Rostudirsen
Rostudirsen, one of the components of Zeleciment rostudirsen , is a phosphorodiamidate morpholino oligomer. Zeleciment rostudirsen consists of Rostudirsen (HY-177484) conjugated to a fragment antibody (Fab), Zeleciment (HY-P990780), which binds to the transferrin receptor 1 (TfR1) that is highly expressed in muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create internally shortened, near full-length dystrophin protein.
Product Specifications
CAS Number
3028750-59-3
Product Name Alternative
DYNE-251 Oligomer
UNSPSC
12352208
Target
Antibody-Oligonucleotide Conjugates (AOCs) ; Transferrin Receptor
Related Pathways
Antibody-drug Conjugate/ADC Related; Apoptosis
Field of Research
Others
Smiles
[Rostudirsen]
Molecular Weight
10451.91
References & Citations
[1]WHO Drug Information, Vol. 37, No. 2, 2023
Shipping Conditions
Room temperature
Scientific Category
Oligonucleotides
Clinical Information
Phase 2
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