AU2021313839A1 - Composition and method for treating eye diseases - Google Patents

Composition and method for treating eye diseases Download PDF

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Publication number
AU2021313839A1
AU2021313839A1 AU2021313839A AU2021313839A AU2021313839A1 AU 2021313839 A1 AU2021313839 A1 AU 2021313839A1 AU 2021313839 A AU2021313839 A AU 2021313839A AU 2021313839 A AU2021313839 A AU 2021313839A AU 2021313839 A1 AU2021313839 A1 AU 2021313839A1
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AU
Australia
Prior art keywords
promoter
sequence
seq
composition
polynucleotide
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Pending
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AU2021313839A
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English (en)
Inventor
Zhongdong SHI
Wei Zhao
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Ftgen Corp
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Ftgen Corp
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Filing date
Publication date
Priority claimed from CN202010706505.5A external-priority patent/CN113952473A/zh
Priority claimed from CN202010706658.XA external-priority patent/CN113952474A/zh
Application filed by Ftgen Corp filed Critical Ftgen Corp
Publication of AU2021313839A1 publication Critical patent/AU2021313839A1/en
Assigned to Frontera Therapeutics, Inc. reassignment Frontera Therapeutics, Inc. Request for Assignment Assignors: INSPIRAR LIMITED
Pending legal-status Critical Current

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    • 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

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  • 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)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
AU2021313839A 2020-07-21 2021-07-20 Composition and method for treating eye diseases Pending AU2021313839A1 (en)

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.X 2020-07-21
CN202010706505.5 2020-07-21
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
PCT/IB2021/000498 WO2022018516A1 (en) 2020-07-21 2021-07-20 Composition and method for treating eye diseases

Publications (1)

Publication Number Publication Date
AU2021313839A1 true AU2021313839A1 (en) 2023-03-23

Family

ID=79728536

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021313839A Pending AU2021313839A1 (en) 2020-07-21 2021-07-20 Composition and method for treating eye diseases

Country Status (7)

Country Link
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=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020006435A (es) 2017-12-19 2021-02-09 Akouos Inc Administracion de anticuerpos terapeuticos mediada por vector de virus adenoasociado (aav) en el oido interno.
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
JP2025506383A (ja) * 2022-02-02 2025-03-11 アドヴェラム バイオテクノロジーズ， インコーポレイテッド アフリベルセプトをコードするａａｖ２バリアントを使用して眼内血管新生疾患を処置する方法
JP2026509829A (ja) * 2023-03-10 2026-03-25 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 昆虫細胞における組換えアデノ随伴ウイルス（ｒＡＡＶ）産生
AU2024315262A1 (en) * 2023-08-03 2026-02-26 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
DE102004037611B4 (de) * 2004-08-03 2013-10-02 Geneart Ag Induzierbare Genexpression
WO2006015789A2 (de) * 2004-08-03 2006-02-16 Geneart Ag Verfahren zur modulation der genexpression durch änderung des cpg gehalts
CN105026433B (zh) * 2014-01-24 2018-10-19 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
DK3471780T3 (da) * 2016-06-16 2020-11-23 Adverum Biotechnologies Inc Behandling af amd med aav2-variant med 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
DK3717636T5 (da) * 2017-11-27 2024-08-26 4D Molecular Therapeutics Inc Adeno-associeret-virus-variantcapsider og anvendelse til inhibering af angiogenese
MX2020006435A (es) * 2017-12-19 2021-02-09 Akouos Inc Administracion de anticuerpos terapeuticos mediada por vector de virus adenoasociado (aav) en el oido interno.
CA3094465A1 (en) * 2018-04-03 2019-10-10 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
FI4027983T3 (fi) * 2019-09-11 2026-02-03 Adverum Biotechnologies Inc Silmän neovaskulaaristen sairauksien hoito afliberseptiä koodaavilla aav2-varianteilla
MX2023004377A (es) * 2020-10-16 2023-06-28 Gyroscope Therapeutics Ltd Acido nucleico que codifica una entidad anti-vegf y un regulador del complemento negativo y sus usos para el tratamiento de la degeneracion macular relacionada con la edad.
CA3210368A1 (en) * 2021-04-09 2022-10-13 Shengjiang Liu Compositions and methods for ocular transgene expression
KR20240004316A (ko) * 2021-04-27 2024-01-11 4디 몰레큘러 테라퓨틱스 아이엔씨. 혈관신생과 관련된 안구 질환의 치료를 위한 조성물 및 방법
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
US20230295243A1 (en) 2023-09-21
EP4185333A1 (en) 2023-05-31
JP2023540464A (ja) 2023-09-25
WO2022018516A1 (en) 2022-01-27
JP7842082B2 (ja) 2026-04-07
CA3186830A1 (en) 2022-01-27
CN116323949A (zh) 2023-06-23
EP4185333A4 (en) 2024-09-04

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PC1 Assignment before grant (sect. 113)

Owner name: FRONTERA THERAPEUTICS, INC.

Free format text: FORMER APPLICANT(S): INSPIRAR LIMITED

DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS: TO AMEND THE APPLICANT NAME TO FTGEN CORP