Skyline Therapeutics Novel Gene Therapy SKG1108 Gains FDA Orphan Drug Designation for Retinitis Pigmentosa
Skyline Therapeutics has received Orphan Drug Designation (ODD) from the US Food and Drug Administration for SKG1108. This new gene therapy, delivered through a single intravitreal injection, aims to treat Retinitis Pigmentosa (RP).
The ODD signifies the FDA's recognition of SKG1108's potential to help patients with RP, providing it with special status and various benefits to expedite its development and improve patient access.
SKG1108 is a recombinant adeno-associated virus (rAAV) vector that uses the new intravitreal capsid AAV.0106 to deliver single-stranded DNA encoding light-activatable proteins directly to the retina.
This therapy seeks to enhance or restore visual function by generating new photo-sensing cells, compensating for the loss of rod and cone photoreceptors in patients with advanced RP, regardless of the specific genetic mutation causing the condition.
Retinitis Pigmentosa is a group of inherited retinal diseases (IRDs) associated with mutations in over 100 different genes, leading to gradual vision loss. The condition starts with the degeneration of rod cells, followed by the gradual loss of cone cells, ultimately resulting in nearly complete photoreceptor cell loss and blindness.
Currently, there are no effective global treatments to stop or reverse this degeneration, and existing therapies targeting specific genetic mutations are applicable to only a small fraction of RP patients. SKG1108 offers a novel approach that has the potential to improve or restore vision for a wider range of patients, independent of their genetic defects.
