Healthcare professionals Inherited retinal disease resources

Ophthalmologist doing eye measurements

Our mission is to help people living with rare genetic diseases that currently have no treatment options. We focus on developing intravitreal RNA therapies that treat the underlying cause of inherited retinal diseases (IRDs).


To advance research and ensure the best care for patients, we believe having close partnerships with healthcare professionals (HCPs) is key. We therefore welcome you to reach out to us with any query or if you are interested in trial participation email us directly at

RNA therapy – a fundamentally new approach to IRDs

For the vast majority of the more than 2 million people living with an IRD, there is no treatment available. We are working hard to change that. We are developing several RNA therapies that could potentially stop disease progression or reverse vision loss in currently untreatable diseases.

RNA illustration

What is RNA therapy?

RNA therapy is different from gene therapy and gene editing because it doesn't alter DNA. RNA therapy aims to repair RNA, which is a product of DNA, therefore any changes made to the RNA are reversible. The routine intravitreal (IVT) injection of RNA therapies and the potential to treat the whole retina, make it an innovative and accessible approach with a manageable benefit/risk profile.

Learn about RNA therapy