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
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)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Abstract
La presente invención se refiere a oligonucleótidos antisentido, composiciones y métodos que se dirigen al exón 6 de ATP7B o un intrón flanqueante, modulando así el empalme de pre-ARNm de ATP7B para aumentar el nivel de moléculas de ARNm de ATP7B que tienen exón 6, por ejemplo, para proporcionar una terapia para la enfermedad de Wilson. La presente descripción proporciona un oligonucleótido antisentido que incluye una secuencia de nucleobase al menos 70% complementaria a una secuencia objetivo de ATP7B en el exón 6, un intrón flanqueante en 5’, un intrón flanqueante en 3’ o una combinación del exón 6 y el intrón flanqueante en 5’ o flanqueante en 3’.
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 (14)
Country | Link |
---|---|
US (1) | US11578327B2 (es) |
EP (1) | EP3752614A4 (es) |
JP (1) | JP2021513851A (es) |
KR (1) | KR20200120675A (es) |
CN (1) | CN112041439A (es) |
AU (1) | AU2019222767A1 (es) |
BR (1) | BR112020016524A2 (es) |
CA (1) | CA3090517A1 (es) |
CL (1) | CL2020002100A1 (es) |
CO (1) | CO2020010226A2 (es) |
IL (1) | IL276701A (es) |
MX (1) | MX2020008533A (es) |
SG (1) | SG11202007644PA (es) |
WO (1) | WO2019161105A1 (es) |
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 |
CN1120707C (zh) | 1995-11-22 | 2003-09-10 | 约翰斯·霍普金斯大学 | 增强生物分子的细胞摄取的配体 |
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 |
US20070280927A1 (en) * | 2004-01-29 | 2007-12-06 | Cellzome Ag | Treatment Of Neurodegenerative Diseases By The Use Of Atp7a |
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 |
WO2005111211A2 (en) | 2004-05-14 | 2005-11-24 | Rosetta Genomics Ltd. | Micronas 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 |
US20090247608A1 (en) | 2007-12-04 | 2009-10-01 | Alnylam Pharmaceuticals, Inc. | Targeting Lipids |
US10023862B2 (en) * | 2012-01-09 | 2018-07-17 | Arrowhead Pharmaceuticals, Inc. | 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 |
RU2019110030A (ru) | 2013-05-01 | 2019-05-06 | Ионис Фармасьютикалз, Инк. | Композиции и способы |
WO2015002978A2 (en) * | 2013-07-02 | 2015-01-08 | The Trustees Of The University Of Pennsylvania | Methods for rapid ribonucleic acid fluorescence in situ hybridization |
US20160257961A1 (en) | 2013-11-06 | 2016-09-08 | 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 |
US20170151339A1 (en) | 2014-06-30 | 2017-06-01 | Tarveda Therapeutics, Inc. | Targeted conjugates and particles and formulations thereof |
WO2016077837A1 (en) | 2014-11-14 | 2016-05-19 | Ionis Pharmaceuticals, Inc. | Compounds and methods for the modulation of proteins |
US20180002379A1 (en) | 2015-01-21 | 2018-01-04 | 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 |
US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
CA3005090A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of liver diseases |
KR101743211B1 (ko) | 2016-06-24 | 2017-06-15 | 주식회사 녹십자지놈 | 선천성 기능장애 진단용 조성물 및 이의 용도 |
WO2020037415A1 (en) * | 2018-08-21 | 2020-02-27 | Deep Genomics Incorporated | Therapeutic splice-switching oligonucleotides |
AU2022271873A1 (en) * | 2021-05-10 | 2024-01-04 | 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 WO PCT/US2019/018076 patent/WO2019161105A1/en unknown
- 2019-02-14 KR KR1020207025862A patent/KR20200120675A/ko unknown
- 2019-02-14 JP JP2020543576A patent/JP2021513851A/ja not_active Withdrawn
- 2019-02-14 BR BR112020016524-7A patent/BR112020016524A2/pt not_active IP Right Cessation
- 2019-02-14 AU AU2019222767A patent/AU2019222767A1/en not_active Abandoned
- 2019-02-14 CN CN201980018495.1A patent/CN112041439A/zh active Pending
- 2019-02-14 EP EP19754072.7A patent/EP3752614A4/en active Pending
- 2019-02-14 CA CA3090517A patent/CA3090517A1/en active Pending
- 2019-02-14 US US16/970,088 patent/US11578327B2/en active Active
- 2019-02-14 MX MX2020008533A patent/MX2020008533A/es unknown
- 2019-02-14 SG SG11202007644PA patent/SG11202007644PA/en unknown
-
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 |
---|---|
EP3752614A1 (en) | 2020-12-23 |
KR20200120675A (ko) | 2020-10-21 |
US11578327B2 (en) | 2023-02-14 |
WO2019161105A1 (en) | 2019-08-22 |
CA3090517A1 (en) | 2019-08-22 |
US20210032629A1 (en) | 2021-02-04 |
IL276701A (en) | 2020-09-30 |
CL2020002100A1 (es) | 2021-01-29 |
CN112041439A (zh) | 2020-12-04 |
JP2021513851A (ja) | 2021-06-03 |
AU2019222767A1 (en) | 2020-08-27 |
BR112020016524A2 (pt) | 2021-09-28 |
SG11202007644PA (en) | 2020-09-29 |
MX2020008533A (es) | 2020-11-06 |
EP3752614A4 (en) | 2021-11-10 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
NZ733882A (en) | Compositions for modulating c9orf72 expression | |
CO2019003846A2 (es) | Tratamiento aav de la enfermedad de huntington | |
CL2019000107A1 (es) | Composiciones de arni contra el componente c5 del complemento y métodos para su uso.(divisional solicitud 201702014) | |
CL2021001490A1 (es) | Constructos de iarn modificados químicamente y usos de estos | |
CO2018006590A2 (es) | Métodos y composiciones para el tratamiento de un trastorno asociado a serpinc1 | |
CR20190294A (es) | COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO | |
NZ747685A (en) | Oligonucleotide analogues targeting human lmna | |
CL2021002326A1 (es) | Composiciones de arni contra el angiotensinógeno (agt) y métodos para su uso (divisional de solicitud n° 202002865) | |
MX2017002144A (es) | Agentes de ácido ribonucleico (arn) de cadena doble modificados. | |
AR107579A1 (es) | Composiciones y métodos para disminuir la expresión de tau | |
DOP2021000126A (es) | COMPOSICIONES DE AGENTE DE ARNi DE PROTEÍNA PRECURSORA DE AMILOIDE (APP) Y MÉTODO DE USO DE LAS MISMAS | |
CY1123639T1 (el) | Μονοκλωνα ολιγονουκλεοτιδια για χρηση στην ιατρικη θεραπευτικη αντιμετωπιση διαταραχων του δερματος | |
AR067395A1 (es) | Inhibicion de htra1 mediada por iarn para el tratamiento de la degeneracion macular | |
EA201791986A1 (ru) | Олигонуклеотиды, соответствующие экзону 73 гена col7a1, для терапии буллезного эпидермолиза | |
CL2021001488A1 (es) | Transposasa de piggybac mutada | |
NZ741841A (en) | Antisense oligonucleotides for use in treating alzheimer’s disease | |
CY1123599T1 (el) | Συνθεσεις και μεθοδοι για τη διορθωση ζωνιαιας μυικης δυστροφιας τυπου 2c με τη χρησιμοποιηση παραλειψης εξονιων | |
CO2020010226A2 (es) | Terapia con oligonucleotidos para la enfermedad de wilson | |
AR120341A1 (es) | COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO | |
BR112022009772A2 (pt) | Métodos e composições para neuroproteção | |
CL2023000949A1 (es) | Oligonucleótidos antisentido que se dirigen al exón 51 del gen de la distrofina | |
BR112021010856A2 (pt) | Vibegron para o tratamento de sintomas de bexiga hiperativa | |
AR111587A1 (es) | Combinación sinérgica de oligonucleótidos de ácidos nucleicos y agentes quimioterapéuticos alquilantes | |
AR111681A1 (es) | Polinucleótidos moduladores | |
AR111410A1 (es) | Combinación sinérgica de oligonucleótidos de ácidos nucleicos y agentes quimioterapéuticos inhibidores de proteína quinasas |