CA3060187A1 - Gene therapy for ciliopathies - Google Patents

Gene therapy for ciliopathies Download PDF

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Publication number
CA3060187A1
CA3060187A1 CA3060187A CA3060187A CA3060187A1 CA 3060187 A1 CA3060187 A1 CA 3060187A1 CA 3060187 A CA3060187 A CA 3060187A CA 3060187 A CA3060187 A CA 3060187A CA 3060187 A1 CA3060187 A1 CA 3060187A1
Authority
CA
Canada
Prior art keywords
vector
ciliopathy
seq
gene
protein
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
CA3060187A
Other languages
English (en)
French (fr)
Inventor
Phillip Beales
Victor Hernandez
Original Assignee
UCL Business 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 UCL Business Ltd filed Critical UCL Business Ltd
Publication of CA3060187A1 publication Critical patent/CA3060187A1/en
Pending legal-status Critical Current

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3060187A 2017-05-05 2018-05-04 Gene therapy for ciliopathies Pending CA3060187A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1707212.5A GB201707212D0 (en) 2017-05-05 2017-05-05 Gene therapy for ciliopathies
GB1707212.5 2017-05-05
PCT/GB2018/051219 WO2018203092A1 (en) 2017-05-05 2018-05-04 Gene therapy for ciliopathies

Publications (1)

Publication Number Publication Date
CA3060187A1 true CA3060187A1 (en) 2018-11-08

Family

ID=59065740

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3060187A Pending CA3060187A1 (en) 2017-05-05 2018-05-04 Gene therapy for ciliopathies

Country Status (8)

Country Link
US (2) US12171794B2 (https=)
EP (1) EP3628011A1 (https=)
JP (2) JP7759174B2 (https=)
CN (1) CN110753559A (https=)
AU (1) AU2018262427B2 (https=)
CA (1) CA3060187A1 (https=)
GB (1) GB201707212D0 (https=)
WO (1) WO2018203092A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12171794B2 (en) 2017-05-05 2024-12-24 Ucl Business Ltd Gene therapy for ciliopathies

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118949075A (zh) * 2019-02-14 2024-11-15 埃泽瑞斯公司 纤毛疾病的治疗
KR20220083714A (ko) * 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
GB202005641D0 (en) * 2020-04-17 2020-06-03 Ucl Business Plc Gene therapy for bardet-biedl syndrome
CN116042719B (zh) * 2022-12-28 2025-11-11 蓝图生物医药(广州)有限公司 一种重组腺相关病毒载体及其应用
WO2025049923A2 (en) * 2023-08-31 2025-03-06 The Board Of Trustees Of The Leland Stanford Junior University Novel treatments of ciliopathies

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6962788B2 (en) 2002-05-30 2005-11-08 University Of Iowa Research Foundation Identification of a gene causing the most common form of bardet-biedl syndrome and uses thereof
US8163482B2 (en) * 2006-02-23 2012-04-24 Nicholas Katsanis BBS10 related diagnostic methods for Bardet-Biedl syndrome
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
ES2697249T3 (es) * 2013-03-11 2019-01-22 Fond Telethon miR-204 y miR-211 y usos de los mismos
GB201707212D0 (en) 2017-05-05 2017-06-21 Ucl Business Plc Gene therapy for ciliopathies

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12171794B2 (en) 2017-05-05 2024-12-24 Ucl Business Ltd Gene therapy for ciliopathies

Also Published As

Publication number Publication date
GB201707212D0 (en) 2017-06-21
AU2018262427B2 (en) 2024-02-29
EP3628011A1 (en) 2020-04-01
CN110753559A (zh) 2020-02-04
US20250057899A1 (en) 2025-02-20
JP2025120285A (ja) 2025-08-15
AU2018262427A1 (en) 2019-11-14
US20200069753A1 (en) 2020-03-05
JP7759174B2 (ja) 2025-10-23
JP2020518269A (ja) 2020-06-25
US12171794B2 (en) 2024-12-24
WO2018203092A1 (en) 2018-11-08

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