JP2021500049A5 - - Google Patents
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- JP2021500049A5 JP2021500049A5 JP2020523011A JP2020523011A JP2021500049A5 JP 2021500049 A5 JP2021500049 A5 JP 2021500049A5 JP 2020523011 A JP2020523011 A JP 2020523011A JP 2020523011 A JP2020523011 A JP 2020523011A JP 2021500049 A5 JP2021500049 A5 JP 2021500049A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- isolated nucleic
- vector
- set forth
- 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.)
- Granted
Links
- 150000007523 nucleic acids Chemical class 0.000 claims 40
- 108020004707 nucleic acids Proteins 0.000 claims 37
- 102000039446 nucleic acids Human genes 0.000 claims 37
- 230000002401 inhibitory effect Effects 0.000 claims 23
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 20
- 238000002347 injection Methods 0.000 claims 12
- 239000007924 injection Substances 0.000 claims 12
- 239000013598 vector Substances 0.000 claims 11
- 102000043334 C9orf72 Human genes 0.000 claims 8
- 108700030955 C9orf72 Proteins 0.000 claims 8
- 102100033342 Lysosomal acid glucosylceramidase Human genes 0.000 claims 8
- 108010017544 Glucosylceramidase Proteins 0.000 claims 6
- 230000000694 effects Effects 0.000 claims 6
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 5
- 101150014718 C9orf72 gene Proteins 0.000 claims 5
- 108090000565 Capsid Proteins Proteins 0.000 claims 5
- 102100023321 Ceruloplasmin Human genes 0.000 claims 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 4
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 108010016626 Dipeptides Proteins 0.000 claims 4
- 102000007370 Ataxin2 Human genes 0.000 claims 3
- 108010032951 Ataxin2 Proteins 0.000 claims 3
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 3
- 101000834926 Homo sapiens Transmembrane protein 106B Proteins 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 102100026232 Transmembrane protein 106B Human genes 0.000 claims 3
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 3
- 210000003169 central nervous system Anatomy 0.000 claims 3
- 230000000295 complement effect Effects 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 3
- 230000004770 neurodegeneration Effects 0.000 claims 3
- 208000015122 neurodegenerative disease Diseases 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- 108091026890 Coding region Proteins 0.000 claims 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 2
- 101000997662 Homo sapiens Lysosomal acid glucosylceramidase Proteins 0.000 claims 2
- 108091023045 Untranslated Region Proteins 0.000 claims 2
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims 2
- 210000002569 neuron Anatomy 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 239000013603 viral vector Substances 0.000 claims 2
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims 1
- 108020005345 3' Untranslated Regions Proteins 0.000 claims 1
- 108020003589 5' Untranslated Regions Proteins 0.000 claims 1
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 208000015872 Gaucher disease Diseases 0.000 claims 1
- 101000934372 Homo sapiens Macrosialin Proteins 0.000 claims 1
- 208000009829 Lewy Body Disease Diseases 0.000 claims 1
- 201000002832 Lewy body dementia Diseases 0.000 claims 1
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 1
- 102100025136 Macrosialin Human genes 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 108091027967 Small hairpin RNA Proteins 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 108091036066 Three prime untranslated region Proteins 0.000 claims 1
- 230000008499 blood brain barrier function Effects 0.000 claims 1
- 210000001218 blood-brain barrier Anatomy 0.000 claims 1
- 230000015556 catabolic process Effects 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 238000006731 degradation reaction Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000007914 intraventricular administration Methods 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 108091070501 miRNA Proteins 0.000 claims 1
- 239000002679 microRNA Substances 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 239000002924 silencing RNA Substances 0.000 claims 1
- 239000004055 small Interfering RNA Substances 0.000 claims 1
- 241000701447 unidentified baculovirus Species 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023220260A JP2024045131A (ja) | 2017-10-23 | 2023-12-27 | 神経変性疾患の遺伝子治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762575795P | 2017-10-23 | 2017-10-23 | |
| US62/575,795 | 2017-10-23 | ||
| US201862742723P | 2018-10-08 | 2018-10-08 | |
| US62/742,723 | 2018-10-08 | ||
| PCT/US2018/057187 WO2019084068A1 (en) | 2017-10-23 | 2018-10-23 | GENE THERAPIES FOR NEURODEGENERATIVE DISEASE |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023220260A Division JP2024045131A (ja) | 2017-10-23 | 2023-12-27 | 神経変性疾患の遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021500049A JP2021500049A (ja) | 2021-01-07 |
| JP2021500049A5 true JP2021500049A5 (enExample) | 2021-11-25 |
| JP7413256B2 JP7413256B2 (ja) | 2024-01-15 |
Family
ID=66247008
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020523011A Active JP7413256B2 (ja) | 2017-10-23 | 2018-10-23 | 神経変性疾患の遺伝子治療 |
| JP2023220260A Pending JP2024045131A (ja) | 2017-10-23 | 2023-12-27 | 神経変性疾患の遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023220260A Pending JP2024045131A (ja) | 2017-10-23 | 2023-12-27 | 神経変性疾患の遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20210261981A1 (enExample) |
| EP (1) | EP3701030A4 (enExample) |
| JP (2) | JP7413256B2 (enExample) |
| KR (1) | KR20200075865A (enExample) |
| CN (2) | CN111819281B (enExample) |
| AU (1) | AU2018354195A1 (enExample) |
| BR (1) | BR112020008033A2 (enExample) |
| CA (2) | CA3083582A1 (enExample) |
| IL (2) | IL274129A (enExample) |
| MX (1) | MX2020004207A (enExample) |
| WO (1) | WO2019084068A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7361037B2 (ja) | 2017-10-03 | 2023-10-13 | プリベイル セラピューティクス，インコーポレーテッド | ライソゾーム病の遺伝子治療 |
| US11802294B2 (en) * | 2017-10-03 | 2023-10-31 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR20200078513A (ko) | 2017-10-03 | 2020-07-01 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| US20210000929A1 (en) * | 2018-03-09 | 2021-01-07 | Avrobio, Inc. | Compositions and methods for treating parkinson's disease |
| CN114174324A (zh) | 2019-04-10 | 2022-03-11 | 普利维尔治疗公司 | 用于溶酶体病症的基因疗法 |
| BR112021019880A2 (pt) | 2019-04-10 | 2022-02-15 | Prevail Therapeutics Inc | Terapias gênicas para distúrbios lisossomais |
| WO2021081236A1 (en) * | 2019-10-22 | 2021-04-29 | Applied Genetic Technologies Corporation | Triple function adeno-associated virus (aav) vectors for the treatment of c9orf72 associated diseases |
| US20240294906A1 (en) * | 2020-07-29 | 2024-09-05 | Alnylam Pharmaceuticals, Inc. | Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases |
| CN116710567A (zh) | 2020-10-15 | 2023-09-05 | 普利维尔治疗公司 | 重组腺相关病毒组合物及其产生方法 |
| WO2022081985A1 (en) | 2020-10-15 | 2022-04-21 | Prevail Therapeutics, Inc. | Assay for measuring potency of gene therapy drug product |
| JPWO2022097727A1 (enExample) | 2020-11-06 | 2022-05-12 | ||
| KR20220109347A (ko) | 2021-01-28 | 2022-08-04 | 주식회사 에이브레인 | 신경 퇴행성 질환의 치료를 위한 유전자 요법 |
| KR102711715B1 (ko) | 2021-01-28 | 2024-09-30 | 주식회사 에이브레인 | 신경 퇴행성 질환의 치료를 위한 유전자 요법 |
| KR102707250B1 (ko) | 2021-10-28 | 2024-09-13 | 한국해양과학기술원 | 신규 화합물 및 이를 유효성분으로 포함하는 신경염증성 뇌질환 예방 또는 치료용 조성물 |
| WO2023133574A1 (en) * | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for treatment of c9orf72-mediated disorders |
| WO2024257847A1 (ja) | 2023-06-16 | 2024-12-19 | 住友ファーマ株式会社 | Ｒｐｓ２５遺伝子の発現及び／又はその機能を調節するアンチセンスオリゴヌクレオチド |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6204059B1 (en) * | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US20040053876A1 (en) * | 2002-03-26 | 2004-03-18 | The Regents Of The University Of Michigan | siRNAs and uses therof |
| US7829694B2 (en) * | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| WO2004069173A2 (en) * | 2003-01-31 | 2004-08-19 | The Trustees Of The University Of Pennsylvania | Methods for modulating an inflammatory response |
| AU2006210973A1 (en) * | 2005-01-31 | 2006-08-10 | University Of Iowa Research Foundation | Nucleic acid silencing of Huntington's Disease gene |
| WO2010055290A1 (en) * | 2008-11-11 | 2010-05-20 | Oxford Biomedica (Uk) Limited | Method |
| US20110142789A1 (en) * | 2009-12-10 | 2011-06-16 | The Trustees Of The University Of Pennsylvania | Compositions and Methods for the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis |
| EP2678433B1 (en) * | 2011-02-22 | 2017-05-03 | California Institute of Technology | Delivery of proteins using adeno-associated virus (aav) vectors |
| HRP20171254T1 (hr) * | 2012-04-23 | 2018-01-12 | Biomarin Technologies B.V. | Oligonukleotidi koji moduliraju rnk s poboljšanim karakteristikama za liječenje neuromišićnih poremećaja |
| MA38144A1 (fr) * | 2012-11-05 | 2018-08-31 | Genzyme Corp | Compositions et procédés pour le traitement de protéinopathies |
| US20140271550A1 (en) * | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
| RU2748426C2 (ru) * | 2013-10-11 | 2021-05-25 | Ионис Фармасьютикалз, Инк. | Композиции для модуляции экспрессии c90rf72 |
| EP4410805A3 (en) * | 2014-03-18 | 2024-11-27 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| PL3119888T3 (pl) * | 2014-03-19 | 2021-12-20 | Ionis Pharmaceuticals, Inc. | Kompozycje do modulacji ekspresji ataksyny 2 |
| WO2015153760A2 (en) * | 2014-04-01 | 2015-10-08 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a nervous system disorder |
| WO2016024205A1 (en) * | 2014-08-15 | 2016-02-18 | Pfizer Inc. | Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene |
| US10597660B2 (en) * | 2014-11-14 | 2020-03-24 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| WO2016172155A1 (en) * | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
| EP3292206B8 (en) * | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| CA3016314A1 (en) * | 2016-03-02 | 2017-09-08 | Julianne REIDERS | Therapy for frontotemporal dementia |
| EP3518667B1 (en) * | 2016-09-30 | 2024-10-30 | Regeneron Pharmaceuticals, Inc. | Rodents having a hexanucleotide repeat expansion in a c9orf72 locus |
| EP3850098A1 (en) * | 2018-09-12 | 2021-07-21 | uniQure IP B.V. | Rnai induced c9orf72 suppression for the treatment of als/ftd |
-
2018
- 2018-10-23 US US16/758,077 patent/US20210261981A1/en not_active Abandoned
- 2018-10-23 EP EP18870021.5A patent/EP3701030A4/en not_active Withdrawn
- 2018-10-23 CN CN201880081355.4A patent/CN111819281B/zh active Active
- 2018-10-23 CA CA3083582A patent/CA3083582A1/en active Pending
- 2018-10-23 MX MX2020004207A patent/MX2020004207A/es unknown
- 2018-10-23 WO PCT/US2018/057187 patent/WO2019084068A1/en not_active Ceased
- 2018-10-23 AU AU2018354195A patent/AU2018354195A1/en not_active Abandoned
- 2018-10-23 CN CN202110196181.XA patent/CN113005123A/zh active Pending
- 2018-10-23 JP JP2020523011A patent/JP7413256B2/ja active Active
- 2018-10-23 BR BR112020008033-0A patent/BR112020008033A2/pt not_active Application Discontinuation
- 2018-10-23 KR KR1020207014683A patent/KR20200075865A/ko active Pending
- 2018-10-23 CA CA3177979A patent/CA3177979A1/en active Pending
-
2020
- 2020-04-22 IL IL274129A patent/IL274129A/en unknown
-
2021
- 2021-02-23 IL IL281057A patent/IL281057A/en unknown
-
2023
- 2023-12-27 JP JP2023220260A patent/JP2024045131A/ja active Pending
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