Drug Profile
GTX 102
Alternative Names: GTX-102- GeneTx Biotherapeutics; GTX102Latest Information Update: 07 Feb 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
- 06 Feb 2024 GTX 102 receives Priority Medicine (PRIME) status for Angelman syndrome (In children, In adolescents) in European Union
- 03 Jan 2024 Ultragenyx Pharmaceutical completes enrolment in a phase-I/II trial in Angelman syndrome (In adolescents, In children) in US, Canada, France, Australia, UKFrance, Germany, Israel and Spain (Intrathecal) (NCT04259281)
- 03 Jan 2024 Ultragenyx Pharmaceutical plans a phase III trial for Angelman syndrome