Skyline Therapeutics has received FDA clearance for its investigational new drug (IND) application for a phase 1/2a clinical trial on SKG0106, a gene therapy designed to treat neovascular age-related macular degeneration (nAMD).
Refresh me on the company.
Founded in 2019 and headquartered in Shanghai, China, Skyline Therapeutics is a clinical-stage, fully-integrated gene and cell therapy company focused on developing and advancing a pipeline of gene therapies for ocular, neurological, cardiovascular, and metabolic disorder.
Of note, the company has a U.S. based facility in Cambridge, Massachusetts.
And its technology?
The company’s proprietary adeno-associated virus (AAV) integrated gene therapy platform—called capDRIVE—is based on capsid encoding nucleic acid recovery technology.
The platform combines multiple proprietary technologies for capsid discovery, therapeutic transgene design, vector engineering, process development, and good manufacturing practice (GMP) production.
Now SKG0106.
SKG0106 is a one-time intravitreally-delivered gene therapy that uses a recombinant AAV with a unique transgene configuration.
It’s currently being developed to have a potent inhibition effect on intraocular neovascularization, making it a potential treatment for serious ophthalmic conditions (like nAMD).
Gotcha. So how does this involve capDRIVE?
The platform was used to develop SKG0106 GMP-grade material in order to support both preclinical and clinical programs for the IND application.
Included among the processes capDRIVE integrates are:
- Cell line development
- Plasmid and viral vector process development
- Analytical development
- GMP production
What did these preclinical programs show?
Sort of … preclinical proof-of-concept studies found SKG0106 to have a favorable safety profile and be an effective anti-angiogenic treatment with long-lasting, durable suppression of neovascular lesions when used at a low-dose, single intravitreal injection, according to the company.
So what’s next?
As part of the IND application acceptance, Skyline Therapeutics’ plans to initiate phase 1 of its phase 1/2a global clinical trial on SKG0106.
No specific date for initiating enrollment has been set yet (according to Clinical Trials), so stay tuned for further updates!