4D Molecular Therapeutics, Inc.
For the period ended Dec 31, 2025. Filed Mar 18, 2026.
View on SEC.govAbout this filing
A company's yearly report to the SEC, with audited financial statements. It is the fullest account a company gives of its business, its finances and the risks it sees.
FY2025 · Filed Mar 18, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-K
Period ended 2024-12-31; filed 2025-02-28.
0000950170-25-030056
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-K
Period ended 2025-12-31; filed 2026-03-18.
0001193125-26-114075
Item 7. 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 138 previous paragraphs and 141 current paragraphs.
80 unchanged; 39 reworded; 20 added; 15 removed; 4 previous and 2 current paragraphs rearranged.
Tables are not compared: 3 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 (20)
Current report
In March 2025, we initiated 4FRONT-1, our first Phase 3 trial of 4D-150 in wet AMD. Subsequently in February 2026, we announced enrollment completion within an approximately 11-month period, ahead of initial projections, with the clinical trial overenrolled and expected to exceed 500 patients randomized, reflecting strong interest from investigators and patients. We anticipate topline data with the 52-week primary endpoint in the first half of 2027.
Current report
Additionally, 4FRONT-2, our second Phase 3 trial of 4D-150 in wet AMD, was initiated in June 2025. 4FRONT-2 is a global clinical trial and is enrolling both treatment-naïve and recently diagnosed, treatment-experienced patients. We expect 52-week topline data for 4FRONT-2 in the second half of 2027.
Current report
In November 2025, we announced positive long-term interim results from the ongoing 4D-150 PRISM Phase 1/2 clinical trial in wet AMD. 4D-150 demonstrated consistent and durable benefit across all three patient cohorts as evidenced by maintenance of visual acuity, control of retinal anatomy and reduction of treatment burden at all time points with 1.5 to 2 years of follow-up. In addition, a consistent dose response was observed between 3E10 vg/eye, the selected Phase 3 dose, and the lower dose of 1E10 vg/eye. The Phase 3 dose achieved clinically meaningful reductions in treatment burden. No new cases of intraocular inflammation were reported during this follow-up period with up to approximately 3.5 years of follow-up.
Current report
In July 2025, we presented positive 60-week results from the 4D-150 SPECTRA clinical trial in DME where 4D-150 continued to be well tolerated with no intraocular inflammation observed at any timepoint or dose level. In addition, 4D-150 demonstrated durable and dose-dependent clinical activity with sustained gains in visual acuity and anatomic control between 3E10 vg/eye, the selected Phase 3 dose, and lower doses. The Phase 3 dose achieved clinically meaningful 78% reduction in treatment burden vs. projected on-label aflibercept 2mg Q8W. The FDA and EMA are aligned on a proposed single Phase 3 clinical trial being acceptable for possible future licensure for 4D-150 in DME.
Current report
In October 2025, we entered into a Collaboration and License Agreement with Otsuka Pharmaceutical Co., Ltd. (the “Otsuka Collaboration and License Agreement”), pursuant to which we granted Otsuka exclusive rights to develop and commercialize 4D-150 for retinal vascular diseases, including wet AMD and DME, in Japan, China, Australia, and other APAC markets. Otsuka has agreed to lead all regulatory and commercialization activities in its licensed territories. We have agreed to continue to lead all Phase 3 clinical activity globally, including within the APAC region. Otsuka made an upfront cash payment of $85 million and agreed to provide certain cost sharing for global development activities. In addition, we are eligible for up to $335.5 million in potential regulatory and commercial milestone payments and tiered double-digit royalties depending on net sales in Otsuka’s licensed territories. We retain full development and commercialization rights for 4D-150 outside the APAC region, including the United States, Latin America, and Europe.
Current report
Restructuring and Other Charges
Current report
On July 2, 2025, we announced a workforce reduction of approximately 25% of current and planned roles, primarily in the areas supporting early-stage research and development and support functions following a strategic pipeline prioritization to focus on the development of 4D-150 and 4D-710. In connection with the workforce reduction, the Company recorded total expense of $3.2 million including severance, benefits and related termination costs during the year ended December 31, 2025. There are no future payments in connection with the workforce reduction.
Current report
In October 2025, we entered into the Otsuka Collaboration and License Agreement where we granted Otsuka exclusive rights to develop and commercialize 4D-150 for retinal vascular diseases, including wet AMD and DME, in Japan, China, Australia, and other Asia-Pacific markets. Otsuka made an upfront cash payment of $85 million which we recognized as revenue during the fourth quarter of 2025, andagreed to provide certain cost sharing for global development activities.
Current report
not meaningful
Current report
an $8.9 million increase in facilities and other research and development expenses primarily due to higher rent and increased clinical trial activity for our product candidates.
Current report
Follow-on Offerings
Current report
At-the-Market Offering Program
Current report
Collaboration and License Agreements
Current report
In October 2025, we entered into a Collaboration and License Agreement with Otsuka where we granted Otsuka exclusive rights to develop and commercialize 4D-150 for retinal vascular diseases, including wet AMD and DME, in Japan, China, Australia, and other Asia-Pacific markets. Otsuka made an upfront cash payment of $85 million and agreed to provide certain cost sharing for global development activities.
Current report
Our revenue is primarily derived through its license, research, development and commercialization agreements. The terms of these types of agreements may include (i) licenses to our technology, (ii) research and development services, and (iii) supplies of clinical and commercial materials. A performance obligation is a promise in a contract to transfer a distinct good or service to the customer, and is the unit of account in ASC 606. Significant judgment is required to determine whether the individual promised goods or services are distinct. Items are considered distinct if the customer can benefit from them on their own, or together with other readily available resources, and if they are separately identifiable from other items in the contract.
Current report
We recognize as revenue sales-based royalties and milestone payments at the later of (i) when the related sales occur, or (ii) when the performance obligation to which the royalty has been allocated has been satisfied.
Current report
Other variable amounts, such as cost-sharing and development and regulatory milestones, are included in the transaction price to the extent it is probable a significant reversal of cumulative revenue recognized will not occur when the associated uncertainties are resolved. We use the most likely or expected value amount methods as appropriate to estimate variable consideration.
Current report
We allocate the total transaction price to each performance obligation based on the estimated standalone selling prices. Variable consideration is allocated to the specific performance obligations if it is triggered by our performance or the outcomes from such performance, and if such allocation meets the allocation objective of recognizing revenue in amounts of consideration to which we expect to be entitled in exchange for transferring its promised goods or services to the customer.
Current report
We recognize revenue when, or as, the performance obligation is satisfied. Performance obligations recognized at a point in time, such as distinct licenses our intellectual property, are recognized when control transfers, including commencement of the license term. Performance obligations recognized over time, such as research and development services, are recognized using an appropriate measure of progress, such as total cost incurred.
Current report
We record accounts receivable when our right to consideration is unconditional, i.e. if and only passage of time is required before payment is due. Amounts collected or included in accounts receivable but not yet recognized in revenue are recorded as deferred revenues. Amounts recognized in revenue but not included in accounts receivable are recorded as contract assets.
Removed paragraphs (15)
Previous report
In January 2025, we implemented a strategic pipeline prioritization designed to prioritize the development of 4D-150 and 4D-710. Further, we will be seeking strategic alternatives, including potential partnering, for our other clinical stage product candidates: 4D-175 for the treatment of geographic atrophy, 4D-725 for the treatment of alpha-1 antitrypsin deficiency, and 4D-310 for the treatment of Fabry disease cardiomyopathy. In addition, we terminated the development of 4D-110 for the treatment of choroideremia and 4D-125 for the treatment of X-linked retinitis pigmentosa.
Previous report
We have incurred significant operating losses and expect that our operating losses will increase significantly as we, among other things, continue to advance our product candidates through preclinical and clinical development, seek regulatory approval, and prepare for, and, if approved, proceed to commercialization; broaden and improve our platform; acquire, discover, validate and develop additional product candidates; maintain, protect and enforce our intellectual property portfolio; and hire additional personnel.
Previous report
Recent Changes to Management / Leadership Team
Previous report
Effective as of January 2, 2024, our Board of Directors (the “Board”) appointed Noriyuki Kasahara, M.D., Ph.D., as Chief Scientific Officer. On January 1, 2024, Dr. Kasahara delivered notice of his resignation from our Board, including his role as a member and the Chair of the Science and Technology Committee of the Board, effective on January 1, 2024.
Previous report
On August 5, 2024, we named Dhaval Desai, PharmD as Chief Development Officer who will oversee late-stage Product Development, Medical Affairs, Scientific Communications, Regulatory and Quality operations. Dhaval was most recently SVP & Chief Development Officer at Iveric Bio where he led development and approval of IZERVAY.
Previous report
Also on August 5, 2024, we named Carlos Quezada-Ruiz, M.D., FASRS as SVP, Therapeutic Area Head, Ophthalmology who will lead the Ophthalmology franchise and oversee Early- and Late-stage Clinical Development. Dr. Quezada-Ruiz was most recently Group Medical Director, Ophthalmology at Genentech where he led clinical development and approval of VABYSMO and SUSVIMO.
Previous report
Effective September 25, 2024, Christopher Simms joined as Chief Commercial Officer and will oversee Pre-commercial and Commercial organizations, pre-launch preparations and development. Chris was most recently SVP & Chief Commercial Officer at Iveric Bio where he led commercial strategy and execution for the launch of IZERVAY.
Previous report
We expect our general and administrative expenses to increase as a result of increased personnel-related costs, patent costs for our product candidates, consulting, legal and accounting services associated with maintaining compliance with stock exchange listing and requirements of the SEC, investor relations costs, director and officer insurance premiums, and other costs associated with being a public company.
Previous report
a $4.5 million increase in consulting services, facilities related expenses, software expenses and other research and development related expenses.
Previous report
a $5.1 million increase in personnel costs mainly due to increased headcount of general and administrative personnel, including a $0.8 million increase in employee stock-based compensation;
Previous report
a $5.0 million increase mainly due to increased consulting and outside services, increased non-employee stock compensation expense, increased facilities related expenses, software expenses, audit and tax fees, service fees and business taxes, which was partially offset by lower business insurance.
Previous report
In December 2020, we completed our IPO. We issued and sold 9,660,000 shares of common stock at a price to the public of $23.00 per share. The aggregate net proceeds from our IPO were $204.7 million after deducting underwriting discounts and commissions and other offering costs.
Previous report
In November 2021, we completed an underwritten public offering (“2021 Offering”) in which 4,750,000 shares of our common stock were sold at an offering price of $25.00 per share. The net proceeds from the 2021 Offering were $111.1 million after deducting underwriting discounts and commissions and offering expenses.
Previous report
Net Cash Provided by (Used in) Investing Activities
Previous report
The Company has experienced ownership changes in the past, as well as in 2024. As a result of the ownership changes, approximately $21.1 million of the federal research and development credits are permanently limited and will expire unused for federal income tax purposes, and such amounts are excluded from the federal research and development credit carryforwards as of December 31, 2024. Subsequent ownership changes may result in additional limitations.
Reworded paragraphs (39)
Previous wording
We are a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented benefits to patients. Our core pipeline focus is advancing 4D-150 for the treatment of wet AMD and DME through late-stage studies and 4D-710 for treatment of cystic fibrosis through early-stage studies. We believe these product candidates are differentiated and can support meaningful near-term and long-term value generation.
Current wording
We are a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented benefits to patients. Our primary focus is advancing 4D-150 for wet age-related macular degeneration (“wet AMD”) and diabetic macular edema (“DME”) through late-stage studies and potential commercialization and advancing our other pipeline programs, 4D-175 for geographic atrophy, 4D-710 for CF lung disease, and 4D-725 for A1AT lung disease primarily through external funding including strategic partnerships. We believe we are well positioned to discover, develop, manufacture and if approved, commercialize targeted genetic medicines with the potential to transform the lives of patients suffering from debilitating diseases.
Previous wording
Our lead product candidate 4D-150 utilizes our proprietary R100 vector and a transgene cassette encoding aflibercept and inhibitory miRNA targeting vascular endothelial growth factor-C (“VEGF-C”). 4D-150 was designed to become the first backbone therapy for the treatment of retinal vascular diseases by providing multi-year sustained production of anti-VEGF from the retina with a single, safe, intravitreal injection, substantially reducing treatment burden and improving long-term patient outcomes. 4D-150 is initially being developed for the treatment of wet age-related macular degeneration (“wet AMD”) and diabetic macular edema (“DME”).
Current wording
Our lead product candidate 4D-150 utilizes our proprietary R100 vector and a transgene encoding anti-VEGF biologics: aflibercept and an RNA interference (RNAi) approach targeting VEGF-C. The goal for our development and potential commercialization of 4D-150 is to transform the standard of care for large market retinal vascular diseases with a safe, in-office, and durable lifelong backbone therapy substantially reducing treatment burden and improving long-term vision outcomes. 4D-150 is initially being developed for the treatment of wet AMD and DME.
Previous wording
Our second priority product candidate is 4D-710, which we believe is the first known genetic medicine to demonstrate successful delivery and expression of the CFTR transgene in the lungs of people with cystic fibrosis (CF). We believe these results will translate into durable clinical improvements in people with CF, including improved lung function and quality of life.
Current wording
Our other pipeline programs include 4D-710, which we believe is the first known genetic medicine to demonstrate successful delivery and durable expression of the cystic fibrosis transmembrane conductance regulator (“CFTR”) transgene in the lungs of people with cystic fibrosis ("CF") and is currently in Phase 2 development. We believe these results will translate into durable clinical improvements in people with CF, including improved lung function and quality of life. In October 2025, we announced a funding agreement with the Cystic Fibrosis Foundation ("CFF") to provide up to $11 million in additional funding, including $7.5 million in an initial tranche, which was completed in October 2025. The proceeds of this funding agreement enabled the start of the Phase 2 stage of the AEROW clinical trial, redosing, and Phase 3 readiness activities.
Previous wording
Our net losses were $160.9 million and $100.8 million for the years ended December 31, 2024 and 2023, respectively. As of December 31, 2024, we had an accumulated deficit of $576.2 million. We do not expect positive cash flows from operations in the foreseeable future. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and our expenditures on other research and development activities.
Current wording
Our net losses were $140.1 million and $160.9 million for the years ended December 31, 2025 and 2024, respectively. As of December 31, 2025, we had an accumulated deficit of $716.3 million. We do not expect positive cash flows from operations in the foreseeable future. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and our expenditures on other research and development activities.
Previous wording
We will require substantial additional funding to support our continuing operations and further the development of our product candidates. 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, which could include income from collaborations, strategic partnerships, or other strategic arrangements, for the foreseeable future. Adequate funding may not be available when needed or on terms acceptable to us, or at all. Our ability to raise additional funds may be adversely impacted by potential worsening global economic conditions and the recent disruptions to, and volatility in, the credit and financial markets in the United States and worldwide resulting from conflicts in the Middle East, the lingering impact of the COVID-19 pandemic, the war in Ukraine, rising interest rates, inflation and otherwise. If we fail to obtain necessary capital when needed on acceptable terms, or at all, it could force us to delay, limit, reduce or terminate our product development programs, commercialization efforts or other operations. Insufficient liquidity may also require us to relinquish rights to product candidates at an earlier stage of development or on less favorable terms than we would otherwise choose. We cannot assure you that we will ever be profitable or generate positive cash flow from operating activities.
Current wording
We will require substantial additional funding to support our continuing operations and further the development of our product candidates. 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, which could include income from collaborations, strategic partnerships, or other strategic arrangements, for the foreseeable future. Adequate funding may not be available when needed or on terms acceptable to us, or at all. Our ability to raise additional funds may be adversely impacted by potential worsening global economic conditions and the recent disruptions to, and volatility in, the credit and financial markets in the United States and worldwide resulting from conflicts in the Middle East, the lingering impact of the COVID-19 pandemic, the war in Ukraine, rising interest rates, tariffs, inflation, government shutdowns and otherwise. If we fail to obtain necessary capital when needed on acceptable terms, or at all, it could force us to delay, limit, reduce or terminate our product development programs, commercialization efforts or other operations. Insufficient liquidity may also require us to relinquish rights to product candidates at an earlier stage of development or on less favorable terms than we would otherwise choose. We cannot assure you that we will ever be profitable or generate positive cash flow from operating activities.
Previous wording
In August 2019, we amended our agreement with uniQure and entered into a separate new collaboration and license agreement with uniQure. Neither party was required to pay monetary consideration in connection with the amendment or new agreement. We determined the incremental transaction price of the amendment and new agreement to be $5.1 million and recorded the amount as deferred revenue in August 2019. We began recognizing revenue related to uniQure in 2020 and recognized the remaining revenue under the agreement during the third quarter of 2023. We recognized immaterial revenue during the during the year ended December 31, 2023 related to this agreement. See Note 7, Research and Collaboration Agreements, to our financial statements included elsewhere in this report for further discussion regarding the accounting treatment of this agreement.
Current wording
In August 2019, we amended our agreement with uniQure (the "Amended uniQure Agreement") and entered into a separate new collaboration and license agreement with uniQure (the "Second uniQure Agreement"). Neither party was required to pay monetary consideration in connection with the amendment or new agreement. We determined the incremental transaction price of the amendment and new agreement to be $5.1 million and recorded the amount as deferred revenue in August 2019. We began recognizing revenue related to uniQure in 2020 and recognized the remaining revenue under the agreement during the third quarter of 2023. We recognized immaterial revenue during the year ended December 31, 2023 related to this agreement. See Note 6, Research and Collaboration Agreements, to our financial statements included elsewhere in this report for further discussion regarding the accounting treatment of this agreement. The Amended uniQure Agreement and the Second uniQure Agreement were terminated by mutual agreement in November 2025. We did not incur any charges related to the termination of the uniQure Agreement.
Previous wording
Our research and development expenses primarily consist of costs incurred for the discovery and preclinical and clinical development of our product candidates. These expenses include salaries and personnel-related costs, including stock-based compensation of our scientific personnel performing research and development activities; laboratory supplies; research materials; fees paid to CROs to execute preclinical studies and clinical trials; fees paid to CMOs to manufacture materials for preclinical studies and clinical trials; fees related to obtaining technology licenses; consulting costs; costs related to seeking regulatory approval of our product candidates; and allocated facility-related costs, information technology costs, depreciation expense, and other overhead.
Current wording
Our research and development expenses primarily consist of costs incurred for the discovery and preclinical and clinical development of our product candidates. These expenses include salaries and personnel-related costs, including stock-based compensation of our clinical, medical, chemistry, manufacturing and controls and scientific personnel performing research and development activities; laboratory supplies; research materials; fees paid to CROs to execute preclinical studies and clinical trials; fees paid to CDMOs to manufacture materials for preclinical studies and clinical trials; fees related to obtaining technology licenses; consulting costs; costs related to seeking regulatory approval of our product candidates; and allocated facility-related costs, information technology costs, depreciation expense, and other overhead.
Previous wording
We expense all research and development costs in the periods in which they are incurred. We have entered into various agreements with CROs and CMOs. Costs of certain activities are recognized based on an evaluation of the progress to completion of specific tasks. Payments made prior to the receipt of goods or services that will be used or rendered for future research and development activities are deferred and capitalized as prepaid expenses and other current assets on our balance sheet. The capitalized amounts are recognized as expense as the goods are delivered or the related services are performed.
Current wording
We expense all research and development costs in the periods in which they are incurred. We have entered into various agreements with CROs and CDMOs. Costs of certain activities are recognized based on an evaluation of the progress to completion of specific tasks. Payments made prior to the receipt of goods or services that will be used or rendered for future research and development activities are deferred and capitalized as prepaid expenses and other current assets on our balance sheet. The capitalized amounts are recognized as expense as the goods are delivered or the related services are performed.
Previous wording
At this time, we cannot reasonably estimate or know the nature, timing or estimated costs of the efforts that will be necessary to complete the development of, and obtain regulatory approval for, any of our product candidates. We expect our research and development expenses to increase for the foreseeable future as we continue to invest in research and development activities related to developing our product candidates, as our product candidates advance into later stages of development, as we begin to conduct larger clinical trials, as we seek regulatory approvals for any product candidates that successfully complete clinical trials, and incur expenses associated with hiring additional personnel to support our research and development efforts. We have strengthened our ophthalmology senior leadership team and announced formation of the Ophthalmology Advisory Board, ahead of first Phase 3 clinical trial initiation for 4D-150 in wet AMD. The process of conducting the necessary clinical research to obtain regulatory approval is costly and time-consuming, and the successful development of our product candidates is highly uncertain. See the section titled “Risk Factors” for additional risks regarding regulatory development and approval.
Current wording
At this time, we cannot reasonably estimate or know the nature, timing or estimated costs of the efforts that will be necessary to complete the development of, and obtain regulatory approval for, any of our product candidates. However, we expect our overall research and development expenses to increase in the near term primarily for 4D-150 Phase 3 trials in wet AMD and DME. The process of conducting the necessary clinical development to obtain regulatory approval is costly and time-consuming, and the successful development of our product candidates is highly uncertain. See the section titled “Risk Factors” for additional risks regarding regulatory development and approval.
Previous wording
Revenue for the year ended December 31, 2024 decreased by $20.7 million, or 100%, from the year ended December 31, 2023. The decrease in revenue is due to the upfront fees received from the License Agreement with Astellas in 2023.
Current wording
Revenue for the year ended December 31, 2025 increased by $85.2 million from the year ended December 31, 2024. The increase in revenue was primarily due to the upfront fees received from the Otsuka Collaboration and License Agreement in October 2025.
Previous wording
Research and development expenses for the year ended December 31, 2024 increased by $44.2 million, or 46%, from the year ended December 31, 2023. The increase was due to the following:
Current wording
Research and development expenses for the year ended December 31, 2025 increased by $54.4 million, or 38%, from the year ended December 31, 2024. The increase was due to the following:
Previous wording
a $25.2 million increase in research and development trials and consumables expense mainly due to increased clinical trial activity for our product candidates, primarily 4D-150;
Current wording
a $35.5 million increase in research and development trials and consumables expenses mainly due to increased clinical trial activity for our product candidates, primarily 4D-150;
Previous wording
a $14.5 million increase in payroll and personnel expenses due to increased headcount of research and development personnel, including a $4.1 million increase in employee stock-based compensation; and
Current wording
a $10.0 million increase in payroll and personnel expenses primarily due to increased headcount of research and development personnel and one-time severance costs; and
Previous wording
General and administrative expenses for the year ended December 31, 2024 increased by $10.1 million, or 28%, from the year ended December 31, 2023. The increase was due to:
Current wording
General and administrative expenses for the year ended December 31, 2025 increased by $2.5 million, or 5%, from the year ended December 31, 2024. The increase was primarily due to an increase in legal and consulting services.
Previous wording
Other income, net, increased by $15.0 million, or 124%, from the year ended December 31, 2023 to the year ended December 31, 2024. The increase is attributable to higher balances of cash equivalents and marketable securities, and higher market yields on our cash equivalents and marketable securities.
Current wording
Other income, net, decreased by $7.5 million, or 28%, from the year ended December 31, 2024 to the year ended December 31, 2025. The decrease was due to a reduction in invested balances from transfers out of investment accounts to fund operating expenses and lower market yields on our cash equivalents and marketable securities.
Previous wording
We have funded our operations primarily through the sale and issuance of our equity securities, including from the sale of our common stock in our IPO, Follow-on Offerings, and “at-the-market” offerings and the sale of our Series A, Series A-1, Series B and Series C redeemable preferred stock, and to a lesser extent from cash received pursuant to our collaboration and license agreements.
Current wording
As of December 31, 2025, we had cash, cash equivalents and marketable securities of $514.0 million. We have funded our operations primarily through the sale and issuance of our equity securities, including Follow-on Offerings and our “at-the-market” offering program, and to a lesser extent from cash received pursuant to our collaboration and license agreements. Our recent sources of liquidity include the following transactions:
Previous wording
In February 2024, we completed an underwritten public offering (the “2024 Offering” and, together with the 2023 Offering and the 2021 Offering (as defined below), the “Follow-on Offerings”) in which 6,586,015 shares of our common stock were sold at an offering price of $29.50 per share, as well as pre-funded warrants to purchase 3,583,476 shares of our common stock at an offering price of $29.4999 per underlying share pursuant to our effective registration statement on Form S-3. The net proceeds from the 2024 Offering were $281.2 million, after deducting underwriting discounts and commissions and other offering expenses. We also granted the underwriters the option to purchase up to 1,525,423 additional shares of common stock in connection with the offering.
Current wording
In November 2025, we completed an underwritten offering (the "2025 Offering") in which 8,385,809 shares of our common stock were sold at an offering price of $10.51 per share, as well as pre-funded warrants to purchase 1,128,949 shares of our common stock at an offering price of $10.5099 per underlying share. The net proceeds from the 2025 Offering were approximately $93.3 million, after deducting the underwriting discounts and commissions and other offering expenses.
Previous wording
In February 2024, we completed the 2024 Offering in which 6,586,015 shares of our common stock were sold at an offering price of $29.50 per share, as well as pre-funded warrants to purchase 3,583,476 shares of our common stock at an offering price of $29.4999 per underlying share. The net proceeds from the 2024 Offering were $281.2 million, after deducting underwriting discounts and commissions and other offering expenses. We also granted the underwriters the option to purchase up to 1,525,423 additional shares of common stock in connection with the offering.
Current wording
In February 2024, we completed the 2024 Offering in which 6,586,015 shares of our common stock were sold at an offering price of $29.50 per share, as well as pre-funded warrants to purchase 3,583,476 shares of our common stock at an offering price of $29.4999 per underlying share. The net proceeds from the 2024 Offering were $281.2 million, after deducting underwriting discounts and commissions and other offering expenses. We also granted the underwriters the option to purchase up to 1,525,423 additional shares of common stock in connection with the offering. In March 2024, the underwriters exercised their option and purchased 1,259,299 additional shares of common stock resulting in net proceeds of $34.9 million, after deducting underwriting discounts and commissions.
Previous wording
In June 2024, we entered into a Sales Agreement (the “Leerink Sales Agreement”) with Leerink Partners LLC (“Leerink”) as sales agent to sell shares of our common stock, from time to time, with aggregate gross sales proceeds of up to $250.0 million pursuant to a Registration Statement on Form S-3 that we filed with the SEC in February 2024 as an “at-the-market” offering under the Securities Act (the “2024 ATM Offering Program”). For the year ended December 31, 2024, no shares had been sold pursuant to the Leerink Sales Agreement.
Current wording
In June 2024, we entered into a Sales Agreement (the “Leerink Sales Agreement”) with Leerink Partners LLC (“Leerink”) as sales agent to sell shares of our common stock, from time to time, with aggregate gross sales proceeds of up to $250.0 million pursuant to a Registration Statement on Form S-3 that we filed with the SEC in February 2024 as an “at-the-market” offering under the Securities Act. For the year ended December 31, 2025, 1,175,000 shares of the Company's common stock were sold pursuant to the Leerink Sales Agreement for net proceeds to the Company of $9.6 million, after deducting issuance costs.
Previous wording
In March 2022, we filed a Registration Statement on Form S-3 covering the offering of up to $300.0 million of common stock, preferred stock, debt securities, warrants and units, which was declared effective by the SEC in April 2022 (the “S-3 Registration Statement”). In March 2022, we also entered into an Open Market Sales Agreement (the “Sales Agreement”) with Jefferies LLC as sales agent to sell shares of our common stock, from time to time, with aggregate gross sales proceeds of up to $100.0 million pursuant to the S-3 Registration Statement as an “at-the-market” offering under the Securities Act (the “2022 ATM Offering Program”). 1,684,550 shares of the Company’s common stock have been sold pursuant to the Sales Agreement for net proceeds to the Company of $34.4 million, after deducting issuance costs. On May 31, 2024, we terminated the Sales Agreement and the 2022 ATM Offering Program pursuant to the terms of the Sales Agreement.
Current wording
In March 2022, we also entered into an Open Market Sales Agreement (the “Sales Agreement”) with Jefferies LLC as sales agent to sell shares of our common stock, from time to time, with aggregate gross sales proceeds of up to $100.0 million pursuant to the S-3 Registration Statement as an “at-the-market” offering under the Securities Act (the “2022 ATM Offering Program”). On May 31, 2024, we terminated the Sales Agreement and the 2022 ATM Offering Program pursuant to the terms of the Sales Agreement. At termination, 1,684,550 shares of our common stock had been sold pursuant to the Sales Agreement for net proceeds to us of $34.4 million, after deducting issuance costs.
Previous wording
We have experienced recurring net losses and had an accumulated deficit of $576.2 million at December 31, 2024. Our transition to profitability is dependent upon the successful development, approval and commercialization of our product candidates and those of our collaboration partners and achieving a level of revenue adequate to support our cost structure. We expect to continue to incur losses for the foreseeable future.
Current wording
We have experienced recurring net losses and had an accumulated deficit of $716.3 million at December 31, 2025. Our transition to profitability is dependent upon the successful development, approval and commercialization of our product candidates and those of our collaboration partners and achieving a level of revenue adequate to support our cost structure. We expect to continue to incur losses for the foreseeable future.
Previous wording
We expect that our research and development and general and administrative expenses will continue to increase for the foreseeable future. Additionally, we expect our capital expenditures will increase significantly in the future for costs associated with building commercial manufacturing capacity. As a result, we will need significant additional capital to fund our operations, which we may obtain through one or more equity offerings, debt financings or other third-party funding, including potential strategic alliances and licensing or collaboration arrangements.
Current wording
We expect that our overall research and development and general and administrative expenses will increase. As a result, we will need significant additional capital to fund our operations, which we may obtain through one or more equity offerings, debt financings or other third-party funding, the Otsuka Collaboration and License Agreement, and additional potential strategic alliances and licensing or collaboration arrangements.
Previous wording
expanding our manufacturing facilities and working with our contract manufacturers to scale up the manufacturing processes for our product candidates;
Current wording
working with our contract manufacturers to scale up the manufacturing processes for our product candidates;
Previous wording
We believe that our existing cash and cash equivalents will allow us to fund our planned operations for at least one year from the date of the issuance of this report.
Current wording
We believe that our existing cash, cash equivalents and marketable securities will allow us to fund our planned operations for at least one year from the date of the issuance of the financial statements included in this report.
Previous wording
We do not have any committed external sources of funds. Accordingly, we will be required to obtain further funding through public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources to complete the clinical development for the product candidates for treatment of wet AMD, DME, GA, cystic fibrosis lung disease, alpha-1 antitrypsin deficiency lung disease, Fabry disease cardiomyopathy or any other indication we may pursue. If we raise additional funds by issuing equity securities, our stockholders may experience dilution. Any future debt financing into which we enter would result in fixed payment obligations and may involve agreements that include grants of security interests on our assets and restrictive covenants that limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, granting liens over our assets, redeeming stock or declaring dividends, that could adversely impact our ability to conduct our business. Any debt financing or additional equity that we raise may contain terms that could adversely affect our common stockholders. Further, additional funds may not be available when we need them, on terms that are acceptable to us, or at all. Our ability to raise additional capital may be adversely impacted by potential worsening global economic conditions and the recent disruptions to and volatility in the credit and financial markets in the United States and worldwide resulting from the war in Ukraine, conflicts in the Middle East, any expansion of these conflicts, rising interest rates and inflation, natural disasters and pandemics.
Current wording
We have limited committed external sources of funds. Accordingly, we will be required to obtain further funding through public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources to complete the clinical development for the product candidates for treatment of wet AMD, DME, geographic atrophy, cystic fibrosis lung disease, alpha-1 antitrypsin deficiency lung disease or any other indication we may pursue. If we raise additional funds by issuing equity securities, our stockholders may experience dilution. Any future debt financing into which we enter would result in fixed payment obligations and may involve agreements that include grants of security interests on our assets and restrictive covenants that limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, granting liens over our assets, redeeming stock or declaring dividends, that could adversely impact our ability to conduct our business. Any debt financing or additional equity that we raise may contain terms that could adversely affect our common stockholders. Further, additional funds may not be available when we need them, on terms that are acceptable to us, or at all. Our ability to raise additional capital may be adversely impacted by potential worsening global economic conditions and the recent disruptions to and volatility in the credit and financial markets in the United States and worldwide resulting from the war in Ukraine, conflicts in the Middle East, any expansion of these conflicts, rising interest rates and inflation, natural disasters and pandemics.
Previous wording
If we are unable to obtain additional funding, we expect to delay, reduce or eliminate some or all of our research and development programs, product portfolio expansion or investment in internal manufacturing capabilities, which could adversely affect our business. If we raise additional funds through collaborations or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to future revenue streams or product candidates or grant licenses on terms that may not be favorable to us.
Current wording
If we are unable to obtain additional funding, we expect to delay, reduce or eliminate some or all of our research and development programs, product portfolio expansion or investment in manufacturing capabilities, which could adversely affect our business. If we raise additional funds through collaborations or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to future revenue streams or product candidates or grant licenses on terms that may not be favorable to us.
Previous wording
Net cash used in operating activities was $$75.8 million for the year ended December 31, 2023. This was primarily due to the net loss of $100.8 million partially offset by a change of $24.4 million in noncash charges and by a net change of $0.6 million in our operating assets and liabilities. The noncash charges primarily consisted of stock-based compensation expense of $19.7 million, depreciation and amortization of $4.2 million, amortization of operating lease right-of-use assets of $1.5 million and change in fair value of derivative liability of $0.2 million, partially offset by accretion of discount on marketable securities of $1.2 million. The change in operating assets and liabilities was primarily due to a $3.4 million increase in accrued and other liabilities and a $0.9 million increase in accounts payable, offset by a $0.7 million decrease in deferred revenue, $1.5 million decrease in operating lease liabilities and a $1.4 million increase in prepaid expenses and other current assets.
Current wording
Net cash used in operating activities was $109.1 million for the year ended December 31, 2025. This was primarily due to the net loss of $140.1 million partially offset by a change of $24.8 million in noncash charges and by a net change of $6.2 million in our operating assets and liabilities. The noncash charges primarily consisted of stock-based compensation expense of $22.0 million, depreciation and amortization of $4.7 million and amortization of operating lease right-of-use assets of $2.9 million, partially offset by accretion of discount on marketable securities of $4.7 million and $0.1 million in change in fair value of derivative liability. The change in operating assets and liabilities was primarily due to an $8.1 million increase in accrued and other liabilities, a $6.8 million increase in accounts payable, offset by a $3.2 million decrease in operating lease liabilities, a $0.2 decrease in deferred revenue, a $0.4 million increase in prepaid expenses and other current assets and a $4.9 million increase in other assets.
Previous wording
Net cash provided by investing activities was $115.7 million for the year ended December 31, 2023. This was due to maturities of marketable securities of $173.3 million offset by purchases of marketable securities of $54.8 million and purchases of property and equipment of $2.8 million.
Current wording
Net cash used in investing activities was $93.0 million for the year ended December 31, 2025. This was due to purchases of marketable securities of $442.8 million and purchases of property and equipment of $0.5 million, offset by maturities of marketable securities of $350.4 million.
Previous wording
Net cash provided by financing activities was $156.8 million for the year ended December 31, 2023, which was due to proceeds from the issuance of common stock pursuant to public offerings of $129.2 million, proceeds from the issuance of common stock from the ATM offering program of $19.1 million, proceeds from the issuance of common stock upon exercise of stock options of $7.3 million and proceeds from ESPP purchases of $1.2 million.
Current wording
Net cash provided by financing activities was $113.0 million for the year ended December 31, 2025. This was due to proceeds from the issuance of common stock upon underwritten offering, net of issuance costs, of $93.5 million, proceeds from the issuance of common stock from the ATM Offering Program of $9.7 million, proceeds from the issuance of common stock under a stock purchase agreement of $7.5 million, proceeds from the issuance of common stock from purchases from the Company’s 2020 Employee Stock Purchase Plan (“ESPP”) of $1.3 million, and proceeds from the issuance of common stock from the exercise of stock options and warrants of $1.0 million.
Previous wording
Net cash provided by financing activities was $337.3 million for the year ended December 31, 2024. This was due to proceeds from the issuance of common stock upon public offering, net of issuance costs, of $316.1 million, proceeds from the issuance of common stock from the 2022 ATM Offering Program of $15.3 million, proceeds from the issuance of common stock from the exercise of stock options and warrants of $4.6 million and proceeds from the issuance of common stock from purchases from the Company’s 2020 Employee Stock Purchase Plan (“ESPP”) of $1.3 million.
Current wording
Net cash provided by financing activities was $337.3 million for the year ended December 31, 2024. This was due to proceeds from the issuance of common stock upon public offering, net of issuance costs, of $316.1 million, proceeds from the issuance of common stock from the 2022 ATM Offering Program of $15.3 million, proceeds from the issuance of common stock from the exercise of stock options and warrants of $4.6 million and proceeds from the issuance of common stock from purchases from the ESPP of $1.3 million.
Previous wording
As of December 31, 2024, our principal commitments consisted of obligations under our operating lease for our headquarters. Please see Note 9 to our financial statements included elsewhere in this report.
Current wording
As of December 31, 2025, our principal commitments consisted of obligations under our operating lease for our headquarters. Please see Note 8, Commitments and Contingencies, to our financial statements included elsewhere in this report.
Previous wording
Our significant accounting policies are described in Note 2 to our financial statements included elsewhere in this report. We believe the following accounting policies are critical to the process of making significant judgments and estimates in the preparation of our financial statements and understanding and evaluating our reported financial results.
Current wording
Our significant accounting policies are described in Note 2, Summary of Significant Accounting Policies, to our financial statements included elsewhere in this report. We believe the following accounting policies are critical to the process of making significant judgments and estimates in the preparation of our financial statements and understanding and evaluating our reported financial results.
Previous wording
We determine revenue recognition for arrangements within the scope of ASC 606 by performing the following five steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations based on estimated selling prices; and (v) recognition of revenue when (or as) we satisfy each performance obligation.
Current wording
We determine revenue recognition for arrangements within the scope of Accounting Standards Update 2014-09, Revenue from Contracts with Customers (Topic 606) (“ASC 606”) by performing the following five steps: (i) assessment whether a contract with a customer exists; (ii) determination of whether the promised goods or services are performance obligations; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations based on estimated standalone selling prices; and (v) recognition of revenue when (or as) we satisfy each performance obligation.
Previous wording
Our revenue is primarily derived through our license, research, development and commercialization agreements. The terms of these types of agreements may include (i) licenses to our technology, (ii) research and development services, and (iii) services or obligations in connection with participation in research or steering committees. Payments to us under these arrangements typically include one or more of the following: nonrefundable upfront and license fees, research funding, milestone and other contingent payments to us for the achievement of defined collaboration objectives and certain preclinical, clinical, regulatory and sales-based events, as well as royalties on sales of any commercialized products. Arrangements that include upfront payments are recorded as deferred revenue upon receipt or when due and may require deferral of revenue recognition to a future period until performance obligations are met. The event-based milestone payments, royalties and cost reimbursements represent variable consideration, and we use the most likely amount method to estimate this variable consideration. Royalty payments are recognized when earned or as the sales occur. We record cost reimbursements as accounts receivable when right to consideration is unconditional.
Current wording
Payments to us under these arrangements typically include one or more of the following: nonrefundable upfront and license fees, cost-sharing and other forms of research funding, milestone and other contingent payments to us for the achievement of defined collaboration objectives and certain preclinical, clinical, regulatory and sales-based events, as well as royalties on sales of any commercialized products.
Previous wording
A performance obligation is a promise in a contract to transfer a distinct good or service to the customer and is the unit of account in ASC 606. We allocate the total transaction price to each performance obligation based on the estimated selling price and recognize revenue when, or as, the performance obligation is satisfied. We include the unconstrained amount of estimated variable consideration in the transaction price. At the end of each reporting period, we re-evaluate the estimated variable consideration included in the transaction price and any related constraint, and if necessary, adjust our estimate of the overall transaction price.
Current wording
At the end of each reporting period, we re-evaluate the estimated variable consideration included in the transaction price and any related constraint, and if necessary, adjusts its estimate of the overall transaction price.
Previous wording
Significant management judgment is required to determine the level of effort required under an arrangement, and the period over which the Company expects to complete its performance obligations under the arrangement. Changes in these estimates can have a material effect on revenue recognized.
Current wording
Significant management judgment is required to determine the level of effort required under an arrangement and the period over which we expect to complete our performance obligations under the arrangement. Changes in these estimates can have a material effect on revenue recognized.
Previous wording
Expected Term—The expected term for employee options is calculated using the simplified method as we do not have sufficient historical information to provide a basis for estimate. The simplified method is based on the average of the vesting tranches and the contractual life of each grant.
Current wording
Expected Term—The expected term for employee stock options is calculated using the simplified method as we do not have sufficient historical information to provide a basis for estimate. The simplified method is based on the average of the vesting tranches and the contractual life of each grant.
Previous wording
As of December 31, 2024, the unrecognized stock-based compensation expense related to stock options was $54.5 million and is expected to be recognized as expense over a weighted-average period of approximately 2.1 years. The intrinsic value of all outstanding stock options as of December 31, 2024 was approximately $0.3 million, of which $0.3 million related to vested options and zero related to unvested options.
Current wording
As of December 31, 2025, the unrecognized stock-based compensation expense related to stock options and RSUs was $34.2 million and is expected to be recognized as expense over a weighted-average period of approximately 1.7 years. The intrinsic value of all outstanding stock options as of December 31, 2025 was approximately $4.6 million, of which $1.0 million related to vested stock options and $3.6 million related to unvested stock options.
Previous wording
See Note 2 to our financial statements included elsewhere in this report for information.
Current wording
See Note 2, Summary of Significant Accounting Policies, to our financial statements included elsewhere in this report for information.
Rearranged current paragraphs (2)
Current report — wording carried from elsewhere
receiving milestone, royalty or other payments under any current or future collaboration or license agreements;
Current report — wording carried from elsewhere
Net Cash Used in Investing Activities
Rearranged previous paragraphs (4)
Previous report — wording carried elsewhere
In March 2024, the underwriters exercised their option to purchase 1,259,299 additional shares of common stock resulting in net proceeds of $34.9 million, after deducting underwriting discounts and commissions.
Previous report — wording carried elsewhere
In July 2023, we entered into the License Agreement with AGT where we provided our 4D vector technology to AGT to deliver AGT’s genetic payloads for the treatment of rare monogenic diseases. As partial consideration for the rights and licenses granted to AGT under License Agreement, we received an upfront payment of $20.0 million which we recognized as revenue during the third quarter of 2023.
Previous report — wording carried elsewhere
In March 2024, the underwriters exercised their option and purchased 1,259,299 additional shares of common stock resulting in net proceeds of $34.9 million, after deducting underwriting discounts and commissions.
Previous report — wording carried elsewhere
As of December 31, 2024, we had cash, cash equivalents and marketable securities of $505.5 million.