The STELLAR clinical trial is a research study of a potential new treatment for people with ABCA4 retinopathy – the most common form of Stargardt disease. Mutations in the ABCA4 gene interfere with the metabolism of Vitamin A in the retina causing a buildup of a waste product called lipofuscin.

ACDN-01 is an investigational treatment which has not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory agency for the treatment of Stargardt disease or any other medical condition. It is a new type of drug, called an RNA exon editor. ACDN-01 contains a healthy copy of sections [exons] of ABCA4 RNA. These healthy sections replace faulty sections of the ABCA4 RNA that contain the mutations and so create healthy ABCA4 RNA in the retina. This is intended to produce normal ABCA4 protein that can then help clear the eye of toxic waste products.

Before being treated in the study participants will be tested to confirm that ACDN-01 would edit the specific mutations present in their ABCA4 RNA. The treatment is administered by sub-retinal injection to a central part of the retina called the macula. The company believes that one treatment may last for many years, perhaps even a person’s lifetime.