Design Therapeutics, Inc. Stock Price, News & Analysis

Design Therapeutics (NASDAQ: DSGN) announced nomination of DT-818 as its development candidate for myotonic dystrophy type-1 (DM1) and obtained ex‑US regulatory clearance to start clinical development. The company plans a Phase 1 multiple-ascending dose trial in Australia with patient dosing to begin in the first half of 2026 and splicing data expected in 2027. Design continues dosing in its RESTORE-FA trial of DT-216P2 and an ongoing Phase 2 biomarker trial of DT-168, both with data anticipated in the second half of 2026. Preclinical DT-818 showed >90% reduction in toxic RNA foci and selective targeting of mutant DMPK. Cash, cash equivalents and investment securities totaled $206.0 million as of September 30, 2025, with a reported Q3 2025 net loss of $17.0 million.

Design Therapeutics (Nasdaq: DSGN), a clinical-stage biotech company, has appointed Justin Gover to its Board of Directors. Gover brings over 25 years of biotech leadership experience, notably as the founding CEO of GW Pharmaceuticals, which he led to a $7 billion acquisition by Jazz Pharmaceuticals in 2021.

The appointment comes as Dr. Arsani William steps down from the Board. William, who joined in 2021, helped guide Design through its IPO and clinical pipeline advancement. Gover currently serves on the boards of Compass Pathways and Xenon Pharmaceuticals, bringing valuable experience in transforming innovative science into commercial medicines.

Design Therapeutics (NASDAQ:DSGN), a clinical-stage biotech company focused on developing treatments for degenerative genetic diseases, has announced its participation in the upcoming 2025 Cantor Global Healthcare Conference. The company's management will engage in a fireside chat on September 4, 2025, at 10:55 a.m. ET in New York.

Investors can access the presentation through a live webcast available on the company's website, which will remain archived for at least 30 days after the event.

Design Therapeutics (NASDAQ:DSGN) reported progress in its GeneTAC® programs and Q2 2025 financial results. The company announced encouraging early pharmacokinetics data for DT-216P2 in Friedreich Ataxia (FA), showing favorable translation from non-human primates to humans via both intravenous and subcutaneous administration.

Key developments include the ongoing RESTORE-FA Phase 1/2 trial of DT-216P2 outside the US, while addressing an FDA clinical hold notice. The company also initiated a Phase 2 biomarker trial for DT-168 in Fuchs Endothelial Corneal Dystrophy (FECD) patients. Design reported $216.3 million in cash and securities, with a Q2 net loss of $19.1 million.

[ "Favorable pharmacokinetics data translation from non-human primates to humans for DT-216P2", "DT-216P2 showed improved exposure and PK parameters compared to previous formulation", "Strong cash position of $216.3 million to support pipeline development", "Advancement of multiple clinical programs including FA and FECD treatments" ]

Design Therapeutics announced the dosing of its first Friedreich ataxia (FA) patient in the RESTORE-FA Phase 1/2 multiple-ascending dose trial of DT-216P2 in Australia. The trial aims to evaluate safety, tolerability, and pharmacokinetics of the drug administered through IV and subcutaneous routes. Initial data from the ongoing Phase 1 single-ascending dose trial in healthy volunteers showed favorable safety and improved exposure compared to the first-generation formulation, with no injection site thrombophlebitis reported. However, the company's IND application to expand the trial to U.S. sites has been placed on clinical hold by the FDA due to nonclinical deficiencies. Design expects to report data from the MAD trial, including frataxin expression levels after 12 weeks of dosing, in 2026.

Design Therapeutics (NASDAQ: DSGN), a clinical-stage biotech company focused on developing treatments for serious degenerative genetic diseases, has announced its participation in the upcoming 2025 Jefferies Global Healthcare Conference. The company's management will deliver a presentation on Wednesday, June 4, 2025, at 2:35 p.m. ET in New York. Interested parties can access a live webcast of the presentation through the company's website or the provided link, with the recording remaining available for at least 30 days after the event.

Design Therapeutics (NASDAQ: DSGN), a clinical-stage biotech company focused on developing treatments for serious degenerative genetic diseases, has announced its participation in the 2025 RBC Capital Markets Healthcare Conference. The company's management will engage in a fireside chat on Tuesday, May 20, 2025, at 8:00 a.m. ET in New York. Investors and interested parties can access the live webcast through the company's website at www.designtx.com, where it will remain archived for at least 30 days after the presentation.

Design Therapeutics (NASDAQ: DSGN) reported progress in Q1 2025 across its GeneTAC® programs and financial results. The company's Phase 1 trial for DT-168 in Fuchs Endothelial Corneal Dystrophy (FECD) showed favorable results, with the treatment being well-tolerated and no adverse events reported. A Phase 2 biomarker trial is planned for H2 2025. The ongoing Phase 1 SAD trial of DT-216P2 for Friedreich Ataxia (FA) continues, with plans to initiate a Phase 1/2 MAD patient study in mid-2025. The company appointed Chris Storgard, M.D. as Chief Medical Officer. Financially, Design reported a net loss of $17.7 million for Q1 2025, with R&D expenses at $15.4 million and G&A expenses at $5.0 million. The company maintains a strong cash position of $229.7 million, expected to fund operations into 2029.

Design Therapeutics (DSGN) reported positive Phase 1 data for DT-168, an eye drop treatment for Fuchs Endothelial Corneal Dystrophy (FECD). The Phase 1 SAD/MAD trial in 24 healthy volunteers demonstrated that DT-168 was well-tolerated with no serious adverse events, no ocular adverse events, and no treatment discontinuations. The GeneTAC® small molecule is designed to reduce mutant TCF4 gene expression causing corneal endothelial cell dysfunction. The company plans to advance to a Phase 2 biomarker trial in FECD patients in H2 2025, with data expected in 2026. The Phase 2 trial will evaluate safety, tolerability, and corneal endothelium biomarkers in patients scheduled for corneal transplant surgery, who will receive 0.5% DT-168 eye drops twice daily for approximately 4 weeks before surgery.