Core Insights - Metagenomi, Inc. has made significant advancements in gene editing technologies, particularly for treating neurological disorders through extrahepatic in vivo gene editing [1][2] - The company presented three abstracts at the ASGCT 28 Annual Meeting, showcasing its proprietary gene editing toolbox, including compact nucleases and CRISPR-associated transposases (CAST) [1][2] Gene Editing Technologies - The metagenomics discovery platform utilizes natural microbial evolution and AI-guided protein optimization to identify and enhance novel gene editing systems [2] - Compact Type II and Type V nucleases have been developed for efficient in vivo editing, with a focus on delivering large therapeutic genes [2][9] - The CAST system has shown promise in addressing challenges related to targeted and precise large gene integration [2] Presentation Highlights - A compact type II CRISPR system (MG21-1) demonstrated up to 69% target protein reduction in the CNS with localized AAV9 delivery [6] - An ultra-compact type V nuclease (MG119-28) achieved over 50% restoration of dystrophin protein levels in DMD model myocytes and 64% knockdown of target protein in the motor cortex [6] - The type V-K CAST system has improved integration efficiency by over 50 times compared to natural systems, indicating its potential for therapeutic applications [9] Company Overview - Metagenomi leverages AI and machine learning to develop next-generation genome editing therapeutics, analyzing over 7.4 billion proteins to target various genetic mutations [7] - The company has a lead program in Hemophilia A, aiming to provide lifelong protection from bleeding events and joint damage [7]
Metagenomi Presents Data Highlighting Advancements in Next-Generation Genome Editing Technologies at ASGCT Annual Meeting 2025