PL3041958T4 - Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi - Google Patents
Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymiInfo
- Publication number
- PL3041958T4 PL3041958T4 PL14842672T PL14842672T PL3041958T4 PL 3041958 T4 PL3041958 T4 PL 3041958T4 PL 14842672 T PL14842672 T PL 14842672T PL 14842672 T PL14842672 T PL 14842672T PL 3041958 T4 PL3041958 T4 PL 3041958T4
- Authority
- PL
- Poland
- Prior art keywords
- mrna decay
- mediated mrna
- nonsense
- reducing
- reducing nonsense
- Prior art date
Links
Classifications
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
- A61K31/4245—Oxadiazoles
-
- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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
- A61K31/715—Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
- A61K31/726—Glycosaminoglycans, i.e. mucopolysaccharides
-
- 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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- 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
- 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
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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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
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
-
- 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
-
- 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
- C12N2320/31—Combination therapy
-
- 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
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/106—Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
-
- 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
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/156—Polymorphic or mutational markers
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361873780P | 2013-09-04 | 2013-09-04 | |
| EP14842672.9A EP3041958B1 (en) | 2013-09-04 | 2014-09-04 | Reducing nonsense-mediated mrna decay |
| PCT/US2014/054151 WO2015035091A1 (en) | 2013-09-04 | 2014-09-04 | Reducing nonsense-mediated mrna decay |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| PL3041958T4 true PL3041958T4 (pl) | 2020-11-02 |
| PL3041958T3 PL3041958T3 (pl) | 2020-11-02 |
Family
ID=52628936
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL14842672T PL3041958T3 (pl) | 2013-09-04 | 2014-09-04 | Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US10647983B2 (pl) |
| EP (2) | EP3690048A1 (pl) |
| AU (1) | AU2014315183B2 (pl) |
| CA (1) | CA2930859C (pl) |
| DK (1) | DK3041958T3 (pl) |
| ES (1) | ES2779302T3 (pl) |
| PL (1) | PL3041958T3 (pl) |
| WO (1) | WO2015035091A1 (pl) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2930859C (en) | 2013-09-04 | 2022-05-03 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| US9976143B2 (en) | 2014-10-03 | 2018-05-22 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| EP3359685A1 (en) * | 2015-10-09 | 2018-08-15 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| CA3005254A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13 |
| EP3389725B1 (en) | 2015-12-14 | 2022-04-06 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of central nervous system diseases |
| CA3005245A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of alagille syndrome |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| JP2018538287A (ja) * | 2015-12-14 | 2018-12-27 | コールド スプリング ハーバー ラボラトリー | 多発性嚢胞腎の処置のためのアンチセンスオリゴマー |
| JP2019500345A (ja) | 2015-12-14 | 2019-01-10 | コールド スプリング ハーバー ラボラトリー | 肝臓病の処置のための組成物および方法 |
| CA3005249A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of kidney diseases |
| JP7036723B2 (ja) * | 2015-12-14 | 2022-03-15 | コールド スプリング ハーバー ラボラトリー | 眼疾患の処置のための組成物および方法 |
| CA3005131A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of tuberous sclerosis complex |
| WO2017106377A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| EP3405478A4 (en) | 2015-12-23 | 2019-10-30 | Moonshot Pharma LLC | METHOD FOR INDUCING AN IMMUNE RESPONSE BY INHIBITING NONSENSE-MEDIATED CRASH |
| WO2018011765A1 (en) * | 2016-07-14 | 2018-01-18 | Universidade Do Porto | Biomarker for prenatal diagnosis of twin-to-twin transfusion syndrome |
| EP3512516A1 (en) * | 2016-09-13 | 2019-07-24 | Marco Cipolli | Method of treatment of shwachman-diamond syndrome |
| FR3057774A1 (fr) * | 2016-10-21 | 2018-04-27 | Museum National D'histoire Naturelle | Derives de la purine pour leur utilisation dans le traitement ou la prevention de maladies dues a une mutation non-sens |
| JP2020525434A (ja) | 2017-06-22 | 2020-08-27 | ムーンショット ファーマ エルエルシー | アンレキサノクス及び免疫調節剤を含む組成物で癌を治療する方法 |
| CN111278991B (zh) * | 2017-08-25 | 2022-04-01 | 斯托克制药公司 | 用于治疗病况和疾病的反义寡聚体 |
| US11213537B2 (en) * | 2017-09-18 | 2022-01-04 | Friedrich Miescher Institute For Biomedical Research | Inhibition of autism spectrum disorder using ribosomal read-through compounds |
| GB2610100B (en) * | 2017-10-23 | 2023-08-16 | Stoke Therapeutics Inc | Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases |
| SG11202105621PA (en) * | 2018-11-29 | 2021-06-29 | Eloxx Pharmaceuticals | Methods, compositions, and kits for treating ocular diseases |
| CN114007608A (zh) * | 2019-04-10 | 2022-02-01 | Ptc医疗公司 | 在儿科患者中治疗无义突变介导的杜氏肌营养不良症的方法 |
| AU2020262435A1 (en) * | 2019-04-24 | 2021-12-02 | Stoke Therapeutics, Inc. | Methods and compositions for modulating splicing and translation |
| US11129829B2 (en) | 2019-06-17 | 2021-09-28 | Skyhawk Therapeutics, Inc. | Methods for modulating splicing |
| WO2021061873A1 (en) * | 2019-09-23 | 2021-04-01 | Eloxx Pharmaceuticals, Inc. | Methods, compositions, and kits for treating polycystic kidney disease |
| WO2021113270A1 (en) | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Therapeutic editing |
| CA3173647A1 (en) | 2020-05-11 | 2021-11-18 | Isabel AZNAREZ | Opa1 antisense oligomers for treatment of conditions and diseases |
| WO2022216804A2 (en) * | 2021-04-07 | 2022-10-13 | University Of Massachusetts | Specific oligonucleotide-programmed readthrough of nonsense codons |
| WO2023112048A1 (en) * | 2021-12-13 | 2023-06-22 | Indian Institute Of Science | Crispr-dcas13 system, composition, and method to induce translational readthrough across stop codons |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CH671155A5 (pl) | 1986-08-18 | 1989-08-15 | Clinical Technologies Ass | |
| US5013556A (en) | 1989-10-20 | 1991-05-07 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US5151510A (en) | 1990-04-20 | 1992-09-29 | Applied Biosystems, Inc. | Method of synethesizing sulfurized oligonucleotide analogs |
| WO1992016192A1 (en) | 1991-03-15 | 1992-10-01 | Amgen Inc. | Pulmonary administration of granulocyte colony stimulating factor |
| US5656612A (en) | 1994-05-31 | 1997-08-12 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of raf gene expression |
| WO2004001010A2 (en) | 2002-06-21 | 2003-12-31 | Ptc Therapeutics, Inc. | METHODS FOR IDENTIFYING SMALL MOLECULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY |
| US8258109B2 (en) | 2005-10-20 | 2012-09-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of LMNA expression |
| JP5926475B2 (ja) * | 2006-09-21 | 2016-05-25 | ユニバーシティー オブ ロチェスター | 筋緊張性ジストロフィーのためのタンパク質置換治療に関する組成物および方法 |
| NZ597071A (en) | 2009-06-17 | 2014-05-30 | Isis Pharmaceuticals Inc | Compositions and methods for modulation of smn2 splicing in a subject |
| CA2803525A1 (en) * | 2009-06-23 | 2011-01-13 | University Of Miami | Aptamer-targeted sirna to inhibit nonsense mediated decay |
| CA2930859C (en) | 2013-09-04 | 2022-05-03 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
-
2014
- 2014-09-04 CA CA2930859A patent/CA2930859C/en active Active
- 2014-09-04 ES ES14842672T patent/ES2779302T3/es active Active
- 2014-09-04 PL PL14842672T patent/PL3041958T3/pl unknown
- 2014-09-04 AU AU2014315183A patent/AU2014315183B2/en active Active
- 2014-09-04 EP EP19206009.3A patent/EP3690048A1/en not_active Withdrawn
- 2014-09-04 DK DK14842672.9T patent/DK3041958T3/da active
- 2014-09-04 US US14/916,561 patent/US10647983B2/en active Active
- 2014-09-04 WO PCT/US2014/054151 patent/WO2015035091A1/en active Application Filing
- 2014-09-04 EP EP14842672.9A patent/EP3041958B1/en active Active
-
2020
- 2020-04-02 US US16/839,034 patent/US11352624B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| EP3041958A1 (en) | 2016-07-13 |
| US11352624B2 (en) | 2022-06-07 |
| US10647983B2 (en) | 2020-05-12 |
| EP3041958B1 (en) | 2019-12-04 |
| EP3690048A1 (en) | 2020-08-05 |
| AU2014315183B2 (en) | 2021-03-04 |
| WO2015035091A1 (en) | 2015-03-12 |
| US20170159049A9 (en) | 2017-06-08 |
| EP3041958A4 (en) | 2017-04-26 |
| CA2930859C (en) | 2022-05-03 |
| AU2014315183A1 (en) | 2016-04-21 |
| PL3041958T3 (pl) | 2020-11-02 |
| US20200283762A1 (en) | 2020-09-10 |
| DK3041958T3 (da) | 2020-03-09 |
| US20160194630A1 (en) | 2016-07-07 |
| ES2779302T3 (es) | 2020-08-14 |
| CA2930859A1 (en) | 2015-03-12 |
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