Core Insights - CRISPR Therapeutics (CRSP) shares have increased by 41.4% over the past three months, driven by positive data from the CTX310 study and strong sales of Casgevy in Q2 2025 [1][9]. In Vivo Pipeline Programs - CRISPR Therapeutics is advancing its in-vivo candidates, CTX310 and CTX320, in separate phase I clinical studies targeting ANGPTL3 and lipoprotein(a) respectively [2]. - Updated data from the CTX310 study showed significant reductions in low-density lipoprotein (LDL) and triglyceride (TG) levels, with peak reductions of up to 86% in LDL and 82% in TG [2][9]. - Data for CTX320 is anticipated in the first half of 2026, raising hopes for its potential in treating heart disease [3]. Casgevy Sales Performance - Casgevy, a one-shot gene therapy developed in partnership with Vertex Pharmaceuticals, was approved for sickle cell disease and transfusion-dependent beta-thalassemia [4]. - After a slow start in 2024 with only $10 million in revenue, Casgevy's sales surged to $30.4 million in Q2 2025, marking a 114.1% sequential increase [5][6]. - Over 75 treatment centers have been activated for Casgevy, with expectations for significant growth in new patient starts throughout 2025 [6]. Other Pipeline Candidates and Collaborations - CRISPR Therapeutics is developing next-generation CAR-T therapy candidates, CTX112 and CTX131, in separate phase I/II studies, with data expected later in 2025 [7]. - The company plans to expand its in-vivo pipeline with two additional programs, CTX340 and CTX450, by the end of the year [8]. - A recent collaboration with Sirius Therapeutics aims to diversify CRSP's pipeline into RNA therapeutics, focusing on the development of SRSD107 for thromboembolic disorders [10][11]. Competitive Landscape - CRISPR Therapeutics faces competition from other companies in the gene-editing space, including Beam Therapeutics and Intellia Therapeutics, which are advancing their own therapies [12]. - Casgevy also competes with chronic therapies like Bristol Myers' Reblozyl and Novartis' Adakveo [13]. Stock Performance and Valuation - CRSP shares have risen 34.8% year-to-date, outperforming the industry and the S&P 500 [14]. - The stock is trading at a price-to-book (P/B) ratio of 2.68, lower than the industry average of 2.95, indicating a potential valuation opportunity [15]. Estimate Movements - Estimates for CRISPR's 2025 loss per share have widened from $5.58 to $6.26, while estimates for 2026 have narrowed from $4.30 to $3.98 [18]. Financial Position - CRISPR Therapeutics has a strong cash balance of approximately $1.7 billion as of June-end, which supports ongoing operations and potential late-stage studies [20].

Financial Data and Key Metrics Changes - Total operating loss from continuing operations increased from $6.6 million in 2023 to $6.9 million in 2024 [22] - Research and development expenses rose to $3.7 million in 2024 from $3.4 million in 2023 [22] - General and administrative expenses decreased from $3.5 million in 2023 to $3.2 million in 2024 [22] - Net loss for 2024 was $8.5 million compared to $4.3 million in the previous year [23] - Cash and cash equivalents at year-end 2024 increased to $1 million from $700,000 at the end of 2023 [25] Business Line Data and Key Metrics Changes - The RNA delivery business showed progress with new partnerships and improved manufacturing processes [6] - The company aims to advance at least one flagship program to an investigational new drug IND filing with the FDA in 2026 [14] - The legacy assets, including the nasal spray products, are expected to see significant commercial opportunities with ongoing discussions for partnerships [20] Market Data and Key Metrics Changes - The global radiopharmaceutical market reached $8.4 billion in size in 2024 and is expected to grow to $17.1 billion by 2033 [13] - The company received marketing approval for its nasal spray in Mainland China, indicating a major commercial opportunity [17] Company Strategy and Development Direction - The company plans to spin off a majority of its RNA delivery subsidiary, Altamira Therapeutics AG, to better capitalize on growth opportunities [8] - The strategy includes licensing the RNA delivery platform technology to partners in the biotech and pharma industry [12] - The company is transitioning to a holding company model, focusing on managing and monetizing its participations and assets [25] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the challenges posed by the NASDAQ delisting but expressed optimism about the planned spin-off and private equity involvement [28] - The company is focused on building its RNA delivery business and addressing the potential for RNA therapeutics [28] Other Important Information - The financial statements for 2024 were presented in U.S. dollars for the first time, reflecting a shift in reporting [20] - The company has filed several patent applications to protect its proprietary programs [14] Q&A Session Summary Question: What are the expectations for the RNA delivery business? - Management highlighted the growing momentum in the RNA delivery business and the importance of appropriate funding to continue progress [27] Question: How does the company plan to address the NASDAQ delisting? - Management discussed the painful experience of the delisting but emphasized the planned spin-off and private equity involvement as beneficial alternatives [28]