RNA therapy should prevent further deterioration of vision in patients with Stargardt disease in the future. For the treatment, which is currently still in the developmental phase, the Radboudumc Holding has founded the company Astherna together with professors Rob Collin and Carel Hoyng. Astherna is the first biotech company initiated from the Radboud university medical center in the field of eye diseases. The goal is to offer a unique treatment for, often still young, patients with Stargardt disease.
Stargardt disease is an inherited retinal disease that affects the central part of the retina, the macula. The vision of patients with this disease slowly deteriorates and can eventually cause severe low vision. One in ten thousand people suffer from it, and there is currently no treatment available. The disease is caused by mutations (errors) in the ABCA4 gene. It is the most common form of macular degeneration in adolescents and young adults; the disease often begins in childhood.
RNA therapy that stops vision from deteriorating
RNA therapy can help prevent further vision deterioration in patients with Stargardt disease. In RNA therapy, so-called antisense oligonucleotides (AONs), small synthetic RNA molecules, are used to restore the production of proteins. Promising results have already been achieved with AONs in the hereditary eye disease Leber congenital amaurosis. A huge advantage of AONs over other therapeutic strategies is that it addresses the root cause of the problem, namely correcting incorrectly synthesized RNA. In addition, AONs are relatively small molecules that can easily reach the right cells in the retina.
The therapy was developed by the research group of Rob Collin, professor of Molecular Therapy for Inherited Retinal Diseases at the Radboud university medical center. "For several mutations in the ABCA4 gene, we have already developed AONs that can correct aberrant RNA, at least in cultured cells. Now it's time for the next step," says Collin. The method entails that patients will need to be treated on a regular basis, two to four times a year. He expects to be able to help tens of thousands of people with Stargardt disease worldwide.
Patients who know what to expect at an early age
When the treatment will become available to patients is not yet known. More clinical trials are needed. Astherna, spin-off of the Radboudumc and the first biotech company founded from the Radboud university medical center for eye diseases, will contribute to this. Professor of Ophthalmology Carel Hoyng: "I am delighted and proud that Astherna has been founded. We can now really mean something for the many patients with Stargardt disease. In the Radboudumc we see the most people with this condition, so we are able to make our research directly applicable to patients. I see young patients in my outpatient clinic who know that they are going to lose their sight at some point. It has a huge impact on the life choices they need to make: what education can they do, what kind of job lies ahead for them? The earlier we can help them, the less sight they will lose. These are the people we do it for."