CO2021002505A2 - Tratamiento de mucopolisacaridosis iva - Google Patents

Tratamiento de mucopolisacaridosis iva

Info

Publication number
CO2021002505A2
CO2021002505A2 CONC2021/0002505A CO2021002505A CO2021002505A2 CO 2021002505 A2 CO2021002505 A2 CO 2021002505A2 CO 2021002505 A CO2021002505 A CO 2021002505A CO 2021002505 A2 CO2021002505 A2 CO 2021002505A2
Authority
CO
Colombia
Prior art keywords
treatment
iva
raavs
gene therapy
methods
Prior art date
Application number
CONC2021/0002505A
Other languages
English (en)
Inventor
Shunji Tomatsu
Kazuki Sawamoto
Subha Karumuthil Melethil
Olivier Danos
Original Assignee
Regenxbio Inc
The Nemours Found
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 Regenxbio Inc, The Nemours Found filed Critical Regenxbio Inc
Publication of CO2021002505A2 publication Critical patent/CO2021002505A2/es

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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
    • C12N15/63—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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61Q—SPECIFIC USE OF COSMETICS OR SIMILAR TOILETRY PREPARATIONS
    • A61Q19/00—Preparations for care of the skin
    • 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)
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/054—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06004—N-Acetylgalactosamine-6-sulfatase (3.1.6.4)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

En la presente se proporcionan métodos de terapia génica para el tratamiento de la mucopolisacaridosis tipo IVA (MPS IVA) que implican el uso de virus adenoasociados recombinantes (rAAV) para administrar N-acetilgalactosamina-6-sulfato sulfatasa humana (hGALNS) al hueso de un sujeto humano al que se le diagnosticó MPS IVA. También se proporcionan en la presente rAAV que pueden utilizarse en los métodos de terapia génica y en métodos para elaborar tales rAAV.
CONC2021/0002505A 2018-07-27 2021-02-25 Tratamiento de mucopolisacaridosis iva CO2021002505A2 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862711238P 2018-07-27 2018-07-27
US201862756880P 2018-11-07 2018-11-07
US201962799834P 2019-02-01 2019-02-01
PCT/US2019/043631 WO2020023857A1 (en) 2018-07-27 2019-07-26 Treatment of mucopolysaccharidosis iva

Publications (1)

Publication Number Publication Date
CO2021002505A2 true CO2021002505A2 (es) 2021-03-08

Family

ID=69180546

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0002505A CO2021002505A2 (es) 2018-07-27 2021-02-25 Tratamiento de mucopolisacaridosis iva

Country Status (12)

Country Link
US (1) US20210292789A1 (es)
EP (1) EP3829619A4 (es)
JP (1) JP2021531044A (es)
KR (1) KR20210040984A (es)
AU (1) AU2019312301A1 (es)
BR (1) BR112021001498A2 (es)
CA (1) CA3107800A1 (es)
CO (1) CO2021002505A2 (es)
IL (1) IL280343A (es)
SG (1) SG11202100781VA (es)
TW (1) TW202039857A (es)
WO (1) WO2020023857A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4004214A1 (en) * 2019-07-26 2022-06-01 RegenxBio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
IL294638A (en) * 2020-01-29 2022-09-01 Regenxbio Inc Treatment of mucopolysaccharidosis iva
WO2023077092A1 (en) * 2021-10-28 2023-05-04 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7863238B2 (en) * 2004-06-10 2011-01-04 Saint Louis University Proteins with an attached short peptide of acidic amino acids
US20070207139A1 (en) * 2004-06-10 2007-09-06 Saint Louis University, A Non-Profit Organization Enhancing the effect of therapeutic proteins on the central nervous system
US7972593B2 (en) * 2004-06-10 2011-07-05 Saint Louis University Delivery of therapeutic agents to the bone
CA2750811C (en) * 2009-01-30 2017-08-29 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Alanine-glyoxylate aminotransferase therapeutics
US20140271550A1 (en) * 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
US20160243260A1 (en) * 2013-10-24 2016-08-25 Uniqure Ip B.V. Treatment of neurological diseases using adeno-associated virus (AAV) comprising AAV-5 capsid proteins
EP3101125A1 (en) * 2015-06-05 2016-12-07 Laboratorios Del Dr. Esteve, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
EP3344650A4 (en) * 2015-08-31 2019-04-17 The Trustees Of The University Of Pennsylvania AAV-EPO FOR THE TREATMENT OF PETS
KR20210027266A (ko) * 2018-05-30 2021-03-10 에스테베 파마슈티칼스 에스에이 뮤코다당질축적증 ｉｖ ａ형의 치료를 위한 아데노관련 바이러스 벡터

Also Published As

Publication number Publication date
CA3107800A1 (en) 2020-01-30
AU2019312301A1 (en) 2021-02-18
JP2021531044A (ja) 2021-11-18
EP3829619A1 (en) 2021-06-09
EP3829619A4 (en) 2022-05-18
TW202039857A (zh) 2020-11-01
SG11202100781VA (en) 2021-02-25
US20210292789A1 (en) 2021-09-23
KR20210040984A (ko) 2021-04-14
BR112021001498A2 (pt) 2021-04-27
IL280343A (en) 2021-03-25
WO2020023857A1 (en) 2020-01-30

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