JP2020506695A5 - - Google Patents

Info

Publication number

JP2020506695A5

JP2020506695A5 JP2019540667A JP2019540667A JP2020506695A5 JP 2020506695 A5 JP2020506695 A5 JP 2020506695A5 JP 2019540667 A JP2019540667 A JP 2019540667A JP 2019540667 A JP2019540667 A JP 2019540667A JP 2020506695 A5 JP2020506695 A5 JP 2020506695A5

Authority

JP

Japan

Prior art keywords

composition

vector

raav

recombinant

nucleic acid

Prior art date

2018-01-30

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
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• 239000013598 vector Substances 0.000 claims description 22
• 150000007523 nucleic acids Chemical class 0.000 claims description 16
• 102000039446 nucleic acids Human genes 0.000 claims description 16
• 108020004707 nucleic acids Proteins 0.000 claims description 16
• 238000000034 method Methods 0.000 claims description 13
• 241000700605 Viruses Species 0.000 claims description 9
• 239000002773 nucleotide Substances 0.000 claims description 8
• 125000003729 nucleotide group Chemical group 0.000 claims description 8
• 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 6
• 241000725303 Human immunodeficiency virus Species 0.000 claims description 6
• 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 4
• 241000713666 Lentivirus Species 0.000 claims description 4
• 208000005516 glycogen storage disease Ib Diseases 0.000 claims description 4
• 208000011476 Glycogen storage disease due to glucose-6-phosphatase deficiency type Ib Diseases 0.000 claims description 3
• 206010018464 Glycogen storage disease type I Diseases 0.000 claims description 3
• 239000013607 AAV vector Substances 0.000 claims description 2
• 241000702421 Dependoparvovirus Species 0.000 claims description 2
• 239000003937 drug carrier Substances 0.000 claims description 2
• 208000007345 glycogen storage disease Diseases 0.000 claims description 2
• 238000001990 intravenous administration Methods 0.000 claims description 2
• 239000002245 particle Substances 0.000 claims description 2
• 239000013647 rAAV8 vector Substances 0.000 claims description 2