Metagenomi Reports Business Updates and Second Quarter 2025 Financial Results

Core Insights - Metagenomi, Inc. presented three abstracts at the American Society of Gene & Cell Therapy (ASGCT), showcasing early proof-of-concept data across multiple gene editing technologies [1] - The company reported a strong cash position of $205.0 million as of June 30, 2025, which is expected to support operations into 2027 [1][8] - The company is advancing its MGX-001 program for hemophilia A towards pre-IND and ex-U.S. regulatory meetings in 2025 [2] Financial Results - For the quarter ended June 30, 2025, research and development (R&D) expenses were $22.5 million, down from $28.3 million in the same period of 2024 [8] - General and administrative (G&A) expenses were $7.0 million for the quarter ended June 30, 2025, compared to $8.6 million for the same period in 2024 [8] - The net loss for the quarter was $19.9 million, compared to a net loss of $10.7 million in the same quarter of 2024 [15] Program Updates - The MGX-001 program for hemophilia A demonstrated durable FVIII activity levels through approximately 19 months, with mean FVIII activity of 75%, 8%, and 29% in three nonhuman primate subjects [7] - The company plans to submit an investigational new drug (IND) application in 2026 based on current data [7] - Key advancements in the gene editing platform were presented at ASGCT, including compact nucleases for extrahepatic gene editing and CRISPR-associated transposases (CAST) for large therapeutic gene integration [2][7] Corporate Developments - Laurence Reid, Ph.D., was appointed to the Board of Directors, bringing extensive experience in biotech [7] - A Research and Development Committee was established within the Board to provide insights on scientific and clinical strategies [7] Market Context - Hemophilia A is the most common X-linked inherited bleeding disorder, with an estimated prevalence of up to 26,500 patients in the U.S. and over 500,000 globally [9] - Metagenomi's gene editing toolbox has analyzed over 7.4 billion proteins, indicating its potential to target various genetic mutations across the human genome [10]