Drug Profile

AAV ATP7B gene therapy - MeiraGTx

Alternative Names: AAV-ATP7B gene therapy - MeiraGTx; AAV-ATP7B opti gene therapy - MeiraGTx

Latest Information Update: 30 Mar 2023

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At a glance

Originator MeiraGTx
Class Gene therapies
Mechanism of Action Copper-transporting ATPase replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

Highest Development Phases

Preclinical Hepatolenticular degeneration

Most Recent Events

16 Mar 2023 Preclinical trials in Hepatolenticular degeneration in USA (Parenteral) prior to March 2023 (MeiraGTx pipeline, March 2023)
14 Dec 2022 Pharmacodynamics data from preclinical studies in Hepatolenticular degeneration released by MeiraGTx
02 Feb 2016 MeiraGtx files a PCT application for gene regulation platform worldwide

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