JPWO2021216556A5 - - Google Patents
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- JPWO2021216556A5 JPWO2021216556A5 JP2022563451A JP2022563451A JPWO2021216556A5 JP WO2021216556 A5 JPWO2021216556 A5 JP WO2021216556A5 JP 2022563451 A JP2022563451 A JP 2022563451A JP 2022563451 A JP2022563451 A JP 2022563451A JP WO2021216556 A5 JPWO2021216556 A5 JP WO2021216556A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense strand
- dsrna
- nucleotides
- sense strand
- optionally
- 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
- 230000000692 anti-sense effect Effects 0.000 claims description 205
- 125000003729 nucleotide group Chemical group 0.000 claims description 168
- 108091081021 Sense strand Proteins 0.000 claims description 152
- 239000002773 nucleotide Substances 0.000 claims description 151
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 125
- 150000001875 compounds Chemical class 0.000 claims description 121
- 150000007523 nucleic acids Chemical group 0.000 claims description 78
- 230000000295 complement effect Effects 0.000 claims description 77
- 102100037700 DNA mismatch repair protein Msh3 Human genes 0.000 claims description 69
- 101001027762 Homo sapiens DNA mismatch repair protein Msh3 Proteins 0.000 claims description 69
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 64
- 238000012986 modification Methods 0.000 claims description 56
- 230000004048 modification Effects 0.000 claims description 56
- 239000002336 ribonucleotide Substances 0.000 claims description 43
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 41
- 125000005647 linker group Chemical group 0.000 claims description 36
- 238000000034 method Methods 0.000 claims description 34
- 101150102506 MSH3 gene Proteins 0.000 claims description 32
- 150000004713 phosphodiesters Chemical class 0.000 claims description 24
- 108020004999 messenger RNA Proteins 0.000 claims description 21
- 239000008194 pharmaceutical composition Substances 0.000 claims description 20
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims description 16
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 claims description 16
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims description 16
- UKMSUNONTOPOIO-UHFFFAOYSA-N docosanoic acid Chemical compound CCCCCCCCCCCCCCCCCCCCCC(O)=O UKMSUNONTOPOIO-UHFFFAOYSA-N 0.000 claims description 16
- 238000002347 injection Methods 0.000 claims description 16
- 239000007924 injection Substances 0.000 claims description 16
- 108091034117 Oligonucleotide Proteins 0.000 claims description 13
- PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims description 13
- 108091027568 Single-stranded nucleotide Proteins 0.000 claims description 12
- 229940088594 vitamin Drugs 0.000 claims description 12
- 229930003231 vitamin Natural products 0.000 claims description 12
- 239000011782 vitamin Substances 0.000 claims description 12
- 235000013343 vitamin Nutrition 0.000 claims description 12
- 108020004414 DNA Proteins 0.000 claims description 11
- LYCAIKOWRPUZTN-UHFFFAOYSA-N Ethylene glycol Chemical group OCCO LYCAIKOWRPUZTN-UHFFFAOYSA-N 0.000 claims description 10
- 229910019142 PO4 Inorganic materials 0.000 claims description 10
- 125000000217 alkyl group Chemical group 0.000 claims description 10
- 150000001408 amides Chemical class 0.000 claims description 10
- 238000001990 intravenous administration Methods 0.000 claims description 10
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims description 10
- 239000010452 phosphate Substances 0.000 claims description 10
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 10
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims description 9
- -1 alkylene phosphonate Chemical compound 0.000 claims description 9
- 150000003722 vitamin derivatives Chemical class 0.000 claims description 9
- GVJHHUAWPYXKBD-UHFFFAOYSA-N (±)-α-Tocopherol Chemical compound OC1=C(C)C(C)=C2OC(CCCC(C)CCCC(C)CCCC(C)C)(C)CCC2=C1C GVJHHUAWPYXKBD-UHFFFAOYSA-N 0.000 claims description 8
- UHDGCWIWMRVCDJ-UHFFFAOYSA-N 1-beta-D-Xylofuranosyl-NH-Cytosine Natural products O=C1N=C(N)C=CN1C1C(O)C(O)C(CO)O1 UHDGCWIWMRVCDJ-UHFFFAOYSA-N 0.000 claims description 8
- 235000021357 Behenic acid Nutrition 0.000 claims description 8
- 239000002126 C01EB10 - Adenosine Substances 0.000 claims description 8
- MIKUYHXYGGJMLM-GIMIYPNGSA-N Crotonoside Natural products C1=NC2=C(N)NC(=O)N=C2N1[C@H]1O[C@@H](CO)[C@H](O)[C@@H]1O MIKUYHXYGGJMLM-GIMIYPNGSA-N 0.000 claims description 8
- UHDGCWIWMRVCDJ-PSQAKQOGSA-N Cytidine Natural products O=C1N=C(N)C=CN1[C@@H]1[C@@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-PSQAKQOGSA-N 0.000 claims description 8
- NYHBQMYGNKIUIF-UHFFFAOYSA-N D-guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(O)C1O NYHBQMYGNKIUIF-UHFFFAOYSA-N 0.000 claims description 8
- 208000023105 Huntington disease Diseases 0.000 claims description 8
- SMEROWZSTRWXGI-UHFFFAOYSA-N Lithocholsaeure Natural products C1CC2CC(O)CCC2(C)C2C1C1CCC(C(CCC(O)=O)C)C1(C)CC2 SMEROWZSTRWXGI-UHFFFAOYSA-N 0.000 claims description 8
- 229960005305 adenosine Drugs 0.000 claims description 8
- JAZBEHYOTPTENJ-JLNKQSITSA-N all-cis-5,8,11,14,17-icosapentaenoic acid Chemical compound CC\C=C/C\C=C/C\C=C/C\C=C/C\C=C/CCCC(O)=O JAZBEHYOTPTENJ-JLNKQSITSA-N 0.000 claims description 8
- 229940116226 behenic acid Drugs 0.000 claims description 8
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 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
- UHDGCWIWMRVCDJ-ZAKLUEHWSA-N cytidine Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-ZAKLUEHWSA-N 0.000 claims description 8
- 235000014113 dietary fatty acids Nutrition 0.000 claims description 8
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 8
- KFEVDPWXEVUUMW-UHFFFAOYSA-N docosanoic acid Natural products CCCCCCCCCCCCCCCCCCCCCC(=O)OCCC1=CC=C(O)C=C1 KFEVDPWXEVUUMW-UHFFFAOYSA-N 0.000 claims description 8
- 235000020673 eicosapentaenoic acid Nutrition 0.000 claims description 8
- 229960005135 eicosapentaenoic acid Drugs 0.000 claims description 8
- JAZBEHYOTPTENJ-UHFFFAOYSA-N eicosapentaenoic acid Natural products CCC=CCC=CCC=CCC=CCC=CCCCC(O)=O JAZBEHYOTPTENJ-UHFFFAOYSA-N 0.000 claims description 8
- 229930195729 fatty acid Natural products 0.000 claims description 8
- 239000000194 fatty acid Substances 0.000 claims description 8
- 150000004665 fatty acids Chemical class 0.000 claims description 8
- 229940029575 guanosine Drugs 0.000 claims description 8
- 230000002209 hydrophobic effect Effects 0.000 claims description 8
- 238000000185 intracerebroventricular administration Methods 0.000 claims description 8
- SMEROWZSTRWXGI-HVATVPOCSA-N lithocholic acid Chemical compound C([C@H]1CC2)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)CC1 SMEROWZSTRWXGI-HVATVPOCSA-N 0.000 claims description 8
- POULHZVOKOAJMA-UHFFFAOYSA-N methyl undecanoic acid Natural products CCCCCCCCCCCC(O)=O POULHZVOKOAJMA-UHFFFAOYSA-N 0.000 claims description 8
- 239000000203 mixture Substances 0.000 claims description 8
- 150000003431 steroids Chemical class 0.000 claims description 8
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims description 8
- 229940045145 uridine Drugs 0.000 claims description 8
- 230000002401 inhibitory effect Effects 0.000 claims description 7
- 125000006850 spacer group Chemical group 0.000 claims description 7
- 230000015556 catabolic process Effects 0.000 claims description 6
- 238000006731 degradation reaction Methods 0.000 claims description 6
- 150000002148 esters Chemical class 0.000 claims description 6
- 108020004707 nucleic acids Proteins 0.000 claims description 6
- 102000039446 nucleic acids Human genes 0.000 claims description 6
- UEZVMMHDMIWARA-UHFFFAOYSA-M phosphonate Chemical compound [O-]P(=O)=O UEZVMMHDMIWARA-UHFFFAOYSA-M 0.000 claims description 6
- 150000003852 triazoles Chemical class 0.000 claims description 6
- DVSZKTAMJJTWFG-SKCDLICFSA-N (2e,4e,6e,8e,10e,12e)-docosa-2,4,6,8,10,12-hexaenoic acid Chemical compound CCCCCCCCC\C=C\C=C\C=C\C=C\C=C\C=C\C(O)=O DVSZKTAMJJTWFG-SKCDLICFSA-N 0.000 claims description 4
- FPIPGXGPPPQFEQ-UHFFFAOYSA-N 13-cis retinol Natural products OCC=C(C)C=CC=C(C)C=CC1=C(C)CCCC1(C)C FPIPGXGPPPQFEQ-UHFFFAOYSA-N 0.000 claims description 4
- GZJLLYHBALOKEX-UHFFFAOYSA-N 6-Ketone, O18-Me-Ussuriedine Natural products CC=CCC=CCC=CCC=CCC=CCC=CCCCC(O)=O GZJLLYHBALOKEX-UHFFFAOYSA-N 0.000 claims description 4
- 208000035657 Abasia Diseases 0.000 claims description 4
- KXDHJXZQYSOELW-UHFFFAOYSA-M Carbamate Chemical compound NC([O-])=O KXDHJXZQYSOELW-UHFFFAOYSA-M 0.000 claims description 4
- IELOKBJPULMYRW-NJQVLOCASA-N D-alpha-Tocopheryl Acid Succinate Chemical compound OC(=O)CCC(=O)OC1=C(C)C(C)=C2O[C@@](CCC[C@H](C)CCC[C@H](C)CCCC(C)C)(C)CCC2=C1C IELOKBJPULMYRW-NJQVLOCASA-N 0.000 claims description 4
- 241000702421 Dependoparvovirus Species 0.000 claims description 4
- 150000001200 N-acyl ethanolamides Chemical class 0.000 claims description 4
- 108091028664 Ribonucleotide Proteins 0.000 claims description 4
- SHGAZHPCJJPHSC-NWVFGJFESA-N Tretinoin Chemical compound OC(=O)/C=C(\C)/C=C/C=C(C)C=CC1=C(C)CCCC1(C)C SHGAZHPCJJPHSC-NWVFGJFESA-N 0.000 claims description 4
- FPIPGXGPPPQFEQ-BOOMUCAASA-N Vitamin A Natural products OC/C=C(/C)\C=C\C=C(\C)/C=C/C1=C(C)CCCC1(C)C FPIPGXGPPPQFEQ-BOOMUCAASA-N 0.000 claims description 4
- 229930003427 Vitamin E Natural products 0.000 claims description 4
- 125000003545 alkoxy group Chemical group 0.000 claims description 4
- FPIPGXGPPPQFEQ-OVSJKPMPSA-N all-trans-retinol Chemical compound OC\C=C(/C)\C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C FPIPGXGPPPQFEQ-OVSJKPMPSA-N 0.000 claims description 4
- 210000004556 brain Anatomy 0.000 claims description 4
- 210000001638 cerebellum Anatomy 0.000 claims description 4
- 235000012000 cholesterol Nutrition 0.000 claims description 4
- OEYIOHPDSNJKLS-UHFFFAOYSA-N choline Chemical compound C[N+](C)(C)CCO OEYIOHPDSNJKLS-UHFFFAOYSA-N 0.000 claims description 4
- 229960001231 choline Drugs 0.000 claims description 4
- 229940099418 d- alpha-tocopherol succinate Drugs 0.000 claims description 4
- 201000010099 disease Diseases 0.000 claims description 4
- 208000035475 disorder Diseases 0.000 claims description 4
- 235000020669 docosahexaenoic acid Nutrition 0.000 claims description 4
- KAUVQQXNCKESLC-UHFFFAOYSA-N docosahexaenoic acid (DHA) Natural products COC(=O)C(C)NOCC1=CC=CC=C1 KAUVQQXNCKESLC-UHFFFAOYSA-N 0.000 claims description 4
- 239000002621 endocannabinoid Substances 0.000 claims description 4
- ZTWTYVWXUKTLCP-UHFFFAOYSA-L ethenyl-dioxido-oxo-$l^{5}-phosphane Chemical compound [O-]P([O-])(=O)C=C ZTWTYVWXUKTLCP-UHFFFAOYSA-L 0.000 claims description 4
- WIGCFUFOHFEKBI-UHFFFAOYSA-N gamma-tocopherol Natural products CC(C)CCCC(C)CCCC(C)CCCC1CCC2C(C)C(O)C(C)C(C)C2O1 WIGCFUFOHFEKBI-UHFFFAOYSA-N 0.000 claims description 4
- 150000002270 gangliosides Chemical class 0.000 claims description 4
- 125000005843 halogen group Chemical group 0.000 claims description 4
- 210000001320 hippocampus Anatomy 0.000 claims description 4
- 125000004356 hydroxy functional group Chemical group O* 0.000 claims description 4
- 210000003016 hypothalamus Anatomy 0.000 claims description 4
- 230000003993 interaction Effects 0.000 claims description 4
- 150000002632 lipids Chemical class 0.000 claims description 4
- 125000001921 locked nucleotide group Chemical group 0.000 claims description 4
- 239000002207 metabolite Substances 0.000 claims description 4
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 4
- 210000001577 neostriatum Anatomy 0.000 claims description 4
- 230000004770 neurodegeneration Effects 0.000 claims description 4
- 208000015122 neurodegenerative disease Diseases 0.000 claims description 4
- 239000002777 nucleoside Chemical class 0.000 claims description 4
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 4
- 125000006239 protecting group Chemical group 0.000 claims description 4
- 229930002330 retinoic acid Natural products 0.000 claims description 4
- 125000002652 ribonucleotide group Chemical group 0.000 claims description 4
- 150000003338 secosteroids Chemical class 0.000 claims description 4
- 210000000278 spinal cord Anatomy 0.000 claims description 4
- 238000007920 subcutaneous administration Methods 0.000 claims description 4
- 210000001103 thalamus Anatomy 0.000 claims description 4
- 229960001727 tretinoin Drugs 0.000 claims description 4
- 235000019155 vitamin A Nutrition 0.000 claims description 4
- 239000011719 vitamin A Substances 0.000 claims description 4
- 229940046009 vitamin E Drugs 0.000 claims description 4
- 235000019165 vitamin E Nutrition 0.000 claims description 4
- 239000011709 vitamin E Substances 0.000 claims description 4
- 229940045997 vitamin a Drugs 0.000 claims description 4
- 238000007385 chemical modification Methods 0.000 claims description 3
- 239000003937 drug carrier Substances 0.000 claims description 3
- 230000001105 regulatory effect Effects 0.000 claims description 3
- 241000894007 species Species 0.000 claims description 3
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 2
- 238000012230 antisense oligonucleotides Methods 0.000 claims description 2
- 210000000234 capsid Anatomy 0.000 claims description 2
- 229920002477 rna polymer Polymers 0.000 claims description 2
- 238000000338 in vitro Methods 0.000 claims 3
- 150000008298 phosphoramidates Chemical class 0.000 claims 1
- 238000007726 management method Methods 0.000 description 5
- 230000001225 therapeutic effect Effects 0.000 description 2
- 108020005004 Guide RNA Proteins 0.000 description 1
- 108020004459 Small interfering RNA Proteins 0.000 description 1
- 108091070501 miRNA Proteins 0.000 description 1
- 239000002679 microRNA Substances 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063012603P | 2020-04-20 | 2020-04-20 | |
| US63/012,603 | 2020-04-20 | ||
| PCT/US2021/028166 WO2021216556A2 (en) | 2020-04-20 | 2021-04-20 | Oligonucleotides for msh3 modulation |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023522701A JP2023522701A (ja) | 2023-05-31 |
| JPWO2021216556A5 true JPWO2021216556A5 (xx) | 2024-05-02 |
Family
ID=78270051
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022563451A Pending JP2023522701A (ja) | 2020-04-20 | 2021-04-20 | Ｍｓｈ３調節のためのオリゴヌクレオチド |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20210355491A1 (xx) |
| EP (1) | EP4138860A2 (xx) |
| JP (1) | JP2023522701A (xx) |
| AU (1) | AU2021260581A1 (xx) |
| CA (1) | CA3176204A1 (xx) |
| WO (1) | WO2021216556A2 (xx) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL307239A (en) | 2021-03-29 | 2023-11-01 | Alnylam Pharmaceuticals Inc | Preparations containing Huntingtin IRNA factor (HTT) and methods of using them |
| TW202334418A (zh) * | 2021-10-29 | 2023-09-01 | 美商艾拉倫製藥股份有限公司 | 杭丁頓（ＨＴＴ）ｉＲＮＡ劑組成物及其使用方法 |
| WO2023114989A2 (en) * | 2021-12-17 | 2023-06-22 | University Of Massachusetts | Oligonucleotides for mlh3 modulation |
| US20240035027A1 (en) * | 2022-05-04 | 2024-02-01 | University Of Massachusetts | Oligonucleotides for pms2 modulation |
| WO2023225495A2 (en) * | 2022-05-16 | 2023-11-23 | Atalanta Therapeutics, Inc. | Compositions and methods for treatment of microsatellite dna expansion disorders |
| WO2024064954A2 (en) * | 2022-09-23 | 2024-03-28 | The Children's Hospital Of Philadelphia | Compositions and methods for treating huntington's disease and related disorders |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AUPO974597A0 (en) * | 1997-10-10 | 1997-11-06 | Rhone-Poulenc Agro | Methods for obtaining plant varieties |
| US7361468B2 (en) * | 2004-07-02 | 2008-04-22 | Affymetrix, Inc. | Methods for genotyping polymorphisms in humans |
| WO2015171918A2 (en) * | 2014-05-07 | 2015-11-12 | Louisiana State University And Agricultural And Mechanical College | Compositions and uses for treatment thereof |
| US10450563B2 (en) * | 2015-02-10 | 2019-10-22 | Genzyme Corporation | Variant RNAi |
| LT3277814T (lt) * | 2015-04-03 | 2020-10-26 | University Of Massachusetts | Oligonukleotidų junginiai, skirti nukreipimui į hantingtino mrnr |
| US20170009304A1 (en) * | 2015-07-07 | 2017-01-12 | Splicingcodes.Com | Method and kit for detecting fusion transcripts |
| AU2017210726B2 (en) * | 2016-01-31 | 2023-08-03 | University Of Massachusetts | Branched oligonucleotides |
| MX2021006463A (es) * | 2018-12-03 | 2021-09-28 | Triplet Therapeutics Inc | Metodos para el tratamiento de trastornos de expansion repetida de trinucleotidos asociados con actividad msh3. |
-
2021
- 2021-04-20 JP JP2022563451A patent/JP2023522701A/ja active Pending
- 2021-04-20 CA CA3176204A patent/CA3176204A1/en active Pending
- 2021-04-20 US US17/235,153 patent/US20210355491A1/en active Pending
- 2021-04-20 EP EP21792058.6A patent/EP4138860A2/en active Pending
- 2021-04-20 WO PCT/US2021/028166 patent/WO2021216556A2/en unknown
- 2021-04-20 AU AU2021260581A patent/AU2021260581A1/en active Pending
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