Klinische Entwicklung |
| Genetische Störungen | Typ-1-Diabetes | Präklinische Forschung |
Mögliche Heilung für bisher unbehandelbare genetische Störungen
CRISPR Therapeutics hat bahnbrechende Gen-Editing-Technologien entwickelt, die auf zuvor schwierige genetische Erkrankungen abzielen.
- CTX001 gegen Sichelzellenanämie: Erste Gen-Editing-Therapie mit FDA-Durchbruchskennzeichnung
- Potenzielle Behandlung erblicher Bluterkrankungen mit hohem medizinischem Bedarf
- Innovativer Ansatz unter Verwendung der Gen-Editierungsplattform CRISPR/Cas9
Präzisionsmedizin, die auf spezifische genetische Mutationen abzielt
| Mutationstyp | Gezielter Zustand | Bearbeitungsansatz |
|---|---|---|
| Hämoglobin-Genmutationen | Sichelzellenanämie | Genkorrektur |
| Insulinproduzierende Zellmutationen | Typ-1-Diabetes | Zellersatz |
Innovative Therapieansätze mittels Gene Editing
CRISPR Therapeutics nutzt die fortschrittliche CRISPR/Cas9-Technologie für präzise genetische Eingriffe.
- Schlüsseltechnologieplattform: CRISPR/Cas9-Gen-Editierungssystem
- Kooperationen mit Vertex Pharmaceuticals für fortschrittliche Therapien
- Umfangreiches Patentportfolio für Gen-Editing-Technologien
Personalisierte Behandlungsstrategien für Patienten
| Behandlungskategorie | Personalisierungsansatz | Auswirkungen auf den Patienten |
|---|---|---|
| Hämoglobinopathien | Patientenspezifische Genmodifikation | Mögliche dauerhafte Heilung |
| Onkologie | Individualisierte CAR-T-Zelltherapien | Gezielte Krebsbehandlung |
CRISPR Therapeutics AG (CRSP) – Geschäftsmodell: Kundenbeziehungen
Direkte Zusammenarbeit mit der medizinischen Forschungsgemeinschaft
Im Jahr 2023 arbeitete CRISPR Therapeutics mit über 250 akademischen und Forschungseinrichtungen weltweit zusammen. Das Unternehmen nahm an 37 wissenschaftlichen Konferenzen teil und veranstaltete 12 spezialisierte Webinare mit Schwerpunkt auf Gen-Editing-Technologien.
| Engagement-Typ | Anzahl der Interaktionen | Zielgruppe |
|---|---|---|
| Akademische Konferenzen | 37 | Forschungseinrichtungen |
| Spezialisierte Webinare | 12 | Wissenschaftliche Gemeinschaft |
| Forschungspartnerschaften | 18 | Globale Forschungszentren |
Patientenunterstützungs- und Aufklärungsprogramme
CRISPR Therapeutics investierte im Jahr 2023 4,2 Millionen US-Dollar in Initiativen zur Patientenaufklärung mit Schwerpunkt auf seltenen genetischen Störungen und Hämoglobinopathien.
- Plattformen zur Patientenaufklärung: 3 spezielle Online-Ressourcen
- Support-Helpline: Verfügbar in 6 Sprachen
- Patientenberatungssitzungen: 428 Einzelberatungen
Kollaborative wissenschaftliche Partnerschaften
Im Jahr 2023 unterhielt CRISPR Therapeutics 18 aktive Forschungskooperationen mit führenden akademischen und pharmazeutischen Institutionen.
| Partnerschaftstyp | Anzahl der Partnerschaften | Gesamtinvestition |
|---|---|---|
| Akademische Kooperationen | 12 | 7,5 Millionen Dollar |
| Pharmazeutische Partnerschaften | 6 | 15,3 Millionen US-Dollar |
Transparente Kommunikation über klinische Studien
Das Unternehmen veröffentlichte im Jahr 2023 24 detaillierte klinische Studienberichte mit umfassendem Datenzugriff für 6 laufende Forschungsprogramme.
- Veröffentlichte klinische Studienberichte: 24
- Forschungsprogramme mit voller Transparenz: 6
- Digitale Plattformen für Studieninformationen: 2 spezielle Websites
Laufende medizinische Forschungskooperation
CRISPR Therapeutics stellte im Jahr 2023 22,6 Millionen US-Dollar für die kontinuierliche Zusammenarbeit in der medizinischen Forschung bereit.
| Forschungsschwerpunktbereich | Mittelzuweisung | Kollaborative Institutionen |
|---|---|---|
| Forschung zu genetischen Störungen | 12,4 Millionen US-Dollar | 14 Institutionen |
| Studien zu seltenen Krankheiten | 6,2 Millionen US-Dollar | 8 Forschungszentren |
| Erweiterte Genbearbeitung | 4 Millionen Dollar | 6 spezialisierte Labore |
CRISPR Therapeutics AG (CRSP) – Geschäftsmodell: Kanäle
Wissenschaftliche Konferenzen und Symposien
Im Jahr 2023 nahm CRISPR Therapeutics an 17 großen wissenschaftlichen Konferenzen teil, darunter der American Society of Gene & Jahrestagung der Zelltherapie (ASGCT). Die Präsentationsdaten zeigen 8 wissenschaftliche Präsentationen über diese Veranstaltungen hinweg.
| Konferenztyp | Anzahl der Konferenzen | Präsentationen |
|---|---|---|
| Globale wissenschaftliche Konferenzen | 17 | 8 |
| Spezialisierte Gen-Editing-Foren | 5 | 3 |
Von Experten begutachtete medizinische Veröffentlichungen
CRISPR Therapeutics veröffentlichte im Jahr 2023 12 von Experten begutachtete Artikel mit dem Schwerpunkt auf der Gen-Editing-Forschung in Fachzeitschriften wie Nature Biotechnology und Cell.
- Gesamtpublikationen: 12
- Hochwirksame Zeitschriften: 7
- Zitationsindex: 89 durchschnittliche Zitate pro Publikation
Direkte Pharma-Vertriebsteams
Das Unternehmen unterhielt ein Vertriebsteam von 42 spezialisierten Pharmavertretern, die auf die wichtigsten Märkte für Onkologie und genetische Krankheiten in Nordamerika und Europa abzielten.
| Region | Vertriebsmitarbeiter | Zielmärkte |
|---|---|---|
| Nordamerika | 26 | Onkologie, genetische Krankheiten |
| Europa | 16 | Seltene genetische Störungen |
Digitale Gesundheitsplattformen
CRISPR Therapeutics investierte 3,2 Millionen US-Dollar in digitale Gesundheitskommunikationsplattformen und erreichte damit monatlich etwa 5.700 medizinische Fachkräfte.
- Investition in die digitale Plattform: 3,2 Millionen US-Dollar
- Monatliche Reichweite medizinischer Fachkräfte: 5.700
- Plattformtypen: Webinare, interaktive Forschungsportale
Kommunikation des medizinischen Forschungsnetzwerks
Das Unternehmen pflegte aktive Kooperationen mit 37 Forschungseinrichtungen und ermöglichte so den direkten Wissensaustausch und potenzielle Partnerschaften bei klinischen Studien.
| Netzwerktyp | Anzahl der Institutionen | Fokus auf Zusammenarbeit |
|---|---|---|
| Akademische Forschungsnetzwerke | 24 | Grundlagenforschung |
| Klinische Forschungszentren | 13 | Klinische Studien |
CRISPR Therapeutics AG (CRSP) – Geschäftsmodell: Kundensegmente
Patienten mit genetischen Störungen
Zielgruppe der Gen-Editing-Therapien von CRISPR Therapeutics:
| Störungskategorie | Geschätzte Patientenpopulation | Potenzieller Marktwert |
|---|---|---|
| Sichelzellenanämie | 100.000 Patienten in den USA | Potenzieller Markt von 1,5 Milliarden US-Dollar |
| Beta-Thalassämie | 60.000 Patienten weltweit | 1,2 Milliarden US-Dollar potenzieller Markt |
Pharmazeutische Forschungseinrichtungen
Wichtige institutionelle Kunden:
- National Institutes of Health (NIH)
- Harvard Medical School
- Forschungszentren der Stanford University
- Forschungsabteilung für Biotechnologie des MIT
Gesundheitsdienstleister
Zielsegmente im Gesundheitswesen:
| Gesundheitssegment | Anzahl potenzieller Kunden | Jährliches Forschungsbudget |
|---|---|---|
| Akademische medizinische Zentren | 150 Zentren | 500 Millionen Dollar |
| Spezialisierte genetische Kliniken | 250 Kliniken | 350 Millionen Dollar |
Biotechnologieforscher
Segmente der Forschungsgemeinschaft:
- Genomforscher
- Spezialisten für Molekularbiologie
- Experten für Gentechnik
Organisationen zur Finanzierung medizinischer Forschung
Finanzierungsquellen und Zuweisungen:
| Förderorganisation | Jährliches Budget für genetische Forschung | Mögliche CRISPR-Investition |
|---|---|---|
| Bill & Melinda Gates Stiftung | 250 Millionen Dollar | 50 Millionen Dollar |
| Willkommen, Vertrauen | 200 Millionen Dollar | 40 Millionen Dollar |
CRISPR Therapeutics AG (CRSP) – Geschäftsmodell: Kostenstruktur
Umfangreiche Forschungs- und Entwicklungskosten
Die CRISPR Therapeutics AG meldete F&E-Ausgaben in Höhe von 612,8 Millionen US-Dollar für das Geschäftsjahr 2022. Die Aufschlüsselung der wichtigsten F&E-Investitionen umfasst:
| F&E-Kategorie | Ausgabenbetrag |
|---|---|
| Entwicklung der Gen-Editing-Technologie | 287,4 Millionen US-Dollar |
| Forschung zu seltenen Krankheiten | 156,2 Millionen US-Dollar |
| Onkologische Forschung | 169,2 Millionen US-Dollar |
Kosten für klinische Studien
Die Ausgaben für klinische Studien für CRISPR Therapeutics beliefen sich im Jahr 2022 auf rund 345,6 Millionen US-Dollar, mit spezifischen Zuweisungen:
- CTX001-Hämoglobinopathie-Studien: 178,3 Millionen US-Dollar
- Klinische Onkologieprogramme: 112,5 Millionen US-Dollar
- Immunonkologische Studien: 54,8 Millionen US-Dollar
Aufrechterhaltung des geistigen Eigentums
Die Kosten für geistiges Eigentum für CRISPR Therapeutics beliefen sich im Jahr 2022 auf 42,3 Millionen US-Dollar, darunter:
| IP-Kategorie | Ausgabenbetrag |
|---|---|
| Patentanmeldung und -verfolgung | 26,7 Millionen US-Dollar |
| Patentpflege | 15,6 Millionen US-Dollar |
Fortschrittliche Laborinfrastruktur
Die Infrastruktur- und Anlagenkosten für 2022 beliefen sich auf 87,5 Millionen US-Dollar, darunter:
- Laborausrüstung: 52,3 Millionen US-Dollar
- Instandhaltung der Anlage: 21,7 Millionen US-Dollar
- Technologieinfrastruktur: 13,5 Millionen US-Dollar
Talentakquise und -bindung
Die Personalausgaben für CRISPR Therapeutics beliefen sich im Jahr 2022 auf 224,6 Millionen US-Dollar, mit folgender Zuteilung:
| Personalkategorie | Ausgabenbetrag |
|---|---|
| Grundgehälter | 156,3 Millionen US-Dollar |
| Aktienbasierte Vergütung | 48,2 Millionen US-Dollar |
| Rekrutierung und Schulung | 20,1 Millionen US-Dollar |
CRISPR Therapeutics AG (CRSP) – Geschäftsmodell: Einnahmequellen
Verbundforschungsförderung
Im Jahr 2023 meldete CRISPR Therapeutics eine gemeinsame Forschungsfinanzierung in Höhe von 292,4 Millionen US-Dollar aus Partnerschaften mit Vertex Pharmaceuticals.
Lizenzierung von Gentechnologie
| Technologie | Lizenzeinnahmen 2023 | Partner |
|---|---|---|
| CRISPR/Cas9-Plattform | 47,6 Millionen US-Dollar | Vertex Pharmaceuticals |
| Techniken zur Genbearbeitung | 22,3 Millionen US-Dollar | Bayer AG |
Potenzielle Verkäufe therapeutischer Produkte
CTX001 zur Behandlung von Sichelzellanämie und Beta-Thalassämie prognostizierte einen potenziellen Jahresumsatz von etwa 1,5 Milliarden US-Dollar.
Forschungsstipendien
- Zuschuss der National Institutes of Health (NIH): 8,2 Millionen US-Dollar
- Zuschuss des Europäischen Forschungsrats: 3,7 Millionen US-Dollar
- Bill & Zuschuss der Melinda Gates Foundation: 5,6 Millionen US-Dollar
Strategische Pharmapartnerschaften
| Partner | Partnerschaftswert | Fokusbereich |
|---|---|---|
| Vertex Pharmaceuticals | 900 Millionen US-Dollar Vorab- und Meilensteinzahlungen | Hämoglobinopathien |
| Bayer AG | Kooperationsvereinbarung über 300 Millionen US-Dollar | Onkologie und genetische Störungen |
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.
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.Disclaimer
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