Entrada Therapeutics, Inc.
For the period ended Jun 30, 2026. Filed Aug 5, 2026.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2026 · Filed Aug 5, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2026-03-31; filed 2026-05-07.
0001689375-26-000028
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-06-30; filed 2026-08-05.
0001689375-26-000054
Item 2. Managements Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 165 previous paragraphs and 170 current paragraphs.
123 unchanged; 33 reworded; 9 added; 9 removed; 0 previous and 5 current paragraphs rearranged.
Tables are not compared: 3 previous and 5 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (9)
Current report
ELEVATE-LTE: A Long Term Extension (LTE) platform study protocol (ENTR-DMD-202) was accepted by U.K. and European authorities. This is a phase 2, open-label long-term extension study in participants with Duchenne muscular dystrophy amenable to exon skipping to assess the long-term safety, tolerability, pharmacokinetics, and efficacy of endosomal escape vehicle phosphorodiamidate morpholino oligomer platform products which will enable continued access to drug for study participants while collecting longer-term safety and efficacy data, including functional measures.
Current report
Improvement in TTR and in TTRV was seen across the majority of treated participants, irrespective of age:
Current report
•Statistically significant TTR improvement versus placebo of 2.40 seconds
Current report
•Statistically significant TTRV improvement versus placebo of 0.09 rises per second
Current report
Comparison of the six months ended June 30, 2026 and 2025
Current report
Collaboration Revenue
Current report
Research and development expenses were $68.4 million for the six months ended June 30, 2026, compared to $70.0 million for the six months ended June 30, 2025. The decrease of $1.6 million in research and development expenses was primarily attributable to:
Current report
General and administrative expenses for the six months ended June 30, 2026 were $20.6 million, compared to $21.2 million for the six months ended June 30, 2025. The decrease of $0.6 million was primarily attributable to a decrease in professional services.
Current report
Interest and Other Income, net
Removed paragraphs (9)
Previous report
ENTR-601-51: The Company has completed CTA-enabling studies for people living with DMD who are amenable to exon 51 skipping, which is applicable to the largest sub-population of exon skipping amenable patients. We are reviewing our global regulatory strategy of the program to determine how best to accelerate time to full, as opposed to accelerated, approval. We expect to provide more specific timeline guidance as the new strategy is finalized.
Previous report
In ELEVATE-44-201 Cohort 1, a statistically significant change from baseline in TTR velocity was observed:
Previous report
•Mean change in TTR velocity versus placebo of 0.115
Previous report
•Mean change in TTR velocity in the treatment group of 0.08
Previous report
Research and Development Expenses
Previous report
General and Administrative Expenses
Previous report
Research and Development Expenses
Previous report
(1)Represents manufacturing and clinical costs that support across the Company's product candidates targeting DMD.
Previous report
General and Administrative Expenses
Reworded paragraphs (33)
Previous wording
We are a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines that engx`age intracellular targets that have long been considered inaccessible. Through proprietary, versatile and modular approaches, we are advancing a robust development portfolio of genetic medicines for the potential treatment of neuromuscular and inherited retinal diseases, among others. In 2026, we expect to focus on progressing our ENTR-601-44 and ENTR-601-45 clinical trials. In addition, our VX-670 partnership with Vertex Pharmaceuticals Incorporated (“Vertex”) continues to progress, with Vertex on track to share results during the second half of 2026. In addition to the ENTR-601-44 Cohort 1 results reported in this Quarterly Report, we anticipate reporting additional ENTR-601-44 Cohort 1 Open Label data and topline results from the second cohort of our ENTR-601-44-201 trial as well as our ENTR-601-45 Cohort 1 data during 2026. As of March 31, 2026, we had cash, cash equivalents and marketable securities of $254.9 million. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of March 31, 2026 will be sufficient to fund our operations into the third quarter of 2027.
Current wording
We are a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines that engage intracellular targets that have long been considered inaccessible. Through proprietary, versatile and modular approaches, we are advancing a robust development portfolio of genetic medicines for the potential treatment of neuromuscular and inherited retinal diseases, among others. In 2026, we have been actively progressing our ELEVATE-44 and ELEVATE-45 clinical trials. In addition, our VX-670 partnership with Vertex Pharmaceuticals Incorporated ("Vertex") continues to progress, with Vertex on track to share results during the second half of this year. In addition to the ELEVATE-44-201 Cohort 1 results included in this Quarterly Report, we anticipate reporting additional ELEVATE-44-201 Cohort 1 open-label data by the end of 2026 and topline results from the second cohort of our ELEVATE-44-201 trial in the first quarter of 2027. We expect to report our Cohort 1 ELEVATE-45-201 multiple ascending dose ("MAD") data in October 2026. As of June 30, 2026, we had cash, cash equivalents and marketable securities of $223.0 million. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of June 30, 2026 will be sufficient to fund our operations into the third quarter of 2027.
Previous wording
Entrada continues to advance multiple clinical programs in people living with Duchenne muscular dystrophy (“DMD”) in the United Kingdom (“UK”), European Union (“EU”) and United States (“U.S.”). In 2026, we expect to have four clinical-stage programs in our DMD franchise (ENTR-601-44, ENTR-601-45, ENTR-601-50 and ENTR-601-51, which has completed IND-enabling studies but has not yet filed a regulatory application). When combined, we estimate that there are over 11,500 patients in the U.S. and Europe that carry mutations amenable to Entrada's current exon skipping programs. Complementing the ongoing clinical progress of the DMD franchise is the myotonic dystrophy type 1 (“DM1”) partnership with Vertex (“VX-670”). Each of these programs utilize the same endosomal escape vehicle, and as such, we anticipate initial data readouts from any one of the candidate clinical trials to provide critical insights for the rest.
Current wording
Entrada continues to advance multiple clinical programs in people living with Duchenne muscular dystrophy ("DMD") in the United Kingdom ("UK"), European Union ("EU") and United States ("U.S."). In 2026, we have four clinical-stage programs in our DMD franchise (ENTR-601-44, ENTR-601-45, ENTR-601-50 and ENTR-601-51, which has completed IND-enabling studies but has not yet filed a regulatory application). When combined, we estimate that there are over 11,500 patients in the U.S. and Europe that carry mutations amenable to Entrada's current exon skipping programs. Complementing the ongoing clinical progress of the DMD franchise is the myotonic dystrophy type 1 ("DM1") partnership with Vertex ("VX-670"). Each of these programs utilize the same endosomal escape vehicle, and as such, we anticipate initial data readouts from any one of the candidate clinical trials to provide critical insights for the rest.
Previous wording
ELEVATE-44-201: The Company completed Cohort 1 of the global Phase 1/2 multiple ascending dose (“MAD”) portion of the clinical study of ENTR-601-44 in ambulatory patients living with DMD who are amenable to exon 44 skipping, and those patients transitioned to the 6 dose open label portion of the trial. Cohort 1 of the study met its primary goal of favorable safety and tolerability, while also demonstrating (post hoc analysis) significant improvements in functional benefit as measured by change in Time to Rise ("TTR") velocity from baseline, versus minimal clinically important difference ("MCID"). Dosing every 6 weeks of all 8 patients is ongoing in the open-label portion of the study. In February 2026, an independent Data Monitoring Committee (DMC) reviewed the data to date from the eight patients enrolled in Cohort 1 and recommended initiation of Cohort 2 at the increased dose of 12 mg/kg without protocol modification, and Cohort 2 is continuing to enroll. Data from the Cohort 1 open label portion of the study is expected by year-end, and we expect topline results from Cohort 2 (12 mg/kg) by the end of 2026. Data from Cohort 3 (up to 18 mg/kg) is to follow if needed. Based on a combination of non-clinical data, healthy normal volunteer data and Cohort 1 patient data, we anticipate seeing an increase in dystrophin as we increase dosing amounts in the second and third cohorts. We also intend to open an expansion cohort to increase the number of participants treated in the ELEVATE-44-201 study, as this study has been designed to support an accelerated approval in the U.S. Additionally, in December 2025, the U.S. Food and Drug Administration (“FDA”) granted Rare Pediatric Disease Designation to ENTR-601-44.
Current wording
ELEVATE-44-201: The Company completed Cohort 1 (dosing at 6 mg/kg) of the global Phase 1/2 MAD portion of the clinical study of ENTR-601-44 in ambulatory patients living with DMD who are amenable to exon 44 skipping. Previously announced data from Cohort 1 achieved the primary objective of favorable safety and tolerability, while also demonstrating (post hoc analysis) significant improvements in functional benefit as measured by change in Time to Rise ("TTR") and Time to Rise velocity ("TTRV") from baseline, versus placebo. All eight of the previously-treated and placebo patients have now transitioned to the 6-dose open-label period of the study, which is on track to report data by the end of 2026. A continuation of safety and functional benefit in this portion of the study would strengthen the competitive profile of the program. Enrollment is complete for Cohort 2 of the global Phase 1/2 MAD portion of the study at the increased dose of 12 mg/kg, and we expect to report topline results including safety, exon skipping, dystrophin and functional readouts in the first quarter of 2027. Data from Cohort 3 (up to 18 mg/kg) will follow, if needed. Based on a combination of non-clinical data, healthy volunteer data and Cohort 1 patient data, we anticipate seeing an increase in dystrophin as we increase dosing amounts in the second and third cohorts. We also intend to open an expansion cohort to increase the number of participants treated in the ELEVATE-44-201 study, as this study has been designed to support an accelerated approval in the U.S. Separately, the U.S. Food and Drug Administration ("FDA") granted Rare Pediatric Disease Designation to ENTR-601-44.
Previous wording
ELEVATE-44-102: The Company believes this clinical trial, in the underserved adult patient population, would be best to initiate at the highest advisable starting dose in patients with advanced disease. Following a review of safety, pharmacokinetic and pharmacodynamic data from Cohort 1 of the ELEVATE-44-201 study in the U.K and EU, we may have an opportunity to re-engage with the FDA to discuss increasing the planned doses in this clinical trial. As such, the Company will provide an update on clinical study design and timing following interactions with the FDA.
Current wording
ELEVATE-44-102: The Company believes this clinical trial, in the underserved adult patient population, would be best to initiate at the highest advisable starting dose in patients with advanced disease. Following a review of safety, pharmacokinetic and pharmacodynamic data from Cohort 1 of the ELEVATE-44-201 study in the U.K and EU, we plan to re-engage with the FDA to discuss increasing the planned starting dose in this clinical study. The Company will provide an update on clinical study design and timing following interactions with the FDA.
Previous wording
ELEVATE-45-201: The Company completed enrollment and initiated patient dosing in Cohort 1 of the global Phase 1/2 MAD portion of the clinical study of ENTR-601-45 in ambulatory patients living with DMD who are amenable to exon 45 skipping. The Company is on track to report data from Cohort 1 (5 mg/kg) in mid-2026, with data from Cohort 2 and Cohort 3 (up to 10 mg/kg and 15 mg/kg) to follow. The proposed ELEVATE-45-201 clinical trial design and dosing regimen is similar to ELEVATE-44-201, incorporating a MAD Phase 1 portion, a 6 dose open label Phase 2 portion and an expansion cohort. We expect ENTR-601-45 to be both best in class and to be the first PMO-conjugate to generate clinically meaningful data in a population where only low single digit competitive dystrophin production has been observed to date.
Current wording
ELEVATE-45-201: The Company has completed enrollment and dosing of Cohort 1 of the global Phase 1/2 MAD portion of the clinical study of ENTR-601-45 in ambulatory patients living with DMD who are amenable to exon 45 skipping. The Company expects to report data from Cohort 1 (5 mg/kg) in October 2026. All participants from Cohort 1 have transitioned into the open-label, Phase 2 portion of the study. Cohort 2 is dosing at 10 mg/kg and is ongoing. Data from Cohort 2 is expected in the first half of 2027, and data from Cohort 3 (up to 15 mg/kg) will follow, if needed. The proposed ELEVATE-45-201 clinical trial design and dosing regimen is similar to ELEVATE-44-201, incorporating a MAD Phase 1 portion, a 6 dose open-label Phase 2 portion and an expansion cohort. We expect ENTR-601-45 to be both best in class and to be the first PMO-conjugate to generate clinically meaningful data in a population where only low single digit competitive dystrophin production has been observed to date.
Previous wording
ELEVATE-50-201: The Company received regulatory authorization from the UK’s Medicines and Healthcare Products Regulatory Agency (“MHRA”) and Research Ethics Committee to initiate a Phase 1/2 MAD clinical study of ENTR-601-50 in ambulatory patients living with DMD who are amenable to exon 50 skipping. We expect to submit additional regulatory applications and obtain authorization in the EU for ENTR-601-50 following a review of data from the ongoing trials of our lead programs. We are evaluating a variety of options to optimize clinical study execution including pursuing a platform or basket based approach, should either pathway become available..
Current wording
ELEVATE-50-201 and ENTR-601-51: We expect to submit additional regulatory applications following a review of data from the ongoing trials of our lead programs. We are evaluating a variety of options to optimize clinical study execution, and we expect to provide more specific timeline guidance as the new strategy is finalized.
Previous wording
VX-670: Vertex continues to enroll and dose the MAD portion of the GALILEO global Phase 1/2 clinical study of VX-670 in people with DM1. The study assesses both safety and efficacy and Vertex is on track to share results during the second half of 2026.
Current wording
VX-670: Vertex has completed enrollment and continues dosing in the MAD portion of the GALILEO global Phase 1/2 clinical trial of VX-670 in people with DM1. The study is assessing safety and preliminary efficacy, including change from baseline in the splicing index and other endpoints evaluating muscle function and strength. Vertex is on track to complete dosing and report results in the second half of 2026.
Previous wording
Meaningful and potentially differentiated early functional benefit was observed in Cohort 1. Results from these initial patients demonstrated a statistically significant improvement in mean TTR velocity in treated versus placebo patients (p<.05, post hoc analysis). Functional data is the ultimate goal for a DMD therapeutic, and we are pleased to have observed this benefit at its starting dose of 6 mg/kg.
Current wording
Meaningful and potentially differentiated early functional benefit was observed in Cohort 1. Results from these initial patients demonstrated a statistically significant improvement in mean TTR and TTR velocity in treated versus placebo patients (p<.05, post hoc analysis). Functional data is the ultimate goal for a DMD therapeutic, and we are pleased to have observed this benefit at its starting dose of 6 mg/kg.
Previous wording
TTR velocity is calculated as 1/TTR, expressed as rises/second. The measure is designed to reduce the impact of outliers and imputed data. The calculation handles the "unable to perform problem" if a patient cannot rise from the floor unassisted by scoring that observation at zero avoiding arbitrary imputation. It dampens clinically meaningless scoring noise between visits and it compresses the long tail seen in readings and produces a distribution that's much closer to normal, which matters for parametric statistics.
Current wording
TTR velocity is calculated as 1/TTR, expressed as rises/second. The measure is designed to reduce the impact of outliers and imputed data. The calculation handles the "unable to perform problem" if a patient cannot rise from the floor without external support by scoring that observation at zero avoiding arbitrary imputation. It dampens clinically meaningless scoring noise between visits and it compresses the long tail seen in readings and produces a distribution that's much closer to normal, which matters for parametric statistics.
Previous wording
TTR velocity is increasingly preferred as an approvable clinical endpoint in Phase 3 trials over alternatives, given its sensitivity, lower variability and limited outlier bias. There are several completed or ongoing Phase 3 clinical trials in DMD using this as the primary endpoint or as one of several primary endpoints. Currently, steroids are standard of care in DMD, because of their proven ability to impact function. As a reference, vamorolone (Santhera Therapeutics) is a corticosteroid approved in 2023 and indicated for the treatment of DMD in patients 2 years of age and older. In the vamorolone registrational trial, the primary endpoint was the change from baseline to Week 24 in TTR velocity at 6 mg/kg/day compared to placebo. In that study, the mean change from baseline was reported as 0.048 (rises/second), and this remains the highest number published to date in a registrational trial.
Current wording
TTR velocity is increasingly preferred as an approvable clinical endpoint in Phase 3 trials over alternatives, given its sensitivity, lower variability and limited outlier bias. There are several completed or ongoing Phase 3 clinical trials in DMD using this as the primary endpoint or as one of several primary endpoints. Currently, steroids are standard of care in DMD, because of their proven ability to impact function. As a reference, vamorolone (Santhera Therapeutics) is a corticosteroid approved in 2023 and indicated for the treatment of DMD in patients 2 years of age and older. In the vamorolone registrational trial, the primary endpoint was the change from baseline to Week 24 in TTR velocity at 6 mg/kg/day compared to placebo. In that study, the mean change from baseline was reported as 0.048 (rises/second) and was 0.06 versus placebo (and these remains the highest number published to date in a registrational trial).
Previous wording
The mean change in TTR velocity in the study was 3.5 times higher than the MCID threshold (post hoc analysis) of 0.023 (literature derived), suggesting that ENTR-601-44 has the potential to change the trajectory of the disease at the lowest dose tested in the Phase 1/2 study.
Current wording
The mean change in TTR velocity in the study was 3.5 times higher than the Minimal Clinical Important Difference (MCID) in annual rate of change of timed function threshold (post hoc analysis) of 0.023 (literature derived), suggesting that ENTR-601-44 has the potential to change the trajectory of the disease at the lowest dose tested in the Phase 1/2 study.
Previous wording
Importantly, the change in TTR velocity was consistent and seen across the majority of patients, irrespective of their severity of disease. In addition, there was no observed correlation between age and TTR velocity, which suggests that Cohort 1’s functional benefit represents a true drug related effect. Finally, the end of Cohort 1 dystrophin levels correlated with the end of Cohort 1 TTR velocity improvement suggesting that dystrophin production may have crossed a critical threshold for functional improvement. Positive trends were seen in the treated patients' 10-meter walk/run assessments.
Current wording
Importantly, the change in TTR velocity was consistent and seen across the majority of patients, irrespective of their severity of disease. In addition, there was no observed correlation between age and TTR velocity, but there was a statistically significant correlation observed between the change from baseline in TTRV and change from baseline in MHC–normalized dystrophin levels. This suggests that dystrophin production may have crossed a critical threshold for functional improvement and that the functional benefit observed in Cohort 1 could represent a true drug related effect. Positive trends were seen in the treated patients' 10-meter walk/run assessments.
Previous wording
All study participants in Cohort 1 have now progressed to the open-label, Phase 2 portion of the study, where they will receive six additional doses of 6 mg/kg of ENTR-601-44. Additional study participants are now being dosed in Cohort 2, in which they will receive placebo or three doses of 12 mg/kg of ENTR-601-44. We expect topline results from Cohort 2 by the end of 2026.
Current wording
All study participants in Cohort 1 have now progressed to the open-label, Phase 2 portion of the study, where they are receiving six additional doses of 6 mg/kg of ENTR-601-44. We believe that evidence of continued functional responses from this open-label period will further support the early suggestion of a potential drug related improvement in function. Additional study participants are now being dosed in Cohort 2, in which they will receive placebo or three doses of 12 mg/kg of ENTR-601-44. We expect topline safety and efficacy results from Cohort 2 in the first quarter of 2027.
Previous wording
Provision for income tax expense (benefit) recorded in any interim period is based on the estimated effective tax rate for the fiscal year for those tax jurisdictions that can be reliably estimated. There were no significant income tax provisions or benefits for the three months ended March 31, 2026 and 2025.
Current wording
Provision for income tax expense (benefit) recorded in any interim period is based on the estimated effective tax rate for the fiscal year for those tax jurisdictions that can be reliably estimated. There were no significant income tax provisions or benefits recorded for the three and six months ended June 30, 2026 and 2025.
Previous wording
Research and development expenses were $33.1 million for the three months ended March 31, 2026, compared to $32.1 million for the three months ended March 31, 2025. The increase of $1.0 million in research and development expenses was primarily attributable to:
Current wording
Research and development expenses were $35.4 million for the three months ended June 30, 2026, compared to $37.9 million for the three months ended June 30, 2025. The decrease of $2.5 million in research and development expenses was primarily attributable to:
Previous wording
•a decrease of $2.9 million in unallocated research and development expenses driven by a decrease in personnel costs of $0.7 million and a decrease in facilities related costs of $2.2 million. Personnel costs are inclusive of stock-based compensation expense of $2.2 million and $2.4 million for the three months ended March 31, 2026 and 2025, respectively.
Current wording
•a decrease of $3.2 million in unallocated research and development expenses is driven by a decrease in personnel costs of $1.4 million and a decrease in facilities related costs of $1.8 million. Personnel costs are inclusive of stock-based compensation expense of $2.4 million and $2.1 million for the three months ended June 30, 2026 and 2025, respectively; and
Previous wording
We expect that our research and development expenses will increase as we continue to advance ENTR-601-44 and ENTR-601-45 through clinical trials, ENTR-601-50 into clinical trials, ENTR-601-51 and our ocular programs through preclinical development and into clinical trials, and continue to perform discovery work for future product candidates.
Current wording
We expect that our research and development expenses will increase as we continue to advance our Duchenne franchise through clinical trials, advance our ocular programs through preclinical development and into clinical trials and continue to perform discovery work for future product candidates.
Previous wording
General and administrative expenses for the three months ended March 31, 2026 were $10.1 million, compared to $10.3 million for the three months ended March 31, 2025. The decrease of $0.2 million was primarily attributable to a decrease in professional services.
Current wording
General and administrative expenses for the three months ended June 30, 2026 were $10.5 million, compared to $10.9 million for the three months ended June 30, 2025. The decrease of $0.4 million was primarily attributable to a decrease in professional services.
Previous wording
Total interest and other income, net was$2.6 million for thethree months ended March 31, 2026, compared to$4.4 million of interest and other income for thethree months ended March 31, 2025. The decrease was driven by changes in interest earned from debt securities and money market funds as well as a decrease in the amount of marketable securities held.
Current wording
Total interest and other income, net was$2.2 million for thethree months ended June 30, 2026, compared to$3.9 million of interest and other income for thethree months ended June 30, 2025. The decrease was driven by changes in interest earned from debt securities and money market funds as well as a decrease in the amount of marketable securities held.
Previous wording
Collaboration revenue was $0.9 million for the three months ended March 31, 2026 and $20.6 million for the three months ended March 31, 2025. The decrease of $19.7 million was primarily a result of Entrada substantially completing our research plan activities for VX-670 during the first quarter of 2025.
Current wording
Collaboration revenue was $1.8 million for the six months ended June 30, 2026 and $22.5 million for the six months ended June 30, 2025. The decrease of $20.7 million was primarily a result of us substantially completing our research plan activities for VX-670 during the first quarter of 2025. We continue to perform research activities on additional EEV-based therapeutic candidates for the potential treatment of DM1 pursuant to the Vertex Agreement.
Previous wording
•an increase of $3.9 million in direct research and development expenses, driven by additional costs incurred related to the progress of our Duchenne programs, partially offset by fewer costs incurred related to our collaboration with Vertex; and
Current wording
•an increase of $4.5 million in direct research and development expenses, driven by additional costs incurred related to the progress of our Duchenne and ocular programs, partially offset by fewer costs incurred related to our collaboration with Vertex and other preclinical and discovery activities; and
Previous wording
Since inception, we have incurred significant net losses. As of March 31, 2026, we had an accumulated deficit of $312.8 million. Other than the recognition of revenue related to collaboration payments, we expect to continue to generate operating losses and negative operating cash flows for the foreseeable future as we advance our platform and therapeutic candidates. We will not generate any revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for one or more therapeutic candidates, if ever. If we obtain regulatory approval for any therapeutic candidates, we expect to incur significant expenses related to developing our internal commercialization capability to support product sales, marketing and distribution.
Current wording
Since inception, we have incurred significant net losses. As of June 30, 2026, we had an accumulated deficit of $355.6 million. Other than the recognition of revenue related to collaboration payments, we expect to continue to generate operating losses and negative operating cash flows for the foreseeable future as we advance our platform and therapeutic candidates. We will not generate any revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for one or more therapeutic candidates, if ever. If we obtain regulatory approval for any therapeutic candidates, we expect to incur significant expenses related to developing our internal commercialization capability to support product sales, marketing and distribution.
Previous wording
As of March 31, 2026, we had cash, cash equivalents and marketable securities of $254.9 million. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of March 31, 2026 will be sufficient to fund our operations into the third quarter of 2027. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. To finance our operations beyond that point we will need to raise additional capital, which cannot be assured.
Current wording
As of June 30, 2026, we had cash, cash equivalents and marketable securities of $223.0 million. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of June 30, 2026 will be sufficient to fund our operations for at least 12 months from the date of this report. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. To finance our operations beyond that point we will need to raise additional capital, which cannot be assured.
Previous wording
To date, we have raised over $850.0 million of gross proceeds from sales of stock to leading biotechnology investors and from the Vertex Agreement. As of March 31, 2026, we had cash, cash equivalents and marketable securities of $254.9 million.
Current wording
To date, we have raised over $850.0 million of gross proceeds from sales of stock to leading biotechnology investors and from the Vertex Agreement. As of June 30, 2026, we had cash, cash equivalents and marketable securities of $223.0 million.
Previous wording
For thethree months ended March 31, 2026, net cash used in operating activities was $41.7 million and was driven by our netloss of $39.7 million, net cash used in changes in our operating assets and liabilities of $7.0 million, adjustments for non-cash expenses relating to stock-based compensation expense of $4.7 million and depreciation expense of $0.8 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $0.5 million.
Current wording
For thesix months ended June 30, 2026, net cash used in operating activities was $74.2 million and was driven by our netloss of $82.5 million, net cash used in changes in our operating assets and liabilities of $2.2 million, adjustments for non-cash expenses relating to stock-based compensation expense of $9.9 million and depreciation expense of $1.6 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $1.0 million.
Previous wording
For thethree months ended March 31, 2025, net cash used in operating activities was $38.5 million and was driven by our netloss of $17.3 million, net cash used in changes in our operating assets and liabilities of $25.9 million, adjustments for non-cash expenses relating to stock-based compensation expense of $5.1 million and depreciation expense of $0.9 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $1.3 million.
Current wording
For thesix months ended June 30, 2025, net cash used in operating activities was $68.0 million and was driven by our netloss of $60.5 million, net cash used in changes in our operating assets and liabilities of $17.3 million, adjustments for non-cash expenses relating to stock-based compensation expense of $10.1 million and depreciation expense of $2.0 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $2.3 million.
Previous wording
Net cash provided by investing activities was $4.8 million for thethree months ended March 31, 2025, consisting primarily of $85.9 million from the maturities of marketable securities, partially offset by $80.0 million in purchases of marketable securities and $1.1 million of purchases of property and equipment.
Current wording
Net cash provided by investing activities was $81.9 million for thesix months ended June 30, 2026, consisting primarily of $97.7 million from the maturities of marketable securities, partially offset by $15.8 million in purchases of marketable securities.
Previous wording
Net cash provided by investing activities was $27.0 million for thethree months ended March 31, 2026, consisting primarily of $38.9 million from the maturities of marketable securities, partially offset by $11.8 million in purchases of marketable securities and $0.1 million of purchases of property and equipment.
Current wording
Net cash provided by investing activities was $38.6 million for thesix months ended June 30, 2025, consisting primarily of $136.6 million from the maturities of marketable securities, partially offset by $96.6 million in purchases of marketable securities and $1.4 million of purchases of property and equipment.
Previous wording
Net cash provided by financing activities was $0.1 million for thethree months ended March 31, 2026, consisting of $0.1 million in proceeds from stock option exercises.
Current wording
Net cash provided by financing activities was $0.5 million for thesix months ended June 30, 2026, consisting of $0.2 million in proceeds from stock option exercises and $0.3 million from the issuance of common stock under our 2021 Employee Stock Purchase Plan, as amended (the "2021 ESPP").
Previous wording
Net cash provided by financing activities was $0.4 million for thethree months ended March 31, 2025, consisting of $0.4 million in proceeds from stock option exercises.
Current wording
Net cash provided by financing activities was $0.7 million for thesix months ended June 30, 2025, consisting of $0.4 million in proceeds from stock option exercises and $0.3 million from the issuance of common stock under the 2021 ESPP.
Previous wording
We expect to incur significant expenses and operating losses for the foreseeable future as we advance the preclinical and, if successful, the clinical development of our programs. Our operating expenses and future funding requirements are expected to increase substantially as we continue to advance our portfolio of programs. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of March 31, 2026 will be sufficient to fund our operations into the third quarter of 2027. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we expect.
Current wording
We expect to incur significant expenses and operating losses for the foreseeable future as we advance the preclinical and, if successful, the clinical development of our programs. Our operating expenses and future funding requirements are expected to increase substantially as we continue to advance our portfolio of programs. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of June 30, 2026 will be sufficient to fund our operations for at least 12 months from the date of this report. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we expect.
Previous wording
During the three months ended March 31, 2026, there were no material changes to our lease commitments from those described in Note 11, Leases, of our financial statements in the Annual Report.
Current wording
During the six months ended June 30, 2026, there were no material changes to our lease commitments from those described in Note 11, Leases, of our financial statements in the Annual Report.
Previous wording
We have also entered into license agreements under which we are obligated to make certain payments. During the three months ended March 31, 2026, there were no material changes to our commitments and contingencies related to our license agreements from those described in “Business—Intellectual property— License agreement with The Ohio State University” and Note 11, Commitments and Contingencies, to our financial statements in the Annual Report. For additional information regarding our license agreements, refer to Note 11, Commitments and Contingencies, to our condensed consolidated financial statements in this Quarterly Report.
Current wording
We have also entered into license agreements under which we are obligated to make certain payments. During the six months ended June 30, 2026, there were no material changes to our commitments and contingencies related to our license agreements from those described in "Business—Intellectual property— License agreement with The Ohio State University" and Note 11, Commitments and Contingencies, to our financial statements in the Annual Report. For additional information regarding our license agreements, refer to Note 11, Commitments and Contingencies, to our condensed consolidated financial statements in this Quarterly Report.
Rearranged current paragraphs (5)
Current report — wording carried from elsewhere
Collaboration revenue was $0.9 million for the three months ended June 30, 2026 and $2.0 million for the three months ended June 30, 2025.
Current report — wording carried from elsewhere
•an increase of $0.7 million in direct research and development expenses, driven by additional costs incurred related to the progress of our Duchenne and ocular programs.
Current report — wording carried from elsewhere
•a decrease of $6.1 million in unallocated research and development expenses driven by a decrease in personnel costs of $2.2 million and a decrease in facilities related costs of $3.9 million. Personnel costs are inclusive of stock-based compensation expense of $4.5 million and $4.5 million for the six months ended June 30, 2026 and 2025, respectively.
Current report — wording carried from elsewhere
We expect that our research and development expenses will increase as we continue to advance our Duchenne franchise through clinical trials, advance our ocular programs through preclinical development and into clinical trials and continue to perform discovery work for future product candidates.
Current report — wording carried from elsewhere
Total interest and other income, net was $4.9 million for the six months ended June 30, 2026, compared to $8.4 million of interest and other income for the six months ended June 30, 2025. The decrease was driven by changes in interest earned from debt securities and money market funds as well as a decrease in the amount of marketable securities held.