CN118355119A - 反义寡核苷酸（aso）与内源性作用于rna的腺苷脱氨酶（adar）用于高效精确rna编辑 - Google Patents
反义寡核苷酸（aso）与内源性作用于rna的腺苷脱氨酶（adar）用于高效精确rna编辑 Download PDFInfo
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- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- 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
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Epidemiology (AREA)
- Saccharide Compounds (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP21211372.4 | 2021-11-30 | ||
| EP21211372 | 2021-11-30 | ||
| PCT/EP2022/083711 WO2023099494A1 (en) | 2021-11-30 | 2022-11-29 | Antisense oligonucleotides (aso) for efficient and precise rna editing with endogenous adenosine deaminase acting on rna (adar) |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN118355119A true CN118355119A (zh) | 2024-07-16 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202280079432.9A Pending CN118355119A (zh) | 2021-11-30 | 2022-11-29 | 反义寡核苷酸（aso）与内源性作用于rna的腺苷脱氨酶（adar）用于高效精确rna编辑 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230235329A1 (https=) |
| EP (1) | EP4441216A1 (https=) |
| JP (1) | JP2024544067A (https=) |
| KR (1) | KR20240111752A (https=) |
| CN (1) | CN118355119A (https=) |
| AU (1) | AU2022399905A1 (https=) |
| CA (1) | CA3238033A1 (https=) |
| IL (1) | IL312762A (https=) |
| WO (1) | WO2023099494A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024114908A1 (en) * | 2022-11-30 | 2024-06-06 | Eberhard Karls Universität Tübingen | Chemically modified antisense oligonucleotides (asos) and compositions comprising the same for rna editing |
| AU2024325709A1 (en) * | 2023-08-15 | 2026-01-29 | Airna Corporation | Chemically modified antisense oligonucleotides (asos) and compositions for rna editing |
| WO2025073902A1 (en) * | 2023-10-06 | 2025-04-10 | Eberhard Karls Universität Tübingen | Chemical and sequence optimisation of antisense oligonucleotides for adar-mediated rna editing |
| WO2025172372A1 (en) | 2024-02-12 | 2025-08-21 | Airna Corporation | Conjugate for targeted delivery of an antisense oligonucleotide |
| WO2025242018A1 (zh) * | 2024-05-20 | 2025-11-27 | 时夕(广州)生物科技有限公司 | 一种经修饰的向导agRNA |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10941402B2 (en) | 2016-09-01 | 2021-03-09 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded RNA-editing oligonucleotides |
| WO2020001793A1 (en) | 2018-06-29 | 2020-01-02 | Eberhard-Karls-Universität Tübingen | Artificial nucleic acids for rna editing |
| CA3158528A1 (en) * | 2019-12-23 | 2021-07-01 | Jim SWILDENS | Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease |
-
2022
- 2022-11-29 WO PCT/EP2022/083711 patent/WO2023099494A1/en not_active Ceased
- 2022-11-29 EP EP22823405.0A patent/EP4441216A1/en active Pending
- 2022-11-29 CN CN202280079432.9A patent/CN118355119A/zh active Pending
- 2022-11-29 AU AU2022399905A patent/AU2022399905A1/en active Pending
- 2022-11-29 CA CA3238033A patent/CA3238033A1/en active Pending
- 2022-11-29 KR KR1020247016720A patent/KR20240111752A/ko active Pending
- 2022-11-29 JP JP2024532266A patent/JP2024544067A/ja active Pending
- 2022-11-29 IL IL312762A patent/IL312762A/en unknown
- 2022-11-30 US US18/071,999 patent/US20230235329A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL312762A (en) | 2024-07-01 |
| JP2024544067A (ja) | 2024-11-27 |
| US20230235329A1 (en) | 2023-07-27 |
| WO2023099494A1 (en) | 2023-06-08 |
| CA3238033A1 (en) | 2023-06-08 |
| AU2022399905A1 (en) | 2024-05-23 |
| WO2023099494A9 (en) | 2024-10-17 |
| EP4441216A1 (en) | 2024-10-09 |
| KR20240111752A (ko) | 2024-07-17 |
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