Risdiplam Powder API CAS No:1825352-65-5
Risdiplam Powder API CAS No:1825352-65-5
Risdiplam Powder CAS No:1825352-65-5,CAS No:1825352-65-5,Risdiplam Powder API
Product Name: Risdiplam API Powder
Product purity: ≥ 99% (pharmaceutical grade); ≥ 98% (research level)
CAS No: 1825352-65-5
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Description
SMN2 Splicing Modifier • CNS PenetratingRisdiplam API & Research Grade Supply
Ultra-High Purity Small Molecule Agonist for Spinal Muscular Atrophy (SMA) Formulation & Therapeutic Development Pipelines
Assay ≥ 99.5% (Pharma Grade)
Copper Catalyst Residue ≤ 1 ppm
Validated Structural Identity (NMR/MS)
CAS 1825352-65-5
⚠ REGULATORY COMPLIANCE DIRECTIVE: INDUSTRIAL B2B SUPPLY ONLY
Shaanxi Sunrise Pharmaceutical Co., Ltd. operates strictly as an industrial active raw material and ingredient manufacturer and supplier. This product is a high-purity small molecule chemical compound intended exclusively for laboratory scientific research, non-clinical assay calibration, and generic drug formulation scale-up development. Shaanxi Sunrise strictly prohibits the distribution or retail of raw materials to private individuals for direct human consumption or therapeutic use.
Scientific Overview
In the highly demanding landscape of neurodegenerative and rare disease therapeutics, maintaining absolute batch-to-batch chemical predictability is critical. Risdiplam (CAS 1825352-65-5) supplied by Shaanxi Sunrise Pharmaceutical Co., Ltd. is a revolutionary, orally bioavailable small-molecule splicing modifier designed to target survival motor neuron (SMN) protein deficiency.
Our premium pharmaceutical-grade Risdiplam powder is synthesized via a proprietary, highly optimized copper-catalyzed coupling pathway. By utilizing precise multi-stage recrystallization matrices, we successfully clear heavy metal catalytic residues down to less than 1 ppm. This strict organic purity threshold actively eliminates background cell toxicity, making our material the premier sourcing standard for generic formulators developing stable oral solutions and solid tablet dosage forms.
A defining pharmacokinetic advantage of Risdiplam is its exceptional ability to efficiently cross the blood-brain barrier (BBB). This allows for uniform, systemic distribution throughout both the central nervous system (CNS) and peripheral tissues, ensuring uncompromised cellular exposure where motor neuron degradation occurs.
SMN2 Gene Splicing Modification Kinetics
The core pathological driver of Spinal Muscular Atrophy (SMA) is the homozygous deletion or mutation of the SMN1 gene. Risdiplam addresses this molecular gap by directly modifying the pre-mRNA splicing pattern of the SMN2 gene—the highly homologous backup gene:
Dual-Site Specificity Coordination: It binds simultaneously to two distinct sites in the SMN2 pre-mRNA transcript: the 5' splice site of intron 7 and the exonic splicing enhancer (ESE) of exon 7.
Exon 7 Systematic Inclusion: This dual-site interaction selectively shifts the splicing equilibrium, successfully forcing the inclusion of Exon 7 into the mature mRNA transcript.
Systemic Functional Upregulation: By preventing the generation of truncated, unstable protein variants, it drives the expression of stable, full-length, functional SMN proteins systemically.
Core Strategic Research Benefits
▶ Survival Rate Optimization: Serving as an essential baseline control agent for investigating survival curve expansions and phenotype corrections in neonatal transgenic models.
▶ Motor Function Metrics: Providing high-accuracy benchmarking for evaluating motor neuron alpha-junction stabilization and measuring functional coordination recovery curves.
▶ Systemic Bio-Efficacy Mapping: Evaluating downstream cytoprotective and therapeutic impacts across non-CNS target organs, including respiratory and swallowing pathways.
▶ Full-Cycle Developmental Studies: Ideally qualified for complex academic modeling extending from early prenatal intervention studies to late-stage adult disease management profiles.
Audited Release Specifications & Quality Metrics
| Analytical Specification Parameter | Pharmacopoeia Limit Standard | Typical Factory Release Result | Validated Methodology |
|---|---|---|---|
| Assay Purity (Dried Basis) | ≥ 99.0% | ≥ 99.68% | Reversed-Phase HPLC-UV |
| Copper Catalyst Residue | ≤ 5 ppm | ≤ 0.8 ppm | ICP-MS Elemental Assay |
| Single Maximum Impurity | ≤ 0.10% | 0.04% | HPLC Area Normalization |
| Total Related Substances | ≤ 0.50% | 0.22% | HPLC Area Normalization |
| Predicted Density Profile | 1.50 ± 0.1 g/cm³ | Conforms | Solid-State Pycnometry |
| Acidity Coefficient (pKa) | 9.41 ± 0.20 | 9.38 | Potentiometric Titration |
| Loss on Drying | ≤ 0.50% | 0.15% | Vacuum Oven Drying |