DSGN

We lease approximately 12,370 square feet of laboratory and office space in Carlsbad, California, pursuant to a lease agreement that commenced in September 2021 and expires in December 2029.
Additionally, we lease approximately 4,900 square feet of additional office space in the same building, pursuant to a lease amendment that commenced in June 2022 and expires in December 2029.
Holders of Common Stock As of March 2, 2026, there were 61,672,279 shares of common stock issued and held by approximately 11 stockholders of record.
As of December 31, 2025, we had used approximately $133.7 million of the net proceeds received from our initial public offering to support our operations.
Previously, we reported clinical data for our lead FA GeneTAC ® small molecule, DT-216, formulated as the prior DT-216 product candidate (DT-216P1) from a Phase 1 single-ascending dose (SAD) clinical trial in December 2022 and a Phase 1 multiple-ascending dose (MAD) clinical trial in August 2023.
Data from the Phase 1 MAD clinical trial for DT-216P1 suggests more sustained exposure to DT-216 is likely needed to achieve a more durable increase in FXN expression.
We then shifted focus to developing DT-216 with a potentially improved formulation to enable more sustained exposure for the treatment of FA.
In nonclinical studies, we observed higher and more sustained DT-216 plasma levels after administration of DT-216P2 than was seen in studies with DT-216P1.
A Phase 1 SAD clinical trial of DT-216P2 in normal healthy volunteers to evaluate single doses using multiple routes of administration, specifically IV infusion and subcutaneous (SC) injection and infusion routes, has shown that DT-216P2 has been generally well-tolerated.
Human plasma pharmacokinetics (PK) profiles of DT-216P2 were consistent with NHP data following both IV and SC single-dose administration and human PK data has demonstrated that DT-216P2 exhibited improved exposure and PK parameters compared to DT-216P1, including higher area under the curve (AUC) and sustained plasma levels at comparable doses.
We are conducting our RESTORE-FA (Reactivating Expression Suppressed Through Overcoming Repeat Expansion for FA) Phase 1/2 MAD clinical trial of DT-216P2.
The RESTORE-FA trial is designed to evaluate the safety, tolerability, PK and pharmacodynamics (PD) of IV and SC of DT-216P2 in patients with FA.
We anticipate providing an update from the RESTORE-FA trial on the effect of DT-216P2 on endogenous frataxin levels following 12 weeks of dosing in the second half of 2026.
In June 2025, we received a clinical hold notice from the FDA regarding our IND application for DT-216P2.
In December 2025, the clinical hold was lifted and we received clearance from the FDA to initiate clinical studies for DT-216P2.
In May 2025, we reported results from a completed Phase 1, double-masked, placebo-controlled, randomized, SAD/MAD clinical trial evaluating the safety, tolerability and systemic PK of DT-168 ophthalmic solution in normal healthy volunteers.
DT-168 eye drops were well-tolerated in all participants with a maximum dose of two 0.5% drops twice-daily for seven days.
There were no serious adverse events, no ocular adverse events (AEs) and no treatment discontinuations due to AEs in the trial.
All observed AEs were deemed not related to DT-168 by the trial investigator.
In parallel with the Phase 1 trial, we conducted reference range studies which showed consistently different splicing in the corneal endothelium between unaffected eye donors and surgical samples from mutant TCF4 FECD patients, supporting the potential for corneal endothelium biomarkers as a clinical proof-of-concept measure of drug activity.
We are conducting a Phase 2 biomarker trial of DT-168 to evaluate safety, tolerability, and corneal endothelium biomarkers in patients with FECD.
We anticipate reporting data from the Phase 2 biomarker trial in the second half of 2026.
In the fourth quarter of 2025, we announced DT-818 as our GeneTAC ® small molecule development candidate for the treatment of DM1.
In preclinical studies, DT-818 has demonstrated a potential best-in-disease profile for DM1, including a greater than 90% reduction in toxic RNA foci in DM1 patient cells, corresponding splicing correction and selective targeting of mutant DMPK.
In an actin repeat mouse model of DM1 (HSA LR mouse model), DT-818 treatment resulted in improved myotonia and foci reduction.
In tissue distribution studies in NHPs, DT-818 levels were observed to be at expected pharmacologic levels in key target tissues at well-tolerated doses.
In the fourth quarter of 2025, we obtained regulatory clearance to initiate clinical development of DT-818 and plan to begin dosing DM1 patients in a Phase 1 MAD trial in the first half of 2026.
The study, with results anticipated in 2027, is expected to assess safety and correction of mis-splicing.
To date, we have incurred net losses and negative cash flows from operations since our inception and as of December 31, 2025, had an accumulated deficit of $297.0 million.
During the year ended December 31, 2025, we sold an aggregate of 3,250,510 shares of our common stock pursuant to our “at-the-market” sales agreement (ATM Program) for aggregate net proceeds of approximately $24.8 million.
Our cash, cash equivalents and investment securities balance as of December 31, 2025, was $219.8 million.
Results of Operations Comparison of the Years Ended December 31, 2025 and 2024 The following table summarizes our operating expenses for the years ended December 31, 2025 and 2024 (in thousands): Year Ended December 31, 2025 2024 Change Operating expenses: Research and development $ 59,129 $ 44,350 $ 14,779 General and administrative 20,338 18,033 2,305 Total operating expenses $ 79,467 $ 62,383 $ 17,084 Research and Development Expenses.
Research and development expenses for our FA program increased in 2025 compared to 2024 primarily due to costs attributable to DT-216P2 clinical activities and costs for additional development activities.
Expenses for our FECD program increased in 2025 compared to 2024 primarily due to costs associated with DT-168 clinical activities and costs for additional development activities.
Other direct expenses increased in 2025 compared to 2024 primarily due to additional activities related to our early stage research programs.
The increase in indirect expenses compared to the same period of the prior year was primarily due to employee related expenses including compensation, stock-based compensation and other support for our ongoing development programs.
The following table summarizes our research and development expenses by program and by direct and indirect costs for the year ended December 31, 2025 and 2024 (in thousands): Year Ended December 31, 2025 2024 Change FA $ 12,950 $ 8,559 $ 4,391 FECD 5,364 5,271 Other direct 19,339 10,849 8,490 Indirect 21,476 19,671 1,805 Total research and development expense $ 59,129 $ 44,350 $ 14,779 General and Administrative Expenses.
The increase in general and administrative expenses in 2025 compared to 2024 was primarily due to a $1.8 million increase in employee compensation, including $1.5 million in stock-based compensation expense, a $0.4 million one-time charge to deferred financing costs and $0.1 million in other expenses incurred during the year ended December 31, 2025 as compared to the same period in 2024.
As of December 31, 2025, we had $219.8 million of combined cash, cash equivalents and investment securities, a decrease of $25.6 million from the $245.5 million of cash, cash equivalents and investment securities at December 31, 2024.
The following table summarizes our cash flow activities (in thousands): Year Ended December 31, 2025 2024 Net cash (used in) provided by: Operating activities $ (54,358 ) $ (43,105 ) Investing activities 22,913 43,955 Financing activities 25,738 Net increase (decrease) in cash and cash equivalents $ (5,707 ) $ 1,363 Operating Activities.

and 130 more.

Gone since FY2024

We lease approximately 12,370 square feet of laboratory and office space in Carlsbad, California, pursuant to a lease agreement, with a related party, that commenced in September 2021 and expires in August 2027.
Additionally, we lease approximately 4,900 square feet of additional office space in the same building, pursuant to a lease amendment, with a related party, that commenced in June 2022 and expires in August 2027.
Holders of Common Stock As of March 6, 2025, there were 56,762,678 shares of common stock issued and held by approximately 11 stockholders of record.
As of December 31, 2024, we had used approximately $102.5 million of the net proceeds received from our initial public offering to support our operations.
In February 2022, the Investigational New Drug Application (IND) for our lead FA GeneTAC ® small molecule, DT-216, formulated as the prior DT-216 product candidate, was cleared by the U.S.
Food and Drug Administration (FDA) to commence Phase 1 clinical trials.
In December 2022, we reported positive initial data from the Phase 1 single-ascending dose (SAD) clinical trial and in August 2023, we reported data from the Phase 1 multiple-ascending dose (MAD) clinical trial of the prior DT-216 product candidate.
Data from the Phase 1 MAD clinical trial suggests more sustained exposure is likely needed to achieve a more durable increase in FXN expression, and we observed five cases of injection site thrombophlebitis in the Phase 1 MAD clinical trial.
We then shifted focus to developing DT-216 with a potentially improved formulation to enable higher exposure and chronic administration for treatment of FA.
In nonclinical studies, we observed higher and more sustained DT-216 plasma levels after administration of DT-216P2 than was seen in studies with the prior DT-216 product candidate.
Additionally, we observed favorable injection site tolerability following multiple intravenous (IV) administrations of DT-216P2.
We have initiated a Phase 1 SAD clinical trial of DT-216P2 in normal healthy volunteers in Australia to evaluate single doses using multiple routes of administration, specifically IV infusion and subcutaneous (SC) injection and infusion routes.
Results from this Phase 1 SAD clinical trial will inform the design of a clinical trial in FA patients, anticipated to begin in mid-2025.
We anticipate providing an update in 2026 on the effect of DT-216P2 on endogenous FXN levels following 12 weeks of dosing.
We have completed dosing in a Phase 1 clinical trial in normal healthy volunteers to evaluate the safety of DT-168 and plan to provide results in the first half of 2025.
Our third program based on the GeneTAC ® platform is focused on DM1.
Our DM1 GeneTAC ® molecules are specifically designed to reduce the formation of CUG repeat hairpin structures that trap splicing factors and form pathogenic nuclear foci that cause DM1.
Blocking the formation of CUG foci has demonstrated phenotypic benefit in DM1 patients.
We plan to continue evaluating the properties of our DM1 GeneTAC ® molecules in preclinical studies in order to nominate a development candidate in 2025.
To date, we have incurred net losses and negative cash flows from operations since our inception and as of December 31, 2024, had an accumulated deficit of $227.2 million.
In January 2021, we issued 19,083,979 shares of Series B convertible preferred stock at $6.55 per share for net proceeds of approximately $124.7 million.
Our cash, cash equivalents and investment securities balance as of December 31, 2024, was $245.5 million.
Results of Operations Comparison of the Years Ended December 31, 2024 and 2023 The following table summarizes our operating expenses for the years ended December 31, 2024 and 2023 (in thousands): Year Ended December 31, 2024 2023 Change Operating expenses: Research and development $ 44,350 $ 57,063 $ (12,713 ) General and administrative 18,033 21,127 (3,094 ) Total operating expenses $ 62,383 $ 78,190 $ (15,807 ) Research and Development Expenses.
The decrease in direct research and development expenses in 2024 compared to 2023 was primarily due to completion of clinical activities for our FA program in 2023.
Expenses associated with our early-stage programs, that are not allocated on a program-by-program basis, decreased due to the breakout of the FECD program, which moved into clinical development.
The decrease in indirect research and development expenses compared to 2023 was primarily due to lower headcount, resulting in a $1.7 million decrease in personnel-related costs inclusive of stock-based compensation, a $1.2 million decrease in laboratory and related supplies, a $2.2 million decrease in indirect research that benefits multiple early-stage programs and a $0.2 million decrease in other expenses.
The following table summarizes our research and development expenses by direct and indirect costs for the year ended December 31, 2024 and 2023 (in thousands): Year Ended December 31, 2024 2023 Change FA $ 8,559 $ 16,159 $ (7,600 ) FECD 5,271 — 5,271 Other direct 10,849 15,889 (5,040 ) Indirect 19,671 25,015 (5,344 ) Total research and development expense $ 44,350 $ 57,063 $ (12,713 ) General and Administrative Expenses.
The decrease in general and administrative expenses in 2024 compared to 2023 was primarily due to lower headcount, resulting in a $2.3 million decrease in personnel-related costs inclusive of stock-based compensation, a $0.6 million decrease in professional services and a $0.2 million decrease in other expenses.
As of December 31, 2024, we had $245.5 million of combined cash and cash equivalents and investment securities, a decrease of $36.3 million from the $281.8 million of cash and cash equivalents and investment securities at December 31, 2023.
The following table summarizes our cash flow activities (in thousands): Year Ended December 31, 2024 2023 Net cash (used in) provided by: Operating activities $ (43,105 ) $ (58,560 ) Investing activities 43,955 52,536 Financing activities Net increase (decrease) in cash and cash equivalents $ 1,363 $ (5,300 ) Operating Activities.
The decrease in our net cash used in operating activities was primarily due to the $17.3 million decrease in net loss, partially offset by an increase in net working capital for the year ended December 31, 2024 compared to the same period of the prior year.
The lower net loss incurred during the 2024 period was primarily due to decreased expenses related to our FA program and lower personnel-related costs.
The decrease in net cash provided by investing activities was primarily due to a net decrease in cash provided from the maturities and purchases of our investment securities during the year ended December 31, 2024 compared to the year ended December 31, 2023.
Net cash provided by financing activities for each of the years ended December 31, 2024 and 2023 was comprised of proceeds received from the issuance of common stock through our employee stock purchase plan and from employee stock option exercises.
Shelf Registration Statement In April 2022, we filed a shelf registration statement on Form S-3 (2022 Shelf Registration Statement), which became effective in May 2022.
The 2022 Shelf Registration Statement permits: (i) the offering, issuance and sale by us of up to a maximum aggregate offering price of $300.0 million of common stock, preferred stock, debt securities and warrants in one or more offerings and in any combination; and (ii) the offering, issuance and sale by us of up to a maximum aggregate offering price of $100.0 million of our common stock that may be issued and sold under an "at-the-market” sales agreement (ATM Program).
The $ 100.0 million of common stock that may be issued and sold under the ATM Program is included in the $ 300.0 million of securities that may be issued and sold under the 2022 Shelf Registration Statement.
As of December 31, 2024, the Company has not sold any shares of its common stock under the ATM Program.
Funding Requirements Based on our current operating plan, we believe that our existing cash, cash equivalents and investments will be sufficient to fund our planned operating expenses and capital expenditure requirements for more than the next 12 months following the date of this Annual Report.
Our ability to raise additional funds may be adversely impacted by potential worsening global economic conditions and disruptions to and volatility in the credit and financial markets in the United States and worldwide as a result of macroeconomic factors, including geopolitical conflicts (such as the geopolitical tensions between the U.S. and China, the ongoing Russia/Ukraine conflict and the Israel-Hamas war), inflation, tariffs, bank failures, global supply chain and labor shortage challenges, and the effects of a health epidemic or pandemic.

and 124 more.

Sentence-level comparison of Item 1A in the two most recent 10-Ks (FY2024 ↗, FY2025 ↗). A reworded sentence counts as one dropped and one added, so heavy edits read as low “kept”. Headings and page furniture are stripped; nothing is summarised by a model.

Watch DSGN. Know when insiders buy.

One email the day a filing lands. Free.