AU2021350024A1 - Compositions and methods for inhibiting gene expression - Google Patents

Compositions and methods for inhibiting gene expression Download PDF

Info

Publication number
AU2021350024A1
AU2021350024A1 AU2021350024A AU2021350024A AU2021350024A1 AU 2021350024 A1 AU2021350024 A1 AU 2021350024A1 AU 2021350024 A AU2021350024 A AU 2021350024A AU 2021350024 A AU2021350024 A AU 2021350024A AU 2021350024 A1 AU2021350024 A1 AU 2021350024A1
Authority
AU
Australia
Prior art keywords
expression
repressor
dna
sequence
targeting moiety
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
AU2021350024A
Other languages
English (en)
Other versions
AU2021350024A9 (en
Inventor
Jeremiah Dale FARELLI
Jodi Michelle KENNEDY
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.)
Flagship Pioneering Innovations V Inc
Original Assignee
Flagship Pioneering Innovations V 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 Flagship Pioneering Innovations V Inc filed Critical Flagship Pioneering Innovations V Inc
Publication of AU2021350024A1 publication Critical patent/AU2021350024A1/en
Publication of AU2021350024A9 publication Critical patent/AU2021350024A9/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/635—Externally inducible repressor mediated regulation of gene expression, e.g. tetR inducible by tetracyline
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1007—Methyltransferases (general) (2.1.1.)
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/01—Methyltransferases (2.1.1)
    • C12Y201/01043—Histone-lysine N-methyltransferase (2.1.1.43)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Plant Substances (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2021350024A 2020-09-24 2021-09-24 Compositions and methods for inhibiting gene expression Pending AU2021350024A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063082555P 2020-09-24 2020-09-24
US63/082,555 2020-09-24
US202163242957P 2021-09-10 2021-09-10
US63/242,957 2021-09-10
PCT/US2021/051945 WO2022067033A1 (en) 2020-09-24 2021-09-24 Compositions and methods for inhibiting gene expression

Publications (2)

Publication Number Publication Date
AU2021350024A1 true AU2021350024A1 (en) 2023-05-04
AU2021350024A9 AU2021350024A9 (en) 2023-07-13

Family

ID=80845810

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021350024A Pending AU2021350024A1 (en) 2020-09-24 2021-09-24 Compositions and methods for inhibiting gene expression

Country Status (7)

Country Link
US (1) US20230399640A1 (https=)
EP (1) EP4217477A4 (https=)
JP (1) JP2023543218A (https=)
KR (1) KR20230074525A (https=)
AU (1) AU2021350024A1 (https=)
MX (1) MX2023003424A (https=)
WO (1) WO2022067033A1 (https=)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170151281A1 (en) 2015-02-19 2017-06-01 Batu Biologics, Inc. Chimeric antigen receptor dendritic cell (car-dc) for treatment of cancer
US20190255106A1 (en) 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
US10980836B1 (en) 2019-12-11 2021-04-20 Myeloid Therapeutics, Inc. Therapeutic cell compositions and methods of manufacturing and use thereof
EP4240367A4 (en) 2020-11-04 2024-10-16 Myeloid Therapeutics, Inc. MODIFIED CHIMERIC FUSION PROTEIN COMPOSITIONS AND METHODS OF USE THEREOF
WO2022197949A2 (en) 2021-03-17 2022-09-22 Myeloid Therapeutics, Inc. Engineered chimeric fusion protein compositions and methods of use thereof
BR112023023642A2 (pt) 2021-05-11 2024-01-30 Myeloid Therapeutics Inc Métodos e composições para integração genômica
WO2023057880A1 (en) * 2021-10-05 2023-04-13 Glaxosmithkline Intellectual Property Development Limited Crispr/cas9-based fusion proteins for modulating gene expression and methods of use
KR20250077608A (ko) * 2022-08-19 2025-05-30 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통한 b형 간염 바이러스 조절용 조성물, 시스템 및 방법
KR20250128349A (ko) * 2022-12-23 2025-08-27 에피제닉 테라퓨틱스 피티이 리미티드 융합체 및 이의 용도
EP4713027A1 (en) 2023-05-16 2026-03-25 Omega Therapeutics, Inc. Methods and compositions for modulating methylation of a target gene
WO2024238723A1 (en) 2023-05-16 2024-11-21 Omega Therapeutics, Inc. Methods and compositions for modulating pcsk9 expression
WO2025019742A1 (en) 2023-07-19 2025-01-23 Omega Therapeutics, Inc. Methods and compositions for modulating ctnnb1 expression
WO2025064469A1 (en) * 2023-09-18 2025-03-27 Omega Therapeutics, Inc. Methods for assessing dosage for epigenetic modifying agents
WO2025171047A1 (en) * 2024-02-05 2025-08-14 Bioventures, Llc Leveraging cancer-evolved resistance mechanisms to enhance ezh2 activity in adoptive t cells
WO2025261475A1 (zh) * 2024-06-21 2025-12-26 益杰立科(上海)生物科技有限公司 靶向血管内皮生长因子的工程化基因转录阻抑工具及用途

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013266968B2 (en) * 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
GB201418965D0 (https=) * 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
WO2017031370A1 (en) * 2015-08-18 2017-02-23 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US9896696B2 (en) * 2016-02-15 2018-02-20 Benson Hill Biosystems, Inc. Compositions and methods for modifying genomes
US20220380760A1 (en) * 2018-10-15 2022-12-01 Flagship Pioneering Innovations V, Inc. Disrupting genomic complex assembly in fusion genes
US20220249625A1 (en) * 2019-01-29 2022-08-11 Flagship Pioneering Innovations V, Inc. Compositions comprising an endonuclease and methods for purifying an endonuclease

Also Published As

Publication number Publication date
EP4217477A4 (en) 2024-10-16
AU2021350024A9 (en) 2023-07-13
US20230399640A1 (en) 2023-12-14
WO2022067033A1 (en) 2022-03-31
JP2023543218A (ja) 2023-10-13
EP4217477A1 (en) 2023-08-02
KR20230074525A (ko) 2023-05-30
MX2023003424A (es) 2023-05-03

Similar Documents

Publication Publication Date Title
US20230399640A1 (en) Compositions and methods for inhibiting gene expression
US20240132559A1 (en) Compositions and methods for modulating myc expression
US20210322577A1 (en) Methods and systems for modifying dna
JP2019531763A (ja) 遺伝子発現をモジュレートするための方法および組成物
US20220403387A1 (en) Modulating genomic complexes
US20250375464A1 (en) Formulations for modulating myc expression
JP2025169372A (ja) フラタキシン発現を調節し、フリードライヒ運動失調症を治療するための方法及び組成物
US20230374549A1 (en) Compositions and methods for inhibiting the expression of multiple genes
WO2021061640A1 (en) Compositions and methods for modulating genomic complex integrity index
CA3196827A1 (en) Compositions and methods for inhibiting gene expression
CN116829175A (zh) 用于调节myc表达的组合物和方法

Legal Events

Date Code Title Description
SREP Specification republished