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XM004 is an investigational gene therapy candidate developed by Hefei Xingmou Biotechnology (also known as StarryGene) for the treatment of severe retinal diseases and blindness. Currently in the preclinical and activity validation stage, XM004 utilizes an adeno-associated virus (AAV) vector to deliver genetic payloads to the retina. The program is specifically targeted at advanced stages of retinal degeneration where significant loss of photoreceptor cells has occurred, potentially employing optogenetic techniques to restore visual perception by making remaining retinal neurons light-sensitive. As part of StarryGene's broader ophthalmic pipeline, XM004 represents a potential single-administration treatment aimed at addressing unmet needs in patients with profound vision loss.
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