CRISPR Therapeutics AG (CRSP) Presents at Citi's 18th Annual BioPharma Conference Transcript

Summary of CRISPR Therapeutics AG Conference Call Company Overview - Company: CRISPR Therapeutics AG (NASDAQ: CRSP) - Event: Citi's 18th Annual BioPharma Conference Call - Date: September 6, 2023 - Participants: - Sam Kulkarni - CEO - Yigal Nochomovitz - Citigroup Analyst Key Points Industry and Company Mission - CRISPR Therapeutics is on the verge of potentially launching the first CRISPR-based medicine, exa-cel, later in 2023, having dosed over 200 patients across various programs [4][5] - The company aims to expand its reach to treat multiple diseases, aspiring to become a $25 billion company in the next three to four years [5][6] Exa-cel and Market Strategy - Exa-cel is targeted at hemoglobinopathies, with PDUFA dates set for December 1 and March [8] - The company is preparing for an Advisory Committee meeting, anticipating standard questions around safety and efficacy [9][10] - The partnership with Vertex is crucial for commercialization, leveraging Vertex's experience in rare diseases [16][33] Market Dynamics and Patient Demand - There is a strong unmet need for therapies in sickle cell disease, with a significant number of patients willing to undergo transplant processes for transformative therapies like exa-cel [21] - The company expects a strong uptake once the drug is launched, with many treatment centers already familiar with the process [20] Competitive Landscape - CRISPR Therapeutics is optimistic about its market share in sickle cell disease, noting a preference among patients for CRISPR-based therapies over other modalities [25] - The company acknowledges competition but emphasizes the safety and efficacy of its CRISPR approach [25] Future Developments - The company is exploring a gentler conditioning regimen that could expand the market for exa-cel from 25,000 severe sickle cell patients to potentially 50,000 or more [30][31] - CRISPR Therapeutics has a 60-40 profit-sharing agreement with Vertex, with milestones tied to the approval of exa-cel [33] Immuno-Oncology Programs - The company is advancing its immuno-oncology platform, particularly with CTX110 and CTX112, which are designed to improve CAR-T cell therapies [36][37] - CTX112 is expected to be significantly more potent than CTX110, with early data showing promising expansion rates [39][40] Diabetes Regenerative Medicine - CRISPR Therapeutics is collaborating with Vertex on diabetes therapies, focusing on editing induced pluripotent stem cells (iPSCs) to create immune-stealth pancreatic cells [59][60] - The company is optimistic about the potential of its VCTX211 program, which aims to eliminate the need for immunosuppression in Type 1 diabetes patients [60][61] In Vivo Programs - The company plans to enter clinical trials for its ANGPTL3 and Lp(a) programs, leveraging advancements in CRISPR delivery to the liver [74][75] - ANGPTL3 targets a rare disease population, while Lp(a) has a broader market potential, with 11 million patients in the U.S. alone [77] Conclusion - CRISPR Therapeutics is positioned to make significant advancements in gene editing therapies, with a strong focus on commercialization, patient needs, and expanding its treatment capabilities across various diseases [5][21][25]