JP2020526514A5 - - Google Patents

Info

Publication number

JP2020526514A5

JP2020526514A5 JP2020500131A JP2020500131A JP2020526514A5 JP 2020526514 A5 JP2020526514 A5 JP 2020526514A5 JP 2020500131 A JP2020500131 A JP 2020500131A JP 2020500131 A JP2020500131 A JP 2020500131A JP 2020526514 A5 JP2020526514 A5 JP 2020526514A5

Authority

JP

Japan

Prior art keywords

immunosuppressant

composition

use according

months

sequence

Prior art date

2018-07-05

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.)

Pending

Links

• Espacenet
• Global Dossier
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• 239000003018 immunosuppressive agent Substances 0.000 claims 36
• 239000000203 mixture Substances 0.000 claims 26
• 229960003444 immunosuppressant agent Drugs 0.000 claims 25
• 230000001861 immunosuppressant effect Effects 0.000 claims 22
• 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 claims 12
• 102000056929 human IDUA Human genes 0.000 claims 11
• 229940125721 immunosuppressive agent Drugs 0.000 claims 11
• 150000003431 steroids Chemical class 0.000 claims 11
• 239000003120 macrolide antibiotic agent Substances 0.000 claims 8
• 229940041033 macrolides Drugs 0.000 claims 8
• 239000013598 vector Substances 0.000 claims 7
• 239000013607 AAV vector Substances 0.000 claims 6
• 210000001744 T-lymphocyte Anatomy 0.000 claims 6
• 230000000340 anti-metabolite Effects 0.000 claims 6
• 229940100197 antimetabolite Drugs 0.000 claims 6
• 239000002256 antimetabolite Substances 0.000 claims 6
• 239000003862 glucocorticoid Substances 0.000 claims 6
• 239000003112 inhibitor Substances 0.000 claims 6
• RTGDFNSFWBGLEC-SYZQJQIISA-N mycophenolate mofetil Chemical compound COC1=C(C)C=2COC(=O)C=2C(O)=C1C\C=C(/C)CCC(=O)OCCN1CCOCC1 RTGDFNSFWBGLEC-SYZQJQIISA-N 0.000 claims 6
• 229960004866 mycophenolate mofetil Drugs 0.000 claims 6
• 229940037128 systemic glucocorticoids Drugs 0.000 claims 6
• 239000003966 growth inhibitor Substances 0.000 claims 5
• 150000007523 nucleic acids Chemical class 0.000 claims 5
• ZAHRKKWIAAJSAO-UHFFFAOYSA-N rapamycin Natural products COCC(O)C(=C/C(C)C(=O)CC(OC(=O)C1CCCCN1C(=O)C(=O)C2(O)OC(CC(OC)C(=CC=CC=CC(C)CC(C)C(=O)C)C)CCC2C)C(C)CC3CCC(O)C(C3)OC)C ZAHRKKWIAAJSAO-UHFFFAOYSA-N 0.000 claims 5
• QFJCIRLUMZQUOT-HPLJOQBZSA-N sirolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 QFJCIRLUMZQUOT-HPLJOQBZSA-N 0.000 claims 5
• 229960002930 sirolimus Drugs 0.000 claims 5
• QJJXYPPXXYFBGM-LFZNUXCKSA-N Tacrolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1\C=C(/C)[C@@H]1[C@H](C)[C@@H](O)CC(=O)[C@H](CC=C)/C=C(C)/C[C@H](C)C[C@H](OC)[C@H]([C@H](C[C@H]2C)OC)O[C@@]2(O)C(=O)C(=O)N2CCCC[C@H]2C(=O)O1 QJJXYPPXXYFBGM-LFZNUXCKSA-N 0.000 claims 4
• 238000009472 formulation Methods 0.000 claims 4
• 108020004707 nucleic acids Proteins 0.000 claims 4
• 102000039446 nucleic acids Human genes 0.000 claims 4
• 229960001967 tacrolimus Drugs 0.000 claims 4
• QJJXYPPXXYFBGM-SHYZHZOCSA-N tacrolimus Natural products CO[C@H]1C[C@H](CC[C@@H]1O)C=C(C)[C@H]2OC(=O)[C@H]3CCCCN3C(=O)C(=O)[C@@]4(O)O[C@@H]([C@H](C[C@H]4C)OC)[C@@H](C[C@H](C)CC(=C[C@@H](CC=C)C(=O)C[C@H](O)[C@H]2C)C)OC QJJXYPPXXYFBGM-SHYZHZOCSA-N 0.000 claims 4
• 108091026890 Coding region Proteins 0.000 claims 3
• 238000002347 injection Methods 0.000 claims 3
• 239000007924 injection Substances 0.000 claims 3
• FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 2
• 150000001413 amino acids Chemical class 0.000 claims 2
• 238000001802 infusion Methods 0.000 claims 2
• 238000007913 intrathecal administration Methods 0.000 claims 2
• 238000001990 intravenous administration Methods 0.000 claims 2
• 238000011282 treatment Methods 0.000 claims 2
• 102000004190 Enzymes Human genes 0.000 claims 1
• 108090000790 Enzymes Proteins 0.000 claims 1
• 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
• 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims 1
• 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 claims 1
• 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
• CBPNZQVSJQDFBE-FUXHJELOSA-N Temsirolimus Chemical compound C1C[C@@H](OC(=O)C(C)(CO)CO)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 CBPNZQVSJQDFBE-FUXHJELOSA-N 0.000 claims 1
• 210000004369 blood Anatomy 0.000 claims 1
• 239000008280 blood Substances 0.000 claims 1
• 210000000133 brain stem Anatomy 0.000 claims 1
• 210000000234 capsid Anatomy 0.000 claims 1
• 238000002648 combination therapy Methods 0.000 claims 1
• 239000003246 corticosteroid Substances 0.000 claims 1
• 229960001334 corticosteroids Drugs 0.000 claims 1
• 238000011262 co‐therapy Methods 0.000 claims 1
• 239000000824 cytostatic agent Substances 0.000 claims 1
• 230000001085 cytostatic effect Effects 0.000 claims 1
• 238000002650 immunosuppressive therapy Methods 0.000 claims 1
• 239000002773 nucleotide Substances 0.000 claims 1
• 125000003729 nucleotide group Chemical group 0.000 claims 1
• 230000001105 regulatory effect Effects 0.000 claims 1
• 239000000725 suspension Substances 0.000 claims 1
• 229960000235 temsirolimus Drugs 0.000 claims 1
• QFJCIRLUMZQUOT-UHFFFAOYSA-N temsirolimus Natural products C1CC(O)C(OC)CC1CC(C)C1OC(=O)C2CCCCN2C(=O)C(=O)C(O)(O2)C(C)CCC2CC(OC)C(C)=CC=CC=CC(C)CC(C)C(=O)C(OC)C(O)C(C)=CC(C)C(=O)C1 QFJCIRLUMZQUOT-UHFFFAOYSA-N 0.000 claims 1
• 238000011285 therapeutic regimen Methods 0.000 claims 1