Our proprietary non-viral gene therapy platform
We are developing a new class of gene therapy that may enable us to treat more people, living with more diseases around the world.
Our proprietary non-viral gene therapy platform is comprised of three essential components:
- our high-capacity DNA construct called closed-ended DNA, or ceDNA, which can accommodate large or multiple genes, as well as native regulatory elements;
- our cell-targeted lipid nanoparticle delivery system, or ctLNP, which enables highly specific delivery of ceDNA to a range of tissues;
- our established, scalable capsid-free manufacturing process, which uses a cost-effective biologics infrastructure.
By creating this new class of gene therapies, we believe we can reach previously untreatable or under-treated patients, and address new indications, including those with large patient populations, thereby unlocking the full potential of genetic medicine.
Our proprietary non-viral gene therapy platform — comprised of our ceDNA construct, our ctLNP delivery system and our established, scalable capsid-free manufacturing process — is designed to provide the following benefits:
Our ctLNP delivery system has been designed to avoid stimulating an antibody response in patients, thereby enabling redosing, which may allow for individualized patient titration to reach desired efficacy levels as well as extension of efficacy as needed throughout a patient’s life.
We believe that the properties of our therapies may allow us to initiate treatment in childhood, enabling a greater therapeutic benefit throughout the patient’s life. Our therapies may also be used to treat patients who are not able to receive current gene therapy treatments due to pre-existing immunity, as well as patients who did not see desired efficacy after being treated with other current gene therapies.
Broad patient reach
We believe ceDNA can deliver a significant majority of the human coding sequences known to be relevant for the treatment of diseases that result from mutations in a single gene or in multiple genes, providing the potential to address many diseases, including prevalent diseases and diseases requiring more than one type of genetic correction. This capacity can also accommodate native or engineered regulatory elements, potentially enabling a powerful new dimension of gene therapy that responds to the body’s own signals.
Delivery of large genetic payloads
Our novel ctLNP delivery system has been engineered to use biological ligands to reach receptors in a specific tissue.
Targeted, multi-tissue delivery
Groundbreaking science. Trailblazing team.
The people behind our revolutionary gene therapies