Mirai Bio to Reveal Two Next-Gen Targeted Nucleic Acid Delivery Programs Beyond the Liver at ASGCT 2026

Mirai Bio to Debut Two Next-Generation Programs for Extrahepatic Targeted Nucleic Acid Delivery at the ASGCT 2026 Annual Meeting

Mirai Bio, a company focused on enabling the next generation of nucleic acid therapeutics through advanced delivery technologies, has announced its participation in the American Society of Gene & Cell Therapy Annual Meeting 2026. At this prominent scientific gathering, the company will showcase three abstracts, including one oral presentation and two poster sessions, underscoring significant advancements across its targeted lipid nanoparticle (LNP) delivery platforms. These presentations collectively reflect both the increasing complexity of nucleic acid medicine delivery beyond the liver and Mirai Bio’s differentiated approach, which leverages extensive in vivo biodistribution data integrated with machine learning (ML) to design more precise, selective, and well-tolerated delivery systems.

As the field of nucleic acid therapeutics continues to evolve—moving beyond traditional liver-targeted approaches toward more complex tissues and cell types—the challenge of efficient and targeted delivery remains a central bottleneck. Mirai Bio is positioning itself at the forefront of solving this challenge by combining experimental biology with computational modeling. According to Jagesh V. Shah, Ph.D., Senior Vice President and Head of Platform at the company, the ability to deliver nucleic acid medicines effectively into diverse tissues is critical to unlocking their full therapeutic potential. He emphasized that Mirai’s upcoming presentations highlight both the ambitious goals of the broader field and the level of precision required in delivery systems to make those goals achievable.

Shah further noted that Mirai’s work spans multiple biological targets and applications, illustrating the breadth of its platform capabilities. One area of focus is adipose tissue, which has historically been difficult to access using conventional delivery technologies. Through iterative in vivo learning—powered by large-scale biodistribution datasets and machine learning—Mirai has been able to significantly improve tissue selectivity in this domain. In parallel, the company is advancing T-cell delivery strategies, demonstrating high levels of precision, favorable tolerability profiles, and translational potential. These advances are designed to support rapid progression of in vivo delivery programs, particularly in the context of immune cell engineering.

The company’s oral presentation (Abstract 2000) will delve into its work on optimizing mRNA LNP delivery to adipocyte tissue. Scheduled for May 14, the session will be presented by Jung Hoon Yang, Associate Director and Lead in LNP Production at Mirai Bio. This research highlights how iterative machine learning models, trained on extensive in vivo datasets, can be used to fine-tune nanoparticle formulations for improved targeting. By reducing off-target accumulation in organs such as the liver and spleen, the approach enhances delivery efficiency to adipose tissue. This has important implications for diseases such as obesity and metabolic disorders, where therapeutic success has been limited in part due to delivery constraints.

In addition to the oral session, Mirai Bio will present two poster abstracts focused on advancing extrahepatic delivery and immune cell targeting. Poster Abstract 1073 explores the development of novel ionizable lipids and long-circulating LNP formulations engineered to enable targeted mRNA delivery to T cells. Presented by Chelsea Martinez, Ph.D., the work demonstrates how innovations in lipid chemistry can improve the pharmacokinetic and biodistribution profiles of LNPs. These next-generation particles show promising specificity, reduced liver accumulation, and early functional activity in preclinical models. Importantly, the platform supports rapid development timelines, with the potential to advance from lead candidate selection to non-human primate (NHP) studies in approximately ten months.

Complementing this research, Poster Abstract 2008 focuses on Mirai’s CD8-targeted LNP platform and its application in generating CAR T cells directly in vivo. Presented by Brian Duke, Ph.D., this work provides preclinical data from both mouse and NHP studies demonstrating the ability of systemically administered LNPs to deliver mRNA encoding CD20 CAR constructs into CD8-positive T cells. The result is the in vivo generation of functional CAR T cells capable of mediating B-cell depletion. This approach represents a significant shift from traditional ex vivo cell therapy manufacturing, offering the potential for more scalable, cost-effective, and accessible immunotherapies.

The findings also highlight key attributes of Mirai’s platform, including targeted activity, reduced off-target delivery, and favorable tolerability in higher-order species. These characteristics are critical for advancing immune cell engineering strategies into clinical development. By addressing long-standing challenges associated with delivery specificity and safety, Mirai Bio is helping to pave the way for broader application of nucleic acid medicines across a range of diseases, including oncology, autoimmune disorders, and metabolic conditions.

Overall, Mirai Bio’s presence at the 2026 ASGCT Annual Meeting underscores its commitment to pushing the boundaries of nucleic acid delivery science. By integrating machine learning with robust in vivo experimentation, the company is building a platform capable of addressing one of the most persistent challenges in the field. As therapeutic ambitions expand to include increasingly complex biological targets, innovations in delivery—such as those being developed by Mirai—will play a decisive role in determining the success of next-generation medicines.

About Mirai Bio

Mirai Bio is delivering genetic medicines from promise to performance. 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 that would otherwise remain constrained by delivery. 

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