ZA202102302B - Methods and compositions for increasing protein expression and/or treating a haploinsufficiency disorder - Google Patents

Methods and compositions for increasing protein expression and/or treating a haploinsufficiency disorder

Info

Publication number
ZA202102302B
ZA202102302B ZA2021/02302A ZA202102302A ZA202102302B ZA 202102302 B ZA202102302 B ZA 202102302B ZA 2021/02302 A ZA2021/02302 A ZA 2021/02302A ZA 202102302 A ZA202102302 A ZA 202102302A ZA 202102302 B ZA202102302 B ZA 202102302B
Authority
ZA
South Africa
Prior art keywords
compositions
treating
methods
protein expression
increasing protein
Prior art date
Application number
ZA2021/02302A
Other languages
English (en)
Inventor
Jeffery M Coller
Thomas Sweet
Harvey Lodish
Original Assignee
Univ Case Western Reserve
Whitehead Inst Biomedical Res
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 Univ Case Western Reserve, Whitehead Inst Biomedical Res filed Critical Univ Case Western Reserve
Publication of ZA202102302B publication Critical patent/ZA202102302B/en

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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N15/63—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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Pain & Pain Management (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
ZA2021/02302A 2018-09-26 2021-04-07 Methods and compositions for increasing protein expression and/or treating a haploinsufficiency disorder ZA202102302B (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862736847P 2018-09-26 2018-09-26
PCT/US2019/053268 WO2020069199A1 (en) 2018-09-26 2019-09-26 Methods and compositions for increasing protein expression and/or treating a haploinsufficiency disorder

Publications (1)

Publication Number Publication Date
ZA202102302B true ZA202102302B (en) 2022-04-28

Family

ID=69949913

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2021/02302A ZA202102302B (en) 2018-09-26 2021-04-07 Methods and compositions for increasing protein expression and/or treating a haploinsufficiency disorder

Country Status (15)

Country Link
US (1) US12281314B2 (enExample)
EP (1) EP3856905A4 (enExample)
JP (2) JP2022502044A (enExample)
KR (1) KR102814622B1 (enExample)
CN (1) CN113195719A (enExample)
AU (1) AU2019350871A1 (enExample)
BR (1) BR112021005788A2 (enExample)
CA (1) CA3114109A1 (enExample)
CL (1) CL2021000747A1 (enExample)
CO (1) CO2021005040A2 (enExample)
EA (1) EA202190736A1 (enExample)
IL (1) IL281848A (enExample)
MX (1) MX2021003567A (enExample)
WO (1) WO2020069199A1 (enExample)
ZA (1) ZA202102302B (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20220128611A (ko) * 2019-11-04 2022-09-21 플래그쉽 파이어니어링, 인크. 맥락적 희귀 코돈용 trem 조성물 및 관련 용도
MX2022015042A (es) * 2020-05-29 2023-03-09 Flagship Pioneering Innovations Vi Llc Composiciones de trem y métodos relacionados con las mismas.
CA3182026A1 (en) * 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Trem compositions and methods relating thereto
CA3217460A1 (en) * 2021-05-05 2022-11-10 Peter M. EIMON Methods and compositions for treating a premature termination codon-mediated disorder
WO2023205606A1 (en) 2022-04-18 2023-10-26 Vertex Pharmaceuticals Incorporated Compositions and methods for enhancing aav therapy and decreasing tropism of aav to the liver
CN117257958B (zh) * 2023-11-21 2024-02-09 四川大学华西医院 Trps1抑制剂的新用途及用于治疗和/或预防雄激素性秃发的药物
WO2025176166A1 (en) * 2024-02-21 2025-08-28 Zhuhai Codone Biotechnology Co., Ltd. Engineered trna expression cassette

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000015775A1 (en) * 1998-09-17 2000-03-23 Smithkline Beecham Corporation Rnase p polypeptides
US20040142325A1 (en) * 2001-09-14 2004-07-22 Liat Mintz Methods and systems for annotating biomolecular sequences
JP4406731B2 (ja) 2005-03-31 2010-02-03 国立大学法人 岡山大学 部位特異的アミノ酸導入法のための新規な直交化ｔＲＮＡ
WO2008127900A1 (en) 2007-04-13 2008-10-23 The Salk Institute For Biological Studies Methods of genetically encoding unnatural amino acids in eukaryotic cells using orthogonal trna/synthetase pairs
US20120283116A1 (en) * 2009-09-11 2012-11-08 Leppert Mark F Mutant Sodium Channel Nav1.7 and Methods Related Thereto
US9676810B2 (en) 2010-07-08 2017-06-13 The Brigham And Women's Hospital, Inc. Neuroprotective molecules and methods of treating neurological disorders and inducing stress granules
US20130129668A1 (en) 2011-09-01 2013-05-23 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics
WO2014052855A1 (en) * 2012-09-27 2014-04-03 Population Diagnostics, Inc. Methods and compositions for screening and treating developmental disorders
EP3052624A1 (en) * 2013-10-02 2016-08-10 Wageningen Universiteit Systematic optimization of coding sequence for functional protein expression
EP3362087A1 (en) * 2015-10-15 2018-08-22 The Scripps Research Institute BINDING ACTIVITY OF AMINOACYL-tRNA SYNTHETASE IN CHARCOT-MARIE-TOOTH (CMT) NEUROPATHY AND CMT-RELATED NEUROLOGICAL DISEASES
US11083745B2 (en) * 2015-12-14 2021-08-10 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and Dravet Syndrome
WO2017193051A1 (en) * 2016-05-06 2017-11-09 The Trustees Of Princeton University Methods of monitoring rnase l activity
DK3458074T3 (da) * 2016-05-16 2024-07-29 Univ Texas COMPOSITIONS FOR THE DELIVERY OF tRNA AS NANOPARTICLES AND METHODS OF USE THEREWITH
US11848074B2 (en) * 2016-12-07 2023-12-19 Gottfried Wilhelm Leibniz Universität Hannover Codon optimization
EP3976782A1 (en) * 2019-05-31 2022-04-06 Flagship Pioneering, Inc. Uses of trem compositions to modulate trna pools

Also Published As

Publication number Publication date
US12281314B2 (en) 2025-04-22
EA202190736A1 (ru) 2021-09-16
CA3114109A1 (en) 2020-04-02
US20220073933A1 (en) 2022-03-10
MX2021003567A (es) 2021-08-24
EP3856905A1 (en) 2021-08-04
KR102814622B1 (ko) 2025-05-29
BR112021005788A2 (pt) 2021-10-26
CN113195719A (zh) 2021-07-30
WO2020069199A1 (en) 2020-04-02
CO2021005040A2 (es) 2021-09-20
EP3856905A4 (en) 2023-06-21
JP2025010148A (ja) 2025-01-20
KR20210091699A (ko) 2021-07-22
CL2021000747A1 (es) 2021-10-22
AU2019350871A1 (en) 2021-05-20
JP2022502044A (ja) 2022-01-11
IL281848A (en) 2021-05-31

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