CAMBRIDGE, Mass., May 14, 2026
Mirai Bio presented new preclinical data at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting 2026 demonstrating that its machine learning-driven lipid nanoparticle (LNP) platform can successfully optimize delivery of nucleic acid medicines to adipocytes, a historically difficult-to-target cell type associated with obesity and metabolic disease. The findings highlight a major advance in expanding genetic medicine delivery beyond the liver into high-value extrahepatic tissues.
Mirai Uses Machine Learning to Improve Adipocyte Delivery
According to Mirai Bio, the company screened more than 400 proprietary ionizable lipids using in vivo biodistribution analysis to identify optimized LNP formulations capable of selectively targeting white adipose tissue while reducing off-target delivery to the liver and spleen. Using iterative machine learning optimization, Mirai developed lead LNP candidates with high adipocyte transfection efficiency, strong white adipose tissue expression, and significantly lower off-target activity in non-adipocyte tissues.
The company reported that immunofluorescence imaging confirmed high on-target adipocyte delivery, while liver immunohistochemistry demonstrated minimal unintended liver accumulation. Additional flow cytometry analysis showed low delivery to non-adipocyte cell populations, reinforcing the selectivity profile of the optimized LNP formulations. These findings suggest that adipose tissue could become a viable therapeutic target for next-generation mRNA and nucleic acid medicines aimed at obesity and metabolic disorders.
Data Strengthen Extrahepatic Genetic Medicine Development
Mirai emphasized that the study demonstrates how delivery optimization itself is becoming a central innovation driver in the genetic medicines sector. Traditionally, many nucleic acid therapies have struggled to efficiently reach tissues beyond the liver, limiting the range of diseases addressable through RNA and gene-based therapeutics.
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Dr. Jagesh V. Shah, Senior Vice President and Head of Platform at Mirai Bio, stated that combining novel lipid chemistry, in vivo biodistribution datasets, and machine learning-guided optimization creates a scalable strategy for designing tissue-specific delivery systems. The company believes its platform can help partners accelerate development of targeted nucleic acid therapies while reducing technical risk associated with delivery challenges.
The oral presentation, delivered by Jung Hoon Yang, Ph.D., focused on maximizing mRNA LNP delivery to adipocyte tissue through iterative in vivo optimization approaches. The data further reinforce growing industry interest in expanding genetic medicine applications into obesity, metabolic disease, and other extrahepatic therapeutic areas.
Source: Mirai Bio press release
