Metagenomi Therapeutics (MGX) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Vision and technology platform

• Focused on developing curative genetic medicines using a proprietary, AI-driven metagenomics platform for programmable genome editing.

• Metagenomics involves extracting DNA from diverse environments and engineering novel cellular machinery for gene editing.

• Toolbox includes nucleases, base editors, RIGs, and Cas systems for a range of genetic corrections, from small to large edits.

• Programmable nucleases and compact SMART systems offer advantages in precision and targeting over CRISPR-Cas9.

• Base editors and RIGs enable single nucleotide changes and large genome integrations, with demonstrated editing in human cells.

Strategic partnerships and business development

• Ionis partnership targets up to eight in vivo gene editing programs for cardiometabolic diseases, with significant milestone and royalty potential.

• Affini-T collaboration focuses on ex vivo immuno-oncology cell therapies, with options for milestones and royalties.

• Actively seeking new partnerships to accelerate clinical development, especially for ex vivo and multiplex cell editing programs.

• Announced plans in Q2 to seek partners for the PH1 program and ex vivo gene editing, while focusing in-house on in vivo programs.

Pipeline and clinical progress

• Pipeline selection prioritizes compelling disease biology, available biomarkers, clear development paths, and unmet medical needs.

• Lead program in Hemophilia A (MGX001) achieved 12-month durability in non-human primates, with plans to expand to other secreted protein disorders.

• Ionis collaboration includes four programs, two announced (transthyretin amyloidosis, refractory hypertension), all in lead optimization.

• Additional programs target alpha-1 antitrypsin, Wilson's disease, and extrahepatic indications such as neuromuscular, lung, and kidney.

• Achieved a milestone with Affini-T supporting regulatory filings and cGMP material supply.