JP2017522046A5 - - Google Patents
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- JP2017522046A5 JP2017522046A5 JP2017516650A JP2017516650A JP2017522046A5 JP 2017522046 A5 JP2017522046 A5 JP 2017522046A5 JP 2017516650 A JP2017516650 A JP 2017516650A JP 2017516650 A JP2017516650 A JP 2017516650A JP 2017522046 A5 JP2017522046 A5 JP 2017522046A5
- Authority
- JP
- Japan
- Prior art keywords
- group
- alkyl
- aryl
- polynucleotide construct
- optionally substituted
- 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.)
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- 239000002157 polynucleotide Substances 0.000 claims description 154
- 102000040430 polynucleotide Human genes 0.000 claims description 154
- 108091033319 polynucleotide Proteins 0.000 claims description 154
- 125000003118 aryl group Chemical group 0.000 claims description 135
- 125000000217 alkyl group Chemical group 0.000 claims description 109
- BWGNESOTFCXPMA-UHFFFAOYSA-N Dihydrogen disulfide Chemical compound SS BWGNESOTFCXPMA-UHFFFAOYSA-N 0.000 claims description 58
- 125000000623 heterocyclic group Chemical group 0.000 claims description 52
- 239000002777 nucleoside Substances 0.000 claims description 49
- 229910052739 hydrogen Inorganic materials 0.000 claims description 38
- 125000003835 nucleoside group Chemical group 0.000 claims description 36
- 239000001257 hydrogen Substances 0.000 claims description 33
- 125000004435 hydrogen atom Chemical class [H]* 0.000 claims description 33
- 239000000178 monomer Substances 0.000 claims description 33
- 125000001072 heteroaryl group Chemical group 0.000 claims description 32
- 229910052760 oxygen Inorganic materials 0.000 claims description 29
- 125000005842 heteroatom Chemical group 0.000 claims description 28
- 229910052717 sulfur Inorganic materials 0.000 claims description 27
- 125000000392 cycloalkenyl group Chemical group 0.000 claims description 23
- 125000006552 (C3-C8) cycloalkyl group Chemical group 0.000 claims description 21
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 21
- 230000008685 targeting Effects 0.000 claims description 19
- 229910052799 carbon Inorganic materials 0.000 claims description 18
- XYFCBTPGUUZFHI-UHFFFAOYSA-N Phosphine Chemical group P XYFCBTPGUUZFHI-UHFFFAOYSA-N 0.000 claims description 16
- 125000005843 halogen group Chemical group 0.000 claims description 16
- 150000001720 carbohydrates Chemical class 0.000 claims description 15
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 15
- 229920001184 polypeptide Polymers 0.000 claims description 14
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 14
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 14
- 125000000732 arylene group Chemical group 0.000 claims description 13
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 13
- 210000004027 cell Anatomy 0.000 claims description 12
- 125000005647 linker group Chemical group 0.000 claims description 12
- 125000003601 C2-C6 alkynyl group Chemical group 0.000 claims description 11
- 125000004104 aryloxy group Chemical group 0.000 claims description 11
- 125000001589 carboacyl group Chemical group 0.000 claims description 11
- 125000005724 cycloalkenylene group Chemical group 0.000 claims description 11
- 125000000000 cycloalkoxy group Chemical group 0.000 claims description 11
- 125000002993 cycloalkylene group Chemical group 0.000 claims description 11
- 125000005309 thioalkoxy group Chemical group 0.000 claims description 11
- 125000004738 (C1-C6) alkyl sulfinyl group Chemical group 0.000 claims description 10
- 125000000882 C2-C6 alkenyl group Chemical group 0.000 claims description 10
- 125000002102 aryl alkyloxo group Chemical group 0.000 claims description 10
- 125000004429 atom Chemical group 0.000 claims description 10
- 125000004093 cyano group Chemical group *C#N 0.000 claims description 10
- 125000001181 organosilyl group Chemical group [SiH3]* 0.000 claims description 10
- 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 8
- 102100041003 Glutamate carboxypeptidase 2 Human genes 0.000 claims description 8
- 101000892862 Homo sapiens Glutamate carboxypeptidase 2 Proteins 0.000 claims description 8
- 229910019142 PO4 Inorganic materials 0.000 claims description 8
- 125000004432 carbon atom Chemical group C* 0.000 claims description 8
- 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 8
- 125000004122 cyclic group Chemical group 0.000 claims description 8
- 239000010452 phosphate Substances 0.000 claims description 8
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims description 8
- 229920000233 poly(alkylene oxides) Polymers 0.000 claims description 8
- 125000006590 (C2-C6) alkenylene group Chemical group 0.000 claims description 7
- 210000001163 endosome Anatomy 0.000 claims description 7
- 230000002427 irreversible effect Effects 0.000 claims description 7
- -1 5 ′ cap Chemical compound 0.000 claims description 6
- 239000003814 drug Substances 0.000 claims description 6
- 230000007935 neutral effect Effects 0.000 claims description 6
- 229910052757 nitrogen Inorganic materials 0.000 claims description 6
- 229920000620 organic polymer Polymers 0.000 claims description 6
- 229920000642 polymer Polymers 0.000 claims description 6
- 125000001424 substituent group Chemical group 0.000 claims description 6
- 229940124597 therapeutic agent Drugs 0.000 claims description 6
- 125000006591 (C2-C6) alkynylene group Chemical group 0.000 claims description 5
- 229920003171 Poly (ethylene oxide) Polymers 0.000 claims description 5
- 125000001841 imino group Chemical group [H]N=* 0.000 claims description 5
- 150000003839 salts Chemical class 0.000 claims description 5
- 102000009027 Albumins Human genes 0.000 claims description 4
- 108010088751 Albumins Proteins 0.000 claims description 4
- FBPFZTCFMRRESA-JGWLITMVSA-N D-glucitol Chemical compound OC[C@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-JGWLITMVSA-N 0.000 claims description 4
- 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 4
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical compound CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-N 0.000 claims description 4
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 claims description 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical compound OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims description 4
- 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 4
- 125000003545 alkoxy group Chemical group 0.000 claims description 4
- 229960002685 biotin Drugs 0.000 claims description 4
- 235000020958 biotin Nutrition 0.000 claims description 4
- 239000011616 biotin Substances 0.000 claims description 4
- 235000012000 cholesterol Nutrition 0.000 claims description 4
- QONQRTHLHBTMGP-UHFFFAOYSA-N digitoxigenin Natural products CC12CCC(C3(CCC(O)CC3CC3)C)C3C11OC1CC2C1=CC(=O)OC1 QONQRTHLHBTMGP-UHFFFAOYSA-N 0.000 claims description 4
- 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 4
- 239000001177 diphosphate Substances 0.000 claims description 4
- XPPKVPWEQAFLFU-UHFFFAOYSA-J diphosphate(4-) Chemical compound [O-]P([O-])(=O)OP([O-])([O-])=O XPPKVPWEQAFLFU-UHFFFAOYSA-J 0.000 claims description 4
- 235000011180 diphosphates Nutrition 0.000 claims description 4
- 210000002472 endoplasmic reticulum Anatomy 0.000 claims description 4
- 229940014144 folate Drugs 0.000 claims description 4
- OVBPIULPVIDEAO-LBPRGKRZSA-N folic acid Chemical compound C=1N=C2NC(N)=NC(=O)C2=NC=1CNC1=CC=C(C(=O)N[C@@H](CCC(O)=O)C(O)=O)C=C1 OVBPIULPVIDEAO-LBPRGKRZSA-N 0.000 claims description 4
- 235000019152 folic acid Nutrition 0.000 claims description 4
- 239000011724 folic acid Substances 0.000 claims description 4
- 239000003446 ligand Substances 0.000 claims description 4
- 125000000311 mannosyl group Chemical group C1([C@@H](O)[C@@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims description 4
- 239000001226 triphosphate Substances 0.000 claims description 4
- 235000011178 triphosphate Nutrition 0.000 claims description 4
- UNXRWKVEANCORM-UHFFFAOYSA-N triphosphoric acid Chemical compound OP(O)(=O)OP(O)(=O)OP(O)(O)=O UNXRWKVEANCORM-UHFFFAOYSA-N 0.000 claims description 4
- WYURNTSHIVDZCO-UHFFFAOYSA-N Tetrahydrofuran Chemical compound C1CCOC1 WYURNTSHIVDZCO-UHFFFAOYSA-N 0.000 claims description 3
- 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 3
- 238000003776 cleavage reaction Methods 0.000 claims description 3
- 229920000359 diblock copolymer Polymers 0.000 claims description 3
- 125000002228 disulfide group Chemical group 0.000 claims description 3
- 125000004433 nitrogen atom Chemical group N* 0.000 claims description 3
- 229920003224 poly(trimethylene oxide) Polymers 0.000 claims description 3
- 229920001748 polybutylene Polymers 0.000 claims description 3
- 229920001451 polypropylene glycol Polymers 0.000 claims description 3
- 230000007017 scission Effects 0.000 claims description 3
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 3
- 229920000428 triblock copolymer Polymers 0.000 claims description 3
- 125000002947 alkylene group Chemical group 0.000 claims description 2
- 150000001540 azides Chemical class 0.000 claims description 2
- 150000004712 monophosphates Chemical class 0.000 claims description 2
- 235000021317 phosphate Nutrition 0.000 claims description 2
- 235000014633 carbohydrates Nutrition 0.000 claims 7
- 125000003396 thiol group Chemical class [H]S* 0.000 claims 5
- 230000002441 reversible effect Effects 0.000 claims 4
- 125000000753 cycloalkyl group Chemical group 0.000 claims 3
- 238000009396 hybridization Methods 0.000 claims 2
- 125000004644 alkyl sulfinyl group Chemical group 0.000 claims 1
- 239000003054 catalyst Substances 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 125000005549 heteroarylene group Chemical group 0.000 description 12
- 125000003161 (C1-C6) alkylene group Chemical group 0.000 description 11
- 150000003573 thiols Chemical class 0.000 description 6
- 101000574648 Homo sapiens Retinoid-inducible serine carboxypeptidase Proteins 0.000 description 4
- 102100025483 Retinoid-inducible serine carboxypeptidase Human genes 0.000 description 4
- 125000005529 alkyleneoxy group Chemical group 0.000 description 4
- 238000000034 method Methods 0.000 description 3
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 description 2
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 description 2
- FBPFZTCFMRRESA-FSIIMWSLSA-N D-Glucitol Natural products OC[C@H](O)[C@H](O)[C@@H](O)[C@H](O)CO FBPFZTCFMRRESA-FSIIMWSLSA-N 0.000 description 2
- 238000012986 modification Methods 0.000 description 2
- 230000004048 modification Effects 0.000 description 2
- 235000010356 sorbitol Nutrition 0.000 description 2
- 229960002920 sorbitol Drugs 0.000 description 2
- 125000003342 alkenyl group Chemical group 0.000 description 1
- 125000000304 alkynyl group Chemical group 0.000 description 1
- 230000001404 mediated effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462009123P | 2014-06-06 | 2014-06-06 | |
| US62/009,123 | 2014-06-06 | ||
| PCT/US2015/034749 WO2015188197A2 (en) | 2014-06-06 | 2015-06-08 | Polynucleotide constructs having bioreversible and non-bioreversible groups |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017522046A JP2017522046A (ja) | 2017-08-10 |
| JP2017522046A5 true JP2017522046A5 (https=) | 2018-07-19 |
Family
ID=54767614
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017516650A Pending JP2017522046A (ja) | 2014-06-06 | 2015-06-08 | 生物可逆的および生物不可逆的基を有するポリヌクレオチド構築物 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US20170114341A1 (https=) |
| EP (1) | EP3152308A4 (https=) |
| JP (1) | JP2017522046A (https=) |
| CN (1) | CN107109405A (https=) |
| AU (1) | AU2015269053A1 (https=) |
| CA (1) | CA2950960A1 (https=) |
| WO (1) | WO2015188197A2 (https=) |
Families Citing this family (79)
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| TW201620526A (zh) | 2014-06-17 | 2016-06-16 | 愛羅海德研究公司 | 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法 |
| US20180133327A1 (en) * | 2015-03-16 | 2018-05-17 | Amal Therapeutics Sa | Cell Penetrating Peptides and Complexes Comprising the Same |
| KR102713216B1 (ko) | 2015-06-25 | 2024-10-02 | 로스웰 엠이 아이엔씨. | 바이오분자 센서들 및 방법들 |
| AU2016365828A1 (en) * | 2015-12-08 | 2018-07-05 | Solstice Biologics, Ltd. | Polynucleotide constructs having an auxiliary moiety non-bioreversibly linked to an internucleoside phosphate or phosphorothioate |
| CN113985017A (zh) | 2016-01-14 | 2022-01-28 | 罗斯韦尔生物技术股份有限公司 | 分子传感器及相关方法 |
| CN109071212A (zh) | 2016-01-28 | 2018-12-21 | 罗斯韦尔生物技术股份有限公司 | 使用大规模分子电子传感器阵列测量分析物的方法和装置 |
| KR102763291B1 (ko) | 2016-01-28 | 2025-02-04 | 로스웰 엠이 아이엔씨. | 대량 병렬 ｄｎａ 시퀀싱 장치 |
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| AU2007249349B2 (en) * | 2006-05-11 | 2012-03-08 | Isis Pharmaceuticals, Inc. | 5'-Modified bicyclic nucleic acid analogs |
| CA2746527A1 (en) * | 2008-09-22 | 2010-03-25 | Rxi Pharmaceuticals Corporation | Rna interference in skin indications |
| US20120142763A1 (en) * | 2009-06-01 | 2012-06-07 | The Regents Of The University Of California | Nucleic acid delivery compositions and methods of use thereof |
| US9950001B2 (en) * | 2012-08-20 | 2018-04-24 | The Regents Of The University Of California | Polynucleotides having bioreversible groups |
| AU2014346658A1 (en) * | 2013-11-06 | 2016-06-02 | Solstice Biologics, Ltd. | Polynucleotide constructs having disulfide groups |
-
2015
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- 2015-06-08 EP EP15803887.7A patent/EP3152308A4/en not_active Withdrawn
- 2015-06-08 AU AU2015269053A patent/AU2015269053A1/en not_active Abandoned
- 2015-06-08 CA CA2950960A patent/CA2950960A1/en not_active Abandoned
- 2015-06-08 US US15/315,608 patent/US20170114341A1/en not_active Abandoned
- 2015-06-08 CN CN201580041889.0A patent/CN107109405A/zh active Pending
- 2015-06-08 WO PCT/US2015/034749 patent/WO2015188197A2/en not_active Ceased
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2020
- 2020-02-03 US US16/780,204 patent/US20200392498A1/en not_active Abandoned
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