Metagenomi (MGX) Earnings Call Presentation

Company Vision and Strategy - Metagenomi's vision is to harness its metagenomics platform to create curative genetic medicines for patients[8] - The company's strategy involves using a diverse and modular genome editing toolbox to precisely target any site in the human genome, addressing the full spectrum of genetic diseases[9] - Metagenomi focuses on investments in development and manufacturing across gene editing technologies to enable rapid advancement to the clinic[11] Disease Targets and Pipeline - Hundreds of millions of people worldwide have diseases potentially treatable with gene editing, including over 180 million with rare diseases of genetic origin and over 200 million with cardiovascular disease tied to genetic risk factors[14] - The company's pipeline includes programs targeting Hemophilia A (affecting more than 500,000 patients globally), Transthyretin Amyloidosis (TTR) (affecting 300,000–500,000 patients globally), and Refractory Hypertension (affecting 900,000 patients in the US)[14, 19] - Metagenomi's Hemophilia A program, MGX-001, has demonstrated sustained Factor VIII activity in non-human primate (NHP) studies for more than 16 months[53] Technology and Platform - Metagenomi's metagenomics platform is the foundation of its gene editing toolbox, involving proprietary sampling, AI-powered screening, and engineering & optimization[20, 22, 24, 25] - The company has a proprietary library of highly precise and efficient nucleases, including ultra-small systems (SMARTs), providing a programmable chassis for other gene editing tools[31] - Metagenomi's base editing platform achieves efficient multiplex editing for cell engineering, with reprogrammed chassis increasing genome targetability[112, 113] Partnerships and Corporate Overview - Metagenomi has strategic collaborations, including one with Ionis focusing on cardiometabolic diseases, with up to 8 targets and potential milestone payments and royalties up to $2.9 billion[50] - The company is well-capitalized with a cash runway into 2027[135]