JP2019500346A5 - - Google Patents
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- Publication number
- JP2019500346A5 JP2019500346A5 JP2018529237A JP2018529237A JP2019500346A5 JP 2019500346 A5 JP2019500346 A5 JP 2019500346A5 JP 2018529237 A JP2018529237 A JP 2018529237A JP 2018529237 A JP2018529237 A JP 2018529237A JP 2019500346 A5 JP2019500346 A5 JP 2019500346A5
- Authority
- JP
- Japan
- Prior art keywords
- mrna
- peroxisome proliferator
- activated receptor
- seq
- pharmaceutical composition
- 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
- 108020004999 messenger RNA Proteins 0.000 claims description 136
- 108090000623 proteins and genes Proteins 0.000 claims description 113
- 230000000717 retained effect Effects 0.000 claims description 96
- 102000004169 proteins and genes Human genes 0.000 claims description 93
- 238000000034 method Methods 0.000 claims description 89
- 239000000203 mixture Substances 0.000 claims description 61
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 52
- 230000000692 anti-sense effect Effects 0.000 claims description 51
- 102100038824 Peroxisome proliferator-activated receptor delta Human genes 0.000 claims description 48
- 239000008194 pharmaceutical composition Substances 0.000 claims description 35
- 108700028369 Alleles Proteins 0.000 claims description 30
- 108091008765 peroxisome proliferator-activated receptors β/δ Proteins 0.000 claims description 26
- 210000004027 cell Anatomy 0.000 claims description 21
- 102000039446 nucleic acids Human genes 0.000 claims description 17
- 108020004707 nucleic acids Proteins 0.000 claims description 17
- 150000007523 nucleic acids Chemical group 0.000 claims description 17
- 230000000295 complement effect Effects 0.000 claims description 14
- 208000017169 kidney disease Diseases 0.000 claims description 12
- 230000001965 increasing effect Effects 0.000 claims description 11
- 108091092195 Intron Proteins 0.000 claims description 10
- 230000002950 deficient Effects 0.000 claims description 10
- 230000000694 effects Effects 0.000 claims description 8
- 230000007812 deficiency Effects 0.000 claims description 6
- 238000010255 intramuscular injection Methods 0.000 claims description 5
- 238000010253 intravenous injection Methods 0.000 claims description 5
- 238000004519 manufacturing process Methods 0.000 claims description 5
- 230000004048 modification Effects 0.000 claims description 5
- 238000012986 modification Methods 0.000 claims description 5
- 238000010254 subcutaneous injection Methods 0.000 claims description 5
- 108091093037 Peptide nucleic acid Proteins 0.000 claims description 4
- ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims description 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 4
- 238000007912 intraperitoneal administration Methods 0.000 claims description 2
- 238000007913 intrathecal administration Methods 0.000 claims description 2
- 238000007914 intraventricular administration Methods 0.000 claims description 2
- 208000020832 chronic kidney disease Diseases 0.000 claims 4
- 239000003814 drug Substances 0.000 claims 2
- 201000000523 end stage renal failure Diseases 0.000 claims 2
- 208000024172 Cardiovascular disease Diseases 0.000 claims 1
- 208000028208 end stage renal disease Diseases 0.000 claims 1
- 230000037356 lipid metabolism Effects 0.000 claims 1
- 239000003614 peroxisome proliferator Substances 0.000 claims 1
- 102000005962 receptors Human genes 0.000 claims 1
- 108020003175 receptors Proteins 0.000 claims 1
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 37
- 101000613577 Homo sapiens Paired box protein Pax-2 Proteins 0.000 description 26
- 108010015181 PPAR delta Proteins 0.000 description 26
- 102100040852 Paired box protein Pax-2 Human genes 0.000 description 26
- 108010026102 Vitamin D3 24-Hydroxylase Proteins 0.000 description 26
- 102000013387 Vitamin D3 24-Hydroxylase Human genes 0.000 description 26
- 102100031089 Cystinosin Human genes 0.000 description 24
- 101000922034 Homo sapiens Cystinosin Proteins 0.000 description 24
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 15
- 201000010099 disease Diseases 0.000 description 10
- 239000002773 nucleotide Substances 0.000 description 9
- 125000003729 nucleotide group Chemical group 0.000 description 9
- 206010011777 Cystinosis Diseases 0.000 description 8
- 102000011138 Cytochrome P450 Family 24 Human genes 0.000 description 5
- 108010037808 Cytochrome P450 Family 24 Proteins 0.000 description 5
- 108010028842 PAX2 Transcription Factor Proteins 0.000 description 5
- 102000016907 PAX2 Transcription Factor Human genes 0.000 description 5
- 229920001184 polypeptide Polymers 0.000 description 5
- 108090000765 processed proteins & peptides Proteins 0.000 description 5
- 102000004196 processed proteins & peptides Human genes 0.000 description 5
- -1 subfamily A Proteins 0.000 description 5
- 208000037147 Hypercalcaemia Diseases 0.000 description 4
- 108091027974 Mature messenger RNA Proteins 0.000 description 4
- 208000035475 disorder Diseases 0.000 description 4
- 201000001126 focal segmental glomerulosclerosis 7 Diseases 0.000 description 4
- 230000000148 hypercalcaemia Effects 0.000 description 4
- 208000030915 hypercalcemia disease Diseases 0.000 description 4
- 210000003734 kidney Anatomy 0.000 description 4
- 208000011580 syndromic disease Diseases 0.000 description 4
- 239000007927 intramuscular injection Substances 0.000 description 3
- 239000007928 intraperitoneal injection Substances 0.000 description 3
- 239000000546 pharmaceutical excipient Substances 0.000 description 3
- 239000002243 precursor Substances 0.000 description 3
- 239000007929 subcutaneous injection Substances 0.000 description 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 2
- 108091034117 Oligonucleotide Proteins 0.000 description 2
- 230000001594 aberrant effect Effects 0.000 description 2
- 239000000074 antisense oligonucleotide Substances 0.000 description 2
- 238000012230 antisense oligonucleotides Methods 0.000 description 2
- 230000001939 inductive effect Effects 0.000 description 2
- 230000035772 mutation Effects 0.000 description 2
- 238000011144 upstream manufacturing Methods 0.000 description 2
- 108700024394 Exon Proteins 0.000 description 1
- 239000002253 acid Substances 0.000 description 1
- 230000001447 compensatory effect Effects 0.000 description 1
- 210000003754 fetus Anatomy 0.000 description 1
- 230000014759 maintenance of location Effects 0.000 description 1
- 210000001161 mammalian embryo Anatomy 0.000 description 1
- 230000016434 protein splicing Effects 0.000 description 1
- 229920002477 rna polymer Polymers 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022013876A JP2022062143A (ja) | 2015-12-14 | 2022-02-01 | 腎臓病の処置のための組成物と方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267242P | 2015-12-14 | 2015-12-14 | |
| US62/267,242 | 2015-12-14 | ||
| PCT/US2016/066576 WO2017106292A1 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of kidney diseases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022013876A Division JP2022062143A (ja) | 2015-12-14 | 2022-02-01 | 腎臓病の処置のための組成物と方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019500346A JP2019500346A (ja) | 2019-01-10 |
| JP2019500346A5 true JP2019500346A5 (enExample) | 2020-01-30 |
| JP7049247B2 JP7049247B2 (ja) | 2022-04-06 |
Family
ID=59057520
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018529237A Active JP7049247B2 (ja) | 2015-12-14 | 2016-12-14 | 腎臓病の処置のための組成物と方法 |
| JP2022013876A Pending JP2022062143A (ja) | 2015-12-14 | 2022-02-01 | 腎臓病の処置のための組成物と方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022013876A Pending JP2022062143A (ja) | 2015-12-14 | 2022-02-01 | 腎臓病の処置のための組成物と方法 |
Country Status (4)
| Country | Link |
|---|---|
| EP (1) | EP3390666A4 (enExample) |
| JP (2) | JP7049247B2 (enExample) |
| CA (1) | CA3005249A1 (enExample) |
| WO (1) | WO2017106292A1 (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| 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 |
| EP3359685A1 (en) | 2015-10-09 | 2018-08-15 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR102604132B1 (ko) | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| KR102712656B1 (ko) | 2017-01-23 | 2024-10-04 | 리제너론 파마슈티칼스 인코포레이티드 | Hsd17b13 변종 및 이것의 용도 |
| US11479802B2 (en) | 2017-04-11 | 2022-10-25 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxy steroid (17-beta) dehydrogenase (HSD17B) family |
| SI3673080T1 (sl) | 2017-08-25 | 2024-03-29 | Stoke Therapeutics, Inc. | Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni |
| AU2018348195B2 (en) | 2017-10-11 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| BR112020018758A2 (pt) | 2018-03-21 | 2021-01-26 | Regeneron Pharmaceuticals, Inc. | agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios |
| BR112020022512A2 (pt) | 2018-05-04 | 2021-05-04 | Stoke Therapeutics, Inc. | métodos e composições para tratamento de doença de armazenamento de éster de colesteril |
| EP3802829A4 (en) * | 2018-06-08 | 2022-10-19 | University of Massachusetts | ANTISENSE OLIGONUCLEOTIDES TO RESTORE DYSFERLIN PROTEIN EXPRESSION IN CELLS FROM PATIENTS WITH DYSFERLINOPATHY |
| US12422440B2 (en) | 2018-10-05 | 2025-09-23 | Seattle Children's Hospital | Newborn screening for primary immunodeficiencies, cystinosis, and Wilson disease |
| AU2020210924A1 (en) * | 2019-01-23 | 2021-09-16 | The Florey Institute Of Neuroscience And Mental Health | Antisense oligonucleotides targeting SCN2A retained introns |
| CN114746550A (zh) * | 2019-08-19 | 2022-07-12 | 斯托克制药公司 | 用于调节剪接和蛋白质表达的组合物和方法 |
| US11940448B2 (en) | 2020-03-31 | 2024-03-26 | Seattle Children's Hospital | Proteomic screening for lysosomal storage diseases |
| AU2021270720A1 (en) | 2020-05-11 | 2022-12-08 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030224514A1 (en) * | 2002-05-31 | 2003-12-04 | Isis Pharmaceuticals Inc. | Antisense modulation of PPAR-delta expression |
| JP2008520244A (ja) * | 2004-11-19 | 2008-06-19 | アカディア ファーマシューティカルズ インコーポレイテッド | ホルモン核内受容体のリガンドの同定方法 |
| US20110269735A1 (en) * | 2010-04-19 | 2011-11-03 | Celera Corporation | Genetic polymorphisms associated with statin response and cardiovascular diseases, methods of detection and uses thereof |
| US20140128449A1 (en) * | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
| US9909128B2 (en) * | 2012-11-15 | 2018-03-06 | The Regents Of The University Of California | Splice modulating oligonucleotides that inhibit cancer |
| CA2930859C (en) * | 2013-09-04 | 2022-05-03 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
| EP3201339A4 (en) * | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
-
2016
- 2016-12-14 EP EP16876548.5A patent/EP3390666A4/en not_active Withdrawn
- 2016-12-14 JP JP2018529237A patent/JP7049247B2/ja active Active
- 2016-12-14 CA CA3005249A patent/CA3005249A1/en active Pending
- 2016-12-14 WO PCT/US2016/066576 patent/WO2017106292A1/en not_active Ceased
-
2022
- 2022-02-01 JP JP2022013876A patent/JP2022062143A/ja active Pending
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