RU2019101298A - Антисмысловые олигонуклеотиды для модулирования экспрессии htra1 - Google Patents
Антисмысловые олигонуклеотиды для модулирования экспрессии htra1 Download PDFInfo
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Claims (19)
1. Антисмысловой олигонуклеотид формулы: TsAsTststsascscstsgsgstsTsGsTsT (SEQ ID NO 143), где заглавная буква представляет нуклеозидное звено LNA, строчная буква представляет нуклеозидное звено ДНК, подстрочный символ s представляет фосфоротиоатную межнуклеозидную связь.
2. Антисмысловой олигонуклеотид по п. 1, где все нуклеозиды LNA представляют собой бета-D-окси нуклеозиды LNA.
3. Антисмысловой олигонуклеотид по п. 1, где антисмысловой олигонуклеотид имеет формулу:
4. Фармацевтически приемлемая соль антисмыслового олигонуклотида по любому из пп. 1-3.
5. Фармацевтически приемлемая соль по п. 4, где данная соль представляет собой натриевую соль.
6. Фармацевтически приемлемая соль по п. 4, где данная соль представляет собой калиевую соль.
7. Фармацевтическая композиция, содержащая антисмысловой олигонуклеотид по любому из пп. 1-3; и фармацевтически приемлемый разбавитель, носитель, соль и/или адъювант.
8. Фармацевтическая композиция по п. 7, где данная фармацевтическая композиция содержит фармацевтически приемлемый разбавитель.
9. Фармацевтическая композиция по п. 8, где фармацевтически приемлемый разбавитель представляет собой фосфатно-солевой буферный раствор.
10. Фармацевтическая композиция по любому из пп. 7-9, где олигонуклеотид находится в форме фармацевтически приемлемой соли.
11. Фармацевтическая композиция, содержащая фармацевтически приемлемую соль по любому из пп. 4-6; и фармацевтически приемлемый разбавитель, носитель, соль и/или адъювант.
12. Фармацевтическая композиция по любому из пп. 7-11, в которой фармацевтически приемлемая соль представляет собой натриевую соль или калиевую соль.
13. Антисмысловой олигонуклеотид по любому из пп. 1-3 или фармацевтически приемлемая соль по любому из пп. 4-6, фармацевтическая композиция по любому из пп. 7-12 для применения в медицине.
14. Антисмысловой олигонуклеотид по любому из пп. 1-3 или фармацевтически приемлемая соль по любому из пп. 4-6, или фармацевтическая композиция по любому из пп. 7-12 для применения в лечении макулодистрофии.
15. Применение антисмыслового олигонуклеотида по любому из пп. 1-3 или фармацевтически приемлемой соли по любому из пп. 4-6, или фармацевтической композиции по любому из пп. 7-12 для получения лекарственного средства для лечения или предупреждения макулодистрофии.
16. Способ лечения или предупреждения макулодистрофии у субъекта, страдающего от макулодистрофии, включающий введение субъекту терапевтически эффективного количества антисмыслового олигонуклеотида по любому из пп. 1-3 или фармацевтически приемлемой соли по любому из пп. 4-6, или фармацевтической композиции по любому из пп. 7-12.
17. Антисмысловой олигонуклеотид, фармацевтически приемлемая соль, фармацевтическая композиция по любому из пп. 13-14, применение по пп. 15 или способ по п. 16, где заболевание выбрано из влажной AMD (возрастная макулодистрофия), сухой AMD, географической атрофии и промежуточной dAMD.
18. Способ in vivo или in vitro модулирования экспрессии HTRA1 в клетке-мишени, которая экспрессирует HTRA1, причем указанный способ включает введение в указанную клетку антисмыслового олигонуклеотида, фармацевтически приемлемой соли или фармацевтической композиции в эффективном количестве.
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
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EP16177508 | 2016-07-01 | ||
EP16177508.5 | 2016-07-01 | ||
EP17170129.5 | 2017-05-09 | ||
EP17170129 | 2017-05-09 | ||
PCT/EP2017/065937 WO2018002105A1 (en) | 2016-07-01 | 2017-06-28 | Antisense oligonucleotides for modulating htra1 expression |
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RU2019101298A true RU2019101298A (ru) | 2020-08-03 |
RU2019101298A3 RU2019101298A3 (ru) | 2021-01-26 |
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RU2019101298A RU2019101298A (ru) | 2016-07-01 | 2017-06-28 | Антисмысловые олигонуклеотиды для модулирования экспрессии htra1 |
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US (2) | US10519450B2 (ru) |
EP (1) | EP3478839A1 (ru) |
JP (3) | JP7060525B2 (ru) |
KR (1) | KR20190025970A (ru) |
CN (1) | CN109415732B (ru) |
AU (1) | AU2017289204A1 (ru) |
BR (1) | BR112018077321A2 (ru) |
CA (1) | CA3026855A1 (ru) |
CL (1) | CL2018003698A1 (ru) |
CO (1) | CO2018013240A2 (ru) |
CR (1) | CR20180606A (ru) |
IL (1) | IL263489A (ru) |
MA (1) | MA45620A (ru) |
MX (1) | MX2018015722A (ru) |
PE (1) | PE20190381A1 (ru) |
PH (1) | PH12018502707A1 (ru) |
RU (1) | RU2019101298A (ru) |
SG (2) | SG10202005885VA (ru) |
TW (1) | TW201803990A (ru) |
WO (1) | WO2018002105A1 (ru) |
ZA (1) | ZA201808291B (ru) |
Families Citing this family (10)
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US10526315B2 (en) | 2016-06-21 | 2020-01-07 | Orion Ophthalmology LLC | Carbocyclic prolinamide derivatives |
EP3472149B1 (en) | 2016-06-21 | 2023-08-30 | Orion Ophthalmology LLC | Heterocyclic prolinamide derivatives |
EP3478839A1 (en) | 2016-07-01 | 2019-05-08 | H. Hoffnabb-La Roche Ag | Antisense oligonucleotides for modulating htra1 expression |
EP3728590A1 (en) | 2017-12-22 | 2020-10-28 | Roche Innovation Center Copenhagen A/S | Novel thiophosphoramidites |
TW201929870A (zh) | 2017-12-22 | 2019-08-01 | 丹麥商羅氏創新中心哥本哈根有限公司 | 包含二硫代磷酸酯核苷間連結之寡核苷酸 |
MX2020005754A (es) | 2017-12-22 | 2020-08-20 | Roche Innovation Ct Copenhagen As | Oligonucleotidos gapmeros que comprenden un enlace internucleosido fosforoditioato. |
EP3752612A4 (en) | 2018-02-12 | 2021-11-10 | Ionis Pharmaceuticals, Inc. | MODIFIED COMPOUNDS AND USES THEREOF |
WO2020007772A1 (en) * | 2018-07-02 | 2020-01-09 | Roche Innovation Center Copenhagen A/S | Antisense oligonucleotides targeting gbp-1 |
WO2020109343A1 (en) * | 2018-11-29 | 2020-06-04 | F. Hoffmann-La Roche Ag | Combination therapy for treatment of macular degeneration |
WO2020109344A1 (en) * | 2018-11-29 | 2020-06-04 | F. Hoffmann-La Roche Ag | Occular administration device for antisense oligonucleotides |
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JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
DE04020014T1 (de) | 1997-09-12 | 2006-01-26 | Exiqon A/S | Bi-zyklische - Nukleosid,Nnukleotid und Oligonukleotid-Analoga |
AU5290499A (en) | 1998-08-03 | 2000-02-28 | Novartis Ag | Human htra serine protease |
IL144338A0 (en) | 1999-02-12 | 2002-05-23 | Sankyo Co | Nucleoside and oligonucleotide analogues and pharmaceutical compositions containing the same |
PT1178999E (pt) | 1999-05-04 | 2007-06-26 | Santaris Pharma As | Análogos de l-ribo-lna |
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DK3222722T3 (da) | 2002-11-18 | 2019-06-17 | Roche Innovation Ct Copenhagen As | Antisense-design |
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WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
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AU2007277125B2 (en) | 2006-07-26 | 2013-08-01 | Yale University | Diagnosis and treatment of age related macular degeneration |
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EP2913341A1 (en) * | 2006-12-22 | 2015-09-02 | University of Utah Research Foundation | Method of detecting ocular diseases and pathologic conditions and treatment of same |
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TW200911290A (en) | 2007-07-02 | 2009-03-16 | Alcon Res Ltd | RNAI-mediated inhibition of HTRA1 for treatment of macular degeneration |
ATE538127T1 (de) | 2007-07-05 | 2012-01-15 | Isis Pharmaceuticals Inc | 6-disubstituierte bicyclische nukleinsäureanaloga |
CA2704787A1 (en) | 2007-11-01 | 2009-05-07 | University Of Iowa Research Foundation | Genes and polymorphisms associated with amd |
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DK2356129T3 (da) | 2008-09-24 | 2013-05-13 | Isis Pharmaceuticals Inc | Substituerede alpha-L-bicykliske nukleosider |
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FR2965278B1 (fr) | 2010-09-23 | 2014-10-10 | Univ Caen Basse Normandie | Procede d'obtention in vitro ou ex vivo de chondrocytes et leurs utilisations |
WO2013036868A1 (en) | 2011-09-07 | 2013-03-14 | Marina Biotech Inc. | Synthesis and uses of nucleic acid compounds with conformationally restricted monomers |
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EP2850184A4 (en) | 2012-05-16 | 2016-01-27 | Rana Therapeutics Inc | COMPOSITIONS AND METHOD FOR MODULATING GENE EXPRESSION |
DK2920307T3 (en) | 2012-11-15 | 2018-07-16 | Roche Innovation Ct Copenhagen As | ANTI-APOB, ANTISENSE CONJUGATE RELATIONSHIPS |
SG10202001808VA (en) | 2015-10-30 | 2020-04-29 | Genentech Inc | Anti-htra1 antibodies and methods of use thereof |
EP3478839A1 (en) | 2016-07-01 | 2019-05-08 | H. Hoffnabb-La Roche Ag | Antisense oligonucleotides for modulating htra1 expression |
US20190284621A1 (en) | 2016-11-11 | 2019-09-19 | Roche Innovation Center Copenhagen A/S | Therapeutic oligonucleotides capture and detection |
US20190055564A1 (en) | 2017-06-01 | 2019-02-21 | F. Hoffmann-La Roche Ag | Antisense oligonucleotides for modulating htra1 expression |
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- 2018-12-04 IL IL263489A patent/IL263489A/en unknown
- 2018-12-06 CO CONC2018/0013240A patent/CO2018013240A2/es unknown
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2019
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2022
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2024
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TW201803990A (zh) | 2018-02-01 |
RU2019101298A3 (ru) | 2021-01-26 |
CN109415732B (zh) | 2024-01-02 |
JP2019522987A (ja) | 2019-08-22 |
CN109415732A (zh) | 2019-03-01 |
JP7416852B2 (ja) | 2024-01-17 |
PH12018502707A1 (en) | 2019-11-11 |
CL2018003698A1 (es) | 2019-04-05 |
WO2018002105A1 (en) | 2018-01-04 |
IL263489A (en) | 2019-01-31 |
PE20190381A1 (es) | 2019-03-08 |
MX2018015722A (es) | 2019-05-27 |
SG10202005885VA (en) | 2020-07-29 |
ZA201808291B (en) | 2019-10-30 |
JP7060525B2 (ja) | 2022-04-26 |
US10519450B2 (en) | 2019-12-31 |
US20200399641A1 (en) | 2020-12-24 |
CO2018013240A2 (es) | 2018-12-14 |
EP3478839A1 (en) | 2019-05-08 |
JP2024029178A (ja) | 2024-03-05 |
KR20190025970A (ko) | 2019-03-12 |
US20180002701A1 (en) | 2018-01-04 |
CR20180606A (es) | 2019-02-14 |
BR112018077321A2 (pt) | 2019-04-24 |
AU2017289204A1 (en) | 2019-01-17 |
SG11201811554SA (en) | 2019-01-30 |
CA3026855A1 (en) | 2018-01-04 |
JP2022106785A (ja) | 2022-07-20 |
MA45620A (fr) | 2019-05-08 |
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