MX2020011745A - Terapias genicas para los trastornos lisosomales. - Google Patents

Terapias genicas para los trastornos lisosomales.

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Publication number
MX2020011745A
MX2020011745A MX2020011745A MX2020011745A MX2020011745A MX 2020011745 A MX2020011745 A MX 2020011745A MX 2020011745 A MX2020011745 A MX 2020011745A MX 2020011745 A MX2020011745 A MX 2020011745A MX 2020011745 A MX2020011745 A MX 2020011745A
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MX
Mexico
Prior art keywords
gene therapies
lysosomal disorders
lysosomal
disorders
therapies
Prior art date
Application number
MX2020011745A
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English (en)
Inventor
Asa Abeliovich
Laura Heckman
Herve Rhinn
Original Assignee
Prevail Therapeutics Inc
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Publication date
Application filed by Prevail Therapeutics Inc filed Critical Prevail Therapeutics Inc
Publication of MX2020011745A publication Critical patent/MX2020011745A/es

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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C—CHEMISTRY; METALLURGY
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
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    • C07—ORGANIC CHEMISTRY
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
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    • 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
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • 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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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vectore
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    • 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

Abstract

La divulgación se refiere, en algunos aspectos, a composiciones y métodos para el tratamiento de las enfermedades asociadas con una función lisosomal aberrante, por ejemplo, la enfermedad de Parkinson (PD) y la enfermedad de Gaucher. En algunas modalidades, la divulgación proporciona las construcciones de expresión que comprenden un transgén que codifica la beta-glucocerebrosidasa (GBA) o una porción de la misma, la proteína de membrana lisosomal 2 (LIMP2), prosaposina, o cualquier combinación de las anteriores. En algunas modalidades, la divulgación proporciona métodos para el tratamiento de la enfermedad de Parkinson por medio de la administración de las construcciones de expresión a un sujeto que lo necesite.
MX2020011745A 2017-10-03 2020-07-13 Terapias genicas para los trastornos lisosomales. MX2020011745A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762567296P 2017-10-03 2017-10-03

Publications (1)

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MX2020011745A true MX2020011745A (es) 2021-01-08

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MX2020003557A MX2020003557A (es) 2017-10-03 2018-10-03 Terapias genicas para los trastornos lisosomales.
MX2020011745A MX2020011745A (es) 2017-10-03 2020-07-13 Terapias genicas para los trastornos lisosomales.

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US (4) US11802294B2 (es)
EP (1) EP3692075A4 (es)
JP (2) JP7254815B2 (es)
KR (1) KR20200078512A (es)
CN (1) CN111542549A (es)
AU (2) AU2018346104B2 (es)
BR (1) BR112020006633A2 (es)
CA (1) CA3078371A1 (es)
IL (2) IL273776A (es)
MX (2) MX2020003557A (es)
WO (1) WO2019070893A1 (es)

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AU2018346104B2 (en) 2023-07-27
EP3692075A4 (en) 2022-02-09
JP2020537543A (ja) 2020-12-24
AU2018346104A1 (en) 2020-04-30
JP7254815B2 (ja) 2023-04-10
WO2019070893A1 (en) 2019-04-11
US10837028B2 (en) 2020-11-17
US20210332385A1 (en) 2021-10-28
MX2020003557A (es) 2020-10-07
KR20200078512A (ko) 2020-07-01
CN111542549A (zh) 2020-08-14
US20200071726A1 (en) 2020-03-05
CA3078371A1 (en) 2019-04-11
AU2020205228B2 (en) 2023-09-07
EP3692075A1 (en) 2020-08-12
IL278868A (en) 2021-01-31
US20200231954A1 (en) 2020-07-23
BR112020006633A2 (pt) 2020-10-06
JP2023086740A (ja) 2023-06-22
US20210010032A1 (en) 2021-01-14
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