KR102946006B1 - 재조합 aav 벡터 및 그 이용 방법 - Google Patents

재조합 aav 벡터 및 그 이용 방법

Info

Publication number
KR102946006B1
KR102946006B1 KR1020217000782A KR20217000782A KR102946006B1 KR 102946006 B1 KR102946006 B1 KR 102946006B1 KR 1020217000782 A KR1020217000782 A KR 1020217000782A KR 20217000782 A KR20217000782 A KR 20217000782A KR 102946006 B1 KR102946006 B1 KR 102946006B1
Authority
KR
South Korea
Prior art keywords
sequence
seq
vector
delete delete
patient
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
KR1020217000782A
Other languages
English (en)
Korean (ko)
Other versions
KR20210123285A (ko
Inventor
세르주 피투씨
바렛 캐츠
Original Assignee
젠사이트 바이올로직스
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
Application filed by 젠사이트 바이올로직스 filed Critical 젠사이트 바이올로직스
Publication of KR20210123285A publication Critical patent/KR20210123285A/ko
Application granted granted Critical
Publication of KR102946006B1 publication Critical patent/KR102946006B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
    • C12N9/0026—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5)
    • C12N9/0028—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5) with NAD or NADP as acceptor (1.5.1)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
    • C12N9/0036—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on NADH or NADPH (1.6)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0053—Oxidoreductases (1.) acting on a heme group of donors (1.9)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y106/00—Oxidoreductases acting on NADH or NADPH (1.6)
    • C12Y106/05—Oxidoreductases acting on NADH or NADPH (1.6) with a quinone or similar compound as acceptor (1.6.5)
    • C12Y106/05003—NADH dehydrogenase (ubiquinone) (1.6.5.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y109/00—Oxidoreductases acting on a heme group of donors (1.9)
    • C12Y109/03—Oxidoreductases acting on a heme group of donors (1.9) with oxygen as acceptor (1.9.3)
    • C12Y109/03001—Cytochrome-c oxidase (1.9.3.1)
    • 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
    • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
KR1020217000782A 2018-06-11 2019-06-11 재조합 aav 벡터 및 그 이용 방법 Active KR102946006B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862683501P 2018-06-11 2018-06-11
US62/683,501 2018-06-11
PCT/US2019/036487 WO2019241206A1 (en) 2018-06-11 2019-06-11 Recombinant aav vectors and methods of using the same

Publications (2)

Publication Number Publication Date
KR20210123285A KR20210123285A (ko) 2021-10-13
KR102946006B1 true KR102946006B1 (ko) 2026-03-30

Family

ID=67138055

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217000782A Active KR102946006B1 (ko) 2018-06-11 2019-06-11 재조합 aav 벡터 및 그 이용 방법

Country Status (8)

Country Link
US (1) US20210189424A1 (https=)
EP (1) EP3802840A1 (https=)
JP (1) JP7470057B2 (https=)
KR (1) KR102946006B1 (https=)
CN (1) CN112384625A (https=)
AU (1) AU2019286386B2 (https=)
CA (1) CA3103231A1 (https=)
WO (1) WO2019241206A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240014102A (ko) 2018-06-29 2024-01-31 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법
CA3109432A1 (en) * 2018-08-20 2020-02-27 Wuhan Neurophth Biotechnology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
CN113025633B (zh) 2019-12-09 2024-08-27 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
CN115710585B (zh) * 2021-08-23 2025-03-28 纽福斯(苏州)生物科技有限公司 编码nd4的核酸及其用途

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006117250A2 (en) 2005-05-03 2006-11-09 Institut National De La Sante Et De La Recherche Medicale (Inserm) Importation of mitochondrial protein by an enhanced allotopic approach

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6218117B1 (en) * 1998-06-15 2001-04-17 Mitokor Compositions and methods for identifying agents that quantitatively alter detectable extramitochondrial DNA:mitochondrial DNA ratios
EP2121914B1 (en) * 2007-02-16 2014-08-20 John Guy Mitochondrial nucleic acid delivery systems
DE102010013829A1 (de) * 2010-03-26 2011-09-29 Carl Zeiss Microlmaging Gmbh Mikroskop und Verfahren zur Erfassung von Probenlicht
CN102634527B (zh) * 2012-04-11 2013-11-06 华中科技大学同济医学院附属同济医院 重组人nadh脱氢酶亚单位4基因及其表达载体构建方法
TWI775096B (zh) * 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
KR102244434B1 (ko) * 2014-08-11 2021-04-23 삼성전자주식회사 재조합 벡터 및 이를 이용한 목적 폴리펩타이드의 생산 방법
CN104450747B (zh) * 2014-09-23 2018-02-09 武汉纽福斯生物科技有限公司 用于治疗Leber遗传性视神经病变的重组腺相关病毒‑NADH脱氢酶亚单位4基因全长以及药剂

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006117250A2 (en) 2005-05-03 2006-11-09 Institut National De La Sante Et De La Recherche Medicale (Inserm) Importation of mitochondrial protein by an enhanced allotopic approach

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Mol. Ther. Methods Clin. Dev., Vol.2, pp.15003(2015.02.25.)*
Sci. Rep., Vol.6, pp.21587(2016.02.19.)*

Also Published As

Publication number Publication date
JP2021526834A (ja) 2021-10-11
AU2019286386B2 (en) 2025-05-29
US20210189424A1 (en) 2021-06-24
CA3103231A1 (en) 2019-12-19
AU2019286386A1 (en) 2021-01-28
WO2019241206A1 (en) 2019-12-19
AU2019286386A8 (en) 2021-02-11
CN112384625A (zh) 2021-02-19
EP3802840A1 (en) 2021-04-14
JP7470057B2 (ja) 2024-04-17
KR20210123285A (ko) 2021-10-13

Similar Documents

Publication Publication Date Title
KR102946006B1 (ko) 재조합 aav 벡터 및 그 이용 방법
KR20220022107A (ko) 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터
AU2019375975B2 (en) Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons
KR102450833B1 (ko) 혈우병 a 치료용 유전자 요법
CN108753824B (zh) 用于治疗视网膜营养不良的病毒载体
AU2017248656B2 (en) Novel AAV8 mutant capsids and compositions containing same
KR101471445B1 (ko) 조류에서 이식유전자 발현
KR20210080375A (ko) 암 면역요법을 위한 재조합 폭스바이러스
CN109069668B (zh) 用于眼病的基因疗法
KR20210151916A (ko) 뒤시엔느 근육 이영양증의 치료를 위한 aav 벡터-매개된 큰 돌연변이 핫스팟의 결실
CN111733174B (zh) 一种分离的核酸分子及其用途
CN113106124B (zh) 表达cyp4v2的aav载体及其用途
KR101470472B1 (ko) 장기 지속형 약물 제형
KR20220161297A (ko) 신규 세포주
KR20220030205A (ko) 면역 세포의 트랜스포존 기반 변형
CN108138159A (zh) 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途
JPH0799989A (ja) ポリペプチドの合成方法
KR20220163950A (ko) Aav 생산을 위한 이중 이기능성 벡터
CN113260704B (zh) CYP4V2和RdCVF在制备药物中的用途
CN114350710A (zh) 编码vegf拮抗剂的多核苷酸表达盒、重组病毒及相关应用
CN111500635B (zh) 一种包含携带核酸分子的载体的试剂盒
KR20130086035A (ko) 장기 지속형 약물 제형
CN112203697A (zh) 编码氨基己糖苷酶alpha和beta亚基的双顺反子AAV载体及其用途
US5955368A (en) Expression system for clostridium species
AU780854B2 (en) Hormone-hormone receptor complexes and nucleic acid constructs and their use in gene therapy

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

A201 Request for examination
PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

D22 Grant of ip right intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D22-EXM-PE0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0701 Decision of registration

St.27 status event code: A-1-2-D10-D22-exm-PE0701

F11 Ip right granted following substantive examination

Free format text: ST27 STATUS EVENT CODE: A-2-4-F10-F11-EXM-PR0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PR0701 Registration of establishment

St.27 status event code: A-2-4-F10-F11-exm-PR0701

PR1002 Payment of registration fee

St.27 status event code: A-2-2-U10-U12-oth-PR1002

Fee payment year number: 1

U12 Designation fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U12-OTH-PR1002 (AS PROVIDED BY THE NATIONAL OFFICE)

Year of fee payment: 1

PG1601 Publication of registration

St.27 status event code: A-4-4-Q10-Q13-nap-PG1601

Q13 Ip right document published

Free format text: ST27 STATUS EVENT CODE: A-4-4-Q10-Q13-NAP-PG1601 (AS PROVIDED BY THE NATIONAL OFFICE)