CARLSBAD, Calif., Sept. 10, 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 industry veteran Justin Gover to its Board of Directors, effective immediately. Mr. Gover has more than 25 years of leadership experience in the biotechnology industry. He served as the founding Chief Executive Officer of GW Pharmaceuticals plc, guiding the company for over two ...

Core Insights - Design Therapeutics has developed a novel class of small molecules that can modulate the expression of specific genes in the genome, functioning similarly to transcription factors [1] - The company is focusing on several monogenic disorders, including Friedreich's ataxia, Fuchs endothelial corneal dystrophy, myotonic dystrophy, and Huntington's disease [2] Group 1 - The unique mechanism of Design Therapeutics' molecules involves recognizing DNA sequences through minor groove interactions and recruiting transcriptional machinery to adjust gene expression levels [1] - Friedreich's ataxia is characterized by low expression levels of the frataxin gene, which is the root cause of the disease [3]

Summary of Design Therapeutics Conference Call Company Overview - **Company**: Design Therapeutics (DSGN) - **Focus**: Development of a new class of small molecules called GeneTACs that modulate gene expression for treating monogenic disorders [2][3] Key Points on GeneTAC Platform - **Mechanism**: GeneTACs can increase or decrease the expression of specific genes by recognizing DNA sequences and recruiting transcriptional machinery [2] - **Targeted Diseases**: - Friedreich's ataxia (FA) - Fuchs' endothelial corneal dystrophy - Myotonic dystrophy - Huntington's disease [2][3] Friedreich's Ataxia (FA) Insights - **Gene Target**: The frataxin gene, where low expression leads to FA [3] - **Current Expression Levels**: - Carriers have about 50% frataxin expression; patients have 20-25% of wild-type levels [9] - **Clinical Benefit**: Potential to increase frataxin expression could provide significant clinical benefits [3][10] - **Clinical Trials**: Two programs are currently in clinical investigation [4] Clinical Development of DT216 - **Clinical Trials**: DT216 was taken into the clinic in 2022-2023, showing the ability to upregulate frataxin expression [16] - **Challenges**: Previous formulation faced issues like injection site thrombophlebitis and short duration of exposure [17] - **New Formulation**: DT216P2 has shown improved pharmacokinetics and resolved previous issues [19][20] - **Next Steps**: Currently in Phase II studies (RESTORE FA trial) with multiple ascending doses [24] Fuchs' Endothelial Corneal Dystrophy Program - **Prevalence**: Approximately 2 million diagnosed cases in the U.S. [34] - **Mechanism**: Caused by a mutation in the TCF4 gene leading to toxic RNA production [35] - **Treatment Approach**: DT168 aims to reduce toxic RNA levels through eye drops, potentially modifying disease progression [36] - **Phase I Study**: Confirmed tolerability and safety of DT168 in healthy volunteers [37] Biomarker Development - **Splice Markers**: Identified splice defects as potential biomarkers for assessing treatment efficacy [38] - **Study Design**: Patients scheduled for corneal transplants will be treated with DT168 to evaluate splicing effects [39] Regulatory Considerations - **Endpoints for Registration Trials**: Evaluating visual quality measures, corneal edema, and imaging endpoints for future trials [44] - **Unmet Need**: Any treatment that slows or stops disease progression would be highly valuable [46] Other Programs - **DM1 Program**: Expected to select a development candidate this year [48] - **Competitive Positioning**: GeneTACs may offer advantages over existing therapies due to their mechanism and administration routes [49] Financial Position - **Capital**: Company ended the quarter with $216 million, sufficient to advance clinical programs [51] Market Potential - **Market Validation**: Skyclaris from Biogen is annualizing around $500 million, indicating a significant market opportunity for therapies targeting the root cause of FA [31]

CARLSBAD, Calif., Aug. 27, 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 at the 2025 Cantor Global Healthcare Conference on Thursday, September 4, 2025, at 10:55 a.m. ET in New York. A live webcast of the fireside chat will be available here and in the investors section of the company’s website at www.designtx.com. Th ...

For the transition period from to Commission File Number: 001-40288 Design Therapeutics, Inc. UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 (Exact Name of Registrant as Specified in its Charter) Delaware 82-3929248 (State or other jurisdi ...

Summary of Design Therapeutics (DSGN) Conference Call Company Overview - **Company**: Design Therapeutics (DSGN) - **Industry**: Biotechnology, focusing on genomic medicines for severe monogenic diseases Key Points and Arguments 1. **Innovative Approach**: Design Therapeutics is pioneering a novel class of small molecule genomic medicines aimed at modulating gene transcription, targeting genetic diseases with validated targets [2][3] 2. **Pipeline Overview**: The company is currently developing treatments for four severe monogenic diseases, including Friedreich ataxia (FA) and Fuchs endothelial corneal dystrophy (FECD) [2][3] 3. **Clinical Development**: - **Friedreich Ataxia (FA)**: The company is conducting clinical studies with DT216P2, aiming to increase endogenous frataxin levels in patients. Initial data shows promise in increasing frataxin expression [4][5][16] - **Fuchs Endothelial Corneal Dystrophy (FECD)**: The DT168 program has shown good tolerability in Phase I trials, with plans for a Phase II proof of concept biomarker trial [5][19][24] 4. **Market Potential**: The genomic medicine platform is positioned to surpass existing modalities like gene editing and therapy, with significant market opportunities in the targeted diseases [3][25] 5. **Financial Position**: As of the first quarter, the company reported approximately $229 million in cash, providing a runway into 2029 to support clinical development across its pipeline [26] Additional Important Content 1. **Regulatory Challenges**: The company faced a clinical hold from the FDA regarding its IND application for US trials, which is under review [18] 2. **Patient Enrollment**: The RESTORE FA trial is currently open for enrollment in Australia, with plans to expand to the US pending resolution of the FDA hold [17][18] 3. **Biomarker Development**: The company is exploring potential biomarkers for FECD, which could facilitate future efficacy studies [24][25] 4. **Therapeutic Mechanism**: The GeneTAC molecules are designed to specifically target and modulate gene expression, addressing the root causes of genetic diseases [12][13][25] This summary encapsulates the core aspects of Design Therapeutics' conference call, highlighting the company's innovative approach, clinical pipeline, market potential, and financial health while also noting regulatory challenges and ongoing research efforts.

Core Insights - Design Therapeutics, Inc. has initiated the RESTORE-FA trial for DT-216P2, targeting Friedreich ataxia (FA) with the first patient dosed via intravenous infusion [1][2] - The trial aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of DT-216P2, with initial results showing no adverse events [2][4] - The company submitted an IND application to the FDA to expand the trial to U.S. sites but received a clinical hold notice due to nonclinical deficiencies [3][5] Group 1: Trial Details - The RESTORE-FA trial is designed to evaluate both intravenous and subcutaneous administration of DT-216P2 in FA patients [2] - Initial data from the ongoing Phase 1 single-ascending dose trial in healthy volunteers indicated that DT-216P2 was well-tolerated, with no cases of injection site thrombophlebitis reported [4] - The company expects to report data from the multiple-ascending dose trial, including frataxin expression levels, in 2026 [2] Group 2: Regulatory and Development Updates - The FDA's clinical hold on the IND application is expected to be addressed by the company once further details are received [3][5] - The company plans to work closely with the FDA to expedite the development process in the U.S. [5] - DT-216P2 is an improved formulation designed to target the GAA repeat expansion mutation causing FA and aims to restore endogenous frataxin production [6] Group 3: Company Overview - Design Therapeutics is focused on developing a new class of therapies using GeneTAC technology, which targets disease-causing genes [7] - The company is advancing multiple programs, including DT-216P2 for FA and DT-168 for Fuchs endothelial corneal dystrophy, along with research in myotonic dystrophy type-1 and Huntington's disease [7]

Company Overview - Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for serious degenerative genetic diseases [3] - The company utilizes a platform of GeneTAC gene targeted chimera small molecules to modulate the expression of disease-causing genes [3] Upcoming Presentation - Management will present at the 2025 Jefferies Global Healthcare Conference on June 4, 2025, at 2:35 p.m. ET in New York [1] - A live webcast of the presentation will be available on the company's website and archived for at least 30 days [2] Product Development - The company is advancing clinical-stage programs including DT-216P2 for Friedreich ataxia and DT-168 for Fuchs endothelial corneal dystrophy [3] - Additional programs are in development for myotonic dystrophy type-1 and Huntington's disease, with ongoing discovery efforts for multiple genomic medicines [3]

Summary of Design Therapeutics (DSGN) Conference Call Company Overview - **Company**: Design Therapeutics (DSGN) - **Event**: RBC's Global Healthcare Conference - **Date**: May 20, 2025 Key Points on Gene Tag Technology - **Gene Tag Molecules**: Represent a new class of small molecules called gene targeted chimeras, which can modulate the expression of individual genes without gene editing or therapy [5][6] - **Therapeutic Potential**: Aims to address the root causes of monogenic diseases, offering a method to distribute treatment widely across all cells in the body [5][6] Lead Program: DT216 for Friedreich's Ataxia (FA) - **Clinical Studies**: DT216 was taken into clinical studies in 2022 and 2023, showing promising results in upregulating endogenous frataxin expression [8][9] - **Optimization**: The molecule has been optimized to DT216P2, addressing previous limitations such as short duration of exposure and injection site thrombophlebitis [9][10] - **Phase I Study**: A single ascending dose study in healthy volunteers is underway, with plans to advance to patient studies if results are encouraging [12][14] - **Administration Routes**: Evaluating multiple administration routes (IV and subcutaneous) to maximize drug exposure and therapeutic effect [15][16] - **Market Opportunity**: The FA market is seen as commercially promising, especially with recent approvals and acquisitions in the space [20][21] Biomarker and Efficacy Measurement - **Biomarker Strategy**: Plans to measure mRNA and protein levels in blood and muscle to assess treatment efficacy [23][24] - **Clinical Benchmark**: A significant increase in endogenous frataxin is expected to be therapeutically beneficial, with a benchmark set against normal frataxin levels [21] Second Program: DT168 for Fuchs Corneal Dystrophy - **Disease Overview**: Fuchs corneal dystrophy affects approximately 2 million diagnosed cases in the U.S., with limited treatment options until severe progression [25][26] - **Mechanism**: DT168 targets a specific mutation (CpG18.1) responsible for the disease, aiming to turn off toxic RNA production [27][28] - **Phase I Results**: Completed Phase I studies showed good tolerance and no significant adverse events, supporting progression to Phase II [28][29] - **Natural History Study**: Ongoing observational study to understand patient characteristics and refine future development plans [30][31] Future Development and Additional Programs - **Exploratory Study**: Phase II study planned with DT168 in patients scheduled for corneal transplant, allowing for tissue analysis post-treatment [33][34] - **Huntington's Disease Program**: A molecule selectively downregulates mutant huntingtin, with enhanced efficacy in cells with longer repeat expansions [36] - **Other Programs**: Additional focus on myotonic dystrophy (DM1) with promising pharmacological profiles [36][37] Conclusion - **Pipeline Potential**: The success of any of the four monogenic programs could create significant value for investors and the company [37]

Core Insights - Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for serious degenerative genetic diseases [3] - The company will participate in a fireside chat at the 2025 RBC Capital Markets Healthcare Conference on May 20, 2025 [1] - A live webcast of the event will be available and archived for at least 30 days [2] Company Overview - Design Therapeutics is developing a new class of therapies using its GeneTAC platform, which targets specific disease-causing genes [3] - Current clinical-stage programs include DT-216P2 for Friedreich ataxia and DT-168 for Fuchs endothelial corneal dystrophy, along with programs for myotonic dystrophy type-1 and Huntington's disease [3] - The company is also engaged in discovery efforts for multiple genomic medicines [3]