AT GTX 701
Alternative Names: AAV based Fabry gene therapy - Amicus Therapeutics/University of Pennsylvania; AT-GTX-701Latest Information Update: 28 Mar 2023
At a glance
- Originator Amicus Therapeutics
- Developer Amicus Therapeutics; University of Pennsylvania
- 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
- No development reported Fabry's disease
Most Recent Events
- 28 Mar 2023 No recent reports of development identified for preclinical development in Fabry's-disease in USA (Parenteral)
- 31 Dec 2022 Amicus Therapeutics and University of Pennsylvania terminates its research, collaboration and license agreement for the development of AAV based gene therapies for Fabry's disease and Pompe disease
- 10 Jan 2022 Amicus Therapeutics anticipates an IND for Fabry's disease, in 2023