KR20240068067A - Pcdh19 관련 장애의 치료를 위한 조성물 및 방법 - Google Patents
Pcdh19 관련 장애의 치료를 위한 조성물 및 방법 Download PDFInfo
- Publication number
- KR20240068067A KR20240068067A KR1020247014017A KR20247014017A KR20240068067A KR 20240068067 A KR20240068067 A KR 20240068067A KR 1020247014017 A KR1020247014017 A KR 1020247014017A KR 20247014017 A KR20247014017 A KR 20247014017A KR 20240068067 A KR20240068067 A KR 20240068067A
- Authority
- KR
- South Korea
- Prior art keywords
- oligonucleotide
- pcdh19
- quot
- oligonucleotides
- nucleosides
- 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
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/08—Antiepileptics; Anticonvulsants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Pain & Pain Management (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Psychiatry (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163248803P | 2021-09-27 | 2021-09-27 | |
| US63/248,803 | 2021-09-27 | ||
| US202263333840P | 2022-04-22 | 2022-04-22 | |
| US63/333,840 | 2022-04-22 | ||
| PCT/AU2022/051153 WO2023044545A1 (en) | 2021-09-27 | 2022-09-27 | Compositions and methods for the treatment of pcdh19 related disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20240068067A true KR20240068067A (ko) | 2024-05-17 |
Family
ID=85719092
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020247014017A Pending KR20240068067A (ko) | 2021-09-27 | 2022-09-27 | Pcdh19 관련 장애의 치료를 위한 조성물 및 방법 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20250002912A1 (https=) |
| EP (1) | EP4408532A4 (https=) |
| JP (1) | JP2024535432A (https=) |
| KR (1) | KR20240068067A (https=) |
| AU (1) | AU2022351990A1 (https=) |
| CA (1) | CA3232862A1 (https=) |
| CL (1) | CL2024000853A1 (https=) |
| CO (1) | CO2024004737A2 (https=) |
| IL (1) | IL311581A (https=) |
| MX (1) | MX2024003947A (https=) |
| PE (1) | PE20250738A1 (https=) |
| WO (1) | WO2023044545A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024206668A1 (en) * | 2023-03-31 | 2024-10-03 | The Board Of Trustees Of The Leland Stanford Junior University | Therapeutic strategy for pcdh19-related encephalopathy |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP5624892B2 (ja) * | 2008-01-04 | 2014-11-12 | セントラル アデレード ローカル ヘルス ネットワーク インコーポレイテッド | Ｅｆｍｒ（女性に限られた癲癇及び精神遅滞）の診断方法及び治療方法 |
| EP3118326B1 (en) * | 2008-10-27 | 2020-04-29 | Sapporo Medical University | Molecular marker for cancer stem cell |
| US9562892B2 (en) * | 2012-10-02 | 2017-02-07 | New York University | Methods and agents for modulating protocadherin-18 activity |
| WO2020191233A1 (en) * | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
-
2022
- 2022-09-27 EP EP22871200.6A patent/EP4408532A4/en active Pending
- 2022-09-27 KR KR1020247014017A patent/KR20240068067A/ko active Pending
- 2022-09-27 US US18/694,295 patent/US20250002912A1/en active Pending
- 2022-09-27 IL IL311581A patent/IL311581A/en unknown
- 2022-09-27 CA CA3232862A patent/CA3232862A1/en active Pending
- 2022-09-27 AU AU2022351990A patent/AU2022351990A1/en active Pending
- 2022-09-27 PE PE2024000576A patent/PE20250738A1/es unknown
- 2022-09-27 JP JP2024519062A patent/JP2024535432A/ja active Pending
- 2022-09-27 MX MX2024003947A patent/MX2024003947A/es unknown
- 2022-09-27 WO PCT/AU2022/051153 patent/WO2023044545A1/en not_active Ceased
-
2024
- 2024-03-22 CL CL2024000853A patent/CL2024000853A1/es unknown
- 2024-04-16 CO CONC2024/0004737A patent/CO2024004737A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP4408532A1 (en) | 2024-08-07 |
| PE20250738A1 (es) | 2025-03-10 |
| MX2024003947A (es) | 2024-04-24 |
| AU2022351990A1 (en) | 2024-05-16 |
| IL311581A (en) | 2024-05-01 |
| JP2024535432A (ja) | 2024-09-30 |
| CA3232862A1 (en) | 2023-03-30 |
| CL2024000853A1 (es) | 2024-10-04 |
| EP4408532A4 (en) | 2026-01-21 |
| US20250002912A1 (en) | 2025-01-02 |
| WO2023044545A1 (en) | 2023-03-30 |
| CO2024004737A2 (es) | 2024-04-29 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
St.27 status event code: A-0-1-A10-A15-nap-PA0105 |
|
| PG1501 | Laying open of application |
St.27 status event code: A-1-1-Q10-Q12-nap-PG1501 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| P11-X000 | Amendment of application requested |
St.27 status event code: A-2-2-P10-P11-nap-X000 |
|
| PN2301 | Change of applicant |
St.27 status event code: A-3-3-R10-R11-asn-PN2301 |
|
| R11 | Change to the name of applicant or owner or transfer of ownership requested |
Free format text: ST27 STATUS EVENT CODE: A-3-3-R10-R11-ASN-PN2301 (AS PROVIDED BY THE NATIONAL OFFICE) |