JPWO2021188626A5 - - Google Patents
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- JPWO2021188626A5 JPWO2021188626A5 JP2022556142A JP2022556142A JPWO2021188626A5 JP WO2021188626 A5 JPWO2021188626 A5 JP WO2021188626A5 JP 2022556142 A JP2022556142 A JP 2022556142A JP 2022556142 A JP2022556142 A JP 2022556142A JP WO2021188626 A5 JPWO2021188626 A5 JP WO2021188626A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotides
- antisense strand
- dsrna
- sense strand
- positions
- 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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- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 64
- 230000000692 anti-sense effect Effects 0.000 claims 60
- 125000003729 nucleotide group Chemical group 0.000 claims 60
- 239000002773 nucleotide Substances 0.000 claims 53
- 108091081021 Sense strand Proteins 0.000 claims 43
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims 38
- 150000007523 nucleic acids Chemical group 0.000 claims 27
- 230000000295 complement effect Effects 0.000 claims 24
- 102100040243 Microtubule-associated protein tau Human genes 0.000 claims 23
- 101710115937 Microtubule-associated protein tau Proteins 0.000 claims 23
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 21
- 150000001875 compounds Chemical class 0.000 claims 19
- 230000004048 modification Effects 0.000 claims 17
- 238000012986 modification Methods 0.000 claims 17
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 15
- 239000002336 ribonucleotide Substances 0.000 claims 14
- 101150070547 MAPT gene Proteins 0.000 claims 10
- 125000005647 linker group Chemical group 0.000 claims 10
- 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 8
- 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 8
- 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 8
- 230000014509 gene expression Effects 0.000 claims 8
- 150000004713 phosphodiesters Chemical class 0.000 claims 7
- 108020004414 DNA Proteins 0.000 claims 6
- 239000000203 mixture Substances 0.000 claims 6
- LYCAIKOWRPUZTN-UHFFFAOYSA-N Ethylene glycol Chemical group OCCO LYCAIKOWRPUZTN-UHFFFAOYSA-N 0.000 claims 5
- 229910019142 PO4 Inorganic materials 0.000 claims 5
- 125000000217 alkyl group Chemical group 0.000 claims 5
- 150000001408 amides Chemical class 0.000 claims 5
- 210000004027 cell Anatomy 0.000 claims 5
- 102000039446 nucleic acids Human genes 0.000 claims 5
- 108020004707 nucleic acids Proteins 0.000 claims 5
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 5
- 239000010452 phosphate Substances 0.000 claims 5
- PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims 5
- 108090000765 processed proteins & peptides Proteins 0.000 claims 5
- 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 4
- 239000002126 C01EB10 - Adenosine Substances 0.000 claims 4
- 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 4
- 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 4
- 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 4
- 108091027568 Single-stranded nucleotide Proteins 0.000 claims 4
- 229960005305 adenosine Drugs 0.000 claims 4
- 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 4
- 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 4
- UKMSUNONTOPOIO-UHFFFAOYSA-N docosanoic acid Chemical compound CCCCCCCCCCCCCCCCCCCCCC(O)=O UKMSUNONTOPOIO-UHFFFAOYSA-N 0.000 claims 4
- 150000002148 esters Chemical class 0.000 claims 4
- 229940029575 guanosine Drugs 0.000 claims 4
- 238000002347 injection Methods 0.000 claims 4
- 239000007924 injection Substances 0.000 claims 4
- 238000000034 method Methods 0.000 claims 4
- UEZVMMHDMIWARA-UHFFFAOYSA-M phosphonate Chemical compound [O-]P(=O)=O UEZVMMHDMIWARA-UHFFFAOYSA-M 0.000 claims 4
- 125000006850 spacer group Chemical group 0.000 claims 4
- 150000003852 triazoles Chemical class 0.000 claims 4
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims 4
- 229940045145 uridine Drugs 0.000 claims 4
- 208000011990 Corticobasal Degeneration Diseases 0.000 claims 3
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 3
- -1 antagomiR Proteins 0.000 claims 3
- 238000001990 intravenous administration Methods 0.000 claims 3
- 108020004999 messenger RNA Proteins 0.000 claims 3
- 230000004770 neurodegeneration Effects 0.000 claims 3
- 229940088594 vitamin Drugs 0.000 claims 3
- 239000011782 vitamin Substances 0.000 claims 3
- 229930003231 vitamin Natural products 0.000 claims 3
- 235000013343 vitamin Nutrition 0.000 claims 3
- 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 2
- 206010003591 Ataxia Diseases 0.000 claims 2
- 235000021357 Behenic acid Nutrition 0.000 claims 2
- 208000004051 Chronic Traumatic Encephalopathy Diseases 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 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 2
- 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 2
- 229940116226 behenic acid Drugs 0.000 claims 2
- 238000007385 chemical modification Methods 0.000 claims 2
- 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 2
- 208000017004 dementia pugilistica Diseases 0.000 claims 2
- 235000014113 dietary fatty acids Nutrition 0.000 claims 2
- KFEVDPWXEVUUMW-UHFFFAOYSA-N docosanoic acid Natural products CCCCCCCCCCCCCCCCCCCCCC(=O)OCCC1=CC=C(O)C=C1 KFEVDPWXEVUUMW-UHFFFAOYSA-N 0.000 claims 2
- 235000020673 eicosapentaenoic acid Nutrition 0.000 claims 2
- 229960005135 eicosapentaenoic acid Drugs 0.000 claims 2
- JAZBEHYOTPTENJ-UHFFFAOYSA-N eicosapentaenoic acid Natural products CCC=CCC=CCC=CCC=CCC=CCCCC(O)=O JAZBEHYOTPTENJ-UHFFFAOYSA-N 0.000 claims 2
- 229930195729 fatty acid Natural products 0.000 claims 2
- 239000000194 fatty acid Substances 0.000 claims 2
- 150000004665 fatty acids Chemical class 0.000 claims 2
- 230000002209 hydrophobic effect Effects 0.000 claims 2
- 230000002401 inhibitory effect Effects 0.000 claims 2
- 230000003993 interaction Effects 0.000 claims 2
- 238000000185 intracerebroventricular administration Methods 0.000 claims 2
- 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 2
- POULHZVOKOAJMA-UHFFFAOYSA-N methyl undecanoic acid Natural products CCCCCCCCCCCC(O)=O POULHZVOKOAJMA-UHFFFAOYSA-N 0.000 claims 2
- 208000015122 neurodegenerative disease Diseases 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 201000002212 progressive supranuclear palsy Diseases 0.000 claims 2
- 230000001105 regulatory effect Effects 0.000 claims 2
- 241000894007 species Species 0.000 claims 2
- 150000003431 steroids Chemical class 0.000 claims 2
- 230000001225 therapeutic effect Effects 0.000 claims 2
- 150000003722 vitamin derivatives Chemical class 0.000 claims 2
- 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 1
- 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 1
- 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 1
- 208000035657 Abasia Diseases 0.000 claims 1
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 208000014644 Brain disease Diseases 0.000 claims 1
- KXDHJXZQYSOELW-UHFFFAOYSA-M Carbamate Chemical compound NC([O-])=O KXDHJXZQYSOELW-UHFFFAOYSA-M 0.000 claims 1
- 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 1
- 208000032274 Encephalopathy Diseases 0.000 claims 1
- 208000001914 Fragile X syndrome Diseases 0.000 claims 1
- 208000024412 Friedreich ataxia Diseases 0.000 claims 1
- 208000032612 Glial tumor Diseases 0.000 claims 1
- 206010018338 Glioma Diseases 0.000 claims 1
- 108020005004 Guide RNA Proteins 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 208000036626 Mental retardation Diseases 0.000 claims 1
- 206010068871 Myotonic dystrophy Diseases 0.000 claims 1
- 150000001200 N-acyl ethanolamides Chemical class 0.000 claims 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 1
- 108010021592 Pantothenate kinase Proteins 0.000 claims 1
- 102100024122 Pantothenate kinase 1 Human genes 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 208000027089 Parkinsonian disease Diseases 0.000 claims 1
- 206010034010 Parkinsonism Diseases 0.000 claims 1
- 208000000609 Pick Disease of the Brain Diseases 0.000 claims 1
- 208000036757 Postencephalitic parkinsonism Diseases 0.000 claims 1
- 108091028664 Ribonucleotide Proteins 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 208000037065 Subacute sclerosing leukoencephalitis Diseases 0.000 claims 1
- 206010042297 Subacute sclerosing panencephalitis Diseases 0.000 claims 1
- 208000034799 Tauopathies Diseases 0.000 claims 1
- 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 1
- 208000026911 Tuberous sclerosis complex Diseases 0.000 claims 1
- 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 1
- 229930003427 Vitamin E Natural products 0.000 claims 1
- 125000003545 alkoxy group Chemical group 0.000 claims 1
- 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 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 230000015556 catabolic process Effects 0.000 claims 1
- 210000001638 cerebellum Anatomy 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 229960001231 choline Drugs 0.000 claims 1
- OEYIOHPDSNJKLS-UHFFFAOYSA-N choline Chemical compound C[N+](C)(C)CCO OEYIOHPDSNJKLS-UHFFFAOYSA-N 0.000 claims 1
- 210000000349 chromosome Anatomy 0.000 claims 1
- 229940099418 d- alpha-tocopherol succinate Drugs 0.000 claims 1
- 238000006731 degradation reaction Methods 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 235000020669 docosahexaenoic acid Nutrition 0.000 claims 1
- KAUVQQXNCKESLC-UHFFFAOYSA-N docosahexaenoic acid (DHA) Natural products COC(=O)C(C)NOCC1=CC=CC=C1 KAUVQQXNCKESLC-UHFFFAOYSA-N 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000002621 endocannabinoid Substances 0.000 claims 1
- ZTWTYVWXUKTLCP-UHFFFAOYSA-L ethenyl-dioxido-oxo-$l^{5}-phosphane Chemical compound [O-]P([O-])(=O)C=C ZTWTYVWXUKTLCP-UHFFFAOYSA-L 0.000 claims 1
- 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 1
- 201000005649 gangliocytoma Diseases 0.000 claims 1
- 201000008361 ganglioneuroma Diseases 0.000 claims 1
- 150000002270 gangliosides Chemical class 0.000 claims 1
- 125000005843 halogen group Chemical group 0.000 claims 1
- 210000001320 hippocampus Anatomy 0.000 claims 1
- 125000004356 hydroxy functional group Chemical group O* 0.000 claims 1
- 210000003016 hypothalamus Anatomy 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 239000002207 metabolite Substances 0.000 claims 1
- 108091070501 miRNA Proteins 0.000 claims 1
- 239000002679 microRNA Substances 0.000 claims 1
- 210000001577 neostriatum Anatomy 0.000 claims 1
- 239000002777 nucleoside Chemical class 0.000 claims 1
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 1
- 150000008298 phosphoramidates Chemical class 0.000 claims 1
- 208000000170 postencephalitic Parkinson disease Diseases 0.000 claims 1
- 125000006239 protecting group Chemical group 0.000 claims 1
- 229930002330 retinoic acid Natural products 0.000 claims 1
- 125000002652 ribonucleotide group Chemical group 0.000 claims 1
- 239000011435 rock Substances 0.000 claims 1
- 150000003338 secosteroids Chemical class 0.000 claims 1
- 210000000278 spinal cord Anatomy 0.000 claims 1
- 201000003594 spinocerebellar ataxia type 12 Diseases 0.000 claims 1
- 238000007920 subcutaneous administration Methods 0.000 claims 1
- 210000001103 thalamus Anatomy 0.000 claims 1
- 229960001727 tretinoin Drugs 0.000 claims 1
- 208000009999 tuberous sclerosis Diseases 0.000 claims 1
- 235000019155 vitamin A Nutrition 0.000 claims 1
- 239000011719 vitamin A Substances 0.000 claims 1
- 229940046009 vitamin E Drugs 0.000 claims 1
- 235000019165 vitamin E Nutrition 0.000 claims 1
- 239000011709 vitamin E Substances 0.000 claims 1
- 229940045997 vitamin a Drugs 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062991405P | 2020-03-18 | 2020-03-18 | |
| US62/991,405 | 2020-03-18 | ||
| US202063071106P | 2020-08-27 | 2020-08-27 | |
| US63/071,106 | 2020-08-27 | ||
| PCT/US2021/022688 WO2021188626A1 (en) | 2020-03-18 | 2021-03-17 | Oligonucleotides for mapt modulation |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023518439A JP2023518439A (ja) | 2023-05-01 |
| JP2023518439A5 JP2023518439A5 (https=) | 2024-03-28 |
| JPWO2021188626A5 true JPWO2021188626A5 (https=) | 2024-03-28 |
Family
ID=77768304
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022556142A Pending JP2023518439A (ja) | 2020-03-18 | 2021-03-17 | Ｍａｐｔ調節のためのオリゴヌクレオチド |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US20210363523A1 (https=) |
| EP (1) | EP4121537A4 (https=) |
| JP (1) | JP2023518439A (https=) |
| KR (1) | KR20230004448A (https=) |
| CN (1) | CN115552006A (https=) |
| AU (1) | AU2021238319A1 (https=) |
| CA (1) | CA3171246A1 (https=) |
| MX (1) | MX2022011516A (https=) |
| WO (1) | WO2021188626A1 (https=) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3277811B1 (en) | 2015-04-03 | 2020-12-23 | University of Massachusetts | Fully stabilized asymmetric sirna |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| JP7406793B2 (ja) | 2017-06-23 | 2023-12-28 | ユニバーシティー オブ マサチューセッツ | ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法 |
| JP7627042B2 (ja) | 2018-08-23 | 2025-02-05 | ユニバーシティー オブ マサチューセッツ | Ｏ－メチルリッチ完全安定化オリゴヌクレオチド |
| CN113614232A (zh) | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| MX2022001710A (es) | 2019-08-09 | 2022-05-10 | Univ Massachusetts | Oligonucleótidos modificados químicamente dirigidos a los snp. |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| AU2021246024A1 (en) * | 2020-03-30 | 2022-10-27 | Alnylam Pharmaceuticals, Inc. | Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof |
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| KR20240067943A (ko) | 2021-09-24 | 2024-05-17 | 알닐람 파마슈티칼스 인코포레이티드 | 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법 |
| US20250340872A1 (en) * | 2021-10-07 | 2025-11-06 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing tau expression |
| EP4448765A1 (en) * | 2021-12-13 | 2024-10-23 | Eli Lilly and Company | Mapt rna interference agents |
| WO2023173061A2 (en) * | 2022-03-11 | 2023-09-14 | University Of Massachusetts | Oligonucleotides for app modulation |
| WO2023193004A2 (en) * | 2022-04-01 | 2023-10-05 | Triplet Therapeutics, Inc. | Methods for the treatment of neurodegenerative disorders |
| CN119173631A (zh) * | 2022-05-12 | 2024-12-20 | 迪克纳制药公司 | 用于抑制mapt表达的组合物和方法 |
| WO2023217437A1 (en) * | 2022-05-13 | 2023-11-16 | Julius-Maximilians-Universität Würzburg | Method and molecules for reducing axonal tau protein accumulation through blocking of hnrnp r-mediated mapt mrna transport for treatment of alzheimer's disease |
| WO2024073618A2 (en) * | 2022-09-29 | 2024-04-04 | Atalanta Therapeutics, Inc. | Sirna compositions and methods targeting microtubule associated protein tau nucleic acids |
| KR20250129743A (ko) * | 2022-12-29 | 2025-08-29 | 보이저 테라퓨틱스, 인크. | Mapt를 조절하기 위한 조성물 및 방법 |
| WO2024201423A2 (en) * | 2023-03-30 | 2024-10-03 | Biorchestra Co., Ltd. | Microtubule-associated protein tau targeting sirnas and uses thereof |
| WO2024220930A2 (en) * | 2023-04-20 | 2024-10-24 | Adarx Pharmaceuticals, Inc. | Mapt-modulating compositions and methods of use thereof |
| AU2024315297A1 (en) * | 2023-08-01 | 2026-02-12 | Tuojie Biotech (Shanghai) Co., Ltd. | Dsrna targeting mapt and pharmaceutical use thereof |
| TW202515584A (zh) * | 2023-10-11 | 2025-04-16 | 大陸商上海舶望製藥有限公司 | 用於抑制微管相關蛋白ｔａｕ（ｍａｐｔ）表達的組合物和方法 |
| US12551569B2 (en) | 2024-03-31 | 2026-02-17 | Arrowhead Pharmaceuticals, Inc. | RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use |
| WO2025233849A2 (en) * | 2024-05-07 | 2025-11-13 | Janssen Pharmaceutica Nv | MAPT siRNA AND USES THEREOF |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050255086A1 (en) * | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
| CA2585970A1 (en) * | 2004-10-27 | 2006-05-04 | Vanderbilt University | Mammalian genes involved in infection |
| GB0605337D0 (en) * | 2006-03-17 | 2006-04-26 | Genomica Sau | Treatment of CNS conditions |
| WO2008124066A1 (en) * | 2007-04-05 | 2008-10-16 | The J. David Gladstone Institutes | Agents that reduce neuronal overexcitation |
| ES2804764T3 (es) * | 2009-06-01 | 2021-02-09 | Halo Bio Rnai Therapeutics Inc | Polinucleótidos para la interferencia de ARN multivalente, composiciones y métodos de uso de los mismos |
| TWI772856B (zh) * | 2013-07-19 | 2022-08-01 | 美商百健Ｍａ公司 | 用於調節τ蛋白表現之組合物 |
| EP3274456B1 (en) * | 2015-03-25 | 2020-06-24 | Università Degli Studi Di Trento | Rna interference mediated therapy for neurodegenerative diseases |
| EP3277811B1 (en) * | 2015-04-03 | 2020-12-23 | University of Massachusetts | Fully stabilized asymmetric sirna |
| WO2017132669A1 (en) * | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
| JP7627042B2 (ja) * | 2018-08-23 | 2025-02-05 | ユニバーシティー オブ マサチューセッツ | Ｏ－メチルリッチ完全安定化オリゴヌクレオチド |
| JP2022545118A (ja) * | 2019-08-23 | 2022-10-25 | ユニバーシティー オブ マサチューセッツ | Ｏ－メチルリッチ完全安定化オリゴヌクレオチド |
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2021
- 2021-03-17 AU AU2021238319A patent/AU2021238319A1/en active Pending
- 2021-03-17 WO PCT/US2021/022688 patent/WO2021188626A1/en not_active Ceased
- 2021-03-17 KR KR1020227031792A patent/KR20230004448A/ko active Pending
- 2021-03-17 MX MX2022011516A patent/MX2022011516A/es unknown
- 2021-03-17 CN CN202180034587.6A patent/CN115552006A/zh active Pending
- 2021-03-17 US US17/204,480 patent/US20210363523A1/en active Pending
- 2021-03-17 JP JP2022556142A patent/JP2023518439A/ja active Pending
- 2021-03-17 EP EP21770963.3A patent/EP4121537A4/en active Pending
- 2021-03-17 CA CA3171246A patent/CA3171246A1/en active Pending
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2025
- 2025-09-24 US US19/338,285 patent/US20260109982A1/en active Pending
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