CRISPR Therapeutics AG (CRSP) Management Presents at Innovation Day Conference (Transcript)

Summary of CRISPR Therapeutics AG Innovation Day Conference Call Company Overview - Company: CRISPR Therapeutics AG (NASDAQ: CRSP) - Date: June 21, 2022 - Key Participants: - Samarth Kulkarni (CEO) - Phuong Khanh Morrow (CMO) - Sumanta Kumar Pal (Co-Director, Kidney Cancer Program) - Swaminathan Iyer (Professor, Lymphoma/Myeloma) - Alireza Rezania (SVP, Head of Regenerative Medicine) Core Industry Focus - Industry: Gene Editing and Biotechnology - Key Areas: Hemoglobinopathies, Immuno-oncology, Regenerative Medicine, In Vivo Therapies Key Points and Arguments Company Progress and Financials - CRISPR Therapeutics has made significant advancements in gene editing, with over 500 employees and a state-of-the-art GMP facility [6] - The company has over $2 billion in its balance sheet, positioning it as a leader in the CRISPR-based revolution [6] - The company is on the verge of filing a Biologics License Application (BLA) for CTX001 (exa-cel) for hemoglobinopathies, which could be the first CRISPR-edited product approved globally [5][6] Hemoglobinopathies - Exa-cel: A gene therapy targeting sickle cell disease and beta-thalassemia, showing promising results: - 42 out of 44 patients with beta-thalassemia became transfusion independent [10] - All patients with sickle cell disease were free of vaso-occlusive crises [10] - The therapy could potentially address around 30,000 patients with these conditions, with plans to expand its reach through targeted conditioning and in vivo delivery [12] - Development of a cKit antibody drug conjugate (ADC) is underway to enhance gene editing delivery [13] Immuno-oncology - CTX110: An allogeneic CAR-T therapy targeting CD19, with ongoing patient dosing and optimization [18] - CTX120: A BCMA-targeting CAR-T therapy, pivoting to a next-generation program due to competitive landscape and efficacy concerns [19] - CTX130: A novel allogeneic CAR-T therapy targeting CD70, with unique edits to enhance efficacy and reduce toxicity: - High CD70 expression in T-cell lymphomas presents a significant unmet need [22][24] - Early data shows a 70% overall response rate and a 30% complete response rate in T-cell lymphoma patients [31][35] Safety and Efficacy Data - CTX130 demonstrated a favorable safety profile with no severe adverse events related to the therapy [29][40] - The efficacy data from the COBALT lymphoma trial showed promising responses across various disease sites, with a 90% disease control rate [31][32] Future Directions - Plans to develop next-generation CAR-T therapies (CTX131) with additional potency edits [35][53] - Ongoing collaborations with academic centers for novel targets beyond CD70, including CD83 and Glypican-3 [56] - The company aims to leverage its CRISPR platform for further innovations in gene editing and therapy development [57] Market Potential - The company believes that the introduction of therapies like Axi-Cel will expand the patient population and improve access to treatments for conditions like sickle cell disease [60] - The predictability and reliability of CRISPR manufacturing provide a competitive advantage over traditional viral methods [64] Additional Important Content - The call emphasized the importance of continuous innovation in gene editing and the potential for CRISPR technology to transform medicine [6][11] - The company is committed to addressing significant unmet medical needs in oncology and hematology through its innovative therapies [24][37]