Research programme: AAV-alpha-sarcoglycan gene therapy - Genethon

Alternative Names: AAV-delivered human alpha sarcoglycan gene therapy - Genethon; Alpha sarcoglycanopathy gene therapy - Genethon; Alpha-sarcoglycan gene therapy - Genethon; LGMD2D gene therapy - Genethon

Latest Information Update: 04 Nov 2017

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At a glance

Originator Genethon
Class Gene therapies
Mechanism of Action Alpha sarcoglycan replacements; Gene transference

Orphan Drug Status

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

Yes - Sarcoglycanopathies
New Molecular Entity No

Highest Development Phases

No development reported Sarcoglycanopathies

Most Recent Events

04 Nov 2017 No recent reports of development identified for preclinical development in Sarcoglycanopathies in France (Parenteral)
01 Aug 2013 Preclinical development is ongoing in France
26 Jul 2011 Preclinical development is ongoing in France

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