Entrada Therapeutics, Inc.
For the period ended Sep 30, 2025. Filed Nov 6, 2025.
View on SEC.govAbout this filing
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.
Sep 30, 2025 · Filed Nov 6, 2025
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 2025-06-30; filed 2025-08-06.
0001689375-25-000043
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent 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 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 146 previous paragraphs and 144 current paragraphs.
98 unchanged; 38 reworded; 5 added; 9 removed; 1 previous and 3 current paragraphs rearranged.
Tables are not compared: 5 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 (5)
Current 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.
Current report
•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 report
Comparison of the nine months ended September 30, 2025 and 2024
Current report
Emerging Growth Company and Smaller Reporting Company Status
Current report
We are also a “smaller reporting company” because the market value of our stock held by non-affiliates was less than $250 million as of June 30, 2025. We may continue to be a smaller reporting company if either (i) the market value of our stock held by non-affiliates is less than $250 million or (ii) our annual revenue was less than $100 million during the most recently completed fiscal year and the market value of our stock held by non-affiliates is less than $700 million. If we are a smaller reporting company at the time we cease to be an emerging growth company, we may continue to rely on exemptions from certain disclosure requirements that are available to smaller reporting companies. Specifically, as a smaller reporting company we may choose to present only the two most recent fiscal years of audited financial statements in our Annual Report and, similar to emerging growth companies, smaller reporting companies have reduced disclosure obligations regarding executive compensation.
Removed paragraphs (9)
Previous report
Organizational Evolution
Previous report
To support its accelerated clinical growth and other strategic priorities, we have enhanced the organization as follows:
Previous report
•Appointed Navid Khan, Ph.D., to the newly created role of Senior Vice President of Medical Affairs in August 2025. Dr. Khan has extensive expertise in directing medical affairs for pre-commercial and commercial therapies for neuromuscular disorders, including DMD.
Previous report
•Expanded our Clinical Development leadership with the appointment of Kiran Patki, M.D., MSc, FFPM, as Senior Vice President of Clinical Development in July 2025. Dr. Patki brings deep expertise in rare diseases including early- and late-stage clinical development, and has a proven track record of securing product regulatory approvals.
Previous report
•Appointed Maha Radhakrishnan, M.D. to our Board of Directors in June 2025. Dr. Radhakrishnan brings extensive experience in global drug development and portfolio management, having served in Executive Medical Officer roles at global development- and commercial-stage biopharmaceutical companies.
Previous report
•an increase of $3.3 million in personnel related costs driven by increased compensation costs for employees in the research and development function, inclusive of $1.7 million in costs associated with our strategic workforce reduction during the three months ended June 30, 2025, and stock-based compensation expense of $2.1 million and $2.0 million for the three months ended June 30, 2025 and 2024, respectively; and
Previous report
Comparison of the six months ended June 30, 2025 and 2024
Previous report
•our ability to implement and achieve the expected cost savings in connection with the new strategic plan, including the reduction in force;
Previous report
Emerging Growth Company Status
Reworded paragraphs (38)
Previous wording
We are a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines which 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. 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 expect to file for regulatory clearance for a third, ENTR-601-50, in the fourth quarter of 2025. We reported positive preliminary data in healthy volunteers from a Phase 1 clinical trial of ENTR-601-44 in 2024 and anticipate reporting data from the global Phase 1/2 multiple ascending dose (MAD) portion of the study in the first half of 2026. We have also partnered with Vertex to develop a clinical-stage program, VX-670, for myotonic dystrophy type 1. As of June 30, 2025, we had cash, cash equivalents and marketable securities of $354.0 million. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of June 30, 2025 will be sufficient to fund our operations into the second 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. 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 wording
During the second quarter of 2025, we continued to progress our portfolio and achieved the important milestone of first patient dosed in ELEVATE-44-201. We anticipate data from the first patient cohort of the multi-ascending dose trial in the first half of 2026. We also initiated ELEVATE-45-201 and remain on track to dose the first patient in the third quarter of 2025. We anticipate data from the first patient cohort in mid-2026. Operationally, we strengthened the team in key areas including clinical development and medical affairs, as we continue to activate multiple clinical trial sites in the U.K. and EU for both ELEVATE-44-201 and ELEVATE-45-201. 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 Pharmaceuticals Incorporated (“Vertex”). This clinical development progress builds upon positive preliminary data in healthy volunteers from a Phase 1 clinical trial, ENTR-601-44-101, as first reported in 2024.
Current 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.
Previous wording
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. These programs include:
Current wording
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 wording
•ELEVATE-44-201: First patient dosed in 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. Multiple clinical trial sites in the U.K. and EU have been activated and are screening patients. We expect to report data from Cohort 1 (6 mg/kg patient dose) during the first half of 2026, with data from Cohort 2 and Cohort 3 (up to 18 mg/kg) to follow.
Current wording
•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 wording
•ELEVATE-45-201: Initiated 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. (March 2025) and EU (May 2025). Multiple clinical study sites have been activated and the study is on track to dose the first patient in the third quarter of 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 15 mg/kg) to follow.
Current wording
•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 wording
•ENTR-601-50 and ENTR-601-51: We expect to submit global regulatory applications for ENTR-601-50 in the fourth quarter of 2025 and for ENTR-601-51 in 2026.
Current wording
•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 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 living with myotonic dystrophy type 1 (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 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.
Previous wording
Collaboration revenue was $2.0 million for the three months ended June 30, 2025 and $94.7 million for the three months ended June 30, 2024. The decrease of $92.7 million was primarily a result of fewer costs incurred for VX-670 during the three months ended June 30, 2025 as compared to the three months ended June 30, 2024, as we substantially completed our research plan activities for VX-670 during the first quarter of 2025.
Current wording
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 wording
Research and development expenses were $37.9 million for the three months ended June 30, 2025, compared to $32.0 million for the three months ended June 30, 2024. The increase of $5.9 million in research and development expenses was primarily attributable
Current 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:
Previous wording
•an increase of $2.7 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 $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
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, and ENTR-601-50 and 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 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 wording
General and administrative expenses for the three months ended June 30, 2025 were $10.9 million, compared to $9.2 million for the three months ended June 30, 2024. The increase of $1.7 million was primarily attributable to an increase in professional services to support our growth, as well as an increase in personnel costs driven by increased compensation costs for employees in the general and administrative function, inclusive of $0.2 million in costs associated with our strategic workforce reduction during the three months ended June 30, 2025, and stock-based compensation expense of $2.9 million and$2.6 millionfor the three months ended June 30, 2025 and 2024, respectively.
Current 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.
Previous wording
Total interest and other income, net was$3.9 million for thethree months ended June 30, 2025, compared to$4.4 million of interest and other income for thethree months ended June 30, 2024. The decrease was driven by changes in interest earned from debt securities and money market funds.
Current 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.
Previous wording
We recorded an income tax provision of $0.2 million for the six months ended June 30, 2025 and an income tax provision of $4.6 million for the six months ended June 30, 2024. Forsix months ended June 30, 2024, the income tax provision recorded was driven largely by the $75 million clinical advancement milestone achieved in the first quarter of 2024.
Current wording
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.
Previous wording
Collaboration revenue was $22.5 million for the six months ended June 30, 2025 and $153.8 million for the six months ended June 30, 2024. The decrease of $131.3 million was primarily a result of fewer costs incurred for VX-670 during the six months ended June 30, 2025, as we substantially completed our research plan activities for VX-670 during the first quarter of 2025.
Current 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.
Previous wording
Research and development expenses were $69.9 million for the six months ended June 30, 2025, compared to $60.6 million for the six months ended June 30, 2024. The increase of $9.3 million in research and development expenses was primarily attributable to:
Current wording
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:
Previous wording
•an increase of $2.6 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.
Current wording
•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 wording
•an increase of $6.2 million in personnel related costs driven by increased compensation costs for employees in the research and development function, inclusive of $1.7 million in costs associated with our strategic workforce reduction for the six months ended June 30, 2025 and stock-based compensation expense of $4.5 million and $3.6 million for the six months ended June 30, 2025 and 2024, respectively;
Current wording
•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
Previous wording
•an increase of $0.4 million in facility related and other costs to support our expanding operations.
Current wording
•an increase of $1.1 million in facility related and other costs to support our expanding operations.
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, and ENTR-601-50 and 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 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 wording
General and administrative expenses for the six months ended June 30, 2025 were $21.2 million, compared to $18.6 million for the six months ended June 30, 2024. The increase of $2.6 million was primarily attributable to an increase in personnel costs driven by increased compensation costs for employees in the general and administrative function, inclusive of $0.2 million in costs associated with our strategic workforce reduction during the six months ended June 30, 2025, and stock-based compensation expense of $5.6 million and $4.7 million for the six months ended June 30, 2025 and 2024, respectively.
Current wording
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 wording
Total interest and other income, net was$8.4 million for thesix months ended June 30, 2025, compared to$8.6 million of interest and other income for thesix months ended June 30, 2024. The decrease was driven by changes in interest earned from debt securities and money market funds.
Current wording
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.
Previous wording
We recorded an income tax provision of $0.2 million for the three months ended June 30, 2025 and an income tax provision of $2.8 million for the three months ended June 30, 2024. Forthree months ended June 30, 2024, the income tax provision recorded was driven largely by the $75 million clinical advancement milestone achieved in the first quarter of 2024.
Current wording
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 wording
Since inception, we have incurred significant net losses. As of June 30, 2025, we had an accumulated deficit of $189.8 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 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.
Previous wording
As of June 30, 2025, we had cash, cash equivalents and marketable securities of $354.0 million. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of June 30, 2025 will be sufficient to fund our operations into the second 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 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.
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 June 30, 2025, we had cash, cash equivalents and marketable securities of $354.0 million.
Current 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.
Previous wording
In September 2023, we entered into a sales agreement (the "Sales Agreement") with 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, shares will be sold pursuant to our shelf registration statement on Form S-3 (File No. 333-268099) filed with the SEC on November 1, 2022, including the base prospectus contained therein, as declared effective by the SEC on November 7, 2022. 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 June 30, 2025 we have not sold any shares of common stock under the 2023 ATM Program.
Current 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
Previous wording
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 shelf registration statement on Form S-3 (File No. 333-268099) previously filed with the SEC on November 1, 2022 and declared effective by the SEC on November 7, 2022. 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.
Current wording
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 wording
For thesix months ended June 30, 2025, net cash used in operating activities was $68.0 million and was driven by our net loss 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.
Current 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.
Previous wording
For thesix months ended June 30, 2024, net cash provided in operating activities was $14.3 million and was driven by our net income of $78.5 million, net cash used in changes in our operating assets and liabilities of $70.1 million, adjustments for non-cash expenses relating to stock-based compensation expense of $8.4 million and depreciation expense of $1.8 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $4.3 million.
Current 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.
Previous 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.
Current 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.
Previous wording
Net cash provided by investing activities was $1.8 million for thesix months ended June 30, 2024, consisting primarily of $211.3 million from the maturities of marketable securities, partially offset by $207.6 million in purchases of marketable securities and $1.9 million of purchases of property and equipment.
Current 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.
Previous 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 our employee stock purchase plan.
Current 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.
Previous wording
Net cash provided by financing activities was $101.6 million for thesix months ended June 30, 2024, consisting of $100.0 million of proceeds from sales of our common stock and pre-funded warrants, $1.3 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 $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 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, 2025 will be sufficient to fund our operations into the second 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 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 utilize our available capital resources sooner than we expect.
Previous wording
During the six months ended June 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 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.
Previous wording
We have also entered into license agreements under which we are obligated to make certain payments. During the six months ended June 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 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.
Previous wording
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 three months ended June 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. As of June 30, 2025, $1.6 million of the costs associated with the Reduction were paid and the remaining $0.3 million were accrued for and recorded in accrued expenses and other current liabilities.
Current wording
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.
Rearranged current paragraphs (3)
Current report — wording carried from elsewhere
(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 report — wording carried from elsewhere
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.
Current report — wording carried from elsewhere
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.
Rearranged previous paragraphs (1)
Previous report — wording carried elsewhere
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"). The Reduction was substantially completed during the second quarter of 2025.