JP2017536092A5 - - Google Patents
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- JP2017536092A5 JP2017536092A5 JP2017517791A JP2017517791A JP2017536092A5 JP 2017536092 A5 JP2017536092 A5 JP 2017536092A5 JP 2017517791 A JP2017517791 A JP 2017517791A JP 2017517791 A JP2017517791 A JP 2017517791A JP 2017536092 A5 JP2017536092 A5 JP 2017536092A5
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- JP
- Japan
- Prior art keywords
- seq
- hepatitis
- nucleic acid
- composition
- lipid
- 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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- 239000002245 particle Substances 0.000 claims description 116
- 239000000203 mixture Substances 0.000 claims description 90
- 241000700721 Hepatitis B virus Species 0.000 claims description 48
- 241000124008 Mammalia Species 0.000 claims description 42
- 239000008194 pharmaceutical composition Substances 0.000 claims description 40
- 108020004459 Small interfering RNA Proteins 0.000 claims description 38
- 238000000034 method Methods 0.000 claims description 34
- 208000029570 hepatitis D virus infection Diseases 0.000 claims description 33
- 210000004027 cell Anatomy 0.000 claims description 32
- 208000002672 hepatitis B Diseases 0.000 claims description 32
- 230000014509 gene expression Effects 0.000 claims description 26
- 108090000623 proteins and genes Proteins 0.000 claims description 26
- 150000002632 lipids Chemical class 0.000 claims description 21
- 230000030279 gene silencing Effects 0.000 claims description 17
- 208000024891 symptom Diseases 0.000 claims description 17
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims description 16
- 150000003904 phospholipids Chemical class 0.000 claims description 14
- 239000003814 drug Substances 0.000 claims description 13
- -1 cationic lipid Chemical class 0.000 claims description 12
- 208000037262 Hepatitis delta Diseases 0.000 claims description 11
- 241000724709 Hepatitis delta virus Species 0.000 claims description 11
- 108020004707 nucleic acids Proteins 0.000 claims description 10
- 150000007523 nucleic acids Chemical class 0.000 claims description 10
- 102000039446 nucleic acids Human genes 0.000 claims description 10
- 235000012000 cholesterol Nutrition 0.000 claims description 8
- 150000001875 compounds Chemical class 0.000 claims description 8
- 208000019423 liver disease Diseases 0.000 claims description 8
- 230000009885 systemic effect Effects 0.000 claims description 8
- KILNVBDSWZSGLL-KXQOOQHDSA-N 1,2-dihexadecanoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCC KILNVBDSWZSGLL-KXQOOQHDSA-N 0.000 claims description 6
- NRJAVPSFFCBXDT-HUESYALOSA-N 1,2-distearoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCCCC NRJAVPSFFCBXDT-HUESYALOSA-N 0.000 claims description 6
- 238000004220 aggregation Methods 0.000 claims description 6
- 230000002776 aggregation Effects 0.000 claims description 6
- 239000000427 antigen Substances 0.000 claims description 5
- 108091007433 antigens Proteins 0.000 claims description 5
- 102000036639 antigens Human genes 0.000 claims description 5
- 230000015572 biosynthetic process Effects 0.000 claims description 5
- 238000002360 preparation method Methods 0.000 claims description 5
- 238000003786 synthesis reaction Methods 0.000 claims description 5
- 239000002202 Polyethylene glycol Substances 0.000 claims description 4
- 125000002091 cationic group Chemical group 0.000 claims description 4
- 210000004962 mammalian cell Anatomy 0.000 claims description 4
- 229920001223 polyethylene glycol Polymers 0.000 claims description 4
- 241000700605 Viruses Species 0.000 claims description 3
- 208000006454 hepatitis Diseases 0.000 claims description 3
- 231100000283 hepatitis Toxicity 0.000 claims description 3
- 230000002401 inhibitory effect Effects 0.000 claims description 3
- 230000010076 replication Effects 0.000 claims description 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 description 2
- FERIUCNNQQJTOY-UHFFFAOYSA-M Butyrate Chemical compound CCCC([O-])=O FERIUCNNQQJTOY-UHFFFAOYSA-M 0.000 claims description 2
- ARJCMRWHZPDQJG-ASSJVFBPSA-N [(6z,16z)-12-[(z)-dec-4-enyl]docosa-6,16-dien-11-yl] 5-(dimethylamino)pentanoate Chemical compound CCCCC\C=C/CCCC(CCC\C=C/CCCCC)C(CCC\C=C/CCCCC)OC(=O)CCCCN(C)C ARJCMRWHZPDQJG-ASSJVFBPSA-N 0.000 claims description 2
- 229940106189 ceramide Drugs 0.000 claims description 2
- 150000001841 cholesterols Chemical class 0.000 claims description 2
- VHRYAHCCVUHIBM-NSOMFCEZSA-N n,n-dimethyl-1,2-bis[(6z,9z,12z)-octadeca-6,9,12-trienoxy]propan-1-amine Chemical compound CCCCC\C=C/C\C=C/C\C=C/CCCCCOC(C)C(N(C)C)OCCCCC\C=C/C\C=C/C\C=C/CCCCC VHRYAHCCVUHIBM-NSOMFCEZSA-N 0.000 claims description 2
- XVUQPECVOGMPRU-ZPPAUJSGSA-N n,n-dimethyl-1,2-bis[(9z,12z)-octadeca-9,12-dienoxy]propan-1-amine Chemical compound CCCCC\C=C/C\C=C/CCCCCCCCOC(C)C(N(C)C)OCCCCCCCC\C=C/C\C=C/CCCCC XVUQPECVOGMPRU-ZPPAUJSGSA-N 0.000 claims description 2
- NFQBIAXADRDUGK-KWXKLSQISA-N n,n-dimethyl-2,3-bis[(9z,12z)-octadeca-9,12-dienoxy]propan-1-amine Chemical compound CCCCC\C=C/C\C=C/CCCCCCCCOCC(CN(C)C)OCCCCCCCC\C=C/C\C=C/CCCCC NFQBIAXADRDUGK-KWXKLSQISA-N 0.000 claims description 2
- 125000001436 propyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])[H] 0.000 claims description 2
- 150000003839 salts Chemical class 0.000 claims description 2
- 230000029812 viral genome replication Effects 0.000 claims description 2
- 238000013160 medical therapy Methods 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462059056P | 2014-10-02 | 2014-10-02 | |
| US62/059,056 | 2014-10-02 | ||
| US201562120149P | 2015-02-24 | 2015-02-24 | |
| US62/120,149 | 2015-02-24 | ||
| PCT/US2015/053569 WO2016054421A1 (en) | 2014-10-02 | 2015-10-01 | Compositions and methods for silencing hepatitis b virus gene expression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017536092A JP2017536092A (ja) | 2017-12-07 |
| JP2017536092A5 true JP2017536092A5 (enExample) | 2018-03-15 |
Family
ID=54325078
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017517791A Pending JP2017536092A (ja) | 2014-10-02 | 2015-10-01 | Ｂ型肝炎ウイルスの遺伝子発現をサイレンシングするための組成物及び方法 |
Country Status (17)
| Country | Link |
|---|---|
| US (1) | US10415037B2 (enExample) |
| EP (1) | EP3201338B1 (enExample) |
| JP (1) | JP2017536092A (enExample) |
| KR (1) | KR20170075742A (enExample) |
| CN (1) | CN107208095B (enExample) |
| AU (1) | AU2015328012A1 (enExample) |
| BR (1) | BR112017006679A2 (enExample) |
| CA (1) | CA2963271A1 (enExample) |
| CL (1) | CL2017000780A1 (enExample) |
| CO (1) | CO2017004290A2 (enExample) |
| CR (1) | CR20170174A (enExample) |
| IL (1) | IL251450A0 (enExample) |
| PH (1) | PH12017500614A1 (enExample) |
| RU (1) | RU2017114964A (enExample) |
| SG (1) | SG11201702662UA (enExample) |
| TW (2) | TW201623614A (enExample) |
| WO (1) | WO2016054421A1 (enExample) |
Families Citing this family (51)
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| WO2011000107A1 (en) | 2009-07-01 | 2011-01-06 | Protiva Biotherapeutics, Inc. | Novel lipid formulations for delivery of therapeutic agents to solid tumors |
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| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| WO2013185067A1 (en) | 2012-06-08 | 2013-12-12 | Shire Human Genetic Therapies, Inc. | Nuclease resistant polynucleotides and uses thereof |
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| KR102559979B1 (ko) | 2014-06-24 | 2023-07-25 | 샤이어 휴먼 지네틱 테라피즈 인크. | 핵산의 전달용 입체화학적으로 풍부한 조성물 |
| BR112016030852A2 (pt) | 2014-07-02 | 2018-01-16 | Shire Human Genetic Therapies | encapsulação de rna mensageiro |
| JP6767976B2 (ja) | 2014-12-05 | 2020-10-14 | トランスレイト バイオ， インコーポレイテッド | 関節疾患の治療のためのメッセンジャーｒｎａ治療法 |
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| EA201991747A1 (ru) | 2017-02-27 | 2020-06-04 | Транслейт Био, Инк. | НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR |
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