This gene therapy is designed to be a one-time intravitreally therapy that has demonstrated potent efficacy and a favorable safety profile in preclinical studies.
The China National Medical Products Administration (NMPA) has approved Skyline Therapeutics’ Investigational New Drug (IND) application for SKG0106. This new treatment compound is a one-time AAV gene therapy for the treatment of neovascular age-related macular degeneration (nAMD) delivered intravitreally.1
According to the company’s press release, SKG0106 is a gene therapy treatment comprised of a proprietary novel recombinant adeno-associated virus (rAAV) capsid and a uniquely configured transgene genome encoding anti-VEGF protein. It is designed to be a one-time intravitreally therapy that has demonstrated potent efficacy and a favorable safety profile in preclinical studies. These initial studies have shown that SKG0106 is effective and shows durable inhibition of neovascular growth, thus reducing of vascular leakage and the mitigation of retinal edema, slowing the progression of nAMD.1
Along with this approval, SKG0106 previously received approval from the FDA in the United States for Phase I/IIa clinical trial in June 2023. The global clinical trial is currently underway in the US.1
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.1