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 PDFInfo
- 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
Links
Images
Classifications
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
- C12N9/80—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-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
- C12N15/1137—Non-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
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-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
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/01—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amides (3.5.1)
- C12Y305/01004—Amidase (3.5.1.4)
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type 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)
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)
| 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)
| 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 |
-
2018
- 2018-02-13 GB GBGB1802326.7A patent/GB201802326D0/en not_active Ceased
-
2019
- 2019-02-12 US US16/969,278 patent/US12241066B2/en active Active
- 2019-02-12 AU AU2019221798A patent/AU2019221798A1/en not_active Abandoned
- 2019-02-12 CN CN201980025731.2A patent/CN112088214A/zh active Pending
- 2019-02-12 WO PCT/GB2019/050368 patent/WO2019158909A1/en not_active Ceased
- 2019-02-12 CA CA3090617A patent/CA3090617A1/en active Pending
- 2019-02-12 JP JP2020542955A patent/JP2021512929A/ja active Pending
- 2019-02-12 EP EP19708599.6A patent/EP3752608B1/en active Active
Non-Patent Citations (3)
| 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 |