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.

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.

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.