Summary of Cariboo's Biopharma Back to School Summit Company Overview - **Company**: Cariboo - **Technology**: CRISPR genome editing, specifically the Chardonnay technology, which allows for more precise genome editing compared to first-generation CRISPR Cas9 [4][4] Key Studies and Developments CB10 (Lymphoma) - **Type**: Off-the-shelf CAR T cell therapy targeting CD19 - **Mechanism**: Utilizes Chardonnay technology to knock out PD-1 to prevent CAR T cell exhaustion [6][6] - **Clinical Trials**: Ongoing phase one studies with significant data readouts expected by the end of the year [4][5] - **Patient Outcomes**: First patient remains in complete response after treatment; data suggests that modest partial HLA matching improves outcomes [7][9] - **Data Sharing**: Plans to share data from a new cohort of 20 patients with at least four matched HLA alleles, aiming for outcomes comparable to autologous CAR Ts [10][12] - **FDA Discussions**: Actively engaging with the FDA regarding pivotal study pathways for CB10 [12][32] CB11 (Multiple Myeloma) - **Type**: Off-the-shelf CAR T cell therapy targeting BCMA - **Mechanism**: Focuses on immune cloaking by knocking out endogenous HLA class one expression and overexpressing HLAE to prevent NK cell rejection [63][65] - **Clinical Trials**: First data update expected this half, with a focus on being on par with bispecific therapies in terms of efficacy [66][70] - **Patient Population**: Only about 10% of myeloma patients currently access autologous CAR Ts, indicating a significant market opportunity [66][67] Market Dynamics and Opportunities - **Access to Therapy**: Approximately 80% of lymphoma patients do not currently benefit from CAR T therapies due to logistical and geographical barriers [35][37] - **Manufacturing Capabilities**: Current processes can produce enough cells for 200 to 300 doses per manufacturing run, indicating readiness for market supply [49][49] - **Community Integration**: Plans to leverage community sites for distribution, making therapies more accessible [100][100] Financial Considerations - **Current Funding**: Approximately $184 million available, sufficient to fund current plans into 2027 but not for pivotal trials [94][94] - **Strategic Investment**: Pfizer made a $25 million investment focused on the myeloma program, with a right of first negotiation for future funding [95][95] Future Directions - **Expansion of Indications**: Potential to explore earlier lines of therapy for both lymphoma and myeloma based on learnings from ongoing studies [89][89] - **Global Reach**: The Antler study has been expanded to include patients in Israel and Australia, providing access to diverse patient populations [102][102] Conclusion Cariboo is positioned to make significant advancements in the CAR T therapy landscape with its innovative CRISPR technology and strategic focus on accessibility and patient outcomes. Upcoming data readouts and FDA discussions will be critical in shaping the future of its therapies.