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RhoNova (GT038)

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Company Genable Technologies Ltd.
DescriptionAdeno-associated virus (AAV) vector containing DNA for an RNAi molecule 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 IndicationRetinitis
Indication DetailsTreat retinitis pigmentosa (RP); Treat rhodopsin-linked autosomal dominant retinitis pigmentosa (adRP)
Regulatory Designation U.S. - Orphan Drug (Treat retinitis pigmentosa (RP));
U.S. - Orphan Drug (Treat rhodopsin-linked autosomal dominant retinitis pigmentosa (adRP))
PartnerSpark Therapeutics Inc.

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