SHANGHAI, May 3, 2023 /PRNewswire/ — Skyline Therapeutics, an innovation-driven gene therapy company dedicated to developing unique and novel solutions to address unmet needs in rare and severe diseases, today announced multiple data presentations at the American Society of Gene and Cell Therapy (ASGCT) 26^th Annual Meeting in Los Angeles, CA, May 16-20, 2023.

Among the exciting findings from several innovative AAV gene therapy programs that the Skyline team will present at ASGCT are SKG0106, a novel recombinant adeno-associated virus (rAAV) vector developed for the treatment of neovascular age-related macular degeneration (nAMD) via intravitreal (IVT) administration, and SKG0201, an innovative next generation SMN1 gene therapy for spinal muscular atrophy (SMA).

SKG0106 is a proprietary AAV vector carrying a transgene genome encoding a novel anti-VEGF protein. Upon delivery, SKG0106 effectively and specifically suppresses the action of human VEGF, a key factor contributing to the pathological angiogenesis in the eyes of nAMD patients. Comprehensive pharmacological studies were conducted to thoroughly evaluate the efficacy of SKG0106 using a DL-α-AAA induced rabbit model of chronic retinal neovascularization (RNV) and a laser photocoagulation induced non-human primate (NHP) model of choroidal neovascularization (CNV). SKG0106 was administered via a single IVT injection.

Data highlights

• Single treatment of SKG0106 via intravitreal delivery effectively reduced fluorescein leakage area and grade 4 lesion percentage in a dose dependent manner; strong efficacy was observed at low dose level
• SKG0106 showed complete and durable inhibition of fluorescein leakage and significant reduction in subretinal hyperreflective material thickness in the 26-week long term study in NHP, suggesting the strong and durable efficacy of the vector in repressing the activity of VEGF
• SKG0106 was well tolerated, and no significant adverse effect observed in ocular nor peripheral tissues

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