WO2026128564A1 - Procédés et compositions pour l'édition d'un gène associé à la galactosémie - Google Patents

Procédés et compositions pour l'édition d'un gène associé à la galactosémie

Info

Publication number
WO2026128564A1
WO2026128564A1 PCT/US2025/058927 US2025058927W WO2026128564A1 WO 2026128564 A1 WO2026128564 A1 WO 2026128564A1 US 2025058927 W US2025058927 W US 2025058927W WO 2026128564 A1 WO2026128564 A1 WO 2026128564A1
Authority
WO
WIPO (PCT)
Prior art keywords
seq
optionally substituted
base editor
polynucleotide
guide rna
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/US2025/058927
Other languages
English (en)
Inventor
Ayan Banerjee
Tom FERNANDEZ
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.)
Beam Therapeutics Inc
Original Assignee
Beam 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 Beam Therapeutics Inc filed Critical Beam Therapeutics Inc
Publication of WO2026128564A1 publication Critical patent/WO2026128564A1/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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)
    • 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)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

L'invention concerne des compositions et des procédés de traitement de galactosémie par introduction d'une ou de plusieurs altérations dans un polynucléotide galactose-1-phosphate uridylyltransférase (GALT) dans une cellule. Plus particulièrement, la présente invention concerne un éditeur de bases (par exemple, une protéine de fusion ou un complexe contenant une protéine de liaison à l'ADN programmable, un éditeur de bases nucléiques et un ARN guide) destiné à modifier un polynucléotide GALT, cette modification étant associée à une augmentation du niveau ou de l'activité du polypeptide GALT codé par ledit polynucléotide.
PCT/US2025/058927 2024-12-11 2025-12-10 Procédés et compositions pour l'édition d'un gène associé à la galactosémie Pending WO2026128564A1 (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202463730807P 2024-12-11 2024-12-11
US63/730,807 2024-12-11
US202563853947P 2025-07-30 2025-07-30
US63/853,947 2025-07-30

Publications (1)

Publication Number Publication Date
WO2026128564A1 true WO2026128564A1 (fr) 2026-06-18

Family

ID=100162113

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2025/058927 Pending WO2026128564A1 (fr) 2024-12-11 2025-12-10 Procédés et compositions pour l'édition d'un gène associé à la galactosémie

Country Status (1)

Country Link
WO (1) WO2026128564A1 (fr)

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210309986A1 (en) * 2018-07-19 2021-10-07 The Board Of Trustees Of The University Of Illinois Methods for exon skipping and gene knockout using base editors
US20230159956A1 (en) * 2018-05-11 2023-05-25 Beam Therapeutics Inc. Methods of editing single nucleotide polymorphism using programmable base editor systems
WO2024019936A1 (fr) * 2022-07-20 2024-01-25 Beam Therapeutics Inc. Nanomatériaux comprenant des triols
WO2024112876A2 (fr) * 2022-11-23 2024-05-30 Prime Medicine, Inc. Synthèse divisée d'arn longs
WO2024227047A2 (fr) * 2023-04-28 2024-10-31 Beam Therapeutics Inc. Arn guide modifié

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230159956A1 (en) * 2018-05-11 2023-05-25 Beam Therapeutics Inc. Methods of editing single nucleotide polymorphism using programmable base editor systems
US20210309986A1 (en) * 2018-07-19 2021-10-07 The Board Of Trustees Of The University Of Illinois Methods for exon skipping and gene knockout using base editors
WO2024019936A1 (fr) * 2022-07-20 2024-01-25 Beam Therapeutics Inc. Nanomatériaux comprenant des triols
WO2024112876A2 (fr) * 2022-11-23 2024-05-30 Prime Medicine, Inc. Synthèse divisée d'arn longs
WO2024227047A2 (fr) * 2023-04-28 2024-10-31 Beam Therapeutics Inc. Arn guide modifié

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