JPWO2019138137A5 - - Google Patents

Info

Publication number

JPWO2019138137A5

JPWO2019138137A5 JP2020538949A JP2020538949A JPWO2019138137A5 JP WO2019138137 A5 JPWO2019138137 A5 JP WO2019138137A5 JP 2020538949 A JP2020538949 A JP 2020538949A JP 2020538949 A JP2020538949 A JP 2020538949A JP WO2019138137 A5 JPWO2019138137 A5 JP WO2019138137A5

Authority

JP

Japan

Prior art keywords

polypeptide

pharmaceutical composition

seq

nucleic acid

composition according

Prior art date

2022-01-25

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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• 108090000765 processed proteins & peptides Proteins 0.000 claims description 120
• 229920001184 polypeptide Polymers 0.000 claims description 119
• 102000004196 processed proteins & peptides Human genes 0.000 claims description 119
• 239000008194 pharmaceutical composition Substances 0.000 claims description 32
• 102000039446 nucleic acids Human genes 0.000 claims description 26
• 108020004707 nucleic acids Proteins 0.000 claims description 26
• 150000007523 nucleic acids Chemical class 0.000 claims description 26
• 201000010099 disease Diseases 0.000 claims description 20
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 20
• 208000002780 macular degeneration Diseases 0.000 claims description 20
• 206010064930 age-related macular degeneration Diseases 0.000 claims description 16
• 150000001413 amino acids Chemical class 0.000 claims description 16
• 239000013598 vector Substances 0.000 claims description 15
• 125000003275 alpha amino acid group Chemical group 0.000 claims description 12
• 230000037361 pathway Effects 0.000 claims description 12
• 230000003248 secreting effect Effects 0.000 claims description 12
• 230000000295 complement effect Effects 0.000 claims description 10
• 102000003689 Complement Factor I Human genes 0.000 claims description 8
• 108090000044 Complement Factor I Proteins 0.000 claims description 8
• 210000001775 bruch membrane Anatomy 0.000 claims description 8
• 208000030533 eye disease Diseases 0.000 claims description 8
• 238000004519 manufacturing process Methods 0.000 claims description 7
• 238000000034 method Methods 0.000 claims description 7
• 230000002265 prevention Effects 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
• 238000006243 chemical reaction Methods 0.000 claims description 4
• 238000003776 cleavage reaction Methods 0.000 claims description 4
• 102000006834 complement receptors Human genes 0.000 claims description 4
• 108010047295 complement receptors Proteins 0.000 claims description 4
• 239000003814 drug Substances 0.000 claims description 4
• 229940079593 drug Drugs 0.000 claims description 4
• 230000000694 effects Effects 0.000 claims description 4
• 230000007017 scission Effects 0.000 claims description 4
• 208000002691 Choroiditis Diseases 0.000 claims description 2
• 208000008069 Geographic Atrophy Diseases 0.000 claims description 2
• 208000010412 Glaucoma Diseases 0.000 claims description 2
• 208000003971 Posterior uveitis Diseases 0.000 claims description 2
• 239000002671 adjuvant Substances 0.000 claims description 2
• 230000033115 angiogenesis Effects 0.000 claims description 2
• 230000002491 angiogenic effect Effects 0.000 claims description 2
• 230000001363 autoimmune Effects 0.000 claims description 2
• 238000012258 culturing Methods 0.000 claims description 2
• 206010012601 diabetes mellitus Diseases 0.000 claims description 2
• 239000003085 diluting agent Substances 0.000 claims description 2
• 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
• 230000002028 premature Effects 0.000 claims description 2
• 239000000790 retinal pigment Substances 0.000 claims description 2
• FVAUCKIRQBBSSJ-UHFFFAOYSA-M sodium iodide Chemical compound [Na+].[I-] FVAUCKIRQBBSSJ-UHFFFAOYSA-M 0.000 description 6
• 241000699670 Mus sp. Species 0.000 description 3
• 201000007737 Retinal degeneration Diseases 0.000 description 2
• 230000004258 retinal degeneration Effects 0.000 description 2
• 235000009518 sodium iodide Nutrition 0.000 description 2
• 239000013607 AAV vector Substances 0.000 description 1
• 241000702423 Adeno-associated virus - 2 Species 0.000 description 1
• 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 1
• 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 1
• 239000003795 chemical substances by application Substances 0.000 description 1
• 230000024203 complement activation Effects 0.000 description 1
• 239000002299 complementary DNA Substances 0.000 description 1
• 230000007850 degeneration Effects 0.000 description 1
• 230000001419 dependent effect Effects 0.000 description 1
• 238000002347 injection Methods 0.000 description 1
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• 210000003583 retinal pigment epithelium Anatomy 0.000 description 1