HRP20210877T1 - Antisens polinukleotidi za induciranje preskakanja egzona i postupci liječenja distrofija - Google Patents
Antisens polinukleotidi za induciranje preskakanja egzona i postupci liječenja distrofija Download PDFInfo
- Publication number
- HRP20210877T1 HRP20210877T1 HRP20210877TT HRP20210877T HRP20210877T1 HR P20210877 T1 HRP20210877 T1 HR P20210877T1 HR P20210877T T HRP20210877T T HR P20210877TT HR P20210877 T HRP20210877 T HR P20210877T HR P20210877 T1 HRP20210877 T1 HR P20210877T1
- Authority
- HR
- Croatia
- Prior art keywords
- antisense polynucleotide
- use according
- polynucleotide
- exon
- modified
- Prior art date
Links
- 108091033319 polynucleotide Proteins 0.000 title claims 29
- 102000040430 polynucleotide Human genes 0.000 title claims 29
- 239000002157 polynucleotide Substances 0.000 title claims 29
- 230000000692 anti-sense effect Effects 0.000 title claims 21
- 108090000765 processed proteins & peptides Proteins 0.000 claims 4
- 102100021792 Gamma-sarcoglycan Human genes 0.000 claims 3
- 201000006938 muscular dystrophy Diseases 0.000 claims 3
- 102000019344 Gamma-sarcoglycan Human genes 0.000 claims 2
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims 2
- 108010083379 Sarcoglycans Proteins 0.000 claims 2
- 210000004027 cell Anatomy 0.000 claims 2
- 230000004700 cellular uptake Effects 0.000 claims 2
- 210000000663 muscle cell Anatomy 0.000 claims 2
- 230000004220 muscle function Effects 0.000 claims 2
- 230000010837 receptor-mediated endocytosis Effects 0.000 claims 2
- 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 1
- 241000713772 Human immunodeficiency virus 1 Species 0.000 claims 1
- 102100034343 Integrase Human genes 0.000 claims 1
- 101710203526 Integrase Proteins 0.000 claims 1
- 239000002202 Polyethylene glycol Substances 0.000 claims 1
- 101710149951 Protein Tat Proteins 0.000 claims 1
- 230000001413 cellular effect Effects 0.000 claims 1
- BOKOVLFWCAFYHP-UHFFFAOYSA-N dihydroxy-methoxy-sulfanylidene-$l^{5}-phosphane Chemical compound COP(O)(O)=S BOKOVLFWCAFYHP-UHFFFAOYSA-N 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 102000034240 fibrous proteins Human genes 0.000 claims 1
- 108091005899 fibrous proteins Proteins 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 102000006495 integrins Human genes 0.000 claims 1
- 108010044426 integrins Proteins 0.000 claims 1
- YACKEPLHDIMKIO-UHFFFAOYSA-L methylphosphonate(2-) Chemical compound CP([O-])([O-])=O YACKEPLHDIMKIO-UHFFFAOYSA-L 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- 210000004165 myocardium Anatomy 0.000 claims 1
- 230000007170 pathology Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000008363 phosphate buffer Substances 0.000 claims 1
- PTMHPRAIXMAOOB-UHFFFAOYSA-N phosphoramidic acid Chemical compound NP(O)(O)=O PTMHPRAIXMAOOB-UHFFFAOYSA-N 0.000 claims 1
- 150000003013 phosphoric acid derivatives Chemical class 0.000 claims 1
- 229920001223 polyethylene glycol Polymers 0.000 claims 1
- 210000003019 respiratory muscle Anatomy 0.000 claims 1
- 239000000758 substrate Substances 0.000 claims 1
- 238000002560 therapeutic procedure Methods 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
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
- A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
- A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
-
- 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
-
- 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
- C12N15/1138—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 receptors or cell surface proteins
-
- 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
-
- 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
- C12N2310/32—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
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/351—Conjugate
-
- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3535—Nitrogen
-
- 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
- C12N2320/33—Alteration of splicing
Claims (14)
1. Antisens polinukleotid za upotrebu u terapiji induciranjem preskakanja egzona RNK gama sarkoglikana u stanici (poželjno humanoj mišićnoj stanici, i posebno gdje je humana mišićna stanica kod pacijenta koji ima mišićnu distrofiju),
pri čemu antisens polinukleotid specifično hibridizira sa ciljnom regijom egzona RNK gama sarkoglikana, gde je egzon izabran iz grupe koja se sastoji iz egzona 4 (SEQ ID NO: 1), egzona 5 (SEQ ID NO: 2), egzona 6 (SEQ ID NO: 3), egzona 7 (SEQ ID NO: 4) i njihove kombinacije.
2. Antisens polinukleotid za upotrebu prema zahtjevu 1, pri čemu polinukleotid ne može formirati supstrat za RNazu H.
3. Antisens polinukleotid za upotrebu prema zahtjevu 1 ili zahtjevu 2, pri čemu se antisens polinukleotid sastoji iz modificirane okosnice polinukleotida koja se sastoji iz modificirane grupe supstituirane šećerom bar jednog od polinukleotida.
4. Antisens polinukleotid za upotrebu prema zahtjevu 3, gdje je modificirana grupa morfolino.
5. Antisens polinukleotid za upotrebu prema zahtjevu 3 ili zahtjevu 4, pri čemu modificirana okosnica polinukleotida bar jednog od polinukleotida se sastoji iz bar jedne modificirane internukleotidne veze.
6. Antisens polinukleotid za upotrebu prema zahtjevu 5, pri čemu se modificirana internukleotidna veza sastoji iz modificiranog fosfata izabranog iz grupe koja se sastoji iz metil fosfonata, metil fosforotioata, fosforomorfolidata, fosforopiperazidata i fosforoamidata.
7. Antisens polinukleotid za upotrebu prema bilo kojem od zahtjeva 3-6, pri čemu je polinukleotid 2’-O-metil-oligoribonukleotid.
8. Antisens polinukleotid za upotrebu prema bilo kojem od zahtjeva 1-7, pri čemu je polinukleotid kemijski vezan sa jednim ili više konjugata koji pojačavaju aktivnost, staničnu distribuciju, ili stanično preuzimanje antisens polinukleotida.
9. Antisens polinukleotid za upotrebu prema zahtjevu 8, pri čemu je polinukleotid kemijski vezan za molekul polietilen glikola.
10. Antisens polinukleotid za upotrebu prema zahtjevu 8 ili zahtjevu 9 pri čemu je konjugat peptid koji pojačava stanično preuzimanje, i gdje je peptid izabran iz grupe koja se sastoji od nuklearnog lokalizacijskog signala (NLS), HIV-1 TAT proteina, peptida koji sadrži domen vezivanja za integrin, oligolizina, adenovirusnog fibrozniog proteina i peptida koji sadrži domen za endocitozu posredovanu receptorom (RME).
11. Antisens polinukleotid za upotrebu prema zahtjevu 1, gdje je stanica u pacijentu koji boluje od pojasne mišićne distrofije tipa 2C (LGMD2C).
12. Antisens polinukleotid za upotrebu prema zahtjevu 11 za upotrebu u ublažavanju, inhibiciji, ili poboljšanju kliničke slike pojasne mišićne distrofije tipa 2C (LGMD2C) kod pacijenta kome je to potrebno, pri čemu se pacijentu primjenjuje terapijski efektivna količina antisens polinukleotida, čime se ublažava LGMD2C, inhibira progresija distrofične patologije ili poboljšava mišićna funkcija.
13. Antisens polinukleotid za upotrebu prema zahtjevu 12, pri čemu poboljšanje obuhvaća poboljšanje funkcije srčanog mišića, poboljšanje snage respiratornih mišića ili poboljšanje motoričke stabilnosti.
14. Antisens polinukleotid za upotrebu prema bilo kojem od prethodnih zahtjeva, pri čemu je antisens polinukleotid u farmaceutskom sastavu koja se sastoji iz antisens polinukleotida i fiziološki kompatibilnog fosfatnog pufera.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261697766P | 2012-09-06 | 2012-09-06 | |
| EP18177365.6A EP3421602B1 (en) | 2012-09-06 | 2013-09-06 | Antisense polynucleotides to induce exon skipping and methods of treating dystrophies |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20210877T1 true HRP20210877T1 (hr) | 2021-07-23 |
Family
ID=50237661
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20181458TT HRP20181458T1 (hr) | 2012-09-06 | 2018-09-12 | Protusmisleni polinukleotidi za induciranje preskakanja egzona i postupci liječenja distrofija |
| HRP20210877TT HRP20210877T1 (hr) | 2012-09-06 | 2021-06-01 | Antisens polinukleotidi za induciranje preskakanja egzona i postupci liječenja distrofija |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20181458TT HRP20181458T1 (hr) | 2012-09-06 | 2018-09-12 | Protusmisleni polinukleotidi za induciranje preskakanja egzona i postupci liječenja distrofija |
Country Status (17)
| Country | Link |
|---|---|
| US (12) | US9499817B2 (hr) |
| EP (2) | EP3421602B1 (hr) |
| BR (1) | BR112015005091B1 (hr) |
| CA (1) | CA2884245C (hr) |
| CY (2) | CY1121037T1 (hr) |
| DK (2) | DK3421602T3 (hr) |
| ES (2) | ES2686727T3 (hr) |
| HK (1) | HK1212272A1 (hr) |
| HR (2) | HRP20181458T1 (hr) |
| HU (2) | HUE039676T2 (hr) |
| LT (1) | LT3421602T (hr) |
| PL (1) | PL3421602T3 (hr) |
| PT (2) | PT3421602T (hr) |
| RS (2) | RS61985B1 (hr) |
| SI (2) | SI2892617T1 (hr) |
| TN (1) | TN2015000079A1 (hr) |
| WO (1) | WO2014039916A1 (hr) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016037163A1 (en) | 2014-09-07 | 2016-03-10 | Selecta Biosciences, Inc. | Methods and compositions for attenuating gene therapy anti-viral transfer vector immune responses |
| CA2981960C (en) * | 2015-04-08 | 2023-09-19 | The University Of Chicago | Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping |
| MX2020000676A (es) | 2017-07-18 | 2021-05-14 | Csl Behring Gene Therapy Inc | Composiciones y metodos para tratar beta-hemoglobinopatias. |
| WO2019075360A1 (en) | 2017-10-13 | 2019-04-18 | Selecta Biosciences, Inc. | METHODS AND COMPOSITIONS FOR MITIGATING ANTI-VECTOR VIRAL TRANSFER IGM RESPONSES |
| CN113347990A (zh) | 2018-12-21 | 2021-09-03 | 西北大学 | 膜联蛋白在预防和治疗肌膜损伤中的用途 |
| CA3123045A1 (en) | 2018-12-23 | 2020-07-02 | Csl Behring L.L.C. | Donor t-cells with kill switch |
| EP3897745A1 (en) | 2018-12-23 | 2021-10-27 | CSL Behring LLC | Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome |
| BR112021023594A2 (pt) | 2019-05-28 | 2022-02-08 | Selecta Biosciences Inc | Métodos e composições para resposta imune de vetor de transferência antiviral atenuada |
| WO2021263070A1 (en) | 2020-06-26 | 2021-12-30 | Csl Behring Llc | Donor t-cells with kill switch |
| WO2023064367A1 (en) | 2021-10-12 | 2023-04-20 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector igm responses |
| WO2023172624A1 (en) | 2022-03-09 | 2023-09-14 | Selecta Biosciences, Inc. | Immunosuppressants in combination with anti-igm agents and related dosing |
Family Cites Families (55)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| 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 |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| JP2828642B2 (ja) | 1987-06-24 | 1998-11-25 | ハワード フローレイ インスティテュト オブ イクスペリメンタル フィジオロジー アンド メディシン | ヌクレオシド誘導体 |
| US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
| US5585362A (en) | 1989-08-22 | 1996-12-17 | The Regents Of The University Of Michigan | Adenovirus vectors for gene therapy |
| US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
| US5130302A (en) | 1989-12-20 | 1992-07-14 | Boron Bilogicals, Inc. | Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same |
| US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| 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 |
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| WO1991018088A1 (en) | 1990-05-23 | 1991-11-28 | The United States Of America, Represented By The Secretary, United States Department Of Commerce | Adeno-associated virus (aav)-based eucaryotic vectors |
| BR9106702A (pt) | 1990-07-27 | 1993-06-08 | Isis Pharmaceuticals Inc | Analogo de oligonucleotideos e processos para modular a producao de uma proteina por um organismo e para tratar um organismo |
| US5328688A (en) | 1990-09-10 | 1994-07-12 | Arch Development Corporation | Recombinant herpes simplex viruses vaccines and methods |
| 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 |
| US5849571A (en) | 1990-10-10 | 1998-12-15 | University Of Pittsburgh Of The Commonwealth System Of Higher Education | Latency active herpes virus promoters and their use |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| US7223833B1 (en) | 1991-05-24 | 2007-05-29 | Isis Pharmaceuticals, Inc. | Peptide nucleic acid conjugates |
| US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
| US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
| US5594121A (en) | 1991-11-07 | 1997-01-14 | Gilead Sciences, Inc. | Enhanced triple-helix and double-helix formation with oligomers containing modified purines |
| US5252479A (en) | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| 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 |
| JP3739785B2 (ja) | 1991-11-26 | 2006-01-25 | アイシス ファーマシューティカルズ，インコーポレイティド | 修飾されたピリミジンを含有するオリゴマーを使用する増強された三重らせんおよび二重らせんの成形 |
| US5879934A (en) | 1992-07-31 | 1999-03-09 | University Of Pittsburgh Of The Commonwealth System Of Higher Education | Herpes simplex virus strains for gene transfer |
| US20040005707A1 (en) * | 2002-07-02 | 2004-01-08 | Isis Pharmaceuticals Inc. | Antisense modulation of integrin beta 5 expression |
| US5661033A (en) | 1992-11-25 | 1997-08-26 | The Board Of Trustees Of The Leland Stanford Junior University | Gene transfer using herpes virus vectors as a tool for neuroprotection |
| US5631237A (en) | 1992-12-22 | 1997-05-20 | Dzau; Victor J. | Method for producing in vivo delivery of therapeutic agents via liposomes |
| DE4311651A1 (de) | 1993-04-08 | 1994-10-13 | Boehringer Ingelheim Int | Virus für den Transport von Fremd-DNA in höhere eukaryotische Zellen |
| US5834441A (en) | 1993-09-13 | 1998-11-10 | Rhone-Poulenc Rorer Pharmaceuticals Inc. | Adeno-associated viral (AAV) liposomes and methods related thereto |
| US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
| AU688428B2 (en) | 1993-11-09 | 1998-03-12 | Johns Hopkins University, The | Generation of high titers of recombinant AAV vectors |
| US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
| FR2716682B1 (fr) | 1994-01-28 | 1996-04-26 | Centre Nat Rech Scient | Procédé de préparation de virus adéno-associés (AAV) recombinants et utilisations. |
| US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
| 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 |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| US5792453A (en) | 1995-02-28 | 1998-08-11 | The Regents Of The University Of California | Gene transfer-mediated angiogenesis therapy |
| US5707618A (en) | 1995-03-24 | 1998-01-13 | Genzyme Corporation | Adenovirus vectors for gene therapy |
| FR2732357B1 (fr) | 1995-03-31 | 1997-04-30 | Rhone Poulenc Rorer Sa | Vecteurs viraux et utilisation pour le traitement des desordres hyperproliferatifs, notamment de la restenose |
| US5773289A (en) | 1995-06-06 | 1998-06-30 | University Of Pittsburgh | AAV directed targeted integration |
| US5622856A (en) | 1995-08-03 | 1997-04-22 | Avigen | High efficiency helper system for AAV vector production |
| US5912340A (en) | 1995-10-04 | 1999-06-15 | Epoch Pharmaceuticals, Inc. | Selective binding complementary oligonucleotides |
| US5830727A (en) | 1995-11-18 | 1998-11-03 | Human Gene Therapy Research Institute | Herpes simplex virus amplicon mini-vector gene transfer system |
| 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 |
| IL135000A0 (en) | 1997-09-12 | 2001-05-20 | Exiqon As | Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues |
| EP1072679A3 (en) | 1999-07-20 | 2002-07-31 | Agilent Technologies, Inc. (a Delaware corporation) | Method of producing nucleic acid molecules with reduced secondary structure |
| US7339051B2 (en) * | 2003-04-28 | 2008-03-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for the treatment of severe acute respiratory syndrome (SARS) |
| CA2566286A1 (en) * | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same |
| CA2981960C (en) * | 2015-04-08 | 2023-09-19 | The University Of Chicago | Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping |
-
2013
- 2013-09-06 BR BR112015005091-3A patent/BR112015005091B1/pt active IP Right Grant
- 2013-09-06 SI SI201331142T patent/SI2892617T1/sl unknown
- 2013-09-06 PT PT181773656T patent/PT3421602T/pt unknown
- 2013-09-06 LT LTEP18177365.6T patent/LT3421602T/lt unknown
- 2013-09-06 ES ES13835266.1T patent/ES2686727T3/es active Active
- 2013-09-06 WO PCT/US2013/058636 patent/WO2014039916A1/en active Application Filing
- 2013-09-06 HU HUE13835266A patent/HUE039676T2/hu unknown
- 2013-09-06 RS RS20210656A patent/RS61985B1/sr unknown
- 2013-09-06 DK DK18177365.6T patent/DK3421602T3/da active
- 2013-09-06 EP EP18177365.6A patent/EP3421602B1/en active Active
- 2013-09-06 US US14/426,348 patent/US9499817B2/en active Active
- 2013-09-06 DK DK13835266.1T patent/DK2892617T3/en active
- 2013-09-06 ES ES18177365T patent/ES2878650T3/es active Active
- 2013-09-06 HU HUE18177365A patent/HUE054260T2/hu unknown
- 2013-09-06 PL PL18177365T patent/PL3421602T3/pl unknown
- 2013-09-06 EP EP13835266.1A patent/EP2892617B1/en active Active
- 2013-09-06 SI SI201331887T patent/SI3421602T1/sl unknown
- 2013-09-06 RS RS20181053A patent/RS57789B1/sr unknown
- 2013-09-06 CA CA2884245A patent/CA2884245C/en active Active
- 2013-09-06 PT PT13835266T patent/PT2892617T/pt unknown
-
2015
- 2015-03-05 TN TNP2015000079A patent/TN2015000079A1/fr unknown
-
2016
- 2016-01-08 HK HK16100188.4A patent/HK1212272A1/xx unknown
- 2016-09-01 US US15/254,817 patent/US9777271B2/en active Active
-
2017
- 2017-09-07 US US15/698,406 patent/US20180037889A1/en not_active Abandoned
-
2018
- 2018-07-20 US US16/041,278 patent/US20180327743A1/en not_active Abandoned
- 2018-09-03 CY CY181100911T patent/CY1121037T1/el unknown
- 2018-09-12 HR HRP20181458TT patent/HRP20181458T1/hr unknown
-
2019
- 2019-03-07 US US16/295,341 patent/US20190194659A1/en not_active Abandoned
- 2019-10-17 US US16/656,177 patent/US20200040340A1/en not_active Abandoned
-
2020
- 2020-05-28 US US16/886,171 patent/US20200291403A1/en not_active Abandoned
-
2021
- 2021-01-07 US US17/143,847 patent/US20210147845A1/en not_active Abandoned
- 2021-06-01 CY CY20211100476T patent/CY1124197T1/el unknown
- 2021-06-01 HR HRP20210877TT patent/HRP20210877T1/hr unknown
- 2021-09-09 US US17/470,892 patent/US20210403910A1/en not_active Abandoned
-
2022
- 2022-04-22 US US17/727,376 patent/US20220251559A1/en not_active Abandoned
- 2022-12-02 US US18/061,345 patent/US20230183700A1/en not_active Abandoned
-
2023
- 2023-10-05 US US18/481,735 patent/US20240076670A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| HRP20210877T1 (hr) | Antisens polinukleotidi za induciranje preskakanja egzona i postupci liječenja distrofija | |
| AU2018201241A1 (en) | Multi-target modulation for treating fibrosis and inflammatory conditions | |
| Ge et al. | Targeted gene delivery by polyplex micelles with crowded PEG palisade and cRGD moiety for systemic treatment of pancreatic tumors | |
| Liu et al. | The use of antibody modified liposomes loaded with AMO-1 to deliver oligonucleotides to ischemic myocardium for arrhythmia therapy | |
| ES2876939T3 (es) | Péptidos penetradores de células que tienen un dominio hidrófobo central | |
| ES2928486T3 (es) | Conjugados de oligómeros con omisión de exón para la distrofia muscular | |
| Gao et al. | Arginine-chitosan/DNA self-assemble nanoparticles for gene delivery: In vitro characteristics and transfection efficiency | |
| JP2019504112A5 (hr) | ||
| US20190040384A1 (en) | Compositions and methods for modulation of smn2 splicing in a subject | |
| CN104781271B (zh) | 具有生物可逆的基团的多核苷酸 | |
| TWI495473B (zh) | 反義抗病毒化合物及治療流感病毒感染的方法 | |
| Cheng et al. | AS1411-induced growth inhibition of glioma cells by up-regulation of p53 and down-regulation of Bcl-2 and Akt1 via nucleolin | |
| JP6944942B2 (ja) | ＩＬ４Ｒα、ＴＲＰＡ１、またはＦ２ＲＬ１を標的とするＲＮＡ複合体を用いたアトピー性皮膚炎および喘息の治療 | |
| JP2014519333A5 (hr) | ||
| JP2010533171A5 (hr) | ||
| JP2015523854A5 (hr) | ||
| JP2018536689A5 (hr) | ||
| US20210389335A1 (en) | Compositions and methods for detection of smn protein in a subject and treatment of a subject | |
| HRP20210139T1 (hr) | Sastavi i postupci za korigiranje mišične distrofije donjih udova tipa 2c upotrebom preskakanja egzona | |
| JP2018530325A (ja) | アンジオテンシノーゲンの発現を調節するための化合物及び方法 | |
| JP2016520312A5 (hr) | ||
| WO2011130458A3 (en) | Rna aptamers against baff-r as cell-type specific delivery agents and methods for their use | |
| Zhou et al. | Improvement of intestinal absorption of forsythoside A and chlorogenic acid by different carboxymethyl chitosan and chito-oligosaccharide, application to Flos Lonicerae-Fructus Forsythiae herb couple preparations | |
| JP2015517466A5 (hr) | ||
| RU2019118041A (ru) | Вещества для нацеливания на различные выбранные органы или ткани |