Mirai Bio's AI Aims Beyond the Liver for New Gene Therapies

CAMBRIDGE, Mass. – April 27, 2026 – Mirai Bio, a company focused on solving the critical delivery challenge for genetic medicines, is set to unveil two next-generation programs at the American Society of Gene & Cell Therapy (ASGCT) 2026 meeting. The presentations will showcase significant progress in using lipid nanoparticles (LNPs) to deliver nucleic acid therapies to tissues beyond the liver, specifically targeting fat cells and T-cells.

The announcement signals a concerted push to overcome one of the primary obstacles that has limited the vast potential of genetic medicine. By leveraging a sophisticated, data-driven platform, Mirai Bio aims to enable new treatments for a range of difficult diseases, from metabolic disorders to cancer.

“Delivery remains one of the defining challenges in nucleic acid medicine, particularly as the field moves toward more complex tissues, cell types, and therapeutic goals,” said Jagesh V. Shah, Ph.D., SVP, Head of Platform at Mirai Bio, in a recent statement. “At ASGCT, Mirai will present work from distinct programs that reflect both the breadth of what the field is trying to achieve and the level of delivery performance needed to make new therapeutic approaches possible for our partners.”

The "Beyond Liver" Challenge and AI's Role

The promise of genetic medicines like mRNA and siRNA has long been tempered by a fundamental biological hurdle: delivery. Lipid nanoparticles, the microscopic fat-based vehicles used to shuttle these genetic instructions into cells, are overwhelmingly filtered out of the bloodstream by the liver. While this natural tendency is advantageous for treating liver diseases, it has severely constrained the application of these powerful therapies to other organs and tissues.

Mirai Bio is positioning itself at the forefront of solving this “extrahepatic” delivery problem. The company’s core strategy combines advanced chemistry with powerful computational tools. Its platform is built upon an extensive, proprietary dataset of in vivo biodistribution results, which feeds a machine learning engine. This AI-driven approach allows the company to predict how different LNP formulations will behave in the body, enabling the generative design of particles that can evade the liver and precisely home in on specific cell types.

This represents a significant evolution in the field, moving away from slower, trial-and-error chemistry toward a data-first design process. The goal is to create LNPs with improved selectivity and enhanced tolerability, thereby unlocking new therapeutic possibilities that were previously out of reach.

Targeting Fat Cells: A New Frontier for Metabolic Disease

One of the most compelling targets for this new technology is adipose tissue, or fat cells. In a planned oral presentation at ASGCT, Mirai will detail its work on optimizing mRNA LNP delivery to adipocytes. This is a crucial area of research, as adipose tissue is a key regulator of energy metabolism and is deeply implicated in widespread conditions like obesity and type 2 diabetes—global health crises with markets valued in the hundreds of billions of dollars.

Historically, delivering therapies directly and efficiently to these cells has been exceptionally difficult. The company’s presentation is set to describe an iterative machine learning process that systematically identified LNP formulations with progressively better delivery to fat cells, all while minimizing unwanted accumulation in the liver and spleen.

“Our adipocyte work highlights how iterative in vivo learning enabled by Mirai’s platform can improve selectivity and expand what is possible in tissues that have historically been difficult to access,” Shah noted. By enabling the direct genetic programming of adipocytes, this technology could pave the way for entirely new classes of treatments for metabolic disorders, potentially correcting the underlying cellular dysfunctions that drive disease progression.

Revolutionizing Immunotherapy with In Vivo T-Cell Engineering

Perhaps even more disruptive is Mirai Bio's second program, focused on delivering genetic cargo directly to T-cells circulating within the body. This work aims to enable in vivo CAR T-cell therapy, a groundbreaking approach that could upend the current treatment paradigm for certain cancers and autoimmune diseases.

Today's CAR T-cell therapies are an arduous and expensive ex vivo process. A patient's T-cells are harvested, shipped to a specialized lab, genetically engineered, multiplied, and then re-infused—a complex logistical chain that can take weeks and cost upwards of $500,000 per dose. This limits access for many eligible patients.

Mirai Bio is pioneering an in vivo approach, essentially turning the patient's body into its own bioreactor. Two poster presentations will highlight the company's progress, detailing novel ionizable lipids and long-circulating LNP compositions designed for precise T-cell targeting. According to the company, preclinical data from mouse and non-human primate (NHP) models show that its CD8-targeted LNP platform can successfully generate CD20 CAR T-cells after a simple systemic administration. These engineered cells then effectively depleted B-cells, a therapeutic mechanism relevant for B-cell-driven cancers and autoimmune disorders.

Critically, the platform promises speed. Mirai claims it can accelerate programs from lead candidate selection to NHP studies in just 10 months. This “off-the-shelf” potential could dramatically reduce costs, shorten treatment timelines, and make powerful cell therapies accessible to a much broader patient population.

A Platform Built for Partnership and Speed

Mirai Bio, which was launched by the prominent life sciences venture firm Flagship Pioneering, is not structured as a traditional drug developer building its own internal pipeline. Instead, it operates as an “innovation delivery partner.” The company's business model is to provide its sophisticated, AI-powered delivery platform to other biotech and pharmaceutical firms, enabling them to pursue high-value programs that would otherwise be stalled by the delivery challenge.

This strategy allows partners to de-risk their development efforts and significantly accelerate timelines. The model is further bolstered by a key strategic collaboration with manufacturing giant Thermo Fisher Scientific, which provides a clear and scalable path from AI-driven design to industrial-scale, GMP-compliant production.

By tackling the fundamental bottlenecks of delivery, design, and manufacturing, Mirai Bio aims to become an essential engine for the entire genetic medicine ecosystem. The upcoming presentations at ASGCT will offer the first public glimpse into whether its technology can deliver on the promise of broadening the impact of these advanced therapies across a wider spectrum of human diseases.