SHANGHAI, May 10, 2025 — Skyline Therapeutics, a gene therapy company in clinical-stage focused on creating innovative solutions for uncommon and critical illnesses, today announced a late-breaking abstract presentation at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting (May 13–17, 2025, New Orleans, Louisiana) on SKG1108, its experimental optogenetic gene therapy for Retinitis Pigmentosa (RP).
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 engineered to restore sight in RP patients who have photoreceptor degeneration. Its unique design incorporates a unique intravitreal capsid and a specifically designed Broad Wavelength Light-sensing Protein (BWLP) composed of opsins from various sources, which showed strong light-responsive activity across a broad spectrum of wavelengths in in vitro electrophysiological studies. In in vivo studies, a single intravitreal injection of SKG1108 exhibited superior effectiveness and safety in rd1 mice, with notable functional improvements in visual pathway electrophysiology, light perception, and visual acuity behavioral tests. The treatment introduces a single-stranded DNA payload encoding light-activated proteins into the retina, removing the need to target particular genetic mutations. By creating new light-sensing cells, SKG1108 seeks to restore visual function in individuals with late-stage RP, irrespective of the genetic cause. SKG1108 was granted U.S. FDA Orphan Drug Designation (ODD) in September 2024.
About Retinitis Pigmentosa (RP)
Retinitis Pigmentosa (RP) refers to a group of inherited retinal diseases (IRDs) associated with mutations in more than 100 different genes. These mutations result in a gradual loss of vision, typically starting with night blindness (rod degeneration), advancing to tunnel vision, and eventually leading to legal blindness because of near-complete photoreceptor death (rod and cone degeneration). Currently, there are no globally available treatments to effectively slow, stop, or reverse photoreceptor degeneration. Existing gene-specific therapies are only applicable to a small percentage of RP patients. SKG1108’s mutation-agnostic optogenetic strategy—designed to restore vision regardless of genetic defects—has the potential to be a transformative treatment option for majority of RP patients, including those with advanced stages of the disease.
About Skyline Therapeutics
Skyline Therapeutics is a clinical-stage, innovation-focused gene therapy company dedicated to creating innovative therapeutic solutions for rare and severe diseases that have significant unmet needs. Utilizing our advanced adeno-associated virus (AAV) platform, which includes multiple proprietary technologies for capsid discovery, vector design and engineering, process development, and GMP manufacturing, we are developing a varied pipeline of gene therapies aimed at treating ocular, neurological, and cardiovascular disorders. Our primary programs are advancing quickly through clinical trials, with regulatory approvals and special designations from the US FDA and China NMPA, bringing us closer to delivering life-changing therapies to patients in need.
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.
SOURCE Skyline Therapeutics
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