Skyline Therapeutics, a biopharmaceutical company based in China, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its proprietary adeno-associated virus (AAV) gene therapy, SKG1108, for the treatment of retinitis pigmentosa (RP). This innovative therapy utilizes a novel AAV vector, including a groundbreaking capsid AAV.0106, designed for intravitreal injection to deliver genes encoding a unique protein directly to the retina. The gene therapy’s cutting-edge design ensures that specific gene elements regulate the expression of photosensitive proteins in the retina’s target cells, aiming to create new photoreceptors that enhance and restore visual function in affected patients.
Retinitis pigmentosa is a genetically diverse inherited retinal disease (IRD) with over a hundred known gene mutations. The disease typically leads to legal blindness by the age of 40, characterized by an initial degeneration of rod cells, followed by a progressive loss of cone cells, culminating in the near-total apoptosis of photoreceptor cells and irreversible visual impairment. Currently, no treatment exists that can prevent or reverse photoreceptor cell degeneration. Existing gene-specific therapies are limited to a small subset of mutations and are not broadly applicable. SKG1108’s innovative mechanism of action is not restricted to specific gene mutations, offering a potential breakthrough for the treatment of severe RP by generating new photoreceptors to compensate for the loss of rod and cone cells, thereby improving and restoring visual function. – Flcube.com
