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.
/cloudfront-us-east-2.images.arcpublishing.com/reuters/U5VZBBJ5SBJARD5TTCJHHDF6VM.jpg "FDA panel says Vertex/CRISPR to assess safety risks of gene therapy in follow-up study")
