CA3171232A1 - Gene therapy - Google Patents

Gene therapy Download PDF

Info

Publication number
CA3171232A1
CA3171232A1 CA3171232A CA3171232A CA3171232A1 CA 3171232 A1 CA3171232 A1 CA 3171232A1 CA 3171232 A CA3171232 A CA 3171232A CA 3171232 A CA3171232 A CA 3171232A CA 3171232 A1 CA3171232 A1 CA 3171232A1
Authority
CA
Canada
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
CA3171232A
Other languages
English (en)
French (fr)
Inventor
Dominic SCHMIDT
Valeryia KUZMUK
Moin SALEEM
Gavin WELSH
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.)
Syncona Ip Holdco 3 Ltd
University of Bristol
Original Assignee
University of Bristol
Syncona Investment Management 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
Application filed by University of Bristol, Syncona Investment Management Ltd filed Critical University of Bristol
Publication of CA3171232A1 publication Critical patent/CA3171232A1/en
Pending legal-status Critical Current

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
    • 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)
CA3171232A 2020-03-12 2021-03-12 Gene therapy Pending CA3171232A1 (en)

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 (1)

Publication Number Publication Date
CA3171232A1 true CA3171232A1 (en) 2021-09-16

Family

ID=70453740

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3171232A Pending CA3171232A1 (en) 2020-03-12 2021-03-12 Gene therapy

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

Family Cites Families (25)

* 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
CA2955481A1 (en) 2014-07-25 2016-01-28 Goldfinch Biopharma, Inc. Collagen iv replacement
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

Also Published As

Publication number Publication date
EP4118216A1 (en) 2023-01-18
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
JP2023517934A (ja) 2023-04-27
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
KR20180012772A (ko) 파브리 병 유전자 치료
JP2022547197A (ja) アデノ随伴ウイルス（ａａｖ）の生体内分布の変更に向けた、ａａｖとａａｖ受容体（ａａｖｒ）との間の相互作用を調節するための方法および組成物
CN103562396A (zh) 用于无脉络膜的基因疗法的aav载体
US11891616B2 (en) Transgene cassettes designed to express a human MECP2 gene
WO2023278305A1 (en) Methods and compositions for tau reduction gene therapy
JP7820422B2 (ja) ジスフェリン二重ベクターを用いた遺伝子治療
BR112021003174A2 (pt) terapia gênica para o tratamento de galactosemia
CN115715185A (zh) 用于治疗神经系统疾病的组合物和方法
US20240376493A1 (en) Optimized ap4m1 polyneucleotides and expression cassettes and their use
CA3130221A1 (en) Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd)
EP4410988A1 (en) An aav2-vector variant for targeted transfer of genes
WO2022238556A1 (en) Vector system
JP7788594B2 (ja) 乳児バッテン病の治療のための髄腔内および静脈内組合せ遺伝子療法
CN121950927A (zh) 基因疗法
WO2024100145A1 (en) Polynucleotide and vector
WO2025168940A1 (en) Vectors
WO2025003710A1 (en) Polynucleotides, aav vectors, and pharmaceutical compositions for treating nephrotic syndrome
WO2025003704A2 (en) Polynucleotides, aav vectors, and pharmaceutical compositions for treating complement-mediated kidney diseases
WO2025193938A1 (en) Delivery of type iv collagen alpha chain using a split intein dual aav vector
WO2023102518A1 (en) Gnao1 gene therapy vectors and uses thereof

Legal Events

Date Code Title Description
D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED

Effective date: 20250228

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W200 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST OR RESPONSE SUBMITTED ONLINE

Effective date: 20250228

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250228

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD

Year of fee payment: 4

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250307

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20250307

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250307

D00 Search and/or examination requested or commenced

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20250310

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT

Effective date: 20250429

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20250512

R00 Party data change recorded

Free format text: ST27 STATUS EVENT CODE: A-2-2-R10-R00-R100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: SMALL ENTITY DECLARATION REQUEST RECEIVED

Effective date: 20251216

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20251216

R00 Party data change recorded

Free format text: ST27 STATUS EVENT CODE: A-2-2-R10-R00-R101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: SMALL ENTITY DECLARATION DETERMINED COMPLIANT

Effective date: 20251219

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251219

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 5TH ANNIV.) - SMALL

Year of fee payment: 5

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260302

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260302