CN119585299A - 编码抗vegf实体和补体负调节剂的核酸及其用于年龄相关性黄斑变性的治疗的用途 - Google Patents

编码抗vegf实体和补体负调节剂的核酸及其用于年龄相关性黄斑变性的治疗的用途 Download PDF

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Publication number
CN119585299A
CN119585299A CN202180039494.2A CN202180039494A CN119585299A CN 119585299 A CN119585299 A CN 119585299A CN 202180039494 A CN202180039494 A CN 202180039494A CN 119585299 A CN119585299 A CN 119585299A
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China
Prior art keywords
complement
vector
vegf
polynucleotide
nucleotide sequence
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Pending
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CN202180039494.2A
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English (en)
Chinese (zh)
Inventor
J·乔伊
J·埃斯特韦-鲁德
L·塔姆
S·埃利斯
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Gilo Kepu Treatment Co ltd
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Gilo Kepu Treatment Co ltd
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Priority claimed from GBGB2016463.8A external-priority patent/GB202016463D0/en
Priority claimed from GBGB2104148.8A external-priority patent/GB202104148D0/en
Application filed by Gilo Kepu Treatment Co ltd filed Critical Gilo Kepu Treatment Co ltd
Publication of CN119585299A publication Critical patent/CN119585299A/zh
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1725—Complement proteins, e.g. anaphylatoxin, C3a or C5a
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K38/179—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • 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
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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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
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    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C07K14/472—Complement proteins, e.g. anaphylatoxin, C3a, C5a
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
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    • C12N15/09—Recombinant DNA-technology
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/32—Fusion polypeptide fusions with soluble part of a cell surface receptor, "decoy receptors"
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    • C12N2750/00011—Details
    • 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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CN202180039494.2A 2020-10-16 2021-10-18 编码抗vegf实体和补体负调节剂的核酸及其用于年龄相关性黄斑变性的治疗的用途 Pending CN119585299A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GBGB2016463.8A GB202016463D0 (en) 2020-10-16 2020-10-16 Product
GB2016463.8 2020-10-16
GBGB2104148.8A GB202104148D0 (en) 2021-03-24 2021-03-24 Product
GB2104148.8 2021-03-24
PCT/GB2021/052689 WO2022079453A1 (en) 2020-10-16 2021-10-18 Nucleic acid encoding an anti-vegf entity and a negative complement regulator and uses thereof for the treatment of age-related macular degeneration

Publications (1)

Publication Number Publication Date
CN119585299A true CN119585299A (zh) 2025-03-07

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ID=78463555

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CN202180039494.2A Pending CN119585299A (zh) 2020-10-16 2021-10-18 编码抗vegf实体和补体负调节剂的核酸及其用于年龄相关性黄斑变性的治疗的用途

Country Status (18)

Country Link
US (1) US20230212275A1 (https=)
EP (1) EP4229076A1 (https=)
JP (1) JP2023545229A (https=)
KR (1) KR20230088630A (https=)
CN (1) CN119585299A (https=)
AU (1) AU2021362770A1 (https=)
CA (1) CA3182337A1 (https=)
CL (1) CL2023001072A1 (https=)
CO (1) CO2023005981A2 (https=)
CR (1) CR20230202A (https=)
DO (1) DOP2023000076A (https=)
EC (1) ECSP23034907A (https=)
IL (1) IL298527A (https=)
JO (1) JOP20230076A1 (https=)
MX (1) MX2023004377A (https=)
PE (1) PE20231747A1 (https=)
PH (1) PH12023551015A1 (https=)
WO (1) WO2022079453A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201800620D0 (en) 2018-01-15 2018-02-28 Univ Manchester C3b Binding Polypeptide
GB201821089D0 (en) 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Codon-optimised complement factor I
JP7842082B2 (ja) * 2020-07-21 2026-04-07 エフティージェン コーポレーション 眼疾患を処置するための組成物および方法
WO2025083169A1 (en) * 2023-10-17 2025-04-24 Tenpoint Therapeutics Limited Combination of a vegf inhibitor and a complement pathway inhibitor for treating ocular disorders
KR20260004635A (ko) * 2024-07-01 2026-01-09 삼성바이오에피스 주식회사 애플리버셉트를 암호화하는 코돈-최적화된 폴리뉴클레오티드를 포함하는 발현 카세트 및 이를 포함하는 rAAV 생산용 플라스미드

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2191001T3 (en) 2007-04-09 2016-09-19 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
BR112014013205A2 (pt) * 2011-12-01 2020-10-27 Protevobio, Inc. proteína de fusão, seu uso e seu método de produção, composição farmacêutica, ácido nucleico, e kit
JP7046828B2 (ja) * 2016-04-15 2022-04-04 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 滲出型加齢性黄斑変性の治療のための組成物
AU2018350990A1 (en) * 2017-10-18 2020-05-21 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap
CN111788311A (zh) * 2017-10-20 2020-10-16 双子治疗公司 用于治疗年龄相关性黄斑变性的组合物和方法
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
GB201821089D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Codon-optimised complement factor I
GB201821082D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Combination of complement factors i and h, and vector encoding thereof
EP4048318A4 (en) * 2019-10-23 2023-11-22 Gemini Therapeutics Sub, Inc. METHOD FOR TREATING PATIENTS WITH CFH MUTATIONS USING RECOMBINANT CFH PROTEINS

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IL298527A (en) 2023-01-01
PH12023551015A1 (en) 2024-02-05
PE20231747A1 (es) 2023-11-06
JP2023545229A (ja) 2023-10-27
KR20230088630A (ko) 2023-06-20
ECSP23034907A (es) 2023-06-30
CR20230202A (es) 2023-06-13
MX2023004377A (es) 2023-06-28
DOP2023000076A (es) 2023-06-15
JOP20230076A1 (ar) 2023-04-16
US20230212275A1 (en) 2023-07-06
CL2023001072A1 (es) 2023-12-01
WO2022079453A1 (en) 2022-04-21
CA3182337A1 (en) 2022-04-21
AU2021362770A1 (en) 2022-12-08
CO2023005981A2 (es) 2023-05-29
EP4229076A1 (en) 2023-08-23

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