CN116457373A - 用于眼部适应症的载体化TNF-α拮抗剂 - Google Patents

用于眼部适应症的载体化TNF-α拮抗剂 Download PDF

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Publication number
CN116457373A
CN116457373A CN202180072567.8A CN202180072567A CN116457373A CN 116457373 A CN116457373 A CN 116457373A CN 202180072567 A CN202180072567 A CN 202180072567A CN 116457373 A CN116457373 A CN 116457373A
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China
Prior art keywords
serotype
aav
tnfα
cells
seq
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CN202180072567.8A
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Chinese (zh)
Inventor
X·王
D·麦克多加尔德
J·布鲁德尔
Y·刘
O·达诺斯
C·乔
W-H·李
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Bio Regeneration Co ltd
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Bio Regeneration Co ltd
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1793—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/715—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
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    • C07K16/24—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
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    • C12N15/09—Recombinant DNA-technology
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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CN202180072567.8A 2020-10-29 2021-10-29 用于眼部适应症的载体化TNF-α拮抗剂 Pending CN116457373A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063107415P 2020-10-29 2020-10-29
US63/107,415 2020-10-29
PCT/US2021/057380 WO2022094295A1 (en) 2020-10-29 2021-10-29 Vectorized tnf-alpha antagonists for ocular indications

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CN116457373A true CN116457373A (zh) 2023-07-18

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US (1) US20240384292A1 (https=)
EP (1) EP4237453A1 (https=)
JP (1) JP2023548145A (https=)
KR (1) KR20230098176A (https=)
CN (1) CN116457373A (https=)
AU (1) AU2021369833A1 (https=)
CA (1) CA3196964A1 (https=)
IL (1) IL302282A (https=)
MX (1) MX2023004843A (https=)
WO (1) WO2022094295A1 (https=)

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WO2024238853A1 (en) * 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy

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