JP2015529073A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
- Authority
- 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
Links
- 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 (enExample) | 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 (enExample) |
| EP (1) | EP2885313A4 (enExample) |
| JP (1) | JP2015529073A (enExample) |
| CN (1) | CN104781271B (enExample) |
| AU (1) | AU2013306006B2 (enExample) |
| CA (1) | CA2880869A1 (enExample) |
| HK (1) | HK1208682A1 (enExample) |
| IN (1) | IN2015DN01765A (enExample) |
| WO (1) | WO2014031575A1 (enExample) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2907184C (en) | 2013-03-15 | 2022-12-06 | Sutter West Bay Hospitals | Falz for use as a target for therapies to treat cancer |
| EP3004131B1 (en) | 2013-06-05 | 2020-07-22 | AM Chemicals Llc | Phosphoramidite building blocks for sugar-conjugated oligonucleotides |
| JP2016537027A (ja) * | 2013-11-06 | 2016-12-01 | ソルスティス バイオロジクス，リミティッド | ジスルフィド基を有するポリヌクレオチド構築物 |
| EP3943500A1 (en) | 2014-04-30 | 2022-01-26 | Agilent Technologies, Inc. | Phosphorous protecting groups and methods of preparation and use thereof |
| EP3152308A4 (en) * | 2014-06-06 | 2017-12-27 | Solstice Biologics, Ltd. | Polynucleotide constructs having bioreversible and non-bioreversible groups |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| EP3201339A4 (en) | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| WO2016100635A1 (en) * | 2014-12-18 | 2016-06-23 | The Regents Of The University Of California | Detection of nucleic acid polymerase conformational changes using a nanotube |
| AU2016334804B2 (en) | 2015-10-09 | 2022-03-31 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| 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 |
| ES2882500T3 (es) | 2015-12-14 | 2021-12-02 | Cold Spring Harbor Laboratory | Oligómeros antisentido para el tratamiento del síndrome de Dravet |
| US10617759B2 (en) | 2016-03-01 | 2020-04-14 | Trustees Of Boston University | Light-stimulated release of cargo from oligonucleotides |
| KR102775461B1 (ko) | 2016-04-01 | 2025-02-28 | 어비디티 바이오사이언시스 인크. | 핵산-폴리펩타이드 조성물 및 이의 용도 |
| WO2018035380A1 (en) | 2016-08-17 | 2018-02-22 | Solstice Biologics, Ltd. | Polynucleotide constructs |
| WO2019006455A1 (en) | 2017-06-30 | 2019-01-03 | Solstice Biologics, Ltd. | AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF |
| SI3673080T1 (sl) | 2017-08-25 | 2024-03-29 | Stoke Therapeutics, Inc. | Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni |
| CN118638788A (zh) | 2017-12-06 | 2024-09-13 | 艾维迪提生物科学公司 | 治疗肌萎缩和强直性肌营养不良的组合物和方法 |
| WO2019213525A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| 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 |
| BR112022022889A2 (pt) | 2020-05-11 | 2023-04-04 | Stoke Therapeutics Inc | Oligômeros antissentido de opa1 para tratamento de condições e doenças |
| 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 |
| MX2024003266A (es) | 2021-09-16 | 2024-04-03 | Avidity Biosciences Inc | Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral. |
| 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 |
| 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 |
| 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 |
| US4415732A (en) | 1981-03-27 | 1983-11-15 | University Patents, Inc. | Phosphoramidite compounds and processes |
| US4973679A (en) | 1981-03-27 | 1990-11-27 | University Patents, Inc. | Process for oligonucleo tide synthesis using phosphormidite intermediates |
| 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 |
| DE3329892A1 (de) | 1983-08-18 | 1985-03-07 | Köster, Hubert, Prof. Dr., 2000 Hamburg | Verfahren zur herstellung von oligonucleotiden |
| USRE34069E (en) | 1983-08-18 | 1992-09-15 | Biosyntech Gmbh | Process for the preparation of oligonucleotides |
| US4672110A (en) | 1983-11-29 | 1987-06-09 | Northwestern University | Method of deprotecting polynucleotide trimethylethyl phosphotriesters |
| 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 |
| US5405938A (en) | 1989-12-20 | 1995-04-11 | 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 |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | 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 |
| 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 |
| 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 |
| 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 |
| EP0314752B1 (en) | 1987-05-19 | 1993-03-17 | Spirac Engineering Ab | A transportation device |
| 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 |
| WO1989009221A1 (en) | 1988-03-25 | 1989-10-05 | University Of Virginia Alumni Patents Foundation | Oligonucleotide n-alkylphosphoramidates |
| 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 |
| US5264564A (en) | 1989-10-24 | 1993-11-23 | Gilead Sciences | Oligonucleotide analogs with novel linkages |
| US5264562A (en) | 1989-10-24 | 1993-11-23 | Gilead Sciences, Inc. | 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 |
| US5804604A (en) | 1989-12-21 | 1998-09-08 | Biogen, Inc. | Tat-derived transport polypeptides and fusion proteins |
| US5674980A (en) | 1989-12-21 | 1997-10-07 | Biogen Inc | Fusion protein comprising tat-derived transport moiety |
| US6316003B1 (en) | 1989-12-21 | 2001-11-13 | Whitehead Institute For Biomedical Research | 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 |
| JPH05505941A (ja) | 1990-03-29 | 1993-09-02 | ギリード サイエンシズ インコーポレーテッド | オリゴヌクレオチド―輸送剤ジスルフィド結合体 |
| 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 |
| US5618704A (en) | 1990-07-27 | 1997-04-08 | Isis Pharmacueticals, Inc. | Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling |
| US5602240A (en) | 1990-07-27 | 1997-02-11 | Ciba Geigy Ag. | Backbone modified oligonucleotide analogs |
| US5610289A (en) | 1990-07-27 | 1997-03-11 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogues |
| US5677437A (en) | 1990-07-27 | 1997-10-14 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
| US5608046A (en) | 1990-07-27 | 1997-03-04 | Isis Pharmaceuticals, Inc. | Conjugated 4'-desmethyl nucleoside analog compounds |
| 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 |
| US5623070A (en) | 1990-07-27 | 1997-04-22 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
| JPH06502300A (ja) | 1990-08-03 | 1994-03-17 | サノフィ | 遺伝子発現の抑制のための化合物及び方法 |
| 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 |
| EP0549686A4 (en) | 1990-09-20 | 1995-01-18 | Gilead Sciences Inc | Modified internucleoside linkages |
| 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 (enExample) | 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 |
| CA2159629A1 (en) | 1993-03-31 | 1994-10-13 | Sanofi | 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 |
| WO1999054459A2 (en) * | 1998-04-20 | 1999-10-28 | 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 |
| US6903077B1 (en) | 1999-01-04 | 2005-06-07 | University Of Vermont And State Agricultural College | Methods and products for delivering nucleic acids |
| AU2405900A (en) | 1999-01-04 | 2000-07-24 | University Of Vermont And State Agricultural College, The | 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 |
| CA2392490A1 (en) | 1999-11-24 | 2001-05-31 | Mcs Micro Carrier Systems Gmbh | 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 |
| US20070026394A1 (en) | 2000-02-11 | 2007-02-01 | Lawrence Blatt | Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies |
| 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 |
| JP4387669B2 (ja) | 2000-10-13 | 2009-12-16 | ザイジェン エス．アー． | 新規なトランスポーターペプチド配列による生物学的エフェクターの細胞内送達 |
| US6858935B1 (en) | 2000-12-07 | 2005-02-22 | Cadence Design Systems, Inc. | Simulating euclidean wiring directions using manhattan and diagonal directional wires |
| MXPA03007358A (es) | 2001-02-16 | 2004-12-13 | Cellgate Inc | Transportadores que contienen porciones de arginina separadas. |
| 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 |
| CN1703229B (zh) | 2002-10-02 | 2011-01-05 | 英属哥伦比亚大学 | 治疗前列腺和其他癌的组合物和其制备药物的用途 |
| ATE465255T1 (de) | 2002-11-26 | 2010-05-15 | Univ Massachusetts | Verabreichung von sirnas |
| US20060251643A1 (en) | 2002-12-09 | 2006-11-09 | Toolgen, Inc. | Regulatory zinc finger proteins |
| US20040147027A1 (en) | 2003-01-28 | 2004-07-29 | Troy Carol M. | Complex for facilitating delivery of dsRNA into a cell and uses thereof |
| 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 |
| 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 |
| WO2004108883A2 (en) | 2003-06-10 | 2004-12-16 | Toolgen, Inc. | Transducible dna-binding proteins |
| CA2529752A1 (en) | 2003-06-20 | 2005-09-15 | The Regents Of The University Of California | Polypeptide transduction and fusogenic peptides |
| WO2005003315A2 (en) | 2003-07-01 | 2005-01-13 | Allele Biotechnology & Pharmaceuticals, Inc. | Compositions and methods for peptide-assisted transfection |
| WO2005056752A2 (en) | 2003-10-24 | 2005-06-23 | Gencia Corporation | Methods and compositions for delivering polynucleotides |
| CA2558408C (en) | 2004-03-03 | 2014-11-18 | Essentia Biosystems, Inc. | Compositions and methods for topical application and transdermal delivery of botulinum toxins |
| US7514530B2 (en) | 2004-04-26 | 2009-04-07 | Centre National De La Recherche Scientifique | Peptide carrier for delivering siRNA into mammalian cells |
| US20060035815A1 (en) | 2004-05-04 | 2006-02-16 | Nastech Pharmaceutical Company Inc. | Pharmaceutical compositions for delivery of ribonucleic acid to a cell |
| EP1750775A2 (en) | 2004-05-04 | 2007-02-14 | Nastech Pharmaceutical Company Inc. | Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in 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 |
| 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 |
| WO2006023491A2 (en) | 2004-08-16 | 2006-03-02 | The Cbr Institute For Biomedical Research, 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 |
| JP2009532017A (ja) | 2006-02-10 | 2009-09-10 | ザ レジェンツ オブ ザ ユニヴァースティ オブ カリフォルニア | ＰＴＤ／ＣＰＰＳへのｄｓＲＮＡ結合ドメイン融合体によるｓｉＲＮＡの導入可能な送達 |
| CA2659103C (en) | 2006-07-12 | 2019-05-21 | The Regents Of The University Of California | Transducible delivery of nucleic acids by reversible phosphotriester charge neutralization protecting groups |
| EP3067359A1 (en) | 2008-09-23 | 2016-09-14 | 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 |
| CN102459302A (zh) * | 2009-06-01 | 2012-05-16 | 加利福尼亚大学董事会 | 核酸递送组合物及其使用方法 |
| EP2451461A4 (en) * | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
-
2013
- 2013-08-20 EP EP13831273.1A patent/EP2885313A4/en not_active Withdrawn
- 2013-08-20 AU AU2013306006A patent/AU2013306006B2/en not_active Ceased
- 2013-08-20 CA CA2880869A patent/CA2880869A1/en not_active Abandoned
- 2013-08-20 JP JP2015528564A patent/JP2015529073A/ja active Pending
- 2013-08-20 IN IN1765DEN2015 patent/IN2015DN01765A/en unknown
- 2013-08-20 HK HK15109270.5A patent/HK1208682A1/xx unknown
- 2013-08-20 US US14/422,970 patent/US9950001B2/en active Active
- 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
-
2018
- 2018-04-16 US US15/954,153 patent/US20180303864A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CN117858948A (zh) | 新的治疗剂递送部分及其用途 | |
| US20200392498A1 (en) | Polynucleotide constructs having bioreversible and non-bioreversible groups | |
| EP3406730B1 (en) | Novel single chemical entities and methods for delivery of oligonucleotides | |
| US9950001B2 (en) | Polynucleotides having bioreversible groups | |
| US20160257961A1 (en) | Polynucleotide constructs having disulfide groups | |
| EP4058032A1 (en) | Compositions for delivery of antisense compounds | |
| JP2022502451A (ja) | 核酸送達のための脂質化カチオン性ペプチド化合物を含む脂質ナノ粒子製剤 | |
| CN107635550A (zh) | 改良的含二硫化物的炔烃交联剂 | |
| US20240417422A1 (en) | Arnatar compounds and methods for enhanced cellular uptake | |
| TW202434200A (zh) | 用於遞送治療劑至脂肪組織之脂質結合物 | |
| Pansuwan et al. | Hydrophilic and cell-penetrable pyrrolidinyl peptide nucleic acid via post-synthetic modification with hydrophilic side chains | |
| US10385090B2 (en) | Nucleotide derivative or salt thereof, nucleotide-derived 5′-phosphate ester or salt thereof, nucleotide-derived 3′-phosphoramidite compound or salt thereof, and polynucleotide | |
| US20060142232A1 (en) | Oligomeric compositions and methods | |
| US10844376B2 (en) | Structurally-enhanced miRNA inhibitor S-TuD | |
| KR20240082344A (ko) | 뒤센 근이영양증에서 엑손 44를 스키핑하기 위한 화합물 및 방법 | |
| WO2011143323A2 (en) | Programmable self-assembled nanostructures based on sidechain-modified pna for the multivalent display of ligands | |
| JP7013626B2 (ja) | 細胞膜透過ペプチド、構築物、及び、カーゴ分子を細胞内に輸送する方法 | |
| Liang et al. | Impurities from Hydroxyproline Derivatives in the Synthesis of Modified Oligonucleotides | |
| US20250171776A1 (en) | Non-canonical cell-penetrating peptides for antisense oligomer delivery | |
| AU2022299173B2 (en) | Novel therapeutic delivery moieties and uses thereof | |
| KR20260056195A (ko) | 올리고뉴클레오타이드의 세포내 표적화 | |
| US10919857B2 (en) | Oligo-guanidines based cellular transporter comprising heterocyclic rings with hydrophobic and/or lipophilic groups at N-terminal for effective delivery of nonpenetrable cargos in-vitro and in-vivo | |
| WO2025261376A1 (zh) | 肝外递送配体及其缀合物 | |
| CN121517358A (zh) | Cb1配体化合物及其缀合物 | |
| Alvira Torre | Modified oligonucleotides for triple helix studies and for the obtention of structures with biomedical and technological interest |