JP2015529073A5 - - Google Patents
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- JP2015529073A5 JP2015529073A5 JP2015528564A JP2015528564A JP2015529073A5 JP 2015529073 A5 JP2015529073 A5 JP 2015529073A5 JP 2015528564 A JP2015528564 A JP 2015528564A JP 2015528564 A JP2015528564 A JP 2015528564A JP 2015529073 A5 JP2015529073 A5 JP 2015529073A5
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- JP
- Japan
- Prior art keywords
- optionally substituted
- group
- peg
- ptd
- alkylene
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
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- 108091033319 polynucleotide Proteins 0.000 claims description 165
- 239000002157 polynucleotide Substances 0.000 claims description 165
- 102000040430 polynucleotide Human genes 0.000 claims description 165
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 118
- 125000003729 nucleotide group Chemical group 0.000 claims description 74
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 74
- 239000002773 nucleotide Substances 0.000 claims description 67
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 65
- 125000002947 alkylene group Chemical group 0.000 claims description 59
- 230000007935 neutral effect Effects 0.000 claims description 56
- 229920000620 organic polymer Polymers 0.000 claims description 55
- 229920001184 polypeptide Polymers 0.000 claims description 52
- 230000008685 targeting Effects 0.000 claims description 51
- 150000001720 carbohydrates Chemical class 0.000 claims description 50
- 235000014633 carbohydrates Nutrition 0.000 claims description 50
- 125000004450 alkenylene group Chemical group 0.000 claims description 49
- 125000004419 alkynylene group Chemical group 0.000 claims description 49
- 229920000642 polymer Polymers 0.000 claims description 47
- 239000003814 drug Substances 0.000 claims description 46
- 229940124597 therapeutic agent Drugs 0.000 claims description 46
- 229910052760 oxygen Inorganic materials 0.000 claims description 35
- 229910052717 sulfur Inorganic materials 0.000 claims description 35
- 125000000217 alkyl group Chemical group 0.000 claims description 29
- 229910052757 nitrogen Inorganic materials 0.000 claims description 29
- 125000003545 alkoxy group Chemical group 0.000 claims description 27
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 27
- 125000005842 heteroatom Chemical group 0.000 claims description 23
- 125000000732 arylene group Chemical group 0.000 claims description 20
- 125000000524 functional group Chemical group 0.000 claims description 19
- 150000003384 small molecules Chemical class 0.000 claims description 18
- 229920001223 polyethylene glycol Polymers 0.000 claims description 17
- 125000003118 aryl group Chemical group 0.000 claims description 16
- 150000002466 imines Chemical class 0.000 claims description 16
- 150000003573 thiols Chemical class 0.000 claims description 16
- 239000001177 diphosphate Substances 0.000 claims description 14
- XPPKVPWEQAFLFU-UHFFFAOYSA-J diphosphate(4-) Chemical compound [O-]P([O-])(=O)OP([O-])([O-])=O XPPKVPWEQAFLFU-UHFFFAOYSA-J 0.000 claims description 14
- 235000011180 diphosphates Nutrition 0.000 claims description 14
- 150000004712 monophosphates Chemical class 0.000 claims description 14
- 239000001226 triphosphate Substances 0.000 claims description 14
- 235000011178 triphosphate Nutrition 0.000 claims description 14
- UNXRWKVEANCORM-UHFFFAOYSA-N triphosphoric acid Chemical compound OP(O)(=O)OP(O)(=O)OP(O)(O)=O UNXRWKVEANCORM-UHFFFAOYSA-N 0.000 claims description 14
- 230000000295 complement effect Effects 0.000 claims description 13
- YBJHBAHKTGYVGT-ZKWXMUAHSA-N (+)-Biotin Chemical compound N1C(=O)N[C@@H]2[C@H](CCCCC(=O)O)SC[C@@H]21 YBJHBAHKTGYVGT-ZKWXMUAHSA-N 0.000 claims description 12
- YDHWWBZFRZWVHO-UHFFFAOYSA-H [oxido-[oxido(phosphonatooxy)phosphoryl]oxyphosphoryl] phosphate Chemical compound [O-]P([O-])(=O)OP([O-])(=O)OP([O-])(=O)OP([O-])([O-])=O YDHWWBZFRZWVHO-UHFFFAOYSA-H 0.000 claims description 12
- QTPILKSJIOLICA-UHFFFAOYSA-N bis[hydroxy(phosphonooxy)phosphoryl] hydrogen phosphate Chemical compound OP(O)(=O)OP(O)(=O)OP(O)(=O)OP(O)(=O)OP(O)(O)=O QTPILKSJIOLICA-UHFFFAOYSA-N 0.000 claims description 12
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims description 12
- 150000002081 enamines Chemical class 0.000 claims description 12
- 125000000623 heterocyclic group Chemical group 0.000 claims description 12
- 125000006681 (C2-C10) alkylene group Chemical group 0.000 claims description 11
- 125000001475 halogen functional group Chemical group 0.000 claims description 11
- 125000006239 protecting group Chemical group 0.000 claims description 11
- 108090000623 proteins and genes Proteins 0.000 claims description 11
- 102000004169 proteins and genes Human genes 0.000 claims description 11
- 125000000882 C2-C6 alkenyl group Chemical group 0.000 claims description 10
- 150000003839 salts Chemical class 0.000 claims description 9
- 125000000753 cycloalkyl group Chemical group 0.000 claims description 8
- 239000001257 hydrogen Substances 0.000 claims description 8
- 229910052739 hydrogen Inorganic materials 0.000 claims description 8
- 125000004435 hydrogen atom Chemical class [H]* 0.000 claims description 8
- 230000003834 intracellular effect Effects 0.000 claims description 8
- KZNICNPSHKQLFF-UHFFFAOYSA-N succinimide Chemical compound O=C1CCC(=O)N1 KZNICNPSHKQLFF-UHFFFAOYSA-N 0.000 claims description 8
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical compound OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims description 7
- 238000006243 chemical reaction Methods 0.000 claims description 7
- 230000026683 transduction Effects 0.000 claims description 7
- 238000010361 transduction Methods 0.000 claims description 7
- 125000006590 (C2-C6) alkenylene group Chemical group 0.000 claims description 6
- 125000006591 (C2-C6) alkynylene group Chemical group 0.000 claims description 6
- 125000003601 C2-C6 alkynyl group Chemical group 0.000 claims description 6
- SHIBSTMRCDJXLN-UHFFFAOYSA-N Digoxigenin Natural products C1CC(C2C(C3(C)CCC(O)CC3CC2)CC2O)(O)C2(C)C1C1=CC(=O)OC1 SHIBSTMRCDJXLN-UHFFFAOYSA-N 0.000 claims description 6
- IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 claims description 6
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 6
- 125000002252 acyl group Chemical group 0.000 claims description 6
- 229960002685 biotin Drugs 0.000 claims description 6
- 235000020958 biotin Nutrition 0.000 claims description 6
- 239000011616 biotin Substances 0.000 claims description 6
- 235000012000 cholesterol Nutrition 0.000 claims description 6
- 125000004093 cyano group Chemical group *C#N 0.000 claims description 6
- 125000000392 cycloalkenyl group Chemical group 0.000 claims description 6
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 6
- QONQRTHLHBTMGP-UHFFFAOYSA-N digitoxigenin Natural products CC12CCC(C3(CCC(O)CC3CC3)C)C3C11OC1CC2C1=CC(=O)OC1 QONQRTHLHBTMGP-UHFFFAOYSA-N 0.000 claims description 6
- SHIBSTMRCDJXLN-KCZCNTNESA-N digoxigenin Chemical compound C1([C@@H]2[C@@]3([C@@](CC2)(O)[C@H]2[C@@H]([C@@]4(C)CC[C@H](O)C[C@H]4CC2)C[C@H]3O)C)=CC(=O)OC1 SHIBSTMRCDJXLN-KCZCNTNESA-N 0.000 claims description 6
- 210000001163 endosome Anatomy 0.000 claims description 6
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 6
- 125000000449 nitro group Chemical group [O-][N+](*)=O 0.000 claims description 6
- SENVMHBTTTXYGJ-UHFFFAOYSA-N s-(1-hydroxyethyl) 2,2-dimethylpropanethioate Chemical compound CC(O)SC(=O)C(C)(C)C SENVMHBTTTXYGJ-UHFFFAOYSA-N 0.000 claims description 6
- 150000007970 thio esters Chemical class 0.000 claims description 6
- 125000002103 4,4'-dimethoxytriphenylmethyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)(C1=C([H])C([H])=C(OC([H])([H])[H])C([H])=C1[H])C1=C([H])C([H])=C(OC([H])([H])[H])C([H])=C1[H] 0.000 claims description 5
- 238000000034 method Methods 0.000 claims description 5
- 125000003161 (C1-C6) alkylene group Chemical group 0.000 claims description 4
- 239000004475 Arginine Substances 0.000 claims description 4
- 108090000790 Enzymes Proteins 0.000 claims description 4
- 102000004190 Enzymes Human genes 0.000 claims description 4
- 108700003968 Human immunodeficiency virus 1 tat peptide (49-57) Proteins 0.000 claims description 4
- 108010003723 Single-Domain Antibodies Proteins 0.000 claims description 4
- 230000029936 alkylation Effects 0.000 claims description 4
- 238000005804 alkylation reaction Methods 0.000 claims description 4
- 150000001413 amino acids Chemical class 0.000 claims description 4
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims description 4
- 238000006254 arylation reaction Methods 0.000 claims description 4
- 125000003178 carboxy group Chemical group [H]OC(*)=O 0.000 claims description 4
- 150000001733 carboxylic acid esters Chemical class 0.000 claims description 4
- 125000002091 cationic group Chemical group 0.000 claims description 4
- 238000006482 condensation reaction Methods 0.000 claims description 4
- 125000006575 electron-withdrawing group Chemical group 0.000 claims description 4
- 125000002485 formyl group Chemical class [H]C(*)=O 0.000 claims description 4
- 125000001072 heteroaryl group Chemical group 0.000 claims description 4
- 150000007857 hydrazones Chemical class 0.000 claims description 4
- 239000003446 ligand Substances 0.000 claims description 4
- 125000005647 linker group Chemical group 0.000 claims description 4
- 125000003588 lysine group Chemical group [H]N([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 claims description 4
- QJGQUHMNIGDVPM-UHFFFAOYSA-N nitrogen group Chemical group [N] QJGQUHMNIGDVPM-UHFFFAOYSA-N 0.000 claims description 4
- 230000000269 nucleophilic effect Effects 0.000 claims description 4
- 150000002923 oximes Chemical class 0.000 claims description 4
- 125000004430 oxygen atom Chemical group O* 0.000 claims description 4
- 238000007149 pericyclic reaction Methods 0.000 claims description 4
- 229960002317 succinimide Drugs 0.000 claims description 4
- 229940124530 sulfonamide Drugs 0.000 claims description 4
- 229930024421 Adenine Natural products 0.000 claims description 3
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 3
- DWRXFEITVBNRMK-UHFFFAOYSA-N Beta-D-1-Arabinofuranosylthymine Natural products O=C1NC(=O)C(C)=CN1C1C(O)C(O)C(CO)O1 DWRXFEITVBNRMK-UHFFFAOYSA-N 0.000 claims description 3
- 108020004635 Complementary DNA Proteins 0.000 claims description 3
- 229960000643 adenine Drugs 0.000 claims description 3
- 125000003172 aldehyde group Chemical group 0.000 claims description 3
- IQFYYKKMVGJFEH-UHFFFAOYSA-N beta-L-thymidine Natural products O=C1NC(=O)C(C)=CN1C1OC(CO)C(O)C1 IQFYYKKMVGJFEH-UHFFFAOYSA-N 0.000 claims description 3
- 229940104302 cytosine Drugs 0.000 claims description 3
- 230000014509 gene expression Effects 0.000 claims description 3
- 230000002441 reversible effect Effects 0.000 claims description 3
- 229940104230 thymidine Drugs 0.000 claims description 3
- 229940035893 uracil Drugs 0.000 claims description 3
- 108020004459 Small interfering RNA Proteins 0.000 claims description 2
- 239000012190 activator Substances 0.000 claims description 2
- 125000003342 alkenyl group Chemical group 0.000 claims description 2
- 150000001732 carboxylic acid derivatives Chemical class 0.000 claims description 2
- 230000021615 conjugation Effects 0.000 claims description 2
- 150000004820 halides Chemical class 0.000 claims description 2
- 239000004615 ingredient Substances 0.000 claims description 2
- 230000001404 mediated effect Effects 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 238000013518 transcription Methods 0.000 claims description 2
- 230000035897 transcription Effects 0.000 claims description 2
- 125000006552 (C3-C8) cycloalkyl group Chemical group 0.000 claims 4
- 125000004169 (C1-C6) alkyl group Chemical group 0.000 claims 1
- 125000006585 (C6-C10) arylene group Chemical group 0.000 claims 1
- 230000010189 intracellular transport Effects 0.000 claims 1
- 239000002777 nucleoside Substances 0.000 description 5
- 150000003833 nucleoside derivatives Chemical class 0.000 description 5
- 238000004519 manufacturing process Methods 0.000 description 4
- 230000002378 acidificating effect Effects 0.000 description 3
- 239000007787 solid Substances 0.000 description 3
- KAESVJOAVNADME-UHFFFAOYSA-N Pyrrole Chemical compound C=1C=CNC=1 KAESVJOAVNADME-UHFFFAOYSA-N 0.000 description 2
- 150000001412 amines Chemical class 0.000 description 2
- 239000000010 aprotic solvent Substances 0.000 description 2
- 230000015572 biosynthetic process Effects 0.000 description 2
- 210000004027 cell Anatomy 0.000 description 2
- 230000004048 modification Effects 0.000 description 2
- 238000012986 modification Methods 0.000 description 2
- 238000003786 synthesis reaction Methods 0.000 description 2
- 125000002373 5 membered heterocyclic group Chemical group 0.000 description 1
- XRFPIJCXAHXKDP-UHFFFAOYSA-N CC(C)(CNC(OCc(c([N+]([O-])=O)c1)cc(OC)c1OC)=O)C(SCCI)=O Chemical compound CC(C)(CNC(OCc(c([N+]([O-])=O)c1)cc(OC)c1OC)=O)C(SCCI)=O XRFPIJCXAHXKDP-UHFFFAOYSA-N 0.000 description 1
- 0 CCC1(CC)COC(c(cc2)ccc2C(SCCCC*)=O)OC1 Chemical compound CCC1(CC)COC(c(cc2)ccc2C(SCCCC*)=O)OC1 0.000 description 1
- BDMSHDKIMYBHFP-UHFFFAOYSA-N CCCCCSC(c1ccc(C2OCC(CC)(CC)CO2)cc1)=O Chemical compound CCCCCSC(c1ccc(C2OCC(CC)(CC)CO2)cc1)=O BDMSHDKIMYBHFP-UHFFFAOYSA-N 0.000 description 1
- SUXRTFJVJWTXGO-UHFFFAOYSA-N CCCCCSC(c1ccc(C=O)cc1)=O Chemical compound CCCCCSC(c1ccc(C=O)cc1)=O SUXRTFJVJWTXGO-UHFFFAOYSA-N 0.000 description 1
- NPTZRHBAHSQKJS-UHFFFAOYSA-N COC(NC(N)I)=O Chemical compound COC(NC(N)I)=O NPTZRHBAHSQKJS-UHFFFAOYSA-N 0.000 description 1
- 108091030071 RNAI Proteins 0.000 description 1
- 239000002253 acid Substances 0.000 description 1
- 230000000692 anti-sense effect Effects 0.000 description 1
- 239000003054 catalyst Substances 0.000 description 1
- 230000008878 coupling Effects 0.000 description 1
- 238000010168 coupling process Methods 0.000 description 1
- 238000005859 coupling reaction Methods 0.000 description 1
- 230000009368 gene silencing by RNA Effects 0.000 description 1
- 239000000203 mixture Substances 0.000 description 1
- 239000007800 oxidant agent Substances 0.000 description 1
- 230000001590 oxidative effect Effects 0.000 description 1
- 150000008300 phosphoramidites Chemical class 0.000 description 1
- 239000003586 protic polar solvent Substances 0.000 description 1
- 239000002904 solvent Substances 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261691175P | 2012-08-20 | 2012-08-20 | |
| US61/691,175 | 2012-08-20 | ||
| PCT/US2013/055675 WO2014031575A1 (en) | 2012-08-20 | 2013-08-20 | Polynucleotides having bioreversible groups |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2015529073A JP2015529073A (ja) | 2015-10-05 |
| JP2015529073A5 true JP2015529073A5 (https=) | 2016-10-06 |
Family
ID=50150337
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015528564A Pending JP2015529073A (ja) | 2012-08-20 | 2013-08-20 | 生物可逆性基を有するポリヌクレオチド |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US9950001B2 (https=) |
| EP (1) | EP2885313A4 (https=) |
| JP (1) | JP2015529073A (https=) |
| CN (1) | CN104781271B (https=) |
| AU (1) | AU2013306006B2 (https=) |
| CA (1) | CA2880869A1 (https=) |
| HK (1) | HK1208682A1 (https=) |
| IN (1) | IN2015DN01765A (https=) |
| WO (1) | WO2014031575A1 (https=) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105189786B (zh) | 2013-03-15 | 2018-04-03 | 萨特西湾医院 | 用作治疗癌症的疗法的靶标的falz |
| EP3004131B1 (en) | 2013-06-05 | 2020-07-22 | AM Chemicals Llc | Phosphoramidite building blocks for sugar-conjugated oligonucleotides |
| EP3066105A4 (en) * | 2013-11-06 | 2017-10-11 | Solstice Biologics, Ltd. | Polynucleotide constructs having disulfide groups |
| EP3943500A1 (en) | 2014-04-30 | 2022-01-26 | Agilent Technologies, Inc. | Phosphorous protecting groups and methods of preparation and use thereof |
| CA2950960A1 (en) * | 2014-06-06 | 2015-12-10 | Solstice Biologics, Ltd. | Polynucleotide constructs having bioreversible and non-bioreversible groups |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| AU2015364602B2 (en) * | 2014-12-18 | 2021-12-09 | The Regents Of The University Of California | Detection of nucleic acid polymerase conformational changes using a nanotube |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| WO2017100461A1 (en) * | 2015-12-08 | 2017-06-15 | Solstice Biologics, Ltd. | Polynucleotide constructs having an auxiliary moiety non-bioreversibly linked to an internucleoside phosphate or phosphorothioate |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| 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 |
| WO2017151748A1 (en) * | 2016-03-01 | 2017-09-08 | Trustees Of Boston University | Light-stimulated release of cargo from oligonucleotides |
| JP2019513371A (ja) | 2016-04-01 | 2019-05-30 | アビディティー バイオサイエンシーズ エルエルシー | 核酸ポリペプチド組成物とその使用 |
| CA3033867A1 (en) * | 2016-08-17 | 2018-02-22 | Solstice Biologics, Ltd. | Polynucleotide constructs |
| US11597744B2 (en) | 2017-06-30 | 2023-03-07 | Sirius Therapeutics, Inc. | Chiral phosphoramidite auxiliaries and methods of their use |
| KR102318434B1 (ko) | 2017-08-25 | 2021-11-01 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| AU2018378812B2 (en) | 2017-12-06 | 2025-05-22 | Avidity Biosciences, Inc. | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
| CA3099280A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| AU2020227825B2 (en) | 2019-02-27 | 2026-03-26 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| US11510939B1 (en) | 2019-04-19 | 2022-11-29 | Apellis Pharmaceuticals, Inc. | RNAs for complement inhibition |
| WO2021188390A1 (en) | 2020-03-19 | 2021-09-23 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
| CN115867657A (zh) | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
| CN116568334A (zh) * | 2020-10-12 | 2023-08-08 | 加利福尼亚大学董事会 | 用于将大分子递送至细胞中的内体逃逸结构域 |
| CN118265544A (zh) | 2021-09-16 | 2024-06-28 | 艾维迪提生物科学公司 | 治疗面肩肱型肌营养不良的组合物和方法 |
| WO2024097310A2 (en) * | 2022-11-01 | 2024-05-10 | Impilo Therapeutics, Inc. | Targeted non-charged-nucleic acid (ncna) delivery and related tumor penetrating nanocomplexes |
Family Cites Families (138)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| US3673612A (en) | 1970-08-28 | 1972-07-04 | Massachusetts Inst Technology | Non-thrombogenic materials and methods for their preparation |
| US5132418A (en) | 1980-02-29 | 1992-07-21 | University Patents, Inc. | Process for preparing polynucleotides |
| US4500707A (en) | 1980-02-29 | 1985-02-19 | University Patents, Inc. | Nucleosides useful in the preparation of polynucleotides |
| US4458066A (en) | 1980-02-29 | 1984-07-03 | 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 |
| US4668777A (en) | 1981-03-27 | 1987-05-26 | University Patents, Inc. | Phosphoramidite nucleoside compounds |
| 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 |
| US4517338A (en) | 1983-06-20 | 1985-05-14 | Chiron Corporation | Multiple reactor system and method for polynucleotide synthesis |
| USRE34069E (en) | 1983-08-18 | 1992-09-15 | Biosyntech Gmbh | Process for the preparation of oligonucleotides |
| DE3329892A1 (de) | 1983-08-18 | 1985-03-07 | Köster, Hubert, Prof. Dr., 2000 Hamburg | Verfahren zur herstellung von oligonucleotiden |
| US4672110A (en) | 1983-11-29 | 1987-06-09 | Northwestern University | Method of deprotecting polynucleotide trimethylethyl phosphotriesters |
| WO1988009301A1 (en) | 1987-05-19 | 1988-12-01 | Spirac Engineering Ab | A transportation device |
| 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 |
| US5235033A (en) | 1985-03-15 | 1993-08-10 | Anti-Gene Development Group | Alpha-morpholino ribonucleoside derivatives and polymers thereof |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5405938A (en) | 1989-12-20 | 1995-04-11 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US4751180A (en) | 1985-03-28 | 1988-06-14 | Chiron Corporation | Expression using fused genes providing for protein product |
| US4888278A (en) | 1985-10-22 | 1989-12-19 | University Of Massachusetts Medical Center | In-situ hybridization to detect nucleic acid sequences in morphologically intact cells |
| US4659774A (en) | 1985-11-01 | 1987-04-21 | American Hoechst Corporation | Support for solid-phase oligonucleotide synthesis |
| US4935233A (en) | 1985-12-02 | 1990-06-19 | G. D. Searle And Company | Covalently linked polypeptide cell modulators |
| US5153319A (en) | 1986-03-31 | 1992-10-06 | University Patents, Inc. | Process for preparing polynucleotides |
| US5319079A (en) | 1986-05-15 | 1994-06-07 | Beckman Instruments, Inc. | Process for terminal substituting of a polynucleotide |
| US4806463A (en) | 1986-05-23 | 1989-02-21 | Worcester Foundation For Experimental Biology | Inhibition of HTLV-III by exogenous oligonucleotides |
| 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 |
| 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 |
| US5188897A (en) | 1987-10-22 | 1993-02-23 | Temple University Of The Commonwealth System Of Higher Education | Encapsulated 2',5'-phosphorothioate oligoadenylates |
| 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 |
| US5218103A (en) | 1988-05-26 | 1993-06-08 | University Patents, Inc. | Nucleoside thiophosphoramidites |
| US5216141A (en) | 1988-06-06 | 1993-06-01 | Benner Steven A | Oligonucleotide analogs containing sulfur linkages |
| US4958013A (en) * | 1989-06-06 | 1990-09-18 | Northwestern University | Cholesteryl modified oligonucleotides |
| US5399676A (en) | 1989-10-23 | 1995-03-21 | Gilead Sciences | Oligonucleotides with inverted polarity |
| 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 |
| US6316003B1 (en) | 1989-12-21 | 2001-11-13 | Whitehead Institute For Biomedical Research | Tat-derived transport polypeptides |
| US5804604A (en) | 1989-12-21 | 1998-09-08 | Biogen, Inc. | Tat-derived transport polypeptides and fusion proteins |
| US5670617A (en) | 1989-12-21 | 1997-09-23 | Biogen Inc | Nucleic acid conjugates of tat-derived transport polypeptides |
| US5587361A (en) | 1991-10-15 | 1996-12-24 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| US5321131A (en) | 1990-03-08 | 1994-06-14 | Hybridon, Inc. | Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling |
| AU7759291A (en) | 1990-03-29 | 1991-10-21 | Gilead Sciences, Inc. | Oligonucleotide-transport agent disulfide conjugates |
| US5470967A (en) | 1990-04-10 | 1995-11-28 | The Dupont Merck Pharmaceutical Company | Oligonucleotide analogs with sulfamate linkages |
| US6610841B1 (en) | 1997-12-18 | 2003-08-26 | Gilead Sciences, Inc. | Nucleotide-based prodrugs |
| US5608046A (en) | 1990-07-27 | 1997-03-04 | Isis Pharmaceuticals, Inc. | Conjugated 4'-desmethyl nucleoside analog compounds |
| US5623070A (en) | 1990-07-27 | 1997-04-22 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
| US5602240A (en) | 1990-07-27 | 1997-02-11 | Ciba Geigy Ag. | Backbone modified oligonucleotide analogs |
| US5541307A (en) | 1990-07-27 | 1996-07-30 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs and solid phase synthesis thereof |
| US5489677A (en) | 1990-07-27 | 1996-02-06 | Isis Pharmaceuticals, Inc. | Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms |
| US5618704A (en) | 1990-07-27 | 1997-04-08 | Isis Pharmacueticals, Inc. | Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling |
| US5677437A (en) | 1990-07-27 | 1997-10-14 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
| US5610289A (en) | 1990-07-27 | 1997-03-11 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogues |
| MY107332A (en) | 1990-08-03 | 1995-11-30 | Sterling Drug Inc | Compounds and methods for inhibiting gene expression. |
| 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 |
| JPH06505704A (ja) | 1990-09-20 | 1994-06-30 | ギリアド サイエンシズ，インコーポレイテッド | 改変ヌクレオシド間結合 |
| US5733523A (en) | 1990-12-10 | 1998-03-31 | Akzo Nobel N.V. | Targeted delivery of a therapeutic entity using complementary oligonucleotides |
| US5571799A (en) | 1991-08-12 | 1996-11-05 | Basco, Ltd. | (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response |
| NZ244306A (en) | 1991-09-30 | 1995-07-26 | Boehringer Ingelheim Int | Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation |
| TW323284B (https=) | 1992-03-23 | 1997-12-21 | Novartis Ag | |
| US5633360A (en) | 1992-04-14 | 1997-05-27 | Gilead Sciences, Inc. | Oligonucleotide analogs capable of passive cell membrane permeation |
| FR2692265B1 (fr) * | 1992-05-25 | 1996-11-08 | Centre Nat Rech Scient | Composes biologiquement actifs de type phosphotriesters. |
| US5434257A (en) | 1992-06-01 | 1995-07-18 | Gilead Sciences, Inc. | Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages |
| US5489525A (en) | 1992-10-08 | 1996-02-06 | The United States Of America As Represented By The Department Of Health And Human Services | Monoclonal antibodies to prostate cells |
| 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 |
| HU9501974D0 (en) | 1993-03-31 | 1995-09-28 | Sterling Winthrop Inc | Oligonucleotides with amide linkages replacing phosphodiester linkages |
| US5955591A (en) | 1993-05-12 | 1999-09-21 | Imbach; Jean-Louis | Phosphotriester oligonucleotides, amidites and method of preparation |
| JPH0799976A (ja) * | 1993-09-30 | 1995-04-18 | Takeda Chem Ind Ltd | 修飾オリゴヌクレオチド |
| US5625050A (en) | 1994-03-31 | 1997-04-29 | Amgen Inc. | Modified oligonucleotides and intermediates useful in nucleic acid therapeutics |
| US5622867A (en) | 1994-10-19 | 1997-04-22 | Lifecell Corporation | Prolonged preservation of blood platelets |
| US6051429A (en) | 1995-06-07 | 2000-04-18 | Life Technologies, Inc. | Peptide-enhanced cationic lipid transfections |
| US6017700A (en) | 1995-08-04 | 2000-01-25 | Bayer Corporation | Cationic oligonucleotides, and related methods of synthesis and use |
| US6251433B1 (en) | 1996-08-13 | 2001-06-26 | Chiron Corporation | Polycationic polymers |
| WO1998007734A1 (en) * | 1996-08-21 | 1998-02-26 | Hybridon, Inc. | Oligonucleotide prodrugs |
| US6214366B1 (en) | 1999-06-01 | 2001-04-10 | The Regents Of The University Of California | Clearance and inhibition of conformationally altered proteins |
| US6720355B2 (en) | 1997-02-21 | 2004-04-13 | The Regents Of The University Of California | Sodium dodecyl sulfate compositions for inactivating prions |
| US5891641A (en) | 1997-02-21 | 1999-04-06 | The Regents Of The University Of California | Assay for disease related conformation of a protein |
| US6423334B1 (en) | 1997-10-01 | 2002-07-23 | Elan Corporation, Plc | Composition and method for enhancing transport across gastrointestinal tract cell layers |
| WO1999029721A1 (en) | 1997-12-10 | 1999-06-17 | Washington University | Anti-pathogen system and methods of use thereof |
| EP1071753A2 (en) * | 1998-04-20 | 2001-01-31 | Ribozyme Pharmaceuticals, Inc. | Nucleic acid molecules with novel chemical compositions capable of modulating gene expression |
| US6531590B1 (en) | 1998-04-24 | 2003-03-11 | Isis Pharmaceuticals, Inc. | Processes for the synthesis of oligonucleotide compounds |
| CA2328457A1 (en) | 1998-06-20 | 1999-12-29 | Washington University | Membrane-permeant peptide complexes for medical imaging, diagnostics, and pharmaceutical therapy |
| US6407077B1 (en) | 1998-11-05 | 2002-06-18 | Emory University | β-L nucleosides for the treatment of HIV infection |
| WO2000040723A2 (en) | 1999-01-04 | 2000-07-13 | University Of Vermont And State Agricultural College | Methods and products for delivering nucleic acids |
| US6903077B1 (en) | 1999-01-04 | 2005-06-07 | University Of Vermont And State Agricultural College | Methods and products for delivering nucleic acids |
| US6656730B1 (en) | 1999-06-15 | 2003-12-02 | Isis Pharmaceuticals, Inc. | Oligonucleotides conjugated to protein-binding drugs |
| US6309663B1 (en) | 1999-08-17 | 2001-10-30 | Lipocine Inc. | Triglyceride-free compositions and methods for enhanced absorption of hydrophilic therapeutic agents |
| US20030104622A1 (en) | 1999-09-01 | 2003-06-05 | Robbins Paul D. | Identification of peptides that facilitate uptake and cytoplasmic and/or nuclear transport of proteins, DNA and viruses |
| EP1235914A2 (en) | 1999-11-24 | 2002-09-04 | Joseph Rosenecker | Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells |
| US7491805B2 (en) | 2001-05-18 | 2009-02-17 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
| JP5610659B2 (ja) | 2000-07-21 | 2014-10-22 | ルバンス セラピュティックス インク．Ｒｅｖａｎｃｅ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 多成分生物学的輸送システム |
| US6841535B2 (en) | 2000-07-31 | 2005-01-11 | Active Motif | Peptide-mediated transfection agents and methods of use |
| US7034109B2 (en) | 2000-10-13 | 2006-04-25 | Christophe Bonny | Intracellular delivery of biological effectors |
| US6900540B1 (en) | 2000-12-07 | 2005-05-31 | Cadence Design Systems, Inc. | Simulating diagonal wiring directions using Manhattan directional wires |
| DE60233137D1 (de) | 2001-02-16 | 2009-09-10 | Univ R | Transporter mit beabstandeten arginin-teilchen |
| EP1386004A4 (en) | 2001-04-05 | 2005-02-16 | Ribozyme Pharm Inc | MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID |
| KR20030062789A (ko) | 2002-01-19 | 2003-07-28 | 포휴먼텍(주) | 생체분자 전달 펩타이드 ｓｉｍ2-ｂｔｍ 및 이것을포함하는 생명공학제품 |
| KR100468316B1 (ko) | 2002-01-29 | 2005-01-27 | 주식회사 웰진 | Ｄｎａ의 세포 또는 조직 내 전달 효율을 높이는 펩타이드 |
| WO2003097671A1 (en) | 2002-03-29 | 2003-11-27 | Creagene Inc. | Cytoplasmic transduction peptides and uses thereof |
| US6835810B2 (en) | 2002-05-13 | 2004-12-28 | Geneshuttle Biopharma, Inc. | Fusion protein for use as vector |
| AU2003281078A1 (en) | 2002-07-17 | 2004-02-02 | University Of Otago | Conjugate for double-stranded rna sequence releasing and methods thereof |
| ATE465743T1 (de) | 2002-10-02 | 2010-05-15 | Univ British Columbia | Oligonucleotide zur behandlung von prostatakrebs und anderen krebserkrankungen |
| EP1585756B1 (en) | 2002-11-26 | 2010-04-21 | University of Massachusetts | Delivery of sirnas |
| US20060251643A1 (en) | 2002-12-09 | 2006-11-09 | Toolgen, Inc. | Regulatory zinc finger proteins |
| US20060178297A1 (en) | 2003-01-28 | 2006-08-10 | Troy Carol M | Systems and methods for silencing expression of a gene in a cell and uses thereof |
| US20040147027A1 (en) | 2003-01-28 | 2004-07-29 | Troy Carol M. | Complex for facilitating delivery of dsRNA into a cell and uses thereof |
| US7166692B2 (en) | 2003-03-04 | 2007-01-23 | Canbrex Bio Science Walkersville, Inc. | Intracellular delivery of small molecules, proteins, and nucleic acids |
| US7329638B2 (en) | 2003-04-30 | 2008-02-12 | The Regents Of The University Of Michigan | Drug delivery compositions |
| US20060182736A1 (en) | 2003-06-10 | 2006-08-17 | Kim Jin-Soo | Transducible dna-binding proteins |
| WO2005084158A2 (en) | 2003-06-20 | 2005-09-15 | The Regents Of The University Of California | Polypeptide transduction and fusogenic peptides |
| US20050042603A1 (en) | 2003-07-01 | 2005-02-24 | Allele Biotechnology & Pharmaceuticals, Inc. | Compositions and methods for peptide-assisted transfection |
| CA2543257C (en) | 2003-10-24 | 2013-12-31 | Gencia Corporation | Methods and compositions for delivering polynucleotides |
| BRPI0508410B8 (pt) | 2004-03-03 | 2021-05-25 | Revance Therapeutics Inc | composições e métodos para a aplicação tópica e liberação transdérmica de toxinas botulínicas |
| US7514530B2 (en) | 2004-04-26 | 2009-04-07 | Centre National De La Recherche Scientifique | Peptide carrier for delivering siRNA into mammalian cells |
| US20060040882A1 (en) | 2004-05-04 | 2006-02-23 | Lishan Chen | Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells |
| JP2007536253A (ja) | 2004-05-04 | 2007-12-13 | ナステック・ファーマシューティカル・カンパニー・インコーポレーテッド | 細胞内への核酸の送達を増強し、細胞中の標的遺伝子の発現を修飾するための組成物及び方法 |
| US20060035815A1 (en) | 2004-05-04 | 2006-02-16 | Nastech Pharmaceutical Company Inc. | Pharmaceutical compositions for delivery of ribonucleic acid to a cell |
| US20060030003A1 (en) | 2004-05-12 | 2006-02-09 | Simon Michael R | Composition and method for introduction of RNA interference sequences into targeted cells and tissues |
| US20050288246A1 (en) | 2004-05-24 | 2005-12-29 | Iversen Patrick L | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
| FR2871548B1 (fr) | 2004-06-14 | 2006-08-11 | Renault Sas | Dispositif de retenue de deux elements l'un par rapport a l'autre |
| US7084248B2 (en) | 2004-07-14 | 2006-08-01 | Gene Tools, Llc | Peptide composition and method for delivering substances into the cytosol of cells |
| EP1789447B1 (en) | 2004-08-16 | 2012-04-25 | Immune Disease Institute, Inc. | Method of delivering rna interference and uses thereof |
| US20080027025A1 (en) | 2004-08-27 | 2008-01-31 | The Regents Of The University Of California | Protection From And Treatment Of Prion Protein Infection |
| WO2006029078A2 (en) | 2004-09-07 | 2006-03-16 | The Regents Of The University Of California | Targeting transducible molecules to specific cell types |
| US8273867B2 (en) | 2006-02-10 | 2012-09-25 | The Regents Of The University Of California | Transducible delivery of siRNA by dsRNA binding domain fusions to PTD/CPPS |
| AU2007272906B2 (en) | 2006-07-12 | 2013-01-31 | The Regents Of The University Of California | Transducible delivery of nucleic acids by reversible phosphotriester charge neutralization protecting groups |
| US8691971B2 (en) | 2008-09-23 | 2014-04-08 | Scott G. Petersen | Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating RNA interference |
| US9260493B2 (en) | 2009-05-07 | 2016-02-16 | The Regents Of The University Of California | Transducible delivery of nucleic acids using modified dsRNA binding domains |
| US20120142763A1 (en) * | 2009-06-01 | 2012-06-07 | The Regents Of The University Of California | Nucleic acid delivery compositions and methods of use thereof |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
-
2013
- 2013-08-20 CN CN201380054525.7A patent/CN104781271B/zh not_active Expired - Fee Related
- 2013-08-20 WO PCT/US2013/055675 patent/WO2014031575A1/en not_active Ceased
- 2013-08-20 CA CA2880869A patent/CA2880869A1/en not_active Abandoned
- 2013-08-20 EP EP13831273.1A patent/EP2885313A4/en not_active Withdrawn
- 2013-08-20 US US14/422,970 patent/US9950001B2/en active Active
- 2013-08-20 HK HK15109270.5A patent/HK1208682A1/xx unknown
- 2013-08-20 AU AU2013306006A patent/AU2013306006B2/en not_active Ceased
- 2013-08-20 JP JP2015528564A patent/JP2015529073A/ja active Pending
- 2013-08-20 IN IN1765DEN2015 patent/IN2015DN01765A/en unknown
-
2018
- 2018-04-16 US US15/954,153 patent/US20180303864A1/en not_active Abandoned
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