cceAAV production

A conventional recombinant single-stranded AAV (ssAAV) and self-complementary AAV (scAAV) package the genome with wild-type ITRs or mutated ITRs flanking an expression cassette encoding a gene of interest. Upon uncoating in target cells, scAAV can rapidly form an intra-molecular double-stranded DNA, facilitating high-level transgene expression.

In our proprietary cceAAV technology, a plasmid with hairpin telomeres forms a hairpin loop and is packaged into the AAV virus vector. cceAAV allows for an increased packaging capacity compared to scAAV vectors due to a higher formation rate of hairpin loops in vitro and fewer nucleotides than the mutated ITR. Analysis on Alkaline Agarose gel indicates that cceAAV viruses have a lower fraction of unexpected partial genomes compared to scAAV, suggesting potential enhancement in potency and reduced safety concerns.”