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Company ProQR Therapeutics N.V.
DescriptionRNA-based therapeutic that repairs centrosomal protein 290kDa (CEP290) mRNA
Molecular Target Centrosomal protein 290kDa (CEP290)
Mechanism of Action 
Therapeutic Modality 
Latest Stage of DevelopmentPreclinical
Standard IndicationBlindness
Indication DetailsTreat Leber's congenital amaurosis (LCA); Treat leber's congenital amaurosis (LCA) Type 10
Regulatory Designation U.S. - Orphan Drug (Treat leber's congenital amaurosis (LCA) Type 10);
EU - Orphan Drug (Treat leber's congenital amaurosis (LCA) Type 10)

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