Pharma Focus Asia

China Nmpa Approves Skyline Therapeutics SKG0106 for Neovascular Age-related Macular Degeneration Gene Therapy

Friday, October 20, 2023

Skyline Therapeutics, an innovative gene therapy company focused on developing novel solutions for rare and severe diseases, has announced that the China National Medical Products Administration (NMPA) has granted approval for its Investigational New Drug (IND) application for SKG0106. This unique therapy is a one-time intravitreally delivered AAV gene therapy designed for the treatment of neovascular age-related macular degeneration (nAMD).

SKG0106 is an inventive gene therapy for ocular diseases, featuring a distinctive recombinant adeno-associated virus (rAAV) capsid and a specially designed transgene genome encoding an anti-VEGF protein. As an exceptional one-time intravitreal AAV gene therapy, SKG0106 has shown significant efficacy and a favorable safety profile in preclinical studies. It effectively and durably inhibits neovascular growth, leading to reduced vascular leakage, alleviation of retinal edema, and a slowdown in the progression of nAMD.

SKG0106 received approval from the U.S. Food and Drug Administration (FDA) for a Phase I/IIa clinical trial in June 2023. The global clinical trial is currently underway in the United States.

Age-related macular degeneration (AMD) is a progressive condition that can severely damage central vision and is a leading cause of irreversible blindness in the elderly. Neovascular age-related macular degeneration (nAMD), characterized by the development of choroidal neovascularization (CNV), bleeding, and exudation, accounts for up to 90% of blindness in AMD patients. Current treatment for nAMD involves frequent ocular injections of anti-VEGF drugs, which can be burdensome for patients and lead to 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 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, achieving durable suppression of neovascular lesions at a low-dose single intravitreal injection. This results in the prevention of choroidal or retinal neovascularization, alleviation of retinal edema, and the reduction of vascular leakage. SKG0106 has received IND approvals from the US FDA and China NMPA. GMP-grade materials used to support 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.



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