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DSGN

Design Therapeutics, Inc.

DSGN

Design Therapeutics, Inc. • NASDAQ
$9.39 ▲ 0.21% (+0.02)
Market Cap $534.86 M
52w High $9.85
52w Low $2.60
Dividend Yield 0%
P/E -7.89
Volume 124.21K
Outstanding Shares 56.96M

Income Statement

Period Revenue Operating Expense Net Income Net Profit Margin Earnings Per Share EBITDA
Q3-2025 $0 $4.722M ▼ $-16.997M ▲ 0% $-0.3 ▲ $-16.842M ▲
Q2-2025 $0 $21.417M ▲ $-19.083M ▼ 0% $-0.34 ▼ $-18.931M ▲
Q1-2025 $0 $20.418M ▲ $-17.715M ▼ 0% $-0.31 ▼ $-20.259M ▼
Q4-2024 $0 $16.694M ▲ $-13.651M ▼ 0% $-0.24 ▼ $-16.545M ▼
Q3-2024 $0 $16.246M $-13.039M 0% $-0.23 $-16.093M

Balance Statement

Period Cash & Short-term Total Assets Total Liabilities Total Equity
Q3-2025 $205.97M ▼ $211.77M ▼ $12.05M ▲ $199.72M ▼
Q2-2025 $216.276M ▼ $222.886M ▼ $9.849M ▲ $213.037M ▼
Q1-2025 $229.674M ▼ $237.475M ▼ $9.556M ▼ $227.919M ▼
Q4-2024 $245.477M ▼ $252.093M ▼ $9.996M ▲ $242.097M ▼
Q3-2024 $254.074M $261.629M $9.176M $252.453M

Cash Flow Statement

Period Net Income Cash From Operations Cash From Investing Cash From Financing Net Change Free Cash Flow
Q3-2025 $-16.997M ▲ $-11.162M ▲ $6.51M ▼ $37K ▼ $-4.615M ▼ $-11.162M ▲
Q2-2025 $-19.083M ▼ $-14.441M ▲ $20.116M ▲ $361K ▲ $6.036M ▲ $-14.528M ▲
Q1-2025 $-17.715M ▼ $-16.789M ▼ $7.87M ▲ $23K ▼ $-8.896M ▼ $-16.861M ▼
Q4-2024 $-13.651M ▼ $-9.86M ▼ $1.832M ▲ $263K ▲ $-7.765M ▲ $-9.86M ▼
Q3-2024 $-13.039M $-9.546M $-5.826M $1K $-15.371M $-9.53M

Five-Year Company Overview

Income Statement

Income Statement Design Therapeutics is still purely a research-stage biotech: it has not generated product revenue over the past several years. The income statement is driven almost entirely by research and development and administrative costs. Losses have been consistent year after year, which is typical for an early-stage biotech without approved drugs. The scale of the losses has grown as the company has ramped up its pipeline, but not in a way that suggests spending is out of control; it looks more like a measured build-out of R&D activity. The key point is that the company remains in an investment phase, with all expenses flowing to the bottom line as net loss because there is no offsetting revenue yet.

Balance Sheet

Balance Sheet The balance sheet shows a business funded by equity, with no debt and a solid cushion of assets relative to its annual losses. Total assets peaked a few years ago and have since trended down as cash is used to fund operations, but shareholders’ equity remains clearly positive, which is a sign of financial health at this stage. The company’s reported cash balance looks much lower now than shortly after the IPO, suggesting that a meaningful portion of its formerly large cash pile has been spent or moved into other types of securities. Still, there is no leverage, no structural debt risk, and the capital structure is simple: equity-funded R&D with a gradually shrinking asset base as trials progress.

Cash Flow

Cash Flow Cash flows are negative and driven by operating activities, reflecting ongoing spending on R&D and corporate costs without incoming revenue. Free cash flow has been consistently negative but reasonably stable, meaning there have been no sudden surges in cash burn. Capital expenditure requirements are minimal, which fits a biotech company that outsources much of its manufacturing and relies on labs rather than heavy infrastructure. Management disclosures outside these raw numbers suggest they believe they still have multiple years of runway, but that cushion ultimately depends on how quickly clinical trials expand and whether spending needs to increase to support more programs in the clinic.

Competitive Edge

Competitive Edge Design Therapeutics is operating in one of the most competitive and fast-moving areas of biotech: genetic medicines for serious inherited diseases. Its edge is a highly differentiated approach—small-molecule GeneTACs that tune gene activity up or down without permanently editing DNA. This contrasts with many peers that rely on viral gene therapy or gene editing tools. The potential advantages are meaningful: simpler delivery, the possibility of oral or local administration (like eye drops), and easier manufacturing compared with complex biologics. At the same time, the target diseases—such as Friedreich’s ataxia, myotonic dystrophy, and Huntington’s disease—are contested spaces, with other companies pursuing different mechanisms. The company’s moat will depend on whether it can show clear, clinically meaningful improvements in patients with a favorable safety profile. Until then, its position is promising but unproven, and it competes not only on science but also on speed to data and quality of execution in trials.

Innovation and R&D

Innovation and R&D Innovation is the core of Design Therapeutics. The GeneTAC platform is a novel way to control gene expression using a chimeric small molecule that both recognizes specific DNA sequences and recruits the cell’s own machinery to increase or decrease gene activity. This is conceptually powerful because it tackles root genetic causes while avoiding permanent changes to the genome. The pipeline reflects this versatility: an improved candidate for Friedreich’s ataxia, an eye-drop therapy for Fuchs endothelial corneal dystrophy, and early programs in myotonic dystrophy and Huntington’s disease. Early clinical signals, such as tolerability in eye trials and improved drug properties for the FA program, are encouraging but still preliminary. There are also clear development risks: one FA trial faced a U.S. clinical hold, and much of the pipeline is preclinical or early clinical, where failure rates in biotech are high. The real test for the platform will be upcoming proof-of-concept data showing that GeneTACs can meaningfully change disease-relevant biomarkers and, eventually, patient outcomes.

Summary

Design Therapeutics is a classic early-stage biotech: no revenue, steady research-driven losses, and a balance sheet funded by equity with no debt. Financially, it looks like a controlled cash-burning R&D engine rather than a commercial business, with negative but relatively stable cash flow and modest capital needs. The strategic story centers on a differentiated platform for gene regulation using small molecules, aiming to address serious genetic repeat disorders at the root-cause level while avoiding the complexities of traditional gene therapy. The company’s strengths lie in its scientific ambition, unique modality, and reported multi-year funding runway. The key risks are the usual biotech ones, but amplified by the novelty of the approach: clinical trial setbacks, regulatory uncertainties, and the need to prove that early scientific promise can translate into clear clinical benefit in competitive disease areas. Future inflection points will come from first meaningful human data in Friedreich’s ataxia and Fuchs dystrophy, and from advancing additional programs into the clinic, all of which will shape how durable and valuable the GeneTAC platform may be over time.

NEWS

November 26, 2025 · 8:00 AM UTC

Design Therapeutics to Participate in Upcoming Investor Conferences

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November 5, 2025 · 4:01 PM UTC

Design Therapeutics Announces Plans to Initiate Patient Dosing of DT-818 in Myotonic Dystrophy Type-1 (DM1) in the First Half of 2026 and Reports Third Quarter 2025 Financial Results

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September 10, 2025 · 4:01 PM UTC

Design Therapeutics Appoints Justin Gover to Board of Directors

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