JP2020510447A5 - - Google Patents

Info

Publication number

JP2020510447A5

JP2020510447A5 JP2019571814A JP2019571814A JP2020510447A5 JP 2020510447 A5 JP2020510447 A5 JP 2020510447A5 JP 2019571814 A JP2019571814 A JP 2019571814A JP 2019571814 A JP2019571814 A JP 2019571814A JP 2020510447 A5 JP2020510447 A5 JP 2020510447A5

Authority

JP

Japan

Prior art keywords

aav vector

recombinant aav

composition

muscular dystrophy

muscle

Prior art date

2018-03-16

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.)

Granted

Links

• Espacenet
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• 239000013607 AAV vector Substances 0.000 claims description 31
• 239000000203 mixture Substances 0.000 claims description 27
• 201000006938 muscular dystrophy Diseases 0.000 claims description 17
• 210000003205 muscle Anatomy 0.000 claims description 14
• 108010059343 MM Form Creatine Kinase Proteins 0.000 claims description 8
• 239000003795 chemical substances by application Substances 0.000 claims description 8
• 238000000034 method Methods 0.000 claims description 8
• 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 6
• 239000003623 enhancer Substances 0.000 claims description 6
• 239000002773 nucleotide Substances 0.000 claims description 6
• 125000003729 nucleotide group Chemical group 0.000 claims description 6
• 238000001802 infusion Methods 0.000 claims description 5
• 238000002347 injection Methods 0.000 claims description 5
• 239000007924 injection Substances 0.000 claims description 5
• 238000002054 transplantation Methods 0.000 claims description 5
• 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 description 4
• 108010085238 Actins Proteins 0.000 claims description 4
• 108090000362 Lymphotoxin-beta Proteins 0.000 claims description 4
• 102000005604 Myosin Heavy Chains Human genes 0.000 claims description 4
• 108010084498 Myosin Heavy Chains Proteins 0.000 claims description 4
• 239000003814 drug Substances 0.000 claims description 3
• 238000010255 intramuscular injection Methods 0.000 claims description 3
• 238000010253 intravenous injection Methods 0.000 claims description 3
• 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 2
• 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 2
• 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 2
• 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 2
• 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 2
• 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 2
• 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 2
• 241000649045 Adeno-associated virus 10 Species 0.000 claims description 2
• 241000649046 Adeno-associated virus 11 Species 0.000 claims description 2
• 241000649047 Adeno-associated virus 12 Species 0.000 claims description 2
• 241000300529 Adeno-associated virus 13 Species 0.000 claims description 2
• 102000004420 Creatine Kinase Human genes 0.000 claims description 2
• 108010042126 Creatine kinase Proteins 0.000 claims description 2
• 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 2
• 206010021143 Hypoxia Diseases 0.000 claims description 2
• 108091027981 Response element Proteins 0.000 claims description 2
• 108010065729 Troponin I Proteins 0.000 claims description 2
• 108010051583 Ventricular Myosins Proteins 0.000 claims description 2
• 239000003470 adrenal cortex hormone Substances 0.000 claims description 2
• 230000000747 cardiac effect Effects 0.000 claims description 2
• RKHQGWMMUURILY-UHRZLXHJSA-N cortivazol Chemical compound C([C@H]1[C@@H]2C[C@H]([C@]([C@@]2(C)C[C@H](O)[C@@H]1[C@@]1(C)C2)(O)C(=O)COC(C)=O)C)=C(C)C1=CC1=C2C=NN1C1=CC=CC=C1 RKHQGWMMUURILY-UHRZLXHJSA-N 0.000 claims description 2
• 238000007918 intramuscular administration Methods 0.000 claims description 2
• 239000007927 intramuscular injection Substances 0.000 claims description 2
• 238000001990 intravenous administration Methods 0.000 claims description 2
• 210000000663 muscle cell Anatomy 0.000 claims description 2
• 230000002107 myocardial effect Effects 0.000 claims description 2
• 238000007911 parenteral administration Methods 0.000 claims description 2
• 238000012385 systemic delivery Methods 0.000 claims description 2
• 229940124597 therapeutic agent Drugs 0.000 claims description 2
• 239000013598 vector Substances 0.000 claims description 2
• 102100024108 Dystrophin Human genes 0.000 description 1
• 102000011856 Utrophin Human genes 0.000 description 1
• 108010075653 Utrophin Proteins 0.000 description 1
• 229940079593 drug Drugs 0.000 description 1