GEMMABio Awarded ARPA-H Funding to Pursue Innovative Base-editing Strategy for Rare Liver Diseases

●     The goals of this project will be enabled through a partnership with Profluent Bio who will deploy its frontier AI models and its scalable base editor platform

●     GEMMABio will utilize a nonviral approach, encapsulating mRNA coding for Profluent's base-editing machinery into lipid nanoparticles to target hepatocytes

●     The award will support research & development that should lead to new personalized medicines for founder mutations that cause homozygous familial hypercholesterolemia and maple syrup urine disease