Summary of Prime Medicine Conference Call - October 14, 2025 Company Overview - **Company**: Prime Medicine (NasdaqGM:PRME) - **Focus**: Development of novel gene and cell therapies utilizing a prime editing platform, which is described as a next-generation gene editing technology that is both versatile and safe [2][4][5] Core Technology and Advantages - **Prime Editing**: - Makes single-stranded breaks in DNA, avoiding double-stranded breaks, which reduces off-target effects and chromosomal rearrangements [5][6] - Capable of writing new genetic code directly into the genome, allowing for permanent changes [6][7] - Can address various mutations, including transition, transversion, and frameshift mutations [7][8] Pipeline Focus - **Current Pipeline**: - Focused on liver diseases, specifically: - **Wilson's Disease**: Expected to enter the clinic in the first half of 2026 [8][9] - **Alpha-1 Antitrypsin Deficiency**: Anticipated IND filing in mid-2026 [9][10] - Future programs include cystic fibrosis, with a goal to treat over 90% of patients [10][11] Discontinued Programs - **Chronic Granulomatous Disease (CGD)**: - Program discontinued due to limited patient population (approximately 1,000 patients in the US) and commercial viability concerns despite strong initial clinical data [12][14] Market Opportunity - **Wilson's Disease**: - Estimated patient population in the US: 10,000 to 11,000 [28] - Potential to treat over 60% of patients with various mutations, leading to a global market opportunity exceeding 20 billion dollars [30][29] Clinical Development and Data Expectations - **IND Filing**: - Guidance for IND filing for Wilson's Disease in the first half of 2026, with clinical data expected in 2027 [31][50] - **Trial Design**: - Biomarkers for Wilson's Disease include copper PET scans and ceruloplasmin levels to assess treatment efficacy [35][36] Competitive Landscape - **Alpha-1 Antitrypsin Deficiency**: - Acknowledgment of competition in the space, but confidence in prime editing's ability to provide a best-in-class therapy due to its endogenous control mechanism [37][38] Delivery Mechanism - **Lipid Nanoparticle (LNP) Technology**: - Universal liver LNP has shown high hepatocyte editing efficiency and is expected to facilitate rapid progression of multiple programs [42][44] - Potential for scalability beyond liver applications, with ongoing exploration of delivery methods for other organs [44][45] Collaboration and Partnerships - **Strategic Collaborations**: - Emphasis on the importance of partnerships for funding and expanding the reach of prime editing technologies [46][48] - Successful collaboration with BMS in ex vivo CAR T therapies, securing $110 million upfront [48] Financial Position - **Cash Runway**: - Approximately $260 million at the end of Q2 2025, expected to sustain operations into 2027 [50] Key Catalysts - **Upcoming Milestones**: - Initiation of clinical programs for Wilson's Disease and Alpha-1 Antitrypsin Deficiency in 2026, with significant data readouts anticipated in 2027 [50]