Skyline Therapeutics (Shanghai) Co. Ltd.’s SKG-1108, a novel one-time intravitreally delivered gene therapy, has been awarded U.S. orphan drug designation for the treatment of retinitis pigmentosa.

Skyline Therapeutics (Shanghai) Co. Ltd. has received FDA clearance for its IND application for a phase I/IIa trial of SKG-0106, a one-time intravitreally delivered recombinant adeno-associated virus (AAV) gene therapy for the treatment of neovascular age-related macular degeneration (wet AMD). A global phase I trial will be initiated soon.

Spinal muscular atrophy (SMA) is caused by mutations in the SMN1 gene, which encodes survival motor neuron 1, leading to reduced protein expression levels and degeneration of motor neurons in the spinal cord, with the consequent muscle atrophy. There is thus a need for new AAV gene therapies for SMA that confer better safety and efficacy.

Researchers from Skyline Therapeutics (Shanghai) Co. Ltd. presented preclinical data for the new recombinant AAV vector therapeutic SKG-0106, being developed for the treatment of neovascular (wet) age-related macular degeneration (AMD).