PHILADELPHIA, Pennsylvania, USA, July 9, 2026
GEMMA Biotherapeutics (GEMMABio) has been awarded funding from the Advanced Research Projects Agency for Health (ARPA-H) to accelerate the development of AI-designed base-editing therapies for rare inherited liver diseases. The funding, awarded under ARPA-H’s Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines (THRIVE) program, will support the company’s Regional-based Personalized Medicines Development (RAPID) project in collaboration with Profluent Bio, an artificial intelligence-driven biotechnology company specializing in next-generation gene-editing technologies. The project aims to develop nonviral, lipid nanoparticle (LNP)-delivered mRNA-based precision genetic medicines targeting founder mutations responsible for homozygous familial hypercholesterolemia (HoFH) and maple syrup urine disease (MSUD). By combining AI-designed base editors, advanced mRNA delivery technologies, and scalable manufacturing approaches, the collaboration seeks to establish a new platform capable of delivering personalized genetic medicines with improved accessibility, reduced development costs, and broader global impact for patients living with rare genetic disorders.
AI-Designed Base Editors Target Rare Genetic Liver Diseases
Under the RAPID initiative, GEMMABio and Profluent Bio will develop four lipid nanoparticle-delivered, mRNA-encoded base-editing therapies designed to precisely correct disease-causing founder mutations in the LDLR and BCKDHB genes associated with HoFH and MSUD, respectively. Profluent will contribute its proprietary AI-powered protein language models and advanced base-editing platform, generating highly specific editors capable of correcting transition mutations while minimizing unwanted off-target effects.
GEMMABio will utilize a nonviral delivery strategy, encapsulating mRNA encoding the base-editing machinery into lipid nanoparticles optimized for delivery to hepatocytes, the primary cells of the liver. This precision approach aims to enable one-time, personalized genetic treatments that directly repair disease-causing DNA mutations without relying on viral vectors, representing an important advancement in the evolution of next-generation gene editing technologies.
Innovative Platform Supports Personalized Genetic Medicines
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A major objective of the project is the development of a scalable base-editing platform built around a common Cas nickase scaffold combined with programmable guide RNAs tailored to individual patient mutations. By using a standardized editing system where only the guide RNA changes for each mutation, researchers aim to simplify manufacturing, streamline clinical development, reduce production costs, and improve regulatory efficiency for personalized therapies. In parallel, GEMMABio will identify clinically validated lipid nanoparticle formulations capable of efficiently delivering the editing machinery directly to liver cells.
The project also includes collaboration with international medicinal product regulators, coordinated by the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA), to support regulatory harmonization and accelerate future clinical trial development. Additionally, GEMMABio plans to establish genetic screening programs to identify eligible patients carrying the targeted founder mutations responsible for these devastating rare diseases.
ARPA-H Funding Accelerates Precision Gene Editing Innovation
The ARPA-H THRIVE award highlights growing federal support for innovative precision genetic medicine platforms capable of rapidly translating scientific discoveries into clinical therapies for patients with severe rare diseases. Homozygous familial hypercholesterolemia and maple syrup urine disease are life-threatening inherited disorders with limited treatment options, often requiring intensive lifelong management or liver transplantation. By integrating artificial intelligence, base editing, mRNA therapeutics, and lipid nanoparticle delivery, GEMMABio and Profluent aim to establish proof-of-concept for a versatile gene-editing platform that can be expanded to numerous additional monogenic diseases.
The initiative aligns with ARPA-H’s vision of accelerating safe, scalable, and personalized in vivo precision genetic medicines while improving affordability and expanding patient access worldwide. If successful, the RAPID program could help transform the future of rare disease therapeutics, demonstrating how AI-enabled genome engineering can deliver durable, one-time treatments for previously untreatable inherited disorders.
Source: GEMMABio press release
