The rAAVen way

Methodology

rAAVen Therapeutics utilizes a unique rational design and DNA barcoding combination for screening of AAV capsids for the next generation of gene therapies with superior efficacy over existing AAV vectors. Our rAAptr (rational AAV peptide research) platform combines DNA barcoding strategy and is paired with next generation sequencing as well as single cell sequencing to establish the viral variants with highest efficacy for the targeted cell population.
Conserved mechanism of infection across species
At rAAVen, we believe that nature has already evolved highly efficient protein-protein interactions which are conserved to a high degree across mammalian species. rAAVen leverages these interactions for viral cell entry of designed AAV capsids with high efficiency and specificity for targeted tissue types and cell populations. Unlike directed evolution, our unique rAAptr platform allows for fast and cheap single screens of AAV capsids with high cell type specificity.
Libraries for future clinical trials
rAAVen Therapeutics also offers screens of large random peptide libraries if requested by our clients. We offer screens in a variety of animal models ranging from rodents to non-human primates in order to offer a product with superior translational value for future clinical trials. Please contact us for further information about custom AAV capsid modification libraries.
Extended screening
To further increase expression selectivity, rAAVen is also pleased to offer screens of promotor/enhancer elements for the screened AAV products using our DNA barcoding platform. Screening can be selected for specific tissues as well as specific cell populations to determine the best possible combination of AAV capsid and promotor/enhancers for our client’s needs.

Indepth methodology and overview of our platform

1.

AAV Peptide display, combined with unique DNA barcoding and next generation sequencing allows for easy screening of AAV libraries containing millions of rationally designed, targeted AAV variants in a single screen.

2.

Choose your target, tissue type, cell population or application. rAAVen designs a targeted, rationally designed AAV peptide display library which can be combined by a generated library of promotor & enhancer elements for increased cell and tissue specific expression.

3.

rAAVen offers screening of the AAV peptide library in animal models ranging from rodents to non-human primates, ensuring that the delivered product translates within mammalian species and are prime candidates for clinical trials.

rAAptr platform workflow

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