JP2021512929A - Faah偽遺伝子の遺伝子療法 - Google Patents

Faah偽遺伝子の遺伝子療法 Download PDF

Info

Publication number
JP2021512929A
JP2021512929A JP2020542955A JP2020542955A JP2021512929A JP 2021512929 A JP2021512929 A JP 2021512929A JP 2020542955 A JP2020542955 A JP 2020542955A JP 2020542955 A JP2020542955 A JP 2020542955A JP 2021512929 A JP2021512929 A JP 2021512929A
Authority
JP
Japan
Prior art keywords
faah
inhibitor
pain
sequence
seq
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
JP2020542955A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021512929A5 (https=
Inventor
ジョン コックス,ジェームズ
ジョン コックス,ジェームズ
スリバスタヴァ,デヴジト
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
Original Assignee
UCL Biomedica PLC
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 Biomedica PLC, UCL Business Ltd filed Critical UCL Biomedica PLC
Publication of JP2021512929A publication Critical patent/JP2021512929A/ja
Publication of JP2021512929A5 publication Critical patent/JP2021512929A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C12N9/80Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/01Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amides (3.5.1)
    • C12Y305/01004Amidase (3.5.1.4)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2020542955A 2018-02-13 2019-02-12 Faah偽遺伝子の遺伝子療法 Pending JP2021512929A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1802326.7A GB201802326D0 (en) 2018-02-13 2018-02-13 Gene therapy
GB1802326.7 2018-02-13
PCT/GB2019/050368 WO2019158909A1 (en) 2018-02-13 2019-02-12 Gene therapy of the faah pseudogene

Publications (2)

Publication Number Publication Date
JP2021512929A true JP2021512929A (ja) 2021-05-20
JP2021512929A5 JP2021512929A5 (https=) 2022-02-21

Family

ID=61731413

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020542955A Pending JP2021512929A (ja) 2018-02-13 2019-02-12 Faah偽遺伝子の遺伝子療法

Country Status (8)

Country Link
US (1) US12241066B2 (https=)
EP (1) EP3752608B1 (https=)
JP (1) JP2021512929A (https=)
CN (1) CN112088214A (https=)
AU (1) AU2019221798A1 (https=)
CA (1) CA3090617A1 (https=)
GB (1) GB201802326D0 (https=)
WO (1) WO2019158909A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022018638A1 (en) 2020-07-21 2022-01-27 Crispr Therapeutics Ag Genome-editing compositions and methods to modulate faah for treatment of neurological disorders
WO2022020756A1 (en) * 2020-07-23 2022-01-27 John Mansell Compositions and methods for treating pain and anxiety disorders
CN119932015B (zh) * 2024-12-20 2025-11-25 中国科学院生物物理研究所 一种lncATRIG抑制剂及其在制备治疗胶质瘤的药物中的应用

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7541359B2 (en) 2005-06-30 2009-06-02 Janssen Pharmaceutica N.V. N-heteroarylpiperazinyl ureas as modulators of fatty acid amide hydrolase
US20070088073A1 (en) * 2005-10-19 2007-04-19 Allergan, Inc. Method for treating pain
US9920317B2 (en) * 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP2670245B1 (en) 2011-02-04 2015-09-09 The Scripps Research Institute Alpha-ketoheterocycles and methods of making and using
KR102079404B1 (ko) 2011-08-19 2020-02-19 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 메타-치환된 비페닐 말초 제한형 faah 억제제

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BRITISH JOURNAL OF PHARMACOLOGY, vol. 148(1), JPN6023007873, 2006, pages 102 - 113, ISSN: 0005000759 *
DRUG DISCOVERY TODAY, vol. 14(23/24), JPN6023007872, 2009, pages 1098 - 1111, ISSN: 0005000761 *
EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES, vol. 52, JPN6023007874, 2014, pages 173 - 179, ISSN: 0005000760 *

Also Published As

Publication number Publication date
GB201802326D0 (en) 2018-03-28
CN112088214A (zh) 2020-12-15
US20210002648A1 (en) 2021-01-07
EP3752608B1 (en) 2026-03-18
EP3752608A1 (en) 2020-12-23
US12241066B2 (en) 2025-03-04
WO2019158909A1 (en) 2019-08-22
AU2019221798A1 (en) 2020-09-03
CA3090617A1 (en) 2019-08-22

Similar Documents

Publication Publication Date Title
Hou et al. Aged bone marrow macrophages drive systemic aging and age-related dysfunction via extracellular vesicle-mediated induction of paracrine senescence
Ge et al. Increased miR-21-3p in injured brain microvascular endothelial cells after traumatic brain injury aggravates blood–brain barrier damage by promoting cellular apoptosis and inflammation through targeting MAT2B
RU2683772C2 (ru) Композиции и способы модуляции smn2 сплайсинга у субъекта
Stavrou et al. A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice
CN113728230B (zh) 检测、预防、逆转和治疗神经系统疾病的方法
EP3449001A1 (en) Inhibition of mir-22 mirna by apt-110
US10822400B2 (en) Dynamin 2 inhibitor for the treatment of Duchenne's muscular dystrophy
JP2023538284A (ja) Plp1発現を阻害するための組成物及び方法
CA2986913A1 (en) Mir-155 inhibitors for treating amyotrophic lateral sclerosis (als)
Forand et al. Combined treatment with peptide-conjugated phosphorodiamidate morpholino oligomer-PPMO and AAV-U7 rescues the severe DMD phenotype in mice
JP2021512929A (ja) Faah偽遺伝子の遺伝子療法
JP2018517412A (ja) 皮膚T細胞リンパ腫(CTCL)を処置するためのmiR−155阻害剤
WO2023278449A1 (en) Engineered guide rnas and polynucleotides
ES2685346T3 (es) ARNip y su uso en métodos y composiciones para el tratamiento y/o la prevención de afecciones oculares
JP2026021335A (ja) apo(a)を標的化するコンジュゲートアンチセンス化合物を用いて心血管事象のリスクを低減する方法
US20240279656A1 (en) Engineered Guide RNAs and Polynucleotides
JP6049143B2 (ja) オリゴヌクレオチド、グルココルチコイド感受性増強剤、医薬組成物、及び発現ベクター
KR20240032998A (ko) 신경근육장애를 위한 올리고뉴클레오티드 및 이의 조성물
US20150231203A1 (en) Treatment of pain by inhibition of usp5 de-ubiquitinase
JP2025501269A (ja) アンチセンスオリゴヌクレオチド及びその使用
WO2026006483A1 (en) Snca vectors and engineered guide rnas
Trundle The Manipulation of Alternative Splicing of Postn mRNA to Address Skeletal Muscle Fibrosis
WO2024238584A1 (en) Engineered constructs with cassette arrangements for increased transcription of rna payloads

Legal Events

Date Code Title Description
RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20211110

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20211110

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220210

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220210

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230228

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230525

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230825

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20231031