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.

Design Therapeutics is a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC gene targeted chimera small molecules. The company's GeneTAC molecules are designed to either dial up or dial down the expression of a specific disease-causing gene to address the underlying cause of disease. In addition to its clinical-stage GeneTAC programs, DT-216P2, in development for patients with Friedreich ataxia, and DT-168, for Fuchs endothelial corneal dystrophy, the c ...

Design Therapeutics (DSGN) 2025 Conference May 20, 2025 08:00 AM ET Speaker0 We're on the first day of RBC's Global Healthcare Conference. We're really excited to kick this off with Design Therapeutics represented by Prateek, who's their CEO. Thanks for being here. Speaker1 Thank you so much for the invitation. Great to do that. Speaker0 So maybe just starting off a little bigger picture. Speaker1 Can you Speaker0 remind us and walk us through what a gene tag is and how that molecule has special properties ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q 6005 Hidden Valley Road, Suite 110 Carlsbad, California 92011 (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-40288 Design Therapeutics, Inc. (Exact Name of Registrant as Specif ...

Design Therapeutics Highlights Momentum Across Lead GeneTAC® Programs and Reports First Quarter 2025 Financial Results 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 7, 2025 - Design Therapeutics, Inc. (N ...

Phase 1 Single Ascending Dose Trial of DT-216P2 for Friedreich Ataxia (FA) Program Ongoing 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 clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced progress across its portfolio of GeneTAC candidates and reported ...

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 American Academy of Ophthalmology highlighting industry advancements and innovative new ...

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 presentation will include safety findings from the Phase 1 single- and multiple-ascendi ...

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 from preclinical stages through global regulatory approvals. “We are thrilled to we ...

Design Therapeutics, Inc. (DSGN) could be a solid addition to your portfolio given its recent upgrade to a Zacks Rank #2 (Buy). This upgrade primarily reflects an upward trend in earnings estimates, which is one of the most powerful forces impacting stock prices.A company's changing earnings picture is at the core of the Zacks rating. The system tracks the Zacks Consensus Estimate -- the consensus measure of EPS estimates from the sell-side analysts covering the stock -- for the current and following years. ...