JPWO2022018171A5 - - Google Patents
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- Publication number
- JPWO2022018171A5 JPWO2022018171A5 JP2023504517A JP2023504517A JPWO2022018171A5 JP WO2022018171 A5 JPWO2022018171 A5 JP WO2022018171A5 JP 2023504517 A JP2023504517 A JP 2023504517A JP 2023504517 A JP2023504517 A JP 2023504517A JP WO2022018171 A5 JPWO2022018171 A5 JP WO2022018171A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- eef1a2
- polynucleotide sequence
- functional variant
- sequence encoding
- 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
- 108091033319 polynucleotide Proteins 0.000 claims description 81
- 102000040430 polynucleotide Human genes 0.000 claims description 81
- 239000002157 polynucleotide Substances 0.000 claims description 81
- 210000002845 virion Anatomy 0.000 claims description 64
- 238000000034 method Methods 0.000 claims description 36
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 claims description 34
- 101000821100 Homo sapiens Synapsin-1 Proteins 0.000 claims description 30
- 208000012902 Nervous system disease Diseases 0.000 claims description 24
- 239000008194 pharmaceutical composition Substances 0.000 claims description 18
- 230000008488 polyadenylation Effects 0.000 claims description 18
- 108020005345 3' Untranslated Regions Proteins 0.000 claims description 14
- 102000018146 globin Human genes 0.000 claims description 14
- 108060003196 globin Proteins 0.000 claims description 14
- 208000025966 Neurological disease Diseases 0.000 claims description 12
- 108090000623 proteins and genes Proteins 0.000 claims description 10
- 102000004169 proteins and genes Human genes 0.000 claims description 10
- 108010044091 Globulins Proteins 0.000 claims description 8
- 102000006395 Globulins Human genes 0.000 claims description 8
- 210000000234 capsid Anatomy 0.000 claims description 8
- 238000007914 intraventricular administration Methods 0.000 claims description 8
- 210000003205 muscle Anatomy 0.000 claims description 8
- 108050005241 Synapsin-1 Proteins 0.000 claims description 6
- 102000017299 Synapsin-1 Human genes 0.000 claims description 6
- 239000003623 enhancer Substances 0.000 claims description 6
- 206010003805 Autism Diseases 0.000 claims description 4
- 208000020706 Autistic disease Diseases 0.000 claims description 4
- 101150049307 EEF1A2 gene Proteins 0.000 claims description 4
- 201000006347 Intellectual Disability Diseases 0.000 claims description 4
- 241000124008 Mammalia Species 0.000 claims description 4
- 108700019146 Transgenes Proteins 0.000 claims description 4
- 108091023045 Untranslated Region Proteins 0.000 claims description 4
- 230000007423 decrease Effects 0.000 claims description 4
- 206010015037 epilepsy Diseases 0.000 claims description 4
- 230000035772 mutation Effects 0.000 claims description 4
- 230000000926 neurological effect Effects 0.000 claims description 4
- 238000010825 rotarod performance test Methods 0.000 claims description 4
- 241000283690 Bos taurus Species 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims description 2
- 108091036066 Three prime untranslated region Proteins 0.000 claims description 2
- 210000004556 brain Anatomy 0.000 claims description 2
- 210000002569 neuron Anatomy 0.000 claims description 2
- 210000004129 prosencephalon Anatomy 0.000 claims description 2
- 238000010253 intravenous injection Methods 0.000 claims 4
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 238000012512 characterization method Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 description 4
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063055775P | 2020-07-23 | 2020-07-23 | |
| US63/055,775 | 2020-07-23 | ||
| PCT/EP2021/070455 WO2022018171A1 (en) | 2020-07-23 | 2021-07-21 | GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023535576A JP2023535576A (ja) | 2023-08-18 |
| JPWO2022018171A5 true JPWO2022018171A5 (enExample) | 2024-07-30 |
Family
ID=71786970
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023504517A Pending JP2023535576A (ja) | 2020-07-23 | 2021-07-21 | ｅＥＦ１Ａ２の遺伝子治療ベクターおよびその使用 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20230330265A1 (enExample) |
| EP (1) | EP4185332A1 (enExample) |
| JP (1) | JP2023535576A (enExample) |
| KR (1) | KR20230039669A (enExample) |
| CN (1) | CN116171325A (enExample) |
| AU (1) | AU2021312320A1 (enExample) |
| CA (1) | CA3186700A1 (enExample) |
| IL (1) | IL299955A (enExample) |
| MX (1) | MX2023000995A (enExample) |
| WO (2) | WO2022017630A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7045362B2 (ja) | 2016-04-20 | 2022-03-31 | セントロ デ インベスティガシオンス エネルジェチカス メディオアンビエンタゥス イェ テクノロジカス オー．エイ． エム．ピー． | Ｐｋｌｒの遺伝子発現増強のための組成物および方法 |
| KR102726267B1 (ko) | 2017-10-16 | 2024-11-11 | 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. | 피루베이트 키나제 결핍증을 치료하기 위한 pklr 전달용 렌티바이러스 벡터 |
| US12378576B2 (en) | 2018-04-27 | 2025-08-05 | Spacecraft Seven, Llc | Gene therapy for CNS degeneration |
| CA3185267A1 (en) | 2020-08-07 | 2022-02-10 | Spacecraft Seven, Llc | Plakophilin-2 (pkp2) gene therapy using aav vector |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0755454B1 (en) | 1994-04-13 | 2008-02-13 | The Rockefeller University | Aav-mediated delivery of dna to cells of the nervous system |
| WO2001083692A2 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| SG168422A1 (en) | 2001-11-13 | 2011-02-28 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| US7122350B2 (en) * | 2003-12-12 | 2006-10-17 | Zymogenetics, Inc. | Methods for enhancing expression of recombinant proteins |
| EP2176283B1 (en) | 2007-07-14 | 2016-11-02 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| AU2011235890B2 (en) | 2010-04-02 | 2014-07-31 | Ceregene, Inc. | Methods for treating Parkinson's disease and other disorders of dopaminergic neurons of the brain |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| EP3564379A1 (en) | 2013-09-13 | 2019-11-06 | California Institute of Technology | Selective recovery |
| LT3137497T (lt) | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| JP2018531926A (ja) * | 2015-09-17 | 2018-11-01 | コーダ バイオセラピューティクス， インコーポレイテッド | 神経学的障害を処置するための組成物および方法 |
| WO2017100671A1 (en) | 2015-12-11 | 2017-06-15 | California Institute Of Technology | TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) |
| MX2021000443A (es) * | 2018-07-12 | 2021-05-28 | Spacecraft Seven Llc | Vectores de terapia génica para el tratamiento de la enfermedad de danon. |
-
2020
- 2020-07-24 WO PCT/EP2020/071028 patent/WO2022017630A1/en not_active Ceased
-
2021
- 2021-07-21 CN CN202180056421.4A patent/CN116171325A/zh active Pending
- 2021-07-21 US US18/017,502 patent/US20230330265A1/en active Pending
- 2021-07-21 WO PCT/EP2021/070455 patent/WO2022018171A1/en not_active Ceased
- 2021-07-21 MX MX2023000995A patent/MX2023000995A/es unknown
- 2021-07-21 KR KR1020237003445A patent/KR20230039669A/ko active Pending
- 2021-07-21 IL IL299955A patent/IL299955A/en unknown
- 2021-07-21 JP JP2023504517A patent/JP2023535576A/ja active Pending
- 2021-07-21 EP EP21755896.4A patent/EP4185332A1/en active Pending
- 2021-07-21 CA CA3186700A patent/CA3186700A1/en active Pending
- 2021-07-21 AU AU2021312320A patent/AU2021312320A1/en active Pending
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