A new class of gene therapy
We are committed to enabling generations of people with genetic diseases to live long, full lives.
We are advancing a broad and expansive portfolio, including eight programs for both rare and prevalent diseases of the liver and retina.
We are focused on diseases with significant unmet need for which our non-viral gene therapy platform may substantially improve clinical efficacy relative to current gene therapy approaches. We are prioritizing rare monogenic diseases of the liver and retina, including phenylketonuria (PKU), hemophilia A, Leber’s congenital amaurosis 10 (LCA10) and Stargardt disease.
We plan to expand our portfolio by pursuing additional programs in rare and prevalent diseases of the liver and retina, as well as in skeletal muscle, the central nervous system and oncology by developing discrete ctLNP delivery systems, each engineered to reach a different tissue.
Our liver disease programs include:
Antibody gene therapy
Our retinal disease programs includes: