JP2015529635A5 - - Google Patents
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- Publication number
- JP2015529635A5 JP2015529635A5 JP2015520410A JP2015520410A JP2015529635A5 JP 2015529635 A5 JP2015529635 A5 JP 2015529635A5 JP 2015520410 A JP2015520410 A JP 2015520410A JP 2015520410 A JP2015520410 A JP 2015520410A JP 2015529635 A5 JP2015529635 A5 JP 2015529635A5
- Authority
- JP
- Japan
- Prior art keywords
- ube3a
- compound
- ats
- seq
- region
- 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
- 150000001875 compounds Chemical class 0.000 claims description 168
- 101000772888 Homo sapiens Ubiquitin-protein ligase E3A Proteins 0.000 claims description 114
- 102100030434 Ubiquitin-protein ligase E3A Human genes 0.000 claims description 114
- 108091060203 Ube3a-ATS Proteins 0.000 claims description 106
- 230000000692 anti-sense effect Effects 0.000 claims description 88
- 108091034117 Oligonucleotide Proteins 0.000 claims description 86
- 150000007523 nucleic acids Chemical group 0.000 claims description 62
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 60
- 230000000295 complement effect Effects 0.000 claims description 55
- 108020004999 messenger RNA Proteins 0.000 claims description 53
- 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 description 36
- 230000001939 inductive effect Effects 0.000 claims description 35
- 238000011144 upstream manufacturing Methods 0.000 claims description 26
- 230000008685 targeting Effects 0.000 claims description 21
- 239000002777 nucleoside Substances 0.000 claims description 12
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 8
- 125000004400 (C1-C12) alkyl group Chemical group 0.000 claims description 4
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 4
- 125000002619 bicyclic group Chemical group 0.000 claims description 4
- 125000003835 nucleoside group Chemical group 0.000 claims description 4
- 125000006239 protecting group Chemical group 0.000 claims description 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 4
- 208000009575 Angelman syndrome Diseases 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 238000004132 cross linking Methods 0.000 claims 1
- 230000008775 paternal effect Effects 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 155
- 210000004027 cell Anatomy 0.000 description 29
- 241001465754 Metazoa Species 0.000 description 28
- 108020003224 Small Nucleolar RNA Proteins 0.000 description 13
- 102000042773 Small Nucleolar RNA Human genes 0.000 description 13
- 108091030111 Small nucleolar RNA SNORD115 Proteins 0.000 description 7
- 230000003834 intracellular effect Effects 0.000 description 3
- 108091032955 Bacterial small RNA Proteins 0.000 description 2
- 102000007999 Nuclear Proteins Human genes 0.000 description 2
- 108010089610 Nuclear Proteins Proteins 0.000 description 2
- 108091033400 Small nucleolar RNA SNORD116 Proteins 0.000 description 2
- 101150069235 Snrpn gene Proteins 0.000 description 2
- 210000004748 cultured cell Anatomy 0.000 description 2
- 230000002401 inhibitory effect Effects 0.000 description 2
- 241000699670 Mus sp. Species 0.000 description 1
- 208000003251 Pruritus Diseases 0.000 description 1
- 238000003556 assay Methods 0.000 description 1
- 230000003750 conditioning effect Effects 0.000 description 1
- 230000007423 decrease Effects 0.000 description 1
- 230000001419 dependent effect Effects 0.000 description 1
- 230000035939 shock Effects 0.000 description 1
- 229910001220 stainless steel Inorganic materials 0.000 description 1
- 239000010935 stainless steel Substances 0.000 description 1
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261664083P | 2012-06-25 | 2012-06-25 | |
| US61/664,083 | 2012-06-25 | ||
| US201261738959P | 2012-12-18 | 2012-12-18 | |
| US61/738,959 | 2012-12-18 | ||
| US201361750939P | 2013-01-10 | 2013-01-10 | |
| US61/750,939 | 2013-01-10 | ||
| US201361755617P | 2013-01-23 | 2013-01-23 | |
| US61/755,617 | 2013-01-23 | ||
| US201361772925P | 2013-03-05 | 2013-03-05 | |
| US61/772,925 | 2013-03-05 | ||
| PCT/US2013/047701 WO2014004572A2 (en) | 2012-06-25 | 2013-06-25 | Modulation of ube3a-ats expression |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015529635A JP2015529635A (ja) | 2015-10-08 |
| JP2015529635A5 true JP2015529635A5 (https=) | 2016-08-12 |
| JP6294876B2 JP6294876B2 (ja) | 2018-03-14 |
Family
ID=49784000
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015520410A Active JP6294876B2 (ja) | 2012-06-25 | 2013-06-25 | Ｕｂｅ３ａ−ａｔｓ発現の調節 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US9617539B2 (https=) |
| EP (4) | EP3461895B1 (https=) |
| JP (1) | JP6294876B2 (https=) |
| AU (1) | AU2013280474B2 (https=) |
| CA (1) | CA2877905A1 (https=) |
| DK (2) | DK3461895T3 (https=) |
| ES (3) | ES2987225T3 (https=) |
| HU (1) | HUE051698T2 (https=) |
| LT (1) | LT3461895T (https=) |
| PL (1) | PL3461895T3 (https=) |
| PT (1) | PT3461895T (https=) |
| SI (1) | SI3461895T1 (https=) |
| WO (1) | WO2014004572A2 (https=) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014004572A2 (en) | 2012-06-25 | 2014-01-03 | Isis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| US10400243B2 (en) | 2014-11-25 | 2019-09-03 | Ionis Pharmaceuticals, Inc. | Modulation of UBE3A-ATS expression |
| EP3291843B1 (en) | 2015-05-07 | 2023-03-22 | University of South Florida | Modified ube3a gene for a gene therapy approach for angelman syndrome |
| LT3374509T (lt) * | 2015-11-12 | 2021-03-10 | F. Hoffmann-La Roche Ag | Oligonukleotidai, skirti sukelti iš tėvo paveldėto ube3a raišką |
| CA3043768A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| KR102723989B1 (ko) | 2017-12-01 | 2024-11-01 | 더 텍사스 에이 & 엠 유니버시티 시스템 | 엔젤만 증후군 안티센스 치료 |
| WO2019168950A1 (en) * | 2018-02-27 | 2019-09-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for treating angelman syndrome |
| KR20210040358A (ko) | 2018-06-14 | 2021-04-13 | 오비드 테라퓨틱스 인크. | 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용 |
| WO2020061528A1 (en) * | 2018-09-21 | 2020-03-26 | University Of Connecticut | Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome |
| EP3911957A1 (en) * | 2019-01-17 | 2021-11-24 | F. Hoffmann-La Roche AG | E3 ubiquitin ligase (ube3a) protein targets |
| PT3947684T (pt) * | 2019-03-29 | 2025-05-19 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular ube3a‑ats |
| US12465655B2 (en) * | 2019-05-22 | 2025-11-11 | The University Of North Carolina At Chapel Hill | UBE3A genes and expression cassettes and their use |
| WO2021011802A1 (en) * | 2019-07-16 | 2021-01-21 | The University Of North Carolina At Chapel Hill | Methods and compositions for unsilencing imprinted genes |
| GB201912103D0 (en) * | 2019-08-22 | 2019-10-09 | Univ Oxford Innovation Ltd | Method of haplotyping |
| US20230042176A1 (en) | 2019-12-18 | 2023-02-09 | National University Of Singapore | Method for treating angelman syndrome and related disorders |
| JP2023524247A (ja) * | 2020-04-28 | 2023-06-09 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アンジェルマン症候群の治療のための組成物及びその使用 |
| WO2022093835A1 (en) * | 2020-10-26 | 2022-05-05 | Remix Therapeutics Inc. | Oligonucleotides useful for modulation of splicing |
| US20240318198A1 (en) * | 2021-02-03 | 2024-09-26 | The Board Of Regents Of The University Of Texas System | Gene therapy for angelman syndrome |
| US20240240179A1 (en) * | 2021-05-05 | 2024-07-18 | The General Hospital Corporation | Therapy for treatment of prader-willi syndrome |
| WO2023168000A1 (en) * | 2022-03-03 | 2023-09-07 | The Regents Of The University Of California | Compositions and methods for treatment of angelman syndrome |
| JP2025518138A (ja) | 2022-05-27 | 2025-06-12 | ゼロ カーボン システムズ インコーポレイテッド | ハイスループット可動パネル直接空気回収システム |
| WO2025101533A1 (en) * | 2023-11-09 | 2025-05-15 | Encoded Therapeutics, Inc. | Methods and compositions for increasing expression of ube3a |
| WO2025101991A1 (en) * | 2023-11-09 | 2025-05-15 | Ionis Pharmaceuticals, Inc. | Methods for modulating ube3a-ats |
| WO2025213123A1 (en) * | 2024-04-05 | 2025-10-09 | Encoded Therapeutics, Inc. | Methods and compositions for increasing expression of ube3a |
| WO2025217466A1 (en) * | 2024-04-11 | 2025-10-16 | Ultragenyx Pharmaceutical Inc. | Dosing regimens for antisense treatment of angelman syndrome |
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-
2013
- 2013-06-25 WO PCT/US2013/047701 patent/WO2014004572A2/en not_active Ceased
- 2013-06-25 AU AU2013280474A patent/AU2013280474B2/en active Active
- 2013-06-25 DK DK18188705.0T patent/DK3461895T3/da active
- 2013-06-25 LT LTEP18188705.0T patent/LT3461895T/lt unknown
- 2013-06-25 ES ES20182895T patent/ES2987225T3/es active Active
- 2013-06-25 HU HUE18188705A patent/HUE051698T2/hu unknown
- 2013-06-25 SI SI201331770T patent/SI3461895T1/sl unknown
- 2013-06-25 ES ES13809694.6T patent/ES2688831T3/es active Active
- 2013-06-25 EP EP18188705.0A patent/EP3461895B1/en not_active Revoked
- 2013-06-25 EP EP13809694.6A patent/EP2864479B1/en not_active Revoked
- 2013-06-25 CA CA2877905A patent/CA2877905A1/en active Pending
- 2013-06-25 JP JP2015520410A patent/JP6294876B2/ja active Active
- 2013-06-25 EP EP20182895.1A patent/EP3770258B1/en active Active
- 2013-06-25 US US14/409,348 patent/US9617539B2/en active Active
- 2013-06-25 PT PT181887050T patent/PT3461895T/pt unknown
- 2013-06-25 EP EP24188665.4A patent/EP4458361A3/en active Pending
- 2013-06-25 PL PL18188705T patent/PL3461895T3/pl unknown
- 2013-06-25 DK DK13809694.6T patent/DK2864479T3/en active
- 2013-06-25 ES ES18188705T patent/ES2809199T3/es active Active
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