This section highlights FDA-related milestones and regulatory updates for drugs developed by Design Therapeutics (DSGN).
Over the past two years, Design Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
DT-216P2, DT-818, and DT-168. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
DT-216P2 FDA Regulatory Timeline and Events
DT-216P2 is a drug developed by Design Therapeutics for the following indication: In patients with Friedreich ataxia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- DT-216P2
- Announced Date:
- May 18, 2026
- Indication:
- In patients with Friedreich ataxia
Announcement
Design Therapeutics, Inc. announced positive biomarker and clinical data from the ongoing Phase 1/2 RESTORE-FA trial evaluating DT-216P2 in patients with Friedreich ataxia (FA). DT-216P2 is a GeneTAC® small-molecule therapeutic candidate designed to increase frataxin (FXN) expression by targeting the GAA repeat expansion in the FXN gene, the genetic root cause of FA.
AI Summary
Design Therapeutics said its Phase 1/2 RESTORE-FA trial of DT-216P2 showed encouraging results in patients with Friedreich ataxia, a rare genetic disease caused by low frataxin levels. After four weeks of weekly IV dosing, the treatment led to dose-dependent improvements in several clinical measures and increased frataxin mRNA and protein in both blood and muscle tissue. These findings suggest DT-216P2 is reaching the disease target and may be helping restore the underlying problem in FA.
The drug was generally well-tolerated across the dose groups studied, which included 16 patients in total. DT-216P2 is a GeneTAC small-molecule therapy designed to act on the GAA repeat expansion in the FXN gene, the genetic root cause of FA. Based on the data, the company plans to move forward with registrational development and expects to share more details on its strategy in the fourth quarter of 2026.
Read Announcement- Drug:
- DT-216P2
- Announced Date:
- May 17, 2026
- Indication:
- In patients with Friedreich ataxia
Announcement
Design Therapeutics, Inc. will announce data from the ongoing Phase 1/2 RESTORE-FA trial evaluating DT-216P2 in patients with Friedreich's ataxia (FA) on Monday, May 18, 2026. Management will host a conference call and webcast at 8:00 a.m. ET.
AI Summary
Design Therapeutics, Inc. said it will release new data from its ongoing Phase 1/2 RESTORE-FA trial of DT-216P2 in patients with Friedreich’s ataxia (FA) on Monday, May 18, 2026. The company expects the update to add more information about how the treatment is performing in this early-stage study. Friedreich’s ataxia is a rare genetic disease that affects the nervous system and can lead to muscle weakness, balance problems, and trouble with coordination.
Management will discuss the results during a conference call and webcast at 8:00 a.m. ET on the same day. Investors and others following the program will be looking for details on the trial’s progress, including safety and early signs of benefit. The announcement reflects continued development work for DT-216P2 as a possible treatment option for people living with FA.
Read Announcement- Drug:
- DT-216P2
- Announced Date:
- June 4, 2025
- Indication:
- In patients with Friedreich ataxia
Announcement
Design Therapeutics, Inc. announced that the first Friedreich ataxia (FA) patient has been dosed via intravenous (IV) infusion in its RESTORE-FA (Reactivating Expression Suppressed Through Overcoming Repeat Expansion for FA) open-label Phase 1/2 multiple-ascending dose (MAD) clinical trial of DT-216P2.
AI Summary
Design Therapeutics, Inc. announced a significant milestone in its RESTORE-FA trial by dosing the first Friedreich ataxia (FA) patient via intravenous infusion. This open-label Phase 1/2 multiple-ascending dose (MAD) study is focused on evaluating DT-216P2, a gene-targeted small molecule therapy that aims to overcome the GAA repeat expansion mutation at the root of FA. The trial will closely monitor the safety, tolerability, pharmacokinetics, and pharmacodynamics of this treatment, with a particular emphasis on its ability to boost the production of the frataxin protein, which is crucial for cell function. To date, no adverse events, including injection site complications, have been reported. Researchers expect that data collected over 12 weeks of dosing will provide valuable insights into increasing frataxin expression and offer new hope for improved treatment options for patients with Friedreich ataxia.
Read Announcement
DT-818 FDA Regulatory Events
DT-818 is a drug developed by Design Therapeutics for the following indication: GeneTAC® candidates.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- DT-818
- Announced Date:
- November 5, 2025
- Indication:
- GeneTAC® candidates
Announcement
Design Therapeutics, Inc. announced progress and updated milestones across its portfolio of GeneTAC® candidates in addition to reporting financial results for the third quarter of 2025.
AI Summary
Design Therapeutics, Inc. reported progress and updated milestones for its GeneTAC® drug candidates, along with third-quarter 2025 financial results. The company advanced DT-818, a small molecule for myotonic dystrophy type-1 (DM1), gaining ex-U.S. regulatory clearance. A Phase 1 multiple-ascending dose trial in Australia is set to begin in early 2026, with splicing correction data expected in 2027.
Trials for DT-216P2 in Friedreich ataxia and DT-168 in Fuchs endothelial corneal dystrophy are ongoing outside the U.S. Both programs aim for data readouts in the second half of 2026. Design continues preclinical work on Huntington’s disease candidates to expand its pipeline.
Financially, R&D expenses for Q3 were $14.6 million, and general and administrative costs were $4.7 million. The net loss totaled $17.0 million. As of September 30, 2025, Design held $206.0 million in cash, cash equivalents and investment securities to support further development.
Read Announcement
DT-168 FDA Regulatory Events
DT-168 is a drug developed by Design Therapeutics for the following indication: For Fuchs endothelial corneal dystrophy (FECD).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- DT-168
- Announced Date:
- May 1, 2025
- Target Action Date:
- May 2, 2025
- Indication:
- For Fuchs endothelial corneal dystrophy (FECD)
Announcement
Design Therapeutics, Inc. announced favorable data from a Phase 1 single- and multiple-ascending dose (SAD/MAD) trial of DT-168 in healthy volunteers, which will be presented on May 2, 2025 at Eyecelerator @ Park City 2025, an event backed by the American Academy of Ophthalmology highlighting industry advancements and innovative new products disrupting eye care.
AI Summary
Design Therapeutics, Inc. announced promising results from its Phase 1 SAD/MAD (single- and multiple-ascending dose) trial of DT-168 in healthy volunteers. DT-168 is an innovative eye drop designed to target and lower the expression of the mutant TCF4 gene, which is a key factor in Fuchs endothelial corneal dystrophy (FECD), a condition that causes progressive vision loss.
The trial showed that DT-168 was well-tolerated, with no serious or ocular adverse events reported and no withdrawals due to side effects. These favorable findings highlight DT-168’s potential as the first disease-modifying treatment for FECD. The data will be formally presented on May 2, 2025, at Eyecelerator @ Park City 2025, an event supported by the American Academy of Ophthalmology that focuses on breakthroughs and innovation in eye care.
Read Announcement- Drug:
- DT-168
- Announced Date:
- April 21, 2025
- Indication:
- For Fuchs endothelial corneal dystrophy (FECD)
Announcement
Design Therapeutics, Inc. announced that it will present an update on the progress of its DT-168 program for Fuchs endothelial corneal dystrophy (FECD) at Eyecelerator @ Park City 2025 on Friday, May 2, 2025, at 1:30 p.m. MT in Park City, UT.
AI Summary
Design Therapeutics, Inc. announced that it will update progress on its DT-168 program for Fuchs endothelial corneal dystrophy (FECD) at the Eyecelerator @ Park City 2025 conference. The presentation is scheduled for Friday, May 2, 2025, at 1:30 p.m. MT in Park City, UT. During this update, the company plans to share safety findings from its Phase 1 trial, which involved both single- and multiple-ascending doses of DT-168 in healthy adult volunteers. Additionally, the company will outline plans for advancing to a Phase 2 clinical trial in FECD patients.
DT-168 is formulated as an eye drop and aims to target the CTG repeat expansion in the TCF4 gene. This targeted approach seeks to reduce the mutant gene product that causes corneal endothelial cell dysfunction, offering hope for a potential treatment for FECD, a condition that currently lacks disease-modifying therapies.
Read Announcement
