AU2018262427B2 - Gene therapy for ciliopathies - Google Patents

Gene therapy for ciliopathies Download PDF

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Publication number
AU2018262427B2
AU2018262427B2 AU2018262427A AU2018262427A AU2018262427B2 AU 2018262427 B2 AU2018262427 B2 AU 2018262427B2 AU 2018262427 A AU2018262427 A AU 2018262427A AU 2018262427 A AU2018262427 A AU 2018262427A AU 2018262427 B2 AU2018262427 B2 AU 2018262427B2
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AU
Australia
Prior art keywords
bbs1
seq
vector
gene
ciliopathy
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Active
Application number
AU2018262427A
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English (en)
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AU2018262427A1 (en
Inventor
Phillip BEALES
Victor Hernandez
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.)
UCL Business Ltd
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UCL Business Ltd
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Publication of AU2018262427A1 publication Critical patent/AU2018262427A1/en
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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
    • A61K48/0083—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
    • 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
    • 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)
AU2018262427A 2017-05-05 2018-05-04 Gene therapy for ciliopathies Active AU2018262427B2 (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 (2)

Publication Number Publication Date
AU2018262427A1 AU2018262427A1 (en) 2019-11-14
AU2018262427B2 true AU2018262427B2 (en) 2024-02-29

Family

ID=59065740

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2018262427A Active AU2018262427B2 (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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007101094A2 (en) * 2006-02-23 2007-09-07 The Johns Hopkins University Bbs10 related diagnostic methods and reagents for bardet-biedl syndrome

Family Cites Families (4)

* 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
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007101094A2 (en) * 2006-02-23 2007-09-07 The Johns Hopkins University Bbs10 related diagnostic methods and reagents for bardet-biedl syndrome

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
COREY L. WILLIAMS ET AL: "Gene Therapeutic Reversal of Peripheral Olfactory Impairment in Bardet-Biedl Syndrome", MOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY, vol. 25, no. 4, 1 April 2017, pages 904 - 916 *
FRANCESCO T. ET AL: "Evaluation of Italian Patients with Leber Congenital Amaurosis due to AIPL1 Mutations Highlights the Potential Applicability of Gene Therapy", INVST. OPTHAL.& VIS.SCI., vol. 52, no. 8, 2011, pages 5618 - 5624 *
SEONGJIN SEO ET AL: "Subretinal Gene Therapy of Mice With Bardet-Biedl Syndrome Type 1", INVESTIGATIVE OPTHALMOLOGY & VISUAL SCIENCE, vol. 54, no. 9, 11 September 2013, pages 6118 - 6132 *
WENSHENG LI ET AL: "Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye", MOLECULAR VISION, 1 January 2009 (2009-01-01), pages 267 - 275 *

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
EP3628011A1 (en) 2020-04-01
CN110753559A (zh) 2020-02-04
US20250057899A1 (en) 2025-02-20
CA3060187A1 (en) 2018-11-08
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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