PE20190401A1 - Novedosas proteinas de la capside del virus adenoasociado - Google Patents

Novedosas proteinas de la capside del virus adenoasociado

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Publication number
PE20190401A1
PE20190401A1 PE2019000266A PE2019000266A PE20190401A1 PE 20190401 A1 PE20190401 A1 PE 20190401A1 PE 2019000266 A PE2019000266 A PE 2019000266A PE 2019000266 A PE2019000266 A PE 2019000266A PE 20190401 A1 PE20190401 A1 PE 20190401A1
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PE
Peru
Prior art keywords
capsid proteins
adeno
associated virus
seq
region
Prior art date
Application number
PE2019000266A
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English (en)
Inventor
Peter Cameron Colosi
Michael Lochrie
Robert Ng
Original Assignee
Biomarin Pharm Inc
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.)
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Publication date
Application filed by Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of PE20190401A1 publication Critical patent/PE20190401A1/es

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    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
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    • C12N15/86—Viral vectors
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    • 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
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)

Abstract

Referido a proteinas de la capside del virus adenoasociado (AAV) que comprenden una secuencia de aminoacidos que es al menos 95% identica a (i) cualquiera de las SEQ ID NO: 15-89, (ii) la region VP2 de cualquiera de las SEQ ID NO: 15-89, o (iii) la region VP3 de cualquiera de las SEQ ID NO: 15-89; ademas comprende un transgen que comprende un gen heterologo ligado operativamente a secuencias reguladoras que controlan la expresion del gen heterologo en una celula huesped. Tambien se refiere a metodos para elaborar los vectores de AAV que expresan las proteinas de la capside y sus usos para el tratamiento de un trastorno o enfermedad como la distrofia muscular de Duchenne.
PE2019000266A 2016-07-26 2017-07-25 Novedosas proteinas de la capside del virus adenoasociado PE20190401A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662366838P 2016-07-26 2016-07-26

Publications (1)

Publication Number Publication Date
PE20190401A1 true PE20190401A1 (es) 2019-03-13

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PE2019000266A PE20190401A1 (es) 2016-07-26 2017-07-25 Novedosas proteinas de la capside del virus adenoasociado

Country Status (15)

Country Link
US (2) US11584780B2 (es)
EP (1) EP3491008A2 (es)
JP (4) JP6994018B2 (es)
KR (2) KR102604510B1 (es)
CN (1) CN109715650B (es)
AU (2) AU2017301600B2 (es)
BR (1) BR112019001532A2 (es)
CA (1) CA3031026A1 (es)
CL (6) CL2019000167A1 (es)
IL (2) IL305149A (es)
MX (1) MX2019000962A (es)
PE (1) PE20190401A1 (es)
PH (1) PH12019500068A1 (es)
SG (1) SG11201811603WA (es)
WO (1) WO2018022608A2 (es)

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