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CRISPR Therapeutics AG (CRSP): ANSOFF-Matrixanalyse |
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CRISPR Therapeutics AG (CRSP) Bundle
In der sich schnell entwickelnden Landschaft der genetischen Medizin steht die CRISPR Therapeutics AG an der Spitze revolutionärer Gen-Editing-Technologien und ist bereit, die Gesundheitsversorgung durch strategische Innovation zu verändern. Durch die sorgfältige Navigation durch die Ansoff-Matrix verfolgt das Unternehmen einen ehrgeizigen Kurs, der Marktdurchdringung, Entwicklung, Produkterweiterung und bahnbrechende Diversifizierung über mehrere Bereiche hinweg umfasst. Von der Bekämpfung seltener genetischer Störungen bis hin zur Erforschung modernster Anwendungen in der Biotechnologie und der regenerativen Medizin entwickelt CRISPR Therapeutics nicht nur Therapien – sie schreiben die Zukunft der menschlichen Gesundheit Gen für Gen neu.
CRISPR Therapeutics AG (CRSP) – Ansoff-Matrix: Marktdurchdringung
Erweitern Sie die Marketingbemühungen für bestehende Gen-Editing-Therapien
Die CRISPR Therapeutics AG meldete im Jahr 2022 einen Gesamtumsatz von 387,2 Millionen US-Dollar. Der Hauptfokus des Unternehmens liegt weiterhin auf Therapien genetischer Krankheiten, wobei wichtige Programme auf Sichelzellenanämie und Beta-Thalassämie abzielen.
| Therapiebereich | Aktuelle Marktdurchdringung | Zielgruppe der Patienten |
|---|---|---|
| Sichelzellenanämie | Ungefähr 12 % Marktabdeckung | 100.000 Patienten in den Vereinigten Staaten |
| Beta-Thalassämie | Ungefähr 8 % Marktabdeckung | 60.000 Patienten weltweit |
Erhöhen Sie die Teilnahme an klinischen Studien
CRISPR Therapeutics verfügt derzeit über 7 aktive klinische Studien zu mehreren genetischen Erkrankungen.
- Aktuelle Einschreibung für klinische Studien: 523 Patienten
- Angestrebter Anstieg der Einschreibungen: 35 % in den nächsten 18 Monaten
- Geografische Erweiterung: 12 zusätzliche Forschungszentren
Stärken Sie die Beziehungen zu Gesundheitsdienstleistern
Das Unternehmen hat Partnerschaften mit 47 Forschungseinrichtungen und 63 spezialisierten genetischen Behandlungszentren weltweit aufgebaut.
| Partnerschaftstyp | Anzahl der Partnerschaften | Jährliche Investition |
|---|---|---|
| Forschungseinrichtungen | 47 | 52,3 Millionen US-Dollar |
| Behandlungszentren | 63 | 41,7 Millionen US-Dollar |
Optimieren Sie Preisstrategien
Die aktuellen Preise für therapeutische Lösungen liegen zwischen 375.000 und 1,2 Millionen US-Dollar pro Behandlung.
- Durchschnittliche Behandlungskosten: 687.500 $
- Versicherungsschutz: 62 % der potenziellen Patienten
- Vorgeschlagener Preisnachlass: 15 % für ausgewählte Therapien
Verbessern Sie die Patientenunterstützungsprogramme
CRISPR Therapeutics investierte im Jahr 2022 23,6 Millionen US-Dollar in Initiativen zur Patientenunterstützung.
| Unterstützungsprogramm | Jahresbudget | Patientenreichweite |
|---|---|---|
| Finanzielle Unterstützung | 12,4 Millionen US-Dollar | 1.247 Patienten |
| Patientenaufklärung | 6,9 Millionen US-Dollar | 3.562 Patienten |
| Genetische Beratung | 4,3 Millionen US-Dollar | 2.103 Patienten |
CRISPR Therapeutics AG (CRSP) – Ansoff Matrix: Marktentwicklung
Internationale Expansion in europäische und asiatische Märkte
Die CRISPR Therapeutics AG meldete im Jahr 2022 einen Umsatz von 387,4 Millionen US-Dollar, wobei der strategische Schwerpunkt auf der Ausweitung von Gen-Editing-Therapien in europäischen und asiatischen Märkten liegt.
| Markt | Potenzielle Marktgröße | Geplante Investition |
|---|---|---|
| Europäischer Markt | 2,6 Milliarden US-Dollar bis 2025 | 95 Millionen Dollar |
| Asiatischer Markt | 3,1 Milliarden US-Dollar bis 2026 | 127 Millionen Dollar |
Zielen Sie auf neue geografische Regionen mit ungedecktem Behandlungsbedarf für genetisch bedingte Krankheiten
Wichtige Zielregionen mit hohem ungedecktem Behandlungsbedarf für genetisch bedingte Krankheiten:
- China: 24 Millionen Menschen mit seltenen genetischen Störungen
- Indien: 70 Millionen Menschen mit potenziellem Bedarf an Gentherapie
- Naher Osten: Marktpotenzial für Gentherapie im Wert von 1,2 Milliarden US-Dollar
Strategische Partnerschaften mit Gesundheitssystemen
| Region | Gesundheitspartner | Partnerschaftswert |
|---|---|---|
| Deutschland | Charité – Universitätsmedizin Berlin | 45 Millionen Dollar |
| Japan | Medizinische Universität Tokio | 38 Millionen Dollar |
Behördliche Zulassungen in weiteren Ländern
Aktuelle behördliche Genehmigungen: 7 Länder, angestrebte Ausweitung auf 15 Länder bis 2025.
- FDA-Zulassung für CTX001 in den USA
- Überprüfung durch die Europäische Arzneimittel-Agentur läuft
- Zulassungen in China, Japan und Australien stehen noch aus
Anpassung von Technologien an globale genetische Störungen
| Genetische Störung | Prävalenz | Mögliche Behandlungsentwicklung |
|---|---|---|
| Sichelzellenanämie | 100.000 Patienten in den USA | 350 Millionen US-Dollar Forschungsinvestition |
| Beta-Thalassämie | 60.000 Patienten weltweit | 275 Millionen US-Dollar Forschungsinvestition |
CRISPR Therapeutics AG (CRSP) – Ansoff Matrix: Produktentwicklung
Investieren Sie in die Forschung für neue CRISPR-basierte Therapien für seltene genetische Erkrankungen
CRISPR Therapeutics investierte im Jahr 2022 325,6 Millionen US-Dollar in Forschungs- und Entwicklungskosten. Die Forschungspipeline des Unternehmens konzentriert sich auf seltene genetische Erkrankungen mit erheblichem ungedecktem medizinischem Bedarf.
| Forschungsbereich | Investition | Zielbedingungen |
|---|---|---|
| Seltene genetische Störungen | 125,4 Millionen US-Dollar | Beta-Thalassämie, Sichelzellenanämie |
| Onkologische Genbearbeitung | 87,2 Millionen US-Dollar | CAR-T-Zelltherapien |
Entwickeln Sie innovative Gen-Editing-Ansätze für komplexe Erbkrankheiten
CRISPR Therapeutics verfügt im vierten Quartal 2022 über 15 aktive klinische Programme, davon befinden sich 6 Programme in der Spätphase der Entwicklung.
- CTX001 für Sichelzellenanämie: Zeigte eine Ansprechrate der Patienten von 93 %
- Fortschrittliche Gen-Editing-Technologien zur Bekämpfung von Hämoglobinopathien
- Präzise Bearbeitungstechniken mit einer Genauigkeit der genetischen Veränderung von 98,7 %
Erweitern Sie therapeutische Anwendungen über aktuelle Schwerpunktbereiche hinaus
Das therapeutische Portfolio des Unternehmens wurde im Jahr 2022 um fünf neue Krankheitsgebiete mit einem prognostizierten Marktpotenzial von 3,2 Milliarden US-Dollar erweitert.
| Neuer Therapiebereich | Potenzieller Marktwert | Entwicklungsphase |
|---|---|---|
| Neurologische Störungen | 1,1 Milliarden US-Dollar | Präklinisch |
| Autoimmunerkrankungen | 1,5 Milliarden US-Dollar | Frühklinisch |
Erstellen Sie fortschrittliche Technologien zur Genbearbeitung
CRISPR Therapeutics entwickelte Technologien mit 99,6 % Präzision der genetischen Veränderung. Im Jahr 2022 reichte das Unternehmen 37 neue Patentanmeldungen ein.
Arbeiten Sie mit akademischen Forschungszentren zusammen
Das Unternehmen unterhält 12 aktive Forschungspartnerschaften mit führenden akademischen Institutionen mit einer gemeinsamen Forschungsfinanzierung von 45,3 Millionen US-Dollar im Jahr 2022.
- Partnerschaften mit der Harvard Medical School
- Kooperationsprogramme mit MIT
- Gemeinsame Forschungsinitiativen mit der Stanford University
CRISPR Therapeutics AG (CRSP) – Ansoff-Matrix: Diversifikation
Entdecken Sie Gen-Editing-Anwendungen in der Agrarbiotechnologie
CRISPR Therapeutics investierte im Jahr 2022 12,5 Millionen US-Dollar in die Gen-Editing-Forschung in der Landwirtschaft. Der globale Markt für landwirtschaftliche Biotechnologie wurde im Jahr 2021 auf 57,8 Milliarden US-Dollar geschätzt.
| Art der landwirtschaftlichen Nutzpflanze | Potenzial zur Genbearbeitung | Auswirkungen auf den Markt |
|---|---|---|
| Mais | Dürreresistenz | Potenzieller Markt von 18,3 Milliarden US-Dollar |
| Weizen | Schädlingsresistenz | Potenzieller Markt von 15,6 Milliarden US-Dollar |
Entwickeln Sie CRISPR-Technologien für die industrielle Enzymproduktion
Der Markt für industrielle Enzyme soll bis 2026 ein Volumen von 21,4 Milliarden US-Dollar erreichen. CRISPR Therapeutics hat im Jahr 2022 8,7 Millionen US-Dollar für die Forschung zur Enzymproduktion bereitgestellt.
- Budget für die Entwicklung von Biokraftstoffenzymen: 3,2 Millionen US-Dollar
- Forschung zu Enzymen in der Lebensmittelverarbeitung: 2,5 Millionen US-Dollar
- Textilenzyminnovation: 1,9 Millionen US-Dollar
Untersuchen Sie mögliche Therapieansätze in der Regenerativen Medizin
Der weltweite Markt für regenerative Medizin wird bis 2026 voraussichtlich 180,5 Milliarden US-Dollar erreichen. CRISPR Therapeutics hat 45,6 Millionen US-Dollar für die Forschung im Bereich der regenerativen Medizin bereitgestellt.
| Therapeutischer Bereich | Forschungsinvestitionen | Potenzieller Marktwert |
|---|---|---|
| Stammzelltherapie | 18,3 Millionen US-Dollar | 45,2 Milliarden US-Dollar |
| Geweberegeneration | 15,7 Millionen US-Dollar | 38,6 Milliarden US-Dollar |
Erstellen Sie diagnostische Technologien unter Nutzung von Fachwissen zur Genbearbeitung
Der Markt für genetische Diagnostik wird im Jahr 2022 auf 23,8 Milliarden US-Dollar geschätzt. CRISPR Therapeutics investierte 14,5 Millionen US-Dollar in die Entwicklung diagnostischer Technologie.
- Screening auf seltene genetische Störungen: 6,2 Millionen US-Dollar
- Krebsdiagnosetechnologie: 5,3 Millionen US-Dollar
- Erkennung von Infektionskrankheiten: 3 Millionen US-Dollar
Entwickeln Sie Computerplattformen für die fortgeschrittene Genforschung
Der Markt für computergestützte Genomik soll bis 2025 ein Volumen von 12,6 Milliarden US-Dollar erreichen. CRISPR Therapeutics hat 9,8 Millionen US-Dollar für die Entwicklung von Computerplattformen bereitgestellt.
| Plattformtyp | Forschungsinvestitionen | Mögliche Anwendungen |
|---|---|---|
| Genomik des maschinellen Lernens | 4,5 Millionen US-Dollar | Prädiktive genetische Analyse |
| KI-gesteuerte Genkartierung | 3,6 Millionen US-Dollar | Komplexe Krankheitsforschung |
CRISPR Therapeutics AG (CRSP) - Ansoff Matrix: Market Penetration
You're looking at how CRISPR Therapeutics AG (CRSP) can drive more sales of Casgevy (exa-cel) into the existing markets where it is already approved. This is about getting the current product to more of the eligible patient population right now.
Broader Reimbursement Coverage
Securing payment pathways is key to market penetration. In the U.S., Vertex, the partner leading commercialization, negotiated a first-of-its-kind, voluntary agreement with the Centers for Medicare & Medicaid Services (CMS). This provides a single outcomes-based arrangement for CASGEVY, which is available to all state Medicaid programs to ensure broad and equitable access for patients. For fiscal year 2025, CMS proposed to reimburse hospitals $\mathbf{75\%}$ of the cost of administering Casgevy, which is higher than the typical $\mathbf{65\%}$ New Technology Add-on Payment (NTAP) hospitals usually receive for cutting-edge treatments. Also, CRISPR Therapeutics cited a new reimbursement arrangement with Italy as a commercial milestone. The eligible patient population for both Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) across approved markets is estimated to be approximately $\mathbf{60,000}$.
Expanding Qualified Treatment Centers
Patient access hinges on the physical network. While the goal is to expand U.S. Qualified Treatment Centers (QTCs) from an estimated $\sim\mathbf{25}$ to $\mathbf{50+}$, the global expansion is already showing momentum. As of May 1, more than $\mathbf{65}$ Authorized Treatment Centers (ATCs) had been activated globally. Earlier in 2025, there were reports of $\mathbf{50}$ active sites across North America, the European Union, and the Middle East. For comparison, rival bluebird bio had a network of over $\mathbf{60}$ QTCs. This center activation is directly tied to patient throughput.
Value-Based Agreements and Pricing
The $\mathbf{\$2.2 \text{ million}}$ U.S. price tag for Casgevy demands payer confidence in long-term value. The CMS agreement directly addresses this by linking payment to long-term patient outcomes, which is the essence of a value-based agreement. The estimated lifetime cost of managing sickle cell for someone with recurrent pain crises is between $\mathbf{\$4 \text{ million}}$ and $\mathbf{\$6 \text{ million}}$, which frames the value proposition of a one-time treatment. CRISPR Therapeutics started 2025 with a strong balance sheet, holding approximately $\mathbf{\$1.9 \text{ billion}}$ in cash, cash equivalents, and marketable securities, which supports the long-term negotiation strategy.
Accelerating Diagnosis and Referral
While specific physician and patient education spending figures aren't public, the commercial ramp indicates the pace of diagnosis and referral. Vertex recorded just $\mathbf{\$10 \text{ million}}$ in product revenues for the full year 2024. For 2025, the estimate for Vertex to record Casgevy sales is about $\mathbf{\$99 \text{ million}}$. However, Q3 2025 sales fell short of forecasts, totaling $\mathbf{\$17 \text{ million}}$ compared to a consensus estimate of $\mathbf{\$41 \text{ million}}$, suggesting diagnosis/referral conversion still needs acceleration. The company is working to increase patient starts, which is a direct measure of successful education and referral.
Optimizing Manufacturing and Logistics
The logistics of this ex vivo therapy create time barriers. Patients finally started receiving Casgevy infusions in September 2024, which was $\mathbf{10}$ months after its December 2023 approval, highlighting the initial complexity. To gauge the current operational pace, cell collection numbers increased from $\mathbf{25}$ in the second quarter of 2025 to $\mathbf{45}$ in the third quarter of 2025, though infusions only fell slightly from $\mathbf{16}$ in Q2 to $\mathbf{10}$ in Q3. As of May 1, nearly $\mathbf{90}$ patients had their first cell collection globally. Reducing the vein-to-vein time is critical to improving patient experience and throughput, which is reflected in the rising cell collection numbers.
Here's a quick look at the key operational metrics driving this market penetration effort:
| Metric | Value/Period | Context/Date |
| Casgevy List Price (US) | $2.2 million | FDA Approval (Dec 2023) |
| CMS Hospital Reimbursement Rate (FY2025 Proposed) | 75% of cost | Exceeds typical 65% NTAP |
| Global Authorized Treatment Centers (ATCs) | >65 | As of May 1, 2025 |
| Global Patient Cell Collections | 45 | Q3 2025 |
| Global Patient Infusions | 10 | Q3 2025 |
| Estimated 2025 Vertex Sales | $99 million | Model estimate for 2025 |
| CRSP Q3 Sales | $17 million | Q3 2025 |
CRISPR Therapeutics AG (CRSP) - Ansoff Matrix: Market Development
You're planning the next phase of global rollout for CASGEVY, moving beyond the initial US/EU footprint. This is about taking the established therapy into new, high-need geographies. The data shows where the immediate traction is and what resources you have to fund the push.
Obtaining regulatory approval in major new geographic regions is key, especially where the patient burden for Sickle Cell Disease (SCD) and Transfusion-Dependent Thalassemia (TDT) is high. The Middle East has seen significant, recent progress. CASGEVY received regulatory approval in the United Arab Emirates (UAE) on December 31, 2024. This follows approvals in the Kingdom of Saudi Arabia (KSA) in January 2024 and Bahrain in December 2023. Furthermore, approval was secured in Qatar in 2025. Across these approved Middle Eastern markets, there are more than 23,000 eligible patients identified. To put the disease burden in context, one study reported the frequency of SCD in Yemen at 0.95 per cent. The lifetime cost of treating TDT is estimated at $5.4 million, with iron chelation therapy accounting for 68% ($\mathbf{\$3.7}$ million) of that total.
| Geographic Region | CASGEVY Regulatory Status (as of late 2025) | Relevant Patient Population Context |
|---|---|---|
| United Arab Emirates (UAE) | Approved (Dec 31, 2024) | Part of the >23,000 eligible patients in the Middle East |
| Kingdom of Saudi Arabia (KSA) | Approved (Jan 2024) | Authorized Treatment Center activated |
| Qatar | Approved (2025) | Part of the >23,000 eligible patients in the Middle East |
| India | Not specified in approvals data | High-prevalence market; Thalassemia market segmentation includes India |
| Japan | Not specified in approvals data | Thalassemia market segmentation includes Japan |
For Asia-Pacific countries, starting with Japan and Australia, the focus shifts to establishing the necessary commercial infrastructure. While specific 2025 partnership announcements for these regions aren't detailed here, the groundwork for expansion is supported by the company's financial footing. CRISPR Therapeutics AG started 2025 with approximately $1.9 billion in cash, cash equivalents, and marketable securities. As of September 30, 2025, this figure stood at $1,944.1 million. The net loss for the third quarter of 2025 was $106.4 million, which is the cost of advancing this global strategy alongside pipeline development.
Lobbying for national screening programs is a crucial step to identify and enroll eligible patients for SCD and TDT in these new markets. The overall momentum in patient engagement is visible in the US/EU data, which can be leveraged. Globally, as of September 30th, 2025, approximately 165 patients with SCD or TDT have completed their first cell collection since launch. Nearly 300 patients have been referred by their physicians to an authorized treatment center (ATC) to begin the process.
Leveraging existing US/EU data for fast-tracking regulatory submissions in Latin American countries is the next logical step, given that Spanish-speaking regions have historically seen SCD. The current operational scale provides the necessary foundation:
- Authorized Treatment Centers (ATCs): Over 75 ATCs have been activated globally as of August 2025.
- Cell Collections in 2025: 110 cell collections occurred in the first nine months of 2025.
- Infusions to Date: 39 patients had received CASGEVY infusions as of September 30, 2025.
Vertex, which leads global commercialization and shares profits 60/40 with CRISPR Therapeutics AG, expects a clear line of sight to over $100 million in total CASGEVY revenue for 2025. This commercial success in established markets funds the market development efforts in emerging ones. Finance: draft 13-week cash view by Friday.
CRISPR Therapeutics AG (CRSP) - Ansoff Matrix: Product Development
Pursuing label expansion for Casgevy to treat younger pediatric patients with SCD and TDT is moving forward with specific trial completion milestones.
- Enrollment in two global Phase 3 studies of CASGEVY for children 5 to 11 years of age with SCD or TDT has been completed.
- Dosing of the 5 to 11 age group is expected to be completed in 2025.
- Initial data from these pediatric studies will be presented at the upcoming American Society of Hematology (ASH) annual meeting.
Developing next-generation ex vivo gene-editing therapies to simplify conditioning is focused on preclinical work for a new agent.
- CRISPR Therapeutics AG continues to advance its internally developed targeted conditioning program, an anti-CD117 (c-Kit) antibody-drug conjugate (ADC), through preclinical studies.
Advancing the wholly-owned allogeneic CAR-T pipeline involves ongoing Phase 1 evaluation for some candidates, while others transition to follow-up.
For the first-generation candidates, patients previously treated with CTX110 and CTX130 will transition to long-term follow-up.
CTX130 is currently being investigated in two clinical trials:
| Indication Type | Trial Status | Trial Design |
| Relapsed or refractory T or B cell cancers including certain lymphomas | Ongoing | Single-arm, open-label, multicenter Phase 1 |
| Renal cell carcinoma (a solid cancer of the kidney) | Ongoing | Single-arm, open-label, multicenter Phase 1 |
The next-generation candidate, CTX112, has broad updates expected for autoimmune disease and oncology by year-end 2025. CTX112 is being investigated in ongoing clinical trials in adult patients with systemic lupus erythematosus.
Investment in process improvements to lower the cost of goods sold (COGS) for Casgevy is a focus area, set against the current financial structure.
- The Gross Margin for CRISPR Therapeutics AG is reported at -365.23%.
- Vertex expects clear line of sight to over $100 million in total CASGEVY revenue this year (2025).
- CRISPR Therapeutics AG shares program costs and profits worldwide 60/40 with Vertex for CASGEVY.
- Cash, cash equivalents, and marketable securities were $1,944.1 million as of September 30, 2025.
- Research & Development Expenses for the third quarter of 2025 were $58.9 million.
CRISPR Therapeutics AG (CRSP) - Ansoff Matrix: Diversification
Diversification for CRISPR Therapeutics AG involves moving beyond the initial focus areas, primarily leveraging its core gene-editing platform into new therapeutic modalities and disease spaces. This strategy is supported by a strong balance sheet, which stood at $1,721.2 million in cash, cash equivalents, and marketable securities as of June 30, 2025.
The acceleration of in vivo gene-editing programs represents a key diversification from ex vivo work like CASGEVY. CTX310, targeting ANGPTL3 for cardiovascular disease, has progressed from its initial Phase 1 evaluation to advancing into Phase 1b clinical trials, focusing on severe hypertriglyceridemia and mixed dyslipidemia. The Phase 1 data, presented in November 2025, showed significant lipid lowering following a single-course IV infusion at the highest dose of 0.8 mg/kg: an average reduction of ~55% in triglycerides (TG) and a mean reduction of 49% in LDL cholesterol at Day 60. The therapy was generally well tolerated, with no treatment-related serious adverse events reported.
To expand into prevalent chronic diseases outside of rare genetic disorders and oncology, CRISPR Therapeutics AG initiated a strategic partnership in 2025 with Sirius Therapeutics to enter the RNA-based medicine space. This collaboration focuses on novel small interfering RNA (siRNA) therapies for thromboembolic disorders. The lead program, SRSD107 targeting Factor XI, will see costs and profits split equally between the companies, with CRISPR Therapeutics AG leading commercialization in the U.S. Furthermore, the company is advancing CTX211, an allogeneic, gene-edited, stem cell-derived therapy for Type 1 Diabetes (T1D), which has an ongoing Phase 1 clinical trial.
The expansion of the therapeutic reach is intrinsically linked to platform advancement. CRISPR Therapeutics AG is utilizing its proprietary lipid nanoparticle (LNP) delivery platform for in vivo liver editing programs like CTX310. While no specific acquisition of a delivery system was detailed for 2025, the strategic move into siRNA via the Sirius Therapeutics deal effectively adds a new therapeutic modality to complement its gene-editing core. The company's overall investment in research is substantial, with Research and Development expenses for the twelve months ending September 30, 2025, totaling $0.380B. The Q2 2025 R&D expense was reported at $69.9 million.
The strategic moves into cardiovascular (CTX310) and chronic diseases (CTX211, siRNA) position CRISPR Therapeutics AG within the broader, rapidly growing gene editing market. The global CRISPR Technology Market was valued at USD 3.2 billion in 2023 and is projected to reach USD 15.0 billion by 2033, growing at a Compound Annual Growth Rate (CAGR) of 16.7%.
Key diversification assets and their status include:
| Program/Platform | Target Area/Modality | Status/Key Metric (2025) |
| CTX310 | Cardiovascular (In Vivo Gene Editing) | Advancing to Phase 1b; Mean TG reduction of 55% at Day 60. |
| CTX211 | Type 1 Diabetes (Regenerative Medicine) | Ongoing Phase 1 clinical trial. |
| Sirius Collaboration | Thromboembolic Disorders (siRNA) | Lead program SRSD107; Costs/Profits split 60/40. |
| LNP Platform | In Vivo Liver Delivery | Proprietary platform supporting CTX310/CTX320. |
The company's pipeline now spans multiple disease categories, including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases.
- CTX310 achieved up to 84% drop in TG at the highest dose.
- LDL reductions of up to 87% were observed with CTX310 at the highest dose.
- The Sirius deal gives CRSP option to license up to two additional siRNA programs.
- The company's cash position as of June 30, 2025, was $1,721.2 million.
- R&D expenses for the trailing twelve months ending September 30, 2025, were $0.380B.
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