A Smarter Way to Deliver CRISPR to the Brain

Gene editing has the power to transform how we treat disease, but delivery remains the biggest challenge, especially in the central nervous system. Traditional approaches like AAV and lipid nanoparticles fall short in the brain, limiting the therapeutic reach of CRISPR.

The Problem with Current Delivery Technologies

AAV Vectors

AAV vectors are widely used, but they bring major drawbacks: limited cargo capacity, prolonged expression, immunogenicity, and manufacturing complexity. These issues are especially acute for CRISPR, where dose control and transient expression are essential.

Lipid Nanoparticles

Lipid nanoparticles have limited penetration in brain tissue, resulting in poor distribution and reduced editing efficiency, even with local or direct administration.

Most CRISPR therapeutics can’t reach the brain, or can’t do so safely.

Introducing
PERC™

(Peptide-enabled RNP delivery of CRISPR)

PERC™ is a next-generation, non-viral delivery platform designed for potent, precise genome editing in vivo, starting in the brain and built to scale beyond it.

PERC™ delivers CRISPR as a fully assembled RNP, functionalized with engineered peptides that drive cellular uptake, endosomal escape, and efficient genome editing in vivo.

Why It Matters

PERC™ addresses the limitations of viral and lipid-based delivery with a fundamentally different approach:

  • Potent in vivo efficacy: high editing efficiency in neurons with broad tissue distribution

  • Built for safety: transient, non-integrating delivery minimizes toxicity, immunogenicity, and off-target risk

  • Cargo agnostic: compatible with a wide range of CRISPR effectors, including base editors and nucleases

  • Scalable & GMP-ready: simple to manufacture, designed for clinical scale-up, and optimized for cost-effective development

Select Publications

  • Foss et al., Nature Biomedical Engineering (2023)

    Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes.

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  • Sahu et al., Nature Protocols (2025)

    Peptide-enabled ribonucleoprotein delivery for CRISPR engineering (PERC) in primary human immune cells and hematopoietic stem cells.

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  • Manohar et al., BioRxiv (2025)

    Efficient in vivo mammalian neuron editing using peptide-mediated CRISPR enzyme delivery.

    Read more

Enabling CRISPR therapies in the brain, where they weren't possible before.

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