How ProQR aims to use RNA therapy to target genetic forms of blindness

The rare disease biotech ProQR (NSDQ:PRQR) has a mission of reversing blindness from inherited retinal diseases. The company seems to be on track to realize that ambition, having recently announced promising data in treating a condition known as Leber congenital amaurosis type 10 in clinical studies.

One patient with the childhood form of blindness gained durable vision improvement after a single injection of ProQR’s investigational RNA therapy sepofarsen. That case study was published in Nature Medicine and featured in Drug Discovery & Development.

To learn more about the company behind sepofarsen, we spoke with its CEO, Daniel de Boer. In the following interview, De Boer shed light on ProQR’s approach to treating genetic forms of blindness, sharing how the firm can grow artificial retinas.

Drug Discovery & Development: What led you to found ProQR Therapeutics?

Daniel De Bo…

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