ASTHERNA is a RNA therapy company developing treatments for retinal diseases. It is our ambition to prevent individuals from getting blind and have an impact on patients’ lives. We have developed a unique therapeutic approach to stop the progression of inherited retinal diseases, collectively affecting hundreds of thousands individuals worldwide. We then also apply this technology to other ocular indications, to help even a larger number of patients worldwide to maintain their vision.
RNA is the intermediate product between DNA, the building blocks of life, and protein, the functional entities within each cell of our body. Several DNA defects, also known as genetic mutations, cause wrong processing of RNA, thereby leading to disrupted protein synthesis. RNA therapy is a strategy that acts at the RNA level, by converting wrong RNA into correct RNA. This ultimately results in the restoration of protein synthesis and cellular function.
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.