Drug intelligence / Profile preview

SKG1310

Development stage
Preclinical
Lead developer
Skyline Therapeutics
Modality
Gene Therapies
Administration
Intravitreal
01

Overview

SKG1310 is a first-in-class, dual-target AAV gene therapy developed by Skyline Therapeutics for the treatment of geographic atrophy (GA), an advanced stage of age-related macular degeneration (AMD). It utilizes a proprietary retinal-tropic AAV capsid to deliver a bicistronic vector that co-expresses a chimeric complement inhibitor (CCI) and an optimized neurotrophic factor (NF). The CCI is designed to inhibit both the classical and alternative complement pathways, addressing the chronic dysregulation of the complement cascade that drives retinal pigment epithelium (RPE) and photoreceptor degeneration. Simultaneously, the neurotrophic factor provides support to retinal cells to preserve structure and function. Administered via a single intravitreal injection, SKG1310 aims to provide a durable, disease-modifying therapeutic effect by addressing the multifactorial pathology of GA.

02

Targets

Complement (Complement system)NTF3 (Neurotrophin-3)

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