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 在人成纤维细胞中表现出高效且特异的 53 号外显子跳跃能力，并能够显著恢复 DMD 转录本的失衡^[1]。
Golodirsen sodium 可恢复人成纤维细胞中抗肌萎缩蛋白的表达，但蛋白恢复水平存在显著的个体差异，与跳跃效率无相关性^[1]。

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

Golodirsen sodium (12-960 mg/kg (小鼠)、100-900 mg/kg (大鼠)、5-320 mg/kg (食蟹猴)，静脉注射，单剂量) 在多个物种中表现出一致的药代动力学特征 (血浆半衰期：2.1-8.7 小时)，并且在目标骨骼肌组织和肾脏中观察到广泛的生物分布^[2]。

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

8647.40

C₃₀₅H₄₈₁N₁₃₈Na₂₅O₁₁₂P₂₅

固体

White to off-white

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.