JP2020516244A5 - - Google Patents
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- JP2020516244A5 JP2020516244A5 JP2019554630A JP2019554630A JP2020516244A5 JP 2020516244 A5 JP2020516244 A5 JP 2020516244A5 JP 2019554630 A JP2019554630 A JP 2019554630A JP 2019554630 A JP2019554630 A JP 2019554630A JP 2020516244 A5 JP2020516244 A5 JP 2020516244A5
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- JP
- Japan
- Prior art keywords
- seq
- sequence
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- variant
- 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.)
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- 239000000203 mixture Substances 0.000 claims 38
- 239000002773 nucleotide Substances 0.000 claims 23
- 125000003729 nucleotide group Chemical group 0.000 claims 23
- 239000012634 fragment Substances 0.000 claims 21
- 108091026890 Coding region Proteins 0.000 claims 19
- 125000003275 alpha amino acid group Chemical group 0.000 claims 11
- 125000006850 spacer group Chemical group 0.000 claims 11
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 9
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 9
- 201000010099 disease Diseases 0.000 claims 8
- 108090000765 processed proteins & peptides Proteins 0.000 claims 6
- 102000004219 Brain-derived neurotrophic factor Human genes 0.000 claims 5
- 108090000715 Brain-derived neurotrophic factor Proteins 0.000 claims 5
- 102000015534 trkB Receptor Human genes 0.000 claims 5
- 108010064880 trkB Receptor Proteins 0.000 claims 5
- 108010029485 Protein Isoforms Proteins 0.000 claims 4
- 102000001708 Protein Isoforms Human genes 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 125000001493 tyrosinyl group Chemical group [H]OC1=C([H])C([H])=C(C([H])=C1[H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 claims 4
- 102000003683 Neurotrophin-4 Human genes 0.000 claims 3
- 108090000099 Neurotrophin-4 Proteins 0.000 claims 3
- 125000000539 amino acid group Chemical group 0.000 claims 3
- 230000007850 degeneration Effects 0.000 claims 3
- 208000015122 neurodegenerative disease Diseases 0.000 claims 3
- 229940097998 neurotrophin 4 Drugs 0.000 claims 3
- 239000000018 receptor agonist Substances 0.000 claims 3
- 229940044601 receptor agonist Drugs 0.000 claims 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 2
- 208000024827 Alzheimer disease Diseases 0.000 claims 2
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 208000004986 Diffuse Cerebral Sclerosis of Schilder Diseases 0.000 claims 2
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 208000000609 Pick Disease of the Brain Diseases 0.000 claims 2
- 239000000556 agonist Substances 0.000 claims 2
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 2
- ZPUCINDJVBIVPJ-LJISPDSOSA-N cocaine Chemical compound O([C@H]1C[C@@H]2CC[C@@H](N2C)[C@H]1C(=O)OC)C(=O)C1=CC=CC=C1 ZPUCINDJVBIVPJ-LJISPDSOSA-N 0.000 claims 2
- 208000005264 motor neuron disease Diseases 0.000 claims 2
- 150000007523 nucleic acids Chemical group 0.000 claims 2
- XOAAWQZATWQOTB-UHFFFAOYSA-N taurine Chemical compound NCCS(O)(=O)=O XOAAWQZATWQOTB-UHFFFAOYSA-N 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 206010065040 AIDS dementia complex Diseases 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 208000011403 Alexander disease Diseases 0.000 claims 1
- 208000036022 Alpers' disease Diseases 0.000 claims 1
- 206010003694 Atrophy Diseases 0.000 claims 1
- 241000283690 Bos taurus Species 0.000 claims 1
- 208000014644 Brain disease Diseases 0.000 claims 1
- 206010068597 Bulbospinal muscular atrophy congenital Diseases 0.000 claims 1
- 206010008025 Cerebellar ataxia Diseases 0.000 claims 1
- 206010012289 Dementia Diseases 0.000 claims 1
- 208000012661 Dyskinesia Diseases 0.000 claims 1
- 206010049020 Encephalitis periaxialis diffusa Diseases 0.000 claims 1
- 208000032274 Encephalopathy Diseases 0.000 claims 1
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Natural products OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- 101000821100 Homo sapiens Synapsin-1 Proteins 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 208000027747 Kennedy disease Diseases 0.000 claims 1
- 208000016604 Lyme disease Diseases 0.000 claims 1
- 208000002569 Machado-Joseph Disease Diseases 0.000 claims 1
- 208000026072 Motor neurone disease Diseases 0.000 claims 1
- 208000008955 Mucolipidoses Diseases 0.000 claims 1
- 206010052057 Neuroborreliosis Diseases 0.000 claims 1
- 208000010577 Niemann-Pick disease type C Diseases 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 208000032319 Primary lateral sclerosis Diseases 0.000 claims 1
- 208000024777 Prion disease Diseases 0.000 claims 1
- 102000004278 Receptor Protein-Tyrosine Kinases Human genes 0.000 claims 1
- 108090000873 Receptor Protein-Tyrosine Kinases Proteins 0.000 claims 1
- 208000021811 Sandhoff disease Diseases 0.000 claims 1
- 208000021235 Schilder disease Diseases 0.000 claims 1
- 208000010112 Spinocerebellar Degenerations Diseases 0.000 claims 1
- 208000007930 Type C Niemann-Pick Disease Diseases 0.000 claims 1
- 206010046298 Upper motor neurone lesion Diseases 0.000 claims 1
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims 1
- 208000006269 X-Linked Bulbo-Spinal Atrophy Diseases 0.000 claims 1
- 238000009825 accumulation Methods 0.000 claims 1
- 230000001154 acute effect Effects 0.000 claims 1
- 208000007502 anemia Diseases 0.000 claims 1
- 230000037444 atrophy Effects 0.000 claims 1
- 230000002490 cerebral effect Effects 0.000 claims 1
- 206010008129 cerebral palsy Diseases 0.000 claims 1
- 229960003920 cocaine Drugs 0.000 claims 1
- 230000001054 cortical effect Effects 0.000 claims 1
- 230000007812 deficiency Effects 0.000 claims 1
- 230000003205 diastolic effect Effects 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 206010015037 epilepsy Diseases 0.000 claims 1
- 235000013922 glutamic acid Nutrition 0.000 claims 1
- 239000004220 glutamic acid Substances 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 102000056115 human SYN1 Human genes 0.000 claims 1
- 201000010901 lateral sclerosis Diseases 0.000 claims 1
- 230000003211 malignant effect Effects 0.000 claims 1
- 201000007769 mucolipidosis Diseases 0.000 claims 1
- 201000006417 multiple sclerosis Diseases 0.000 claims 1
- 201000003631 narcolepsy Diseases 0.000 claims 1
- 230000001537 neural effect Effects 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 230000003647 oxidation Effects 0.000 claims 1
- 238000007254 oxidation reaction Methods 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 201000002212 progressive supranuclear palsy Diseases 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 230000028327 secretion Effects 0.000 claims 1
- 210000000278 spinal cord Anatomy 0.000 claims 1
- 208000002320 spinal muscular atrophy Diseases 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
- 229960003080 taurine Drugs 0.000 claims 1
- 210000003478 temporal lobe Anatomy 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1705484.2A GB201705484D0 (en) | 2017-04-05 | 2017-04-05 | Genetic construct |
| GB1705484.2 | 2017-04-05 | ||
| PCT/GB2018/050824 WO2018185468A1 (en) | 2017-04-05 | 2018-03-28 | Genetic construct for use in the treatment of neurodegenerative disorder or stroke |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020516244A JP2020516244A (ja) | 2020-06-11 |
| JP2020516244A5 true JP2020516244A5 (https=) | 2021-05-06 |
| JP7296321B2 JP7296321B2 (ja) | 2023-06-22 |
Family
ID=58682577
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019554630A Active JP7296321B2 (ja) | 2017-04-05 | 2018-03-28 | 神経変性障害または脳卒中の治療に用いる遺伝子構築物 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20200046850A1 (https=) |
| EP (1) | EP3606546B1 (https=) |
| JP (1) | JP7296321B2 (https=) |
| KR (1) | KR102616629B1 (https=) |
| CN (1) | CN110809476B (https=) |
| AU (1) | AU2018248651B2 (https=) |
| CA (1) | CA3058549A1 (https=) |
| ES (1) | ES2881176T3 (https=) |
| GB (1) | GB201705484D0 (https=) |
| MX (1) | MX2019012000A (https=) |
| PL (1) | PL3606546T3 (https=) |
| PT (1) | PT3606546T (https=) |
| RU (1) | RU2757932C2 (https=) |
| WO (1) | WO2018185468A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3687580A1 (en) * | 2017-09-27 | 2020-08-05 | Sigilon Therapeutics, Inc. | Methods, compositions, and implantable elements comprising active cells |
| AU2019407921A1 (en) * | 2018-12-19 | 2021-06-10 | Versameb Ag | RNA encoding a protein |
| JP7524158B2 (ja) | 2019-03-04 | 2024-07-29 | 公益財団法人東京都医学総合研究所 | Trk断片をコードする核酸構築物、及びその利用 |
| AU2020248099A1 (en) * | 2019-03-27 | 2021-10-07 | Sigilon Therapeutics, Inc. | Compositions, devices and methods for treating Fabry disease |
| GB202004832D0 (en) * | 2020-04-01 | 2020-05-13 | Instituto De Medicina Molecular Faculdade De Medicina Univ De Lisboa | Therapeutic agaents, pharmaceutical compositions, and associated biomarkers |
| US20240050524A1 (en) * | 2020-12-18 | 2024-02-15 | Baylor College Of Medicine | Delivery of abeta variants for aggregation inhibition |
| CN114933657B (zh) * | 2021-08-25 | 2024-02-02 | 上海交通大学医学院 | 神经生长因子突变体重组蛋白及其应用 |
| CN118256562A (zh) * | 2022-12-26 | 2024-06-28 | 科辉智药(深圳)新药研究中心有限公司 | 一种用于中枢神经系统疾病治疗的基因序列构建体及其应用 |
| AU2024214634A1 (en) | 2023-02-02 | 2025-06-26 | Quethera Limited | Recombinant adeno-associated virus vector |
| EP4413993A1 (en) * | 2023-02-10 | 2024-08-14 | Dompe' Farmaceutici S.P.A. | Method of obtaining recombinant human brain-derived neurotrophic factor |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2588221C (en) * | 1999-06-08 | 2013-07-09 | Regeneron Pharmaceuticals, Inc. | Modified chimeric polypeptides with improved pharmacokinetic properties |
| US20030124095A1 (en) * | 2001-12-31 | 2003-07-03 | Regents Of The University Of California | Methods for therapeutic use of brain derived neurotrophic factor in the entorhinal cortex |
| US9265843B2 (en) * | 2008-03-27 | 2016-02-23 | The Ohio State University | Treatment of metabolic-related disorders using hypothalamic gene transfer of BDNF and compositions therefor |
| EP3165537A1 (en) * | 2008-12-19 | 2017-05-10 | H. Lundbeck A/S | Modulation of the vps 10-domain receptor family for the treatment of mental and behavioural disorders |
| HK1210211A1 (en) * | 2012-03-15 | 2016-04-15 | 科纳公司 | Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf |
| GB2547179A (en) * | 2015-10-26 | 2017-08-16 | Quethera Ltd | Genetic construct |
-
2017
- 2017-04-05 GB GBGB1705484.2A patent/GB201705484D0/en not_active Ceased
-
2018
- 2018-03-28 PT PT187159116T patent/PT3606546T/pt unknown
- 2018-03-28 CN CN201880023931.XA patent/CN110809476B/zh active Active
- 2018-03-28 JP JP2019554630A patent/JP7296321B2/ja active Active
- 2018-03-28 RU RU2019131098A patent/RU2757932C2/ru active
- 2018-03-28 MX MX2019012000A patent/MX2019012000A/es unknown
- 2018-03-28 WO PCT/GB2018/050824 patent/WO2018185468A1/en not_active Ceased
- 2018-03-28 PL PL18715911T patent/PL3606546T3/pl unknown
- 2018-03-28 ES ES18715911T patent/ES2881176T3/es active Active
- 2018-03-28 KR KR1020197032606A patent/KR102616629B1/ko active Active
- 2018-03-28 US US16/497,412 patent/US20200046850A1/en not_active Abandoned
- 2018-03-28 AU AU2018248651A patent/AU2018248651B2/en active Active
- 2018-03-28 CA CA3058549A patent/CA3058549A1/en active Pending
- 2018-03-28 EP EP18715911.6A patent/EP3606546B1/en active Active
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