ETX 201 - Encoded Therapeutics
Alternative Names: ETX-201 - Encoded TherapeuticsLatest Information Update: 31 Jul 2026
At a glance
- Originator Encoded Therapeutics
- Class Gene therapies
- Mechanism of Action RNA interference; UBE3A protein expression stimulants
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Orphan Drug Status
No
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Preclinical Angelman syndrome
Most Recent Events
- 13 May 2026 Pharmacodynamics and adverse event data from a preclinical study in Angelman syndrome released by Encoded Therapeutics
- 13 May 2025 Pharmacodynamics data from a preclinical study in Angelman syndrome released by Encoded Therapeutics
- 28 Apr 2025 Pharmacodynamics data from a preclinical studies in Angelman syndrome released by Encoded Therapeutics