AU2017254718A1 - Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four - Google Patents

Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four Download PDF

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Publication number
AU2017254718A1
AU2017254718A1 AU2017254718A AU2017254718A AU2017254718A1 AU 2017254718 A1 AU2017254718 A1 AU 2017254718A1 AU 2017254718 A AU2017254718 A AU 2017254718A AU 2017254718 A AU2017254718 A AU 2017254718A AU 2017254718 A1 AU2017254718 A1 AU 2017254718A1
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Australia
Prior art keywords
seq
nuclease
guide rna
directs
grna
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Abandoned
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AU2017254718A
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Inventor
Sean Michael BURNS
Sarah Beth HESSE
Bradley Andrew MURRAY
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Intellia Therapeutics Inc
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Intellia Therapeutics Inc
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    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A61K9/0051—Ocular inserts or implants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5184—Virus capsids or envelopes enclosing drugs
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • 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]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychology (AREA)
  • Botany (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
AU2017254718A 2016-04-22 2017-04-21 Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four Abandoned AU2017254718A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662326700P 2016-04-22 2016-04-22
US62/326,700 2016-04-22
PCT/US2017/028981 WO2017185054A1 (en) 2016-04-22 2017-04-21 Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four

Publications (1)

Publication Number Publication Date
AU2017254718A1 true AU2017254718A1 (en) 2018-11-15

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
AU2017254718A Abandoned AU2017254718A1 (en) 2016-04-22 2017-04-21 Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four

Country Status (11)

Country Link
US (2) US20190142972A1 (https=)
EP (1) EP3445375A1 (https=)
JP (1) JP2019515914A (https=)
KR (1) KR20180134412A (https=)
CN (1) CN109414450A (https=)
AU (1) AU2017254718A1 (https=)
BR (1) BR112018071439A2 (https=)
CA (1) CA3021647A1 (https=)
CO (1) CO2018012433A2 (https=)
MX (1) MX2018012873A (https=)
WO (1) WO2017185054A1 (https=)

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CA2989331A1 (en) * 2015-07-02 2017-01-05 The Johns Hopkins University Crispr/cas9-based treatments
WO2022072458A1 (en) * 2020-09-29 2022-04-07 Avellino Lab Usa, Inc. Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy
AU2017379073B2 (en) 2016-12-22 2023-12-14 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2018165541A1 (en) * 2017-03-10 2018-09-13 The Board Of Regents Of The University Of Texas System Treatment of fuchs' endothelial corneal dystrophy
EP3723813A4 (en) * 2017-12-15 2021-10-06 Regents of the University of Minnesota GENOMIC CRISPR MEDIATION EDITING WITH VECTORS
CN109929872A (zh) * 2017-12-18 2019-06-25 中国科学院遗传与发育生物学研究所 一种通过基因编辑技术创制番茄白果材料的方法
WO2019122302A1 (en) 2017-12-21 2019-06-27 Max-Delbrück-Centrum Für Molekulare Medizin In Der Helmholtz-Gemeinschaft Nucleic acid sequence replacement by nhej
US20190151470A1 (en) * 2018-01-21 2019-05-23 RoverMed BioSciences, LLC Nanoparticles comprising protein-polynucleotide complexes and for delivering protein based complexes
KR20210139225A (ko) * 2019-02-28 2021-11-22 프리드리히 이. 크루제 세포외 기질 조정제
AU2020229871B2 (en) * 2019-02-28 2023-05-18 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
JP2023515710A (ja) * 2020-04-27 2023-04-13 デューク ユニバーシティ ＣＲＩＳＰＲ媒介式エクソン欠失用の最適なｇＲＮＡ対を発見するためのハイスループットスクリーニング法
CN111944813B (zh) * 2020-08-24 2022-07-15 武汉纽福斯生物科技有限公司 核苷酸、病毒载体及其应用和RNAi药物制剂
CN111944814B (zh) * 2020-08-24 2022-07-15 武汉纽福斯生物科技有限公司 寡核苷酸、病毒载体及其应用和RNAi药物制剂
CR20230305A (es) 2020-12-11 2023-11-10 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación
EP4288541A1 (en) 2021-02-05 2023-12-13 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
EP4430185A1 (en) * 2021-11-09 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy
US12297285B2 (en) 2022-06-24 2025-05-13 Orna Therapeutics, Inc. Circular RNA encoding chimeric antigen receptors targeting BCMA
CN120390656A (zh) 2022-11-08 2025-07-29 欧纳医疗公司 环状rna组合物
WO2024226536A1 (en) * 2023-04-24 2024-10-31 The General Hospital Corporation Methods and compositions for modifying genetic repeats
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (10)

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WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
WO2013101711A1 (en) * 2011-12-30 2013-07-04 Mayo Foundation For Medical Education And Research Assessing likelihood of developing fuchs' corneal dystrophy
EP3241902B1 (en) * 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
EP2931899A1 (en) * 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP3608308B1 (en) 2013-03-08 2021-07-21 Novartis AG Lipids and lipid compositions for the delivery of active agents
AU2014346559B2 (en) * 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
EP3470089A1 (en) * 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
ES2895651T3 (es) 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
CA2945335A1 (en) * 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
CA2989331A1 (en) * 2015-07-02 2017-01-05 The Johns Hopkins University Crispr/cas9-based treatments

Also Published As

Publication number Publication date
US20250025580A1 (en) 2025-01-23
JP2019515914A (ja) 2019-06-13
CN109414450A (zh) 2019-03-01
KR20180134412A (ko) 2018-12-18
BR112018071439A2 (pt) 2019-03-19
CO2018012433A2 (es) 2019-04-30
MX2018012873A (es) 2019-08-05
EP3445375A1 (en) 2019-02-27
CA3021647A1 (en) 2017-10-26
WO2017185054A1 (en) 2017-10-26
US20190142972A1 (en) 2019-05-16

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MK4 Application lapsed section 142(2)(d) - no continuation fee paid for the application