KR102043422B1 - 프라탁신 (fxn)에 대한 자연 안티센스 전사체의 저해에 의한 프라탁신 (fxn) 관련된 질환의 치료 - Google Patents
프라탁신 (fxn)에 대한 자연 안티센스 전사체의 저해에 의한 프라탁신 (fxn) 관련된 질환의 치료 Download PDFInfo
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- KR102043422B1 KR102043422B1 KR1020137034424A KR20137034424A KR102043422B1 KR 102043422 B1 KR102043422 B1 KR 102043422B1 KR 1020137034424 A KR1020137034424 A KR 1020137034424A KR 20137034424 A KR20137034424 A KR 20137034424A KR 102043422 B1 KR102043422 B1 KR 102043422B1
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- 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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- 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
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P39/00—General protective or antinoxious agents
- A61P39/06—Free radical scavengers or antioxidants
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
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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
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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
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
- C12N2310/30—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
- C12N2310/32—Chemical structure of the sugar
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Biochemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Analytical Chemistry (AREA)
- Rheumatology (AREA)
- Toxicology (AREA)
- Neurosurgery (AREA)
- Pain & Pain Management (AREA)
- Neurology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161494928P | 2011-06-09 | 2011-06-09 | |
| US61/494,928 | 2011-06-09 | ||
| PCT/US2012/041484 WO2012170771A1 (en) | 2011-06-09 | 2012-06-08 | Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| KR20140033165A KR20140033165A (ko) | 2014-03-17 |
| KR102043422B1 true KR102043422B1 (ko) | 2019-11-11 |
Family
ID=47296462
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020137034424A Expired - Fee Related KR102043422B1 (ko) | 2011-06-09 | 2012-06-08 | 프라탁신 (fxn)에 대한 자연 안티센스 전사체의 저해에 의한 프라탁신 (fxn) 관련된 질환의 치료 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US9593330B2 (https=) |
| EP (1) | EP2718439B1 (https=) |
| JP (1) | JP6188686B2 (https=) |
| KR (1) | KR102043422B1 (https=) |
| CN (1) | CN103620036B (https=) |
| CA (1) | CA2838588C (https=) |
| ES (1) | ES2653247T3 (https=) |
| RU (1) | RU2620980C2 (https=) |
| TW (1) | TWI678375B (https=) |
| WO (1) | WO2012170771A1 (https=) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20100233270A1 (en) | 2009-01-08 | 2010-09-16 | Northwestern University | Delivery of Oligonucleotide-Functionalized Nanoparticles |
| CA2817256A1 (en) | 2010-11-12 | 2012-05-18 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| EA201492116A1 (ru) | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
| SG11201407483YA (en) | 2012-05-16 | 2014-12-30 | Rana Therapeutics Inc | Compositions and methods for modulating smn gene family expression |
| AU2013262699A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating ATP2A2 expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| BR112014028631A2 (pt) | 2012-05-16 | 2017-10-17 | Rana Therapeutics Inc | composições e métodos para modulação da expressão da família de genes da hemoglobina |
| EA201492122A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии utrn |
| CA2921556A1 (en) * | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Compositions and methods for modulating rna |
| CA2921457A1 (en) * | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Heterochromatin forming non-coding rnas |
| WO2015023938A1 (en) * | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Epigenetic regulators of frataxin |
| JP2016531570A (ja) | 2013-08-16 | 2016-10-13 | ラナ セラピューティクス インコーポレイテッド | ユークロマチン領域を標的とするオリゴヌクレオチド |
| US20160201064A1 (en) * | 2013-08-16 | 2016-07-14 | Rana Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin |
| US20160271090A1 (en) * | 2013-10-29 | 2016-09-22 | Tokyo University Of Agriculture | Frataxin enhancer |
| SI3071696T1 (sl) | 2013-11-22 | 2019-11-29 | Mina Therapeutics Ltd | C/EBP alfa kratko delujoči RNA sestavki in postopki uporabe |
| WO2016028940A1 (en) | 2014-08-19 | 2016-02-25 | Northwestern University | Protein/oligonucleotide core-shell nanoparticle therapeutics |
| WO2016070060A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
| US10758558B2 (en) | 2015-02-13 | 2020-09-01 | Translate Bio Ma, Inc. | Hybrid oligonucleotides and uses thereof |
| EP3256590A4 (en) * | 2015-02-13 | 2018-10-03 | Translate Bio Ma, Inc. | Targeting oligonucleotides and uses thereof to modulate gene expression |
| WO2016149455A2 (en) | 2015-03-17 | 2016-09-22 | The General Hospital Corporation | The rna interactome of polycomb repressive complex 1 (prc1) |
| EP3368089B1 (en) | 2015-10-26 | 2025-11-05 | Translate Bio Ma, Inc. | Nanoparticle formulations for delivery of nucleic acid complexes |
| SG11201802408RA (en) * | 2015-11-05 | 2018-05-30 | Bamboo Therapeutics Inc | Modified friedreich ataxia genes and vectors for gene therapy |
| WO2018089527A1 (en) | 2016-11-09 | 2018-05-17 | Intrexon Corporation | Frataxin expression constructs |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| EP4089173A1 (en) * | 2017-10-17 | 2022-11-16 | Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol | Vectors for the treatment of friedreich's ataxia |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| BR112022001657A2 (pt) * | 2019-07-29 | 2022-03-22 | Univ Indiana Trustees | Polipeptídeo de fusão, sequência de ácidos nucleicos, cassete de expressão, vetor de expressão, célula hospedeira, composição farmacêutica, e, métodos para tratar a ataxia de friedreich e para tratar uma doença associada à ataxia de friedreich |
| WO2021021964A2 (en) | 2019-07-29 | 2021-02-04 | Chondrial Therapeutics, Inc. | Methods of quantifying frataxin and frataxin fusion proteins |
| US20220348893A1 (en) * | 2019-09-23 | 2022-11-03 | Omega Therapeutics, Inc | Methods and compositions for modulating frataxin expression and treating friedrich's ataxia |
| KR20220108094A (ko) | 2019-11-25 | 2022-08-02 | 라리마 테라퓨틱스, 인코포레이티드 | 프라탁신 활성의 정량화 방법 |
| EP4103715A4 (en) * | 2020-02-14 | 2024-03-06 | University Of Massachusetts | FRATAXIN-TARGETING OLIGONUCLEOTIDES AND RELATED METHODS |
| WO2021202557A1 (en) * | 2020-03-30 | 2021-10-07 | Exicure Operating Company | Spherical nucleic acids (snas) for regulation of frataxin |
| WO2021262919A2 (en) | 2020-06-26 | 2021-12-30 | The Research Foundation For The State University Of New York | 5-halouracil-modified micrornas and their use in the treatment of cancer |
| WO2024069235A2 (en) * | 2022-09-30 | 2024-04-04 | Sixfold Bioscience Ltd. | Compositions containing oligonucleotides with theranostic applications |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2007087113A2 (en) * | 2005-12-28 | 2007-08-02 | The Scripps Research Institute | Natural antisense and non-coding rna transcripts as drug targets |
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- 2012-06-08 RU RU2013153487A patent/RU2620980C2/ru active
- 2012-06-08 JP JP2014514869A patent/JP6188686B2/ja active Active
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Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2007087113A2 (en) * | 2005-12-28 | 2007-08-02 | The Scripps Research Institute | Natural antisense and non-coding rna transcripts as drug targets |
| JP2009521934A (ja) | 2005-12-28 | 2009-06-11 | ザ スクリップス リサーチ インスティテュート | 薬物標的としての天然アンチセンスおよび非コードrna転写物 |
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| PLoS ONE,제4권,11호,e7914,1-8면(2009.11.)* |
| Science,제309권,1564-1566면(2005.09.)* |
Also Published As
| Publication number | Publication date |
|---|---|
| US20140187606A1 (en) | 2014-07-03 |
| JP6188686B2 (ja) | 2017-08-30 |
| RU2013153487A (ru) | 2015-07-20 |
| CA2838588C (en) | 2021-09-14 |
| TW201302781A (zh) | 2013-01-16 |
| CA2838588A1 (en) | 2012-12-13 |
| CN103620036B (zh) | 2016-12-21 |
| EP2718439A1 (en) | 2014-04-16 |
| US20170145414A1 (en) | 2017-05-25 |
| KR20140033165A (ko) | 2014-03-17 |
| JP2014519333A (ja) | 2014-08-14 |
| US9902959B2 (en) | 2018-02-27 |
| RU2620980C2 (ru) | 2017-05-30 |
| TWI678375B (zh) | 2019-12-01 |
| WO2012170771A1 (en) | 2012-12-13 |
| EP2718439A4 (en) | 2014-05-21 |
| EP2718439B1 (en) | 2017-08-09 |
| CN103620036A (zh) | 2014-03-05 |
| US9593330B2 (en) | 2017-03-14 |
| ES2653247T3 (es) | 2018-02-06 |
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