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Company Horama S.A.S.
DescriptionSerotype 5 recombinant adeno-associated virus (rAAV) delivering the phosphodiesterase-6B (PDE-6B) gene
Molecular Target Phosphodiesterase-6B (PDE-6B)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationRetinitis
Indication DetailsTreat PDE-6B-mediated retinitis pigmentosa
Regulatory Designation EU - Orphan Drug (Treat PDE-6B-mediated retinitis pigmentosa)

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