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XLRP

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Company Applied Genetic Technologies Corp.
DescriptionAdeno-associated virus (AAV) vector expressing the retinitis pigmentosa GTPase regulator (RPGR) gene
Molecular Target Retinitis pigmentosa GTPase regulator (RPGR)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationRetinitis
Indication DetailsTreat X-linked retinitis pigmentosa; Treat X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene
Regulatory Designation EU - Orphan Drug (Treat X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene)
PartnerBiogen Inc.

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