Skyline Therapeutics, a Shanghai-based cell and gene therapy (CGT) developer, has announced encouraging preclinical study results for its AAV gene therapies, SKG0106 and SKG0201, targeting neovascular age-related macular degeneration (nAMD) and spinal muscular atrophy (SMA), respectively.
SKG0106: A Novel AAV Therapy for nAMD
SKG0106 is a self-developed novel AAV vector carrying a transgenic sequence encoding a unique anti-VEGF protein. Administered through the vitreous cavity, it efficiently transduces retinal cells to express anti-VEG0F protein, which has a specific inhibitory effect on human VEGF, a key factor in pathological angiogenesis and leakage in nAMD patients. The preclinical data indicates that SKG0106 has shown significant preliminary efficacy in rabbit chronic retinal neovascularization (RNV) models and non-human primate choroidal neovascularization (CNV) models, and has demonstrated good safety in toxicological studies, with no observed side effects.
SKG0201: Gene Replacement Therapy for SMA
SKG0201 is an AAV-mediated gene replacement therapy that includes human SMN1 cDNA regulated and comprehensively optimized by a unique promoter. Preclinical data suggest that a single intravenous injection of SKG0201 can quickly and persistently restore functional SMN protein expression, significantly increase body weight, and effectively prolong the lifespan of SMA disease model mice at low dose levels. In terms of safety, SKG0201 shows significant efficacy and no hepatotoxicity at low doses, indicating its potential for low effective dose application in clinical practice and its promise as a new generation of AAV gene therapy for SMA.-Fineline Info & Tech
