Core Insights - Metagenomi, Inc. is advancing its gene editing candidate MGX-001 for hemophilia A, with regulatory interactions anticipated in 2025 and a focus on achieving proof-of-concept for secreted protein deficiencies [1][2][6] - The company is on track to nominate one to two development candidates from its collaboration with Ionis in 2025, targeting large cardiometabolic indications [1][12] - Metagenomi's cash runway is expected to support its operational plans into 2027, indicating financial stability for ongoing projects [1] Hemophilia A Program - MGX-001 has achieved sustained Factor VIII (FVIII) activity in a nonhuman primate study over 16 months, demonstrating its potential efficacy [6] - The company has engaged with the FDA for initial regulatory discussions and has initiated GxP manufacturing activities for MGX-001 [6] - Plans for 2025 include finalizing the ongoing nonhuman primate durability study and continuing Investigational New Drug (IND) enabling efforts [6] Secreted Protein Deficiencies - In 2024, Metagenomi identified targets for wholly-owned therapeutic programs leveraging the albumin approach used in MGX-001 [4] - The company anticipates demonstrating proof-of-concept for its lead secreted protein deficiency target in nonhuman primates in 2025 [7] Cardiometabolic Programs - Metagenomi has advanced all four Wave 1 Ionis collaboration programs to lead optimization, achieving in vivo proof-of-concept in rodents [12] - The company plans to nominate development candidates from these programs in 2025 and disclose remaining therapeutic indications [12] Technology Development - Metagenomi has made advancements in its gene editing toolbox, including next-generation base editors and ultra-small nucleases [2][12] - The company presented novel Adenine Base Editors (ABEs) that target over 95% of the human genome, demonstrating high specificity and no adverse effects on cell health [12]
Metagenomi Highlights Progress Across Therapeutic Portfolio and Outlines Anticipated Milestones