SHANGHAI, China, July 3, 2023 – Skyline Therapeutics, an innovation-driven gene therapy company committed to developing unique and novel solutions for rare and severe diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (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.
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.
In support of the IND application, SKG0106 GMP-grade material was produced utilizing Skyline Therapeutics' proprietary manufacturing platform that integrates cell line development, plasmid and viral vector process development, analytical development and GMP production, ensuring unwavering support for the Company’s preclinical and clinical programs in its pipeline.
Investigational SKG0106 is a proprietary novel recombinant adeno-associated viral vector containing a uniquely configured transgene with potent inhibition effect on intraocular neovascularization, an innovative one-time intravitreally delivered AAV gene therapy that is being developed for the treatment of serious ophthalmic conditions including nAMD. Robust preclinical proof-of-concept studies validated SKG0106 as a safe and effective anti-angiogenic treatment with durable suppression of neovascular lesions at a low-dose single intravitreal injection, which results in the prevention of choroidal or retinal neovascularization and relief of retinal edema and vascular leakage.
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