- Findings show Mirai’s in vivo data-driven machine learning platform can optimize lipid nanoparticles for adipocyte delivery while reducing off-target delivery to liver and spleen
- Data highlight high adipocyte transfection and low off-target signal, supporting adipose tissue as a high-value delivery frontier for nucleic acid medicines in obesity and metabolic diseases
- Results demonstrate the broader potential of Mirai’s platform to turn delivery into a scalable strategic advantage across tissue-specific nucleic acid medicine programs
CAMBRIDGE, Mass.--(BUSINESS WIRE)--#ASGCT26--Mirai Bio, the innovation delivery partner for companies developing next generation nucleic acid medicines, today announced new preclinical data from an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting 2026 demonstrating that its in vivo data-driven machine learning platform can optimize lipid nanoparticles (LNPs) for delivery to adipocytes, a historically difficult-to-reach cell type central to obesity and related metabolic diseases. The findings reinforce that delivery is a core area of therapeutic innovation, expanding the tissues nucleic acid medicines can reach and what disease biology they can address beyond the liver.
“Nucleic acid medicines have enormous therapeutic potential, but that potential is only realized when the right cargo reaches the right cells at the right level of activity,” said Jagesh V. Shah, Ph.D., SVP, Head of Platform at Mirai Bio. “These data show that delivery is not an enabling step after cargo design; it is a core area of scientific innovation. By combining novel lipid chemistry, in vivo biodistribution data, and machine learning, Mirai is building a repeatable approach for multiparameter optimization of LNPs against the delivery attributes that determine whether a nucleic acid medicine can advance.”
Adipocytes represent a high-value extrahepatic delivery frontier because of their central role in obesity and metabolic disease. Mirai’s adipocyte program applies iterative in vivo machine learning optimization across LNP components and composition, with the goal of increasing on-target white adipose tissue delivery while reducing off-target delivery to liver and spleen. In the data presented at ASGCT, Mirai screened more than 400 proprietary ionizable lipids for in vivo biodistribution and selected top-performing lipids for machine learning-guided optimization. Using in vivo readouts, the platform identified optimized LNPs with distinct component and composition profiles that improved adipocyte delivery and selectivity.
Lead LNPs achieved high white adipose tissue expression, low off-target signal, and significant adipocyte transfection. Cellular profiling confirmed adipocyte delivery, with immunofluorescence showing high on-target adipocyte delivery and liver immunohistochemistry showing minimal off-target liver delivery for lead LNPs. Flow cytometry further showed low off-target delivery to non-adipocyte cell populations.
Shah continued, “For partners, these findings create a path to cargo-ready delivery vehicles for adipocyte-directed programs in obesity and related metabolic diseases. For the broader field, they show how Mirai can extend its platform across tissue-specific delivery challenges, creating scalable value from one of the most important constraints facing nucleic acid medicines.”
The data were presented by Jung Hoon Yang, Ph.D., Associate Director, Lead in LNP Productions at Mirai Bio, in an oral presentation titled “Maximizing delivery of mRNA lipid nanoparticles to adipocyte tissue through an iterative in vivo machine learning approach” at the ASGCT Annual Meeting 2026.
About Mirai Bio
Mirai Bio is delivering genetic medicines from promise to performance. Founded by Flagship Pioneering, Mirai was built to solve one of the field’s central challenges: achieving precise, scalable delivery of diverse nucleic acid payloads to specific cells and tissues, particularly beyond the liver. By combining a large and growing proprietary in vivo biodistribution dataset, machine learning-guided lipid nanoparticle optimization, modular targeted particle design, and integrated translation and manufacturing capabilities, Mirai helps partners accelerate development, reduce technical risk, and pursue high-value programs making delivery a strategic advantage. For more info, visit www.miraibio.com.
Contacts