CAMBRIDGE, Mass., April 27, 2026

Mirai Bio announced it will unveil two next-generation programs focused on targeted nucleic acid delivery beyond the liver at ASGCT 2026, highlighting machine learning–driven lipid nanoparticle (LNP) innovation and in vivo immune cell engineering. The presentations demonstrate how the company is addressing one of the field’s most critical challenges—precise delivery of genetic medicines to complex tissues and specific cell types.

Machine Learning Optimizes Adipose Tissue Delivery

A key highlight from Mirai Bio’s oral presentation is its machine learning (ML)-guided optimization of LNPs to achieve selective delivery of mRNA to adipose tissue, an area historically difficult to target. Using a large proprietary in vivo biodistribution dataset, the company applied iterative ML modeling to refine nanoparticle design, resulting in enhanced tissue selectivity while minimizing off-target delivery to the liver and spleen.

This advancement is particularly significant for therapeutic areas such as obesity and metabolic disorders, where effective delivery to adipocytes has remained a major barrier. The findings demonstrate that data-driven design can unlock new therapeutic possibilities, enabling more precise and efficient delivery of nucleic acid medicines. By improving both selectivity and tolerability, Mirai’s approach represents a major step forward in expanding genetic medicine applications beyond traditional liver-targeted therapies.

Targeted T-Cell Delivery Enables In Vivo CAR-T Innovation

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In addition to adipose tissue targeting, Mirai Bio presented two poster studies focused on T-cell delivery, showcasing its CD8-targeted LNP platform for in vivo immune cell engineering. The data highlight the development of novel ionizable lipids and long-circulating nanoparticle compositions, designed to achieve extrahepatic delivery directly to T cells.

Preclinical findings demonstrate efficient in vivo generation of CAR-T cells, including CD20-targeted CAR-T cells, leading to robust B-cell depletion in both mouse and non-human primate (NHP) models. Importantly, the platform achieved high specificity, reduced off-target effects, and strong tolerability, addressing key limitations of traditional delivery systems. The ability to move from candidate design to NHP validation in just 10 months underscores the platform’s speed and translational potential.

This approach represents a paradigm shift in CAR-T therapy development, enabling in vivo programming of immune cells without complex ex vivo manufacturing. Such advancements could significantly improve scalability, accessibility, and cost-effectiveness of cell-based therapies, particularly for B-cell-driven diseases and broader immunological indications.

Expanding the Frontier of Nucleic Acid Medicines

Mirai Bio’s ASGCT presentations collectively highlight its leadership in next-generation delivery technologies, combining machine learning, advanced lipid chemistry, and in vivo validation to solve critical challenges in genetic medicine. The company’s platform is designed to deliver precise, scalable, and functional nucleic acid therapies across diverse tissues and cell types, supporting a wide range of therapeutic applications.

By focusing on extrahepatic delivery, Mirai is helping to expand the boundaries of what is possible in RNA and gene-based therapeutics. The ability to target adipose tissue and immune cells with high precision opens new opportunities in metabolic disease, oncology, and immunology, areas with significant unmet medical need.

As the field continues to evolve, Mirai Bio’s integrated approach to particle design, data analytics, and translational development positions it as a key enabler for partners developing next-generation nucleic acid medicines. The data presented at ASGCT 2026 reinforce the importance of delivery innovation as a cornerstone of therapeutic success, paving the way for more effective and accessible genetic treatments.

Source: Mirai Bio press release