Description

Nusinersen is a synthetic antisense oligonucleotide designed to modulate RNA splicing. This compound is of critical importance as the first approved therapeutic agent for the treatment of spinal muscular atrophy (SMA), a severe neuromuscular disorder. It is essential for pharmaceutical manufacturers and research institutions engaged in developing and producing targeted therapies for genetic diseases, particularly within the neurology and rare disease sectors. Nusinersen CAS NO 1258984-36-9 represents a significant advancement in precision medicine.

Application

• Active Pharmaceutical Ingredient (API): Primary use as the active component in the commercial drug Spinraza® for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
• Pharmaceutical Research & Development: Serves as a key reference standard and investigational compound in preclinical and clinical studies for SMA and related neuromuscular conditions.
• Biomedical Research: Used as a tool compound to study RNA-targeting mechanisms, antisense oligonucleotide (ASO) pharmacology, and splice modulation in cellular and animal models.
• Drug Formulation Development: Employed in the development of novel delivery systems and formulations for intrathecal administration of oligonucleotide therapeutics.
• Contract Manufacturing: Supplied to CDMOs (Contract Development and Manufacturing Organizations) for the GMP-compliant production of finished dosage forms.
• Regulatory & Quality Control: Utilized as a high-purity standard for analytical method development, validation, and quality control testing in pharmaceutical laboratories.

Basic Information

Product Name	Nusinersen
CAS No.	1258984-36-9
Molecular Formula	C234H323N61O128P19S19
Molecular Weight	7501.0 g/mol (approximate)
Synonyms	ISIS 396443; ISIS-SMNRx; IONIS-SMNRx; Spinraza (brand name); 2'-O-Methoxyethyl Phosphorothioate Oligonucleotide; Antisense Oligonucleotide (ASO) targeting SMN2; 5'-d(T*A*G*G*A*A*A*T*A*T*A*A*A*C*T*T*C*A*A*T*A*A*T)-3' (sequence with modifications); Nusinersen sodium
EINECS	Contact for details

Quality Control

Our Nusinersen is manufactured under strict quality management systems. Each batch is subjected to comprehensive analytical testing, including identity confirmation by mass spectrometry, purity assessment by advanced HPLC methods, and stringent controls for related substances and residual solvents. We provide full traceability and Certificates of Analysis (COA) that detail compliance with relevant specifications. Our quality commitment supports applications requiring research-grade to cGMP standards.

Storage

Preserve in a tightly closed container, protected from light. Store frozen at -20°C or below. This product is hygroscopic (moisture-sensitive). Allow the vial to equilibrate to room temperature in a dry environment before opening to prevent condensation. For long-term storage, consider under an inert atmosphere.

Specification

Item	Specification
Appearance	White to off-white solid or lyophilized powder
Identification (MS)	Conforms
Identification (HPLC)	Conforms to reference standard
Assay (HPLC)	≥ 95.0%
Purity (Total Oligonucleotide)	≥ 85.0%
Related Substances (HPLC)	Individual impurity ≤ 2.0%; Total impurities ≤ 10.0%
Water Content (KF)	≤ 5.0%
Endotoxin	< 10.0 EU/mg
Residual Solvents (GC)	Meets ICH guidelines

Note: Specifications can be tailored. Please contact us for the detailed technical data sheet of a specific grade.