FDA Approves Skyline Therapeutics' Novel Gene Therapy for Macular Degeneration
Skyline Therapeutics, a company focused on developing solutions for rare and severe diseases, has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application. The clearance allows for the initiation of Phase I/IIa clinical trials of SKG0106, a onetime intravitreally delivered AAV gene therapy for neovascular age-related macular degeneration (nAMD).
SKG0106 is a unique recombinant adeno-associated virus (AAV) gene therapy designed to treat ocular diseases. It features a proprietary AAV capsid and a specially designed transgene genome encoding an anti-VEGF protein. Preclinical studies have shown that SKG0106 effectively inhibits intraocular neovascularization, with a favorable safety profile and long-lasting durability following a single intravitreal administration.
To support the IND application, Skyline Therapeutics produced GMP-grade material for SKG0106 using its proprietary manufacturing platform. This platform integrates various processes such as cell line development, plasmid and viral vector process development, analytical development, and GMP production, ensuring consistent support for the company's preclinical and clinical programs.
SKG0106 is a novel recombinant adeno-associated viral vector containing a uniquely configured transgene that exhibits potent inhibition of intraocular neovascularization. It represents an innovative one-time intravitreally delivered AAV gene therapy under development for serious ophthalmic conditions, including nAMD. Robust preclinical studies have demonstrated the safety and efficacy of SKG0106 as an anti-angiogenic treatment. A low-dose single intravitreal injection provides durable suppression of neovascular lesions, preventing choroidal or retinal neovascularization and reducing retinal edema and vascular leakage.
