JP7842082B2 - 眼疾患を処置するための組成物および方法 - Google Patents

眼疾患を処置するための組成物および方法

Info

Publication number
JP7842082B2
JP7842082B2 JP2023512663A JP2023512663A JP7842082B2 JP 7842082 B2 JP7842082 B2 JP 7842082B2 JP 2023512663 A JP2023512663 A JP 2023512663A JP 2023512663 A JP2023512663 A JP 2023512663A JP 7842082 B2 JP7842082 B2 JP 7842082B2
Authority
JP
Japan
Prior art keywords
sequence
promoter
aav
cells
seq
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.)
Active
Application number
JP2023512663A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2022018516A5 (https=
JP2023540464A (ja
JP2023540464A5 (https=
Inventor
シー，ジョンドン
チャオ，ウェイ
Original Assignee
エフティージェン コーポレーション
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 CN202010706505.5A external-priority patent/CN113952473A/zh
Priority claimed from CN202010706658.XA external-priority patent/CN113952474A/zh
Application filed by エフティージェン コーポレーション filed Critical エフティージェン コーポレーション
Publication of JP2023540464A publication Critical patent/JP2023540464A/ja
Publication of JPWO2022018516A5 publication Critical patent/JPWO2022018516A5/ja
Publication of JP2023540464A5 publication Critical patent/JP2023540464A5/ja
Application granted granted Critical
Publication of JP7842082B2 publication Critical patent/JP7842082B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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)
  • 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)
JP2023512663A 2020-07-21 2021-07-20 眼疾患を処置するための組成物および方法 Active JP7842082B2 (ja)

Applications Claiming Priority (3)

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

Publications (4)

Publication Number Publication Date
JP2023540464A JP2023540464A (ja) 2023-09-25
JPWO2022018516A5 JPWO2022018516A5 (https=) 2024-07-26
JP2023540464A5 JP2023540464A5 (https=) 2024-07-26
JP7842082B2 true JP7842082B2 (ja) 2026-04-07

Family

ID=79728536

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023512663A Active JP7842082B2 (ja) 2020-07-21 2021-07-20 眼疾患を処置するための組成物および方法

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

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008507986A (ja) 2004-08-03 2008-03-21 ジーンアート・アクチエンゲゼルシャフト ＣｐＧ含量を変化させることにより遺伝子発現を調整する方法
WO2020102645A1 (en) 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease
JP2021500071A (ja) 2017-10-18 2021-01-07 リジェネックスバイオ インコーポレイテッド ヒト翻訳後修飾ｖｅｇｆ−ｔｒａｐによる眼疾患および転移性大腸がんの処置
JP2021503914A (ja) 2017-11-27 2021-02-15 ４ディー モレキュラー セラピューティクス インコーポレイテッド アデノ随伴ウイルス変異キャプシドおよび血管新生の阻害のための使用
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
WO2021050649A1 (en) 2019-09-11 2021-03-18 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept

Family Cites Families (9)

* 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
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
CA3094465A1 (en) * 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
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.

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008507986A (ja) 2004-08-03 2008-03-21 ジーンアート・アクチエンゲゼルシャフト ＣｐＧ含量を変化させることにより遺伝子発現を調整する方法
JP2021500071A (ja) 2017-10-18 2021-01-07 リジェネックスバイオ インコーポレイテッド ヒト翻訳後修飾ｖｅｇｆ−ｔｒａｐによる眼疾患および転移性大腸がんの処置
JP2021503914A (ja) 2017-11-27 2021-02-15 ４ディー モレキュラー セラピューティクス インコーポレイテッド アデノ随伴ウイルス変異キャプシドおよび血管新生の阻害のための使用
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
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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
ウイルス，2007年，vol.57, no.1，p.47-56

Also Published As

Publication number Publication date
US20230295243A1 (en) 2023-09-21
AU2021313839A1 (en) 2023-03-23
EP4185333A1 (en) 2023-05-31
JP2023540464A (ja) 2023-09-25
WO2022018516A1 (en) 2022-01-27
CA3186830A1 (en) 2022-01-27
CN116323949A (zh) 2023-06-23
EP4185333A4 (en) 2024-09-04

Similar Documents

Publication Publication Date Title
JP7842082B2 (ja) 眼疾患を処置するための組成物および方法
JP7492556B2 (ja) 滲出型加齢性黄斑変性の治療のための組成物
JP7410522B2 (ja) 抗ｆａｍ１９ａ５抗体のアデノ関連ウイルス（ａａｖ）伝達
CN113966236A (zh) 眼睛病状的基因疗法
JP2021513355A (ja) 非ウイルス性ｄｎａベクター、ならびに抗体および融合タンパク質の産生のためのその使用
US20250340900A1 (en) Aav drug for treating angiogenesis-related fundus diseases
CN108103104B (zh) 一种预防和治疗脉络膜新生血管相关眼部疾病的基因药物
US20130090375A1 (en) Virus-mediated delivery of bevacizumab for therapeutic applications
JPWO2022018516A5 (https=)
US20230321281A1 (en) Compositions and methods for the treatment of eye diseases
CN118139628A (zh) 用于转基因表达的组合物和方法
CN113952473A (zh) 用于治疗眼部疾病的组合物和方法
CN113952474A (zh) 用于治疗眼部疾病的组合物和方法
AU2024322589A1 (en) Compositions and methods for expressing therapeutics
HK40090167A (zh) 一种抗vegf抗体体内表达系统的构建和应用
HK40091511A (zh) 一种抗vegf抗体体内表达系统的构建和应用
HK40092768A (zh) 用於治疗眼部疾病的组合物和方法
WO2025103416A1 (zh) 用于老年相关性黄斑变性的治疗的基因治疗药物
WO2024238853A1 (en) Adeno-associated viruses for ocular delivery of gene therapy
TW202530261A (zh) 類胰島素生長因子-1受體(igf1r)抗體及相關治療方法
CN113952471A (zh) 用于治疗眼部疾病的组合物和方法
HK40092791A (zh) 用於治疗眼疾病的组合物和方法
JPWO2022018518A5 (https=)
NZ746729B2 (en) Compositions for treatment of wet age-related macular degeneration
NZ787237A (en) Compositions For Treatment of Wet Age-Related Macular Degeneration

Legal Events

Date Code Title Description
A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20231030

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20231030

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231030

A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20240130

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240717

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240717

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250827

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251127

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20260218

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20260319

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20260326

R150 Certificate of patent or registration of utility model

Ref document number: 7842082

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150