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GT038

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Collapse Summary General Information
Company Genable Technologies Ltd.
DescriptionAdeno-associated viral (AAV) vector encoding an RNAi targeting rhodopsin (Rho; Opn2) in combination with an AAV vector containing a Rho gene
Molecular Target Not available
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard Indication Ophthalmic (unspecified)
Indication DetailsTreat retinitis pigmentosa (RP); Treat rhodopsin-linked autosomal dominant retinitis pigmentosa (adRP)
Regulatory Designation

U.S. - Orphan Drug (Treat retinitis pigmentosa (RP));
EU - Orphan Drug (Treat rhodopsin-linked autosomal dominant retinitis pigmentosa (adRP))

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