GENE THERAPY REIMAGINED
Who is DNA Nanobots?
The DNA Nanobot Story
Solving delivery. Expanding what gene therapy can reach.
DNA Nanobots was founded in 2021 in Columbus, Ohio as a spin-out from The Ohio State University, built on more than a decade of foundational work in DNA nanotechnology by Professor Carlos E. Castro and colleagues. What began as a platform technology has evolved into a focused gene therapy company with a single, central conviction: the limitations holding back gene therapy are not biological. They are structural.
From that insight, we developed the Genobot, a structurally engineered DNA delivery system designed to overcome the core constraints facing the field: large gene payloads, repeat dosing, and controlled targeting.
The Genobot Platform
Where others see genetic information, we see construction material. Using DNA origami as our foundation, the Genobot is a programmable structure in which the gene itself becomes the delivery system. Functional elements are precisely positioned on the construct, enabling design-guided control over biological behavior.
Current gene delivery systems were not designed for large or structured payloads. They carry inherent limitations that are a function of their biology, not the genes they carry. We take a different approach. Engineer the structure, and let the structure solve the problem.
Our Vision
To expand the reach of gene therapy by solving the delivery constraints that have limited it to a narrow set of diseases and patient populations. We are building the platform that enables a new generation of large-gene therapeutics.
Our Mission
We are advancing a programmable, non-viral gene delivery platform from preclinical validation toward disease-focused development. Our near-term pathfinder asset is Wilson’s Disease. Our lead long-term objective is Duchenne Muscular Dystrophy. We are also pursuing Hemophilia B as an additional pipeline program. Our goal is to deliver genes that current modalities simply cannot.
Where We’re Focused
One platform. Engineered to deliver what existing modalities cannot.
Wilson’s Disease
A liver-accessible rare disease with measurable biomarkers. Being developed as a pathfinder asset to validate systemic delivery, gene expression, and repeat dosing with the Genobot platform.
Duchenne Muscular Dystrophy (DMD)
Our lead long-term objective. Full-length dystrophin exceeds the payload capacity of AAV and current truncation-based approaches. The Genobot is being designed to deliver it.
Hemophilia B
A liver-targeted rare disease caused by Factor IX deficiency. Hemophilia B is being explored as an additional program within the Genobot platform, given the liver’s accessibility and the potential for a single-administration gene delivery approach to restore clotting function.
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