Product description
Golodirsen is an active pharmaceutical ingredient (API) designed for exon-skipping therapy targeting the DMD gene. It is a next-generation gene repair material for rare muscular disorders, featuring a charge-neutral phosphorodiamidate morpholino oligomer (PMO) backbone with high stability and an excellent safety profile. Unlike charged oligonucleotide therapeutics, its neutral morpholino backbone significantly reduces nonspecific binding and immune stimulation in vivo, providing superior biocompatibility and enhanced tissue penetration for efficient accumulation in skeletal and cardiac muscle.
Through a precise exon 53-skipping mechanism, Golodirsen corrects abnormal DMD pre-mRNA splicing and restores the expression of a shortened yet biologically functional dystrophin protein. This targeted approach helps slow progressive skeletal muscle degeneration and delay the loss of motor function while offering high specificity, a well-defined mechanism of action, durable therapeutic activity, and minimal systemic adverse effects.
Manufactured using a proprietary precision PMO synthesis platform, the product achieves a purity of ≥99%, with trace synthetic by-products and residual impurities strictly controlled in accordance with pharmacopeial and ICH quality standards. It provides outstanding batch-to-batch consistency, sequence uniformity, molecular stability, and precisely controlled gene-repair activity, fully meeting the quality requirements for the development and large-scale production of advanced genetic medicines for rare diseases.
Produced on standardized GMP-compliant manufacturing lines, the product can be supplied with a complete Certificate of Analysis (COA), comprehensive impurity profiles, and DMF documentation to support research, process development, regulatory submission, and commercial manufacturing of targeted muscle injection formulations, therapies for inherited muscular dystrophies, and gene-targeted biologics for rare diseases.
The product efficiently mediates DMD exon 53 skipping, restores functional dystrophin expression, suppresses degenerative apoptosis of skeletal muscle cells, delays the progression of muscle atrophy, and helps preserve motor function. It combines precise gene-splicing correction, muscle cell protection, muscle tissue regeneration, and long-term intervention for rare muscular disorders, making it a high-quality pharmaceutical intermediate for the development of innovative gene therapies for Duchenne muscular dystrophy (DMD).
This product is intended solely as a pharmaceutical and biopharmaceutical intermediate and is not intended for direct human use.