Entrada Therapeutics, Inc.
For the period ended Mar 31, 2026. Filed May 7, 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.
Mar 31, 2026 · Filed May 7, 2026
TRDA$4.89+7.5%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-09-30; filed 2025-11-06.
0001689375-25-000050
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent 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 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 144 previous paragraphs and 165 current paragraphs.
87 unchanged; 26 reworded; 51 added; 28 removed; 3 previous and 1 current paragraphs rearranged.
Tables are not compared: 5 previous and 3 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 (51)
Current report
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 report
Clinical-Stage Development Pipeline:
Current report
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 report
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 report
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 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.
Current report
Expanding Preclinical Pipeline:
Current report
The Company has advanced two ocular programs into lead optimization for the potential treatment of inherited retinal diseases. Both programs are novel oligonucleotide-based therapeutics with the potential to address areas of high unmet need. In December 2025, Entrada announced its first ocular clinical candidate, ENTR-801, for the potential treatment of Usher syndrome type 2A (“USH2A”). The Company plans to announce a second clinical candidate in ocular diseases in the second half of 2026 and continues to explore novel targets to address both rare and more common retinal conditions. From a strategic point of view, progress in a new therapeutic area enables portfolio diversification in the form of tissue type, route of administration and regulatory pathway. At the same time, however, as in our neuromuscular franchise, initial targets are selected based on well-understood biology, significant unmet need, translational clarity and the potential to differentiate based on our proprietary technologies and capabilities.
Current report
ENTR-801: The Company’s first ocular candidate is an optimized, proprietary oligonucleotide-based therapy for the potential treatment of a subgroup of patients with USH2A, who are amenable to exon 13 skipping. The clinical candidate was designed to restore functional usherin protein production with the goal of preserving photoreceptors (the light-sensing cells in the eye) to stabilize the overall retinal architecture and preserve function. ENTR-801 was selected from a library of 200 sequences based on its robust exon skipping and usherin protein production, as well as initial safety data in multiple animal models.
Current report
We continue to believe that the robust supporting data and ongoing progress of our growing portfolio of clinical and preclinical candidates has the potential to make a significant difference in the lives of patients.
Current report
ENTR-601-44-201 (ELEVATE-44-201) Phase 1/2 Cohort 1 Results
Current report
Clinical Trial Overview
Current report
ELEVATE-44-201 is a global, two-part, randomized, double-blind, placebo-controlled Phase 1/2 study evaluating the safety, tolerability and effectiveness of ENTR-601-44 in ambulatory participants ages four to twenty with DMD who are exon 44 skipping amenable. The multiple ascending dose Part A portion of the study is evaluating the safety, pharmacokinetics, pharmacodynamics and functional parameters following intravenous administration of ENTR-601-44 to study participants in three cohorts at sites in the U.K. and EU. The MAD portion of the study enrolled eight participants with DMD in Cohort 1. They were randomized 3:1 to receive ENTR-601-44 at a dose of 6 mg/kg or placebo, administered intravenously. During this double-blind period, doses were administered on days one, 43 and 85, and muscle biopsies were performed at the time of screening and at six weeks after the last dose. Following the initial three doses administered in Part A, all participants continued into the Phase 2, open-label portion in which the safety and efficacy of ENTR-601-44 are evaluated over a longer period of time.
Current report
Summary of Demographics and Baseline Patient Characteristics
Current report
The average age of treated participants in Cohort 1 was 9.3 years old with a mean age of disease onset of 2.2 years. Per protocol, all participants were ambulatory and all were on a stable dose of steroids. Baseline dystrophin in both the placebo and treatment population was also lower than that reported in competitive exon 44 skipping clinical trials. This is notable as treatment response is generally considered to correlate with higher baseline dystrophin levels.
Current report
Safety and Tolerability Data
Current report
Cohort 1 achieved its primary endpoint, demonstrating a generally favorable safety and tolerability profile. All treatment emergent adverse events (TEAEs) were mild to moderate. There were no discontinuations and no serious adverse events reported. The most common adverse event (AE) seen was headache.
Current report
Importantly, clinically relevant markers of kidney function including eGFR, Cystatin-C and magnesium were all within normal ranges and treated subjects were comparable to placebo. Participant level data is presented below.
Current report
We believe these results to be encouraging, given that the kidney has historically been the organ of toxicity concern for PMO-based oligonucleotides and peptide conjugates in particular, and because similarly favorable safety was observed at 6 mg/kg in the Phase 1 healthy normal volunteer trial disclosed in 2024.
Current report
Biodistribution and Biomarkers
Current report
Lower than expected plasma Cmax and AUC (area under the curve) was observed in the ENTR-601-44-201 pediatric DMD patient study when compared with our prior ENTR-601-44-101 Phase 1 healthy adult volunteer study. Our current understanding is that this difference is due to a combination of age and disease status, and that higher doses of drug may result in higher levels of drug concentration in circulation. This lower than expected drug concentration, along with lower than expected baseline dystrophin value and a high number of patients with multi-exon deletions or more complex arrangements other than exon 45 deletion mutation may have impacted initial biomarker responses. There was a demonstrated mean increase of 2.36% in dystrophin over a baseline of 4.00% (total mean 6.36% dystrophin) and a demonstrated mean increase of 2.31% in exon skipping over baseline of 2.66% (total mean of 4.97%) in treated patients. Importantly, however, even at this first dose with lower than expected exposures, there was a correlation observed between exon skipping, dystrophin production and functional improvement.
Current report
Functional Improvement
Current report
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 report
"Time To Rise" (referred to as TTSTAND, TTR, and TRF in the literature) is standardized procedure and double-blind evaluative process conducted under GCP conditions and is expressed as the number of seconds taken to rise from a supine position without assistance to standing upright. The clinical evaluators (CEs) are physiotherapists with experience evaluating neuromuscular patients, trained as specified in the protocol, and as detailed in the Evaluator Manual. All CE assessments are videotaped and videos are reviewed by a Master Physiotherapist to ensure that the tests are performed correctly and to remove inter site evaluator variability. TTR declines rapidly over time in patients with DMD and has been previously shown to be an early prognostic factor for disease progression and loss of ambulation.
Current report
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 report
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 report
Functional Improvement Results
Current report
In ELEVATE-44-201 Cohort 1, a statistically significant change from baseline in TTR velocity was observed:
Current report
•Mean change in TTR velocity versus placebo of 0.115
Current report
•Mean change in TTR velocity in the treatment group of 0.08
Current report
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 report
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 report
Discussion of Functional Improvement Results
Current report
An ideal treatment for DMD is regenerative – replacing damaged, dystrophic muscle with healthy muscle; This requires satellite cell correction which then promotes asymmetric differentiation.
Current report
The DMD challenge: Damage and impaired repair
Current report
•A lack of dystrophin not only leaves mature tissue prone to damage but also inhibits the body’s repair mechanism; This “double hit” results in an inevitable decline in function as damaged tissues fail and are replaced with fat and fibrosis
Current report
•Satellite (stem) cells are responsible for muscle regeneration and a lack of dystrophin inhibits the proliferation and differentiation of these progenitor cells which in turn limits muscle repair
Current report
The significant unmet need in DMD: Muscle repair
Current report
•Competitive treatments target mature, damaged cells to slow decline but may not significantly or substantially promote repair via enhanced satellite cell proliferation and asymmetric division
Current report
•Biomarkers (Dystrophin, CK) will improve as damaged tissue is protected, but functional benefit is likely to come back slowly as the repair mechanism is still impaired
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Removed paragraphs (28)
Previous report
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. Our Endosomal Escape Vehicle (EEV™)-therapeutics are designed to enable the efficient intracellular delivery of a wide range of therapeutics into a variety of organs and tissues, resulting in an improved therapeutic index. Leveraging this proprietary, versatile and modular approach, we are advancing a robust development portfolio of RNA- and protein-based programs for the potential treatment of neuromuscular and ocular diseases, among others. Our lead oligonucleotide programs are in development for the potential treatment of people living with Duchenne muscular dystrophy (DMD) who are exon 44, 45, 50 and 51 skipping amenable. We currently have two product candidates, ENTR-601-44 and ENTR-601-45, in clinical development and have filed for UK regulatory clearance for a third, ENTR-601-50, in the third quarter of 2025. We expect to file for EU regulatory clearance in the second half of 2026 and initiate the study by the end of 2026. We expect to submit for regulatory clearance for a fourth product candidate, ENTR-601-51 in 2026. We reported positive preliminary data in healthy volunteers from a Phase 1 clinical trial of ENTR-601-44 in 2024, have fully enrolled the first cohort of patients in the global Phase 1/2 multiple ascending dose (MAD) portion of the study and anticipate reporting data in the second quarter of 2026. We have dosed the first patient in our Phase 1/2 clinical trial for ENTR-601-45, and we anticipate data from the first patient cohort in mid-2026. We have also partnered with Vertex Pharmaceuticals Incorporated (“Vertex”) to develop a clinical-stage program, VX-670, for myotonic dystrophy type 1. As of September 30, 2025, we had cash, cash equivalents and marketable securities of $326.8 million. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of September 30, 2025 will be sufficient to fund our operations into the third quarter of 2027.
Previous report
Key business updates include:
Previous report
Development Pipeline
Previous report
Since January 2025, Entrada has advanced multiple clinical programs in people living with Duchenne muscular dystrophy (DMD) in the U.K., EU and U.S. The clinical trial programs include:
Previous report
•ELEVATE-44-201: Completed enrollment of 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. An independent Data Monitoring Committee (DMC) has reviewed initial data from the eight patients enrolled in Cohort 1 of the blinded, placebo-controlled MAD portion of the study and supports continuation without any protocol modifications. Multiple clinical trial sites in the U.K. and EU have been activated and are dosing patients. We expect to report data from Cohort 1 (6 mg/kg patient dose) during the second quarter of 2026, with data from Cohort 2 and Cohort 3 (up to 12 mg/kg and 18 mg/kg) to follow.
Previous report
•ELEVATE-44-102: Planned Phase 1b MAD clinical study of ENTR-601-44 in ambulatory and non-ambulatory adults living with DMD. The study will run in the U.S. and is on track to initiate in the first half of 2026.
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•ELEVATE-45-201: Initiated dosing in a global Phase 1/2 MAD clinical study of ENTR-601-45 in ambulatory patients living with DMD who are amenable to exon 45 skipping. The study received authorization in the U.K.
Previous report
•ENTR-601-50 and ENTR-601-51: We filed for UK regulatory clearance for ENTR-601-50 in the third quarter of 2025. We expect to file in the EU in the second half of 2026 and initiate the study by the end of 2026. We expect to submit regulatory applications for ENTR-601-51 in 2026.
Previous report
Entrada has also filed multiple regulatory drug designation requests that support the development of drugs for rare, serious, and unmet medical needs. In 2022, the FDA granted Orphan Drug Designation to ENTR-601-44.
Previous report
Beyond DMD, we see continued progress in both partnered and wholly-owned efforts:
Previous report
•We have generated positive preclinical data from programs outside of our neuromuscular franchise, which include new moieties. We now have a growing ocular franchise, with two ocular programs in lead optimization and additional targets in the research phase. We expect to share our first clinical candidate in ocular disease later in 2025.
Previous report
Collaboration revenue was $1.6 million for the three months ended September 30, 2025 and $19.6 million for the three months ended September 30, 2024. The decrease of $18.0 million was primarily a result of fewer costs incurred for VX-670 during the three months ended September 30, 2025 as compared to the three months ended September 30, 2024, as we substantially completed our research plan activities for VX-670 during the first quarter of 2025.
Previous report
We recorded an income tax provision of $0.7 million for the three months ended September 30, 2025 and an income tax benefit of $1.9 million for the three months ended September 30, 2024. For the three months ended September 30, 2025 the income tax provision recorded was primarily driven by adjustments made upon the finalization of tax returns. For the three months ended September 30, 2024, the income tax benefit recorded was primarily driven by the$75 million clinical advancement milestone achieved in the first quarter of 2024 and the application of ASC 740 interim reporting guidance.
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Comparison of the nine months ended September 30, 2025 and 2024
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Collaboration Revenue
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Research and development expenses were $108.3 million for the nine months ended September 30, 2025, compared to $91.9 million for the nine months ended September 30, 2024. The increase of $16.4 million in research and development expenses was primarily attributable to:
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•an increase of $6.8 million in personnel related costs, inclusive of $1.7 million in costs associated with our strategic workforce reduction for the nine months ended September 30, 2025 and stock-based compensation expense of $6.8 million and $5.8 million for the nine months ended September 30, 2025 and 2024, respectively; and
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•an increase of $1.1 million in facility related and other costs to support our expanding operations.
Previous report
General and administrative expenses for the nine months ended September 30, 2025 were $31.5 million, compared to $28.6 million for the nine months ended September 30, 2024. The increase of $2.9 million was primarily attributable to an increase in personnel costs, inclusive of $0.2 million in costs associated with our strategic workforce reduction during the nine months ended September 30, 2025, and stock-based compensation expense of $8.5 million and $7.5 million for the nine months ended September 30, 2025 and 2024, respectively.
Previous report
Interest and Other Income, net
Previous report
We recorded an income tax provision of $0.8 million for the nine months ended September 30, 2025 and an income tax provision of $2.7 million for the nine months ended September 30, 2024. For the nine months ended September 30, 2025, the income tax provision recorded was primarily driven by adjustments made upon the finalization of tax returns.
Previous report
Fornine months ended September 30, 2024, the income tax provision recorded was primarily driven by the $75 million clinical advancement milestone achieved in the first quarter of 2024.
Previous report
Registration Statement that will expire on November 7, 2025, in accordance with applicable SEC regulations. Pursuant to Rule 415(a)(6) under the Securities Act, the offering of securities under the Prior Registration Statement will be deemed terminated as of the date of effectiveness of the new registration statement. Our common stock will be sold at prevailing market prices at the time of the sale, and as a result, prices may vary. As of September 30, 2025 we have not sold any shares of common stock under the 2023 ATM Program.
Previous report
In June 2024, we entered into a securities purchase agreement with a limited number of investors relating to a registered direct offering (the “June 2024 Offering”) of 3,367,003 shares of our common stock at a purchase price of $14.85 per share and, in lieu of common stock to certain investors who so chose, pre-funded warrants (the “Pre-Funded Warrants”) to purchase 3,367,003 shares of common stock at a purchase price of $14.8499 per Pre-Funded Warrant, which represents the price per share at which the shares of common stock were sold to the investors in the June 2024 Offering, minus $0.0001, which is the exercise price of each Pre-Funded Warrant. The June 2024 Offering was made pursuant to the Prior Registration Statement. The aggregate net proceeds from the sale of common stock and Pre-Funded Warrants in the June 2024 Offering were approximately $99.6 million, after deducting offering expenses payable by us. We will receive nominal proceeds, if any, from the exercise of the Pre-Funded Warrants.
Previous report
Net cash provided by financing activities was $101.6 million for thenine months ended September 30, 2024, consisting of $99.6 million of proceeds from sales of our common stock and pre-funded warrants, $1.7 million in proceeds from stock option exercises and $0.3 million from the issuance of common stock under our employee stock purchase plan.
Previous report
Reduction in Force
Previous report
On April 29, 2025, our board of directors approved a strategic plan, designed to increase the focus of our resources on our expanding portfolio of clinical candidates in Duchenne muscular dystrophy (“DMD”) and key preclinical programs. In connection with the new strategic plan, we reduced our workforce by approximately 20% (the "Reduction"). As a result of the Reduction, we incurred charges of $1.9 million, primarily consisting of one-time severance payments and employee termination-related benefits during the nine months ended September 30, 2025. $1.7 million of the charge was recorded as research and development expenses and $0.2 million of the charge was recorded as general and administrative expenses. We do not expect to incur additional charges related to the Reduction.
Previous report
•we may present only two years of audited financial statements and only two years of related Management’s Discussion and Analysis of Financial Condition and Results of Operations;
Reworded paragraphs (26)
Previous wording
By year end, we expect to have three clinical-stage programs in our DMD franchise (ENTR-601-44, ENTR-601-45, ENTR-601-50), complementing the ongoing progress of our VX-670 partnership with Vertex.
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 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.
Previous wording
(March 2025) and EU (May 2025). We expect 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.
Current 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.
Previous wording
•VX-670: Vertex continues to enroll and dose the MAD portion of the global Phase 1/2 clinical trial of VX-670 in people with DM1, which will assess both safety and efficacy. Vertex is on track to complete enrollment and dosing in the trial in the first half of 2026.
Current 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.
Previous wording
Our research and development costs are primarily devoted to supporting our neuromuscular program development efforts. Our direct, external research and development expenses consist primarily of fees paid to outside consultants, CROs, CMOs and research laboratories in connection with our process development, manufacturing and clinical development activities. Our direct external research and development expenses also include fees incurred under license and intellectual property purchase agreements. We expect to track these external research and development costs on a program-by-program basis as we identify specific programs and product candidates to advance into clinical development.
Current wording
Our research and development costs are primarily devoted to supporting our neuromuscular program development efforts. Our direct, external research and development expenses consist primarily of fees paid to outside consultants, CROs, CMOs and research laboratories in connection with our process development, manufacturing and clinical development activities. Our direct external research and development expenses also include fees incurred under license and intellectual property purchase agreements. Where specifically identifiable, we expect to track these external research and development costs on a program-by-program basis as we identify product candidates to advance into clinical development.
Previous wording
We anticipate that our general and administrative expenses will increase in the future as we increase our headcount and expand our facilities to support our continued research activities and development of our programs and EEV Platform.
Current wording
We anticipate that our general and administrative expenses will increase in the future as we increase our headcount and expand our facilities to support our continued research activities and development of our programs and our EEV platform (“EEV Platform”).
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. Our calculation of the estimated effective tax rate requires us to estimate pre‑tax income by tax jurisdiction as well as total tax expense (benefit) for the fiscal year. Accordingly, the annual estimated effective tax rate is subject to adjustment if there are changes to the initial estimates of total tax expense (benefit) or pre‑tax income.
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 for the three months ended March 31, 2026 and 2025.
Previous wording
Collaboration revenue was $24.1 million for the nine months ended September 30, 2025 and $173.4 million for the nine months ended September 30, 2024. The decrease of $149.3 million was primarily a result of fewer costs incurred for VX-670 during the nine months ended September 30, 2025, as we substantially completed our research plan activities for VX-670 during the first quarter of 2025.
Current 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.
Previous wording
Research and development expenses were $38.4 million for the three months ended September 30, 2025, compared to $31.3 million for the three months ended September 30, 2024. The increase of $7.1 million in research and development expenses was primarily attributable to:
Current 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:
Previous wording
•an increase of $5.8 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 $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
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•an increase of $1.3 million in unallocated research and development expenses driven by an increase in personnel costs of $0.7 million and an increase in facilities related costs of $0.7 million. Personnel costs are inclusive of stock-based compensation expense of $2.3 million and $2.1 million for the three months ended September 30, 2025 and 2024, respectively.
Current 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.
Previous wording
We expect that our research and development expenses will increase as we advance ENTR-601-44 and ENTR-601-45 through clinical trials, ENTR-601-50 into clinical trials, ENTR-601-51 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 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.
Previous wording
General and administrative expenses for the three months ended September 30, 2025 were $10.3 million, compared to $10.0 million for the three months ended September 30, 2024. The increase of $0.3 million was primarily attributable to an increase in professional services to support our growth, as well as an increase in personnel costs, inclusive of stock-based compensation expense of $2.8 million and$2.7 millionfor the three months ended September 30, 2025 and 2024, respectively.
Current 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.
Previous wording
Total interest and other income, net was$3.6 million for thethree months ended September 30, 2025, compared to$5.8 million of interest and other income for thethree months ended September 30, 2024. 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$2.6 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.
Previous wording
Since our inception, we have devoted substantially all our resources to research and development efforts relating to our EEV Platform, advancing development of our portfolio of programs and general and administrative support for these operations, including raising capital.
Current wording
Since our inception, we have devoted substantially all our resources to research and development efforts relating to our EEV Platform, advancing development of our portfolio of genetic medicines for the potential treatment of neuromuscular and inherited retinal diseases and administrative support for these operations, including raising capital.
Previous wording
Since inception, we have incurred significant net losses. As of September 30, 2025, we had an accumulated deficit of $233.9 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 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.
Previous wording
As of September 30, 2025, we had cash, cash equivalents and marketable securities of $326.8 million. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of September 30, 2025 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 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.
Previous wording
Since our inception, 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 September 30, 2025, we had cash, cash equivalents and marketable securities of $326.8 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 March 31, 2026, we had cash, cash equivalents and marketable securities of $254.9 million.
Previous wording
In September 2023, we entered into a sales agreement (the "Sales Agreement") with TD Securities (USA) LLC (f/k/a Cowen and Company, LLC) (the "Sales Agent") under which we may, from time to time, issue and sell shares of our common stock having aggregate sales proceeds of up to $150.0 million, in a series of one or more ATM equity offerings (the "2023 ATM Program"). The Sales Agent is not required to sell any specific share amounts but acts as our sales agent, using commercially reasonable efforts consistent with its normal trading and sales practices. Pursuant to the Sales Agreement, any shares will be sold pursuant to the shelf registration statement on Form S-3 (File No. 333-268099) previously filed by the Company with the Securities and Exchange Commission (the “SEC”) on November 1, 2022 and declared effective by the SEC on November 7, 2022 (the “Prior Registration Statement”). The Company plans to file a shelf registration statement on Form S-3, including a sales agreement prospectus contained therein, to replace the Prior
Current wording
In November 2025, we entered into a sales agreement (the "Sales Agreement"), with TD Securities (USA) LLC (f/k/a Cowen and Company, LLC), acting as our agent and/or principal (the "Sales Agent"), with respect to an "at the market offering" program under which we may, from time to time, at our sole discretion, issue and sell shares of our common stock having an aggregate offering price of up to $150.0 million through the Sales Agent. During the three months ended March 31, 2026, there have been no sales of common stock pursuant to the Sales Agreement.
Previous wording
For thenine months ended September 30, 2025, net cash used in operating activities was $96.3 million and was driven by our net loss of $104.6 million, net cash used in changes in our operating assets and liabilities of $7.2 million, adjustments for non-cash expenses relating to stock-based compensation expense of $15.3 million and depreciation expense of $3.2 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $3.0 million.
Current 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.
Previous wording
For thenine months ended September 30, 2024, net cash used in operating activities was $10.0 million and was driven by our net income of $64.5 million, net cash used in changes in our operating assets and liabilities of $83.0 million, adjustments for non-cash expenses relating to stock-based compensation expense of $13.2 million and depreciation expense of $2.8 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $7.5 million.
Current 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.
Previous wording
Net cash provided by investing activities was $87.7 million for thenine months ended September 30, 2025, consisting primarily of $212.1 million from the maturities of marketable securities, partially offset by $123.1 million in purchases of marketable securities and $1.3 million of purchases of property and equipment.
Current 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.
Previous wording
Net cash used in investing activities was $81.2 million for thenine months ended September 30, 2024, consisting primarily of $373.2 million in purchases of marketable securities and $2.5 million of purchases of property and equipment, partially offset by $294.5 million from the maturities of marketable securities.
Current 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.
Previous wording
Net cash provided by financing activities was $0.7 million for thenine months ended September 30, 2025, consisting of $0.4 million in proceeds from stock option exercises and $0.3 million from the issuance of common stock under our employee stock purchase plan.
Current 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.
Previous wording
•the ongoing costs of operating as a public company and recent increases in inflationary rates.
Current wording
•the ongoing costs of operating as a public company.
Previous wording
During the nine months ended September 30, 2025, 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 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.
Previous wording
We have also entered into license agreements under which we are obligated to make certain payments. During the nine months ended September 30, 2025, 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 10, 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 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.
Rearranged current paragraphs (1)
Current report — wording carried from elsewhere
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.
Rearranged previous paragraphs (3)
Previous report — wording carried elsewhere
•an increase of $8.5 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;
Previous report — wording carried elsewhere
We expect that our research and development expenses will increase as we advance ENTR-601-44 and ENTR-601-45 through clinical trials, ENTR-601-50 into clinical trials, ENTR-601-51 through preclinical development and into clinical trials, and continue to perform discovery work for future product candidates.
Previous report — wording carried elsewhere
Total interest and other income, net was$11.9 million for thenine months ended September 30, 2025, compared to$14.3 million of interest and other income for thenine months ended September 30, 2024. 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.