GEMMABio Secures ARPA-H Funding to Advance AI-Driven Gene Editing

The collaboration focuses on addressing homozygous familial hypercholesterolemia and maple syrup urine disease, two conditions historically requiring invasive procedures like liver transplants. By utilizing a nonviral delivery method, researchers aim to encapsulate mRNA-encoded base editors into lipid nanoparticles that target hepatocytes directly. This approach bypasses traditional viral vectors, potentially streamlining manufacturing and reducing costs.

Profluent Bio will contribute its proprietary protein language models to generate base editors capable of correcting transition mutations with high specificity. The project is part of the ARPA-H THRIVE program, which emphasizes the development of scalable, in vivo genetic medicines. Beyond the technical development, the team is establishing a regulatory consortium led by the United Kingdom’s Medicines and Healthcare products Regulatory Agency to synchronize data sharing and accelerate the path toward future clinical trials.