Agalsidase alfa, Recombinant human alpha-galactosidase A

Specification

• Cat#: THP-0115
• Product Name: Agalsidase alfa, Recombinant human alpha-galactosidase A
• Description: Recombinant human alpha-galactosidase A, a mature protein composed of 2 subunits of 398 residues, also glycosylated and produced in CHO cells.
• Sequences: LDNGLARTPTMGWLHWERFMCNLDCQEEPDSCISEKLFMEMAELMVSEGWKDAGYEYLCIDDCWMAPQRDSEGRLQADPQRFPHGIRQLANYVHSKGLKLGIYADVGNKTCAGFPGSFGYYDIDAQTFADWGVDLLKFDGCYCDSLENLADGYKHMSLALNRTGRSIVYSCEWPLYMWPFQKPNYTEIRQYCNHWRNFADIDDSWKSIKSILDWTSFNQERIVDVAGPGGWNDPDMLVIGNFGLSWNQQVTQMALWAIMAAPLFMSNDLRHISPQAKALLQDKDVIAINQDPLGKQGYQLRQGDNFEVWERPLSGLAWAVAMINRQEIGGPRSYTIAVASLGKGVACNPACFITQLLPVKRKLGFYEWTSRLRSHINPTGTVLLQLENTMQMSLKDLL
• Species: Human
• Molecular Weight: 45351.6 Da
• Source: CHO
• Purity: >99% by SDS-Page and HPLC analysis
• Cas No: 104138-64-9
• Formula: C2029H3080N544O587S27
• Endotoxin Level: <0.001 EU per 1 μg of the protein by the LAL method
• Biological Activity: 1mg/ml
• Storage: Lyophilized protein should be stored at < -20°C, though stable at room temperature for 3 weeks. Reconstituted protein solution can be stored at 2-8 °C for 1 week. Aliquots of reconstituted samples are stable at < -20°C for 3 months.

Pharmaceutical Information

• Agalsidase alfa: alpha-galactosidase
• Applications: For treatment of Fabry's disease (alpha-galactosidase A deficiency).
• Examples of Clinical Use: Fabry disease
• Pharmacodynamics: Used in the treatment of Fabry disease, an X-linked genetic disorder of glycosphingolipid metabolism. The disease is characterized by a deficiency of the lysosomal enzyme alpha-galactosidase A, which leads to progressive accumulation of glycosphingolipids, predominantly GL-3, in many body tissues. Clinical manifestations of Fabry disease include renal failure, cardiomyopathy, and cerebrovascular accidents. Recombinant human alpha-galactosidase A is intended to provide an exogenous source of alpha-galactosidase A and to limit the accumulation of these glycolipids in the tissues. Studies show unconclusive evidence for the clinical efficacy of either agalasidase alfa or agalasidase beta in preventing morbidity.
• Mechanism of action: Alpha-galactosidase A catalyzes the hydrolysis of globotriaosylceramide (GL-3) and other a-galactyl-terminated neutral glycosphingolipids, such as galabiosylceramide and blood group B substances to ceramide dihexoside and galactose.
• Affected organisms: Humans and other mammals