Skyline Therapeutics has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for SKG1108, a gene therapy being developed for the treatment of retinitis pigmentosa (RP). This designation aims to accelerate the development of SKG1108 and improve patient access to this potential therapeutic solution for this inherited retinal disease.
SKG1108 is a recombinant adeno-associated virus (rAAV) vector designed for a one-time intravitreal delivery. It utilizes the intravitreal capsid AAV.0106 to deliver single-stranded DNA encoding light-activatable proteins directly to the retina. According to Skyline Therapeutics, the protein, regulated by specific genetic elements, is intended to improve or restore visual function by generating new photo-sensing cells.
The Orphan Drug Designation status offers several benefits, including tax credits for qualified clinical trials, exemption from prescription drug user fees, and the potential for seven years of market exclusivity upon approval. These incentives are designed to encourage innovation and research into treatments for rare diseases.
Retinitis Pigmentosa and SKG1108's Approach
Retinitis pigmentosa (RP) encompasses a group of inherited retinal diseases linked to mutations in over 100 different genes or loci, all leading to progressive vision loss. The disease typically begins with the degeneration of rod cells, followed by the gradual loss of cone cells, ultimately leading to near-complete loss of all photoreceptor cells and blindness. SKG1108 is designed to compensate for the loss of both rod and cone photoreceptors in patients with late-stage RP, irrespective of the specific gene or genetic mutation responsible for the condition. This approach offers a potential treatment option for a broad range of RP patients, regardless of the underlying genetic cause.
