JP2019500345A - 肝臓病の処置のための組成物および方法 - Google Patents
肝臓病の処置のための組成物および方法 Download PDFInfo
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- JP2019500345A JP2019500345A JP2018529229A JP2018529229A JP2019500345A JP 2019500345 A JP2019500345 A JP 2019500345A JP 2018529229 A JP2018529229 A JP 2018529229A JP 2018529229 A JP2018529229 A JP 2018529229A JP 2019500345 A JP2019500345 A JP 2019500345A
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Genetics & Genomics (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Investigating Or Analysing Biological Materials (AREA)
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562267238P | 2015-12-14 | 2015-12-14 | |
US62/267,238 | 2015-12-14 | ||
US201662319015P | 2016-04-06 | 2016-04-06 | |
US62/319,015 | 2016-04-06 | ||
PCT/US2016/066564 WO2017106283A1 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of liver diseases |
Publications (2)
Publication Number | Publication Date |
---|---|
JP2019500345A true JP2019500345A (ja) | 2019-01-10 |
JP2019500345A5 JP2019500345A5 (zh) | 2020-01-30 |
Family
ID=59057460
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2018529229A Pending JP2019500345A (ja) | 2015-12-14 | 2016-12-14 | 肝臓病の処置のための組成物および方法 |
Country Status (4)
Country | Link |
---|---|
EP (1) | EP3389672A4 (zh) |
JP (1) | JP2019500345A (zh) |
CA (1) | CA3005090A1 (zh) |
WO (1) | WO2017106283A1 (zh) |
Families Citing this family (22)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
JP6867945B2 (ja) | 2014-10-03 | 2021-05-12 | コールド スプリング ハーバー ラボラトリー | 核内遺伝子出力の標的とされた増強 |
WO2017060731A1 (en) | 2015-10-09 | 2017-04-13 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
EP3933041B1 (en) | 2015-12-14 | 2024-01-31 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant retardation |
US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
KR102619197B1 (ko) | 2017-01-23 | 2024-01-03 | 리제너론 파마슈티칼스 인코포레이티드 | Hsd17b13 변종 및 이것의 용도 |
AU2018250727A1 (en) | 2017-04-11 | 2019-10-31 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (HSD17B) family |
PL3673080T3 (pl) | 2017-08-25 | 2024-03-11 | Stoke Therapeutics, Inc. | Oligomery antysensowne do leczenia stanów i chorób |
EP3461837A1 (en) * | 2017-09-28 | 2019-04-03 | Secarna Pharmaceuticals GmbH & Co. KG | Inhibitor inhibiting the expression of pprx1 |
EP3695012B1 (en) | 2017-10-11 | 2023-03-08 | Regeneron Pharmaceuticals, Inc. | Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation |
KR20200120675A (ko) * | 2018-02-14 | 2020-10-21 | 딥 지노믹스 인코포레이티드 | 윌슨병을 위한 올리고뉴클레오티드 요법 |
SG11202007652UA (en) * | 2018-02-21 | 2020-09-29 | Bristol Myers Squibb Co | Camk2d antisense oligonucleotides and uses thereof |
TW202023573A (zh) | 2018-09-19 | 2020-07-01 | 美商Ionis製藥公司 | Pnpla3表現之調節劑 |
US10913951B2 (en) * | 2018-10-31 | 2021-02-09 | University of Pittsburgh—of the Commonwealth System of Higher Education | Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure |
CN111849968A (zh) * | 2019-04-30 | 2020-10-30 | 中美瑞康核酸技术(南通)研究院有限公司 | 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用 |
EP4017979A4 (en) * | 2019-08-19 | 2024-03-27 | Stoke Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR MODULATING SPLICING AND PROTEIN EXPRESSION |
AU2020349991A1 (en) * | 2019-09-20 | 2022-04-07 | Ractigen Therapeutics | Nucleic acid molecule for treating immune thrombocytopenia and application thereof |
CN114728017A (zh) * | 2019-10-14 | 2022-07-08 | 阿斯利康(瑞典)有限公司 | Pnpla3表达的调节剂 |
KR20230022409A (ko) | 2020-05-11 | 2023-02-15 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머 |
WO2021231698A1 (en) * | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl) |
CN116209761A (zh) * | 2020-07-23 | 2023-06-02 | 豪夫迈·罗氏有限公司 | 靶向rna结合蛋白位点的寡核苷酸 |
WO2022109030A1 (en) * | 2020-11-17 | 2022-05-27 | Skyhawk Therapeutics, Inc. | Rna-targeting splicing modifiers for treatment of pnpla3-associated conditions and diseases |
Citations (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20140128449A1 (en) * | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
Family Cites Families (15)
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US4866042A (en) | 1987-11-18 | 1989-09-12 | Neuwelt Edward A | Method for the delivery of genetic material across the blood brain barrier |
US6294520B1 (en) | 1989-03-27 | 2001-09-25 | Albert T. Naito | Material for passage through the blood-brain barrier |
US5151510A (en) | 1990-04-20 | 1992-09-29 | Applied Biosystems, Inc. | Method of synethesizing sulfurized oligonucleotide analogs |
WO1994008026A1 (en) | 1992-09-25 | 1994-04-14 | Rhone-Poulenc Rorer S.A. | Adenovirus vectors for the transfer of foreign genes into cells of the central nervous system, particularly in brain |
US5656612A (en) | 1994-05-31 | 1997-08-12 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of raf gene expression |
FR2727867B1 (fr) | 1994-12-13 | 1997-01-31 | Rhone Poulenc Rorer Sa | Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux |
US6936589B2 (en) | 2001-09-28 | 2005-08-30 | Albert T. Naito | Parenteral delivery systems |
US7291461B2 (en) * | 2002-06-21 | 2007-11-06 | Ptc Therapeutics, Inc. | Methods for identifying small molecules that modulate premature translation termination and nonsense mRNA decay |
JP5202296B2 (ja) * | 2005-04-01 | 2013-06-05 | ユニバーシティ オブ フロリダ リサーチ ファウンデーション,インコーポレイティド | 肝臓傷害のバイオマーカー |
WO2007047913A2 (en) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions and methods for modulation of lmna expression |
CA2910760C (en) | 2007-12-04 | 2019-07-09 | Muthiah Manoharan | Targeting lipids |
CN106983768B (zh) | 2009-06-17 | 2023-04-07 | 冷泉港实验室 | 用于在对象中调节smn2剪接的组合物和方法 |
WO2012075040A2 (en) * | 2010-11-30 | 2012-06-07 | Shire Human Genetic Therapies, Inc. | mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES |
US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
EP3690048A1 (en) | 2013-09-04 | 2020-08-05 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
-
2016
- 2016-12-14 JP JP2018529229A patent/JP2019500345A/ja active Pending
- 2016-12-14 CA CA3005090A patent/CA3005090A1/en active Pending
- 2016-12-14 EP EP16876543.6A patent/EP3389672A4/en not_active Withdrawn
- 2016-12-14 WO PCT/US2016/066564 patent/WO2017106283A1/en active Application Filing
Patent Citations (1)
Publication number | Priority date | Publication date | Assignee | Title |
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US20140128449A1 (en) * | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
Non-Patent Citations (9)
Title |
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LLEWELLYN, D. H. ET AL., J MED GENET, vol. 33, JPN6020050338, 1996, pages 437 - 8, ISSN: 0004416760 * |
MARTINEZ DI MONTEMUROS, F. ET AL., BLOOD CELL MOL DIS, vol. 27, JPN6020050337, 2001, pages 961 - 70, ISSN: 0004416759 * |
MUSTAJOKI, S. ET AL., HUM GENET, vol. 102, JPN6020050336, 1998, pages 541 - 8, ISSN: 0004416758 * |
PNAS, vol. 93, JPN6020044617, 1996, pages 12840 - 12844, ISSN: 0004416766 * |
PRICE, L. ET AL., NEUROMUSCUL DISORD, vol. 22, JPN6020050344, 2012, pages 862, ISSN: 0004416764 * |
RIVERA-BARAHONA, A. ET AL., PLOS ONE, vol. 10, JPN6020050343, April 2015 (2015-04-01), pages 0122966 - 1, ISSN: 0004416763 * |
ROBREAU-FRAOLINI, A. M. ET AL., HUM GENET, vol. 107, JPN6020050342, 2000, pages 150 - 9, ISSN: 0004416762 * |
VAN BLITTERSWIJK, M. ET AL., AMYOTROPH LATERAL SCLER FRONTOTEMPORAL DEGENER, vol. 165, JPN6020050346, October 2015 (2015-10-01), pages 9, ISSN: 0004416765 * |
五十嵐正樹, 外9名, 臨床血液, vol. 45, JPN6020050339, 2004, pages 562 - 7, ISSN: 0004416761 * |
Also Published As
Publication number | Publication date |
---|---|
EP3389672A1 (en) | 2018-10-24 |
CA3005090A1 (en) | 2017-06-22 |
EP3389672A4 (en) | 2019-08-14 |
WO2017106283A1 (en) | 2017-06-22 |
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