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Under the agreement, OBiO leverages its capabilities of process development, large-scale cGMP commercial production and global business development for Refreshgene's RRG-001 gene therapy product.
HG004 is a gene replacement therapy drug which uses the recombinant non-adeno-associated virus serotype 2 vector to deliver a functional human RPE65 gene to the retina to restore, treat, and prevent blindness of children and adults with RPE65 mutation-associated IRDs.
HG004 is an ophthalmic injection intended for the treatment of RPE65 gene mutation-related retinopathy. Based on direct preclinical comparative studies of HG004 and AAV2 in retinal function, HG004 is at least 10 times more efficient at transducing retinal pigment epithelium.