BR112018071439A2 - composições e métodos para o tratamento de doenças associadas com repetições de três nucleotídeos no fator de transcrição quatro - Google Patents

composições e métodos para o tratamento de doenças associadas com repetições de três nucleotídeos no fator de transcrição quatro

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Publication number
BR112018071439A2
BR112018071439A2 BR112018071439-9A BR112018071439A BR112018071439A2 BR 112018071439 A2 BR112018071439 A2 BR 112018071439A2 BR 112018071439 A BR112018071439 A BR 112018071439A BR 112018071439 A2 BR112018071439 A2 BR 112018071439A2
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BR
Brazil
Prior art keywords
compositions
methods
nucleotide repeats
transcription factor
diseases associated
Prior art date
Application number
BR112018071439-9A
Other languages
English (en)
Inventor
Michael Burns Sean
Andrew Murray Bradley
Beth Hesse Sarah
Original Assignee
Intellia 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.)
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Publication date
Application filed by Intellia Therapeutics, Inc. filed Critical Intellia Therapeutics, Inc.
Publication of BR112018071439A2 publication Critical patent/BR112018071439A2/pt

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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
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • A—HUMAN NECESSITIES
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61K9/5107—Excipients; Inactive ingredients
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
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    • 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
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    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
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    • 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 [CRISPRs]

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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)
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  • Animal Behavior & Ethology (AREA)
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  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
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  • Organic Chemistry (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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  • Ophthalmology & Optometry (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nanotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Psychology (AREA)
  • Botany (AREA)
  • Optics & Photonics (AREA)
  • Immunology (AREA)
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  • Microbiology (AREA)

Abstract

a presente invenção refere-se a composições e métodos para remoção por excisão de repetições de três nucleotídeos (tnrs) contidas no intron 3 de tcf4, tal como é visto em indivíduos portadores de distrofia endotelial da córnea ou distrofia de fuchs (fecd), colangite esclerosante primária (cep) e esquizofrenia. além disso, são descritas também composições compreendendo sequências-guia que têm como alvo a subunidade alfa 2 do colágeno viii para o tratamento de mutações nesta subunidade que podem contribuir para distrofia endotelial da córnea ou de fuchs (fecd).
BR112018071439-9A 2016-04-22 2017-04-21 composições e métodos para o tratamento de doenças associadas com repetições de três nucleotídeos no fator de transcrição quatro BR112018071439A2 (pt)

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
BR112018071439A2 true BR112018071439A2 (pt) 2019-03-19

Family

ID=58692575

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018071439-9A BR112018071439A2 (pt) 2016-04-22 2017-04-21 composições e métodos para o tratamento de doenças associadas com repetições de três nucleotídeos no fator de transcrição quatro

Country Status (11)

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

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108350446A (zh) * 2015-07-02 2018-07-31 约翰霍普金斯大学 基于crispr/cas9的治疗
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
US11512312B2 (en) 2017-03-10 2022-11-29 The Board Of Regents Of The University Of Texas System Treatment of Fuchs' endothelial corneal dystrophy
US20230201373A1 (en) * 2017-12-15 2023-06-29 Regents Of The University Of Minnesota Crispr-mediated genome 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
US20220047618A1 (en) * 2019-02-28 2022-02-17 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
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
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
EP4259792A1 (en) 2020-12-11 2023-10-18 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination
WO2023086842A1 (en) * 2021-11-09 2023-05-19 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
WO2013101711A1 (en) * 2011-12-30 2013-07-04 Mayo Foundation For Medical Education And Research Assessing likelihood of developing fuchs' corneal dystrophy
DE202013012242U1 (de) * 2012-05-25 2016-02-02 Emmanuelle Charpentier Zusammensetzungen für die durch RNA gesteuerte Modifikation einer Ziel-DNA und für die durch RNA gesteuerte Modulation der Transkription
WO2014093701A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EA030650B1 (ru) 2013-03-08 2018-09-28 Новартис Аг Липиды и липидные композиции для доставки активных агентов
DK3066201T3 (en) * 2013-11-07 2018-06-06 Editas Medicine Inc CRISPR-RELATED PROCEDURES AND COMPOSITIONS WITH LEADING GRADES
EP3079726B1 (en) * 2013-12-12 2018-12-05 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
US10059655B2 (en) 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
JP2017513485A (ja) * 2014-04-18 2017-06-01 エディタス・メディシン，インコーポレイテッド がん免疫療法のためのｃｒｉｓｐｒ−ｃａｓ関連方法、組成物および構成要素
CN108350446A (zh) * 2015-07-02 2018-07-31 约翰霍普金斯大学 基于crispr/cas9的治疗

Also Published As

Publication number Publication date
EP3445375A1 (en) 2019-02-27
MX2018012873A (es) 2019-08-05
KR20180134412A (ko) 2018-12-18
CO2018012433A2 (es) 2019-04-30
JP2019515914A (ja) 2019-06-13
US20190142972A1 (en) 2019-05-16
AU2017254718A1 (en) 2018-11-15
WO2017185054A1 (en) 2017-10-26
CN109414450A (zh) 2019-03-01
CA3021647A1 (en) 2017-10-26

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