BR112018076127A2 - disferlina truncada para tratamento da disferlinopatia - Google Patents

disferlina truncada para tratamento da disferlinopatia

Info

Publication number
BR112018076127A2
BR112018076127A2 BR112018076127-3A BR112018076127A BR112018076127A2 BR 112018076127 A2 BR112018076127 A2 BR 112018076127A2 BR 112018076127 A BR112018076127 A BR 112018076127A BR 112018076127 A2 BR112018076127 A2 BR 112018076127A2
Authority
BR
Brazil
Prior art keywords
dysferlinopathy
treatment
dysferlin
truncated
nucleic acid
Prior art date
Application number
BR112018076127-3A
Other languages
English (en)
Inventor
Louis Hirsch Matthew
Bryan Sutton R.
Original Assignee
The University Of North Carolina At Chapel Hill
Texas Tech University System
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 The University Of North Carolina At Chapel Hill, Texas Tech University System filed Critical The University Of North Carolina At Chapel Hill
Publication of BR112018076127A2 publication Critical patent/BR112018076127A2/pt

Links

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • 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
    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2750/14051—Methods of production or purification of viral material
    • C12N2750/14052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Environmental Sciences (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

esta invenção refere-se a ácido nucleico e proteína de disferlina truncada, vetores (por exemplo, vetores do vírus adeno-associado) compreendendo o ácido nucleico e métodos de uso do mesmo para liberação de disferlina a uma célula ou a um indivíduo e tratamento de disferlinopatia.
BR112018076127-3A 2016-06-17 2017-06-16 disferlina truncada para tratamento da disferlinopatia BR112018076127A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662351701P 2016-06-17 2016-06-17
US62/351,701 2016-06-17
PCT/US2017/037822 WO2017218866A1 (en) 2016-06-17 2017-06-16 Truncated dysferlin for treatment of dysferlinopathy

Publications (1)

Publication Number Publication Date
BR112018076127A2 true BR112018076127A2 (pt) 2019-03-26

Family

ID=60663818

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018076127-3A BR112018076127A2 (pt) 2016-06-17 2017-06-16 disferlina truncada para tratamento da disferlinopatia

Country Status (11)

Country Link
US (1) US20200010521A1 (pt)
EP (1) EP3472320A4 (pt)
JP (1) JP7202895B2 (pt)
KR (1) KR20190028389A (pt)
CN (1) CN109563512A (pt)
AU (1) AU2017285423A1 (pt)
BR (1) BR112018076127A2 (pt)
CA (1) CA3027149A1 (pt)
IL (1) IL263577A (pt)
RU (1) RU2019100990A (pt)
WO (1) WO2017218866A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202208407A (zh) * 2020-05-13 2022-03-01 美國全美兒童醫院之研究學會 使用質膜修復蛋白(dysferlin)雙載體之基因療法
EP4086276A1 (en) * 2021-05-03 2022-11-09 Université d'Aix-Marseille Composition for treating dysferlinopathy

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000011157A1 (en) * 1998-08-25 2000-03-02 The General Hospital Corporation Dysferlin, a gene mutated in distal myopathy and limb girdle muscular dystrophy
WO2007089632A2 (en) * 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US20090104155A1 (en) * 2006-04-21 2009-04-23 Goodrich Laurie R Treatment of connective tissue disorders
FR2919305B1 (fr) * 2007-07-26 2009-09-18 Genethon Ass Loi De 1901 Vecteurs viraux adeno-associes pour l'expression de la dysferline.
RU2527073C2 (ru) * 2012-12-24 2014-08-27 Общество с ограниченной ответственностью "НекстГен" Кодон-оптимизированная кднк, кодирующая дисферлин человека, генно-инженерная конструкция, рекомбинантный аденовирус и фармацевтическая композиция для лечения дисферлинопатий

Also Published As

Publication number Publication date
CN109563512A (zh) 2019-04-02
AU2017285423A1 (en) 2019-01-03
JP7202895B2 (ja) 2023-01-12
US20200010521A1 (en) 2020-01-09
EP3472320A1 (en) 2019-04-24
IL263577A (en) 2019-01-31
KR20190028389A (ko) 2019-03-18
WO2017218866A1 (en) 2017-12-21
RU2019100990A (ru) 2020-07-17
RU2019100990A3 (pt) 2020-11-18
EP3472320A4 (en) 2020-04-08
JP2019517816A (ja) 2019-06-27
CA3027149A1 (en) 2017-12-21

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Legal Events

Date Code Title Description
B350 Update of information on the portal [chapter 15.35 patent gazette]
B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]
B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

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