Product Details:

CasNo： 1258984-36-9

Molecular Formula： C234H340N61O128P17S17

Appearance： White to off‑white solid

Packing： Bottle

Throughput： 1KG/Month

Purity： 99%

Nusinersen（诺西那生钠）

Brand Name: Spinraza®

CAS‑No.: 1258984‑36‑9

Chemical class: 2′‑O‑Methoxyethyl‑modified phosphorothioate antisense oligonucleotide (ASO)

Molecular formula: C₂₃₄H₃₂₃N₆₁O₁₂₈P₂₁S₂₁

Molecular weight: 7501.4 Da

Mechanism of Action（FDA SPL）

Nusinersen is a synthetic, phosphorothioate‑modified antisense oligonucleotide designed to modulate SMN2 pre‑mRNA splicing. It hybridizes to the intron‑splicing silencer element within intron 7 of SMN2 pre‑mRNA, promoting inclusion of exon 7 into mature SMN2‑derived messenger RNA. This yields full‑length, functional survival motor neuron (SMN) protein, compensating for deficient SMN1‑derived protein production in spinal muscular atrophy (SMA) caused by biallelic loss‑of‑function mutations or deletions of the SMN1 gene. Nusinersen is administered intrathecally to achieve CNS exposure, as it poorly crosses the intact blood‑brain barrierPMC.

Indications and Usage (Approved Clinical Label)

Spinal Muscular Atrophy (SMA) Nusinersen is indicated for the treatment of paediatric and adult patients with spinal muscular atrophy (SMA), caused by homozygous deletion or pathogenic variants in the SMN1 gene, including:

• Infantile‑onset SMA (Type 1)
• Later‑onset SMA (Type 2 and Type 3)
• Presymptomatic SMA patients with genetically confirmed diagnosis of SMA

Dosing regimen (clinical): Intrathecal injection; loading doses at Days 0, 14, 28, 63; followed by maintenance dose every 4 months thereafter.

Disclaimer for research‑grade material This oligonucleotide active pharmaceutical ingredient is supplied For Research Use Only (RUO). It is not manufactured under cGMP for human clinical use. Formulation, intrathecal administration or any human therapeutic application requires full local regulatory approval and compliant cGMP manufacturing.