JPWO2022169947A5 - - Google Patents
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- Publication number
- JPWO2022169947A5 JPWO2022169947A5 JP2023547237A JP2023547237A JPWO2022169947A5 JP WO2022169947 A5 JPWO2022169947 A5 JP WO2022169947A5 JP 2023547237 A JP2023547237 A JP 2023547237A JP 2023547237 A JP2023547237 A JP 2023547237A JP WO2022169947 A5 JPWO2022169947 A5 JP WO2022169947A5
- Authority
- JP
- Japan
- Prior art keywords
- mrna
- nucleobases
- nucleotides
- agent
- 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.)
- Pending
Links
- 108020004999 messenger RNA Proteins 0.000 claims description 196
- 239000003795 chemical substances by application Substances 0.000 claims description 104
- 108090000623 proteins and genes Proteins 0.000 claims description 85
- 102000004169 proteins and genes Human genes 0.000 claims description 65
- 239000000203 mixture Substances 0.000 claims description 51
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 48
- 239000002773 nucleotide Substances 0.000 claims description 48
- 125000003729 nucleotide group Chemical group 0.000 claims description 48
- 230000008685 targeting Effects 0.000 claims description 48
- 102100036142 Polycystin-2 Human genes 0.000 claims description 43
- 230000001105 regulatory effect Effects 0.000 claims description 39
- 201000010099 disease Diseases 0.000 claims description 38
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 38
- 101710146368 Polycystin-2 Proteins 0.000 claims description 36
- 230000014509 gene expression Effects 0.000 claims description 36
- 150000007523 nucleic acids Chemical class 0.000 claims description 35
- 102000039446 nucleic acids Human genes 0.000 claims description 29
- 108020004707 nucleic acids Proteins 0.000 claims description 29
- 239000008194 pharmaceutical composition Substances 0.000 claims description 29
- 239000003814 drug Substances 0.000 claims description 28
- 230000000692 anti-sense effect Effects 0.000 claims description 22
- 230000001404 mediated effect Effects 0.000 claims description 21
- 230000001965 increasing effect Effects 0.000 claims description 20
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 19
- 229940079593 drug Drugs 0.000 claims description 19
- 239000013598 vector Substances 0.000 claims description 19
- 108700028369 Alleles Proteins 0.000 claims description 16
- 238000011144 upstream manufacturing Methods 0.000 claims description 15
- 239000013603 viral vector Substances 0.000 claims description 14
- 101150112863 pkd2 gene Proteins 0.000 claims description 13
- 230000035772 mutation Effects 0.000 claims description 12
- 108020004485 Nonsense Codon Proteins 0.000 claims description 8
- 102000039471 Small Nuclear RNA Human genes 0.000 claims description 8
- 108020004688 Small Nuclear RNA Proteins 0.000 claims description 8
- 230000007812 deficiency Effects 0.000 claims description 8
- 230000000694 effects Effects 0.000 claims description 8
- 108091033319 polynucleotide Proteins 0.000 claims description 8
- 102000040430 polynucleotide Human genes 0.000 claims description 8
- 101001074439 Homo sapiens Polycystin-2 Proteins 0.000 claims description 7
- 101001026882 Homo sapiens Serine/threonine-protein kinase D2 Proteins 0.000 claims description 7
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 6
- 239000002157 polynucleotide Substances 0.000 claims description 6
- 108091026823 U7 small nuclear RNA Proteins 0.000 claims description 5
- 230000000295 complement effect Effects 0.000 claims description 5
- 108020003589 5' Untranslated Regions Proteins 0.000 claims description 4
- 208000010061 Autosomal Dominant Polycystic Kidney Diseases 0.000 claims description 4
- 101100029888 Homo sapiens PKD1 gene Proteins 0.000 claims description 4
- 201000008450 Intracranial aneurysm Diseases 0.000 claims description 4
- 101150056230 PKD1 gene Proteins 0.000 claims description 4
- 208000022185 autosomal dominant polycystic kidney disease Diseases 0.000 claims description 4
- 230000001939 inductive effect Effects 0.000 claims description 4
- 208000030761 polycystic kidney disease Diseases 0.000 claims description 4
- 108700024394 Exon Proteins 0.000 claims description 3
- 108091093037 Peptide nucleic acid Proteins 0.000 claims description 3
- 101710146367 Polycystin-1 Proteins 0.000 claims description 3
- 102100036143 Polycystin-1 Human genes 0.000 claims description 3
- 108091026838 U1 spliceosomal RNA Proteins 0.000 claims description 3
- 238000012986 modification Methods 0.000 claims description 3
- 230000004048 modification 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
- ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims description 2
- 210000004185 liver Anatomy 0.000 claims description 2
- 230000001737 promoting effect Effects 0.000 claims description 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 154
- 238000002347 injection Methods 0.000 description 8
- 239000007924 injection Substances 0.000 description 8
- 229940124597 therapeutic agent Drugs 0.000 description 8
- 108091028043 Nucleic acid sequence Proteins 0.000 description 6
- 230000003993 interaction Effects 0.000 description 4
- 238000007913 intrathecal administration Methods 0.000 description 4
- 238000010253 intravenous injection Methods 0.000 description 4
- 241000701161 unidentified adenovirus Species 0.000 description 4
- 238000002513 implantation Methods 0.000 description 3
- 238000007914 intraventricular administration Methods 0.000 description 3
- 108700020942 nucleic acid binding protein Proteins 0.000 description 3
- 102000044158 nucleic acid binding protein Human genes 0.000 description 3
- 229920001184 polypeptide Polymers 0.000 description 3
- 108090000765 processed proteins & peptides Proteins 0.000 description 3
- 102000004196 processed proteins & peptides Human genes 0.000 description 3
- 230000000699 topical effect Effects 0.000 description 3
- 108091081024 Start codon Proteins 0.000 description 2
- 208000021033 autosomal dominant polycystic liver disease Diseases 0.000 description 2
- 230000007717 exclusion Effects 0.000 description 2
- 210000003754 fetus Anatomy 0.000 description 2
- 238000007918 intramuscular administration Methods 0.000 description 2
- 238000010255 intramuscular injection Methods 0.000 description 2
- 239000007927 intramuscular injection Substances 0.000 description 2
- 238000007912 intraperitoneal administration Methods 0.000 description 2
- 239000007928 intraperitoneal injection Substances 0.000 description 2
- 210000001161 mammalian embryo Anatomy 0.000 description 2
- 208000028589 polycystic liver disease Diseases 0.000 description 2
- 150000003384 small molecules Chemical group 0.000 description 2
- 238000007920 subcutaneous administration Methods 0.000 description 2
- 238000010254 subcutaneous injection Methods 0.000 description 2
- 239000007929 subcutaneous injection Substances 0.000 description 2
- 230000003612 virological effect Effects 0.000 description 2
- 102000001708 Protein Isoforms Human genes 0.000 description 1
- 108010029485 Protein Isoforms Proteins 0.000 description 1
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 description 1
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 description 1
- 230000008030 elimination Effects 0.000 description 1
- 238000003379 elimination reaction Methods 0.000 description 1
- 238000000185 intracerebroventricular administration Methods 0.000 description 1
- 230000004777 loss-of-function mutation Effects 0.000 description 1
- 230000014759 maintenance of location Effects 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 210000000056 organ Anatomy 0.000 description 1
- 239000011701 zinc Substances 0.000 description 1
- 229910052725 zinc Inorganic materials 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163145288P | 2021-02-03 | 2021-02-03 | |
| US63/145,288 | 2021-02-03 | ||
| PCT/US2022/015074 WO2022169947A2 (en) | 2021-02-03 | 2022-02-03 | Compositions for treatment of conditions and diseases associated with polycystin expression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024507717A JP2024507717A (ja) | 2024-02-21 |
| JPWO2022169947A5 true JPWO2022169947A5 (enrdf_load_stackoverflow) | 2025-02-12 |
Family
ID=82741748
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023547237A Pending JP2024507717A (ja) | 2021-02-03 | 2022-02-03 | ポリシスチン発現に関連する状態及び疾患の治療のための組成物 |
Country Status (12)
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024097138A1 (en) * | 2022-10-31 | 2024-05-10 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6916619B2 (en) * | 2001-10-12 | 2005-07-12 | Athena Diagnostics, Inc. | Compositions and methods for genetic analysis of polycystic kidney disease |
| US11096956B2 (en) * | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| SMT202500127T1 (it) * | 2017-10-23 | 2025-05-12 | Stoke Therapeutics Inc | Oligomeri antisenso per il trattamento di malattie e condizioni basate sul decadimento dell’rna mediato da un nonsenso |
-
2022
- 2022-02-03 WO PCT/US2022/015074 patent/WO2022169947A2/en active Application Filing
- 2022-02-03 KR KR1020237029596A patent/KR20230150973A/ko active Pending
- 2022-02-03 BR BR112023015636A patent/BR112023015636A2/pt unknown
- 2022-02-03 AR ARP220100222A patent/AR124809A1/es unknown
- 2022-02-03 AU AU2022215577A patent/AU2022215577A1/en active Pending
- 2022-02-03 CN CN202280019548.3A patent/CN117413061A/zh active Pending
- 2022-02-03 EP EP22750375.2A patent/EP4288543A2/en active Pending
- 2022-02-03 CA CA3207341A patent/CA3207341A1/en active Pending
- 2022-02-03 MX MX2023009151A patent/MX2023009151A/es unknown
- 2022-02-03 JP JP2023547237A patent/JP2024507717A/ja active Pending
- 2022-02-07 TW TW111104414A patent/TW202242113A/zh unknown
-
2023
- 2023-08-02 US US18/364,244 patent/US20240117353A1/en active Pending
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