Design Therapeutics, Inc.
Business
Design Therapeutics, Inc. is a clinical-stage biopharmaceutical company pioneering GeneTAC® small-molecule gene-targeted chimera therapeutics to address inherited nucleotide repeat expansion diseases. It develops GeneTAC® molecules that selectively target expanded repeat sequences to modulate mRNA transcription and restore cellular function, with lead candidates including DT-216/DT-216P2 for Friedreich ataxia and DT-168 for Fuchs endothelial corneal dystrophy. Key business segments comprise discovery and preclinical research, clinical development across programs for FA, FECD, DM1 and HD, and reliance on third-party cGMP manufacturers. Its programs operate in multiple jurisdictions through clinical trial networks and employ conventional small-molecule distribution and administration routes, including topical ocular and systemic IV and subcutaneous delivery.
Summary from filing dated 2025-03-10
Financials
Consolidated Statement of Income
| Concept | Trend | FY 2025 2025-12-31 | FY 2024 2024-12-31 | FY 2023 2023-12-31 | FY 2022 2022-12-31 | FY 2021 2021-12-31 |
|---|---|---|---|---|---|---|
| Research and development (including related party amounts of $915 and $891, respectively) | 59 | 44 | 57 | 49 | 25 | |
| General and administrative (including related party amounts of $528 and $537, respectively) | 20 | 18 | 21 | 19 | 11 | |
| Total operating expenses | 79 | 62 | 78 | 68 | 36 | |
| Loss from operations | −79 | −62 | −78 | −68 | −36 | |
| Interest income | 10 | 13 | 11 | — | — | |
| Net loss | −70 | −50 | −67 | −63 | −36 | |
| Net loss per share, basic | −1.22 | −0.88 | −1.19 | −1.14 | −0.77 | |
| Net loss per share, diluted | −1.22 | −0.88 | −1.19 | −1.14 | −0.77 | |
| Weighted-average shares of common stock outstanding, basic | 57 | 57 | 56 | 56 | 46 | |
| Weighted-average shares of common stock outstanding, diluted | 57 | 57 | 56 | 56 | 46 |
Consolidated Balance Sheet
| Concept | Trend | FY 2025 2025-12-31 | FY 2024 2024-12-31 | FY 2023 2023-12-31 | FY 2022 2022-12-31 | FY 2021 2021-12-31 |
|---|---|---|---|---|---|---|
| Cash and cash equivalents | 17 | 23 | 21 | 27 | 299 | |
| Investment securities | 203 | 223 | 261 | 304 | 85 | |
| Prepaid expenses and other current assets | 4 | 3 | 3 | 5 | 1 | |
| Total current assets | 224 | 248 | 285 | 335 | 385 | |
| Property and equipment, net | 981,000 | 1 | 2 | 2 | 2 | |
| Right-of-use asset, related party | 1 | 2 | 3 | 4 | 4 | |
| Other assets | 0 | 427,000 | 430,000 | 459,000 | — | |
| Total assets | 226 | 252 | 290 | 341 | 391 | |
| Accounts payable | 2 | 2 | 2 | 3 | 2 | |
| Accrued expenses and other current liabilities (including related party amounts of $890 and $800, respectively) | 11 | 6 | 8 | 8 | 4 | |
| Total current liabilities | 13 | 8 | 10 | 11 | 5 | |
| Operating lease liability, net, related party | 645,000 | 2 | 2 | 3 | 3 | |
| Total liabilities | 14 | 10 | 12 | 14 | 8 | |
| Commitments and contingencies (See Note 11) | — | — | — | — | — | |
| Common stock: $0.0001 par value; 200,000,000 shares authorized, 60,398,051 and 56,754,341 shares issued and outstanding at December 31, 2025 and 2024, respectively | 6,000 | 6,000 | 6,000 | 6,000 | 6,000 | |
| Additional paid-in capital | 509 | 469 | 455 | 441 | 430 | |
| Accumulated deficit | −297 | −227 | −178 | −111 | −47 | |
| Accumulated other comprehensive income | 403,000 | 475,000 | 62,000 | −3 | −244,000 | |
| Total stockholders' equity | 213 | 242 | 278 | 327 | 382 | |
| Total liabilities and stockholders' equity | 226 | 252 | 290 | 341 | 391 |
Consolidated Statement of Cash Flows
| Concept | Trend | FY 2025 2025-12-31 | FY 2024 2024-12-31 | FY 2023 2023-12-31 | FY 2022 2022-12-31 | FY 2021 2021-12-31 |
|---|---|---|---|---|---|---|
| Net loss | −70 | −50 | −67 | −63 | −36 | |
| Depreciation | 622,000 | 596,000 | 537,000 | 406,000 | 129,000 | |
| Stock-based compensation | 15 | 13 | 13 | 11 | 5 | |
| Amortization of premiums on investment securities, net | −3 | −6 | −6 | −1 | −77,000 | |
| Non-cash lease expense | −21,000 | 6,000 | — | — | — | |
| Deferred financing costs | 427,000 | 0 | — | — | — | |
| Prepaid expense and other assets | −1 | 226,000 | 2 | −3 | −1 | |
| Accounts payable and other liabilities | 4 | −1 | −1 | 5 | 2 | |
| Net cash used in operating activities | −54 | −43 | −59 | −51 | −29 | |
| Purchases of investment securities | −292 | −230 | −225 | −313 | −86 | |
| Proceeds from maturities of investment securities | 315 | 274 | 277 | 93 | 34 | |
| Purchases of property and equipment | −193,000 | −340,000 | −256,000 | −918,000 | −2 | |
| Net cash provided by investing activities | 23 | 44 | 53 | −221 | −54 | |
| Proceeds from the issuance of common stock in at-the-market offering, net of issuance costs | 25 | 0 | — | — | — | |
| Proceeds from the exercise of stock options | 200,000 | 36,000 | 400,000 | 300,000 | 34,000 | |
| Issuance of common stock through employee stock purchase plan | 649,000 | 477,000 | 305,000 | 339,000 | 194,000 | |
| Net cash provided by financing activities | 26 | 513,000 | 724,000 | 235,000 | 379 | |
| Net decrease in cash and cash equivalents | −6 | 1 | −5 | −272 | 296 | |
| Issuance costs included in accrued expenses | 17,000 | 0 | — | — | — |