CO2020005116A2 - Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr - Google Patents

Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr

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Publication number
CO2020005116A2
CO2020005116A2 CONC2020/0005116A CO2020005116A CO2020005116A2 CO 2020005116 A2 CO2020005116 A2 CO 2020005116A2 CO 2020005116 A CO2020005116 A CO 2020005116A CO 2020005116 A2 CO2020005116 A2 CO 2020005116A2
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CO
Colombia
Prior art keywords
methods
compositions
treatment
ttr gene
gene editing
Prior art date
Application number
CONC2020/0005116A
Other languages
English (en)
Inventor
Walter Strapps
Shobu Odate
Reynald Michael Lescarbeau
Arti Mahendra Prakash Kanjolia
Jessica Lynn Seitzer
Original Assignee
Intellia Therapeutics Inc
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Publication date
Application filed by Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of CO2020005116A2 publication Critical patent/CO2020005116A2/es

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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
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • 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
    • 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
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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
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
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  • Biomedical Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Manufacturing & Machinery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

RESUMEN Se proporcionan las composiciones y los métodos para editar, por ejemplo, introducir ruptura bicatenaria, dentro del gen TTR. Se proporcionan las composiciones y los métodos para tratar sujetos que tienen amiloidosis asociada con la transtiretina (ATTR).
CONC2020/0005116A 2017-09-29 2020-04-24 Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr CO2020005116A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762566236P 2017-09-29 2017-09-29
US201862671902P 2018-05-15 2018-05-15
PCT/US2018/053382 WO2019067872A1 (en) 2017-09-29 2018-09-28 COMPOSITIONS AND METHODS FOR EDITING THE TTR GENE AND TREATING AMYLOID DOSE ATTR

Publications (1)

Publication Number Publication Date
CO2020005116A2 true CO2020005116A2 (es) 2020-05-15

Family

ID=63858214

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2020/0005116A CO2020005116A2 (es) 2017-09-29 2020-04-24 Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr

Country Status (15)

Country Link
US (3) US20200248180A1 (es)
EP (1) EP3688161A1 (es)
JP (2) JP2021500864A (es)
KR (1) KR20200058509A (es)
CN (2) CN111417728A (es)
AU (1) AU2018338787A1 (es)
BR (1) BR112020005287A2 (es)
CA (1) CA3077251A1 (es)
CO (1) CO2020005116A2 (es)
IL (2) IL311170A (es)
MX (1) MX2020003608A (es)
PH (1) PH12020550364A1 (es)
SG (1) SG11202002565YA (es)
TW (1) TW201932479A (es)
WO (1) WO2019067872A1 (es)

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CA3137761A1 (en) 2019-06-04 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use
CN115176001A (zh) * 2019-12-11 2022-10-11 因特利亚治疗公司 用于基因编辑的修饰的引导rna
EP4237005A1 (en) * 2020-10-28 2023-09-06 Novo Nordisk A/S Anti-transthyretin antibodies and methods of use thereof
TW202237845A (zh) 2020-12-11 2022-10-01 美商英特利亞醫療公司 用於涉及去胺作用之基因體編輯之多核苷酸、組合物及方法
CN112795595A (zh) * 2020-12-24 2021-05-14 中山大学 一种遗传性转甲状腺素蛋白淀粉样变性疾病的基因治疗系统
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WO2023028469A2 (en) * 2021-08-23 2023-03-02 The Board Of Trustees Of The Leland Stanford Junior University Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells
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Also Published As

Publication number Publication date
TW201932479A (zh) 2019-08-16
WO2019067872A1 (en) 2019-04-04
US11965165B2 (en) 2024-04-23
IL311170A (en) 2024-04-01
KR20200058509A (ko) 2020-05-27
CA3077251A1 (en) 2019-04-04
CN118530993A (zh) 2024-08-23
US20230257747A1 (en) 2023-08-17
US11795460B2 (en) 2023-10-24
JP2023029966A (ja) 2023-03-07
MX2020003608A (es) 2020-09-25
JP2021500864A (ja) 2021-01-14
BR112020005287A2 (pt) 2020-09-24
SG11202002565YA (en) 2020-04-29
AU2018338787A1 (en) 2020-04-16
US20230118592A1 (en) 2023-04-20
PH12020550364A1 (en) 2021-02-15
US20200248180A1 (en) 2020-08-06
IL273317A (en) 2020-04-30
CN111417728A (zh) 2020-07-14
EP3688161A1 (en) 2020-08-05

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