JP2023517934A - 遺伝子治療 - Google Patents

遺伝子治療 Download PDF

Info

Publication number
JP2023517934A
JP2023517934A JP2022554473A JP2022554473A JP2023517934A JP 2023517934 A JP2023517934 A JP 2023517934A JP 2022554473 A JP2022554473 A JP 2022554473A JP 2022554473 A JP2022554473 A JP 2022554473A JP 2023517934 A JP2023517934 A JP 2023517934A
Authority
JP
Japan
Prior art keywords
viral vector
seq
promoter
aav
col4a5
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
JP2022554473A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023517934A5 (https=
JPWO2021181118A5 (https=
Inventor
シュミット，ドミニク
クズムク，ヴァレリイア
サレーム，モイン
ウェルシュ，ギャビン
Original Assignee
ザ ユニバーシティ オブ ブリストル
シンコナ インヴェストメント マネージメント リミティッド
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 ザ ユニバーシティ オブ ブリストル, シンコナ インヴェストメント マネージメント リミティッド filed Critical ザ ユニバーシティ オブ ブリストル
Publication of JP2023517934A publication Critical patent/JP2023517934A/ja
Publication of JP2023517934A5 publication Critical patent/JP2023517934A5/ja
Publication of JPWO2021181118A5 publication Critical patent/JPWO2021181118A5/ja
Pending legal-status Critical Current

Links

Images

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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • 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
    • 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
    • C12N2750/14145—Special targeting system for 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
JP2022554473A 2020-03-12 2021-03-12 遺伝子治療 Pending JP2023517934A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB2003618.2 2020-03-12
GBGB2003618.2A GB202003618D0 (en) 2020-03-12 2020-03-12 Gene Therapy
PCT/GB2021/050633 WO2021181118A1 (en) 2020-03-12 2021-03-12 Gene therapy

Publications (3)

Publication Number Publication Date
JP2023517934A true JP2023517934A (ja) 2023-04-27
JP2023517934A5 JP2023517934A5 (https=) 2024-03-21
JPWO2021181118A5 JPWO2021181118A5 (https=) 2024-03-21

Family

ID=70453740

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022554473A Pending JP2023517934A (ja) 2020-03-12 2021-03-12 遺伝子治療

Country Status (11)

Country Link
US (1) US12589167B2 (https=)
EP (1) EP4118216A1 (https=)
JP (1) JP2023517934A (https=)
CN (1) CN115427578B (https=)
AU (1) AU2021234169A1 (https=)
BR (1) BR112022018036A2 (https=)
CA (1) CA3171232A1 (https=)
GB (1) GB202003618D0 (https=)
IL (1) IL296255A (https=)
MX (1) MX2022011295A (https=)
WO (1) WO2021181118A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB202003618D0 (en) 2020-03-12 2020-04-29 Univ Bristol Gene Therapy
EP4273243A1 (en) 2022-05-02 2023-11-08 Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca Nucleic acid constructs and vectors for podocyte specific expression
GB202304841D0 (en) * 2023-03-31 2023-05-17 Purespring Therapeutics Ltd Promoter
CN116590345B (zh) * 2023-05-06 2024-01-30 北京中医药大学 永生化小鼠足细胞系及其制备方法、分化方法和应用
GB202309913D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Polynucleotides, avv vectors, and pharmaceutical compositions for treating nephrotic syndrome
GB202309908D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Polynucleotides, aav vectors, and pharmaceutical compositions for treating complement-mediated kidney diseases
GB202309914D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Promoters
GB202309909D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Regulatory elements
WO2025168940A1 (en) * 2024-02-07 2025-08-14 Purespring Therapeutics Limited Vectors
WO2025193938A1 (en) * 2024-03-13 2025-09-18 Oregon Health & Science University Delivery of type iv collagen alpha chain using a split intein dual aav vector

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017532365A (ja) * 2014-07-25 2017-11-02 ゴールドフィンチ バイオファーマ，インク． コラーゲンｉｖの置き換え

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL332875A1 (en) 1996-10-17 1999-10-25 Oxford Biomedica Ltd Retroviral vectors
US20060281090A1 (en) 2003-05-01 2006-12-14 University Of Washington Uw Tech Transfer- Invention Licensing Capsid-modified adenovirus vectors and methods of using the same
WO2005012351A2 (en) 2003-08-01 2005-02-10 The Brigham And Women's Hospital, Inc. Novel functions for decay accelerating factor (daf) in inflammation
EP2357254A1 (en) 2005-02-14 2011-08-17 University of Iowa Research Foundation Methods and reagents for treatment and diagnosis of age-related macular degeneration
WO2007056435A2 (en) 2005-11-08 2007-05-18 The General Hospital Corporation Dynamin mediated diseases and associated methods and products
WO2008136547A1 (en) 2007-05-07 2008-11-13 University Of Ulsan Foundation For Industry Cooperation Method of preventing or treating body weight disorders by employing clusterin
ES2457822T3 (es) 2007-11-08 2014-04-29 The General Hospital Corporation Procedimientos y composiciones de tratamiento de enfermedades proteinúricas
EP2601288B1 (en) 2010-08-05 2016-04-06 Wisconsin Alumni Research Foundation Simplified basic media for human pluripotent cell culture
US8852936B2 (en) 2012-09-21 2014-10-07 Laurantis Pharma Oy Autologous lymph node transfer in combination with VEGF-C or VEGF-D growth factor therapy to treat lymphedema and to improve reconstructive surgery
TW201506036A (zh) 2013-08-14 2015-02-16 Laurantis Pharma Oy Vegf-c及ccbe1之治療用途
EP3526333A4 (en) * 2016-10-13 2020-07-29 University of Massachusetts AAV CAPSIDE DESIGNS
CN107011424B (zh) 2017-03-14 2020-07-03 华南师范大学 斜带石斑鱼补体c1inh基因、载体、重组菌株和蛋白及其应用
GB201706808D0 (en) 2017-04-28 2017-06-14 Univ Newcastle Modified complement proteins and uses thereof
EP3635121A1 (en) * 2017-06-02 2020-04-15 Institut National de la Sante et de la Recherche Medicale (INSERM) Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders
CN111788311A (zh) 2017-10-20 2020-10-16 双子治疗公司 用于治疗年龄相关性黄斑变性的组合物和方法
RU2675861C1 (ru) 2017-11-22 2018-12-25 Лев Иосифович Шагам Способ прогнозирования наличия мутации гена col4a5 у пациентов с предполагаемым диагнозом "синдром альпорта"
EP3856763A1 (en) * 2018-09-28 2021-08-04 Massachusetts Institute of Technology Collagen-localized immunomodulatory molecules and methods thereof
EP3861010A1 (en) * 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
CA3117551A1 (en) 2018-10-23 2020-04-30 Gemini Therapeutics Inc. Compositions and methods for treating age-related macular degeneration and other diseases
RU2714763C1 (ru) 2018-11-08 2020-02-19 Генетик Диагностикс Энд Терапи 21 Лтд Генотерапевтический ДНК-вектор для таргетной генной терапии, способ его получения (варианты), штамм для его производства, способ его получения
GB201900702D0 (en) 2019-01-18 2019-03-06 Univ Bristol Therapy
GB202003618D0 (en) 2020-03-12 2020-04-29 Univ Bristol Gene Therapy
GB202010009D0 (en) 2020-06-30 2020-08-12 Syncona Investment Man Ltd Vector
GB202103470D0 (en) 2021-03-12 2021-04-28 Univ Bristol Promoter

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017532365A (ja) * 2014-07-25 2017-11-02 ゴールドフィンチ バイオファーマ，インク． コラーゲンｉｖの置き換え

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
GENE THERAPY, vol. 8, JPN6025009485, 2001, pages 882 - 890, ISSN: 0005546895 *
HEARING RESEARCH, vol. 394, 107912, JPN6025009486, February 2020 (2020-02-01), pages 1 - 13, ISSN: 0005546896 *
HUMAN GENE THERAPY, vol. 10, JPN6025009488, 1999, pages 2295 - 2305, ISSN: 0005546898 *
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, vol. 1, 14014, JPN6025009487, 2014, ISSN: 0005546897 *

Also Published As

Publication number Publication date
EP4118216A1 (en) 2023-01-18
CA3171232A1 (en) 2021-09-16
US12589167B2 (en) 2026-03-31
CN115427578A (zh) 2022-12-02
WO2021181118A1 (en) 2021-09-16
AU2021234169A1 (en) 2022-10-06
US20240197917A1 (en) 2024-06-20
IL296255A (en) 2022-11-01
MX2022011295A (es) 2022-11-14
BR112022018036A2 (pt) 2022-11-01
CN115427578B (zh) 2026-03-17
GB202003618D0 (en) 2020-04-29

Similar Documents

Publication Publication Date Title
US12589167B2 (en) AAV gene therapy vector with podocyte-specific promoter
JP7349931B2 (ja) 高ビリルビン血症の処置
US12370268B2 (en) Fabry disease gene therapy
US20250333763A1 (en) Gene therapy constructs for treating wilson disease
HK1249548A1 (zh) 用於无脉络膜的基因疗法的aav载体
JP2020536589A (ja) Ａａｖベクター
JP2019523648A (ja) Ａｂｃ４ａを発現させるためのデュアル重複アデノ随伴ウイルスベクターシステム
JP7616668B2 (ja) Ｕｂｅ３ａ遺伝子および発現カセットならびにそれらの使用
JP2021533801A (ja) 組換えヒトＩＩ型ミトコンドリアダイニン様ＧＴＰａｓｅの遺伝子配列及びその使用
EP3390623B1 (en) Wilson's disease gene therapy
EP4410988A1 (en) An aav2-vector variant for targeted transfer of genes
US20220389450A1 (en) Vector system
CN121950927A (zh) 基因疗法
RU2807158C2 (ru) Генотерапевтические конструкции для лечения болезни вильсона
WO2025003710A1 (en) Polynucleotides, aav vectors, and pharmaceutical compositions for treating nephrotic syndrome
EP4615492A1 (en) Polynucleotide and vector
WO2025003704A2 (en) Polynucleotides, aav vectors, and pharmaceutical compositions for treating complement-mediated kidney diseases
WO2025006957A2 (en) Non-neutralizing aav constructs and methods of generating and using same

Legal Events

Date Code Title Description
RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20221209

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20221209

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240308

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240308

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20241112

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20241112

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20250214

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250311

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250516

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250909

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20251111

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260310