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Company Horama S.A.S.
DescriptionRecombinant adeno-associated virus (rAAV) delivering the rab escort protein 1 (CHM; REP1) gene
Molecular Target Rab escort protein 1 (CHM) (REP1)
Mechanism of Action 
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationBlindness
Indication DetailsTreat choroideremia
Regulatory Designation

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