Aavecule® DNA platform
Gene therapies based on adeno-associated virus (AAV) are reaching market maturity; however, concerns about patient access and safety challenge further development. In contrast, synthetic, lipid nanoparticle (LNP)-based mRNA vaccines have safely transferred nucleic acid material to billions of people. Still, their effect is transient, making them unsuitable for many gene therapy applications.
Our patent-pending Aavecule® DNA Platform can bridge the gap between the two technologies, paving the way toward accessible gene therapy. Aavecule® DNA is synthetically manufactured in a cell-free reaction to mimic the single-stranded DNA genomes of AAV vectors, maintaining their unique physical and biological properties. Packaged in lipid nanoparticles, Aavecule® DNA provides the stable gene expression of AAV from a completely synthetic therapeutic. Various other applications, e.g., in AAV and CAR-T-cell manufacturing, provide exciting possibilities for continued development and collaboration.
