Drug Profile
Research programme: CNS disorder gene therapies - PTC Therapeutics/Gene Therapy Research Institution
Alternative Names: AAV based CNS disorder gene therapies - PTC Therapeutics/GTRI; AAV gene therapies - PTC Therapeutics/Gene Therapy Research Institution; CNS disorder gene therapies - GTRI/PTC Therapeutics; CNS disorder gene therapies - PTC Therapeutics/GTRI; GT-RLN; Sf9 baculovirus vector gene therapy - PTC Therapeutics/GTRILatest Information Update: 30 May 2023
Price :
$50
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At a glance
- Originator Agilis Biotherapeutics; Gene Therapy Research Institution
- Developer PTC Therapeutics
- Class Gene therapies; Neuroprotectants
- 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 CNS disorders