WO2024020484A3 - Inactivation biallélique d'angptl3 - Google Patents

Inactivation biallélique d'angptl3 Download PDF

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Publication number
WO2024020484A3
WO2024020484A3 PCT/US2023/070583 US2023070583W WO2024020484A3 WO 2024020484 A3 WO2024020484 A3 WO 2024020484A3 US 2023070583 W US2023070583 W US 2023070583W WO 2024020484 A3 WO2024020484 A3 WO 2024020484A3
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WO
WIPO (PCT)
Prior art keywords
angptl3
biallelic knockout
nucleotides
biallelic
knockout
Prior art date
Application number
PCT/US2023/070583
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English (en)
Other versions
WO2024020484A2 (fr
Inventor
Rafi EMMANUEL
Original Assignee
Emendobio 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 Emendobio Inc. filed Critical Emendobio Inc.
Publication of WO2024020484A2 publication Critical patent/WO2024020484A2/fr
Publication of WO2024020484A3 publication Critical patent/WO2024020484A3/fr

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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
    • 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 RNAses, DNAses
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/515—Angiogenesic factors; Angiogenin
    • 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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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 [CRISPRs]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Vascular Medicine (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

L'invention concerne des molécules d'ARN contenant une partie de séquence de guidage constituée de 17 à 50 nucléotides et comprenant 17 à 22 nucléotides dans la séquence présentée dans l'une quelconque des SEQ ID No : 1-20347, ainsi que des compositions, des méthodes et des utilisations associées.
PCT/US2023/070583 2022-07-20 2023-07-20 Inactivation biallélique d'angptl3 WO2024020484A2 (fr)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202263368926P 2022-07-20 2022-07-20
US63/368,926 2022-07-20
US202263385944P 2022-12-02 2022-12-02
US63/385,944 2022-12-02
US202363492808P 2023-03-29 2023-03-29
US63/492,808 2023-03-29

Publications (2)

Publication Number Publication Date
WO2024020484A2 WO2024020484A2 (fr) 2024-01-25
WO2024020484A3 true WO2024020484A3 (fr) 2024-05-10

Family

ID=89618483

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/070583 WO2024020484A2 (fr) 2022-07-20 2023-07-20 Inactivation biallélique d'angptl3

Country Status (1)

Country Link
WO (1) WO2024020484A2 (fr)

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018154387A1 (fr) * 2017-02-22 2018-08-30 Crispr Therapeutics Ag Compositions et méthodes pour l'édition génétique
US20210363547A1 (en) * 2018-05-06 2021-11-25 Emendobio Inc. Differential knockout of an allele of a heterozygous elane gene

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018154387A1 (fr) * 2017-02-22 2018-08-30 Crispr Therapeutics Ag Compositions et méthodes pour l'édition génétique
US20210363547A1 (en) * 2018-05-06 2021-11-25 Emendobio Inc. Differential knockout of an allele of a heterozygous elane gene

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
XU ET AL.: "Role of angiopoietin-like 3 (ANGPTL3) in regulating plasma level of low-density lipoprotein s", ATHEROSCLEROSIS, vol. 268, January 2018 (2018-01-01), pages 196 - 206, XP085322060, DOI: 10.1016/j.atherosclerosis.2017.08.031 *

Also Published As

Publication number Publication date
WO2024020484A2 (fr) 2024-01-25

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