CN118434758A - 肠肽的多顺反子表达 - Google Patents

肠肽的多顺反子表达 Download PDF

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Publication number
CN118434758A
CN118434758A CN202280082853.7A CN202280082853A CN118434758A CN 118434758 A CN118434758 A CN 118434758A CN 202280082853 A CN202280082853 A CN 202280082853A CN 118434758 A CN118434758 A CN 118434758A
Authority
CN
China
Prior art keywords
expression construct
seq
sequence
peptide
tricistronic
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
CN202280082853.7A
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English (en)
Chinese (zh)
Inventor
郭雪翠
A·福布斯
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.)
Meritus Gene Regulation Ltd
Original Assignee
MeiraGTx UK II Ltd
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 MeiraGTx UK II Ltd filed Critical MeiraGTx UK II Ltd
Publication of CN118434758A publication Critical patent/CN118434758A/zh
Pending legal-status Critical Current

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Classifications

    • 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
    • C07K14/435—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
    • C07K14/4713—Autoimmune diseases, e.g. Insulin-dependent diabetes mellitus, multiple sclerosis, rheumathoid arthritis, systemic lupus erythematosus; Autoantigens
    • 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
    • A61P3/04—Anorexiants; Antiobesity agents
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • C07K14/575—Hormones
    • C07K14/605—Glucagons
    • 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
    • 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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Diabetes (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Endocrinology (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Obesity (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Emergency Medicine (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Child & Adolescent Psychology (AREA)
  • Immunology (AREA)
  • Rehabilitation Therapy (AREA)
CN202280082853.7A 2021-12-15 2022-12-15 肠肽的多顺反子表达 Pending CN118434758A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163361399P 2021-12-15 2021-12-15
US63/361399 2021-12-15
PCT/IB2022/000815 WO2023131811A2 (en) 2021-12-15 2022-12-15 Polycistronic expression of gut peptides

Publications (1)

Publication Number Publication Date
CN118434758A true CN118434758A (zh) 2024-08-02

Family

ID=86732042

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280082853.7A Pending CN118434758A (zh) 2021-12-15 2022-12-15 肠肽的多顺反子表达

Country Status (10)

Country Link
US (1) US20250302992A1 (enExample)
EP (1) EP4448555A2 (enExample)
JP (1) JP2025500874A (enExample)
KR (1) KR20240133712A (enExample)
CN (1) CN118434758A (enExample)
AU (1) AU2022431978B2 (enExample)
CA (1) CA3239128A1 (enExample)
IL (1) IL313462A (enExample)
MX (1) MX2024007519A (enExample)
WO (1) WO2023131811A2 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026092369A1 (zh) * 2024-10-31 2026-05-07 派格生物医药(杭州)股份有限公司 一种环状rna分子及其应用

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ511037A (en) 1998-09-17 2005-02-25 Univ Florida Methods for treatment of degenerative retinal diseases
AU2002327430A1 (en) * 2001-08-08 2003-02-24 Genzyme Corporation Methods for treating diabetes and other blood sugar disorders
ATE514776T1 (de) * 2004-10-05 2011-07-15 California Inst Of Techn Aptamer-regulierte nukleinsäuren und verwendungen davon
US7829664B2 (en) * 2007-06-01 2010-11-09 Boehringer Ingelheim International Gmbh Modified nucleotide sequence encoding glucagon-like peptide-1 (GLP-1), nucleic acid construct comprising same for production of glucagon-like peptide-1 (GLP-1), human cells comprising said construct and insulin-producing constructs, and methods of use thereof
EA037120B1 (ru) 2015-02-02 2021-02-09 МЕИРЭДжТиЭкс ЮКей II ЛИМИТЕД Регулирование экспрессии генов посредством аптамер-опосредованного модулирования альтернативного сплайсинга
MX2018009370A (es) 2016-02-02 2018-11-12 Meiragtx Uk Ii Ltd Regulacion de expresion genica a través de poliadenilacion modulada por aptámeros.
SG11201806589UA (en) 2016-02-02 2018-09-27 Meiragtx Uk Ii Ltd Regulation of gene expression via aptamer-mediated control of self-cleaving ribozymes
US11311633B2 (en) * 2016-04-16 2022-04-26 University Of Florida Research Foundation, Incorporated Satiation peptides for weight loss and altered taste sensitivity
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
WO2018156658A1 (en) 2017-02-21 2018-08-30 MEIRAGTX, UK II Limited Regulation of gene expression by aptamer-mediated accessibility of polyadenylation signals
US11739333B2 (en) 2017-03-02 2023-08-29 Meiragtx Uk Ii Limited Regulation of gene expression by aptamer-modulated RNase p cleavage

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026092369A1 (zh) * 2024-10-31 2026-05-07 派格生物医药(杭州)股份有限公司 一种环状rna分子及其应用

Also Published As

Publication number Publication date
US20250302992A1 (en) 2025-10-02
JP2025500874A (ja) 2025-01-15
KR20240133712A (ko) 2024-09-04
MX2024007519A (es) 2024-08-28
WO2023131811A3 (en) 2023-09-14
AU2022431978B2 (en) 2026-05-07
AU2022431978A1 (en) 2024-07-11
CA3239128A1 (en) 2023-07-13
EP4448555A2 (en) 2024-10-23
IL313462A (en) 2024-08-01
WO2023131811A2 (en) 2023-07-13

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Effective date of registration: 20260109

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Applicant after: Meritus Gene Regulation Ltd.

Country or region after: United Kingdom

Address before: London, England

Applicant before: MEIRAGTX UK II LTD.

Country or region before: United Kingdom