dna microscopic view

Why STELLAR™ Matters

A Safer, More Flexible Approach to Genetic Medicine

STELLAR™ was engineered to solve the biggest challenges holding gene therapy back — from immune activation to payload limitations to the inability to re-dose.

By leveraging a natural trafficking pathway used by the cell’s own nucleolin protein, STELLAR™ delivers genetic material directly to the nucleus without triggering innate immunity.

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How STELLAR™ Works

“The Three-Step Delivery Pathway”

nuclear pore

1

Targeted Binding

o Nanoparticle binds to nucleolin on the cell surface.

Visual: cell membrane with “ligand-to-nucleolin” connection.

2

Protected Uptake

o Nanoparticle moves through a non-degradative endosomal pathway.

Visual: vesicle carrying intact particle.

3

Active Nuclear Delivery

o DNA/RNA enters nucleus and initiates expression.

Visual: nucleus with transcription spark icon.

Side Benefits

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No toll receptor
activation
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No immune response
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Works in dividing &
non-dividing cells
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Repeat dosing possible
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No payload size limit

What Makes STELLAR™ Different

Designed for Repeatability, Scalability, and Real-World Use
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Compacted nanoparticles (8–22 nm) enable efficient nuclear entry.
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Fully non-viral: no capsids, no antibodies, no immunogenic memory.
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Capable of delivering very large plasmids or mRNA.
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Manufactured from off-the-shelf, cGMP raw materials.
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Highly stable with multi-year shelf life.
3 d representation dna

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How STELLAR™ Compares

 
Copernicus (STELLAR)
copernicus diff
Non-Viral LNP mRNA
(Arcturus / ReCode / Vertex)
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Viral Vector
(4DMT AAV / Spirovant AAV / BI Lenti)
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Modality Compacted DNA/RNA nanoparticles, non-viral mRNA in LNPs, non-viral AAV / Lenti viral vectors
Mutation Coverage Agnostic (full-length CFTR DNA or mRNA) Agnostic (Arcturus, Vertex); nonsense-focused first (ReCode) Agnostic (full CFTR)
Re-Dosing Agnostic (full-length CFTR DNA or mRNA) Repeatable in principle (innate-immune/tolerability constraints) Constrained by anti-capsid or integration (AAV NAbs; lenti typically one-and-done)
Innate Immunity / TLR Reported minimal / no TLR activation Known TLR/innate activation possible with mRNA/LNP Innate & adaptive responses to capsid; pre-existing NAbs common
Durability DNA can enable longer expression vs mRNA mRNA short-lived; requires frequent dosing AAV/lenti can be long-lasting after one dose
Stage of Development Completed Ph.1 human POC; Ph.2 Ready Arcturus (Ph.2); ReCode (Ph.1/1b); Vertex (Ph 1/2 paused MAD) 4DMT (Ph 1/2 with Ph 3 plan); Spirovant (Ph 1/2); BI (Ph 1/2)

Explore Our Clinical Pipeline