WO2024254519A3 - Compositions et procédés pour la modification de gènes humains exprimés par des cellules hépatiques - Google Patents

Compositions et procédés pour la modification de gènes humains exprimés par des cellules hépatiques Download PDF

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Publication number
WO2024254519A3
WO2024254519A3 PCT/US2024/033100 US2024033100W WO2024254519A3 WO 2024254519 A3 WO2024254519 A3 WO 2024254519A3 US 2024033100 W US2024033100 W US 2024033100W WO 2024254519 A3 WO2024254519 A3 WO 2024254519A3
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WO
WIPO (PCT)
Prior art keywords
methods
compositions
modification
liver cells
human genes
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
Application number
PCT/US2024/033100
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English (en)
Other versions
WO2024254519A2 (fr
Inventor
Sean Chen
Lucas Benjamin HARRINGTON
William Douglass WRIGHT
Aaron DELOUGHERY
Wiputra Jaya HARTONO
Benjamin Julius RAUCH
Stepan TYMOSHENKO
Lauren Kelli UYESAKA
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Mammoth Biosciences Inc
Original Assignee
Mammoth Biosciences Inc
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Filing date
Publication date
Application filed by Mammoth Biosciences Inc filed Critical Mammoth Biosciences Inc
Publication of WO2024254519A2 publication Critical patent/WO2024254519A2/fr
Publication of WO2024254519A3 publication Critical patent/WO2024254519A3/fr
Anticipated expiration legal-status Critical
Pending legal-status Critical Current

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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
    • 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
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • C12N15/1137—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 enzymes
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)

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

Abstract

L'invention concerne des compositions, des systèmes et des procédés comprenant des protéines effectrices, et leurs utilisations. Ces protéines effectrices peuvent être caractérisées en tant que protéines associées à CRISPR (Cas). Diverses compositions, systèmes et procédés de la présente divulgation peuvent tirer profit des activités de ces protéines effectrices pour l'édition et/ou l'ingénierie d'acides nucléiques.
PCT/US2024/033100 2023-06-08 2024-06-07 Compositions et procédés pour la modification de gènes humains exprimés par des cellules hépatiques Pending WO2024254519A2 (fr)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202363507030P 2023-06-08 2023-06-08
US63/507,030 2023-06-08
US202363586743P 2023-09-29 2023-09-29
US63/586,743 2023-09-29
US202363603618P 2023-11-28 2023-11-28
US63/603,618 2023-11-28

Publications (2)

Publication Number Publication Date
WO2024254519A2 WO2024254519A2 (fr) 2024-12-12
WO2024254519A3 true WO2024254519A3 (fr) 2025-04-17

Family

ID=93796423

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/033100 Pending WO2024254519A2 (fr) 2023-06-08 2024-06-07 Compositions et procédés pour la modification de gènes humains exprimés par des cellules hépatiques

Country Status (1)

Country Link
WO (1) WO2024254519A2 (fr)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190247517A1 (en) * 2017-10-17 2019-08-15 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing for hemophilia a
US20200270618A1 (en) * 2018-10-18 2020-08-27 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2023039378A1 (fr) * 2021-09-08 2023-03-16 Metagenomi, Inc. Systèmes crispr de classe ii, de type v

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190247517A1 (en) * 2017-10-17 2019-08-15 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing for hemophilia a
US20200270618A1 (en) * 2018-10-18 2020-08-27 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2023039378A1 (fr) * 2021-09-08 2023-03-16 Metagenomi, Inc. Systèmes crispr de classe ii, de type v

Also Published As

Publication number Publication date
WO2024254519A2 (fr) 2024-12-12

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