MX2020004005A - Terapias genicas para los trastornos lisosomales. - Google Patents

Terapias genicas para los trastornos lisosomales.

Info

Publication number
MX2020004005A
MX2020004005A MX2020004005A MX2020004005A MX2020004005A MX 2020004005 A MX2020004005 A MX 2020004005A MX 2020004005 A MX2020004005 A MX 2020004005A MX 2020004005 A MX2020004005 A MX 2020004005A MX 2020004005 A MX2020004005 A MX 2020004005A
Authority
MX
Mexico
Prior art keywords
disease
gene therapies
expression constructs
disclosure provides
lysosomal disorders
Prior art date
Application number
MX2020004005A
Other languages
English (en)
Spanish (es)
Inventor
Asa Abeliovich
Laura Heckman
Herve Rhinn
Original Assignee
Prevail 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 Prevail Therapeutics Inc filed Critical Prevail Therapeutics Inc
Publication of MX2020004005A publication Critical patent/MX2020004005A/es

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    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • 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
    • 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
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    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • 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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    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
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    • 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
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MX2020004005A 2017-10-03 2018-10-03 Terapias genicas para los trastornos lisosomales. MX2020004005A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762567305P 2017-10-03 2017-10-03
US201762567303P 2017-10-03 2017-10-03
PCT/US2018/054223 WO2019070891A1 (en) 2017-10-03 2018-10-03 GENE THERAPIES FOR LYSOSOMAL DISORDERS

Publications (1)

Publication Number Publication Date
MX2020004005A true MX2020004005A (es) 2020-10-05

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MX2020004005A MX2020004005A (es) 2017-10-03 2018-10-03 Terapias genicas para los trastornos lisosomales.
MX2020011748A MX2020011748A (es) 2017-10-03 2020-07-13 Terapias genicas para los trastornos lisosomales.

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MX2020011748A MX2020011748A (es) 2017-10-03 2020-07-13 Terapias genicas para los trastornos lisosomales.

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Country Link
US (3) US11807849B2 (https=)
EP (1) EP3692151A4 (https=)
JP (4) JP2020537542A (https=)
KR (2) KR20200078513A (https=)
CN (2) CN111465691A (https=)
AU (3) AU2018346102B2 (https=)
BR (1) BR112020006671A2 (https=)
CA (1) CA3078501A1 (https=)
IL (2) IL273768A (https=)
MX (2) MX2020004005A (https=)
WO (1) WO2019070891A1 (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3692075A4 (en) 2017-10-03 2022-02-09 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
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