Golodirsen sodium  (Synonyms: SRP-4053 sodium)

Golodirsen (SRP-4053) sodium is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen sodium restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen sodium can restore the expression of the anti-myostatin protein. Golodirsen sodium can be used for the research of duchenne muscular dystrophy (DMD)^[1][2].

Golodirsen sodium demonstrates efficient and specific exon 53 skipping ability in human fibroblasts, and is able to significantly restore the imbalance of DMD transcripts^[1].
Golodirsen sodium restores the expression of the anti-myostatin protein in human fibroblasts, but there are significant individual differences in the level of protein recovery, and it has no correlation with the jumping efficiency^[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Golodirsen sodium (12-960 mg/kg (mice), 100-900 mg/kg (rats), 5-320 mg/kg (cynomolgus monkeys), i.v., single dose) exhibits consistent pharmacokinetic characteristics in multiple species (plasma half-lives: 2.1-8.7 h) and extensive biodistribution is observed in the target skeletal muscle tissues and the kidney^[2].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

8647.40

C₃₀₅H₄₈₁N₁₃₈Na₂₅O₁₁₂P₂₅

Solid

White to off-white

[Golodirsen (sodium)]

Room temperature in continental US; may vary elsewhere.

-20°C, stored under nitrogen, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)

• [1]. Rossi R, et al. Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells. Skelet Muscle. 2024 Nov 29;14(1):28.  [Content Brief]

  [2]. Goey, Andrew KL, et al. Characterization of Nonclinical Drug Metabolism and Pharmacokinetic Properties of Phosphorodiamidate Morpholino Oligonucleotides, a Novel Drug Class for Duchenne Muscular Dystrophy. Drug Metabolism and Disposition 52.12 (2024): 1396-1406.