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

Terapias genicas para los trastornos lisosomales.

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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)
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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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 uno o más ácidos nucleicos inhibidores que se dirigen hacia el SCNA o una porción del mismo, hacia el TMEM106B o una porción del mismo, o hacia cualquier combinación de los anteriores. En algunas modalidades, la divulgación proporciona los 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.
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
US201762567303P 2017-10-03 2017-10-03
US201762567305P 2017-10-03 2017-10-03
PCT/US2018/054223 WO2019070891A1 (en) 2017-10-03 2018-10-03 GENE THERAPIES FOR LYSOSOMAL DISORDERS

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

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US11802294B2 (en) 2017-10-03 2023-10-31 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
MX2020004005A (es) 2017-10-03 2020-10-05 Prevail Therapeutics Inc Terapias genicas para los trastornos lisosomales.
CN114026115A (zh) 2019-04-10 2022-02-08 普利维尔治疗公司 用于溶酶体病症的基因疗法
KR20210150486A (ko) 2019-04-10 2021-12-10 프리베일 테라퓨틱스, 인크. 리소좀 장애에 대한 유전자 요법
WO2021030522A1 (en) * 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
WO2022035900A1 (en) 2020-08-10 2022-02-17 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
JP2023537903A (ja) 2020-08-10 2023-09-06 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害に対する遺伝子療法
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