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 Penetrating
Risdiplam (API & Research Grade)
High-Purity Small Molecule for Spinal Muscular Atrophy (SMA) Research
RESEARCH NOTICE:
This product is a high-purity chemical intended for Laboratory R&D and Industrial Use ONLY. It is not a finished drug product and not for direct human consumption. Shaanxi Sunrise Pharmaceutical adheres to strict compliance protocols.
Scientific Overview
Shaanxi Sunrise Pharmaceutical provides ultra-high-purity Risdiplam, a revolutionary small-molecule splicing modifier. Our product is synthesized via a specialized copper-catalyzed process, ensuring heavy metal residues are kept below 1ppm, making it an ideal choice for large-scale formulation research of oral solutions and tablets.
Risdiplam is unique for its ability to penetrate the blood-brain barrier (BBB), allowing for systemic distribution and targeting SMN protein deficiency in both the central nervous system and peripheral tissues.
Mechanism of Action (MOA)
SMN2 Gene Modification
The core cause of SMA is the loss of the SMN1 gene. Risdiplam addresses this by:
Dual-Site Specificity: Modulates the splicing of the SMN2 gene (the homologous gene to SMN1).
Exon-7 Inclusion: Promotes the inclusion of Exon 7 into the SMN2 mRNA transcript.
Protein Expression: Significantly increases the levels of functional, full-length SMN protein systemically.
Core Research Benefits
✅ Survival Breakthrough: Critical for studying survival rate improvements in neonatal models.
✅ Motor Function: Benchmarking motor neuron stabilization and motor function recovery.
✅ Systemic Impact: Protective effects across respiratory and swallowing functions.
✅ Full-Cycle Study: Applicable from prenatal intervention studies to adult disease management.
Chemical & Structural Properties
Validated via advanced analytical techniques to ensure batch-to-batch consistency (>99.5% stability):
Density: 1.50 ± 0.1 g/cm³ (Predicted)
Acidity (pKa): 9.41 ± 0.20
Verification: HPLC-UV, NMR, and LC-MS structural confirmation provided.
Traceability: Batch stability data and COA available upon request.
Quality & Compliance
Our raw materials follow strict cold-chain protocols during storage and transportation. We provide customized packaging (5g to 100g+) and pre-batch supply to support our global partners in accelerating formulation research and regulatory compliance declarations.