A New Era for Gene Therapy
In creating our GeneWave platform, we sought to do what no other gene therapy platform has yet accomplished: to deliver titratable, re-dosable genetic medicines with drug-like properties to patients with a wide range of genetic diseases.
Our ceDNA technology has a unique combination of properties that derive from its ability to translocate from the cytoplasm to the nucleus without a viral capsid. Once in the nucleus, ceDNA forms stable, non-integrating episomes that result in durable, high levels of gene expression. This capsid-free technology allows us to deliver larger transgenes than traditional AAV vectors and enables repeated dosing. We have demonstrated durable expression for ceDNA in multiple tissues including liver, eye, brain, and muscle.
GeneWave is our therapeutic platform that uses state-of-the-art lipid nanoparticles to deliver ceDNA to the liver via systemic administration. This allows GeneWave to deliver genes to correct intrinsic liver diseases, and to address systemic diseases by transforming the liver into a biofactory to express proteins with systemic effects.
Sustainable Genetic Medicines
ceDNA is manufactured in a serum-free eukaryotic system without the use of animal-derived products. Our manufacturing platform is scalable, allowing us to address rare diseases and more prevalent chronic diseases.