ES3037550T3 - Dystrophin gene exon deletion using engineered nucleases - Google Patents

Dystrophin gene exon deletion using engineered nucleases

Info

Publication number
ES3037550T3
ES3037550T3 ES20189872T ES20189872T ES3037550T3 ES 3037550 T3 ES3037550 T3 ES 3037550T3 ES 20189872 T ES20189872 T ES 20189872T ES 20189872 T ES20189872 T ES 20189872T ES 3037550 T3 ES3037550 T3 ES 3037550T3
Authority
ES
Spain
Prior art keywords
nuclease
exon
dna
gene
sequence
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.)
Active
Application number
ES20189872T
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English (en)
Spanish (es)
Inventor
Derek Jantz
James Jefferson Smith
Michael G Nicholson
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.)
Precision Biosciences Inc
Original Assignee
Precision Biosciences Inc
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Publication date
Application filed by Precision Biosciences Inc filed Critical Precision Biosciences Inc
Application granted granted Critical
Publication of ES3037550T3 publication Critical patent/ES3037550T3/es
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    • 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
    • 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
    • A61K38/54—Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification 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
    • 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 [RNase]; Deoxyribonucleases [DNase]
    • 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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
ES20189872T 2014-03-12 2015-03-12 Dystrophin gene exon deletion using engineered nucleases Active ES3037550T3 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201461951648P 2014-03-12 2014-03-12

Publications (1)

Publication Number Publication Date
ES3037550T3 true ES3037550T3 (en) 2025-10-03

Family

ID=54072591

Family Applications (2)

Application Number Title Priority Date Filing Date
ES20189872T Active ES3037550T3 (en) 2014-03-12 2015-03-12 Dystrophin gene exon deletion using engineered nucleases
ES15761320T Active ES2821149T3 (es) 2014-03-12 2015-03-12 Eliminación del exón del gen de la distrofina mediante nucleasas modificadas genéticamente

Family Applications After (1)

Application Number Title Priority Date Filing Date
ES15761320T Active ES2821149T3 (es) 2014-03-12 2015-03-12 Eliminación del exón del gen de la distrofina mediante nucleasas modificadas genéticamente

Country Status (8)

Country Link
US (6) US20170106055A1 (Direct)
EP (3) EP3858376B1 (Direct)
JP (5) JP2017512767A (Direct)
AU (3) AU2015229299A1 (Direct)
CA (1) CA2942268A1 (Direct)
DK (1) DK3116533T3 (Direct)
ES (2) ES3037550T3 (Direct)
WO (1) WO2015138739A2 (Direct)

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Also Published As

Publication number Publication date
WO2015138739A3 (en) 2015-10-22
JP2021088563A (ja) 2021-06-10
WO2015138739A2 (en) 2015-09-17
US20240156919A1 (en) 2024-05-16
JP2017512767A (ja) 2017-05-25
US20250152679A1 (en) 2025-05-15
EP3116533A4 (en) 2018-04-18
ES2821149T3 (es) 2021-04-23
AU2015229299A1 (en) 2016-09-22
WO2015138739A8 (en) 2015-11-19
EP3858376C0 (en) 2025-07-30
EP3116533A2 (en) 2017-01-18
CA2942268A1 (en) 2015-09-17
AU2020260387A1 (en) 2020-11-19
EP3116533B1 (en) 2020-08-12
JP2025013784A (ja) 2025-01-28
EP3858376B1 (en) 2025-07-30
JP6832995B2 (ja) 2021-02-24
JP2019214609A (ja) 2019-12-19
US20170106055A1 (en) 2017-04-20
EP4659765A3 (en) 2026-02-18
DK3116533T3 (da) 2020-08-24
US20190269762A1 (en) 2019-09-05
AU2024202080A1 (en) 2024-04-18
EP4659765A2 (en) 2025-12-10
US20210145940A1 (en) 2021-05-20
EP3858376A1 (en) 2021-08-04
US20190365870A1 (en) 2019-12-05
JP2023002555A (ja) 2023-01-10

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