Drug Profile
GTX 102
Alternative Names: GTX-102- GeneTx Biotherapeutics; GTX102Latest Information Update: 22 Apr 2024
Price :
$50
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At a glance
- Originator Texas A&M AgriLife Research; Texas A&M University
- Developer GeneTx Biotherapeutics; Ultragenyx Pharmaceutical
- Class Antisense oligonucleotides; Neuroprotectants
- Mechanism of Action Gene silencing; UBE3A protein expression stimulants
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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 Yes
Highest Development Phases
- Phase I/II Angelman syndrome
Most Recent Events
- 15 Apr 2024 GTX 102 receives Orphan Drug status for Angelman syndrome in European Union
- 15 Apr 2024 Ultragenyx Pharmaceutical plans an end-of-phase II meeting with the US FDA and other health authorities for phase III trial in Angelman syndrome
- 15 Apr 2024 Efficacy and adverse events data from a phase I/II trial in Angelman syndrome released by Ultragenyx Pharmaceutical