CN116323949A - 用于治疗眼部疾病的组合物和方法 - Google Patents

用于治疗眼部疾病的组合物和方法 Download PDF

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CN116323949A
CN116323949A CN202180064547.6A CN202180064547A CN116323949A CN 116323949 A CN116323949 A CN 116323949A CN 202180064547 A CN202180064547 A CN 202180064547A CN 116323949 A CN116323949 A CN 116323949A
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sequence
seq
polynucleotide
composition
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施中东
赵巍
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Fangtuo Biotechnology Co ltd
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Fangtuo Biotechnology Co ltd
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Priority claimed from CN202010706505.5A external-priority patent/CN113952473A/zh
Priority claimed from CN202010706658.XA external-priority patent/CN113952474A/zh
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Publication of CN116323949A publication Critical patent/CN116323949A/zh
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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
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    • C12N15/86—Viral vectors
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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    • C07—ORGANIC CHEMISTRY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • 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
    • A01K2207/00—Modified animals
    • A01K2207/30—Animals modified by surgical methods
    • 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
    • A01K2267/035—Animal model for multifactorial diseases
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/075—Adenoviridae
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • 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
    • C07K14/4703—Inhibitors; Suppressors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Microbiology (AREA)
  • Epidemiology (AREA)
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  • Gastroenterology & Hepatology (AREA)
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CN202180064547.6A 2020-07-21 2021-07-20 用于治疗眼部疾病的组合物和方法 Pending CN116323949A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
CN202010706505.5A CN113952473A (zh) 2020-07-21 2020-07-21 用于治疗眼部疾病的组合物和方法
CN202010706658.XA CN113952474A (zh) 2020-07-21 2020-07-21 用于治疗眼部疾病的组合物和方法
CN202010706658X 2020-07-21
CN2020107065055 2020-07-21
PCT/IB2021/000498 WO2022018516A1 (en) 2020-07-21 2021-07-20 Composition and method for treating eye diseases

Publications (1)

Publication Number Publication Date
CN116323949A true CN116323949A (zh) 2023-06-23

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CN202180064547.6A Pending CN116323949A (zh) 2020-07-21 2021-07-20 用于治疗眼部疾病的组合物和方法

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US (1) US20230295243A1 (https=)
EP (1) EP4185333A4 (https=)
JP (1) JP7842082B2 (https=)
CN (1) CN116323949A (https=)
AU (1) AU2021313839A1 (https=)
CA (1) CA3186830A1 (https=)
WO (1) WO2022018516A1 (https=)

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* Cited by examiner, † Cited by third party
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EP3727468A4 (en) 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED THERAPEUTIC ANTIBODIES DELIVERY TO THE INTERNAL EAR
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
US20250137011A1 (en) * 2022-02-02 2025-05-01 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
CN120826472A (zh) * 2023-03-10 2025-10-21 阿德夫拉姆生物技术股份有限公司 昆虫细胞中重组腺相关病毒（rAAV）的生产
WO2025030169A2 (en) * 2023-08-03 2025-02-06 Arcum Vision Inc. Enhanced formulation of polymyxin b/ trimethoprim and rifampin and methods for ophthalmic uses

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Publication number Priority date Publication date Assignee Title
DE102004037611B4 (de) * 2004-08-03 2013-10-02 Geneart Ag Induzierbare Genexpression
ATE453716T1 (de) * 2004-08-03 2010-01-15 Geneart Ag Verfahren zur modulation der genexpression durch änderung des cpg gehalts
WO2015109898A1 (zh) * 2014-01-24 2015-07-30 上海恒瑞医药有限公司 VEGF与PDGFRβ双特异性融合蛋白及其用途
AU2016219789B2 (en) * 2015-02-20 2021-03-25 Fondazione Telethon Methods and compositions for treating genetic eye diseases
ES2840059T3 (es) * 2016-06-16 2021-07-06 Adverum Biotechnologies Inc Tratamiento de la DMRE mediante el uso de la variante AAV2 con aflibercept
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
CN111770999A (zh) 2017-11-27 2020-10-13 4D分子治疗有限公司 腺相关病毒变体衣壳和用于抑制血管生成的应用
EP3727468A4 (en) * 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED THERAPEUTIC ANTIBODIES DELIVERY TO THE INTERNAL EAR
MX2020010465A (es) * 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
JP2022513067A (ja) 2018-11-16 2022-02-07 アスクレピオス バイオファーマシューティカル， インコーポレイテッド ポンペ病を処置するための治療用アデノ随伴ウイルス
HRP20260137T1 (hr) 2019-09-11 2026-03-13 Adverum Biotechnologies, Inc. Liječenje očnih neovaskularnih bolesti upotrebom aav2 varijanti koje kodiraju aflibercept
CR20230202A (es) * 2020-10-16 2023-06-13 Gyroscope Therapeutics Ltd Ácido nucleico que codifica una entidad anti-vegf y un regulador del complemento negativo y sus usos para el tratamiento de la degeneración macular relacionada con la edad
BR112023020490A2 (pt) * 2021-04-09 2023-11-21 Avirmax Biopharma Inc Composições e métodos para expressão de transgenes oculares
IL307416A (en) * 2021-04-27 2023-12-01 4D Molecular Therapeutics Inc Preparations and methods for the treatment of eye disease related to the formation of blood vessels
US20240252679A1 (en) * 2021-05-28 2024-08-01 Shanghai Regenelead Therapies Co., Ltd. Recombinant adeno-associated virus having variant capsid, and application thereof

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Publication number Publication date
EP4185333A4 (en) 2024-09-04
EP4185333A1 (en) 2023-05-31
AU2021313839A1 (en) 2023-03-23
WO2022018516A1 (en) 2022-01-27
JP7842082B2 (ja) 2026-04-07
US20230295243A1 (en) 2023-09-21
JP2023540464A (ja) 2023-09-25
CA3186830A1 (en) 2022-01-27

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