Drug intelligence / Profile preview

SKG1108

Development stage
Preclinical
Lead developer
Skyline Therapeutics
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravitreal
01

Overview

SKG1108 is a novel, one-time intravitreally delivered gene therapy developed for the treatment of retinitis pigmentosa (RP), a progressive inherited retinal disease that leads to vision loss and blindness. It utilizes a recombinant adeno-associated virus (rAAV) vector with an innovative intravitreal capsid (AAV.0106) to deliver single-stranded DNA encoding light-activatable proteins directly to the retina. These proteins, regulated by specific genetic elements, are designed to generate new photo-sensing cells in the retina, thereby compensating for the loss of rod and cone photoreceptors in patients with late-stage RP. This approach is agnostic to the underlying genetic mutation causing RP and aims to improve or restore visual function across a broad range of patients[1][2][4][5][6][7].

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