October 2023 , Skyline Therapeutics announced that the clinical trial application (IND) of SKG0106 is approved by the China National Medical Products Administration (NMPA) for the treatment of neovascular age-related macular degeneration (nAMD).
SKG0106 was approved by the U.S. Food and Drug Administration (FDA) earlier this year to conduct a global Phase I/IIa clinical trial for the treatment of neovascular age-related macular degeneration (nAMD). The global clinical trial has been launched.
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 Age-related macular degeneration (AMD)
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.
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. www.skytx.com