Gene therapy company Skyline Therapeutics has received Investigational New Drug application clearance from the US FDA for its gene therapy candidate for the treatment of neovascular age-related macular degeneration (nAMD). The company is now preparing to initiate a global Phase I clinical trial for SKG0106 – a one-time intravitreally delivered adeno-associated virus (AAV) gene therapy.
SKG0106 is an investigational gene therapy that uses a recombinant adeno-associated viral vector with a unique transgene configuration. It has demonstrated potent inhibition of intraocular neovascularization, leading to the prevention of neovascular lesions and relief of retinal edema and vascular leakage. The therapy is administered through a one-time intravitreal injection.
SKG0106 features a unique AAV capsid and a specially designed transgene genome encoding an anti-VEGF protein. In preclinical studies, the gene therapy demonstrated strong efficacy in inhibiting intraocular neovascularization, a positive safety profile, and long-lasting durability after a single intravitreal administration.
To support the IND application, Skyline produced GMP-grade material of SKG0106 using its proprietary manufacturing platform. The platform integrates various processes such as cell line development, plasmid and viral vector process development, analytical development, and GMP production.
Skyline says it is advancing a diverse pipeline of gene therapies for ocular, neurological, cardiovascular, and metabolic disorders. The company’s AAV platform combines proprietary technologies for capsid discovery, transgene design, and vector engineering.
