HK Neopharm Limited is one of the most reliable manufacturers and suppliers of nusinersen in China, featured by quality products and good price. Please rest assured to buy nusinersen in stock here and get quotation from our factory. Customized orders are welcome.
Nusinersen (CAS 1258984-36-9) is the world's first approved antisense oligonucleotide (ASO) drug for spinal muscular atrophy (SMA), with nusinersen FDA approval for clinical application. Administered via intrathecal injection, it targets and modulates SMN2 gene splicing to promote full-length SMN protein synthesis and enhance motor neuron function. It is suitable for SMA patients of all ages and types, offering a vital therapeutic option for rare genetic diseases.
Technical Specifications
|
Product Name |
Nusinersen |
|
Synonyms |
Nusinersen powder, Nusinersen API |
|
Characteristics |
A white or off-white lyophilized powder |
|
CAS Number |
1258984-36-9 |
|
Assay |
≥99.0% (HPLC, Subject to the COA) |
|
Formula |
C234H340N61O128P17S17 |
|
Package |
Technical Package |
|
Storage |
1. API (Powder)Store frozen at -20°C, tightly sealed, dry and protected from light; storage under nitrogen is recommended.Solution: stable for 6 months at -80°C; 1 month at -20°C. 2.Injection (SPINRAZA)Store refrigerated at 2–8°C, protected from light in original package; do not freeze.Stable for up to 14 days at ≤30°C; up to 30 hours at ≤25°C after removal from package. |
|
Structure |
|
Nusinersen API is a highly specific antisense oligonucleotide raw material for rare disease treatment, mainly used in clinical R&D of SMA therapies and serving as a core product in RNA-targeted rare disease drugs. The free acid form of nusinersen powder differs distinctly from its salt form in physicochemical properties, enabling precise regulation of SMN2 mRNA splicing to efficiently produce functional SMN protein and support clinical SMA treatment. As one of the trusted China nusinersen manufacturers, we provide reliable raw material support.
Product Features
Adopting advanced oligonucleotide synthesis and purification technologies, nusinersen API achieves a purity of ≥99.0% (HPLC), complying with pharmaceutical-grade API standards with strictly controlled impurities, ensuring safety for clinical use.
As an ASO drug, nusinersen (CAS 1258984-36-9) binds specifically to SMN2 gene mRNA, accurately regulating splicing to generate full-length functional SMN protein and fundamentally improving SMA pathological progression.
This product is a white to off-white lyophilized powder with stable structure, requiring storage at -20°C in a light-proof sealed condition to retain long-term activity, matching preparation production and intrathecal administration processes.
This raw material supports therapy development for SMA patients of all ages and subtypes, delivering long-term treatment plans for rare disease patients.This offers broad clinical and market value.
Full-Cycle Quality Control & Consistent Quality
Full-Process Traceability Control
We establish a full-chain quality traceability system from raw material feeding to nusinersen powder release, with complete COA for each batch and real-time monitoring of key processes to ensure verifiable quality.
Strict Standard Compliance
In line with ICH and pharmaceutical API quality specifications, multi-dimensional tests (HPLC purity, impurities, moisture, etc.) are performed. Nusinersen API maintains stable purity ≥99.0%, with impurities far below the pharmacopoeia limits to meet global pharmaceutical standards.
Continuous Quality System Optimization
Relying on mature production platforms and regular quality audits, we continuously optimize processes and testing methods to ensure batch-to-batch consistency of nusinersen (CAS 1258984-36-9), providing customers with long-term stable raw material supply.
Application Scenarios
Nusinersen (CAS 1258984-36-9), the world's first targeted raw material for SMA, features precise RNA regulation and holds core value in rare disease diagnosis, treatment and drug R&D.
Core Raw Material for Clinical Therapy: As the key API of Spinraza, it is used to prepare intrathecal preparations, providing targeted treatment for SMA patients of all ages and subtypes to improve motor function.
Rare Disease Drug R&D Platform
As a benchmark ASO product, it offers process references and mechanism verification for similar RNA-targeted drugs, driving the advancement of rare disease treatment technologies.
Carrier for Formulation Process Development
With stable lyophilized powder properties, it optimizes ASO drug formulations and delivery systems, exploring more convenient administration routes to improve patient compliance.
Tool for Scientific & Basic Research
It is widely used in SMA pathogenesis and SMN protein function research, supporting scientists in analyzing pathological mechanisms and exploring new targets and therapies.
Global Supply Chain Support
We supply high-purity pharmaceutical-grade API to global pharmaceutical enterprises, ensuring large-scale production of SMA drugs as professional China nusinersen suppliers.
Packaging and Transportation
Nusinersen (CAS 1258984-36-9), a highly active pharmaceutical-grade ASO API, is packaged and transported in strict accordance with cold-chain standards to fully protect product activity and stability. Specifically as follows:
Customized Protective Packaging
Pharmaceutical-grade light-proof aluminum foil bags with vacuum sealing, moisture-proof buffers and dry ice containers are used, adapting to -20°C storage to isolate light, moisture and temperature fluctuations.
Full-Process Temperature-Controlled Cold-Chain Logistics
A full-link temperature monitoring network is built, with real-time temperature tracking during delivery to keep nusinersen API at -20°C and avoid quality risks.
Compliant International Storage & Transportation
In strict compliance with international API transportation regulations and customs declaration requirements, complete temperature records and documents are provided for safe global delivery.
Flexible Specification Adaptation
Multi-specification customized packaging and sub-packing plans are available for Customized nusinersen, adjusted to meet R&D and production needs of different scales.
Our Advantages
Stable Production Capacity
With mature large-scale production lines, we ensure long-term stable supply of nusinersen in stock with consistent batch quality for R&D and industrial production.
High-Standard Quality Control
Full-process strict quality inspection ensures purity and impurity indexes of nusinersen powder meet international pharmaceutical standards for safe and reliable products.
Customized Technical Support
Professional teams provide customized synthesis and sub-packing services with quick response and complete technical documents to accelerate project progress.
Safe & Efficient Logistics
Professional cold-chain transportation with traceable temperature control ensures efficient global delivery, offering customers a hassle-free purchasing experience.
FAQ
Nusinersen (CAS 1258984-36-9) is produced with mature synthesis and strict quality control, featuring high purity and batch consistency for R&D to industrial applications. It supports SMA drug development, formulation optimization and large-scale preparation, backing precision medicine. As a provider of nusinersen made in China, we offer bulk nusinersen powder and supply solutions to accelerate rare disease drug projects. Clients can buy nusinersen from us, and nusinersen free sample is available for evaluation upon request.
Hot Tags: nusinersen, China nusinersen manufacturers, suppliers, factory
