CA2946392A1 - Ldlr variants and their use in compositions for reducing cholesterol levels - Google Patents

Ldlr variants and their use in compositions for reducing cholesterol levels Download PDF

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Publication number
CA2946392A1
CA2946392A1 CA2946392A CA2946392A CA2946392A1 CA 2946392 A1 CA2946392 A1 CA 2946392A1 CA 2946392 A CA2946392 A CA 2946392A CA 2946392 A CA2946392 A CA 2946392A CA 2946392 A1 CA2946392 A1 CA 2946392A1
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Prior art keywords
hldlr
amino acid
seq
modified
vector
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Pending
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CA2946392A
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English (en)
French (fr)
Inventor
James M. Wilson
Daniel J. Rader
Suryanrayan SOMANATHAN
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA2946392A 2014-04-25 2015-04-24 Ldlr variants and their use in compositions for reducing cholesterol levels Pending CA2946392A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461984620P 2014-04-25 2014-04-25
US61/984,620 2014-04-25
US201462022627P 2014-07-09 2014-07-09
US62/022,627 2014-07-09
PCT/US2015/027572 WO2015164778A1 (en) 2014-04-25 2015-04-24 Ldlr variants and their use in compositions for reducing cholesterol levels

Publications (1)

Publication Number Publication Date
CA2946392A1 true CA2946392A1 (en) 2015-10-29

Family

ID=53055129

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2946392A Pending CA2946392A1 (en) 2014-04-25 2015-04-24 Ldlr variants and their use in compositions for reducing cholesterol levels

Country Status (7)

Country Link
US (2) US11555059B2 (enExample)
EP (2) EP3134431B1 (enExample)
JP (3) JP6741590B2 (enExample)
BR (1) BR112016024379A2 (enExample)
CA (1) CA2946392A1 (enExample)
ES (1) ES2876409T3 (enExample)
WO (1) WO2015164778A1 (enExample)

Cited By (1)

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CN110769845A (zh) * 2017-04-21 2020-02-07 精密生物科学公司 对pcsk9基因中的识别序列具有特异性的工程化大范围核酸酶

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CA3008142A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
WO2017100676A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
KR20190118163A (ko) 2017-02-20 2019-10-17 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법

Cited By (2)

* Cited by examiner, † Cited by third party
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CN110769845A (zh) * 2017-04-21 2020-02-07 精密生物科学公司 对pcsk9基因中的识别序列具有特异性的工程化大范围核酸酶
CN110769845B (zh) * 2017-04-21 2023-08-15 精密生物科学公司 对pcsk9基因中的识别序列具有特异性的工程化大范围核酸酶

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JP7280853B2 (ja) 2023-05-24
BR112016024379A2 (pt) 2017-10-10
EP3919508A1 (en) 2021-12-08
ES2876409T3 (es) 2021-11-12
EP3134431B1 (en) 2021-04-07
JP2020120673A (ja) 2020-08-13
EP3134431A1 (en) 2017-03-01
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