MX2020012077A - Composiciones y metodos para el tratamiento de la enfermedad de parkinson. - Google Patents

Composiciones y metodos para el tratamiento de la enfermedad de parkinson.

Info

Publication number
MX2020012077A
MX2020012077A MX2020012077A MX2020012077A MX2020012077A MX 2020012077 A MX2020012077 A MX 2020012077A MX 2020012077 A MX2020012077 A MX 2020012077A MX 2020012077 A MX2020012077 A MX 2020012077A MX 2020012077 A MX2020012077 A MX 2020012077A
Authority
MX
Mexico
Prior art keywords
methods
disease
treatment
parkinson
formulations
Prior art date
Application number
MX2020012077A
Other languages
English (en)
Inventor
Bernard Ravina
Adrian Philip Kells
Original Assignee
Voyager Therapeutics 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.)
Filing date
Publication date
Application filed by Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of MX2020012077A publication Critical patent/MX2020012077A/es

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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51—Lyases (4)
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • C12Y—ENZYMES
    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01028—Aromatic-L-amino-acid decarboxylase (4.1.1.28), i.e. tryptophane-decarboxylase
    • 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

Abstract

La presente divulgación se refiere a métodos, formulaciones y dispositivos para el suministro y la administración terapéutica de polinucleótidos que codifican AADC. La presente divulgación se refiere a métodos, formulaciones y dispositivos para el suministro y la administración terapéutica de vectores de AAV que incluyen polinucleótidos que codifican AADC. La presente divulgación se refiere a métodos, formulaciones y dispositivos para el suministro y la administración terapéutica de polinucleótidos que codifican AADC en el tratamiento de enfermedades, trastornos y afecciones neurológicas, incluida la Enfermedad de Parkinson.
MX2020012077A 2018-05-15 2019-05-15 Composiciones y metodos para el tratamiento de la enfermedad de parkinson. MX2020012077A (es)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US201862671944P 2018-05-15 2018-05-15
US201862681891P 2018-06-07 2018-06-07
US201862684384P 2018-06-13 2018-06-13
US201862691748P 2018-06-29 2018-06-29
US201862698419P 2018-07-16 2018-07-16
US201862703137P 2018-07-25 2018-07-25
US201862741021P 2018-10-04 2018-10-04
US201862748119P 2018-10-19 2018-10-19
US201862756897P 2018-11-07 2018-11-07
US201962789909P 2019-01-08 2019-01-08
US201962831400P 2019-04-09 2019-04-09
PCT/US2019/032384 WO2019222328A1 (en) 2018-05-15 2019-05-15 Compositions and methods for the treatment of parkinson's disease

Publications (1)

Publication Number Publication Date
MX2020012077A true MX2020012077A (es) 2021-03-09

Family

ID=67060450

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020012077A MX2020012077A (es) 2018-05-15 2019-05-15 Composiciones y metodos para el tratamiento de la enfermedad de parkinson.

Country Status (13)

Country Link
US (1) US20210198691A1 (es)
EP (1) EP3794126A1 (es)
JP (1) JP2021523914A (es)
KR (1) KR20210019996A (es)
CN (1) CN112424359A (es)
AU (1) AU2019268330A1 (es)
BR (1) BR112020023082A2 (es)
CA (1) CA3099306A1 (es)
MA (1) MA52631A (es)
MX (1) MX2020012077A (es)
SG (1) SG11202011296VA (es)
TW (1) TW202012628A (es)
WO (1) WO2019222328A1 (es)

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WO2018191450A2 (en) 2017-04-14 2018-10-18 National Taiwan University Hospital Gene therapy for aadc deficiency
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2021007382A1 (en) * 2019-07-09 2021-01-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
CA3168055A1 (en) 2020-02-21 2021-08-26 Emmanuel John Simons Compositions and methods for treating non-age-associated hearing impairment in a human subject
CN115607684A (zh) * 2021-07-15 2023-01-17 华中科技大学 一种内耳药物纳米载体及其应用
CN116554278A (zh) * 2022-01-30 2023-08-08 上海玮美基因科技有限责任公司 变异型腺相关病毒及其在疾病治疗中的应用

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