Skyline Therapeutics’ Novel AAV Gene Therapy for Ocular Disease Enters Clinical Trial
December 16, 2022

Shanghai, China, Dec. 16, 2022 – Skyline Therapeutics today announced that the Company’s innovative ocular AAV gene therapy SKG0106 is entering human clinical trials, for the treatment of neovascular or wet age-related macular degeneration (nAMD or wAMD).


SKG0106 is a distinctively designed recombinant adeno-associated virus (rAAV) mediated gene therapy for ocular disease, containing a proprietary novel capsid and an uniquely designed transgene for neovascularization inhibition. Through transduction of the retinal cells following a one-time intravitreal delivery, the SKG0106 vector expresses a lasting therapeutic agent that blocks the growth of neovascular lesions in the retina and relieves retinal edema and vascular leakage, thus slowing down the progression of nAMD. The company owns patents of SKG0106 including but not limited to AAV capsid and transgene/vector design in multiple regions & countries.


In the comprehensive preclinical studies, SKG0106 showed excellent target tissue tropism, penetration and biodistribution, as well as potent and durable choroidal neovascularization (CNV) lesion suppression, demonstrating favorable safety and efficacy profile.


An open label, does escalation study is being initiated in the Department of Ophthalmology, Peking Union Medical College Hospital (PUMCH). The study is to evaluate the safety, preliminary efficacy, immunogenicity and pharmacokinetic profile of a single intravitreal injection of SKG0106 intraocular solution in patients with nAMD.


The current clinical standard of care of nAMD involves long-term anti-VEGF treatment, which requires frequent intraocular injections, resulting in a large burden on patients and poor compliance. Innovative gene therapy with favorable safety and long-lasting anti-angiogenic effects through a single intravitreal administration has the potential to bring promising new paradigm of treatment for nAMD patients.


About SKG0106

Investigational SKG0106 is a proprietary novel recombinant adeno-associated viral vector containing a uniquely configured transgene with potent inhibition effect on neovascularization, an innovative one-time intravitreally delivered AAV gene therapy that is being developed for the treatment of serious ophthalmic conditions including nAMD. Robust preclinical proof-of-concept studies validated SKG0106 as a safe and effective anti-angiogenic treatment with durable suppression of neovascular lesions 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.


About neovascular age-related macular degeneration (nAMD)

Age-related macular degeneration (AMD) is a progressive disease that leads to severe impairment of central vision. It is a leading cause of irreversible blindness in the elderly population. nAMD is characterized by CNV formation, or growth of abnormal vessels most often from the choroid, causing acute vision loss through leakage, edema and bleeding. As high as 90% of patients blinded by AMD are those with nAMD.


About Skyline Therapeutics

Skyline Therapeutics is a fully integrated gene therapy company dedicated to developing novel gene therapies to address major unmet needs in severe diseases. Skyline has established an advanced adeno-associated virus (AAV) based platform that seamlessly integrates cutting-edge capsid discovery, transgene design, vector engineering, process development and GMP manufacturing up to commercial-grade. The Skyline team of world-class experts and leaders in science and technology is advancing a deep and diverse pipeline of AAV-mediated gene therapies targeting ocular, neurological, cardiovascular, metabolic and blood disorders. Skyline Therapeutics has research, development and manufacturing capabilities in Shanghai and Hangzhou, China, and Boston, MA, US.