CA2880880C - Cyclodextrin for the treatment of lysosomal storage diseases - Google Patents

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Publication number

CA2880880C

CA2880880C CA2880880A CA2880880A CA2880880C CA 2880880 C CA2880880 C CA 2880880C CA 2880880 A CA2880880 A CA 2880880A CA 2880880 A CA2880880 A CA 2880880A CA 2880880 C CA2880880 C CA 2880880C

Authority

CA

Canada

Prior art keywords

cyclodextrin

disease

tocopherol

compound

cholesterol

Prior art date

2012-08-03

Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)

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• 208000015439 Lysosomal storage disease Diseases 0.000 title claims abstract description 47
• 229920000858 Cyclodextrin Polymers 0.000 title claims description 194
• 238000011282 treatment Methods 0.000 title claims description 49
• HFHDHCJBZVLPGP-UHFFFAOYSA-N schardinger α-dextrin Chemical compound O1C(C(C2O)O)C(CO)OC2OC(C(C2O)O)C(CO)OC2OC(C(C2O)O)C(CO)OC2OC(C(O)C2O)C(CO)OC2OC(C(C2O)O)C(CO)OC2OC2C(O)C(O)C1OC2CO HFHDHCJBZVLPGP-UHFFFAOYSA-N 0.000 title abstract description 52
• -1 cyclodextrin compound Chemical class 0.000 claims description 141
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 36
• 150000003839 salts Chemical class 0.000 claims description 33
• 239000012453 solvate Substances 0.000 claims description 25
• 150000002148 esters Chemical class 0.000 claims description 24
• 201000010099 disease Diseases 0.000 claims description 22
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• 239000000546 pharmaceutical excipient Substances 0.000 claims description 11
• 208000008955 Mucolipidoses Diseases 0.000 claims description 7
• 206010053185 Glycogen storage disease type II Diseases 0.000 claims description 6
• 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 claims description 6
• ODLHGICHYURWBS-LKONHMLTSA-N trappsol cyclo Chemical compound CC(O)COC[C@H]([C@H]([C@@H]([C@H]1O)O)O[C@H]2O[C@@H]([C@@H](O[C@H]3O[C@H](COCC(C)O)[C@H]([C@@H]([C@H]3O)O)O[C@H]3O[C@H](COCC(C)O)[C@H]([C@@H]([C@H]3O)O)O[C@H]3O[C@H](COCC(C)O)[C@H]([C@@H]([C@H]3O)O)O[C@H]3O[C@H](COCC(C)O)[C@H]([C@@H]([C@H]3O)O)O3)[C@H](O)[C@H]2O)COCC(O)C)O[C@@H]1O[C@H]1[C@H](O)[C@@H](O)[C@@H]3O[C@@H]1COCC(C)O ODLHGICHYURWBS-LKONHMLTSA-N 0.000 claims description 6
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• 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 claims description 3
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• 238000000034 method Methods 0.000 abstract description 43
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• GVJHHUAWPYXKBD-UHFFFAOYSA-N (±)-α-Tocopherol Chemical compound OC1=C(C)C(C)=C2OC(CCCC(C)CCCC(C)CCCC(C)C)(C)CCC2=C1C GVJHHUAWPYXKBD-UHFFFAOYSA-N 0.000 abstract description 33
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• GZIFEOYASATJEH-VHFRWLAGSA-N δ-tocopherol Chemical compound OC1=CC(C)=C2O[C@@](CCC[C@H](C)CCC[C@H](C)CCCC(C)C)(C)CCC2=C1 GZIFEOYASATJEH-VHFRWLAGSA-N 0.000 description 88
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• 125000002887 hydroxy group Chemical group [H]O* 0.000 description 15
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• HFHDHCJBZVLPGP-RWMJIURBSA-N alpha-cyclodextrin Chemical compound OC[C@H]([C@H]([C@@H]([C@H]1O)O)O[C@H]2O[C@@H]([C@@H](O[C@H]3O[C@H](CO)[C@H]([C@@H]([C@H]3O)O)O[C@H]3O[C@H](CO)[C@H]([C@@H]([C@H]3O)O)O[C@H]3O[C@H](CO)[C@H]([C@@H]([C@H]3O)O)O3)[C@H](O)[C@H]2O)CO)O[C@@H]1O[C@H]1[C@H](O)[C@@H](O)[C@@H]3O[C@@H]1CO HFHDHCJBZVLPGP-RWMJIURBSA-N 0.000 description 13
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