IL323452A - עריכה מבוססת ניקאז בגוף החי של הגן lpa לטיפול במחלות לב וכלי דם - Google Patents
עריכה מבוססת ניקאז בגוף החי של הגן lpa לטיפול במחלות לב וכלי דםInfo
- Publication number
- IL323452A IL323452A IL323452A IL32345225A IL323452A IL 323452 A IL323452 A IL 323452A IL 323452 A IL323452 A IL 323452A IL 32345225 A IL32345225 A IL 32345225A IL 323452 A IL323452 A IL 323452A
- Authority
- IL
- Israel
- Prior art keywords
- sequence
- spacer
- nucleotides
- guide
- gene
- Prior art date
Links
Classifications
-
- 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
- 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
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
- C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
- C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
-
- 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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202363453207P | 2023-03-20 | 2023-03-20 | |
| US202463554838P | 2024-02-16 | 2024-02-16 | |
| PCT/US2024/020769 WO2024197065A2 (en) | 2023-03-20 | 2024-03-20 | In vivo nickase-based editing of the lpa gene for treatment of cardiovascular disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL323452A true IL323452A (he) | 2025-11-01 |
Family
ID=92842450
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL323452A IL323452A (he) | 2023-03-20 | 2025-09-18 | עריכה מבוססת ניקאז בגוף החי של הגן lpa לטיפול במחלות לב וכלי דם |
Country Status (9)
| Country | Link |
|---|---|
| EP (1) | EP4684017A2 (he) |
| JP (1) | JP2026511099A (he) |
| KR (1) | KR20250163949A (he) |
| CN (1) | CN121358859A (he) |
| AU (1) | AU2024241162A1 (he) |
| GB (1) | GB2643844A (he) |
| IL (1) | IL323452A (he) |
| MX (1) | MX2025011034A (he) |
| WO (1) | WO2024197065A2 (he) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025166323A2 (en) * | 2024-02-02 | 2025-08-07 | Editas Medicine, Inc. | Crispr-related methods and compositions targeting lipoprotein (a) expression |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2748495C2 (ru) * | 2012-05-24 | 2021-05-26 | Ионис Фармасьютикалз, Инк. | Способы и композиции для модулирования экспрессии аполипопротеина (а) |
| DK2931897T3 (en) * | 2012-12-12 | 2018-02-05 | Broad Inst Inc | CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS |
| GB2632565B (en) * | 2020-04-09 | 2025-06-04 | Verve Therapeutics Inc | Base editing of PCSK9 and methods of using same for treatment of disease |
| KR102812602B1 (ko) * | 2020-08-05 | 2025-05-28 | 다이서나 파마수이티컬, 인크. | Lpa 발현을 저해하기 위한 조성물 및 방법 |
-
2024
- 2024-03-20 KR KR1020257034729A patent/KR20250163949A/ko active Pending
- 2024-03-20 JP JP2025555424A patent/JP2026511099A/ja active Pending
- 2024-03-20 GB GB2517438.4A patent/GB2643844A/en active Pending
- 2024-03-20 WO PCT/US2024/020769 patent/WO2024197065A2/en not_active Ceased
- 2024-03-20 AU AU2024241162A patent/AU2024241162A1/en active Pending
- 2024-03-20 CN CN202480029064.6A patent/CN121358859A/zh active Pending
- 2024-03-20 EP EP24775657.0A patent/EP4684017A2/en active Pending
-
2025
- 2025-09-18 IL IL323452A patent/IL323452A/he unknown
- 2025-09-18 MX MX2025011034A patent/MX2025011034A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2024197065A2 (en) | 2024-09-26 |
| JP2026511099A (ja) | 2026-04-10 |
| KR20250163949A (ko) | 2025-11-21 |
| CN121358859A (zh) | 2026-01-16 |
| MX2025011034A (es) | 2026-03-02 |
| AU2024241162A1 (en) | 2025-09-25 |
| EP4684017A2 (en) | 2026-01-28 |
| WO2024197065A3 (en) | 2024-11-21 |
| GB2643844A (en) | 2026-03-04 |
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