KR20240035503A - 석출 억제제 - Google Patents
석출 억제제 Download PDFInfo
- Publication number
- KR20240035503A KR20240035503A KR1020247003689A KR20247003689A KR20240035503A KR 20240035503 A KR20240035503 A KR 20240035503A KR 1020247003689 A KR1020247003689 A KR 1020247003689A KR 20247003689 A KR20247003689 A KR 20247003689A KR 20240035503 A KR20240035503 A KR 20240035503A
- Authority
- KR
- South Korea
- Prior art keywords
- precipitation
- sugar
- antisense oligomer
- pharmaceutical composition
- antisense
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
-
- 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
-
- 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
- A61K31/7016—Disaccharides, e.g. lactose, lactulose
-
- 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
- A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
- A61K31/047—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates having two or more hydroxy groups, e.g. sorbitol
-
- 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
- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- 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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- 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
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- C12N2310/314—Phosphoramidates
-
- 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
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Urology & Nephrology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021113562 | 2021-07-08 | ||
| JPJP-P-2021-113562 | 2021-07-08 | ||
| PCT/JP2022/027105 WO2023282346A1 (ja) | 2021-07-08 | 2022-07-08 | 析出抑制剤 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20240035503A true KR20240035503A (ko) | 2024-03-15 |
Family
ID=84800729
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020247003689A Pending KR20240035503A (ko) | 2021-07-08 | 2022-07-08 | 석출 억제제 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20240285770A1 (https=) |
| EP (1) | EP4368187A4 (https=) |
| JP (3) | JPWO2023282346A1 (https=) |
| KR (1) | KR20240035503A (https=) |
| CN (1) | CN118434424A (https=) |
| AU (1) | AU2022306542A1 (https=) |
| CA (1) | CA3225573A1 (https=) |
| IL (1) | IL310001A (https=) |
| TW (1) | TW202317147A (https=) |
| WO (1) | WO2023282346A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN118475355A (zh) * | 2021-07-08 | 2024-08-09 | 日本新药株式会社 | 肾毒性减轻剂 |
| CA3225573A1 (en) * | 2021-07-08 | 2023-01-12 | Nippon Shinyaku Co., Ltd. | Precipitation suppressing agent |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2002036767A2 (en) | 2000-11-02 | 2002-05-10 | Inex Pharmaceuticals Corporation | Therapeutic oligonucleotides of reduced toxicity |
| WO2010009038A2 (en) | 2008-07-18 | 2010-01-21 | Oncogenex Technologies Inc. | Antisense formulation |
| JP2015091229A (ja) | 2013-10-04 | 2015-05-14 | 国立大学法人神戸大学 | 福山型筋ジストロフィー治療用アンチセンス核酸 |
Family Cites Families (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU655164B2 (en) | 1989-12-20 | 1994-12-08 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| WO2006038608A1 (ja) | 2004-10-05 | 2006-04-13 | Nippon Shinyaku Co., Ltd. | オリゴ二本鎖rna及び医薬組成物 |
| JP2008520600A (ja) * | 2004-11-19 | 2008-06-19 | ノヴォソム アクチェンゲゼルシャフト | 局所投与のための医薬組成物におけるまたはそれに関する改善 |
| EP1886688A4 (en) | 2005-05-30 | 2013-01-09 | Nippon Shinyaku Co Ltd | METHOD FOR PRODUCING PREPARATION OF A NUCLEIC ACID-CONTAINING COMPLEX |
| PL2735568T3 (pl) | 2006-05-10 | 2018-02-28 | Sarepta Therapeutics, Inc. | Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami |
| CN1868456A (zh) * | 2006-06-25 | 2006-11-29 | 沈阳药科大学 | 反义脱氧寡核苷酸isis23665脂质体制剂及制备方法 |
| WO2009005793A2 (en) | 2007-06-29 | 2009-01-08 | Avi Biopharma, Inc. | Tissue specific peptide conjugates and methods |
| RU2606627C2 (ru) | 2007-11-15 | 2017-01-10 | Серепта Терапьютикс,Инк. | Способ синтеза морфолиновых олигомеров |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| CA3092114A1 (en) | 2011-05-05 | 2012-11-08 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| CN102989007B (zh) * | 2011-06-29 | 2015-12-16 | 湖州市中心医院 | 大规模制备注射用中期因子反义寡核苷酸纳米脂质体的方法 |
| EP3730618A1 (en) | 2011-11-18 | 2020-10-28 | Alnylam Pharmaceuticals, Inc. | Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases |
| CA2861247C (en) | 2011-12-28 | 2021-11-16 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acids |
| DK2992098T3 (da) | 2013-05-01 | 2019-06-17 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression |
| WO2015006740A2 (en) | 2013-07-11 | 2015-01-15 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide-ligand conjugates and process for their preparation |
| BR112016013516A8 (pt) | 2013-12-12 | 2020-05-19 | Life Technologies Corp | complexo de transfecção e seu uso para aumentar transporte de molécula de carga em células e para o tratamento de câncer e terapia gênica, bem como método in vitro para fornecer um poliânion a uma célula |
| JP6482475B2 (ja) | 2014-01-07 | 2019-03-13 | レナセラピューティクス株式会社 | アンチセンスオリゴヌクレオチド及び糖誘導体を含む二本鎖オリゴヌクレオチド |
| AU2016264471B2 (en) | 2015-05-19 | 2022-01-27 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| WO2017010575A1 (ja) | 2015-07-16 | 2017-01-19 | 協和発酵キリン株式会社 | β2GPI遺伝子発現抑制核酸複合体 |
| KR102810425B1 (ko) | 2016-12-19 | 2025-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| PT3554552T (pt) | 2016-12-19 | 2022-10-03 | Sarepta Therapeutics Inc | Conjugados de oligómero de skipping de exão para distrofia muscular |
| KR20240006023A (ko) | 2016-12-19 | 2024-01-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| CA3225573A1 (en) * | 2021-07-08 | 2023-01-12 | Nippon Shinyaku Co., Ltd. | Precipitation suppressing agent |
| CN118475355A (zh) * | 2021-07-08 | 2024-08-09 | 日本新药株式会社 | 肾毒性减轻剂 |
-
2022
- 2022-07-08 CA CA3225573A patent/CA3225573A1/en active Pending
- 2022-07-08 JP JP2022555691A patent/JPWO2023282346A1/ja not_active Withdrawn
- 2022-07-08 IL IL310001A patent/IL310001A/en unknown
- 2022-07-08 AU AU2022306542A patent/AU2022306542A1/en active Pending
- 2022-07-08 US US18/577,142 patent/US20240285770A1/en active Pending
- 2022-07-08 CN CN202280047891.9A patent/CN118434424A/zh active Pending
- 2022-07-08 KR KR1020247003689A patent/KR20240035503A/ko active Pending
- 2022-07-08 WO PCT/JP2022/027105 patent/WO2023282346A1/ja not_active Ceased
- 2022-07-08 TW TW111125775A patent/TW202317147A/zh unknown
- 2022-07-08 EP EP22837760.2A patent/EP4368187A4/en active Pending
- 2022-11-07 JP JP2022178017A patent/JP7466609B2/ja active Active
-
2025
- 2025-07-29 JP JP2025126561A patent/JP2025142303A/ja active Pending
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2002036767A2 (en) | 2000-11-02 | 2002-05-10 | Inex Pharmaceuticals Corporation | Therapeutic oligonucleotides of reduced toxicity |
| WO2010009038A2 (en) | 2008-07-18 | 2010-01-21 | Oncogenex Technologies Inc. | Antisense formulation |
| JP2015091229A (ja) | 2013-10-04 | 2015-05-14 | 国立大学法人神戸大学 | 福山型筋ジストロフィー治療用アンチセンス核酸 |
Non-Patent Citations (3)
| Title |
|---|
| Carver MP, Charleston JS, Shanks C, Zhang J, Mense M, Sharma AK, Kaur H, Sazani P. Toxicological Characterization of Exon Skipping Phosphorodiamidate Morpholino Oligomers (PMOs) in Non-human Primates. J Neuromuscul Dis. 2016 Aug 30;3(3):381-393. doi:10.3233/JND-160157. |
| Heemskerk HA, de Winter CL, de Kimpe SJ, van Kuik-Romeijn P, Heuvelmans N, Platenburg GJ, van Ommen GJ, van Deutekom JC, Aartsma-Rus A. In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping. J Gene Med. 2009 Mar;11(3):257-66. doi:10.1002/jgm.1288. |
| Sazani P, Ness KP, Weller DL, Poage D, Nelson K, Shrewsbury AS. Chemical and mechanistic toxicology evaluation of exon skipping phosphorodiamidate morpholino oligomers in mdx mice. Int J Toxicol. 2011 May;30(3):322-33. doi:10.1177/1091581811403504. Epub 2011 May 3. |
Also Published As
| Publication number | Publication date |
|---|---|
| EP4368187A1 (en) | 2024-05-15 |
| JP7466609B2 (ja) | 2024-04-12 |
| EP4368187A4 (en) | 2025-07-02 |
| TW202317147A (zh) | 2023-05-01 |
| JP2023133099A (ja) | 2023-09-22 |
| JPWO2023282346A1 (https=) | 2023-01-12 |
| AU2022306542A1 (en) | 2024-01-18 |
| CA3225573A1 (en) | 2023-01-12 |
| IL310001A (en) | 2024-03-01 |
| WO2023282346A8 (ja) | 2024-02-15 |
| WO2023282346A1 (ja) | 2023-01-12 |
| CN118434424A (zh) | 2024-08-02 |
| JP2025142303A (ja) | 2025-09-30 |
| US20240285770A1 (en) | 2024-08-29 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2025142303A (ja) | 析出抑制剤 | |
| JP2025142302A (ja) | 腎毒性軽減剤 | |
| US12595477B2 (en) | Complement Factor B-modulating compositions and methods of use thereof | |
| JP2022551269A5 (https=) | ||
| US20240401045A1 (en) | Angiotensinogen-modulating compositions and methods of use thereof | |
| JP2025142304A (ja) | 腎毒性軽減剤 | |
| US20240041913A1 (en) | Angiotensinogen-modulating compositions and methods of use thereof | |
| HK40104301A (en) | Precipitation suppressing agent | |
| HK40103946A (en) | Nephrotoxicity reducing agent | |
| AU2024301682A1 (en) | Coagulation factor xi-modulating compositions and methods of use thereof | |
| HK40107109A (zh) | 肾毒性减轻剂 | |
| HK40107255A (zh) | 析出抑制剂 | |
| US12042509B2 (en) | Prekallikrein-modulating compositions and methods of use thereof | |
| TW202346589A (zh) | 抗病毒反義寡聚物 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
Patent event date: 20240131 Patent event code: PA01051R01D Comment text: International Patent Application |
|
| PG1501 | Laying open of application | ||
| A201 | Request for examination | ||
| PA0201 | Request for examination |
Patent event code: PA02012R01D Patent event date: 20250701 Comment text: Request for Examination of Application |