Refining delivery to transform treatment

The therapy isn’t just the gene — it's also the vehicle

Our goal is to revolutionize AAV (adeno-associated virus) gene therapy through capsid engineering and optimized routes of administration

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Pursuing Efficacy

Striving to increase substructure and
cell-specific targeting

Prioritizing Well-Being

Aiming to limit or avoid on-tissue and off-tissue toxicity

Lowering Dose Requirements

Optimizing delivery and specificity to enable targeted treatments at lower doses

ADDRESSING UNMET NEED

19% of people globally are affected by monogenic diseases, yet many go untreated due to the absence of effective therapies. Our innovative gene therapy approaches aim to revolutionize treatment and bring hope to millions of people worldwide.

19% of people globally have monogenic disease

* Sourced from the National Institutes of Health
PROBLEM / Solution

We aim to solve a leading problem with AAV gene therapy – high dose requirements

  • High AAV doses lead to toxicities, manufacturing challenges, and high cost of goods
  • We identify novel AAV capsid variants matched to optimized routes of administration to target the right cell types in the right tissues, potentially leading to lower doses, volumes and costs
Our procedures Tissue optimized
Our procedure times 2–3 hours
Our doses 1/100 to 1/10,000 of the competition
Our capsids Targeted and cell-specific
Our off-tissue distribution Minimal to none
Our cost of goods <1/10 of the competition
Our Approach

Deploying advanced computational methods and unbiased screening

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ABOUT US

Translational medicine expertise that spans from pipette to patient

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PIPELINE

We are mining a proprietary database of unique delivery zip codes to enable targeting of a broad range of central nervous system (CNS) and peripheral diseases

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NEWS & PUBLICATIONS

Explore our latest news releases, publications, and scientific updates

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