Skyline Therapeutics to Present Novel Optogenetic Gene Therapy for Retinitis Pigmentosa at ASGCT 2025
Key Insights
Skyline Therapeutics (search) will present data on SKG1108, a mutation-agnostic optogenetic gene therapy designed to restore vision in patients with Retinitis Pigmentosa (search), at the ASGCT Annual Meeting in New Orleans.
SKG1108 combines a proprietary intravitreal capsid with a uniquely engineered Broad Wavelength Light-sensing Protein (search), showing promising results in preclinical studies with a single intravitreal injection.
The therapy received FDA Orphan Drug Designation in September 2024 and offers potential treatment for the majority of RP patients regardless of their specific genetic mutation.
Skyline Therapeutics (search) announced today that it will present a late-breaking abstract on SKG1108, its investigational optogenetic gene therapy for Retinitis Pigmentosa (search) (RP), at the upcoming American Society of Gene and Cell Therapy (search) (ASGCT) 28th Annual Meeting in New Orleans, Louisiana, scheduled for May 13-17, 2025.
The presentation, titled "SKG1108: Optogenetic Gene Therapy with AAV-BWLP for Visual Restoration in Retinitis Pigmentosa (search) via a Single Intravitreal Injection," will take place on May 13, 2025, at 6:00 PM CT, highlighting the company's innovative approach to treating this rare genetic eye disorder.
Novel Mechanism of Action
SKG1108 represents a significant advancement in the treatment landscape for RP, a condition currently lacking effective therapies for most patients. Unlike conventional gene therapies that target specific genetic mutations, SKG1108 employs a mutation-agnostic approach using optogenetics to potentially restore vision regardless of the underlying genetic defect.
The therapy utilizes an adeno-associated virus (AAV) vector to deliver a single-stranded DNA payload encoding light-activatable proteins to the retina. Its design combines a proprietary intravitreal capsid with a uniquely engineered Broad Wavelength Light-sensing Protein (search) (BWLP) comprising opsins from multiple sources.
"This optogenetic approach has the potential to address a significant unmet need in the RP patient community," said a spokesperson from Skyline Therapeutics (search). "By bypassing the need to target specific genetic mutations, SKG1108 could offer hope to a much broader patient population, including those with advanced disease."
Promising Preclinical Results
In preclinical studies, SKG1108 has demonstrated encouraging results. In vitro electrophysiological studies showed strong and broad wavelength range light-responsive activity. More significantly, in vivo studies in rd1 mice revealed that a single intravitreal injection of SKG1108 produced superior efficacy and safety profiles.
The treatment led to striking functional improvements in visual pathway electrophysiology, light perception, and visual acuity behavioral tests. These results suggest that by generating new photo-sensing cells, SKG1108 may restore visual function in late-stage RP patients who have experienced significant photoreceptor degeneration.
Retinitis Pigmentosa: A Challenging Genetic Disorder
Retinitis Pigmentosa (search) represents a group of inherited retinal diseases linked to mutations in over 100 different genes. The condition leads to progressive vision loss, typically beginning with night blindness due to rod photoreceptor degeneration, followed by tunnel vision, and eventually legal blindness as both rod and cone photoreceptors deteriorate.
Currently, there are no widely effective treatments that can slow, halt, or reverse photoreceptor degeneration in RP. Existing gene-specific therapies only apply to a small subset of patients with particular genetic mutations, leaving the majority without viable treatment options.
Approximately 1 in 4,000 people worldwide are affected by RP, translating to roughly 2 million individuals globally who could potentially benefit from a mutation-agnostic approach like SKG1108.
Regulatory Progress and Development Timeline
SKG1108 received U.S. FDA Orphan Drug Designation (ODD) in September 2024, highlighting the therapy's potential significance for this rare disease population. The designation provides various development incentives, including tax credits for clinical trials, exemption from user fees, and potential market exclusivity upon approval.
Skyline Therapeutics (search) is leveraging its proprietary adeno-associated virus (AAV) platform, which encompasses technologies for capsid discovery, vector design and engineering, process development, and GMP manufacturing to advance SKG1108 through clinical development.
About Skyline Therapeutics
Shanghai-based Skyline Therapeutics (search) is a clinical-stage gene therapy company focused on developing novel therapeutic solutions for rare and severe diseases with high unmet needs. The company is advancing a diverse pipeline of gene therapies targeting ocular, neurological, and cardiovascular disorders.
Their lead programs are progressing through clinical trials with regulatory approvals and special designations from both the U.S. FDA and China NMPA (search). Skyline's approach combines cutting-edge AAV technology with innovative therapeutic designs to address challenging conditions like Retinitis Pigmentosa (search).
The upcoming presentation at ASGCT, the largest association of individuals involved in gene and cell therapy research with more than 5,000 members worldwide, represents an important opportunity for Skyline to share its progress with the scientific community and potentially accelerate the development of this promising therapy for patients with limited treatment options.