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 דם

Info

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
Application number
IL323452A
Other languages
English (en)
Original Assignee
Verve Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Verve Therapeutics Inc filed Critical Verve Therapeutics Inc
Publication of IL323452A publication Critical patent/IL323452A/he

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226Class 2 CAS enzyme complex, e.g. single CAS protein
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type 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)
IL323452A 2023-03-20 2025-09-18 עריכה מבוססת ניקאז בגוף החי של הגן lpa לטיפול במחלות לב וכלי דם IL323452A (he)

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

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

Similar Documents

Publication Publication Date Title
US20240390523A1 (en) Base editing of pcsk9 and methods of using same for treatment of disease
US12263227B2 (en) Optimized mRNA encoding CAS9 for use in LNPs
US20070281900A1 (en) COMPOSITIONS AND METHODS FOR LIPID AND POLYPEPTIDE BASED siRNA INTRACELLULAR DELIVERY
JP2019516351A (ja) Crispr/cas構成成分のための脂質ナノ粒子製剤
IL323452A (he) עריכה מבוססת ניקאז בגוף החי של הגן lpa לטיפול במחלות לב וכלי דם
IL301973A (he) תהליך משופר להכנת ננו-חלקיקי ליפידים הנושאים mRNA
IL292420A (he) מבנים אורניתין טרנסקרבאמילאז (otc) ושיטות לשימוש בהם
US20250312491A1 (en) Base editing of transthyretin gene
US20240124536A1 (en) Base editing of transthyretin gene
WO2026035968A1 (en) In vivo nickase-based editing of the angiotensinogen gene
CN117916368A (zh) 甲状腺素转运蛋白基因的碱基编辑
JP2025084707A (ja) アンチセンス核酸およびその利用
WO2026055228A1 (en) Methods and compositions for editing a gene associated with phenylketonuria (pku)
WO2024112775A1 (en) Compositions and methods for editing a transthyretin gene
WO2025159966A1 (en) Compositions and methods for altering a nucleobase in a transthyretin polynucleotide