Skyline Therapeutics' Novel Gene Therapy SKG1108 Receives FDA Orphan Drug Designation for Retinitis Pigmentosa

BOSTON and SHANGHAI, Sept. 3, 2024 /PRNewswire/ -- Skyline Therapeutics, an innovation-driven gene therapy company committed to developing unique and novel solutions for rare and severe diseases, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SKG1108, a novel one-time intravitreally delivered gene therapy for the treatment of Retinitis Pigmentosa (RP). This designation reflects the recognition by the FDA of the potential of SKG1108 for treating RP. The ODD grants special status to SKG1108 and qualifies it for various benefits, which will greatly accelerate the development of the drug and enhance patient access to the leading-edge therapeutic solution.

SKG1108 is a recombinant adeno-associated virus (rAAV) vector with innovative design, utilizing the novel intravitreal capsid AAV.0106 to deliver single-stranded DNA encoding light-activatable proteins directly to the retina. The protein, regulated by specific genetic elements, aims to improve or restore visual function by generating new photo-sensing cells, thereby compensating for the loss of rod and cone photoreceptors in patients with late-stage RP, agnostic on the specific gene or genetic mutation responsible for the condition.