JP2018537089A5 - - Google Patents

Info

Publication number

JP2018537089A5

JP2018537089A5 JP2018524806A JP2018524806A JP2018537089A5 JP 2018537089 A5 JP2018537089 A5 JP 2018537089A5 JP 2018524806 A JP2018524806 A JP 2018524806A JP 2018524806 A JP2018524806 A JP 2018524806A JP 2018537089 A5 JP2018537089 A5 JP 2018537089A5

Authority

JP

Japan

Prior art keywords

polynucleotide

nucleotide sequence

seq

factor viii

aav

Prior art date

2016-11-11

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

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• 239000002157 polynucleotide Substances 0.000 claims description 67
• 102000040430 polynucleotide Human genes 0.000 claims description 67
• 108091033319 polynucleotide Proteins 0.000 claims description 67
• 239000002773 nucleotide Substances 0.000 claims description 42
• 125000003729 nucleotide group Chemical group 0.000 claims description 42
• 229920001184 polypeptide Polymers 0.000 claims description 42
• 108090000765 processed proteins & peptides Proteins 0.000 claims description 42
• 102000004196 processed proteins & peptides Human genes 0.000 claims description 42
• 108010054218 Factor VIII Proteins 0.000 claims description 34
• 102000001690 Factor VIII Human genes 0.000 claims description 34
• 229960000301 factor viii Drugs 0.000 claims description 34
• 241000702421 Dependoparvovirus Species 0.000 claims description 15
• 239000002245 particle Substances 0.000 claims description 13
• 238000000034 method Methods 0.000 claims description 7
• 102100026735 Coagulation factor VIII Human genes 0.000 claims description 5
• 201000003542 Factor VIII deficiency Diseases 0.000 claims description 5
• 208000009292 Hemophilia A Diseases 0.000 claims description 5
• 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims description 5
• 210000004185 liver Anatomy 0.000 claims description 4
• 238000004519 manufacturing process Methods 0.000 claims description 4
• 230000002463 transducing effect Effects 0.000 claims description 3
• 238000011144 upstream manufacturing Methods 0.000 claims description 2
• 239000008194 pharmaceutical composition Substances 0.000 claims 1
• 125000003275 alpha amino acid group Chemical group 0.000 description 8
• 102000004961 Furin Human genes 0.000 description 3
• 108090001126 Furin Proteins 0.000 description 3
• 238000003776 cleavage reaction Methods 0.000 description 3
• 230000007017 scission Effects 0.000 description 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 description 2
• 239000003814 drug Substances 0.000 description 2
• 150000001413 amino acids Chemical class 0.000 description 1
• 238000001415 gene therapy Methods 0.000 description 1