CA3186830A1 - Composition and method for treating eye diseases - Google Patents

Composition and method for treating eye diseases

Info

Publication number
CA3186830A1
CA3186830A1 CA3186830A CA3186830A CA3186830A1 CA 3186830 A1 CA3186830 A1 CA 3186830A1 CA 3186830 A CA3186830 A CA 3186830A CA 3186830 A CA3186830 A CA 3186830A CA 3186830 A1 CA3186830 A1 CA 3186830A1
Authority
CA
Canada
Prior art keywords
promoter
sequence
seq
composition
polynucleotide
Prior art date
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
Application number
CA3186830A
Other languages
English (en)
French (fr)
Inventor
Zhongdong SHI
Wei Zhao
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Frontera Therapeutics Inc
Original Assignee
Inspirar Ltd
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Priority claimed from CN202010706658.XA external-priority patent/CN113952474A/zh
Priority claimed from CN202010706505.5A external-priority patent/CN113952473A/zh
Application filed by Inspirar Ltd filed Critical Inspirar Ltd
Publication of CA3186830A1 publication Critical patent/CA3186830A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • 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
    • 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
    • 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
    • C07K14/705—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Emergency Medicine (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Obesity (AREA)
  • Endocrinology (AREA)
  • Hematology (AREA)
CA3186830A 2020-07-21 2021-07-20 Composition and method for treating eye diseases Pending CA3186830A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
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
CN202010706505.5 2020-07-21
CN202010706658.X 2020-07-21
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
PCT/IB2021/000498 WO2022018516A1 (en) 2020-07-21 2021-07-20 Composition and method for treating eye diseases

Publications (1)

Publication Number Publication Date
CA3186830A1 true CA3186830A1 (en) 2022-01-27

Family

ID=79728536

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3186830A Pending CA3186830A1 (en) 2020-07-21 2021-07-20 Composition and method for treating eye diseases

Country Status (7)

Country Link
US (1) US20230295243A1 (enExample)
EP (1) EP4185333A4 (enExample)
JP (1) JP2023540464A (enExample)
CN (1) CN116323949A (enExample)
AU (1) AU2021313839A1 (enExample)
CA (1) CA3186830A1 (enExample)
WO (1) WO2022018516A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021506861A (ja) 2017-12-19 2021-02-22 アコーオス インコーポレイテッド 内耳への治療用抗体のａａｖ媒介送達
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
CA3243256A1 (en) * 2022-02-02 2023-08-10 Adverum Biotechnologies Inc Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
AU2024234810A1 (en) * 2023-03-10 2025-09-04 Adverum Biotechnologies, Inc. Recombinant adeno-associated virus (raav) production in insect cells
WO2025030169A2 (en) * 2023-08-03 2025-02-06 Arcum Vision Inc. Enhanced formulation of polymyxin b/ trimethoprim and rifampin and methods for ophthalmic uses

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK1776460T3 (da) * 2004-08-03 2010-04-12 Geneart Ag Fremgangsmåde til modulering af genekspression ved ændring af CpG-indholdet
DE102004037611B4 (de) * 2004-08-03 2013-10-02 Geneart Ag Induzierbare Genexpression
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
CA2977355A1 (en) * 2015-02-20 2016-08-25 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases
HUE051953T2 (hu) * 2016-06-16 2021-04-28 Adverum Biotechnologies Inc Idõskori makula degeneráció kezelése AAV2 variáns és aflibercept alkalmazásával
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
AU2018372235B9 (en) * 2017-11-27 2022-03-10 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
JP2021506861A (ja) * 2017-12-19 2021-02-22 アコーオス インコーポレイテッド 内耳への治療用抗体のａａｖ媒介送達
EP3773743A1 (en) * 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2020102645A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease
WO2021050649A1 (en) * 2019-09-11 2021-03-18 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
AU2021362770A1 (en) * 2020-10-16 2022-12-08 Gyroscope Therapeutics Limited Nucleic acid encoding an anti-VEGF entity and a negative complement regulator and uses thereof for the treatment of age-related macular degeneration
JP2024516128A (ja) * 2021-04-09 2024-04-12 アビラマックス・バイオファーマ・インコーポレイテッド 眼での導入遺伝子発現のための組成物および方法
WO2022232178A1 (en) * 2021-04-27 2022-11-03 4D Molecular Therapeutics Inc. Compositions and methods for treatment of ocular disease associated with angiogenesis
MX2023014041A (es) * 2021-05-28 2023-12-15 Shanghai Regenelead Therapies Co Ltd Virus adenoasociado recombinante que tiene una capsida variante y su aplicacion.

Also Published As

Publication number Publication date
JP2023540464A (ja) 2023-09-25
US20230295243A1 (en) 2023-09-21
AU2021313839A1 (en) 2023-03-23
EP4185333A1 (en) 2023-05-31
EP4185333A4 (en) 2024-09-04
CN116323949A (zh) 2023-06-23
WO2022018516A1 (en) 2022-01-27

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