Drug Profile
Research programme: AAV-alpha-sarcoglycan gene therapy - Genethon
Alternative Names: AAV-delivered human alpha sarcoglycan gene therapy - Genethon; AAV-vectored gene therapy; Alpha sarcoglycanopathy gene therapy - Genethon; Alpha-sarcoglycan gene therapy - Genethon; LGMD2D gene therapy - GenethonLatest Information Update: 28 Sep 2021
Price :
$50
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At a glance
- Originator Genethon
- Class Gene therapies
- Mechanism of Action Alpha sarcoglycan replacements; Gene transference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- No development reported Sarcoglycanopathies
Most Recent Events
- 28 Sep 2021 No recent reports of development identified for preclinical development in Sarcoglycanopathies in France (Parenteral)
- 18 Sep 2019 Preclinical development is ongoing in France (Genethon pipeline, September 2019)
- 01 Aug 2013 Preclinical development is ongoing in France