GEMMABio Secures ARPA-H Funding to Advance LNP-Delivered mRNA Base Editing Strategy for Rare Liver Diseases

July 12, 2026

BioSpace USA

Overview

GEMMABio announced securing ARPA-H funding to develop four mRNA-encoded base editing products delivered via lipid nanoparticles (LNPs) in collaboration with Profluent. This innovative strategy targets four pathogenic mutations across two rare monogenic liver diseases: homozygous familial hypercholesterolemia (HoFH) and maple syrup urine disease (MSUD). LNP-mediated mRNA base editing offers a highly specific gene therapy approach for the liver, potentially providing the first definitive treatments for these severe rare conditions.

In Depth

Key Findings

GEMMABio has been awarded funding from ARPA-H (Advanced Research Projects Agency for Health) to accelerate its groundbreaking base editing strategy for rare liver diseases. In a strategic collaboration with Profluent, GEMMABio will develop four novel base editing products encoded by messenger RNA (mRNA) and delivered via lipid nanoparticles (LNPs). This initiative aims to precisely correct specific genetic mutations, offering new hope for severe rare liver diseases where treatment options have been severely limited.

Technical & Clinical Details

The program targets a total of four pathogenic mutations in two distinct rare monogenic liver diseases: homozygous familial hypercholesterolemia (HoFH) and maple syrup urine disease (MSUD). HoFH is characterized by severe hypercholesterolemia due to mutations in the LDL receptor gene, increasing the risk of premature cardiovascular events. MSUD, caused by genetic defects in the branched-chain alpha-keto acid dehydrogenase complex, leads to amino acid metabolism disorders and the accumulation of neurotoxic metabolites. GEMMABio’s approach involves LNP-encapsulated mRNA being specifically delivered to liver cells. Once inside, the encoded base editing tools (Cas proteins and guide RNAs) are expressed, directly and irreversibly converting specific genomic DNA bases, such as A to G or C to T, with high efficiency and precision. This aims to permanently correct the single-base mutations responsible for these diseases, leading to a lasting therapeutic effect.

Background & Context

Rare liver diseases represent a high unmet medical need, with limited effective treatments due to the small patient populations hindering research and development. Monogenic diseases, in particular, hold the promise of curative treatment through genetic-level correction. LNPs, widely recognized for their efficacy and safety in COVID-19 mRNA vaccines, have shown high tropism for the liver, making them a promising carrier for liver-targeted gene therapies. Base editing technology is a next-generation genome editing tool that reduces the risk of off-target effects associated with double-stranded DNA breaks in traditional CRISPR-Cas9 systems, enabling safer and more precise genomic modifications. The ARPA-H funding is crucial support to accelerate the clinical application of this innovative technology.

Strategic Significance & Outlook

The collaboration between GEMMABio and Profluent has the potential to fundamentally transform the treatment paradigm for rare liver diseases. Initial development success could provide definitive therapeutic options for patients with HoFH and MSUD. Furthermore, this LNP-delivered mRNA-based base editing platform holds broad potential for expansion to numerous other genetic diseases, especially those targeting the liver. The advancement of this technology will play a critical role in shaping the future of personalized and precision medicine.

Source: https://www.biospace.com/press-releases/gemmabio-awarded-arpa-h-funding-to-pursue-innovative-base-editing-strategy-for-rare-liver-diseases

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