Expanding the reach of gene therapy
We are developing a new approach to gene therapy that will enable us to treat more people, living with more diseases, in more places.
Our goal is to bring the benefits of these therapies to people around the world, including children, so they can move past their genetic diseases and live long, full lives.
Our targeted lipid nanoparticle (LNP) technology enables delivery of ceDNA to hepatocytes, resulting in durable high-level expression of the transgene. Because we do not use a viral construct for delivery, we should be able to treat patients who could not receive or did not respond to AAV-based therapy, and to re-dose as needed.
By combining a novel construct with targeted delivery, our platform enables us to expand the reach of gene therapy across multiple tissue types and disease areas. We are developing a deep initial portfolio of liver-directed therapies with the potential to move into other tissues, including the eye, muscle and brain.
Our therapies have the potential to transcend the limitations of current approaches by enabling repeat dosing, delivering larger transgenes, and by scaling to treat larger diseases and global populations.
Groundbreaking science. Trailblazing team.
The science behind our revolutionary gene therapies