JP2020506714A5 - - Google Patents
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- JP2020506714A5 JP2020506714A5 JP2019542703A JP2019542703A JP2020506714A5 JP 2020506714 A5 JP2020506714 A5 JP 2020506714A5 JP 2019542703 A JP2019542703 A JP 2019542703A JP 2019542703 A JP2019542703 A JP 2019542703A JP 2020506714 A5 JP2020506714 A5 JP 2020506714A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- nucleic acid
- ids
- apoeii
- 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.)
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- 102000039446 nucleic acids Human genes 0.000 claims 17
- 108020004707 nucleic acids Proteins 0.000 claims 17
- 150000007523 nucleic acids Chemical class 0.000 claims 17
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 claims 15
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 claims 15
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 10
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 8
- 125000005647 linker group Chemical group 0.000 claims 6
- 239000000203 mixture Substances 0.000 claims 6
- 239000008194 pharmaceutical composition Substances 0.000 claims 6
- 108090000623 proteins and genes Proteins 0.000 claims 6
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims 5
- 230000007812 deficiency Effects 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 101710095339 Apolipoprotein E Proteins 0.000 claims 3
- 102100029470 Apolipoprotein E Human genes 0.000 claims 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 3
- 238000010606 normalization Methods 0.000 claims 3
- 230000008499 blood brain barrier function Effects 0.000 claims 2
- 210000001218 blood-brain barrier Anatomy 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 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 1
- 102000007592 Apolipoproteins Human genes 0.000 claims 1
- 108010071619 Apolipoproteins Proteins 0.000 claims 1
- 101150110586 IDS gene Proteins 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 238000001415 gene therapy Methods 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1701968.8A GB201701968D0 (en) | 2017-02-07 | 2017-02-07 | Gene therapy |
| GB1701968.8 | 2017-02-07 | ||
| PCT/GB2018/050347 WO2018146473A1 (en) | 2017-02-07 | 2018-02-07 | Gene therapy |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020506714A JP2020506714A (ja) | 2020-03-05 |
| JP2020506714A5 true JP2020506714A5 (enExample) | 2021-03-25 |
| JP7237364B2 JP7237364B2 (ja) | 2023-03-13 |
Family
ID=58462471
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019542703A Active JP7237364B2 (ja) | 2017-02-07 | 2018-02-07 | 遺伝子治療 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US11701390B2 (enExample) |
| EP (2) | EP3960188B1 (enExample) |
| JP (1) | JP7237364B2 (enExample) |
| ES (2) | ES2895263T3 (enExample) |
| GB (1) | GB201701968D0 (enExample) |
| WO (1) | WO2018146473A1 (enExample) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112020005271A2 (pt) | 2017-10-02 | 2020-09-15 | Denali Therapeutics Inc. | proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica |
| KR20250110842A (ko) | 2022-11-01 | 2025-07-21 | 에라스무스 유니버시티 메디컬 센터 로테르담 | 대사질환을 위한 유전자 요법 구성체 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2004108071A2 (en) | 2003-06-05 | 2004-12-16 | Salk Institute For Biological Studies | Targeting polypeptides to the central nervous system |
| JP2007513188A (ja) | 2003-12-04 | 2007-05-24 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | リソソーム蓄積症の処置のための組成物および方法 |
| US10227387B2 (en) | 2011-05-18 | 2019-03-12 | Children's Hospital Medical Center | Targeted delivery of proteins across the blood-brain barrier |
| RU2014126482A (ru) | 2011-12-01 | 2016-01-27 | Ангиочем Инк. | Нацеленные соединения ферментов и их применение |
| EP3218000A2 (en) * | 2014-11-10 | 2017-09-20 | Alexion Pharmaceuticals, Inc. | Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof |
-
2017
- 2017-02-07 GB GBGB1701968.8A patent/GB201701968D0/en not_active Ceased
-
2018
- 2018-02-07 ES ES18705462T patent/ES2895263T3/es active Active
- 2018-02-07 WO PCT/GB2018/050347 patent/WO2018146473A1/en not_active Ceased
- 2018-02-07 US US16/484,311 patent/US11701390B2/en active Active
- 2018-02-07 ES ES21187825T patent/ES3035262T3/es active Active
- 2018-02-07 EP EP21187825.1A patent/EP3960188B1/en active Active
- 2018-02-07 EP EP18705462.2A patent/EP3579849B1/en active Active
- 2018-02-07 JP JP2019542703A patent/JP7237364B2/ja active Active
-
2022
- 2022-12-22 US US18/086,719 patent/US20230390340A1/en active Pending
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