SHANGHAI, May 11, 2025 /PRNewswire/ — Skyline Therapeutics, an innovation-driven clinical-stage gene therapy company focused on developing unique and novel solutions for rare and severe diseases, today announced a late-breaking abstract presentation on SKG1108, its investigational optogenetic gene therapy for Retinitis Pigmentosa (RP), at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting (May 13–17, 2025, New Orleans, Louisiana).
ASGCT 2025 Late-Breaking Abstract
Abstract Title: SKG1108: Optogenetic Gene Therapy with AAV-BWLP for Visual Restoration in Retinitis Pigmentosa via a Single Intravitreal Injection
Abstract No.：2023
Date & Time: May 13, 2025 | 6:00 PM – 7:30 PM CT
About SKG1108
SKG1108 is an optogenetic adeno-associated virus (AAV) gene therapy vector designed to restore vision in RP patients with photoreceptor degeneration. Its novel design combines a proprietary intravitreal capsid and a uniquely engineered Broad Wavelength Light-sensing Protein (BWLP) comprising opsins of multiple sources, which demonstrated strong and broad wave-length range light-responsive activity in in vitro electrophysiological studies. In in vivo studies, a single intravitreal injection of SKG1108 showed superior efficacy and safety in rd1 mice, with striking functional improvements in visual pathway electrophysiology, light perception and visual acuity behavioral tests. The therapy delivers a single-stranded DNA payload encoding light-activatable proteins to the retina, bypassing the need to target specific genetic mutations. By generating new photo-sensing cells, SKG1108 aims to restore visual function in late-stage RP patients, regardless of their underlying genetic defect. SKG1108 received U.S. FDA Orphan Drug Designation (ODD) in September 2024.
About Retinitis Pigmentosa (RP)
Retinitis Pigmentosa (RP) is a type of inherited retinal diseases (IRDs) linked to mutations in over 100 different genes, leading to progressive vision loss that typically begins with night blindness (rod degeneration), followed by tunnel vision and eventual legal blindness due to near-total photoreceptor death (rod and cone degeneration). Currently, no effective treatments worldwide can effectively slow, halt, or reverse photoreceptor degeneration, and existing gene-specific therapies only apply to a small subset of RP patients. SKG1108’s mutation-agnostic optogenetic approach—designed to restore vision independent of genetic defects—has the potential to become a transformative treatment for the vast majority of RP patients, including those with advanced disease.
About Skyline Therapeutics
Skyline Therapeutics is an innovation-driven, clinical-stage gene therapy company focused on developing unique and novel therapeutic solutions for rare and severe diseases with high unmet needs. Leveraging our cutting-edge adeno-associated virus (AAV) platform, which encompasses multiple proprietary technologies for capsid discovery, vector design and engineering, process development and GMP manufacturing, we are advancing a diverse pipeline of gene therapies targeting ocular, neurological, and cardiovascular disorders. Our lead programs are rapidly progressing through clinical trials with regulatory approvals and special designations from the US FDA and China NMPA, bringing us closer to providing life-changing therapies to patients in need. www.skytx.com
About ASGCT
The American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of gene, related cell and nucleic acid therapies, as well as promotion of professional and public education in the field. With more than 5,000 members in the US and worldwide, ASGCT is the largest association of individuals involved in gene and cell therapy research. The ASGCT Annual Meeting is the premier event for gene and cell therapy professionals to learn from the latest scientific research and stay current on new technologies.
