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Company Adverum Biotechnologies Inc.
DescriptionOptimized adeno-associated virus (AAV) vector that utilizes Avalanche's directed evolution technology to intravitreally deliver the RS1 gene in the 83 eye
Molecular Target Retinoschisis X-linked juvenile 1 (RS1) (XLRS1)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard Indication Ophthalmic (unspecified)
Indication DetailsTreat X-linked juvenile retinoschisis
Regulatory Designation
PartnerRegeneron Pharmaceuticals Inc.

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