CO2020010226A2 - Terapia con oligonucleotidos para la enfermedad de wilson - Google Patents
Terapia con oligonucleotidos para la enfermedad de wilsonInfo
- Publication number
- CO2020010226A2 CO2020010226A2 CONC2020/0010226A CO2020010226A CO2020010226A2 CO 2020010226 A2 CO2020010226 A2 CO 2020010226A2 CO 2020010226 A CO2020010226 A CO 2020010226A CO 2020010226 A2 CO2020010226 A2 CO 2020010226A2
- Authority
- CO
- Colombia
- Prior art keywords
- exon
- atp7b
- wilson
- disease
- flanking
- Prior art date
Links
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 title abstract 2
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 2
- 208000018839 Wilson disease Diseases 0.000 title abstract 2
- 238000002560 therapeutic procedure Methods 0.000 title abstract 2
- 102100027591 Copper-transporting ATPase 2 Human genes 0.000 abstract 4
- 101000936280 Homo sapiens Copper-transporting ATPase 2 Proteins 0.000 abstract 4
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 2
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 2
- 108020004999 messenger RNA Proteins 0.000 abstract 2
- 108020000948 Antisense Oligonucleotides Proteins 0.000 abstract 1
- 230000000295 complement effect Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
Classifications
-
- 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
- C12N15/1137—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 against enzymes
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N2310/33—Chemical structure of the base
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
- C12Y306/03004—Cu2+-exporting ATPase (3.6.3.4)
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862630565P | 2018-02-14 | 2018-02-14 | |
| PCT/US2019/018076 WO2019161105A1 (en) | 2018-02-14 | 2019-02-14 | Oligonucleotide therapy for wilson disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2020010226A2 true CO2020010226A2 (es) | 2020-12-10 |
Family
ID=67619598
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2020/0010226A CO2020010226A2 (es) | 2018-02-14 | 2020-08-19 | Terapia con oligonucleotidos para la enfermedad de wilson |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US11578327B2 (https=) |
| EP (1) | EP3752614A4 (https=) |
| JP (1) | JP2021513851A (https=) |
| KR (1) | KR20200120675A (https=) |
| CN (1) | CN112041439A (https=) |
| AU (1) | AU2019222767A1 (https=) |
| BR (1) | BR112020016524A2 (https=) |
| CA (1) | CA3090517A1 (https=) |
| CL (1) | CL2020002100A1 (https=) |
| CO (1) | CO2020010226A2 (https=) |
| IL (1) | IL276701A (https=) |
| MX (1) | MX2020008533A (https=) |
| PH (1) | PH12020551219A1 (https=) |
| SG (1) | SG11202007644PA (https=) |
| WO (1) | WO2019161105A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025110984A1 (en) * | 2023-11-21 | 2025-05-30 | Hydrogene Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| CN117925624B (zh) * | 2024-03-22 | 2024-05-31 | 上海凌医生物科技有限公司 | 一种金属响应调控元件 |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| US5578493A (en) * | 1993-09-01 | 1996-11-26 | The Trustees Of Columbia University In The City Of New York | Wilson's disease gene |
| CA2108927C (en) | 1993-09-21 | 2008-09-02 | Peter Bull | Wilson disease gene |
| AU1039397A (en) | 1995-11-22 | 1997-06-27 | Johns Hopkins University, The | Ligands to enhance cellular uptake of biomolecules |
| US20040101854A1 (en) * | 2002-11-21 | 2004-05-27 | Isis Pharmaceuticals Inc. | Modulation of BCL2-associated athanogene expression |
| US7491805B2 (en) | 2001-05-18 | 2009-02-17 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
| US20030180953A1 (en) | 2000-12-29 | 2003-09-25 | Elitra Pharmaceuticals, Inc. | Gene disruption methodologies for drug target discovery |
| US20040126761A1 (en) * | 2002-12-10 | 2004-07-01 | Isis Pharmaceuticals Inc. | Modulation of alpha-methylacyl-CoA racemase expression |
| WO2004035819A2 (en) | 2002-10-21 | 2004-04-29 | Exiqon A/S | Oligonucleotide analogues for detecting and analyzing nucleic acids |
| US20050244851A1 (en) | 2004-01-13 | 2005-11-03 | Affymetrix, Inc. | Methods of analysis of alternative splicing in human |
| CN1934251A (zh) * | 2004-01-29 | 2007-03-21 | 塞尔卓姆股份公司 | 使用atp7a－调控剂治疗神经变性疾病 |
| EP1752536A4 (en) | 2004-05-11 | 2008-04-16 | Alphagen Co Ltd | POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME |
| CA2566519C (en) | 2004-05-14 | 2020-04-21 | Rosetta Genomics Ltd. | Micrornas and uses thereof |
| US20070048756A1 (en) | 2005-04-18 | 2007-03-01 | Affymetrix, Inc. | Methods for whole genome association studies |
| EP1783645A1 (en) | 2005-11-08 | 2007-05-09 | Actigenics | Methods for the identification of microRNA and their applications in research and human health |
| US8178503B2 (en) | 2006-03-03 | 2012-05-15 | International Business Machines Corporation | Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes |
| JP4841279B2 (ja) | 2006-03-23 | 2011-12-21 | 一般財団法人 化学物質評価研究機構 | 被検物質の発がん性予測方法 |
| WO2009082607A2 (en) | 2007-12-04 | 2009-07-02 | Alnylam Pharmaceuticals, Inc. | Targeting lipids |
| CA2860676A1 (en) * | 2012-01-09 | 2013-07-18 | Novartis Ag | Organic compositions to treat beta-catenin-related diseases |
| WO2014036301A1 (en) * | 2012-08-30 | 2014-03-06 | Isis Pharmaceuticals, Inc. | Modulation of copper related diseases by ctr1 |
| DK2991656T3 (da) | 2013-05-01 | 2020-03-23 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression |
| US20160258005A1 (en) * | 2013-07-02 | 2016-09-08 | The Trustees Of The University Of Pennsylvania | Methods for rapid ribonucleic acid fluorescence in situ hybridization |
| AU2014346658A1 (en) | 2013-11-06 | 2016-06-02 | Solstice Biologics, Ltd. | Polynucleotide constructs having disulfide groups |
| JP2017522046A (ja) | 2014-06-06 | 2017-08-10 | ソルスティス バイオロジクス，リミティッド | 生物可逆的および生物不可逆的基を有するポリヌクレオチド構築物 |
| WO2016004043A1 (en) | 2014-06-30 | 2016-01-07 | Blend Therapeutics, Inc. | Targeted conjugates and particles and formulations thereof |
| JP2017533721A (ja) | 2014-11-14 | 2017-11-16 | アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． | タンパク質の調節のための化合物及び方法 |
| EP3247366A4 (en) | 2015-01-21 | 2018-10-31 | Sangamo Therapeutics, Inc. | Methods and compositions for identification of highly specific nucleases |
| CN105986015B (zh) * | 2015-02-05 | 2020-10-23 | 大连晶泰生物技术有限公司 | 一种基于高通量测序的多样本的一个或多个靶序列的检测方法和试剂盒 |
| EP3286318A2 (en) | 2015-04-22 | 2018-02-28 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| AU2016365828A1 (en) | 2015-12-08 | 2018-07-05 | Solstice Biologics, Ltd. | Polynucleotide constructs having an auxiliary moiety non-bioreversibly linked to an internucleoside phosphate or phosphorothioate |
| CA3005090A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of liver diseases |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR101743211B1 (ko) | 2016-06-24 | 2017-06-15 | 주식회사 녹십자지놈 | 선천성 기능장애 진단용 조성물 및 이의 용도 |
| EP3840758A1 (en) * | 2018-08-21 | 2021-06-30 | Deep Genomics Incorporated | Therapeutic splice-switching oligonucleotides |
| US20240358845A1 (en) * | 2021-05-10 | 2024-10-31 | Entrada Therapeutics, Inc. | Compositions and methods for intracellular therapeutics |
| WO2022256283A2 (en) * | 2021-06-01 | 2022-12-08 | Korro Bio, Inc. | Methods for restoring protein function using adar |
-
2019
- 2019-02-14 CN CN201980018495.1A patent/CN112041439A/zh active Pending
- 2019-02-14 BR BR112020016524-7A patent/BR112020016524A2/pt not_active IP Right Cessation
- 2019-02-14 SG SG11202007644PA patent/SG11202007644PA/en unknown
- 2019-02-14 AU AU2019222767A patent/AU2019222767A1/en not_active Abandoned
- 2019-02-14 EP EP19754072.7A patent/EP3752614A4/en active Pending
- 2019-02-14 CA CA3090517A patent/CA3090517A1/en active Pending
- 2019-02-14 KR KR1020207025862A patent/KR20200120675A/ko not_active Withdrawn
- 2019-02-14 JP JP2020543576A patent/JP2021513851A/ja not_active Withdrawn
- 2019-02-14 WO PCT/US2019/018076 patent/WO2019161105A1/en not_active Ceased
- 2019-02-14 MX MX2020008533A patent/MX2020008533A/es unknown
- 2019-02-14 PH PH1/2020/551219A patent/PH12020551219A1/en unknown
- 2019-02-14 US US16/970,088 patent/US11578327B2/en active Active
-
2020
- 2020-08-13 CL CL2020002100A patent/CL2020002100A1/es unknown
- 2020-08-13 IL IL276701A patent/IL276701A/en unknown
- 2020-08-19 CO CONC2020/0010226A patent/CO2020010226A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| SG11202007644PA (en) | 2020-09-29 |
| PH12020551219A1 (en) | 2021-04-19 |
| AU2019222767A1 (en) | 2020-08-27 |
| MX2020008533A (es) | 2020-11-06 |
| US11578327B2 (en) | 2023-02-14 |
| CA3090517A1 (en) | 2019-08-22 |
| EP3752614A4 (en) | 2021-11-10 |
| CL2020002100A1 (es) | 2021-01-29 |
| IL276701A (en) | 2020-09-30 |
| WO2019161105A1 (en) | 2019-08-22 |
| CN112041439A (zh) | 2020-12-04 |
| US20210032629A1 (en) | 2021-02-04 |
| JP2021513851A (ja) | 2021-06-03 |
| EP3752614A1 (en) | 2020-12-23 |
| BR112020016524A2 (pt) | 2021-09-28 |
| KR20200120675A (ko) | 2020-10-21 |
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