Abstract scientific visualization of a lipid nanoparticle crossing the blood-brain barrier, with glowing teal particles traversing a cellular membrane layer
Preclinical LNP Delivery Platform

LNP Delivery That Crosses
the Blood-Brain Barrier

Biopathio engineers lipid nanoparticle delivery vehicles for gene therapy developers targeting CNS rare diseases — the organs mRNA alone can't reach.

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~85 nm
Mean LNP particle diameter (PDI < 0.15)
>78%
Encapsulation efficiency for sgRNA cargo
CNS-targeted
Biodistribution profile across the BBB in preclinical models
The Delivery Challenge

The Delivery Problem Gene Therapy Can't Afford to Ignore

Most LNP platforms are optimized for hepatic delivery. The CNS is different: the blood-brain barrier excludes virtually all biologics > 1 nm. Biopathio's ionizable lipid formulations are designed from first principles for BBB transit, CRISPR cargo encapsulation, and endosomal escape in neural tissue.

Ionizable lipid composition

pKa-tuned for endosomal acidification in neural cells, not hepatocytes.

BBB receptor targeting

Surface ligands engineered to engage transferrin and LRP1 receptors for transcytosis.

CRISPR cargo compatibility

Formulated for RNP, mRNA + sgRNA dual-cargo, and base editor delivery.

Characterization-grade formulation

Every batch verified: DLS, cryo-TEM, EE assay, and in vitro CNS cell uptake.

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Mechanism of Action

How Biopathio LNPs Cross the Blood-Brain Barrier

A 5-step pathway from systemic circulation to intracellular CRISPR cargo release in neural tissue.

STEP 1 Bloodstream Circulation STEP 2 BBB Receptor Binding STEP 3 Transcytosis Pathway STEP 4 Brain Parenchyma STEP 5 Endosomal Escape + CRISPR Release Systemic TfR / LRP1 Vesicle transport Neural uptake Schematic — not to scale
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From the Lab

Grounded in Peer-Reviewed Science

Our formulation approach draws on published research in ionizable lipid chemistry, CNS drug delivery, and CRISPR nucleic acid encapsulation.

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Preprint
Ionizable Lipid pKa Optimization for CNS-Targeted Nanoparticle Delivery Across the Blood-Brain Barrier
Watts S, Mehta P, Chen M. bioRxiv. 2024.
Conference
Receptor-Mediated BBB Transcytosis of Engineered Lipid Nanoparticles: In Vitro Evidence in hCMEC/D3 Models
Mehta P, Watts S. ASGCT Annual Meeting, Poster Presentation. 2024.
Preprint
Dual-Cargo CRISPR LNP Formulation for Base Editor Delivery in Primary Cortical Neurons
Chen M, Watts S, Mehta P. bioRxiv. 2025.
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Building a CNS Gene Therapy Program?

If your team has a validated target but no delivery mechanism for the CNS, Biopathio offers formulation collaboration, feasibility studies, and custom LNP engineering for your payload.

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