JP2018501816A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018501816A5 JP2018501816A5 JP2017544992A JP2017544992A JP2018501816A5 JP 2018501816 A5 JP2018501816 A5 JP 2018501816A5 JP 2017544992 A JP2017544992 A JP 2017544992A JP 2017544992 A JP2017544992 A JP 2017544992A JP 2018501816 A5 JP2018501816 A5 JP 2018501816A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense
- disease
- sequence
- oligonucleotide
- seq
- 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.)
- Granted
Links
- 239000000074 antisense oligonucleotide Substances 0.000 claims 24
- 238000012230 antisense oligonucleotides Methods 0.000 claims 24
- 108091034117 Oligonucleotide Proteins 0.000 claims 21
- 239000002773 nucleotide Substances 0.000 claims 9
- 125000003729 nucleotide group Chemical group 0.000 claims 9
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 7
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims 5
- 206010016654 Fibrosis Diseases 0.000 claims 5
- 201000010099 disease Diseases 0.000 claims 5
- 230000004761 fibrosis Effects 0.000 claims 4
- 108020004999 messenger RNA Proteins 0.000 claims 4
- 206010012289 Dementia Diseases 0.000 claims 3
- 206010021143 Hypoxia Diseases 0.000 claims 3
- 210000003169 central nervous system Anatomy 0.000 claims 3
- 206010014599 encephalitis Diseases 0.000 claims 3
- 208000014674 injury Diseases 0.000 claims 3
- 201000011475 meningoencephalitis Diseases 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 230000008733 trauma Effects 0.000 claims 3
- OVONXEQGWXGFJD-UHFFFAOYSA-N 4-sulfanylidene-1h-pyrimidin-2-one Chemical compound SC=1C=CNC(=O)N=1 OVONXEQGWXGFJD-UHFFFAOYSA-N 0.000 claims 2
- OIVLITBTBDPEFK-UHFFFAOYSA-N 5,6-dihydrouracil Chemical compound O=C1CCNC(=O)N1 OIVLITBTBDPEFK-UHFFFAOYSA-N 0.000 claims 2
- RYVNIFSIEDRLSJ-UHFFFAOYSA-N 5-(hydroxymethyl)cytosine Chemical compound NC=1NC(=O)N=CC=1CO RYVNIFSIEDRLSJ-UHFFFAOYSA-N 0.000 claims 2
- -1 6- Methylguanine Chemical compound 0.000 claims 2
- PNWOYKVCNDZOLS-UHFFFAOYSA-N 6-amino-5-chloro-1h-pyrimidin-2-one Chemical compound NC=1NC(=O)N=CC=1Cl PNWOYKVCNDZOLS-UHFFFAOYSA-N 0.000 claims 2
- PEHVGBZKEYRQSX-UHFFFAOYSA-N 7-deaza-adenine Chemical compound NC1=NC=NC2=C1C=CN2 PEHVGBZKEYRQSX-UHFFFAOYSA-N 0.000 claims 2
- LRFVTYWOQMYALW-UHFFFAOYSA-N 9H-xanthine Chemical compound O=C1NC(=O)NC2=C1NC=N2 LRFVTYWOQMYALW-UHFFFAOYSA-N 0.000 claims 2
- 208000020406 Creutzfeldt Jacob disease Diseases 0.000 claims 2
- 208000003407 Creutzfeldt-Jakob Syndrome Diseases 0.000 claims 2
- 208000010859 Creutzfeldt-Jakob disease Diseases 0.000 claims 2
- 208000001089 Multiple system atrophy Diseases 0.000 claims 2
- 206010028980 Neoplasm Diseases 0.000 claims 2
- 208000036110 Neuroinflammatory disease Diseases 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical group OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims 2
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 2
- 239000003795 chemical substances by application Substances 0.000 claims 2
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 2
- 230000007850 degeneration Effects 0.000 claims 2
- NAGJZTKCGNOGPW-UHFFFAOYSA-N dithiophosphoric acid Chemical group OP(O)(S)=S NAGJZTKCGNOGPW-UHFFFAOYSA-N 0.000 claims 2
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 2
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims 2
- 230000001146 hypoxic effect Effects 0.000 claims 2
- 230000004770 neurodegeneration Effects 0.000 claims 2
- 208000015122 neurodegenerative disease Diseases 0.000 claims 2
- 230000003287 optical effect Effects 0.000 claims 2
- PXQPEWDEAKTCGB-UHFFFAOYSA-N orotic acid Chemical compound OC(=O)C1=CC(=O)NC(=O)N1 PXQPEWDEAKTCGB-UHFFFAOYSA-N 0.000 claims 2
- 201000010041 presbyopia Diseases 0.000 claims 2
- 230000008929 regeneration Effects 0.000 claims 2
- 238000011069 regeneration method Methods 0.000 claims 2
- 210000000278 spinal cord Anatomy 0.000 claims 2
- 230000009885 systemic effect Effects 0.000 claims 2
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 2
- 229940035893 uracil Drugs 0.000 claims 2
- HASUWNAFLUMMFI-UHFFFAOYSA-N 1,7-dihydropyrrolo[2,3-d]pyrimidine-2,4-dione Chemical compound O=C1NC(=O)NC2=C1C=CN2 HASUWNAFLUMMFI-UHFFFAOYSA-N 0.000 claims 1
- UHUHBFMZVCOEOV-UHFFFAOYSA-N 1h-imidazo[4,5-c]pyridin-4-amine Chemical compound NC1=NC=CC2=C1N=CN2 UHUHBFMZVCOEOV-UHFFFAOYSA-N 0.000 claims 1
- WYDKPTZGVLTYPG-UHFFFAOYSA-N 2,8-diamino-3,7-dihydropurin-6-one Chemical compound N1C(N)=NC(=O)C2=C1N=C(N)N2 WYDKPTZGVLTYPG-UHFFFAOYSA-N 0.000 claims 1
- HTOVHZGIBCAAJU-UHFFFAOYSA-N 2-amino-2-propyl-1h-purin-6-one Chemical compound CCCC1(N)NC(=O)C2=NC=NC2=N1 HTOVHZGIBCAAJU-UHFFFAOYSA-N 0.000 claims 1
- CRYCZDRIXVHNQB-UHFFFAOYSA-N 2-amino-8-bromo-3,7-dihydropurin-6-one Chemical compound N1C(N)=NC(=O)C2=C1N=C(Br)N2 CRYCZDRIXVHNQB-UHFFFAOYSA-N 0.000 claims 1
- YCFWZXAEOXKNHL-UHFFFAOYSA-N 2-amino-8-chloro-3,7-dihydropurin-6-one Chemical compound N1C(N)=NC(=O)C2=C1N=C(Cl)N2 YCFWZXAEOXKNHL-UHFFFAOYSA-N 0.000 claims 1
- GBSSBTKECSBHSE-UHFFFAOYSA-N 2-amino-8-fluoro-3,7-dihydropurin-6-one Chemical compound O=C1NC(N)=NC2=C1NC(F)=N2 GBSSBTKECSBHSE-UHFFFAOYSA-N 0.000 claims 1
- SXGFECRAKVVEJT-UHFFFAOYSA-N 2-amino-8-iodo-3,7-dihydropurin-6-one Chemical compound N1C(N)=NC(=O)C2=C1N=C(I)N2 SXGFECRAKVVEJT-UHFFFAOYSA-N 0.000 claims 1
- USCCECGPGBGFOM-UHFFFAOYSA-N 2-propyl-7h-purin-6-amine Chemical compound CCCC1=NC(N)=C2NC=NC2=N1 USCCECGPGBGFOM-UHFFFAOYSA-N 0.000 claims 1
- LMNPKIOZMGYQIU-UHFFFAOYSA-N 5-(trifluoromethyl)-1h-pyrimidine-2,4-dione Chemical compound FC(F)(F)C1=CNC(=O)NC1=O LMNPKIOZMGYQIU-UHFFFAOYSA-N 0.000 claims 1
- SVXNJCYYMRMXNM-UHFFFAOYSA-N 5-amino-2h-1,2,4-triazin-3-one Chemical compound NC=1C=NNC(=O)N=1 SVXNJCYYMRMXNM-UHFFFAOYSA-N 0.000 claims 1
- LQLQRFGHAALLLE-UHFFFAOYSA-N 5-bromouracil Chemical compound BrC1=CNC(=O)NC1=O LQLQRFGHAALLLE-UHFFFAOYSA-N 0.000 claims 1
- ZFTBZKVVGZNMJR-UHFFFAOYSA-N 5-chlorouracil Chemical compound ClC1=CNC(=O)NC1=O ZFTBZKVVGZNMJR-UHFFFAOYSA-N 0.000 claims 1
- KSNXJLQDQOIRIP-UHFFFAOYSA-N 5-iodouracil Chemical compound IC1=CNC(=O)NC1=O KSNXJLQDQOIRIP-UHFFFAOYSA-N 0.000 claims 1
- ZLAQATDNGLKIEV-UHFFFAOYSA-N 5-methyl-2-sulfanylidene-1h-pyrimidin-4-one Chemical compound CC1=CNC(=S)NC1=O ZLAQATDNGLKIEV-UHFFFAOYSA-N 0.000 claims 1
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
- UJBCLAXPPIDQEE-UHFFFAOYSA-N 5-prop-1-ynyl-1h-pyrimidine-2,4-dione Chemical compound CC#CC1=CNC(=O)NC1=O UJBCLAXPPIDQEE-UHFFFAOYSA-N 0.000 claims 1
- KXBCLNRMQPRVTP-UHFFFAOYSA-N 6-amino-1,5-dihydroimidazo[4,5-c]pyridin-4-one Chemical compound O=C1NC(N)=CC2=C1N=CN2 KXBCLNRMQPRVTP-UHFFFAOYSA-N 0.000 claims 1
- DCPSTSVLRXOYGS-UHFFFAOYSA-N 6-amino-1h-pyrimidine-2-thione Chemical compound NC1=CC=NC(S)=N1 DCPSTSVLRXOYGS-UHFFFAOYSA-N 0.000 claims 1
- OHILKUISCGPRMQ-UHFFFAOYSA-N 6-amino-5-(trifluoromethyl)-1h-pyrimidin-2-one Chemical compound NC1=NC(=O)NC=C1C(F)(F)F OHILKUISCGPRMQ-UHFFFAOYSA-N 0.000 claims 1
- QFVKLKDEXOWFSL-UHFFFAOYSA-N 6-amino-5-bromo-1h-pyrimidin-2-one Chemical compound NC=1NC(=O)N=CC=1Br QFVKLKDEXOWFSL-UHFFFAOYSA-N 0.000 claims 1
- UFVWJVAMULFOMC-UHFFFAOYSA-N 6-amino-5-iodo-1h-pyrimidin-2-one Chemical compound NC=1NC(=O)N=CC=1I UFVWJVAMULFOMC-UHFFFAOYSA-N 0.000 claims 1
- QNNARSZPGNJZIX-UHFFFAOYSA-N 6-amino-5-prop-1-ynyl-1h-pyrimidin-2-one Chemical compound CC#CC1=CNC(=O)N=C1N QNNARSZPGNJZIX-UHFFFAOYSA-N 0.000 claims 1
- XZWMZFQOHTWGQE-UHFFFAOYSA-N 6-azathymine Chemical compound CC1=NNC(=O)NC1=O XZWMZFQOHTWGQE-UHFFFAOYSA-N 0.000 claims 1
- SSPYSWLZOPCOLO-UHFFFAOYSA-N 6-azauracil Chemical compound O=C1C=NNC(=O)N1 SSPYSWLZOPCOLO-UHFFFAOYSA-N 0.000 claims 1
- SFXXRVSPZSOREJ-UHFFFAOYSA-N 6-ethoxy-7h-purin-2-amine Chemical compound CCOC1=NC(N)=NC2=C1NC=N2 SFXXRVSPZSOREJ-UHFFFAOYSA-N 0.000 claims 1
- AIDIRIDKERNCFT-UHFFFAOYSA-N 6-ethylpurin-6-amine Chemical compound C(C)C1(C2=NC=NC2=NC=N1)N AIDIRIDKERNCFT-UHFFFAOYSA-N 0.000 claims 1
- CKOMXBHMKXXTNW-UHFFFAOYSA-N 6-methyladenine Chemical compound CNC1=NC=NC2=C1N=CN2 CKOMXBHMKXXTNW-UHFFFAOYSA-N 0.000 claims 1
- CLGFIVUFZRGQRP-UHFFFAOYSA-N 7,8-dihydro-8-oxoguanine Chemical compound O=C1NC(N)=NC2=C1NC(=O)N2 CLGFIVUFZRGQRP-UHFFFAOYSA-N 0.000 claims 1
- LHCPRYRLDOSKHK-UHFFFAOYSA-N 7-deaza-8-aza-adenine Chemical compound NC1=NC=NC2=C1C=NN2 LHCPRYRLDOSKHK-UHFFFAOYSA-N 0.000 claims 1
- LOSIULRWFAEMFL-UHFFFAOYSA-N 7-deazaguanine Chemical compound O=C1NC(N)=NC2=C1CC=N2 LOSIULRWFAEMFL-UHFFFAOYSA-N 0.000 claims 1
- PFUVOLUPRFCPMN-UHFFFAOYSA-N 7h-purine-6,8-diamine Chemical compound C1=NC(N)=C2NC(N)=NC2=N1 PFUVOLUPRFCPMN-UHFFFAOYSA-N 0.000 claims 1
- HRYKDUPGBWLLHO-UHFFFAOYSA-N 8-azaadenine Chemical compound NC1=NC=NC2=NNN=C12 HRYKDUPGBWLLHO-UHFFFAOYSA-N 0.000 claims 1
- LPXQRXLUHJKZIE-UHFFFAOYSA-N 8-azaguanine Chemical compound NC1=NC(O)=C2NN=NC2=N1 LPXQRXLUHJKZIE-UHFFFAOYSA-N 0.000 claims 1
- 229960005508 8-azaguanine Drugs 0.000 claims 1
- FVXHPCVBOXMRJP-UHFFFAOYSA-N 8-bromo-7h-purin-6-amine Chemical compound NC1=NC=NC2=C1NC(Br)=N2 FVXHPCVBOXMRJP-UHFFFAOYSA-N 0.000 claims 1
- FBVALAOQISRDRY-UHFFFAOYSA-N 8-chloro-7h-purin-6-amine Chemical compound NC1=NC=NC2=C1NC(Cl)=N2 FBVALAOQISRDRY-UHFFFAOYSA-N 0.000 claims 1
- MRZDHXLJHIMNOR-UHFFFAOYSA-N 8-fluoro-7h-purin-6-amine Chemical compound NC1=NC=NC2=C1NC(F)=N2 MRZDHXLJHIMNOR-UHFFFAOYSA-N 0.000 claims 1
- XUMSFQKCBNKNCE-UHFFFAOYSA-N 8-iodo-7h-purin-6-amine Chemical compound NC1=NC=NC2=C1NC(I)=N2 XUMSFQKCBNKNCE-UHFFFAOYSA-N 0.000 claims 1
- MSSXOMSJDRHRMC-UHFFFAOYSA-N 9H-purine-2,6-diamine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 claims 1
- 206010065040 AIDS dementia complex Diseases 0.000 claims 1
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims 1
- 229930024421 Adenine Natural products 0.000 claims 1
- 208000017194 Affective disease Diseases 0.000 claims 1
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 206010003210 Arteriosclerosis Diseases 0.000 claims 1
- 201000001320 Atherosclerosis Diseases 0.000 claims 1
- 208000023275 Autoimmune disease Diseases 0.000 claims 1
- 241000283690 Bos taurus Species 0.000 claims 1
- LRDGSQDXTPBSJZ-UHFFFAOYSA-N CN1C=NC2=C1C(=O)NC(=N2)N.CN1C=NC2=NC=NC(=C21)N Chemical compound CN1C=NC2=C1C(=O)NC(=N2)N.CN1C=NC2=NC=NC(=C21)N LRDGSQDXTPBSJZ-UHFFFAOYSA-N 0.000 claims 1
- 206010008025 Cerebellar ataxia Diseases 0.000 claims 1
- 208000011231 Crohn disease Diseases 0.000 claims 1
- 201000003883 Cystic fibrosis Diseases 0.000 claims 1
- 206010011878 Deafness Diseases 0.000 claims 1
- 201000010374 Down Syndrome Diseases 0.000 claims 1
- 208000001708 Dupuytren contracture Diseases 0.000 claims 1
- 206010015150 Erythema Diseases 0.000 claims 1
- GHASVSINZRGABV-UHFFFAOYSA-N Fluorouracil Chemical compound FC1=CNC(=O)NC1=O GHASVSINZRGABV-UHFFFAOYSA-N 0.000 claims 1
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims 1
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims 1
- 208000024934 IgG4-related mediastinitis Diseases 0.000 claims 1
- 208000002260 Keloid Diseases 0.000 claims 1
- 206010023330 Keloid scar Diseases 0.000 claims 1
- 208000002805 Mediastinal fibrosis Diseases 0.000 claims 1
- 208000019022 Mood disease Diseases 0.000 claims 1
- 208000026072 Motor neurone disease Diseases 0.000 claims 1
- 206010028594 Myocardial fibrosis Diseases 0.000 claims 1
- PJKKQFAEFWCNAQ-UHFFFAOYSA-N N(4)-methylcytosine Chemical compound CNC=1C=CNC(=O)N=1 PJKKQFAEFWCNAQ-UHFFFAOYSA-N 0.000 claims 1
- 208000029726 Neurodevelopmental disease Diseases 0.000 claims 1
- 240000007817 Olea europaea Species 0.000 claims 1
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 208000004362 Penile Induration Diseases 0.000 claims 1
- 208000020758 Peyronie disease Diseases 0.000 claims 1
- 206010036626 Presbyacusis Diseases 0.000 claims 1
- 206010036805 Progressive massive fibrosis Diseases 0.000 claims 1
- 201000007737 Retinal degeneration Diseases 0.000 claims 1
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 1
- 208000009106 Shy-Drager Syndrome Diseases 0.000 claims 1
- 208000009415 Spinocerebellar Ataxias Diseases 0.000 claims 1
- 102000019197 Superoxide Dismutase Human genes 0.000 claims 1
- 108010012715 Superoxide dismutase Proteins 0.000 claims 1
- 201000009594 Systemic Scleroderma Diseases 0.000 claims 1
- 206010042953 Systemic sclerosis Diseases 0.000 claims 1
- 208000030886 Traumatic Brain injury Diseases 0.000 claims 1
- 206010044565 Tremor Diseases 0.000 claims 1
- 201000004810 Vascular dementia Diseases 0.000 claims 1
- 206010063661 Vascular encephalopathy Diseases 0.000 claims 1
- 208000035868 Vascular inflammations Diseases 0.000 claims 1
- 230000001154 acute effect Effects 0.000 claims 1
- 229960000643 adenine Drugs 0.000 claims 1
- 239000002671 adjuvant Substances 0.000 claims 1
- 230000032683 aging Effects 0.000 claims 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- 208000011775 arteriosclerosis disease Diseases 0.000 claims 1
- 230000001746 atrial effect Effects 0.000 claims 1
- 230000001580 bacterial effect Effects 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 208000015114 central nervous system disease Diseases 0.000 claims 1
- 208000025434 cerebellar degeneration Diseases 0.000 claims 1
- 230000007882 cirrhosis Effects 0.000 claims 1
- 208000019425 cirrhosis of liver Diseases 0.000 claims 1
- 229940104302 cytosine Drugs 0.000 claims 1
- 231100000895 deafness Toxicity 0.000 claims 1
- 230000001419 dependent effect Effects 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- NAGJZTKCGNOGPW-UHFFFAOYSA-K dioxido-sulfanylidene-sulfido-$l^{5}-phosphane Chemical compound [O-]P([O-])([S-])=S NAGJZTKCGNOGPW-UHFFFAOYSA-K 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 231100000321 erythema Toxicity 0.000 claims 1
- 239000000835 fiber Substances 0.000 claims 1
- 230000003176 fibrotic effect Effects 0.000 claims 1
- XRECTZIEBJDKEO-UHFFFAOYSA-N flucytosine Chemical compound NC1=NC(=O)NC=C1F XRECTZIEBJDKEO-UHFFFAOYSA-N 0.000 claims 1
- 229960004413 flucytosine Drugs 0.000 claims 1
- 229960002949 fluorouracil Drugs 0.000 claims 1
- 208000016354 hearing loss disease Diseases 0.000 claims 1
- 229910052739 hydrogen Inorganic materials 0.000 claims 1
- 239000001257 hydrogen Substances 0.000 claims 1
- 230000003463 hyperproliferative effect Effects 0.000 claims 1
- 230000007954 hypoxia Effects 0.000 claims 1
- 208000015181 infectious disease Diseases 0.000 claims 1
- 230000000302 ischemic effect Effects 0.000 claims 1
- 210000001117 keloid Anatomy 0.000 claims 1
- 230000003902 lesion Effects 0.000 claims 1
- 208000002780 macular degeneration Diseases 0.000 claims 1
- 208000024714 major depressive disease Diseases 0.000 claims 1
- 206010027175 memory impairment Diseases 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 208000005264 motor neuron disease Diseases 0.000 claims 1
- 201000006417 multiple sclerosis Diseases 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 206010028537 myelofibrosis Diseases 0.000 claims 1
- 208000010125 myocardial infarction Diseases 0.000 claims 1
- 210000000118 neural pathway Anatomy 0.000 claims 1
- 230000010004 neural pathway Effects 0.000 claims 1
- 210000001178 neural stem cell Anatomy 0.000 claims 1
- 208000017376 neurovascular disease Diseases 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 1
- 239000010452 phosphate Substances 0.000 claims 1
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 201000002212 progressive supranuclear palsy Diseases 0.000 claims 1
- 230000001737 promoting effect Effects 0.000 claims 1
- 208000005069 pulmonary fibrosis Diseases 0.000 claims 1
- 230000004258 retinal degeneration Effects 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 201000000980 schizophrenia Diseases 0.000 claims 1
- 239000002904 solvent Substances 0.000 claims 1
- 208000002320 spinal muscular atrophy Diseases 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
- 229940113082 thymine Drugs 0.000 claims 1
- 230000000472 traumatic effect Effects 0.000 claims 1
- 208000019553 vascular disease Diseases 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
- 229940075420 xanthine Drugs 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021014438A JP6983343B2 (ja) | 2014-11-16 | 2021-02-01 | Ｔｇｆ−ｒシグナリング阻害剤としてのアンチセンス−オリゴヌクレオチド |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP14193368.9A EP3020813A1 (en) | 2014-11-16 | 2014-11-16 | Antisense-oligonucleotides as inhibitors of TGF-R signaling |
| EP14193368.9 | 2014-11-16 | ||
| PCT/EP2015/076730 WO2016075339A1 (en) | 2014-11-16 | 2015-11-16 | Antisense-oligonucleotides as inhibitors of tgf-r signaling |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021014438A Division JP6983343B2 (ja) | 2014-11-16 | 2021-02-01 | Ｔｇｆ−ｒシグナリング阻害剤としてのアンチセンス−オリゴヌクレオチド |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018501816A JP2018501816A (ja) | 2018-01-25 |
| JP2018501816A5 true JP2018501816A5 (enExample) | 2019-01-10 |
| JP6832285B2 JP6832285B2 (ja) | 2021-02-24 |
Family
ID=51900289
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017544992A Active JP6832285B2 (ja) | 2014-11-16 | 2015-11-16 | Ｔｇｆ−ｒシグナリング阻害剤としてのアンチセンス−オリゴヌクレオチド |
| JP2021014438A Active JP6983343B2 (ja) | 2014-11-16 | 2021-02-01 | Ｔｇｆ−ｒシグナリング阻害剤としてのアンチセンス−オリゴヌクレオチド |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021014438A Active JP6983343B2 (ja) | 2014-11-16 | 2021-02-01 | Ｔｇｆ−ｒシグナリング阻害剤としてのアンチセンス−オリゴヌクレオチド |
Country Status (22)
| Country | Link |
|---|---|
| US (4) | US11213541B2 (enExample) |
| EP (3) | EP3020813A1 (enExample) |
| JP (2) | JP6832285B2 (enExample) |
| KR (2) | KR102310899B1 (enExample) |
| CN (2) | CN107002082B (enExample) |
| AU (3) | AU2015345006B2 (enExample) |
| BR (1) | BR112017006201B1 (enExample) |
| CA (2) | CA3201845C (enExample) |
| CL (2) | CL2017001233A1 (enExample) |
| CY (1) | CY1121110T1 (enExample) |
| DK (1) | DK3218488T3 (enExample) |
| ES (1) | ES2705066T3 (enExample) |
| HU (1) | HUE041793T2 (enExample) |
| IL (2) | IL251565B (enExample) |
| MX (2) | MX2017006369A (enExample) |
| MY (1) | MY181404A (enExample) |
| PL (1) | PL3218488T3 (enExample) |
| PT (1) | PT3218488T (enExample) |
| RU (1) | RU2717454C2 (enExample) |
| SG (1) | SG11201703127SA (enExample) |
| TR (1) | TR201900844T4 (enExample) |
| WO (1) | WO2016075339A1 (enExample) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3020813A1 (en) * | 2014-11-16 | 2016-05-18 | Neurovision Pharma GmbH | Antisense-oligonucleotides as inhibitors of TGF-R signaling |
| WO2019010226A1 (en) * | 2017-07-03 | 2019-01-10 | The Regents Of The University Of California | CHEMICALLY MODIFIED MICROARN-214 ANTISENS AND USE THEREOF FOR THE TREATMENT OF HEMORRHAGIC RECTOCOLITE AND PROCTITE |
| US20230061455A1 (en) * | 2017-11-01 | 2023-03-02 | Editas Medicine, Inc. | Methods, compositions and components for crispr-cas9 editing of tgfbr2 in t cells for immunotherapy |
| CN111757936B (zh) * | 2017-12-22 | 2025-07-04 | 罗氏创新中心哥本哈根有限公司 | 包含二硫代磷酸酯核苷间连接的寡核苷酸 |
| US20200332289A1 (en) * | 2017-12-22 | 2020-10-22 | Roche Innovation Center Copenhagen A/S | Gapmer oligonucleotides comprising a phosphorodithioate internucleoside linkage |
| US11597926B2 (en) * | 2017-12-22 | 2023-03-07 | Roche Innovation Center Copenhagen A/S | Thiophosphoramidites |
| MY200352A (en) | 2018-05-22 | 2023-12-21 | Ionis Pharmaceuticals Inc | Modulators of apol1 expression |
| CN112513060B (zh) * | 2018-07-31 | 2024-08-06 | 罗氏创新中心哥本哈根有限公司 | 包含三硫代磷酸酯核苷间键的寡核苷酸 |
| US20210221837A1 (en) * | 2018-07-31 | 2021-07-22 | Roche Innovation Center Copenhagen A/S | Oligonucleotides comprising a phosphorotrithioate internucleoside linkage |
| CN113490742A (zh) * | 2019-02-20 | 2021-10-08 | 罗氏创新中心哥本哈根有限公司 | 膦酰基乙酸酯缺口聚物寡核苷酸 |
| EP4237436A4 (en) * | 2020-10-29 | 2025-09-10 | Garcia Blanco Mariano A | SOLUBLE INTERLEUKIN-7 RECEPTOR (SIL7R) MODULATION THERAPY FOR CANCERS |
| CN114588160A (zh) * | 2020-12-07 | 2022-06-07 | 四川大学华西医院 | 具有抗肺纤维化作用的次黄碱衍生物 |
| CN113992687B (zh) * | 2021-12-28 | 2022-04-08 | 浙江宇视科技有限公司 | 智能业务集群调度方法、装置、电子设备及存储介质 |
| TW202442689A (zh) | 2023-03-03 | 2024-11-01 | 美商亞森諾生物科學公司 | 靶向ｐｓｍａ及ｃａ９之系統 |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL125800A0 (en) | 1996-02-19 | 1999-04-11 | Nycomed Imaging As | Thermally stabilized contrast agent |
| US20030064944A1 (en) * | 2001-06-21 | 2003-04-03 | Isis Pharmaceuticals Inc. | Antisense modulation of transforming growth factor beta receptor II expression |
| DK2752488T3 (da) | 2002-11-18 | 2020-04-20 | Roche Innovation Ct Copenhagen As | Antisense-design |
| WO2005019422A2 (en) * | 2003-08-13 | 2005-03-03 | The Board Of Trustees Of The University Of Illinois | Silencing of tgf-beta receptor type ii expression by sirna |
| NZ548926A (en) * | 2004-02-09 | 2009-07-31 | Regenion Gmbh | Inhibitors of TGF-R signaling for treatment of CNS disorders |
| WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
| ES2516815T3 (es) | 2006-01-27 | 2014-10-31 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos modificados en la posición 6 |
| KR101407707B1 (ko) | 2006-04-03 | 2014-06-19 | 산타리스 팔마 에이/에스 | Ａｎｔｉ-ｍｉｒｎａ 안티센스 올리고뉴클레오타이드를 함유하는 약학적 조성물 |
| CA2651453C (en) | 2006-05-11 | 2014-10-14 | Isis Pharmaceuticals, Inc. | 5'-modified bicyclic nucleic acid analogs |
| CN101554074B (zh) | 2006-10-30 | 2012-08-22 | 诺基亚公司 | 为用户设备提供运营商控制的移动性的方法、设备和系统 |
| WO2008109546A2 (en) * | 2007-03-02 | 2008-09-12 | Mdrna, Inc. | Nucleic acid compounds for inhibiting tgfbr gene expression and uses thereof |
| WO2008150729A2 (en) | 2007-05-30 | 2008-12-11 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| EP2173760B2 (en) | 2007-06-08 | 2015-11-04 | Isis Pharmaceuticals, Inc. | Carbocyclic bicyclic nucleic acid analogs |
| AU2008272918B2 (en) | 2007-07-05 | 2012-09-13 | Isis Pharmaceuticals, Inc. | 6-disubstituted bicyclic nucleic acid analogs |
| US8546556B2 (en) | 2007-11-21 | 2013-10-01 | Isis Pharmaceuticals, Inc | Carbocyclic alpha-L-bicyclic nucleic acid analogs |
| CN102089429A (zh) * | 2008-07-15 | 2011-06-08 | 弗·哈夫曼-拉罗切有限公司 | 用于抑制TGF-β受体基因表达的组合物和方法 |
| EP2512491B1 (en) * | 2009-10-16 | 2017-10-11 | Glaxo Group Limited | Hbv antisense inhibitors |
| EP2580326A1 (en) * | 2010-06-11 | 2013-04-17 | Antisense Pharma GmbH | Method for selective oligonucleotide modification |
| US9115371B2 (en) * | 2011-08-18 | 2015-08-25 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Reduction of TGF beta signaling in myeloid cells in the treatment of cancer |
| CN104004762B (zh) * | 2014-05-20 | 2016-03-30 | 南京医科大学附属南京儿童医院 | Tgf-r反义序列及其在制备抗气道炎症反应药物中的应用 |
| EP3020813A1 (en) * | 2014-11-16 | 2016-05-18 | Neurovision Pharma GmbH | Antisense-oligonucleotides as inhibitors of TGF-R signaling |
-
2014
- 2014-11-16 EP EP14193368.9A patent/EP3020813A1/en not_active Ceased
-
2015
- 2015-11-16 EP EP18184873.0A patent/EP3418385A1/en not_active Withdrawn
- 2015-11-16 MY MYPI2017701692A patent/MY181404A/en unknown
- 2015-11-16 ES ES15797640T patent/ES2705066T3/es active Active
- 2015-11-16 RU RU2017120481A patent/RU2717454C2/ru active
- 2015-11-16 PL PL15797640T patent/PL3218488T3/pl unknown
- 2015-11-16 KR KR1020177016366A patent/KR102310899B1/ko active Active
- 2015-11-16 WO PCT/EP2015/076730 patent/WO2016075339A1/en not_active Ceased
- 2015-11-16 EP EP15797640.8A patent/EP3218488B1/en active Active
- 2015-11-16 PT PT15797640T patent/PT3218488T/pt unknown
- 2015-11-16 CN CN201580056200.1A patent/CN107002082B/zh active Active
- 2015-11-16 US US15/525,561 patent/US11213541B2/en active Active
- 2015-11-16 CA CA3201845A patent/CA3201845C/en active Active
- 2015-11-16 CA CA2964834A patent/CA2964834C/en active Active
- 2015-11-16 TR TR2019/00844T patent/TR201900844T4/tr unknown
- 2015-11-16 KR KR1020217028947A patent/KR102417601B1/ko active Active
- 2015-11-16 MX MX2017006369A patent/MX2017006369A/es unknown
- 2015-11-16 CN CN202110048326.1A patent/CN113151261B/zh active Active
- 2015-11-16 US US14/942,899 patent/US10363266B2/en active Active
- 2015-11-16 HU HUE15797640A patent/HUE041793T2/hu unknown
- 2015-11-16 SG SG11201703127SA patent/SG11201703127SA/en unknown
- 2015-11-16 DK DK15797640.8T patent/DK3218488T3/en active
- 2015-11-16 JP JP2017544992A patent/JP6832285B2/ja active Active
- 2015-11-16 BR BR112017006201-1A patent/BR112017006201B1/pt active IP Right Grant
- 2015-11-16 AU AU2015345006A patent/AU2015345006B2/en not_active Ceased
-
2017
- 2017-04-04 IL IL251565A patent/IL251565B/en active IP Right Grant
- 2017-05-15 CL CL2017001233A patent/CL2017001233A1/es unknown
- 2017-05-16 MX MX2020011476A patent/MX2020011476A/es unknown
-
2018
- 2018-12-28 CY CY181101402T patent/CY1121110T1/el unknown
-
2019
- 2019-04-11 CL CL2019000980A patent/CL2019000980A1/es unknown
- 2019-06-14 US US16/442,054 patent/US20190314401A1/en not_active Abandoned
- 2019-09-05 IL IL269151A patent/IL269151B2/en unknown
-
2020
- 2020-10-22 AU AU2020257116A patent/AU2020257116B2/en not_active Ceased
- 2020-10-26 AU AU2020260380A patent/AU2020260380B2/en active Active
-
2021
- 2021-02-01 JP JP2021014438A patent/JP6983343B2/ja active Active
- 2021-11-08 US US17/453,950 patent/US20220143071A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018501816A5 (enExample) | ||
| JP2019524149A5 (enExample) | ||
| EP3353298B1 (en) | Allele selective gene editing and uses thereof | |
| RU2016123086A (ru) | Способы и композиции для лечения болезни хантингтона | |
| JP2020511970A5 (enExample) | ||
| SA517390168B1 (ar) | تركيبات لتعديل التعبير الوراثي عن c9orf72 | |
| JP2010515464A (ja) | Ｈｉｖ細胞表面受容体の標的化不活性化のための組成物および方法 | |
| Nakamura et al. | Exon‐skipping therapy for Duchenne muscular dystrophy | |
| JP2018513682A5 (enExample) | ||
| JP2017536338A5 (enExample) | ||
| JP2019500428A5 (enExample) | ||
| JP2016520310A5 (enExample) | ||
| US20240318180A1 (en) | Antisense oligonucleotides targeting actl6b | |
| MX2024014029A (es) | Moléculas de corte y empalme en trans abca4 | |
| Kawecka et al. | Adeno-associated virus (AAV) mediated dystrophin gene transfer studies and exon skipping strategies for Duchenne muscular dystrophy (DMD) | |
| Choi et al. | Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington’s disease | |
| JP2025500335A (ja) | Ｕｎｃ１３ａを標的とするアンチセンスオリゴヌクレオチド | |
| RU2020125769A (ru) | Олигонуклеотиды для модуляции экспрессии tmem106b | |
| US20240344065A1 (en) | Method | |
| US12104153B2 (en) | Antisense oligonucleotide for targeting progranulin | |
| US20220031730A1 (en) | Enhanced oligonucleotides for modulating fubp1 expression | |
| US20250290068A2 (en) | Improved oligonucleotides targeting rna binding protein sites | |
| US20240182898A1 (en) | ENHANCED OLIGONUCLEOTIDES FOR INHIBITING RTELl EXPRESSION | |
| US20250361507A2 (en) | Antisense oligonucleotides for targeting progranulin | |
| US20210261961A1 (en) | Antisense oligonucleotides targeting tia1 |