Summary of CRISPR Therapeutics Conference Call Company Overview - **Company**: CRISPR Therapeutics (NasdaqGM:CRSP) - **Event**: Virtual Oncology Leadership Summit - **Date**: February 18, 2026 Key Points Industry Focus - CRISPR Therapeutics is engaged in multiple disease areas, with a strong focus on oncology, cardiovascular diseases, autoimmune diseases, and Type 1 diabetes [3][4] Oncology Programs - **ZugoCell (formerly CTX112)**: A next-generation allogeneic CAR-T therapy targeting CD19, designed to improve potency and safety compared to earlier generations [4][5] - **Allogeneic CAR-T**: Made from healthy donor-derived cells, with advanced edits to enhance efficacy and safety, aiming for autologous-like performance [5][6] Key Edits in ZugoCell - **Beta-2M Knockout**: Prevents immune system from recognizing allogeneic cells as foreign, enhancing persistence [8][9] - **RGNASE1 Knockout**: Retains a naive T cell phenotype, allowing for better expansion and reduced exhaustion [10][11] - **TGF-beta Receptor 2 Knockout**: Prevents exhaustion of CAR-T cells, enhancing potency [10][11] Clinical Strategy - **Autoimmune Diseases**: Rapidly enrolling patients for indications like lupus, myositis, and scleroderma, with plans to expand further [17][18] - **Oncology**: Aiming for high complete response (CR) rates and durability, with initial CR rates for ZugoCell reaching nearly 70% [20][21] Regulatory Pathway - Parallel development in oncology and autoimmune indications, with discussions with regulators expected to guide future trials [27][28] Pricing Strategy - Targeting a price point of around $200K for allogeneic CAR-T therapies, significantly lower than current autologous therapies priced at $350K-$400K [52][53] In Vivo CAR-T Development - Developing both transient and permanent CAR-T therapies using LNP-mediated delivery, with a focus on safety and efficacy [54][55] - Permanent CARs could potentially eliminate tumors with a single injection, offering a significant advancement in oncology [56][57] Market Opportunities - Significant potential in both U.S. and international markets, particularly in regions where autologous therapies are not feasible due to cost and availability [48][49] Future Directions - Continued focus on data collection and analysis in both oncology and autoimmune spaces, with a strong emphasis on achieving long-term remissions and safety [31][32][39] Conclusion - CRISPR Therapeutics is positioned to make significant advancements in both oncology and autoimmune therapies through innovative CAR-T technologies, with a clear strategy for regulatory approval and market entry.

Financial Data and Key Metrics Changes - The company has streamlined its pipeline to focus on SC451 for type 1 diabetes and SG293 for in vivo CAR-T therapy, indicating a strategic shift towards high-potential products [2][6] - The company aims to initiate a clinical study for SC451 next year, with an IND submission expected soon, reflecting confidence in its development timeline [21][56] Business Line Data and Key Metrics Changes - The type 1 diabetes program is positioned as a one-time curative therapy, with the goal of eliminating the need for insulin and immunosuppression for patients [6][56] - The in vivo CAR-T platform is being developed to target various blood cancers and autoimmune disorders, with a focus on cell specificity and safety [8][46] Market Data and Key Metrics Changes - There are approximately 10 million people with type 1 diabetes in the U.S., projected to grow to 15 million in 15 years, highlighting a significant market opportunity [4][23] - The company is aware of the competitive landscape in the CAR-T space but believes its differentiated approach could lead to a best-in-class product [9][45] Company Strategy and Development Direction - The primary focus remains on advancing the type 1 diabetes program while exploring partnerships to enhance financial resilience and success probability [29][31] - The company is also considering partnerships for the in vivo CAR-T platform to accelerate development and leverage external expertise [49][50] Management's Comments on Operating Environment and Future Outlook - Management emphasizes the high unmet need in type 1 diabetes and the urgency to deliver a safe and effective therapy [44][56] - The company is optimistic about the regulatory path and believes it can navigate the complexities of manufacturing and clinical trials effectively [15][24] Other Important Information - The company has established a master cell bank for its gene-modified pluripotent stem cells, which is crucial for consistent product manufacturing [20][19] - Safety remains a primary concern, with management focusing on mitigating risks associated with severe hypoglycemia and potential tumor formation [14][18] Q&A Session Summary Question: What is the current status of the IND preparation for type 1 diabetes? - The company is engaged in dialogues with regulators and is working on completing the non-clinical toxicology package and GMP manufacturing to move forward with human testing [11][15] Question: How does the company plan to scale manufacturing for the type 1 diabetes therapy? - The company acknowledges the challenges of scaling but believes it can solve the manufacturing issues internally while also exploring partnerships for scientific support [33][29] Question: What are the key differentiators of the in vivo CAR-T platform? - The in vivo CAR-T platform utilizes virus-like particles for targeted delivery, which allows for cell-specific treatment without the complications associated with traditional methods [46][47] Question: How does the company view potential partnerships for its programs? - The company is open to partnerships that can enhance its financial stability and improve the probability of success, particularly for the in vivo CAR-T platform [29][49] Question: What are the anticipated timelines for clinical trials? - The company aims to initiate a phase 1 trial for the type 1 diabetes therapy next year, with data expected to emerge quickly thereafter [21][56]