Product Description
DT-216 is a new molecule to activate transcription of the FXN gene and restore production of the frataxin protein. More specifically, it is a GeneTAC™ gene targeted chimera small molecule designed to specifically target the GAA repeat expansion mutation and restore FXN gene expression. (Sourced from: https://curefa.org/clinical-trials/clinical-trials-active-enrolling)
Mechanisms of Action: Gene Therapy,FXN
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: Intravenous
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Design Therapeutics
Company Location: CARLSBAD CA 92011
Company CEO: João Siffert
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: United States
Active Clinical Trial Count: 1
Highest Development Phases
Phase 1: Cerebellar Ataxia|Friedreich Ataxia
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| DTX-216-102 | P1 |
Completed |
Cerebellar Ataxia|Friedreich Ataxia |
2023-08-25 |