ATIVAA® - Next Gene Therapeutics™, a start-up initiative from Bielefeld University, Germany, aims to accelerate the development of safe, effective, and affordable treatments for patients with genetic diseases worldwide. We strive to revolutionize gene therapy by bridging the gap between viral and non-viral gene transfer through the power of our innovative Aavecule® DNA platform technology.
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.
Aavecule® DNA acts as a starting material for high-titer, high-quality AAV manufacturing. Unlike plasmid DNA, Aavecule® DNA contains no bacterial sequences, resulting in the highest genetic purity AAV.
Aavecule® DNA can be packaged directly in lipid nanoparticles for a stable, highly scalable gene transfer with AAV-like stability of gene expression.
In most in vivo applications, like CAR-T-cell manufacturing Aavecule® DNA combined with chemical or physical transfection techniques can readily replace expensive AAV preparations for cell line engineering.
Get in touch now to learn about the unique advantages of our approach and the capabilities at our research facility in Bielefeld, Germany. Send us an email at email@example.com.