AMD Gene Therapy Cleared for US Trial

The FDA has granted investigational new drug (IND) application clearance to Skyline Therapeutics’ gene therapy SKG0106 for the treatment of neovascular age-related macular degeneration (nAMD) and the company will soon be initiating a phase 1 clinical trial.¹

SKG0106 is an intravitreally delivered recombinant adeno-associated virus (rAAV) gene therapy that uses a novel AAV capsid and a transgene encoding anti-vascular endothelial growth factor (VEGF) protein. In preclinical studies, the therapy has demonstrated efficacy in inhibiting intraocular neovascularization with long-lasting durability and a favorable safety profile with a single injection, which may translate to preventing choroidal or retinal neovascularization and provide relief of retinal edema and vascular leakage.

Skyline presented positive preclinical data on SKG0106 at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting in May 2023, along with other gene therapy candidates from the company, including SKG0201, SKG0301, and SKG0402, which are being explored for spinal muscular atrophy, Pompe disease, and Fabry disease, respectively. The company also presented data on a novel cell line platform.

READ MORE: Silence and Replace Gene Therapy Cleared for Oculopharyngeal Muscular Dystrophy Clinical Trial

SKG0106 is being explored for treating nAMD and other ophthalmic conditions. Skyline produced good manufacturing practice (GMP)-grade SKG0106 material with its proprietary capDRIVE®AAV manufacturing platform, which is designed to integrate cell line development, plasmid and viral vector process development, GMP production, and analytical development.

Skyline announced that SKG0106 was entering clinical trials for nAMD and wet AMD in China in December 2022.³ The therapy is being evaluated in an open-label, dose escalation study at the department of ophthalmology at Peking Union Medical College Hospital. The study is primarily evaluating the safety, preliminary efficacy, immunogenicity and pharmacokinetic profile of a single intravitreal injection of SKG0106 intraocular solution in patients with nAMD.

REFERENCES

1. Skyline Therapeutics receives FDA clearance of IND for SKG0106, a novel intravitreally delivered AAV gene therapy candidate for neovascular age-related macular degeneration. News release. Skyline Therapeutics. July 3, 2023. https://prnmedia.prnewswire.com/news-releases/skyline-therapeutics-receives-fda-clearance-of-ind-for-skg0106-a-novel-intravitreally-delivered-aav-gene-therapy-candidate-for-neovascular-age-related-macular-degeneration-301868856.html
2. Skyline Therapeutics Brings Multiple Data Presentations to ASGCT 2023. News release. Skyline Therapeutics. May 17, 2023. https://www.skytx.com/news-2023_05_17
3. Skyline Therapeutics’ novel AAV gene therapy for ocular disease enters clinical trial. News release. Skyline Therapeutics. December 16, 2022. https://www.skytx.com/news-2022_12_16