Skyline Therapeutics, an innovation-driven gene therapy company committed to developing unique and novel solutions for rare and severe diseases, today announced that the China National Medical Products Administration (NMPA) has approved its Investigational New Drug (IND) application for SKG0106, a one-time intravitreally delivered AAV gene therapy for the treatment of neovascular age-related macular degeneration (nAMD).
SKG0106 represents an innovative gene therapy for ocular diseases, comprising a proprietary novel recombinant adeno-associated virus (rAAV) capsid and a uniquely configured transgene genome encoding anti-VEGF protein. As a distinctively designed one-time intravitreally delivered novel AAV gene therapy, SKG0106 has demonstrated potent efficacy and a favorable safety profile in preclinical studies, including its effective and durable inhibition of neovascular growth, resulting in the reduction of vascular leakage and the mitigation of retinal edema, thus slowing the progression of nAMD.
SKG0106 received approval from the U.S. Food and Drug Administration (FDA) for Phase I/IIa clinical trial in June 2023. The global clinical trial is currently underway in the US.
About Neovascular Age-related Macular Degeneration (nAMD)
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.
Investigational SKG0106 is a proprietary novel recombinant adeno-associated viral vector containing a uniquely configured transgene with a potent inhibitory effect on intraocular neovascularization. It is an innovative one-time intravitreally delivered AAV gene therapy being developed for the treatment of serious ophthalmic conditions, including nAMD. Robust preclinical proof-of-concept studies have validated SKG0106 as a safe and effective anti-angiogenic treatment, with durable suppression of neovascular lesions achieved 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. SKG0106 has received IND approvals from the US FDA and China NMPA. GMP-grade materials used in support of SKG0106's IND filing and clinical trials were produced by Skyline Therapeutics, utilizing its in-house proprietary manufacturing platform, which integrates cell line development, plasmid and viral vector process development, analytical development, and GMP production.
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, as well as 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 are currently in clinical trials with regulatory approvals from the US FDA and China NMPA, bringing us closer to providing life-changing therapies to patients in need. Skyline Therapeutics has established a global presence for research, development, regulatory, and manufacturing in Shanghai and Hangzhou, China, and Boston, MA, USA. www.skytx.com