JP2017513500A5 - - Google Patents
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- Publication number
- JP2017513500A5 JP2017513500A5 JP2016564019A JP2016564019A JP2017513500A5 JP 2017513500 A5 JP2017513500 A5 JP 2017513500A5 JP 2016564019 A JP2016564019 A JP 2016564019A JP 2016564019 A JP2016564019 A JP 2016564019A JP 2017513500 A5 JP2017513500 A5 JP 2017513500A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- raav
- protein
- pharmaceutical composition
- acid vector
- 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
- 108020004707 nucleic acids Proteins 0.000 claims 27
- 102000039446 nucleic acids Human genes 0.000 claims 27
- 150000007523 nucleic acids Chemical class 0.000 claims 27
- 239000013598 vector Substances 0.000 claims 15
- 102000002233 Myelin-Oligodendrocyte Glycoprotein Human genes 0.000 claims 11
- 108010000123 Myelin-Oligodendrocyte Glycoprotein Proteins 0.000 claims 11
- 241000124008 Mammalia Species 0.000 claims 10
- 239000008194 pharmaceutical composition Substances 0.000 claims 10
- KISWVXRQTGLFGD-UHFFFAOYSA-N 2-[[2-[[6-amino-2-[[2-[[2-[[5-amino-2-[[2-[[1-[2-[[6-amino-2-[(2,5-diamino-5-oxopentanoyl)amino]hexanoyl]amino]-5-(diaminomethylideneamino)pentanoyl]pyrrolidine-2-carbonyl]amino]-3-hydroxypropanoyl]amino]-5-oxopentanoyl]amino]-5-(diaminomethylideneamino)p Chemical compound C1CCN(C(=O)C(CCCN=C(N)N)NC(=O)C(CCCCN)NC(=O)C(N)CCC(N)=O)C1C(=O)NC(CO)C(=O)NC(CCC(N)=O)C(=O)NC(CCCN=C(N)N)C(=O)NC(CO)C(=O)NC(CCCCN)C(=O)NC(C(=O)NC(CC(C)C)C(O)=O)CC1=CC=C(O)C=C1 KISWVXRQTGLFGD-UHFFFAOYSA-N 0.000 claims 6
- 208000023275 Autoimmune disease Diseases 0.000 claims 6
- 102000016202 Proteolipids Human genes 0.000 claims 6
- 108010010974 Proteolipids Proteins 0.000 claims 6
- 230000001225 therapeutic effect Effects 0.000 claims 5
- 241001465754 Metazoa Species 0.000 claims 4
- 239000003112 inhibitor Substances 0.000 claims 3
- 201000006417 multiple sclerosis Diseases 0.000 claims 3
- 239000002245 particle Substances 0.000 claims 3
- 108090000765 processed proteins & peptides Proteins 0.000 claims 3
- 108020003175 receptors Proteins 0.000 claims 3
- 102000005962 receptors Human genes 0.000 claims 3
- 208000024891 symptom Diseases 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
- 102000008847 Serpin Human genes 0.000 claims 2
- 108050000761 Serpin Proteins 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 150000001413 amino acids Chemical class 0.000 claims 2
- -1 antibody Proteins 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 239000003102 growth factor Substances 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 108091033319 polynucleotide Proteins 0.000 claims 2
- 102000040430 polynucleotide Human genes 0.000 claims 2
- 239000002157 polynucleotide Substances 0.000 claims 2
- 239000003001 serine protease inhibitor Substances 0.000 claims 2
- QZAYGJVTTNCVMB-UHFFFAOYSA-N serotonin Chemical compound C1=C(O)C=C2C(CCN)=CNC2=C1 QZAYGJVTTNCVMB-UHFFFAOYSA-N 0.000 claims 2
- 229940124597 therapeutic agent Drugs 0.000 claims 2
- 102000004031 Carboxy-Lyases Human genes 0.000 claims 1
- 108090000489 Carboxy-Lyases Proteins 0.000 claims 1
- 102000014914 Carrier Proteins Human genes 0.000 claims 1
- 108010078791 Carrier Proteins Proteins 0.000 claims 1
- 102000053642 Catalytic RNA Human genes 0.000 claims 1
- 108090000994 Catalytic RNA Proteins 0.000 claims 1
- 101710112752 Cytotoxin Proteins 0.000 claims 1
- 208000016192 Demyelinating disease Diseases 0.000 claims 1
- 206010012305 Demyelination Diseases 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 102000003886 Glycoproteins Human genes 0.000 claims 1
- 108090000288 Glycoproteins Proteins 0.000 claims 1
- 101001115699 Homo sapiens Myelin-oligodendrocyte glycoprotein Proteins 0.000 claims 1
- 206010061218 Inflammation Diseases 0.000 claims 1
- 102000005755 Intercellular Signaling Peptides and Proteins Human genes 0.000 claims 1
- 108010070716 Intercellular Signaling Peptides and Proteins Proteins 0.000 claims 1
- 102000001617 Interferon Receptors Human genes 0.000 claims 1
- 102000014150 Interferons Human genes 0.000 claims 1
- 108010050904 Interferons Proteins 0.000 claims 1
- 102000015696 Interleukins Human genes 0.000 claims 1
- 108010063738 Interleukins Proteins 0.000 claims 1
- 102000047918 Myelin Basic Human genes 0.000 claims 1
- 102000006386 Myelin Proteins Human genes 0.000 claims 1
- 108010083674 Myelin Proteins Proteins 0.000 claims 1
- 101710107068 Myelin basic protein Proteins 0.000 claims 1
- 102000007339 Nerve Growth Factor Receptors Human genes 0.000 claims 1
- 108010032605 Nerve Growth Factor Receptors Proteins 0.000 claims 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 1
- 108091005804 Peptidases Proteins 0.000 claims 1
- 108091093037 Peptide nucleic acid Proteins 0.000 claims 1
- 101710093543 Probable non-specific lipid-transfer protein Proteins 0.000 claims 1
- 239000004365 Protease Substances 0.000 claims 1
- 229940124158 Protease/peptidase inhibitor Drugs 0.000 claims 1
- 102000001253 Protein Kinase Human genes 0.000 claims 1
- 108091030071 RNAI Proteins 0.000 claims 1
- 101710146873 Receptor-binding protein Proteins 0.000 claims 1
- 102100037486 Reverse transcriptase/ribonuclease H Human genes 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 102000001742 Tumor Suppressor Proteins Human genes 0.000 claims 1
- 108010040002 Tumor Suppressor Proteins Proteins 0.000 claims 1
- 239000000556 agonist Substances 0.000 claims 1
- 239000005557 antagonist Substances 0.000 claims 1
- 230000002424 anti-apoptotic effect Effects 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- 239000000427 antigen Substances 0.000 claims 1
- 102000036639 antigens Human genes 0.000 claims 1
- 108091007433 antigens Proteins 0.000 claims 1
- 239000002246 antineoplastic agent Substances 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 102000003675 cytokine receptors Human genes 0.000 claims 1
- 108010057085 cytokine receptors Proteins 0.000 claims 1
- 229940127089 cytotoxic agent Drugs 0.000 claims 1
- 231100000599 cytotoxic agent Toxicity 0.000 claims 1
- 239000002619 cytotoxin Substances 0.000 claims 1
- 201000002491 encephalomyelitis Diseases 0.000 claims 1
- 239000012645 endogenous antigen Substances 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 230000010437 erythropoiesis Effects 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 230000009368 gene silencing by RNA Effects 0.000 claims 1
- 239000005556 hormone Substances 0.000 claims 1
- 229940088597 hormone Drugs 0.000 claims 1
- 108091008039 hormone receptors Proteins 0.000 claims 1
- 102000047972 human MOG Human genes 0.000 claims 1
- 230000006058 immune tolerance Effects 0.000 claims 1
- 208000027866 inflammatory disease Diseases 0.000 claims 1
- 230000004054 inflammatory process Effects 0.000 claims 1
- 229940079322 interferon Drugs 0.000 claims 1
- 229940043355 kinase inhibitor Drugs 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000004962 mammalian cell Anatomy 0.000 claims 1
- 210000005012 myelin Anatomy 0.000 claims 1
- 239000000137 peptide hydrolase inhibitor Substances 0.000 claims 1
- 108010011903 peptide receptors Proteins 0.000 claims 1
- 102000014187 peptide receptors Human genes 0.000 claims 1
- 230000008488 polyadenylation Effects 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108060006633 protein kinase Proteins 0.000 claims 1
- 239000003909 protein kinase inhibitor Substances 0.000 claims 1
- 239000013608 rAAV vector Substances 0.000 claims 1
- 210000003289 regulatory T cell Anatomy 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 108091092562 ribozyme Proteins 0.000 claims 1
- 229940076279 serotonin Drugs 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019235107A JP7430898B2 (ja) | 2014-04-24 | 2019-12-25 | 多発性硬化症のためのａａｖ系遺伝子療法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461983924P | 2014-04-24 | 2014-04-24 | |
| US61/983,924 | 2014-04-24 | ||
| PCT/US2015/027598 WO2015164789A1 (en) | 2014-04-24 | 2015-04-24 | Aav-based gene therapy for multiple sclerosis |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019235107A Division JP7430898B2 (ja) | 2014-04-24 | 2019-12-25 | 多発性硬化症のためのａａｖ系遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017513500A JP2017513500A (ja) | 2017-06-01 |
| JP2017513500A5 true JP2017513500A5 (enExample) | 2018-07-26 |
| JP6646282B2 JP6646282B2 (ja) | 2020-02-14 |
Family
ID=54333299
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016564019A Active JP6646282B2 (ja) | 2014-04-24 | 2015-04-24 | 多発性硬化症のためのａａｖ系遺伝子療法 |
| JP2019235107A Active JP7430898B2 (ja) | 2014-04-24 | 2019-12-25 | 多発性硬化症のためのａａｖ系遺伝子療法 |
| JP2022031064A Pending JP2022081549A (ja) | 2014-04-24 | 2022-03-01 | 多発性硬化症のためのａａｖ系遺伝子療法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019235107A Active JP7430898B2 (ja) | 2014-04-24 | 2019-12-25 | 多発性硬化症のためのａａｖ系遺伝子療法 |
| JP2022031064A Pending JP2022081549A (ja) | 2014-04-24 | 2022-03-01 | 多発性硬化症のためのａａｖ系遺伝子療法 |
Country Status (17)
| Country | Link |
|---|---|
| US (3) | US20170043036A1 (enExample) |
| EP (2) | EP3744177A1 (enExample) |
| JP (3) | JP6646282B2 (enExample) |
| AU (2) | AU2015249328B2 (enExample) |
| CA (1) | CA2945984C (enExample) |
| CY (1) | CY1123312T1 (enExample) |
| DK (1) | DK3133923T3 (enExample) |
| ES (1) | ES2814280T3 (enExample) |
| HR (1) | HRP20201342T1 (enExample) |
| HU (1) | HUE050681T2 (enExample) |
| LT (1) | LT3133923T (enExample) |
| PL (1) | PL3133923T3 (enExample) |
| PT (1) | PT3133923T (enExample) |
| RS (1) | RS60816B1 (enExample) |
| SI (1) | SI3133923T1 (enExample) |
| SM (1) | SMT202000453T1 (enExample) |
| WO (1) | WO2015164789A1 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7270946B2 (ja) * | 2017-08-08 | 2023-05-11 | 国立大学法人浜松医科大学 | 自己免疫疾患の治療又は予防剤 |
| JP2021533805A (ja) * | 2018-08-24 | 2021-12-09 | スパーク セラピューティクス インコーポレイテッドSpark Therapeutics, Inc. | 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法 |
| AU2020289326A1 (en) * | 2019-06-04 | 2022-01-27 | Thomas Jefferson University | Oligodendrocyte-derived extracellular vesicles for therapy of multiple sclerosis |
| US20230263909A1 (en) * | 2020-04-14 | 2023-08-24 | University Of Florida Research Foundation, Incorporated | Enhanced effects of gene-immunotherapy and immunosuppressants in multiple sclerosis |
| EP4135777A4 (en) * | 2020-04-14 | 2025-03-12 | University Of Florida Research Foundation, Incorporated | AAV-BASED GENE THERAPIES FOR THE TREATMENT OF AUTOIMMUNE DISEASES |
| CN116209477A (zh) * | 2020-06-30 | 2023-06-02 | 儿童国家医疗中心 | 使用重组人酸性鞘氨醇酶改善骨骼肌纤维的修复 |
| WO2024081570A1 (en) * | 2022-10-10 | 2024-04-18 | Eli Lilly And Company | Methods and systems for using causal networks to develop models for evaluating biological processes |
| WO2024258925A1 (en) | 2023-06-12 | 2024-12-19 | Children's Hospital Medical Center | Aav-cftr vectors and methods of using same |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU7725894A (en) * | 1993-09-03 | 1995-03-22 | Immulogic Pharmaceutical Corporation | Uses of myelin oligodendrocyte glycoprotein and peptide portions thereof in protocols related to autoimmune disease |
| WO1998024479A1 (en) * | 1996-12-02 | 1998-06-11 | Cell Genesys, Inc. | Adeno-associated viral vector-mediated delivery of dna to cells of the liver |
| ATE270306T1 (de) * | 2000-10-02 | 2004-07-15 | Univ Yonsei Seoul | Einkettige insulinanaloge |
| KR100449454B1 (ko) * | 2000-10-02 | 2004-09-21 | 이현철 | 단일사슬 인슐린 유도체의 유전자를 포함하는 당뇨병치료용 벡터 |
| CA2361462A1 (en) * | 2001-11-07 | 2003-05-08 | Katherine A. High | Induction of tolerance to a therapeutic polypeptide |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| WO2007094003A2 (en) * | 2006-02-15 | 2007-08-23 | Ramot At Tel Aviv University Ltd. | Viral display vehicles for treating multiple sclerosis |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| US9938540B2 (en) * | 2008-11-12 | 2018-04-10 | Ospedale San Raffaele S.R.L. | Gene vector for inducing transgene-specific immune tolerance |
| US9402919B2 (en) * | 2011-03-04 | 2016-08-02 | Intrexon Corporation | Vectors conditionally expressing protein |
| EP2760478A1 (en) * | 2011-09-26 | 2014-08-06 | Universität Zürich Prorektorat MNW | Apc-mediated tolerance induction for therapy of multiple sclerosis |
| ES2719691T3 (es) * | 2013-11-22 | 2019-07-12 | Amarna Holding B V | Procedimiento de restauración de tolerancia inmunitaria in vivo |
| EP4135777A4 (en) * | 2020-04-14 | 2025-03-12 | University Of Florida Research Foundation, Incorporated | AAV-BASED GENE THERAPIES FOR THE TREATMENT OF AUTOIMMUNE DISEASES |
-
2015
- 2015-04-24 DK DK15782332.9T patent/DK3133923T3/da active
- 2015-04-24 PT PT157823329T patent/PT3133923T/pt unknown
- 2015-04-24 SM SM20200453T patent/SMT202000453T1/it unknown
- 2015-04-24 PL PL15782332.9T patent/PL3133923T3/pl unknown
- 2015-04-24 HU HUE15782332A patent/HUE050681T2/hu unknown
- 2015-04-24 CA CA2945984A patent/CA2945984C/en active Active
- 2015-04-24 LT LTEP15782332.9T patent/LT3133923T/lt unknown
- 2015-04-24 WO PCT/US2015/027598 patent/WO2015164789A1/en not_active Ceased
- 2015-04-24 HR HRP20201342TT patent/HRP20201342T1/hr unknown
- 2015-04-24 RS RS20201008A patent/RS60816B1/sr unknown
- 2015-04-24 ES ES15782332T patent/ES2814280T3/es active Active
- 2015-04-24 EP EP20167323.3A patent/EP3744177A1/en active Pending
- 2015-04-24 SI SI201531336T patent/SI3133923T1/sl unknown
- 2015-04-24 AU AU2015249328A patent/AU2015249328B2/en not_active Ceased
- 2015-04-24 JP JP2016564019A patent/JP6646282B2/ja active Active
- 2015-04-24 US US15/306,163 patent/US20170043036A1/en not_active Abandoned
- 2015-04-24 EP EP15782332.9A patent/EP3133923B1/en active Active
-
2019
- 2019-04-17 AU AU2019202958A patent/AU2019202958B2/en not_active Ceased
- 2019-11-08 US US16/679,102 patent/US20200138978A1/en not_active Abandoned
- 2019-12-25 JP JP2019235107A patent/JP7430898B2/ja active Active
-
2020
- 2020-09-02 CY CY20201100820T patent/CY1123312T1/el unknown
-
2022
- 2022-03-01 JP JP2022031064A patent/JP2022081549A/ja active Pending
-
2023
- 2023-03-28 US US18/191,790 patent/US20230381342A1/en active Pending
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