HRP20211397T1 - Pripravci za modulaciju ekspresije ataksina 2 - Google Patents
Pripravci za modulaciju ekspresije ataksina 2 Download PDFInfo
- Publication number
- HRP20211397T1 HRP20211397T1 HRP20211397TT HRP20211397T HRP20211397T1 HR P20211397 T1 HRP20211397 T1 HR P20211397T1 HR P20211397T T HRP20211397T T HR P20211397TT HR P20211397 T HRP20211397 T HR P20211397T HR P20211397 T1 HRP20211397 T1 HR P20211397T1
- Authority
- HR
- Croatia
- Prior art keywords
- modified
- linker
- seq
- stranded oligonucleotide
- internucleoside linker
- Prior art date
Links
- 102000007370 Ataxin2 Human genes 0.000 title claims 4
- 108010032951 Ataxin2 Proteins 0.000 title claims 4
- 239000000203 mixture Substances 0.000 title 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 26
- 239000002777 nucleoside Substances 0.000 claims 13
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 7
- 125000003835 nucleoside group Chemical group 0.000 claims 6
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 4
- 230000000295 complement effect Effects 0.000 claims 4
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 3
- 108020004999 messenger RNA Proteins 0.000 claims 3
- 125000002619 bicyclic group Chemical group 0.000 claims 2
- 239000003085 diluting agent Substances 0.000 claims 2
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical group P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 claims 2
- 239000002953 phosphate buffered saline Substances 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical group [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- 125000004400 (C1-C12) alkyl group Chemical group 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 150000004713 phosphodiesters Chemical group 0.000 claims 1
- 125000006239 protecting group Chemical group 0.000 claims 1
- 159000000000 sodium salts Chemical class 0.000 claims 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
-
- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- 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
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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
- C12N2310/341—Gapmers, i.e. of the type ===---===
-
- 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
- C12N2310/345—Spatial arrangement of the modifications having at least two different backbone 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
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Neurology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Neurosurgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Psychology (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)
Claims (14)
1. Modificirani jednolančani oligonukleotid, naznačen time, što se sastoji od 12 do 30 povezanih nukleozida i ima nukleobazni slijed koji sadrži najmanje 12 susjednih nukleobaza od bilo kojih nukleobaznih slijedova sa SEQ ID NO: 18, 19 ili 98, pri čemu je nukleobazni slijed modificiranog oligonukleotida najmanje 90% komplementaran sa SEQ ID NO: 1 ili SEQ ID NO: 2 i pritom modificirani jednolančani oligonukleotid smanjuje ekspresiju mRNA i/ili proteina ataksina 2.
2. Modificirani jednolančani oligonukleotid prema patentnom zahtjevu 1, naznačen time, što je nukleobazni slijed modificiranog oligonukleotida najmanje 95%, ili je 100% komplementaran sa SEQ ID NO: 1 ili SEQ ID NO: 2.
3. Modificirani jednolančani oligonukleotid prema bilo kojem prethodnom patentnom zahtjevu, naznačen time, što najmanje jedan međunukleozidni veznik jest modificirani međunukleozidni veznik; pri čemu opcionalno:
a) najmanje jedan modificirani međunukleozidni veznik jest fosforotioatni međunukleozidni veznik; ili
b) svaki modificirani međunukleozidni veznik jest fosforotioatni međunukleozidni veznik.
4. Modificirani jednolančani oligonukleotid prema bilo kojem prethodnom patentnom zahtjevu, naznačen time, što:
a) najmanje jedan međunukleozidni veznik jest fosfodiesterski međunukleozidni veznik; i/ili
b) najmanje jedan međunukleozidni veznik jest fosforotioatni veznik i najmanje jedan međunukleozidni veznik jest fosfodiesterski veznik.
5. Modificirani jednolančani olikonukleotid prema bilo kojem prethodnom patentnom zahtjevu, naznačen time, što najmanje jedan nukleozid sadrži modificiranu nukleobazu; pri čemu opcionalno, modificirana nukleobaza jest 5-metilcitozin.
6. Modificirani jednolančani olikonukleotid prema bilo kojem prethodnom patentnom zahtjevu, naznačen time, što najmanje jedan nukleozid od modificiranog oligonukleotida sadrži modificirani šećer.
7. Modificirani jednolančani olikonukleotid prema patentnom zahtjevu 6, naznačen time, što najmanje jedan modificirani šećer jest biciklički šećer; pri čemu opcionalno, biciklički šećer sadrži most 4'-CH(R)-O-2' gdje je R neovisno H, C1-C12 alkil, ili zaštitna skupina; nadalje opcionalno pritom:
a) R je metil; ili
b) R je H.
8. Modificirani jednolančani olikonukleotid prema patentnom zahtjevu 6, naznačen time, što najmanje jedan modificirani šećer sadrži skupinu 2'-O-metoksietil ili skupinu 2'-O-metil.
9. Modificirani jednolančani olikonukleotid prema bilo kojem prethodnom patentnom zahtjevu, naznačen time, što modificirani olikonukleotid sadrži:
prazan segment koji se sastoji od 10 povezanih deoksinukleozida;
5' krilni segment koji se sastoji od 5 povezanih nukleozida; i
3' krilni segment koji se sastoji od 5 povezanih nukleozida;
gdje je prazan segment smješten između 5' krilnog segmenta i 3' krilnog segmenta i pri čemu svaki nukleozid od svakog krilnog segmenta sadrži modificirani šećer.
10. Modificirani jednolančani oligonukleotid prema bilo kojem prethodnom patentnom zahtjevu, naznačen time, što se modificirani oligonukleotid sastoji od 20 povezanih nukleozida.
11. Modificirani jednolančani oligonukleotid prema bilo kojem prethodnom patentnom zahtjevu, naznačen time, što se sastoji od 20 povezanih nukleozida i ima nukleobazni slijed koji sadrži najmanje 15 susjednih nukleobaza sa SEQ ID NO: 18, pri čemu je nukleobazni slijed od modificiranog oligonukleotida 100% komplementaran sa SEQ ID NO: 1 ili SEQ ID NO: 2, gdje najmanje jedan nukleozid od modificiranog oligonukleotida sadrži modificirani šećer, dok najmanje jedan modificirani šećer sadrži skupinu 2'-O-metoksietil, pri čemu najmanje jedan nukleozid sadrži modificiranu nukleobazu koja je 5-metilcitozin, dok je najmanje jedan međunukleozidni veznik fosforotioatni veznik i najmanje jedan međunukleozidni veznik je fosfodiesterski veznik, i pritom modificirani jednolančani oligonukleotid smanjuje ekspresiju mRNA i/ili proteina ataksina 2.
12. Modificirani jednolančani oligonukleotid prema bilo kojem prethodnom patentnom zahtjevu, naznačen time, što se sastoji od 20 povezanih nukleozida i ima nukleobazni slijed koji sadrži najmanje 19 susjednih nukleobaza sa SEQ ID NO: 19, pri čemu je nukleobazni slijed od modificiranog oligonukleotida 100% komplementaran sa SEQ ID NO: 1 ili SEQ ID NO: 2, gdje najmanje jedan nukleozid od modificiranog oligonukleotida sadrži modificirani šećer, dok najmanje jedan modificirani šećer sadrži skupinu 2'-O-metoksietil, pri čemu najmanje jedan nukleozid sadrži modificiranu nukleobazu koja je 5-metilcitozin, dok je najmanje jedan međunukleozidni veznik fosforotioatni veznik i najmanje jedan međunukleozidni veznik je fosfodiesterski veznik, i pritom modificirani jednolančani oligonukleotid smanjuje ekspresiju mRNA i/ili proteina ataksina 2.
13. Pripravak, naznačen time, što sadrži modificirani jednolančani oligonukleotid prema bilo kojem prethodnom patentnom zahtjevu i najmanje jedan farmaceutski prihvatljiv nosač ili razrjeđivač, pri čemu opcionalno, farmaceutski prihvatljiv razrjeđivač jest fosfatno puferirana solna otopina (PBS).
14. Pripravak prema patentnom zahtjevu 13, naznačen time, što modificirani oligonukleotid jest natrijeva sol.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201461955705P | 2014-03-19 | 2014-03-19 | |
US201461982131P | 2014-04-21 | 2014-04-21 | |
EP15765851.9A EP3119888B1 (en) | 2014-03-19 | 2015-03-19 | Compositions for modulating ataxin 2 expression |
PCT/US2015/021608 WO2015143246A1 (en) | 2014-03-19 | 2015-03-19 | Compositions for modulating ataxin 2 expression |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20211397T1 true HRP20211397T1 (hr) | 2021-12-10 |
Family
ID=54145357
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20211397TT HRP20211397T1 (hr) | 2014-03-19 | 2021-09-02 | Pripravci za modulaciju ekspresije ataksina 2 |
Country Status (25)
Country | Link |
---|---|
US (3) | US10308934B2 (hr) |
EP (2) | EP3978610A3 (hr) |
JP (3) | JP6902869B2 (hr) |
KR (1) | KR102352589B1 (hr) |
CN (2) | CN116970607A (hr) |
AU (2) | AU2015231130C1 (hr) |
BR (1) | BR112016020472B1 (hr) |
CA (1) | CA2942340A1 (hr) |
CL (1) | CL2016002346A1 (hr) |
CY (1) | CY1124526T1 (hr) |
DK (1) | DK3119888T3 (hr) |
ES (1) | ES2890677T3 (hr) |
HK (1) | HK1232251A1 (hr) |
HR (1) | HRP20211397T1 (hr) |
HU (1) | HUE055877T2 (hr) |
IL (1) | IL247600B (hr) |
LT (1) | LT3119888T (hr) |
MX (1) | MX2016012123A (hr) |
PL (1) | PL3119888T3 (hr) |
PT (1) | PT3119888T (hr) |
RS (1) | RS62526B1 (hr) |
RU (2) | RU2702838C2 (hr) |
SG (2) | SG10201808063PA (hr) |
SI (1) | SI3119888T1 (hr) |
WO (1) | WO2015143246A1 (hr) |
Families Citing this family (18)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
RS62526B1 (sr) | 2014-03-19 | 2021-11-30 | Ionis Pharmaceuticals Inc | Kompozicije za modulaciju ekspresije ataksina 2 |
WO2015143245A1 (en) | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Methods for modulating ataxin 2 expression |
US10533175B2 (en) | 2015-09-25 | 2020-01-14 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating Ataxin 3 expression |
WO2017087282A1 (en) | 2015-11-18 | 2017-05-26 | Rosalind Franklin University Of Medicine And Science | Antisense compounds targeting leucine-rich repeat kinase 2 (lrrk2) for the treatment of parkinsons disease |
US10370667B2 (en) | 2015-11-18 | 2019-08-06 | Rosalind Franklin University Of Medicine And Science | Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease |
AU2016381174A1 (en) * | 2015-12-31 | 2018-05-31 | Ionis Pharmaceuticals, Inc. | Methods for reducing Ataxin-2 expression |
JOP20190104A1 (ar) | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
WO2019084068A1 (en) * | 2017-10-23 | 2019-05-02 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR NEURODEGENERATIVE DISEASE |
BR112020021253A2 (pt) | 2018-05-09 | 2021-02-02 | Ionis Pharmaceuticals, Inc. | compostos e métodos para redução da expressão de atxn3 |
WO2019233921A1 (en) | 2018-06-05 | 2019-12-12 | F. Hoffmann-La Roche Ag | Oligonucleotides for modulating atxn2 expression |
JP7411632B2 (ja) | 2018-07-25 | 2024-01-11 | 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 | Atxn2発現を低減するための化合物及び方法 |
BR112021010660A2 (pt) | 2018-12-06 | 2021-08-24 | Biogen Ma Inc. | Proteína de neurofilamento para guiar intervenção terapêutica sobre esclerose lateral amiotrófica |
TW202035694A (zh) * | 2018-12-14 | 2020-10-01 | 美商百健Ma公司 | 用於治療及預防肌肉萎縮性脊髓側索硬化症之組合物及方法 |
CN109529018B (zh) * | 2019-01-10 | 2021-11-30 | 南方医科大学南方医院 | Atxn2l用于制备逆转胃癌奥沙利铂耐药的药物的用途 |
EP3947677A1 (en) | 2019-04-04 | 2022-02-09 | F. Hoffmann-La Roche AG | Oligonucleotides for modulating atxn2 expression |
US11286485B2 (en) | 2019-04-04 | 2022-03-29 | Hoffmann-La Roche Inc. | Oligonucleotides for modulating ATXN2 expression |
EP4188390A1 (en) * | 2020-07-29 | 2023-06-07 | Alnylam Pharmaceuticals, Inc. | Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases |
WO2023250368A2 (en) * | 2022-06-24 | 2023-12-28 | Eli Lilly And Company | Atxn2 rna interference agents |
Family Cites Families (220)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
US4500707A (en) | 1980-02-29 | 1985-02-19 | University Patents, Inc. | Nucleosides useful in the preparation of polynucleotides |
US5132418A (en) | 1980-02-29 | 1992-07-21 | University Patents, Inc. | Process for preparing polynucleotides |
US4469863A (en) | 1980-11-12 | 1984-09-04 | Ts O Paul O P | Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof |
US4973679A (en) | 1981-03-27 | 1990-11-27 | University Patents, Inc. | Process for oligonucleo tide synthesis using phosphormidite intermediates |
US4415732A (en) | 1981-03-27 | 1983-11-15 | University Patents, Inc. | Phosphoramidite compounds and processes |
US5023243A (en) | 1981-10-23 | 1991-06-11 | Molecular Biosystems, Inc. | Oligonucleotide therapeutic agent and method of making same |
US4476301A (en) | 1982-04-29 | 1984-10-09 | Centre National De La Recherche Scientifique | Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon |
DE3329892A1 (de) | 1983-08-18 | 1985-03-07 | Köster, Hubert, Prof. Dr., 2000 Hamburg | Verfahren zur herstellung von oligonucleotiden |
US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
USRE34036E (en) | 1984-06-06 | 1992-08-18 | National Research Development Corporation | Data transmission using a transparent tone-in band system |
US5550111A (en) | 1984-07-11 | 1996-08-27 | Temple University-Of The Commonwealth System Of Higher Education | Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof |
FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
US5367066A (en) | 1984-10-16 | 1994-11-22 | Chiron Corporation | Oligonucleotides with selectably cleavable and/or abasic sites |
FR2575751B1 (fr) | 1985-01-08 | 1987-04-03 | Pasteur Institut | Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques |
US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
US5235033A (en) | 1985-03-15 | 1993-08-10 | Anti-Gene Development Group | Alpha-morpholino ribonucleoside derivatives and polymers thereof |
US5405938A (en) | 1989-12-20 | 1995-04-11 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
EP0260032B1 (en) | 1986-09-08 | 1994-01-26 | Ajinomoto Co., Inc. | Compounds for the cleavage at a specific position of RNA, oligomers employed for the formation of said compounds, and starting materials for the synthesis of said oligomers |
US5264423A (en) | 1987-03-25 | 1993-11-23 | The United States Of America As Represented By The Department Of Health And Human Services | Inhibitors for replication of retroviruses and for the expression of oncogene products |
US5276019A (en) | 1987-03-25 | 1994-01-04 | The United States Of America As Represented By The Department Of Health And Human Services | Inhibitors for replication of retroviruses and for the expression of oncogene products |
ATE113059T1 (de) | 1987-06-24 | 1994-11-15 | Florey Howard Inst | Nukleosid-derivate. |
US5188897A (en) | 1987-10-22 | 1993-02-23 | Temple University Of The Commonwealth System Of Higher Education | Encapsulated 2',5'-phosphorothioate oligoadenylates |
US4924624A (en) | 1987-10-22 | 1990-05-15 | Temple University-Of The Commonwealth System Of Higher Education | 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof |
EP0348458B1 (en) | 1987-11-30 | 1997-04-09 | University Of Iowa Research Foundation | Dna molecules stabilized by modifications of the 3'-terminal phosphodiester linkage and their use as nucleic acid probes and as therapeutic agents to block the expression of specifically targeted genes |
US5403711A (en) | 1987-11-30 | 1995-04-04 | University Of Iowa Research Foundation | Nucleic acid hybridization and amplification method for detection of specific sequences in which a complementary labeled nucleic acid probe is cleaved |
JPH03503894A (ja) | 1988-03-25 | 1991-08-29 | ユニバーシィティ オブ バージニア アランミ パテンツ ファウンデイション | オリゴヌクレオチド n‐アルキルホスホラミデート |
US5278302A (en) | 1988-05-26 | 1994-01-11 | University Patents, Inc. | Polynucleotide phosphorodithioates |
US5216141A (en) | 1988-06-06 | 1993-06-01 | Benner Steven A | Oligonucleotide analogs containing sulfur linkages |
US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
US5194599A (en) | 1988-09-23 | 1993-03-16 | Gilead Sciences, Inc. | Hydrogen phosphonodithioate compositions |
US5256775A (en) | 1989-06-05 | 1993-10-26 | Gilead Sciences, Inc. | Exonuclease-resistant oligonucleotides |
US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
US5591722A (en) | 1989-09-15 | 1997-01-07 | Southern Research Institute | 2'-deoxy-4'-thioribonucleosides and their antiviral activity |
US5721218A (en) | 1989-10-23 | 1998-02-24 | Gilead Sciences, Inc. | Oligonucleotides with inverted polarity |
US5399676A (en) | 1989-10-23 | 1995-03-21 | Gilead Sciences | Oligonucleotides with inverted polarity |
DK0497875T3 (da) | 1989-10-24 | 2000-07-03 | Gilead Sciences Inc | 2'-modificerede oligonukleotider |
US5264562A (en) | 1989-10-24 | 1993-11-23 | Gilead Sciences, Inc. | Oligonucleotide analogs with novel linkages |
US5264564A (en) | 1989-10-24 | 1993-11-23 | Gilead Sciences | Oligonucleotide analogs with novel linkages |
US5177198A (en) | 1989-11-30 | 1993-01-05 | University Of N.C. At Chapel Hill | Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates |
US5130302A (en) | 1989-12-20 | 1992-07-14 | Boron Bilogicals, Inc. | Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same |
US6005087A (en) | 1995-06-06 | 1999-12-21 | Isis Pharmaceuticals, Inc. | 2'-modified oligonucleotides |
US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
US5859221A (en) | 1990-01-11 | 1999-01-12 | Isis Pharmaceuticals, Inc. | 2'-modified oligonucleotides |
US5646265A (en) | 1990-01-11 | 1997-07-08 | Isis Pharmceuticals, Inc. | Process for the preparation of 2'-O-alkyl purine phosphoramidites |
US5457191A (en) | 1990-01-11 | 1995-10-10 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
US5670633A (en) | 1990-01-11 | 1997-09-23 | Isis Pharmaceuticals, Inc. | Sugar modified oligonucleotides that detect and modulate gene expression |
US7101993B1 (en) | 1990-01-11 | 2006-09-05 | Isis Pharmaceuticals, Inc. | Oligonucleotides containing 2′-O-modified purines |
US5623065A (en) | 1990-08-13 | 1997-04-22 | Isis Pharmaceuticals, Inc. | Gapped 2' modified oligonucleotides |
US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
US5587361A (en) | 1991-10-15 | 1996-12-24 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
US5220007A (en) | 1990-02-15 | 1993-06-15 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of RNA and production of encoded polypeptides |
US5149797A (en) | 1990-02-15 | 1992-09-22 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of rna and production of encoded polypeptides |
US5321131A (en) | 1990-03-08 | 1994-06-14 | Hybridon, Inc. | Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling |
US5470967A (en) | 1990-04-10 | 1995-11-28 | The Dupont Merck Pharmaceutical Company | Oligonucleotide analogs with sulfamate linkages |
GB9009980D0 (en) | 1990-05-03 | 1990-06-27 | Amersham Int Plc | Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides |
DE69032425T2 (de) | 1990-05-11 | 1998-11-26 | Microprobe Corp., Bothell, Wash. | Teststreifen zum Eintauchen für Nukleinsäure-Hybridisierungsassays und Verfahren zur kovalenten Immobilisierung von Oligonucleotiden |
US5489677A (en) | 1990-07-27 | 1996-02-06 | Isis Pharmaceuticals, Inc. | Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms |
US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
US5541307A (en) | 1990-07-27 | 1996-07-30 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs and solid phase synthesis thereof |
US5608046A (en) | 1990-07-27 | 1997-03-04 | Isis Pharmaceuticals, Inc. | Conjugated 4'-desmethyl nucleoside analog compounds |
US5223618A (en) | 1990-08-13 | 1993-06-29 | Isis Pharmaceuticals, Inc. | 4'-desmethyl nucleoside analog compounds |
US5386023A (en) | 1990-07-27 | 1995-01-31 | Isis Pharmaceuticals | Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling |
ATE154246T1 (de) | 1990-07-27 | 1997-06-15 | Isis Pharmaceuticals Inc | Nuklease resistente, pyrimidin modifizierte oligonukleotide, die die gen-expression detektieren und modulieren |
US5610289A (en) | 1990-07-27 | 1997-03-11 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogues |
US5602240A (en) | 1990-07-27 | 1997-02-11 | Ciba Geigy Ag. | Backbone modified oligonucleotide analogs |
US5677437A (en) | 1990-07-27 | 1997-10-14 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
US5618704A (en) | 1990-07-27 | 1997-04-08 | Isis Pharmacueticals, Inc. | Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling |
US5623070A (en) | 1990-07-27 | 1997-04-22 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
HU217036B (hu) | 1990-08-03 | 1999-11-29 | Sanofi | Eljárás génexpresszió gátlására alkalmas vegyületek előállítására |
US5177196A (en) | 1990-08-16 | 1993-01-05 | Microprobe Corporation | Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof |
US5214134A (en) | 1990-09-12 | 1993-05-25 | Sterling Winthrop Inc. | Process of linking nucleosides with a siloxane bridge |
US5561225A (en) | 1990-09-19 | 1996-10-01 | Southern Research Institute | Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages |
CA2092002A1 (en) | 1990-09-20 | 1992-03-21 | Mark Matteucci | Modified internucleoside linkages |
US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
US6582908B2 (en) | 1990-12-06 | 2003-06-24 | Affymetrix, Inc. | Oligonucleotides |
US5948903A (en) | 1991-01-11 | 1999-09-07 | Isis Pharmaceuticals, Inc. | Synthesis of 3-deazapurines |
US5672697A (en) | 1991-02-08 | 1997-09-30 | Gilead Sciences, Inc. | Nucleoside 5'-methylene phosphonates |
US7015315B1 (en) | 1991-12-24 | 2006-03-21 | Isis Pharmaceuticals, Inc. | Gapped oligonucleotides |
US5571799A (en) | 1991-08-12 | 1996-11-05 | Basco, Ltd. | (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response |
EP0538194B1 (de) | 1991-10-17 | 1997-06-04 | Novartis AG | Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte |
US5594121A (en) | 1991-11-07 | 1997-01-14 | Gilead Sciences, Inc. | Enhanced triple-helix and double-helix formation with oligomers containing modified purines |
TW393513B (en) | 1991-11-26 | 2000-06-11 | Isis Pharmaceuticals Inc | Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines |
US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
ATE226093T1 (de) | 1991-11-26 | 2002-11-15 | Isis Pharmaceuticals Inc | Gesteigerte bildung von triple- und doppelhelices aus oligomeren mit modifizierten pyrimidinen |
US5792608A (en) | 1991-12-12 | 1998-08-11 | Gilead Sciences, Inc. | Nuclease stable and binding competent oligomers and methods for their use |
US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
US5700922A (en) | 1991-12-24 | 1997-12-23 | Isis Pharmaceuticals, Inc. | PNA-DNA-PNA chimeric macromolecules |
FR2687679B1 (fr) | 1992-02-05 | 1994-10-28 | Centre Nat Rech Scient | Oligothionucleotides. |
US5633360A (en) | 1992-04-14 | 1997-05-27 | Gilead Sciences, Inc. | Oligonucleotide analogs capable of passive cell membrane permeation |
US5434257A (en) | 1992-06-01 | 1995-07-18 | Gilead Sciences, Inc. | Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages |
EP0577558A2 (de) | 1992-07-01 | 1994-01-05 | Ciba-Geigy Ag | Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte |
US5652355A (en) | 1992-07-23 | 1997-07-29 | Worcester Foundation For Experimental Biology | Hybrid oligonucleotide phosphorothioates |
JPH08504559A (ja) | 1992-12-14 | 1996-05-14 | ハネウエル・インコーポレーテッド | 個別に制御される冗長巻線を有するモータシステム |
US5476925A (en) | 1993-02-01 | 1995-12-19 | Northwestern University | Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups |
GB9304618D0 (en) | 1993-03-06 | 1993-04-21 | Ciba Geigy Ag | Chemical compounds |
GB9304620D0 (en) | 1993-03-06 | 1993-04-21 | Ciba Geigy Ag | Compounds |
EP0691968B1 (en) | 1993-03-30 | 1997-07-16 | Sanofi | Acyclic nucleoside analogs and oligonucleotide sequences containing them |
WO1994022891A1 (en) | 1993-03-31 | 1994-10-13 | Sterling Winthrop Inc. | Oligonucleotides with amide linkages replacing phosphodiester linkages |
US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
US5801154A (en) | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
US5446137B1 (en) | 1993-12-09 | 1998-10-06 | Behringwerke Ag | Oligonucleotides containing 4'-substituted nucleotides |
ATE196311T1 (de) | 1993-12-09 | 2000-09-15 | Univ Jefferson | Verbindungen und verfahren zur ortsspezifischen mutation in eukaryotischen zellen |
US5519134A (en) | 1994-01-11 | 1996-05-21 | Isis Pharmaceuticals, Inc. | Pyrrolidine-containing monomers and oligomers |
US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
US5627053A (en) | 1994-03-29 | 1997-05-06 | Ribozyme Pharmaceuticals, Inc. | 2'deoxy-2'-alkylnucleotide containing nucleic acid |
US5625050A (en) | 1994-03-31 | 1997-04-29 | Amgen Inc. | Modified oligonucleotides and intermediates useful in nucleic acid therapeutics |
US5646269A (en) | 1994-04-28 | 1997-07-08 | Gilead Sciences, Inc. | Method for oligonucleotide analog synthesis |
US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
US5597909A (en) | 1994-08-25 | 1997-01-28 | Chiron Corporation | Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use |
US5652356A (en) | 1995-08-17 | 1997-07-29 | Hybridon, Inc. | Inverted chimeric and hybrid oligonucleotides |
CA2238379A1 (en) | 1995-11-22 | 1997-06-12 | The Johns-Hopkins University | Ligands to enhance cellular uptake of biomolecules |
US6673535B1 (en) | 1996-05-08 | 2004-01-06 | Cedars-Sinai Medical Center | Methods of detecting spinocerebellar Ataxia-2 nucleic acids |
WO1997042314A1 (en) * | 1996-05-08 | 1997-11-13 | Cedars-Sinai Medical Center | Nucleic acid encoding spinocerebellar ataxia-2 and products related thereto |
US6844431B1 (en) | 1996-05-08 | 2005-01-18 | Cedars-Sinai Medical Center | Nucleic acid encoding spinocerebellar ataxia-2 and products related thereto |
EP0878543A1 (en) | 1996-10-30 | 1998-11-18 | Srl, Inc. | cDNA FRAGMENTS OF GENE CAUSATIVE OF SPINOCEREBELLAR ATAXIA TYPE 2 |
US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
USRE44779E1 (en) | 1997-03-07 | 2014-02-25 | Santaris Pharma A/S | Bicyclonucleoside and oligonucleotide analogues |
JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
US7572582B2 (en) | 1997-09-12 | 2009-08-11 | Exiqon A/S | Oligonucleotide analogues |
EP2253639A1 (en) | 1997-09-12 | 2010-11-24 | Exiqon A/S | Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues |
US20030228597A1 (en) | 1998-04-13 | 2003-12-11 | Cowsert Lex M. | Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation |
US6300319B1 (en) | 1998-06-16 | 2001-10-09 | Isis Pharmaceuticals, Inc. | Targeted oligonucleotide conjugates |
US6043352A (en) | 1998-08-07 | 2000-03-28 | Isis Pharmaceuticals, Inc. | 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides |
WO2000015265A1 (en) * | 1998-09-16 | 2000-03-23 | Vitagenix, Inc. | Compositions and methods of use of uptake mechanisms for diagnostic and therapeutic applications of oligonucleotides targeting bacteria |
EP1178999B1 (en) | 1999-05-04 | 2007-03-14 | Santaris Pharma A/S | L-ribo-lna analogues |
US6525191B1 (en) | 1999-05-11 | 2003-02-25 | Kanda S. Ramasamy | Conformationally constrained L-nucleosides |
WO2000070039A1 (en) * | 1999-05-17 | 2000-11-23 | Mcgill University | METHOD FOR SUBTRACTING cDNAs BY SUPPRESSING THE SYNTHESIS OF SPECIFICALLY TARGETED mRNAs |
US7491805B2 (en) | 2001-05-18 | 2009-02-17 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
US6372433B1 (en) * | 2000-04-28 | 2002-04-16 | Isis Pharmaceuticals, Inc. | Antisense modulation of inhibitor of DNA binding-1 expression |
US6426220B1 (en) | 2000-10-30 | 2002-07-30 | Isis Pharmaceuticals, Inc. | Antisense modulation of calreticulin expression |
AU2002217980A1 (en) | 2000-12-01 | 2002-06-11 | Cell Works Inc. | Conjugates of glycosylated/galactosylated peptide |
WO2003004602A2 (en) | 2001-07-03 | 2003-01-16 | Isis Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
US20030175906A1 (en) | 2001-07-03 | 2003-09-18 | Muthiah Manoharan | Nuclease resistant chimeric oligonucleotides |
US20030158403A1 (en) | 2001-07-03 | 2003-08-21 | Isis Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
WO2003033741A1 (en) * | 2001-10-16 | 2003-04-24 | Aclara Biosciences, Inc. | Universal e-tag primer and probe compositions and methods |
JP2005532052A (ja) | 2002-07-01 | 2005-10-27 | ファルマシア・コーポレーション | Lrh1発現のアンチセンス調節 |
US20050042646A1 (en) * | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
AU2003291753B2 (en) | 2002-11-05 | 2010-07-08 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
AU2003291755A1 (en) | 2002-11-05 | 2004-06-07 | Isis Pharmaceuticals, Inc. | Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use |
ATE517992T1 (de) | 2002-11-14 | 2011-08-15 | Dharmacon Inc | Funktionelle und hyperfunktionelle sirna |
US7250289B2 (en) | 2002-11-20 | 2007-07-31 | Affymetrix, Inc. | Methods of genetic analysis of mouse |
US7829694B2 (en) | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
US7605249B2 (en) * | 2002-11-26 | 2009-10-20 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
WO2004070062A2 (en) * | 2003-02-04 | 2004-08-19 | Wyeth | Compositions and methods for diagnosing and treating cancers |
US7723509B2 (en) | 2003-04-17 | 2010-05-25 | Alnylam Pharmaceuticals | IRNA agents with biocleavable tethers |
US7339051B2 (en) * | 2003-04-28 | 2008-03-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for the treatment of severe acute respiratory syndrome (SARS) |
WO2004106356A1 (en) | 2003-05-27 | 2004-12-09 | Syddansk Universitet | Functionalized nucleotide derivatives |
US7888497B2 (en) | 2003-08-13 | 2011-02-15 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof |
US7427672B2 (en) | 2003-08-28 | 2008-09-23 | Takeshi Imanishi | Artificial nucleic acids of n-o bond crosslinkage type |
AU2004274021B2 (en) | 2003-09-18 | 2009-08-13 | Isis Pharmaceuticals, Inc. | 4'-thionucleosides and oligomeric compounds |
US20050244851A1 (en) | 2004-01-13 | 2005-11-03 | Affymetrix, Inc. | Methods of analysis of alternative splicing in human |
EP1568383A3 (en) | 2004-02-27 | 2005-11-16 | Antisense Pharma GmbH | Use of an oligonucleotide or its active derivative for the preparation of a pharmaceutical composition for inhibiting the formation of metastases in cancer treatment |
US20050209178A1 (en) | 2004-03-16 | 2005-09-22 | Stefan Pulst | Methods and compositions for the treatment of obesity |
US7374927B2 (en) * | 2004-05-03 | 2008-05-20 | Affymetrix, Inc. | Methods of analysis of degraded nucleic acid samples |
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 |
GB0415359D0 (en) * | 2004-07-08 | 2004-08-11 | Aimsco Ltd | Medicament |
BRPI0513062A (pt) | 2004-07-08 | 2008-04-22 | Aimsco Ltd | composição farmacêutica, método de estimular a produção de pomc em um paciente, usos de um peptìdeo de crf isolado e de um peptìdeo de pomc isolado, e, métodos de tratamento para uma doença, de produzir crf e de tratamento curativo, melhorador ou profilático de uma doença |
US20060148740A1 (en) | 2005-01-05 | 2006-07-06 | Prosensa B.V. | Mannose-6-phosphate receptor mediated gene transfer into muscle cells |
US20060270727A1 (en) * | 2005-05-03 | 2006-11-30 | Christian Melander | Small molecule therapeutics and uses therefor |
US20100266574A1 (en) | 2005-06-10 | 2010-10-21 | Orna Mor | Oligoribonucleotides and Methods of Use Thereof for Treatment of Fibrotic Conditions and Other Diseases |
US7569686B1 (en) | 2006-01-27 | 2009-08-04 | Isis Pharmaceuticals, Inc. | Compounds and methods for synthesis of bicyclic nucleic acid analogs |
AU2007211080B9 (en) | 2006-01-27 | 2012-05-03 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
US20110177960A1 (en) * | 2006-03-10 | 2011-07-21 | Ellen Murphy | Microarray for monitoring gene expression in multiple strains of Streptococcus pneumoniae |
ES2389737T3 (es) | 2006-05-11 | 2012-10-31 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos modificados en 5' |
US7666854B2 (en) | 2006-05-11 | 2010-02-23 | Isis Pharmaceuticals, Inc. | Bis-modified bicyclic nucleic acid analogs |
EP2049664B1 (en) | 2006-08-11 | 2011-09-14 | Prosensa Technologies B.V. | Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders |
JP5665317B2 (ja) | 2006-10-18 | 2015-02-04 | アイシス ファーマシューティカルズ, インコーポレーテッド | アンチセンス化合物 |
US20100190837A1 (en) | 2007-02-15 | 2010-07-29 | Isis Pharmaceuticals, Inc. | 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom |
WO2008109379A1 (en) | 2007-03-02 | 2008-09-12 | Mdrna, Inc. | Nucleic acid compounds for inhibiting il17a gene expression and uses thereof |
WO2008109369A2 (en) | 2007-03-02 | 2008-09-12 | Mdrna, Inc. | Nucleic acid compounds for inhibiting tnf gene expression and uses thereof |
EP2170917B1 (en) | 2007-05-30 | 2012-06-27 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
EP2173760B2 (en) | 2007-06-08 | 2015-11-04 | Isis Pharmaceuticals, Inc. | Carbocyclic bicyclic nucleic acid analogs |
US20100273854A1 (en) * | 2007-06-15 | 2010-10-28 | Hagar Kalinski | Compositions and methods for inhibiting nadph oxidase expression |
DK2176280T4 (en) | 2007-07-05 | 2015-07-20 | Isis Pharmaceuticals Inc | 6-Disubstituerede bicykliske nukleinsyreanaloge |
US8088904B2 (en) | 2007-08-15 | 2012-01-03 | Isis Pharmaceuticals, Inc. | Tetrahydropyran nucleic acid analogs |
CA2701128A1 (en) * | 2007-10-01 | 2009-04-09 | Isis Pharmaceuticals, Inc. | Antisense modulation of fibroblast growth factor receptor 4 expression |
WO2009067647A1 (en) | 2007-11-21 | 2009-05-28 | Isis Pharmaceuticals, Inc. | Carbocyclic alpha-l-bicyclic nucleic acid analogs |
EP2231195B1 (en) | 2007-12-04 | 2017-03-29 | Arbutus Biopharma Corporation | Targeting lipids |
EP2265627A2 (en) | 2008-02-07 | 2010-12-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexitol nucleic acid analogs |
JP5524189B2 (ja) * | 2008-06-06 | 2014-06-18 | クォーク ファーマシューティカルズ インコーポレーティッド | 耳障害治療のための組成物および方法 |
EP2317847B1 (en) | 2008-07-29 | 2019-04-17 | The Board of Regents of The University of Texas System | Selective inhibition of polyglutamine protein expression |
WO2010036698A1 (en) | 2008-09-24 | 2010-04-01 | Isis Pharmaceuticals, Inc. | Substituted alpha-l-bicyclic nucleosides |
MX2011013421A (es) * | 2009-06-15 | 2012-03-16 | Alnylam Pharmaceuticals Inc | Arnds formulado con lipido de direccionamiento del gen pcsk9. |
MX2012000468A (es) | 2009-07-09 | 2012-03-07 | Abraxis Bioscience Llc | Composiciones antisentido de proteina acida secretada y rica en cisteina (sparc) y usos de las mismas. |
WO2011017521A2 (en) | 2009-08-06 | 2011-02-10 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
US20110142789A1 (en) | 2009-12-10 | 2011-06-16 | The Trustees Of The University Of Pennsylvania | Compositions and Methods for the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis |
EP3000885B1 (en) * | 2009-12-18 | 2018-07-25 | Arrowhead Pharmaceuticals, Inc. | Organic compositions to treat hsf1-related diseases |
US9574191B2 (en) | 2010-02-03 | 2017-02-21 | The Board Of Regents Of The University Of Texas System | Selective inhibition of polyglutamine protein expression |
WO2011097641A1 (en) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Methods and compositions useful in treatment of diseases or conditions related to repeat expansion |
WO2011133876A2 (en) | 2010-04-22 | 2011-10-27 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising acyclic and abasic nucleosides and analogs |
CN106434648A (zh) | 2010-07-19 | 2017-02-22 | F·C·贝内特 | 肌强直性营养障碍蛋白激酶(dmpk)表达的调节 |
AU2011302152B2 (en) | 2010-09-15 | 2015-06-11 | Alnylam Pharmaceuticals, Inc. | Modified iRNA agents |
WO2012079578A1 (en) | 2010-12-15 | 2012-06-21 | Syddansk Universitet | Selective killing of cancer cells |
EP2673361B1 (en) | 2011-02-08 | 2016-04-13 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
CN106912197B (zh) | 2011-04-28 | 2022-01-25 | 生命技术公司 | 用于多重pcr的方法和组合物 |
WO2012177639A2 (en) * | 2011-06-22 | 2012-12-27 | Alnylam Pharmaceuticals, Inc. | Bioprocessing and bioproduction using avian cell lines |
EP2751270B1 (en) | 2011-08-29 | 2018-08-22 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
WO2013081864A1 (en) * | 2011-11-29 | 2013-06-06 | Life Technologies Corporation | Methods and compositions for multiplex pcr |
EP2785729B1 (en) | 2011-11-30 | 2020-11-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
EP3336189A1 (en) | 2012-04-20 | 2018-06-20 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
RS56319B1 (sr) | 2012-04-23 | 2017-12-29 | Biomarin Tech Bv | Oligonukleotidi koji moduliraju rnk sa poboljšanim karakteristikama za lečenje neuromišićnih poremećaja |
WO2013173645A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating utrn expression |
JP2016522674A (ja) | 2012-05-16 | 2016-08-04 | ラナ セラピューティクス インコーポレイテッド | 遺伝子発現を調節するための組成物及び方法 |
EP2906699A4 (en) | 2012-10-11 | 2016-06-08 | Ionis Pharmaceuticals Inc | OLIGOMER COMPOUNDS WITH BICYCLIC NUCLEOSIDES AND USES THEREOF |
JP6570447B2 (ja) | 2012-10-15 | 2019-09-04 | 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. | C9orf72発現を調節するための組成物 |
CN104884618A (zh) | 2012-11-15 | 2015-09-02 | 罗氏创新中心哥本哈根有限公司 | 寡核苷酸缀合物 |
RU2650510C2 (ru) | 2013-05-01 | 2018-04-16 | Ионис Фармасьютикалз, Инк. | Композиции и способы модулирования экспрессии аполипопротеина c-iii |
CA2917229A1 (en) | 2013-07-02 | 2015-01-08 | Isis Pharmaceuticals, Inc. | Modulators of growth hormone receptor |
JP2015092853A (ja) | 2013-11-12 | 2015-05-18 | 国立大学法人名古屋大学 | Als疾患関連遺伝子配列解析用の補足pcrプライマーセット、als疾患関連遺伝子配列の解析方法、及びals疾患の検査方法 |
WO2015143245A1 (en) | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Methods for modulating ataxin 2 expression |
RS62526B1 (sr) | 2014-03-19 | 2021-11-30 | Ionis Pharmaceuticals Inc | Kompozicije za modulaciju ekspresije ataksina 2 |
AU2016381174A1 (en) | 2015-12-31 | 2018-05-31 | Ionis Pharmaceuticals, Inc. | Methods for reducing Ataxin-2 expression |
JP7411632B2 (ja) | 2018-07-25 | 2024-01-11 | 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 | Atxn2発現を低減するための化合物及び方法 |
-
2015
- 2015-03-19 RS RS20211237A patent/RS62526B1/sr unknown
- 2015-03-19 MX MX2016012123A patent/MX2016012123A/es unknown
- 2015-03-19 PT PT15765851T patent/PT3119888T/pt unknown
- 2015-03-19 WO PCT/US2015/021608 patent/WO2015143246A1/en active Application Filing
- 2015-03-19 IL IL247600A patent/IL247600B/en unknown
- 2015-03-19 PL PL15765851T patent/PL3119888T3/pl unknown
- 2015-03-19 AU AU2015231130A patent/AU2015231130C1/en active Active
- 2015-03-19 JP JP2016558122A patent/JP6902869B2/ja active Active
- 2015-03-19 SG SG10201808063PA patent/SG10201808063PA/en unknown
- 2015-03-19 CN CN202310849672.9A patent/CN116970607A/zh active Pending
- 2015-03-19 RU RU2016140897A patent/RU2702838C2/ru active
- 2015-03-19 LT LTEPPCT/US2015/021608T patent/LT3119888T/lt unknown
- 2015-03-19 CA CA2942340A patent/CA2942340A1/en active Pending
- 2015-03-19 CN CN201580012549.5A patent/CN106103717A/zh active Pending
- 2015-03-19 SG SG11201607761UA patent/SG11201607761UA/en unknown
- 2015-03-19 BR BR112016020472-7A patent/BR112016020472B1/pt active IP Right Grant
- 2015-03-19 DK DK15765851.9T patent/DK3119888T3/da active
- 2015-03-19 EP EP21187734.5A patent/EP3978610A3/en active Pending
- 2015-03-19 SI SI201531693T patent/SI3119888T1/sl unknown
- 2015-03-19 KR KR1020167027552A patent/KR102352589B1/ko active IP Right Grant
- 2015-03-19 ES ES15765851T patent/ES2890677T3/es active Active
- 2015-03-19 EP EP15765851.9A patent/EP3119888B1/en active Active
- 2015-03-19 US US15/127,358 patent/US10308934B2/en active Active
- 2015-03-19 HU HUE15765851A patent/HUE055877T2/hu unknown
- 2015-03-19 RU RU2019130898A patent/RU2019130898A/ru unknown
-
2016
- 2016-09-16 CL CL2016002346A patent/CL2016002346A1/es unknown
-
2017
- 2017-06-09 HK HK17105750.0A patent/HK1232251A1/zh unknown
-
2019
- 2019-04-17 US US16/387,308 patent/US11111494B2/en active Active
-
2021
- 2021-03-30 JP JP2021056879A patent/JP7194222B2/ja active Active
- 2021-08-04 US US17/393,738 patent/US11834660B2/en active Active
- 2021-08-28 AU AU2021221934A patent/AU2021221934B2/en active Active
- 2021-09-02 HR HRP20211397TT patent/HRP20211397T1/hr unknown
- 2021-09-24 CY CY20211100838T patent/CY1124526T1/el unknown
-
2022
- 2022-12-09 JP JP2022196985A patent/JP2023027236A/ja active Pending
Also Published As
Similar Documents
Publication | Publication Date | Title |
---|---|---|
HRP20211397T1 (hr) | Pripravci za modulaciju ekspresije ataksina 2 | |
HRP20201426T1 (hr) | Postupci i pripravci za moduliranje ekspresije apolipoproteina (a) | |
HRP20211051T1 (hr) | Pripravci namijenjeni moduliranju eksprimiranja tau | |
HRP20211365T1 (hr) | Pripravci i postupci za modulaciju ekspresije proteinske kinaze miotonične distrofije (dmpk) | |
RU2016116849A (ru) | Композиции для модуляции экспрессии c90rf72 | |
HRP20201078T1 (hr) | Pripravci namijenjeni moduliranju eksprimiranja sod-1 | |
HRP20201125T1 (hr) | Antisens nukleinska kiselina | |
HRP20191600T1 (hr) | Modulatori faktora b komplementa | |
HRP20200042T1 (hr) | Suprotne nukleinske kiseline za uporabu u liječenju duchennove mišićne distrofije | |
HRP20210227T1 (hr) | Oligonukleotidi za indukciju ekspresije paternalnog gena ube3a | |
JP2017510271A5 (hr) | ||
JP2013226147A5 (hr) | ||
PE20181267A1 (es) | Modulacion de la expresion del virus de la hepatitis b (vhb) | |
RU2017139121A (ru) | Композиции для модулирования экспрессии c9orf72 | |
EA201890494A1 (ru) | Способы лечения вирусных инфекций arenaviridae и coronaviridae | |
NZ710192A (en) | Modulation of apolipoprotein ciii (apociii) expression | |
HRP20160336T1 (hr) | Protusmjerna nukleinska kiselina | |
DK3821011T3 (da) | Transposom-aktiveret dna/rna-sekventering (ted rna-seq) | |
MX2017002144A (es) | Agentes de ácido ribonucleico (arn) de cadena doble modificados. | |
IL288490A (en) | RNA editor complementary strand oligonucleotides containing cytidine analogs | |
EA201490993A1 (ru) | МОДИФИЦИРОВАННЫЕ СРЕДСТВА РНКи | |
BR112014007852A2 (pt) | nucleosídeos, nucleotídeos e ácidos nucleicos modificados e usos dos mesmos | |
SI2379084T1 (en) | Modulation of factor expression 11 | |
EA201692535A1 (ru) | Замещенные нуклеозиды, нуклеотиды и их аналоги | |
ZA201404444B (en) | 4'-azido,3'-fluoro substituted nucleoside derivatives as inhibitors of hcv rna replication |