Entrada Therapeutics, Inc.

Previous wording

We are a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines that engage intracellular targets that have long been considered inaccessible. Through proprietary, versatile and modular approaches, we are advancing a robust development portfolio of genetic medicines for the potential treatment of neuromuscular and inherited retinal diseases, among others. In 2026, we have been actively progressing our ELEVATE-44 and ELEVATE-45 clinical trials. In addition, our VX-670 partnership with Vertex Pharmaceuticals Incorporated ("Vertex") continues to progress, with Vertex on track to share results during the second half of this year. In addition to the ELEVATE-44-201 Cohort 1 results included in this Quarterly Report, we anticipate reporting additional ELEVATE-44-201 Cohort 1 open-label data by the end of 2026 and topline results from the second cohort of our ELEVATE-44-201 trial in the first quarter of 2027. We expect to report our Cohort 1 ELEVATE-45-201 multiple ascending dose ("MAD") data in October 2026. As of June 30, 2026, we had cash, cash equivalents and marketable securities of $223.0 million. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of June 30, 2026 will be sufficient to fund our operations into the third quarter of 2027.

Current wording

We are a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines that engage intracellular targets that have long been considered inaccessible. Through proprietary, versatile and modular approaches, we are advancing a robust development portfolio of genetic medicines for the potential treatment of neuromuscular and inherited retinal diseases, among others. In 2026, we have been actively progressing our ELEVATE-44 and ELEVATE-45 clinical trials. In addition, our VX-670 partnership with Vertex Pharmaceuticals Incorporated ("Vertex") continues to progress, with Vertex on track to share interim results in the second half of 2026. In addition to the ELEVATE-45-201 Cohort 1 results included in this Quarterly Report, we anticipate additional ELEVATE-44-201 Cohort 1 open-label data by the end of 2026. We anticipate topline data from Cohort 2 of our ELEVATE-44-201 multiple ascending dose ("MAD") trial in the first quarter of 2027 and to report our Cohort 2 ELEVATE-45-201 MAD data in the first half of 2027. We expect that our cash, cash equivalents and marketable securities of $193.0 million as of September 30, 2026 will be sufficient to fund our operations and capital expenditure requirements into the fourth quarter of 2027.

Previous wording

Entrada continues to advance multiple clinical programs in people living with Duchenne muscular dystrophy ("DMD") in the United Kingdom ("UK"), European Union ("EU") and United States ("U.S."). In 2026, we have four clinical-stage programs in our DMD franchise (ENTR-601-44, ENTR-601-45, ENTR-601-50 and ENTR-601-51, which has completed IND-enabling studies but has not yet filed a regulatory application). When combined, we estimate that there are over 11,500 patients in the U.S. and Europe that carry mutations amenable to Entrada's current exon skipping programs. Complementing the ongoing clinical progress of the DMD franchise is the myotonic dystrophy type 1 ("DM1") partnership with Vertex ("VX-670"). Each of these programs utilize the same endosomal escape vehicle, and as such, we anticipate initial data readouts from any one of the candidate clinical trials to provide critical insights for the rest.

Current wording

Entrada continues to advance multiple clinical programs in people living with Duchenne muscular dystrophy ("DMD") in the United Kingdom ("U.K."), European Union ("EU") and United States ("U.S."). In 2026, we have four clinical-stage programs in our DMD franchise (ENTR-601-44, ENTR-601-45, ENTR-601-50 and ENTR-601-51, which has completed CTA-enabling studies but has not yet filed a regulatory application). When combined, we estimate that there are over 11,500 patients in the U.S. and Europe that carry mutations amenable to Entrada's current exon skipping programs. Complementing the ongoing clinical progress of the DMD franchise is the myotonic dystrophy type 1 ("DM1") partnership with Vertex ("VX-670"). Each of these programs utilize the same endosomal escape vehicle, and as such, we anticipate initial data readouts from any one of the candidate clinical trials to provide critical insights for the rest.

Previous wording

ELEVATE-44-201: The Company completed Cohort 1 (dosing at 6 mg/kg) of the global Phase 1/2 MAD portion of the clinical study of ENTR-601-44 in ambulatory patients living with DMD who are amenable to exon 44 skipping. Previously announced data from Cohort 1 achieved the primary objective of favorable safety and tolerability, while also demonstrating (post hoc analysis) significant improvements in functional benefit as measured by change in Time to Rise ("TTR") and Time to Rise velocity ("TTRV") from baseline, versus placebo. All eight of the previously-treated and placebo patients have now transitioned to the 6-dose open-label period of the study, which is on track to report data by the end of 2026. A continuation of safety and functional benefit in this portion of the study would strengthen the competitive profile of the program. Enrollment is complete for Cohort 2 of the global Phase 1/2 MAD portion of the study at the increased dose of 12 mg/kg, and we expect to report topline results including safety, exon skipping, dystrophin and functional readouts in the first quarter of 2027. Data from Cohort 3 (up to 18 mg/kg) will follow, if needed. Based on a combination of non-clinical data, healthy volunteer data and Cohort 1 patient data, we anticipate seeing an increase in dystrophin as we increase dosing amounts in the second and third cohorts. We also intend to open an expansion cohort to increase the number of participants treated in the ELEVATE-44-201 study, as this study has been designed to support an accelerated approval in the U.S. Separately, the U.S. Food and Drug Administration ("FDA") granted Rare Pediatric Disease Designation to ENTR-601-44.

Current wording

ELEVATE-44-201: All eight of the previously treated and placebo participants from Cohort 1 have received their final dose in the 6-dose open-label period of the study and have rolled into the long-term extension (LTE) study. Data from the open-label period remains on track to be received by the end of 2026. A continuation of safety and functional benefit in the open-label period of the study would strengthen the competitive profile of the program. Enrollment is complete for Cohort 2 of the global Phase 1/2 MAD portion of the study at the increased dose of 12 mg/kg, and we expect to report topline results including safety, exon skipping, dystrophin and functional readouts in the first quarter of 2027. Data from Cohort 3 will follow. In the third quarter of 2026, an independent Data Monitoring Committee ("DMC"), per study protocol, reviewed all available safety and pharmacokinetics data from Cohort 2 of the double-blind, placebo-controlled, MAD portion of ELEVATE-44-201. The DMC recommended initiation of the Cohort 3 dose at 18 mg/kg, a dose escalation from 12 mg/kg in Cohort 2. We anticipate seeing an increase in dystrophin and improvements in functional responses 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.

Previous wording

ELEVATE-44-102: The Company believes this clinical trial, in the underserved adult patient population, would be best to initiate at the highest advisable starting dose in patients with advanced disease. Following a review of safety, pharmacokinetic and pharmacodynamic data from Cohort 1 of the ELEVATE-44-201 study in the U.K and EU, we plan to re-engage with the FDA to discuss increasing the planned starting dose in this clinical study. The Company will provide an update on clinical study design and timing following interactions with the FDA.

Current wording

ELEVATE-44-102: We believe 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 our lead programs in the U.K. and EU, we will continue to engage with the FDA and will provide an update on the clinical study design and timing when appropriate.

Previous wording

ELEVATE-45-201: The Company has completed enrollment and dosing of Cohort 1 of the global Phase 1/2 MAD portion of the clinical study of ENTR-601-45 in ambulatory patients living with DMD who are amenable to exon 45 skipping. The Company expects to report data from Cohort 1 (5 mg/kg) in October 2026. All participants from Cohort 1 have transitioned into the open-label, Phase 2 portion of the study. Cohort 2 is dosing at 10 mg/kg and is ongoing. Data from Cohort 2 is expected in the first half of 2027, and data from Cohort 3 (up to 15 mg/kg) will follow, if needed. The proposed ELEVATE-45-201 clinical trial design and dosing regimen is similar to ELEVATE-44-201, incorporating a MAD Phase 1 portion, a 6 dose open-label Phase 2 portion and an expansion cohort. We expect ENTR-601-45 to be both best in class and to be the first PMO-conjugate to generate clinically meaningful data in a population where only low single digit competitive dystrophin production has been observed to date.

Current wording

ELEVATE-45-201: We reported data from Cohort 1 (5 mg/kg) in October 2026. Study participants in Cohort 1 received three doses of 5 mg/kg of ENTR-601-45 or placebo. Topline results met the primary objective of a favorable safety and tolerability profile. Importantly, no hypomagnesemia or renal safety concerns were noted. The secondary objectives also met our expectations. A 0.10% mean increase in dystrophin and a 0.15% mean increase in exon skipping compared with baseline levels was observed in treated participants. All participants from Cohort 1 have transitioned into the open-label, Phase 2 portion of the study. Cohort 2 is dosing at 10 mg/kg and is ongoing. Data from Cohort 2 is expected in the first half of 2027, and data from Cohort 3 (up to 15 mg/kg) is expected to follow. The 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, at higher doses, has the potential to be both best in class and to be the first PMO-conjugate to generate clinically meaningful data in a population where low single digit competitive dystrophin production has been observed to date. In October 2026, the U.S. FDA Office of Orphan Products Development (OOPD) granted orphan drug designation for ENTR-601-45 for the treatment of DMD.

Previous wording

ELEVATE-LTE: A Long Term Extension (LTE) platform study protocol (ENTR-DMD-202) was accepted by U.K. and European authorities. This is a phase 2, open-label long-term extension study in participants with Duchenne muscular dystrophy amenable to exon skipping to assess the long-term safety, tolerability, pharmacokinetics, and efficacy of endosomal escape vehicle phosphorodiamidate morpholino oligomer platform products which will enable continued access to drug for study participants while collecting longer-term safety and efficacy data, including functional measures.

Current wording

ELEVATE-LTE: A LTE platform study protocol (ENTR-DMD-202) was accepted by U.K. and European authorities, and patients who have completed the open-label portion of their Phase 1/2 trials are eligible to roll over into the LTE. This is a Phase 2, open-label long-term extension study in participants with Duchenne muscular dystrophy amenable to exon skipping to assess the long-term safety, tolerability, pharmacokinetics, and efficacy of endosomal escape vehicle phosphorodiamidate morpholino oligomer platform products which will enable continued access to drug for study participants while collecting longer-term safety and efficacy data, including functional measures.

Previous wording

VX-670: Vertex has completed enrollment and continues dosing in the MAD portion of the GALILEO global Phase 1/2 clinical trial of VX-670 in people with DM1. The study is assessing safety and preliminary efficacy, including change from baseline in the splicing index and other endpoints evaluating muscle function and strength. Vertex is on track to complete dosing and report results in the second half of 2026.

Current wording

VX-670: Vertex has completed enrollment and dosing in the MAD portion of the GALILEO global Phase 1/2 clinical trial of VX-670 in people with DM1. The study is assessing both safety and preliminary efficacy, as measured by the change from baseline in the splicing index on muscle biopsy as well as other endpoints, including those that evaluate muscle function and strength, such as video hand opening time (vHOT) and quantitative muscle testing (QMT) score. Vertex is on track to share interim results from the GALILEO study in the second half of 2026.

Previous wording

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 wording

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 nominate a second clinical candidate in ocular diseases by year-end 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.

Previous wording

ENTR-601-44-201 (ELEVATE-44-201) Phase 1/2 Cohort 1 Results

Current wording

ENTR-601-45-201 (ELEVATE-45-201) Phase 1/2 Cohort 1 Results

Previous wording

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 wording

ELEVATE-45-201 is a global, two-part, randomized, double-blind, placebo-controlled Phase 1/2 study evaluating the safety, tolerability and effectiveness of ENTR-601-45 in ambulatory participants ages four to twenty with DMD who are exon 45 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-601-45 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-601-45 at a dose of 5 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-601-45 are evaluated over a longer period of time.

Previous wording

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 wording

The average age of treated participants in Cohort 1 was 6.3 years old with a mean age of diagnosis of 1.9 years. Per protocol, all participants were ambulatory and all were on a stable dose of steroids.

Previous wording

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 wording

Cohort 1 achieved its primary objective, demonstrating a generally favorable safety and tolerability profile. Most treatment emergent adverse events ("TEAEs") were mild to moderate. One participant experienced two serious TEAEs (vomiting and pyrexia), both of which resolved. No discontinuations were reported. All study participants in Cohort 1 have now progressed to the open-label, Phase 2 portion of the study, where they will all receive six doses of 5 mg/kg of ENTR-601-45. The most common TEAE seen was headache.

Previous wording

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 wording

Importantly, clinically relevant markers of kidney function including eGFR, Cystatin-C and magnesium were generally within normal ranges and treated subjects were comparable to placebo. Participant level data is presented below.

Previous wording

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 wording

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 for ENTR-601-44 at 6 mg/kg in the Phase 1 healthy normal volunteer trial disclosed in 2024 and in the first cohort of the Phase 1/2 ELEVATE-44-201 trial disclosed in the first quarter of 2026.

Previous wording

All study participants in Cohort 1 have now progressed to the open-label, Phase 2 portion of the study, where they are receiving six additional doses of 6 mg/kg of ENTR-601-44. We believe that evidence of continued functional responses from this open-label period will further support the early suggestion of a potential drug related improvement in function. Additional study participants are now being dosed in Cohort 2, in which they will receive placebo or three doses of 12 mg/kg of ENTR-601-44. We expect topline safety and efficacy results from Cohort 2 in the first quarter of 2027.

Current wording

All study participants in Cohort 1 have now progressed to the open-label, Phase 2 portion of the study, where they are receiving six additional doses of 5 mg/kg of ENTR-601-45. Additional study participants are now being dosed in Cohort 2, in which they will receive placebo or three doses of 10 mg/kg of ENTR-601-45. We expect topline safety and efficacy results from Cohort 2 in the first half of 2027.

Previous wording

Provision for income tax expense (benefit) recorded in any interim period is based on the estimated effective tax rate for the fiscal year for those tax jurisdictions that can be reliably estimated. There were no significant income tax provisions or benefits recorded for the three and six months ended June 30, 2026 and 2025.

Current wording

Provision for income tax expense (benefit) recorded in any interim period is based on the estimated effective tax rate for the fiscal year for those tax jurisdictions that can be reliably estimated. There were no significant income tax provisions or benefits recorded for the three and nine months ended September 30, 2026. The Company recorded income tax expense of$0.7 million for thethree months ended September 30, 2025 and$0.8 million for thenine months ended September 30, 2025. For the three and 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

Collaboration revenue was $0.9 million for the three months ended June 30, 2026 and $2.0 million for the three months ended June 30, 2025.

Current wording

Collaboration revenue was $0.9 million for the three months ended September 30, 2026 and $1.6 million for the three months ended September 30, 2025.

Previous wording

Research and development expenses were $35.4 million for the three months ended June 30, 2026, compared to $37.9 million for the three months ended June 30, 2025. The decrease of $2.5 million in research and development expenses was primarily attributable to:

Current wording

Research and development expenses were $32.4 million for the three months ended September 30, 2026, compared to $38.4 million for the three months ended September 30, 2025. The decrease of $6.0 million in research and development expenses was primarily attributable to:

Previous wording

•an increase of $0.7 million in direct research and development expenses, driven by additional costs incurred related to the progress of our Duchenne and ocular programs.

Current wording

•a decrease of $3.9 million in direct research and development expenses, primarily driven by lower preclinical costs for the Company’s Duchenne programs; and

Previous wording

•a decrease of $6.1 million in unallocated research and development expenses driven by a decrease in personnel costs of $2.2 million and a decrease in facilities related costs of $3.9 million. Personnel costs are inclusive of stock-based compensation expense of $4.5 million and $4.5 million for the six months ended June 30, 2026 and 2025, respectively.

Current wording

•a decrease of $2.1 million in unallocated research and development expenses, primarily driven by a decrease in facility related and other costs of $2.5 million, offset by an increase in personnel costs of $0.4 million. Personnel costs are inclusive of stock-based compensation expense of $2.3 million for the three months ended September 30, 2026 and 2025.

Previous wording

General and administrative expenses for the six months ended June 30, 2026 were $20.6 million, compared to $21.2 million for the six months ended June 30, 2025. The decrease of $0.6 million was primarily attributable to a decrease in professional services.

Current wording

General and administrative expenses for the three months ended September 30, 2026 were $10.2 million, compared to $10.3 million for the three months ended September 30, 2025.

Previous wording

Total interest and other income, net was$2.2 million for thethree months ended June 30, 2026, compared to$3.9 million of interest and other income for thethree months ended June 30, 2025. The decrease was driven by changes in interest earned from debt securities and money market funds as well as a decrease in the amount of marketable securities held.

Current wording

Total interest and other income was$2.0 million for thethree months ended September 30, 2026, compared to$3.6 million of interest and other income for thethree months ended September 30, 2025. The decrease was driven by changes in interest earned from debt securities and money market funds as well as a decrease in the amount of marketable securities held.

Previous wording

Collaboration revenue was $1.8 million for the six months ended June 30, 2026 and $22.5 million for the six months ended June 30, 2025. The decrease of $20.7 million was primarily a result of us substantially completing our research plan activities for VX-670 during the first quarter of 2025. We continue to perform research activities on additional EEV-based therapeutic candidates for the potential treatment of DM1 pursuant to the Vertex Agreement.

Current wording

Collaboration revenue was $2.7 million for the nine months ended September 30, 2026 and $24.1 million for the nine months ended September 30, 2025. The decrease of $21.4 million was primarily a result of us substantially completing our research plan activities for VX-670 during the first quarter of 2025. We continue to perform research activities on additional EEV-based therapeutic candidates for the potential treatment of DM1 pursuant to the Vertex Agreement.

Previous wording

Research and development expenses were $68.4 million for the six months ended June 30, 2026, compared to $70.0 million for the six months ended June 30, 2025. The decrease of $1.6 million in research and development expenses was primarily attributable to:

Current wording

Research and development expenses were $100.8 million for the nine months ended September 30, 2026, compared to $108.3 million for the nine months ended September 30, 2025. The decrease of $7.5 million in research and development expenses was primarily attributable to:

Previous wording

•a decrease of $3.2 million in unallocated research and development expenses is driven by a decrease in personnel costs of $1.4 million and a decrease in facilities related costs of $1.8 million. Personnel costs are inclusive of stock-based compensation expense of $2.4 million and $2.1 million for the three months ended June 30, 2026 and 2025, respectively; and

Current wording

•a decrease of $8.2 million in unallocated research and development expenses driven by a decrease in facility related and other costs of $6.4 million and a decrease in personnel costs of $1.8 million. Personnel costs are inclusive of stock-based compensation expense of $6.9 million and $6.8 million for the nine months ended September 30, 2026 and 2025, respectively; and

Previous wording

•an increase of $4.5 million in direct research and development expenses, driven by additional costs incurred related to the progress of our Duchenne and ocular programs, partially offset by fewer costs incurred related to our collaboration with Vertex and other preclinical and discovery activities; and

Current wording

•an increase of $0.7 million in direct research and development expenses, driven by higher clinical and manufacturing costs for our Duchenne programs, partially offset by lower preclinical costs for our Duchenne programs and lower costs related to our collaboration with Vertex and other preclinical and discovery activities.

Previous wording

General and administrative expenses for the three months ended June 30, 2026 were $10.5 million, compared to $10.9 million for the three months ended June 30, 2025. The decrease of $0.4 million was primarily attributable to a decrease in professional services.

Current wording

General and administrative expenses for the nine months ended September 30, 2026 were $30.8 million, compared to $31.5 million for the nine months ended September 30, 2025. The decrease of $0.7 million was primarily attributable to a decrease in professional services.

Previous wording

Total interest and other income, net was $4.9 million for the six months ended June 30, 2026, compared to $8.4 million of interest and other income for the six months ended June 30, 2025. The decrease was driven by changes in interest earned from debt securities and money market funds as well as a decrease in the amount of marketable securities held.

Current wording

Total interest and other income was $6.8 million for the nine months ended September 30, 2026, compared to $11.9 million of interest and other income for the nine months ended September 30, 2025. The decrease was driven by changes in interest earned from debt securities and money market funds as well as a decrease in the amount of marketable securities held.

Previous wording

Since inception, we have incurred significant net losses. As of June 30, 2026, we had an accumulated deficit of $355.6 million. Other than the recognition of revenue related to collaboration payments, we expect to continue to generate operating losses and negative operating cash flows for the foreseeable future as we advance our platform and therapeutic candidates. We will not generate any revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for one or more therapeutic candidates, if ever. If we obtain regulatory approval for any therapeutic candidates, we expect to incur significant expenses related to developing our internal commercialization capability to support product sales, marketing and distribution.

Current wording

Since inception, we have incurred significant net losses and 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. As of September 30, 2026, we had an accumulated deficit of $395.3 million. 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

Furthermore, we expect to incur additional costs associated with operating as a public company, including significant legal, accounting, investor relations and other expenses. As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy, as we advance therapeutic candidates through preclinical and, if successful, into clinical development, seek regulatory approval, prepare for and, if any therapeutic candidates are approved, proceed to commercialization and operate as a public company. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the sale of equity, debt financings or other capital sources, including potential collaborations with other companies or other strategic transactions.

Current wording

As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy, as we advance therapeutic candidates through preclinical and, if successful, into clinical development, seek regulatory approval, prepare for and, if any therapeutic candidates are approved, proceed to commercialization and operate as a public company. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the sale of equity, debt financings or other capital sources, including potential collaborations with other companies or other strategic transactions.

Previous wording

To date, we have raised over $850.0 million of gross proceeds from sales of stock to leading biotechnology investors and from the Vertex Agreement. As of June 30, 2026, we had cash, cash equivalents and marketable securities of $223.0 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 September 30, 2026, we had cash, cash equivalents and marketable securities of $193.0 million.

Previous wording

For thesix months ended June 30, 2025, net cash used in operating activities was $68.0 million and was driven by our netloss of $60.5 million, net cash used in changes in our operating assets and liabilities of $17.3 million, adjustments for non-cash expenses relating to stock-based compensation expense of $10.1 million and depreciation expense of $2.0 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $2.3 million.

Current wording

For thenine months ended September 30, 2026, net cash used in operating activities was $104.1 million and was driven by our netloss of $122.2 million, net cash provided bychanges in our operating assets and liabilities of $2.4 million, adjustments for non-cash expenses relating to stock-based compensation expense of $14.7 million and depreciation expense of $2.3 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $1.3 million.

Previous wording

For thesix months ended June 30, 2026, net cash used in operating activities was $74.2 million and was driven by our netloss of $82.5 million, net cash used in changes in our operating assets and liabilities of $2.2 million, adjustments for non-cash expenses relating to stock-based compensation expense of $9.9 million and depreciation expense of $1.6 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $1.0 million.

Current wording

For thenine months ended September 30, 2025, net cash used in operating activities was $96.3 million and was driven by our netloss 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

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 $124.9 million for thenine months ended September 30, 2026, consisting primarily of $148.8 million from the maturities of marketable securities, partially offset by $23.7 million in purchases of marketable securities and $0.2 million of purchases of property and equipment.

Previous wording

Net cash provided by investing activities was $81.9 million for thesix months ended June 30, 2026, consisting primarily of $97.7 million from the maturities of marketable securities, partially offset by $15.8 million in purchases of marketable securities.

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 financing activities was $0.5 million for thesix months ended June 30, 2026, consisting of $0.2 million in proceeds from stock option exercises and $0.3 million from the issuance of common stock under our 2021 Employee Stock Purchase Plan, as amended (the "2021 ESPP").

Current wording

Net cash provided by financing activities was $0.5 million for thenine months ended September 30, 2026, consisting of $0.2 million in proceeds from stock option exercises and $0.3 million from the issuance of common stock under our 2021 Employee Stock Purchase Plan, as amended (the "2021 ESPP").

Previous wording

Net cash provided by financing activities was $0.7 million for thesix months ended June 30, 2025, consisting of $0.4 million in proceeds from stock option exercises and $0.3 million from the issuance of common stock under the 2021 ESPP.

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 the 2021 ESPP.

Previous wording

We expect to incur significant expenses and operating losses for the foreseeable future as we advance the preclinical and, if successful, the clinical development of our programs. Our operating expenses and future funding requirements are expected to increase substantially as we continue to advance our portfolio of programs. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of June 30, 2026 will be sufficient to fund our operations for at least 12 months from the date of this report. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we expect.

Current wording

We expect to incur significant expenses and operating losses for the foreseeable future as we advance the preclinical and 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. We expect that our cash, cash equivalents and marketable securities of $193.0 million as of September 30, 2026 will be sufficient to fund our operations and capital expenditure requirements into the fourth quarter of 2027.

Previous wording

During the six months ended June 30, 2026, there were no material changes to our lease commitments from those described in Note 11, Leases, of our financial statements in the Annual Report.

Current wording

During the nine months ended September 30, 2026, there were no material changes to our lease commitments from those described in Note 11, Leases, of our financial statements in the Annual Report.

Previous wording

We have also entered into license agreements under which we are obligated to make certain payments. During the six months ended June 30, 2026, there were no material changes to our commitments and contingencies related to our license agreements from those described in "Business—Intellectual property— License agreement with The Ohio State University" and Note 11, Commitments and Contingencies, to our financial statements in the Annual Report. For additional information regarding our license agreements, refer to Note 11, Commitments and Contingencies, to our condensed consolidated financial statements in this Quarterly Report.

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, 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.