Research programme: gene therapy - MeiraGTxAlternative Names: A-002 - MeiraGTx; A-003 - MeiraGTx; A-004; A-005; A-006 - MeiraGTx; A-007 - MeiraGTx; AAV2/8-hCARp.hCNGB3
Latest Information Update: 23 Dec 2016
At a glance
- Originator MeiraGTx
- Class Gene therapies
- Mechanism of Action Gene transference; Platelet-derived growth factor beta receptor antagonists; Vascular endothelial growth factor receptor-2 antagonists
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
On Fast track
Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.
- New Molecular Entity No
Highest Development Phases
- Preclinical Amyotrophic lateral sclerosis; Colour vision defects; Dry age-related macular degeneration; Retinitis pigmentosa; Wet age-related macular degeneration