July 2023, Skyline Therapeutics announced that the U.S. FDA has cleared its IND application for Phase I/IIa clinical trial of SKG0106, a one-time intravitreally delivered AAV gene therapy for the treatment of neovascular age-related macular degeneration (nAMD). Global Phase I clinical trial will be initiated soon.
Age-related macular degeneration (AMD) is a progressive disease that can severely impair central vision and is a leading cause of irreversible blindness in the elderly. Neovascular age-related macular degeneration (nAMD), characterized by the formation of choroidal neovascularization (CNV), bleeding, and exudation, accounts for up to 90% of blindness in AMD patients. Currently, clinical treatment of nAMD involves frequent ocular injections of anti-VEGF drugs, placing a significant burden on patients and often resulting in compliance challenges. Innovative gene therapies, such as the one-time intravitreally-delivered anti-VEGF AAV gene therapy with potentially enduring anti-angiogenic effects, offer a promising new approach to address these challenges.
SKG0106 is a distinctively designed recombinant adeno-associated virus (rAAV)-mediated gene therapy for ocular disease. It encompasses a proprietary novel AAV capsid and a uniquely configured transgene genome encoding anti-VEGF protein. In comprehensive preclinical studies, SKG0106 has demonstrated potent efficacy of inhibition of intraocular neovascularization, a favorable safety profile, and long-lasting durability following a single intravitreal administration.
About Skyline Therapeutics
Skyline Therapeutics is an innovation-driven clinical-stage gene therapy company dedicated to developing unique and novel solutions to address unmet needs in rare and severe diseases. Our cutting-edge adeno-associated virus (AAV) platform comprises multiple proprietary technologies for capsid discovery, transgene design and vector engineering. Combining our AAV expertise with robust in-house process and analytical development, and GMP manufacturing for clinical-grade plasmid and viral vector production, we are advancing a diverse pipeline of gene therapies for ocular, neurological, cardiovascular and metabolic disorders. Our lead programs, with significant therapeutic potential as validated by rigorous preclinical studies, are now in first-in-human clinical trials, bringing us closer to providing life-changing therapies to patients in need. Skyline Therapeutics has established global presence for research, development, regulatory, and manufacturing in Shanghai and Hangzhou, China, and Boston, MA, USA.