Golodirsen (SRP-4053) is a phosphodiimide morpholino oligomer (PMO) that specifically targets exon 53 of the dystrophin trophin pre-mRNA. Golodirsen may be utilized in study/research of Duchenne muscular dystrophy (DMD).

Physicochemical Properties

CAS #	1422959-91-8
Appearance	White to off-white solid powder
HS Tariff Code	2934.99.9001
Storage	Powder-20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment (e.g. under nitrogen), avoid exposure to moisture.
Shipping Condition	Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)

Biological Activity

ln Vitro	Golodirsen selectively binds to exon 53 of the dystrophin pre-mRNA, preventing it from being included in the finished mRNA product. By omitting exon 53, the mRNA reading frame is restored and a partially internally deleted dystrophin protein with intact C- and N-terminal regions is produced [2].
References	[1]. Heo YA. Golodirsen: First Approval. Drugs. 2020 Feb;80(3):329-333. [2]. The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy. Acta Neuropathol Commun. 2021 Jan 6;9(1):7.

Solubility Data

Solubility (In Vitro)	H2O : ~100 mg/mL (~11.56 mM)
Solubility (In Vivo)	Solubility in Formulation 1: 50 mg/mL (5.78 mM) in PBS (add these co-solvents sequentially from left to right, and one by one), clear solution; with sonication.  (Please use freshly prepared in vivo formulations for optimal results.)