Spark Therapeutics

Genable Technologies Limited is a bio-pharmaceutical company based in Dublin, Ireland, specializing in the development of gene therapies for autosomal dominant genetic diseases. The company focuses on innovative treatments for inherited disorders, notably its RhoNova product aimed at addressing rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa, a progressive form of inherited blindness caused by mutations in the RHO gene. Genable employs a unique approach that combines suppression and replacement of faulty gene copies using adeno-associated virus (AAV) vectors to deliver RNA interference (RNAi) molecules. This method allows the company to tackle the variability of mutations in dominant diseases without the need to target specific mutations. Genable Technologies was incorporated in 1998 and operates as a subsidiary of Spark Therapeutics, Inc. The company's technology is backed by a comprehensive portfolio of patents in the United States, Europe, and globally.