Drugs / DT-216
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT05573698 | Sep 2022 → Aug 2023 | Friedreich ataxia | Design Therapeutics, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT05285540 | Mar → Dec 2022 | Friedreich ataxia | Design Therapeutics, Inc. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2023-08-14 | Design Therapeutics, Inc. | Results Design Therapeutics Reports Initial Results from Phase 1 Multiple-Ascending Dose Study of DT-216 for the Treatment of Friedrich Ataxia designtx.com ↗
As of a data cutoff date of August 7, 2023, results showed that DT-216 was generally well-tolerated and achieved a statistically significant and dose-related increase in frataxin (FXN) mRNA levels in skeletal muscle biopsies. |
| 2023-08-14 | Design Therapeutics, Inc. | Results Design Therapeutics Provides Pipeline Update and Reports Second Quarter 2023 Financial Results designtx.com ↗ |
| 2022-12-07 | Design Therapeutics, Inc. | Results Design Therapeutics Reports Positive Data from Single-Ascending Dose Trial of DT-216 for the Treatment of Friedreich Ataxia and Portfolio Progress designtx.com ↗
The results show that DT-216 was generally well-tolerated and able to overcome the frataxin (FXN) transcription impairment that causes FA, with a greater than two-fold increase in FXN mRNA in the cohort with the highest response. |
| 2022-10-17 | Design Therapeutics, Inc. | Results Design Therapeutics to Present Preclinical Data Highlighting the Potential of its GeneTAC™ Small Molecule, DT-216, for the Treatment of Friedreich Ataxia at ICAR 2022 designtx.com ↗
Administration of DT-216 increased FXN mRNA by approximately 10-fold in peripheral blood mononuclear cells (PBMCs) collected directly from FA patients (N=23 donors with >100 to > 1500 GAA repeats) |
| 2022-02-28 | Design Therapeutics, Inc. | Regulatory Design Therapeutics Announces FDA Clearance of Investigational New Drug Application for First GeneTAC™ Molecule for Friedreich Ataxia designtx.com ↗
today announced that its Investigational New Drug Application (IND) for its lead candidate, DT-216, for the treatment of Friedreich ataxia (FA), was cleared by the U.S. Food and Drug Administration (FDA). |
All press releases naming this drug 6 releases
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | DT-216 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05573698 ↗ |
| Route | Intravenous | “DT-216 will be administered by intravenous (IV) injection” NCT05285540 ↗ |