tgAAG 76
Alternative Names: AAV-RPE65; Leber's hereditary optic atrophy gene therapy - AmpliPhi Biosciences; rAAV2/2.hRPE65p.hRPE65; RPE65 gene therapy - AmpliPhi Biosciences; tgAAG76Latest Information Update: 31 Mar 2022
At a glance
- Originator Targeted Genetics Corporation
- Developer AmpliPhi Biosciences Corporation; University College London
- Class Gene therapies
- Mechanism of Action 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
- Discontinued Leber's hereditary optic atrophy
Most Recent Events
- 08 Apr 2011 Genzyme Corporation has been acquired by sanofi-aventis
- 09 Mar 2011 Targeted Genetics Corporation is now called AmpliPhi Biosciences
- 11 Nov 2010 Phase I/II development is ongoing in United Kingdom