Initial Data from the Ongoing, Blinded Phase 1 Single-Ascending Dose Trial Has Demonstrated Favorable Safety and Pharmacokinetics IND Application Submitted to Expand RESTORE-FA Trial to U.S. Sites; Notice Received from FDA Placing Clinical Hold on IND Application to Open U.S. Sites CARLSBAD, Calif., June 04, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing... Read More

CARLSBAD, Calif., May 28, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that management will present during the 2025 Jefferies Global Healthcare Conference on Wednesday, June 4, 2025, at 2:35 p.m. ET in New York. A live webcast of the presentation will be available here and in the... Read More

CARLSBAD, Calif., May 13, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that management will participate in a fireside chat during the 2025 RBC Capital Markets Healthcare Conference on Tuesday, May 20, 2025, at 8:00 a.m. ET in New York. A live webcast of the fireside chat will be... Read More

Phase 1 Single Ascending Dose Trial of DT-216P2 for Friedreich Ataxia (FA) Program Ongoing Reported Favorable Phase 1 Data for DT-168 for Fuchs Endothelial Corneal Dystrophy (FECD) Program Well-Capitalized with Cash and Securities of $229.7 Million to Fund Operations Through up to Four Potential Clinical Proof-of-Concept Data Sets CARLSBAD, Calif., May 07, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a... Read More

CARLSBAD, Calif., May 01, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today 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... Read More

CARLSBAD, Calif., April 21, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today 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. The... Read More

CARLSBAD, Calif., April 17, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced the appointment of Chris M. Storgard, M.D., as Chief Medical Officer (CMO). Dr. Storgard brings over two decades of leadership and hands-on drug development experience, having successfully advanced multiple assets... Read More

Initiated Phase 1 Single Ascending Dose Trial of DT-216P2 in Healthy Volunteers; Friedreich Ataxia (FA) Patient Dosing to Begin in mid-2025 Dosing Complete in DT-168 Phase 1 Healthy Volunteer Trial, with Data on Track for the First Half of 2025; Enrollment Target Achieved in Fuchs Endothelial Corneal Dystrophy (FECD) Observational Study Selection of Development Candidate for Myotonic Dystrophy Type-1 (DM1) Expected in 2025... Read More

CARLSBAD, Calif., March 03, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that management will participate in a fireside chat during Leerink’s Global Healthcare Conference on Monday, March 10, 2025, at 1:40 p.m. ET in Miami. A live webcast of the fireside chat will be available here and... Read More

CARLSBAD, Calif., Nov. 13, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that management will participate in the following upcoming investor conferences: 2024 Jefferies London Healthcare Conference Presentation: Tuesday, November 19, 2024, at 10:00 a.m. GMT Location: London, UK Piper... Read More

Fuchs Endothelial Corneal Dystrophy (FECD) Phase 1 Trial Initiated with Data Expected in the First Half of 2025 Friedreich Ataxia (FA) Program on Track to Initiate Phase 1 Single Ascending Dose, Healthy Volunteer Study in the First Half of 2025 Active Research Pipeline Advancing with Programs in Myotonic Dystrophy Type-1 (DM1) and Huntington’s Disease (HD) Well-Capitalized with Cash and Securities of $254.1 Million to Fund... Read More

CARLSBAD, Calif., Sept. 10, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious degenerative genetic diseases, today announced that management will participate in a fireside chat at the 2024 Cantor Global Healthcare Conference on Thursday, September 19, 2024, at 12:45 p.m. ET in New York. A live webcast of the fireside chat will be available here and in... Read More

Friedreich Ataxia (FA) and Fuchs Endothelial Corneal Dystrophy (FECD) Programs on Track and Advancing Toward Clinical Trials Active Research Pipeline with Programs Progressing in Myotonic Dystrophy Type-1 (DM1) and Huntington’s Disease (HD) Cash and Securities of $261.0 Million Supports Operations Through Potentially Four Clinical Proof-of-Concept Data Sets CARLSBAD, Calif., Aug. 05, 2024 (GLOBE NEWSWIRE) -- Design... Read More

Advancing DT-216P2, New Drug Product for Friedreich Ataxia (FA), Toward Clinical Trials Starting Phase 1 Development for Fuchs Endothelial Corneal Dystrophy (FECD) in 2024; Observational Study Currently Enrolling Patients Progressing GeneTAC TM Pipeline Programs in Huntington’s Disease (HD) and Myotonic Dystrophy Type-1 (DM1) to Development Candidates Cash and Securities of $270.7 Million Support Multi-Year Operating Runway... Read More

CARLSBAD, Calif., May 07, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious degenerative genetic diseases, today announced that management will participate in a fireside chat at the 2024 RBC Capital Markets Healthcare Conference on Wednesday, May 15, 2024, at 11:30 a.m. ET in New York. A live webcast of the fireside chat will be available here and in... Read More

New Drug Product for Friedreich Ataxia (FA) DT-216P2 with Favorable Nonclinical Pharmacokinetic and Injection Site Safety Profile; Complete GLP Studies by Year-end 2024 to Start Patient Trials in 2025 IND Cleared for DT-168 for Treatment of Fuchs Endothelial Corneal Dystrophy (FECD) with Phase 1 Development to Start in 2024; Observational Study Underway to Confirm Patient Characteristics and Evaluate Potential Endpoints New... Read More

CARLSBAD, Calif., March 12, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious degenerative genetic diseases, today announced that the company will host a conference call and live webcast on Tuesday, March 19, 2024, at 4:30 p.m. ET to discuss its fourth quarter and full year 2023 financial results and provide a comprehensive portfolio update. The event... Read More

Seasoned Biotech Executive Pratik Shah Appointed as Chief Executive Officer to Lead Company Turnaround Current Cash and Securities of ~$290M Support Extended Five-Year Operating Runway Through 2028 Revised Corporate Strategy with Realignment Towards Long-Term Growth to be Presented in Early 2024; Additional Details to Follow CARLSBAD, Calif., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a... Read More

DT-216 Resulted in a Significant Increase in FXN mRNA Levels in Skeletal Muscle of FA Patients Treatment Generally Well-Tolerated; Injection Site Reactions Attributable to Current Formulation Composition Design Plans to Proceed with an Improved DT-216 Formulation and Initiate a Multiple Dose Phase 1 Clinical Study in the Second Half of 2024 Conference Call and Webcast to be Held Today at 4:30pm ET CARLSBAD, Calif., Aug. 14,... Read More

Initial Data from Phase 1 Multiple-Ascending Dose Trial of DT-216 for Friedreich Ataxia Showed Significant Increase in FXN mRNA Levels in Skeletal Muscle Company Expects to Resume a Multiple Dose Phase 1 Clinical Study with DT-216 with an Improved Formulation in the Second Half of 2024 and Report Data in the First Half of 2025 IND Submission for FECD GeneTAC™ Program On-track for the Second Half of 2023 $303.1 Million in... Read More

CARLSBAD, Calif., May 31, 2023 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today reported that management will present at the 2023 Jefferies Healthcare Conference on Wednesday, June 7, 2023 at 3:30 p.m. ET in NYC. A live webcast will be available in the investor section of the company's website at... Read More

Initial Data from Phase 1 Multiple-Ascending Dose Trial of DT-216 for Friedreich Ataxia Expected in the Third Quarter of 2023 Progress Across GeneTAC™ Small Molecule Pipeline with IND Submissions for FECD and DM1 Programs On-track for the Second Half of 2023 and 2024, Respectively $315.4 Million in Cash and Securities Expected to Support Operating Runway through 2025 CARLSBAD, Calif., May 09, 2023 (GLOBE NEWSWIRE) -- Design... Read More

Treatment with DT-168 Eye Drops Reduced Nuclear Foci and Significantly Improved Mis-Splicing In Vitro Company On-track to Submit Investigational New Drug Application for DT-168 in the Second Half of 2023 CARLSBAD, Calif., April 24, 2023 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that... Read More

Phase 1 Multiple-Ascending Dose Trial of DT-216 for Friedreich Ataxia Ongoing with Data Expected Mid-Year Continued Progress Across Pipeline of GeneTAC™ Small Molecules with Three Programs Expected to be in Clinical Development in the Next Two Years Strong Financial Position with $330 Million in Cash and Securities Expected to Support Operating Runway through 2025 CARLSBAD, Calif., March 14, 2023 (GLOBE NEWSWIRE) -- Design... Read More

CARLSBAD, Calif., Feb. 08, 2023 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. ( Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that João Siffert, M.D., president and chief executive officer, will participate in a fireside chat during the SVB Securities Virtual Global Biopharma Conference on Wednesday, February 15, 2023, at 5:00 p.m. ET. A... Read More

DT-216 was Generally Well-Tolerated and Resulted in a More than Doubling of Frataxin mRNA in Patients with Friedreich Ataxia Second GeneTAC TM Development Candidate, DT-168, Selected for the Treatment of Fuchs Endothelial Corneal Dystrophy Administered as an Eye Drop; IND Expected in 2023 Advancing Myotonic Dystrophy Type-1 GeneTAC TM Lead Molecules; IND Expected in 2024 Conference Call to be Held Today at 4:30pm ET... Read More

Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that preclinical data for the company’s novel GeneTAC™ small molecule, DT-216, as a potential treatment for Friedreich ataxia (FA) will be presented during an oral session at the International Congress for Ataxia Research (ICAR) 2022. The data were included in the... Read More

Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today reported that João Siffert, M.D., president and chief executive officer, and Sean Jeffries, Ph.D., chief operating officer, will participate in a fireside chat during the 2022 Jefferies Cell and Genetic Medicine Summit on Thursday, September 29, 2022, at 10:00 a.m. ET in NYC.... Read More

Initial Data from Friedreich Ataxia Phase 1 Trial of DT-216 Expected in the Fourth Quarter of 2022 Strong Financial Position with $359.4 Million in Cash and Securities to Support Multi-Year Operating Runway Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today highlighted anticipated upcoming milestones across its clinical and... Read More

Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that João Siffert, M.D., president and chief executive officer, and Sean Jeffries, Ph.D., chief operating officer, will participate in a fireside chat during the 2022 Wedbush PacGrow Virtual Healthcare Conference on Wednesday, August 10, 2022, at 10:20 a.m. ET. A... Read More

Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that João Siffert, M.D., president and chief executive officer, will present at the 2022 Jefferies Healthcare Conference on Wednesday, June 8, 2022, at 8:30 a.m. ET in New York City. A live webcast will be available in the investor section of the company’s website... Read More

Clinical Execution of Lead GeneTAC™ Molecule, DT-216, with Initiation of Phase 1 Trial for the Treatment of Friedreich Ataxia Robust Research Pipeline of GeneTAC™ Candidates, with Promising New Data for Fuchs Endothelial Corneal Dystrophy Strong Cash Position with $371.2 Million to Support Multi-Year Operating Runway Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious... Read More

Topline Data from Phase 1 Trial Expected in the Second Half of 2022 DT-216 Granted FDA Fast Track Designation for Patients with Friedreich Ataxia Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for degenerative genetic disorders, today announced that it has completed dosing in the first single ascending dose (SAD) cohort of its Phase 1 clinical trial of DT-216 in... Read More

Phase 1 Trial of DT-216, a Novel FA GeneTAC™ Molecule, in Patients with Friedreich Ataxia On Track to Begin Soon Preclinical Data Supporting Development of Novel GeneTAC™ Small Molecules for the Treatment of Fuchs Endothelial Corneal Dystrophy (FECD) to be Presented at ARVO 2022 Well-Capitalized with $384.1 Million in Cash and Investments at the End of 2021 to Support Upcoming Milestones Design Therapeutics, Inc. (Nasdaq:... Read More

Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today announced that preclinical data supporting the potential of the company’s novel GeneTAC™ small molecules to correct the most common underlying genetic cause of Fuchs endothelial corneal dystrophy (FECD) will be presented at the Association for Research in Vision and Ophthalmology 2022 Annual... Read More

Phase 1 Trial of DT-216 in Patients with Friedreich Ataxia to Be Initiated Soon Topline Data from Phase 1 Trial Expected in the Second Half of 2022 Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today announced that its Investigational New Drug Application (IND) for its lead candidate, DT-216, for the treatment of Friedreich ataxia (FA), was cleared... Read More

Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today reported that João Siffert, M.D., president and chief executive officer, and Sean Jeffries, Ph.D., chief operating officer, will participate in a fireside chat during the SVB Leerink 11 th Annual Global Healthcare Conference on Thursday, February 17, 2022 at 11:20 a.m. ET. A live webcast will be... Read More

Clinical Initiation of Lead GeneTAC TM Program for Friedreich Ataxia On-track for the First Half of 2022 Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today reported recent pipeline progress and third quarter 2021 financial results. “At Design, our mission is to bring a new class of small-molecule genomic medicines to patients with inherited... Read More

Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing small molecule treatments for degenerative genetic disorders, today announced new preclinical data from its novel DM1 GeneTAC™ program, which demonstrated a near-complete resolution of disease-causing foci and correction of splicing defects in myotonic dystrophy type-1 (DM1) patient cells. These data will be presented in a poster titled “ Small... Read More

Preclinical Data Support Initiation of Clinical Development of Lead GeneTAC Program for Friedreich Ataxia in the First Half of 2022 Leadership Team Strengthened with Appointment of Elizabeth Gordon, Ph.D., as Senior Vice President of Regulatory Affairs Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today reported recent progress with its portfolio... Read More

Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today reported that João Siffert, M.D., president and chief executive officer, will participate in a fireside chat during the Goldman Sachs 42 nd Annual Global Healthcare Conference on Wednesday, June 9, 2021 at 1:20 p.m. ET. The live webcast will be available in the investor section of the company's... Read More

Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today announced the appointments of industry veterans, Heather Behanna, Ph.D., principal of SR One, and Deepa Prasad, managing director of WestRiver Group, to its board of directors effective June 15, 2021. “We are delighted to further strengthen the Design team with the appointments of both Heather and... Read More

Successful $276 Million IPO Completed to Advance GeneTAC TM Programs for Friedreich Ataxia and Myotonic Dystrophy Type-1 Toward Clinical Development Company On-track to Initiate Phase 1 Clinical Trial for the Treatment of Friedreich Ataxia in the First Half of 2022 Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today reported business highlights and... Read More