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-168 and DT-216P2. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
DT-168 - FDA Regulatory Timeline and 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
- Estimated Event Date Range:
- May 2, 2025 - May 2, 2025
- Target Action Date:
- May 02, 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
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:
- 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
Free DSGN Stock Alerts