Eyestem RDH12 gene therapy

Eyestem RDH12 gene therapy is an investigational AAV-mediated gene augmentation therapy being developed by Eyestem Research for the treatment of Inherited Retinal Dystrophies (IRD), specifically those associated with mutations in the RDH12 gene. The therapy utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the RDH12 gene to the retina. RDH12 encodes a retinol dehydrogenase enzyme essential for the visual cycle; its deficiency leads to early-onset, severe retinal degeneration such as Leber Congenital Amaurosis type 13 (LCA13). By restoring RDH12 expression, the therapy aims to preserve photoreceptor function and prevent vision loss. It is currently in the preclinical stage of development.

Other names

AAV-mediated gene augmentation platform (RDH12)RDH12 gene therapyRDH-12 gene therapyRDH 12 gene therapy