CA3145309A1 - Rna-targeting knockdown and replacement compositions and methods for use - Google Patents

Rna-targeting knockdown and replacement compositions and methods for use

Info

Publication number
CA3145309A1
CA3145309A1 CA3145309A CA3145309A CA3145309A1 CA 3145309 A1 CA3145309 A1 CA 3145309A1 CA 3145309 A CA3145309 A CA 3145309A CA 3145309 A CA3145309 A CA 3145309A CA 3145309 A1 CA3145309 A1 CA 3145309A1
Authority
CA
Canada
Prior art keywords
rna
seq
sequence
protein
disclosure
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
CA3145309A
Other languages
English (en)
French (fr)
Inventor
David A. Nelles
Ranjan BATRA
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.)
Locanabio Inc
Original Assignee
Locanabio 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 Locanabio Inc filed Critical Locanabio Inc
Publication of CA3145309A1 publication Critical patent/CA3145309A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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
    • A61P27/16—Otologicals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • C12N15/861—Adenoviral 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
    • 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]
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Cardiology (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
CA3145309A 2019-07-10 2020-07-10 Rna-targeting knockdown and replacement compositions and methods for use Pending CA3145309A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962872604P 2019-07-10 2019-07-10
US62/872,604 2019-07-10
US202062968819P 2020-01-31 2020-01-31
US62/968,819 2020-01-31
PCT/US2020/041634 WO2021007529A1 (en) 2019-07-10 2020-07-10 Rna-targeting knockdown and replacement compositions and methods for use

Publications (1)

Publication Number Publication Date
CA3145309A1 true CA3145309A1 (en) 2021-01-14

Family

ID=71995058

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3145309A Pending CA3145309A1 (en) 2019-07-10 2020-07-10 Rna-targeting knockdown and replacement compositions and methods for use

Country Status (7)

Country Link
US (1) US20210009987A1 (https=)
EP (1) EP3997227A1 (https=)
JP (1) JP2022540446A (https=)
CN (1) CN114450031A (https=)
AU (1) AU2020310201A1 (https=)
CA (1) CA3145309A1 (https=)
WO (1) WO2021007529A1 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011160052A2 (en) 2010-06-18 2011-12-22 The University Of North Carolina At Chapel Hill Methods and compositions for sequence specific rna endonucleases
EP3901262A1 (en) * 2020-04-20 2021-10-27 Universität Regensburg Compositions for use in treating autosomal dominant best1-related retinopathies
EP4216964A4 (en) * 2020-09-24 2025-02-12 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing apoe expression
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
WO2022221278A1 (en) * 2021-04-12 2022-10-20 Locanabio, Inc. Compositions and methods comprising hybrid promoters
WO2022241059A2 (en) * 2021-05-11 2022-11-17 Mammoth Biosciences, Inc. Effector proteins and methods of use
WO2023015153A2 (en) * 2021-08-02 2023-02-09 President And Fellows Of Harvard College ANTISENSE OLIGONUCLEOTIDE TARGETING APOE ε4 AND USES THEREOF
EP4419688A2 (en) * 2021-10-21 2024-08-28 Prime Medicine, Inc. Genome editing compositions and methods for treatment of usher syndrome type 3
US12173368B2 (en) * 2022-03-11 2024-12-24 New England Biolabs, Inc. Compositions and analysis of dephosphorylated oligoribonucleotides
EP4493702A4 (en) * 2022-03-18 2026-04-01 Univ Florida METHODS AND COMPOSITIONS FOR TREATING TNNT2-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR
WO2023215761A1 (en) * 2022-05-03 2023-11-09 Tacit Therapeutics, Inc. Localization of trans-splicing nucleic acid molecules to and within the cellular nucleus
WO2024196955A2 (en) * 2023-03-20 2024-09-26 Duke University Apoe-targeted microglia-specific gene therapy for neurological diseases
WO2025043118A1 (en) * 2023-08-23 2025-02-27 Ohio State Innovation Foundation Making tiny rnas inside the cell
WO2025043278A1 (en) * 2023-08-25 2025-03-06 PYC Therapeutics Limited Gene-mediated conditions
WO2025159427A1 (ko) * 2024-01-26 2025-07-31 서울대학교산학협력단 신규한 apoe 안티센스 올리고뉴클레오타이드 및 이의 용도
WO2025217591A1 (en) * 2024-04-12 2025-10-16 Strand Therapeutics Inc. Human-derived synthetic regulators and uses thereof
WO2026010243A1 (ko) * 2024-07-03 2026-01-08 주식회사 아바타테라퓨틱스 Crispr/cas 시스템을 사용하는 best1 유전자 편집 방법

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
CN101895633A (zh) 2010-07-14 2010-11-24 中兴通讯股份有限公司 一种移动终端及其解锁方法
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
US9951349B2 (en) * 2011-09-27 2018-04-24 Yale University Compositions and methods for transient expression of recombinant RNA
DK2784157T3 (da) 2011-10-21 2019-10-21 Univ Kyushu Nat Univ Corp Designfremgangsmåde til et rna-bindende protein under anvendelse af ppr-motiv og anvendelse deraf
US9850269B2 (en) * 2014-04-25 2017-12-26 Translate Bio, Inc. Methods for purification of messenger RNA
US10330674B2 (en) 2015-01-13 2019-06-25 Massachusetts Institute Of Technology Pumilio domain-based modular protein architecture for RNA binding
EP3858992A1 (en) * 2015-03-13 2021-08-04 The Jackson Laboratory A three-component crispr/cas complex system and uses thereof
EP3289080B1 (en) * 2015-04-30 2021-08-25 The Trustees of Columbia University in the City of New York Gene therapy for autosomal dominant diseases
US10876101B2 (en) 2017-03-28 2020-12-29 Locanabio, Inc. CRISPR-associated (Cas) protein
CN108690861A (zh) * 2017-04-10 2018-10-23 肖晓莺 用于早期乳腺癌症诊断的微小核糖核酸
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
US10476825B2 (en) 2017-08-22 2019-11-12 Salk Institue for Biological Studies RNA targeting methods and compositions

Also Published As

Publication number Publication date
WO2021007529A1 (en) 2021-01-14
EP3997227A1 (en) 2022-05-18
US20210009987A1 (en) 2021-01-14
JP2022540446A (ja) 2022-09-15
AU2020310201A1 (en) 2022-01-27
CN114450031A (zh) 2022-05-06

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