JPWO2019169004A5 - - Google Patents

Info

Publication number

JPWO2019169004A5

JPWO2019169004A5 JP2020545159A JP2020545159A JPWO2019169004A5 JP WO2019169004 A5 JPWO2019169004 A5 JP WO2019169004A5 JP 2020545159 A JP2020545159 A JP 2020545159A JP 2020545159 A JP2020545159 A JP 2020545159A JP WO2019169004 A5 JPWO2019169004 A5 JP WO2019169004A5

Authority

JP

Japan

Prior art keywords

seq

acid sequence

protein

capsid

amino acid

Prior art date

2022-02-18

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
• Global Dossier
• Discuss

• 108090000623 proteins and genes Proteins 0.000 claims 96
• 235000018102 proteins Nutrition 0.000 claims 94
• 102000004169 proteins and genes Human genes 0.000 claims 94
• 150000001413 amino acids Chemical group 0.000 claims 93
• 241000702421 Dependoparvovirus Species 0.000 claims 48
• 150000007523 nucleic acids Chemical group 0.000 claims 47
• 210000000234 capsid Anatomy 0.000 claims 46
• 235000001014 amino acid Nutrition 0.000 claims 45
• 229940024606 amino acid Drugs 0.000 claims 45
• 108091028043 Nucleic acid sequence Proteins 0.000 claims 32
• 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 24
• 108090000565 Capsid Proteins Proteins 0.000 claims 23
• 102100023321 Ceruloplasmin Human genes 0.000 claims 23
• 239000002773 nucleotide Substances 0.000 claims 21
• 125000003729 nucleotide group Chemical group 0.000 claims 21
• 239000004471 Glycine Substances 0.000 claims 16
• 108020004707 nucleic acids Proteins 0.000 claims 14
• 102000039446 nucleic acids Human genes 0.000 claims 14
• 241001164825 Adeno-associated virus - 8 Species 0.000 claims 13
• 230000004048 modification Effects 0.000 claims 11
• 238000012986 modification Methods 0.000 claims 11
• 230000006240 deamidation Effects 0.000 claims 10
• 239000013598 vector Substances 0.000 claims 9
• 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 6
• CKLJMWTZIZZHCS-REOHCLBHSA-N L-aspartic acid Chemical compound OC(=O)[C@@H](N)CC(O)=O CKLJMWTZIZZHCS-REOHCLBHSA-N 0.000 claims 6
• 235000003704 aspartic acid Nutrition 0.000 claims 6
• OQFSQFPPLPISGP-UHFFFAOYSA-N beta-carboxyaspartic acid Natural products OC(=O)C(N)C(C(O)=O)C(O)=O OQFSQFPPLPISGP-UHFFFAOYSA-N 0.000 claims 6
• 238000004949 mass spectrometry Methods 0.000 claims 6
• 239000013647 rAAV8 vector Substances 0.000 claims 6
• 235000009582 asparagine Nutrition 0.000 claims 5
• 239000004220 glutamic acid Substances 0.000 claims 4
• DCXYFEDJOCDNAF-UHFFFAOYSA-N Asparagine Natural products OC(=O)C(N)CC(N)=O DCXYFEDJOCDNAF-UHFFFAOYSA-N 0.000 claims 3
• DCXYFEDJOCDNAF-REOHCLBHSA-N L-asparagine Chemical compound OC(=O)[C@@H](N)CC(N)=O DCXYFEDJOCDNAF-REOHCLBHSA-N 0.000 claims 3
• FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 claims 3
• QIVBCDIJIAJPQS-VIFPVBQESA-N L-tryptophane Chemical compound C1=CC=C2C(C[C@H](N)C(O)=O)=CNC2=C1 QIVBCDIJIAJPQS-VIFPVBQESA-N 0.000 claims 3
• 239000004472 Lysine Substances 0.000 claims 3
• KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims 3
• AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 claims 3
• 239000004473 Threonine Substances 0.000 claims 3
• QIVBCDIJIAJPQS-UHFFFAOYSA-N Tryptophan Natural products C1=CC=C2C(CC(N)C(O)=O)=CNC2=C1 QIVBCDIJIAJPQS-UHFFFAOYSA-N 0.000 claims 3
• 229960001230 asparagine Drugs 0.000 claims 3
• 150000001508 asparagines Chemical class 0.000 claims 3
• 229930182817 methionine Natural products 0.000 claims 3
• BZQFBWGGLXLEPQ-REOHCLBHSA-N phosphoserine Chemical compound OC(=O)[C@@H](N)COP(O)(O)=O BZQFBWGGLXLEPQ-REOHCLBHSA-N 0.000 claims 3
• 101150066583 rep gene Proteins 0.000 claims 3
• 229960004799 tryptophan Drugs 0.000 claims 3
• 108700019146 Transgenes Proteins 0.000 claims 2
• 239000002253 acid Substances 0.000 claims 2
• 238000004519 manufacturing process Methods 0.000 claims 2
• 238000004806 packaging method and process Methods 0.000 claims 2
• 238000013322 recombinant adeno-associated virus production system Methods 0.000 claims 2
• 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
• 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
• 239000006035 Tryptophane Substances 0.000 claims 1
• 238000004113 cell culture Methods 0.000 claims 1
• 238000012258 culturing Methods 0.000 claims 1
• 239000003085 diluting agent Substances 0.000 claims 1
• 239000003937 drug carrier Substances 0.000 claims 1
• ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Natural products OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 claims 1
• 238000000338 in vitro Methods 0.000 claims 1
• 210000003734 kidney Anatomy 0.000 claims 1
• 238000000034 method Methods 0.000 claims 1
• 239000000546 pharmaceutical excipient Substances 0.000 claims 1
• 239000013612 plasmid Substances 0.000 claims 1
• 230000026683 transduction Effects 0.000 claims 1
• 238000010361 transduction Methods 0.000 claims 1