Re-dosable, titratable genetic medicines
We are committed to enabling generations of people with genetic diseases to live freely and fully. To do this, we are applying our technology to the development of genetic medicines for people with a wide range of genetic diseases, and working to bring these medicines to people around the world.
We are advancing a diverse portfolio of therapeutic candidates, formulated in lipid nanoparticles, for diseases of the liver.
Our ceDNA technology enables a broad portfolio of ophthalmology candidates, which can be delivered to the eye using multiple formulations.
We have also demonstrated durable in vivo expression in two additional tissues: the muscle and the central nervous system. In addition, we continue to work to unlock tractable pharmacology in several other important tissues.