Skyline Therapeutics has received clinical trial approval from China’s National Medical Products Administration (NMPA) for its proprietary AAV gene therapy, SKG0201 injection, which is being developed as a treatment for type I spinal muscular atrophy (SMA). SKG0201 is an SMN1 gene replacement therapy that is administered through a single intravenous infusion.
The drug candidate features an innovative vector design, comprising human SMN1 cDNA with a unique central nervous system (CNS)-specific promoter regulation and comprehensive codon optimization. This design aims to enhance tissue targeting, enabling the normal SMN1 gene to achieve a maximized therapeutic effect within the CNS after introduction into the body. It is intended to rapidly restore the expression of the normal SMN protein in motor neurons at low doses, thereby safely and effectively improving the function of affected cells, including motor neurons.
SKG0201 has initiated an investigator-initiated trial in type 1 SMA, demonstrating good tolerability and therapeutic effects in early evaluations.- Flcube.com
