CRISPR Therapeutics AG (CRSP) Management Presents at Goldman Sachs 43rd Annual Global Healthcare Conference (Transcript)

Summary of CRISPR Therapeutics AG Conference Call Company Overview - Company: CRISPR Therapeutics AG (NASDAQ: CRSP) - Event: Goldman Sachs 43rd Annual Global Healthcare Conference - Date: June 15, 2022 - Participants: Lawrence Klein (COO), Salveen Richter (Goldman Sachs) Key Points Industry Focus - Core Technology: CRISPR editing technology - Main Areas of Focus: - Ex vivo applications (hemoglobinopathies, rare diseases) - Oncology (allogeneic CAR-T therapies) - Regenerative medicine (beta cell replacement for type 1 diabetes) - In vivo applications [3][4] Clinical Programs and Updates - Clinical Programs: - CTX110 (CD19), CTX120 (BCMA), CTX130 (CD70) - Data expected by the end of the month for CTX120 and CTX130 [5][6] - Durable Responses: Achieved durable remissions with a single dose of allogeneic cell therapy [7] - Competitive Dynamics: Acknowledgment of competitive fields, with ongoing efforts to improve immunovasion and potency [8] Financial Position - Cash Balance: Approximately $2.2 billion, providing multiple years of runway [9] - Capital Allocation: Majority of capital directed towards clinical programs, with oncology being the largest area of expenditure [10] In Vivo Programs - Strategic Decision: Initial focus on ex vivo programs due to lower technical hurdles; in vivo programs are now accelerating [12][13] - Potential for Approval: Exa-Cel (formerly CTX001) on track for approval, with significant clinical benefits observed [16] Regulatory and Clinical Strategy - Regulatory Submission Timeline: Confidence in submitting for regulatory approval by year-end, with ongoing discussions with the FDA regarding patient follow-up duration [17][18] - Endpoint Adjustments: Shifted from transfusion reduction to transfusion independence as primary endpoints based on clinical results [19] Competitive Landscape - Comparison with Other Therapies: Observations on the competitive landscape, particularly regarding autologous therapies and the need for off-the-shelf solutions [30][31] - Differentiation Strategy: Focus on beta2M edit for immune evasion, aiming to avoid harsher lymphodepletion [30] Regenerative Medicine - Type 1 Diabetes Program: Ongoing clinical trials with a focus on safety and immunoagent trials, aiming for unencapsulated, injectable cell therapies [45][46] Intellectual Property (IP) Status - IP Landscape: Strong position in ex-U.S. markets; ongoing appeal regarding U.S. patents held by Broad Institute [52][54] Future Outlook - In Vivo Pipeline: Anticipation of unveiling significant progress in in vivo programs at the upcoming Innovation Day [56] Additional Insights - Patient Population: Approximately 30,000 patients identified for current standard of care conditioning in beta thalassemia and sickle cell disease [26] - Innovation in Preconditioning: Ongoing efforts to develop targeted conditioning regimens to enhance patient access [27] - Market Need: Recognition of the unmet need in T-cell lymphomas and the potential for new therapies [38] This summary encapsulates the critical insights and developments discussed during the conference call, highlighting the strategic direction and clinical advancements of CRISPR Therapeutics AG.