ASTHERNA is a RNA therapy company developing treatments for inherited retinal diseases, collectively affecting hundreds of thousands individuals worldwide. We have developed a unique therapeutic approach to stop the progression of these diseases. It is our ambition to prevent individuals from getting blind and have an impact on patients’ lives.
Stargardt disease is the most common retinal dystrophy caused by mutations in a single gene. Currently, there is no treatment available. A patient with Stargardt disease experiences a progressive loss of central vision. Eventually it leads to severe visual impairment or blindness. Stargardt disease is caused by a defect in the ABCA4 gene that disturbs cell function in the retina, ultimately leading to damage to the retina.