The US Food and Drug Administration (FDA) has granted clearance for Skyline Therapeutics’ investigational new drug (IND) application for Phase I/IIa clinical trial of gene therapy SKG0106 to treat neovascular age-related macular degeneration (nAMD).
A single-time, recombinant adeno-associated virus (rAAV)-mediated gene therapy for ocular disease, SKG0106 is administered intravitreally.
The global Phase I trial will begin shortly.
SKG0106 consists of a new AAV capsid along with a distinctively arranged transgene genome that encodes anti-VEGF protein.
Preclinical study data showed that the gene therapy possesses robust efficacy in hindering intraocular neovascularisation, an encouraging safety profile and continued durability after a single intravitreal dosing.
SKG0106 GMP-grade material was manufactured leveraging the company’s platform that merges plasmid and viral vector process development, cell line development, analytical development and GMP manufacturing. This approach guarantees continued support for its preclinical and clinical programmes in the pipeline.
In December last year, SKG0106 entered clinical trials in humans for neovascular or wet age-related macular degeneration indications.
Skyline own patents of SKG0106 including but not limited to AAV capsid and the design of transgene/vector in various regions globally.