MX2020011748A - Terapias genicas para los trastornos lisosomales. - Google Patents

Terapias genicas para los trastornos lisosomales.

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Publication number
MX2020011748A
MX2020011748A MX2020011748A MX2020011748A MX2020011748A MX 2020011748 A MX2020011748 A MX 2020011748A MX 2020011748 A MX2020011748 A MX 2020011748A MX 2020011748 A MX2020011748 A MX 2020011748A MX 2020011748 A MX2020011748 A MX 2020011748A
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MX
Mexico
Prior art keywords
gene therapies
lysosomal disorders
lysosomal
disorders
therapies
Prior art date
Application number
MX2020011748A
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 MX2020011748A publication Critical patent/MX2020011748A/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.
MX2020011748A 2017-10-03 2020-07-13 Terapias genicas para los trastornos lisosomales. MX2020011748A (es)

Applications Claiming Priority (2)

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US201762567303P 2017-10-03 2017-10-03
US201762567305P 2017-10-03 2017-10-03

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MX2020011748A true MX2020011748A (es) 2021-01-08

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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) CN112501208A (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)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020003965A (es) 2017-10-03 2020-10-05 Prevail Therapeutics Inc Terapias genicas para los trastornos lisosomales.
KR20200078513A (ko) 2017-10-03 2020-07-01 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
JP7254815B2 (ja) 2017-10-03 2023-04-10 プリベイル セラピューティクス，インコーポレーテッド ライソゾーム病の遺伝子治療
JP2022526021A (ja) 2019-04-10 2022-05-20 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
EP3953377A1 (en) 2019-04-10 2022-02-16 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
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
WO2022035903A2 (en) 2020-08-10 2022-02-17 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CA3190864A1 (en) 2020-08-10 2022-02-17 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
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