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RNA therapy
to save vision

Loss of vision in Stargardt disease

Our mission

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.

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RNA therapy

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

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.

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