FDMT

MKT CAP $619.7MP/E —DIV YIELD —FCF YIELD —REV TTM $92M +278712.1%NET INCOME −$179.2MNET CASH $50.8M52W 7.19 – 16.76SEC XBRL · TTM TO Invalid Date

How FDMT rewrote its risk factors

10-K ITEM 1A · FY2024 → FY2025

Text kept

80%

of sentences unchanged

Added

150

new sentences

Dropped

112

sentences removed

Length

+925

words, now 45,054

New in FY2025

Risks Related to Our Limited Operating History, Financial Condition and Capital Requirements We are in the late stages of drug development for our lead program and have a limited operating history and no products approved for commercial sale, which may make it difficult to evaluate our current business and predict our future success and viability.
We are a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with the potential to transform treatment paradigms and provide unprecedented benefits to patients.
Our limited operating history as a company and stage of drug development make any assessment of our future success and viability subject to significant uncertainty.
We will encounter risks and difficulties frequently experienced by biopharmaceutical companies in rapidly evolving fields, and we have not yet demonstrated an ability to successfully overcome such risks and difficulties.
We have incurred recurring net losses, including net losses of $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 have devoted substantially all of our financial resources and efforts on research and development activities.
Our expenses may increase substantially if and as we: • progress our current and any future product candidates through preclinical and clinical development; • expand our manufacturing facilities and work with our contract manufacturers to scale up the manufacturing processes for our product candidates; • continue our research and discovery activities; • continue the development of our Therapeutic Vector Evolution platform; • initiate and conduct additional preclinical, clinical or other studies for our product candidates; • change or add additional contract manufacturers or suppliers; • seek regulatory approvals and marketing authorizations for our product candidates; • establish sales, marketing and distribution infrastructure to commercialize any products for which we obtain approval; • acquire or in-license product candidates, intellectual property and technologies; • make milestone, royalty or other payments due under any current or future collaboration or license agreements; • obtain, maintain, expand, protect and enforce our intellectual property portfolio; • attract, hire and retain qualified personnel; • experience any delays or encounter other issues related to our operations; • meet the requirements and demands of being a public company; • are adversely impacted by general economic conditions, such as rising inflation, tariffs and increased interest rates; • defend against any product liability claims or other lawsuits related to our products; and • experience delays in our preclinical studies and clinical trials, whether current or planned, due to pandemics or public health emergencies.
As of December 31, 2025, we had $514.0 million in cash and cash equivalents and marketable securities.
Our ability to raise additional capital may be adversely impacted by global economic conditions affecting credit and financial markets (including due to increased interest rates and high inflation rates) and product supply chains.
If we make incorrect determinations regarding the viability or market potential of any of our product candidates or misread trends in the biopharmaceutical industry, particularly in retina and pulmonology, our business could be seriously harmed.
Our quarterly and annual operating results may fluctuate significantly, making it difficult for us to predict our future operating results.
These fluctuations may occur due to a variety of factors, many of which are outside our control and may be difficult to predict, including: • the timing and success or failure of preclinical studies and clinical trials for our product candidates or competing product candidates, or any other change in the competitive landscape of our industry, including consolidation among our competitors or collaboration partners; • the timing, cost, and level of investment in research, development and commercialization activities, which may change from time to time; • the timing of receipt of approvals from regulatory authorities in the United States and internationally; • the timing and status of enrollment and safety and efficacy readouts for our clinical trials; • the cost of manufacturing, as well as building out our supply chain, which may vary depending on the quantity of production, the cost of continuing to establish and scale up our manufacturing capabilities, and the terms of any agreements we enter into with third-party suppliers; • the timing and amount of any option, milestone, royalty or other payments due under any current or future collaboration or license agreement; • coverage and reimbursement policies with respect to our genetic medicine product candidates and potential future drugs that compete with our products, if approved; • expenditures that we may incur to acquire, develop or commercialize additional products and technologies; • the level of demand for our genetic medicine products, if approved, which may vary significantly over time; • future accounting pronouncements or changes in our accounting policies; and • the impact from general macroeconomic trends, such as higher inflation, tariffs and increased interest rates.
The IBC assesses the safety of the research and identifies any potential risk to public health or the environment, and such review may result in some delay before initiation of a clinical trial.
The role of the CAT is to prepare a draft opinion on an application for marketing authorization for an ATMP candidate that is submitted to the EMA.
We cannot guarantee that any clinical trials will be initiated, conducted as planned or completed on schedule, if at all.
In 2025, we initiated our wet AMD Phase 3 studies comparing a single dose of 4D-150 3E10 vg/eye to on-label aflibercept 2mg Q8 weeks.
If we experience any delays in completing these trials, due to delays in enrollment or other factors, it could result in serious harm to our business.
Events that may prevent successful or timely initiation or completion of clinical trials include: • inability to generate sufficient preclinical, toxicology, or other in vivo or in vitro data to support the initiation or continuation of clinical trials; • delays in reaching a consensus with regulatory agencies on study design or implementation of the clinical trials; • delays or failure in obtaining regulatory authorization to commence a trial; • delays in reaching agreement on acceptable terms with prospective contract research organizations (“CROs”) and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and clinical trial sites; • refusal of the FDA to accept data from clinical trials in geographies outside the United States; • delays in identifying, recruiting and training suitable clinical investigators; • delays in identifying, recruiting, and enrolling patients who meet the requirements of our clinical trials; • delays in obtaining required IRB approval or ethics committee opinion at each clinical trial site; • delays in manufacturing, testing, releasing, validating, or importing/exporting sufficient stable quantities of our product candidates for use in clinical trials or the inability to do any of the foregoing; • insufficient or inadequate supply or quality of product candidates or other materials necessary for use in clinical trials, or delays in sufficiently developing, characterizing or controlling a manufacturing process suitable for clinical trials; • imposition of a temporary or permanent clinical hold by regulatory agencies for a number of reasons, including after review of an IND or amendment, CTA or amendment, or equivalent foreign application or amendment; as a result of a new safety finding that presents unreasonable risk to clinical trial participants; or a negative finding from an inspection of our clinical trial operations or study sites; • developments on trials conducted by competitors for related technology that raise FDA or foreign regulatory authority concerns about risk to patients of the technology broadly; or if the FDA or a foreign regulatory authority finds that the investigational protocol or plan is clearly deficient to meet its stated objectives; • delays caused by patients withdrawing from clinical trials or failing to return for post-treatment follow-up; • difficulty collaborating with patient groups and investigators; • failure by our CROs, other third parties, or us to adhere to clinical trial protocols; • failure to perform in accordance with the FDA’s or any other regulatory authority’s good clinical practice (“GCP”) requirements or applicable regulatory guidelines in other countries; • occurrence of adverse events associated with the product candidate that are viewed to outweigh its potential benefits; • changes in regulatory requirements and guidance (including differing expectations across jurisdictions) that require amending or submitting new clinical protocols, expanding enrollment, modifying statistical assumptions (including non-inferiority margins), or submitting new or amended protocols, including for our Phase 3 trials of 4D-150; • changes in the standard of care on which a clinical development plan was based, which may require new or additional trials; • the cost of clinical trials of our product candidates being greater than we anticipate; • clinical trials of our product candidates producing negative or inconclusive results, which may result in our deciding, or regulators requiring us, to conduct additional clinical trials or abandon development of such product candidates; • transfer of manufacturing processes to larger-scale facilities operated by a CDMO or by us, and delays or failure by our CDMOs or us to make any necessary changes to such manufacturing process; • third parties being unwilling or unable to satisfy their contractual obligations to us; and • adverse public perception or regulatory scrutiny of genetic medicine technology may negatively impact the developmental progress or commercial success of products that we develop alone or with collaborators.
Patient enrollment, a determinative factor in the timing of clinical trials, is affected by many factors including the severity and difficulty of diagnosing the disease under investigation, knowledge of the disease in the medical community and availability of effective diagnostic methods, size and distribution of the patient population and process for identifying subjects, access of patients to medical professionals experienced in their disease, our ability to effectively disseminate information about our clinical trials to the patient population and access of patients to such information, eligibility and exclusion criteria for the trial in question, design of the trial protocol, availability, efficacy of, and our ability to compete with approved and standard of care therapies or other clinical trials for the disease or condition under investigation, perceived risks and benefits of the product candidate under trial or testing, availability of genetic testing for potential patients, efforts to facilitate timely enrollment in clinical trials, patient referral practices of physicians, ability to obtain and maintain subject consent, the risk that enrolled subjects will drop out before completion of the trial, the ability to monitor patients adequately during and after treatment, the time and financial commitments required of patients to enroll in our trials beyond the costs covered by the company, and the proximity and availability of and access to clinical trial sites for prospective patients.
Aside from 4D-150, our product candidates are at an early stage of development.
Failure of a product candidate may occur at any stage of preclinical or clinical development, and, because most of our product candidates and our Therapeutic Vector Evolution platform technology are in an early stage of development, there is a relatively higher risk of failure, and we may never succeed in developing marketable products or generating product revenue.
Aside from 4D-150, each of our product candidates is in the early stages of development and will require significant additional clinical development, management of preclinical, clinical, and manufacturing activities, regulatory approval, adequate manufacturing supply, a commercial organization, and significant marketing efforts before we generate any revenue from product sales, if at all.
If any of our product candidates successfully complete clinical trials, we generally plan to seek regulatory approval to market our product candidates in the United States, the EU, and in additional foreign countries where we believe there is a viable commercial opportunity.
We have never commenced, compiled or submitted an application seeking regulatory approval to market any product candidate, which may impede our ability to secure approval and successfully launch our therapies.
Disruptions at the FDA and other government agencies caused by funding shortages, staffing limitations or policy changes could hinder their ability to hire, retain or deploy key leadership and other personnel, or otherwise prevent new or modified products from being developed, approved or commercialized in a timely manner or at all.
Presidential administration has issued certain policies and Executive Orders directed towards reducing the employee headcount and costs associated with U.S. administrative agencies, including the FDA, and it remains unclear the degree to which these efforts may limit or otherwise adversely affect the FDA's ability to conduct routine activities.
Separately, in response to the novel coronavirus pandemic, the FDA postponed most inspections of domestic and foreign manufacturing facilities at various points.
If a prolonged government shutdown occurs, or if renewed global health concerns, funding shortages or staffing limitations prevent the FDA or other regulatory authorities from conducting their regular inspections, reviews, or other regulatory activities, it could significantly impact the ability of the FDA or such other regulatory authorities to timely review and process our regulatory submissions, which could seriously harm our business.
Compliance with the CTR requirements by us and our third-party service providers, such as CROs, may impact our development plans in the EU.
There are a number of large pharmaceutical and biotechnology companies that are currently pursuing the development of products for the treatment of the indications for which we have product candidates, including wet AMD, DME, GA and cystic fibrosis.
Certain of our competitors have commercially approved products for the treatment of the diseases that we are pursuing or may pursue in the future, including Apellis Pharmaceuticals Inc., Astellas Pharma Inc., Regeneron Pharmaceuticals Inc., Roche and Vertex Pharmaceuticals Incorporated.
Companies that we are aware are developing therapeutics in the retina and pulmonology disease areas include large companies with significant financial resources, such as AbbVie Inc, Astellas Pharma Inc., Eli Lilly and Company, Johnson & Johnson, Merck & Company Inc., Regeneron Pharmaceuticals Inc., Roche, Sanofi and Vertex Pharmaceuticals Incorporated, and biopharmaceutical companies such as Apellis Pharmaceuticals Inc., Arcturus Therapeutics Holdings Inc., EyePoint Inc., Kodiak Sciences Inc., Krystal Biotech Inc., Ocular Therapeutix Inc., Recode Therapeutics Inc., REGENXBIO Inc. and Sionna Therapeutics Inc.
In addition to competition from other companies targeting retina and pulmonology, any products we may develop may also face competition from other types of therapies, such as gene-editing therapies and drug delivery devices.
Furthermore, currently approved products could be discovered to have application for treatment of retina and pulmonology indications, which could give such products significant regulatory and market timing advantages over any of our product candidates.
We currently manufacture and test clinical trial material for our products both internally at our cGMP facility, and externally at our partner CDMOs.
We have approximately 17,000 square feet of laboratory and manufacturing space at our headquarters in Emeryville, California, a large portion of which we plan to devote to manufacturing activities for our clinical trials under cGMP.
We currently rely, and expect to continue to rely, on third parties for the production of some of our planned clinical trial drugs and commercial drug materials and, therefore, we can control only certain aspects of their activities.
The facilities used by our contract manufacturers to manufacture certain of our product candidates must be reviewed by the FDA pursuant to inspections that will be conducted after we submit a BLA to the FDA.
If our contract manufacturers cannot successfully manufacture material that conforms to our specifications and the strict regulatory requirements of the FDA or others, we will not be able to obtain and/or maintain regulatory approval for our products as manufactured at their manufacturing facilities.

and 110 more.

Gone since FY2024

Risk Factor Summary Our ability to implement our business strategy is subject to numerous risks that you should be aware of before making an investment decision.
The following is a summary of the principal risks that could seriously harm our business, all of which are more fully described below.
This summary should be read in conjunction with the other risk factors included in this “Risk Factors” section and should not be relied upon as an exhaustive summary of the material risks facing our business. • We are in the early stages of drug development and have a limited operating history and no products approved for commercial sale, which may make it difficult to evaluate our current business and predict our future success and viability. • We have had recurring net losses, and we expect to continue to incur significant net losses for the foreseeable future. • We will require substantial additional capital to finance our operations.
If we are unable to raise such capital when needed, or on acceptable terms, we may be forced to delay, reduce and/or eliminate one or more of our research and drug development programs or future commercialization efforts. • All of our product candidates are based on a novel AAV genetic medicine technology with which there is limited regulatory and clinical experience to date, which makes it difficult to predict the time and cost of product candidate development and subsequently obtaining regulatory approval.
Further, the regulatory approval process for novel product candidates such as ours can be more expensive and take longer than for other, better known or extensively studied therapeutic modalities. • Adverse public perception or regulatory scrutiny of genetic medicine technology may negatively impact the developmental progress or commercial success of products that we develop alone or with collaborators. • Our clinical trials may fail to demonstrate substantial evidence of the safety and efficacy of our product candidates, which would prevent, delay or limit the scope of regulatory approval and commercialization. • Gene therapies are novel, complex and difficult to manufacture.
We could experience production problems that result in delays in our development or commercialization programs, limit the supply of our products or otherwise seriously harm our business. • The regulatory approval processes of the FDA, European Medicines Agency ("EMA") and comparable foreign regulatory authorities are lengthy, expensive, time consuming, and inherently unpredictable.
If we are ultimately unable to obtain regulatory approval for our product candidates, we will be unable to generate product revenue and our business will be substantially harmed. • Our success depends on our ability to protect our intellectual property and our proprietary technologies. • Our rights to develop and commercialize our product candidates are subject in part to the terms and conditions of licenses granted to us by others, and the patent protection, prosecution and enforcement for some of our product candidates may be dependent on our licensors. • Our employees, independent contractors, consultants, research or commercial partners or collaborators and vendors may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements.
Risks Related to Our Limited Operating History, Financial Condition and Capital Requirements We are in the early stages of drug development and have a limited operating history and no products approved for commercial sale, which may make it difficult to evaluate our current business and predict our future success and viability.
We are a clinical-stage genetic medicine company pioneering the development of product candidates using our targeted and evolved AAV vectors.
Our limited operating history as a company and early stage of drug development make any assessment of our future success and viability subject to significant uncertainty.
We will encounter risks and difficulties frequently experienced by early-stage biopharmaceutical companies in rapidly evolving fields, and we have not yet demonstrated an ability to successfully overcome such risks and difficulties.
We have incurred recurring net losses, including net losses of $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 have devoted substantially all of our financial resources and efforts on research and development activities, including for our product candidates and our Therapeutic Vector Evolution platform.
We anticipate that our expenses will increase substantially if and as we: • progress our current and any future product candidates through preclinical and clinical development; • expand our manufacturing facilities and work with our contract manufacturers to scale up the manufacturing processes for our product candidates; • continue our research and discovery activities; • continue the development of our Therapeutic Vector Evolution platform; • initiate and conduct additional preclinical, clinical or other studies for our product candidates; • change or add additional contract manufacturers or suppliers; • seek regulatory approvals and marketing authorizations for our product candidates; • establish sales, marketing and distribution infrastructure to commercialize any products for which we obtain approval; • acquire or in-license product candidates, intellectual property and technologies; • make milestone, royalty or other payments due under any current or future collaboration or license agreements; • obtain, maintain, expand, protect and enforce our intellectual property portfolio; • attract, hire and retain qualified personnel; • experience any delays or encounter other issues related to our operations; • meet the requirements and demands of being a public company; • are adversely impacted by general economic conditions, such as rising inflation, tariffs and increased interest rates; • defend against any product liability claims or other lawsuits related to our products; and • experience delays in our preclinical studies and clinical trials, whether current or planned, due to pandemics or public health emergencies .
As of December 31, 2024, we had $505.5 million in cash and cash equivalents and marketable securities.
Our ability to raise additional capital may be adversely impacted if global economic conditions continue to worsen as a result of volatility in credit and financial markets (including due to increased interest rates and high inflation rates) and to product supply chains.
If we make incorrect determinations regarding the viability or market potential of any of our product candidates or misread trends in the biopharmaceutical industry, in particular for ophthalmology and pulmonology, our business could be seriously harmed.
Our quarterly and annual operating results may fluctuate significantly, which makes it difficult for us to predict our future operating results.
These fluctuations may occur due to a variety of factors, many of which are outside of our control and may be difficult to predict, including: • the timing and success or failure of preclinical studies and clinical trials for our product candidates or competing product candidates, or any other change in the competitive landscape of our industry, including consolidation among our competitors or collaboration partners; • the timing and cost of, and level of investment in research, development and commercialization activities, which may change from time to time; • the timing of receipt of approvals from regulatory authorities in the United States and internationally; • the timing and status of enrollment and safety and efficacy readouts for our clinical trials; • the cost of manufacturing, as well as building out our supply chain, which may vary depending on the quantity of production, the cost of continuing to establish and scale up our internal manufacturing capabilities, and the terms of any agreements we enter into with third-party suppliers; • timing and amount of any option, milestone, royalty or other payments due under any current or future collaboration or license agreement; • coverage and reimbursement policies with respect to our genetic medicine product candidates and potential future drugs that compete with our products, if approved; • expenditures that we may incur to acquire, develop or commercialize additional products and technologies; • the level of demand for our genetic medicine products, if approved, which may vary significantly over time; • future accounting pronouncements or changes in our accounting policies; and • the impact from general macroeconomic trends, such as higher inflation, tariffs and increased interest rates.
For example, most of our collaboration and license revenue for the year ended December 31, 2023 was from AGT.
The IBC assesses the safety of the research and identifies any potential risk to the public health or the environment, and such review may result in some delay before initiation of a clinical trial.
The role of the CAT is to prepare a draft opinion on an application for marketing authorization for ATMP candidate that is submitted to the EMA.
We cannot guarantee that any clinical trials will be initiated or conducted as planned or completed on schedule, if at all.
For example, in 2025, we plan to initiate Phase 3 studies comparing a single dose of 4D-150 3E10 vg/eye to on-label aflibercept 2mg Q8 weeks.
If we experience any delays in completing these trials, due to delays in enrollment or other factors, it could result in serious harm our business.
Events that may prevent successful or timely initiation or completion of clinical trials include: • inability to generate sufficient preclinical, toxicology, or other in vivo or in vitro data to support the initiation or continuation of clinical trials; • delays in reaching a consensus with regulatory agencies on study design or implementation of the clinical trials; • delays or failure in obtaining regulatory authorization to commence a trial; • delays in reaching agreement on acceptable terms with prospective contract research organizations (“CROs”) and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and clinical trial sites; • refusal of the FDA to accept data from clinical trials in geographies outside the United States; • delays in identifying, recruiting and training suitable clinical investigators; • delays in identifying, recruiting, and enrolling patients who meet the requirements of our clinical trials; • delays in obtaining required IRB approval or ethics committee opinion at each clinical trial site; • delays in manufacturing, testing, releasing, validating, or importing/exporting sufficient stable quantities of our product candidates for use in clinical trials or the inability to do any of the foregoing; • insufficient or inadequate supply or quality of product candidates or other materials necessary for use in clinical trials, or delays in sufficiently developing, characterizing or controlling a manufacturing process suitable for clinical trials; • imposition of a temporary or permanent clinical hold by regulatory agencies for a number of reasons, including after review of an IND or amendment, CTA or amendment, or equivalent foreign application or amendment; as a result of a new safety finding that presents unreasonable risk to clinical trial participants; or a negative finding from an inspection of our clinical trial operations or study sites; • developments on trials conducted by competitors for related technology that raise FDA or foreign regulatory authority concerns about risk to patients of the technology broadly; or if the FDA or a foreign regulatory authority finds that the investigational protocol or plan is clearly deficient to meet its stated objectives; • delays caused by patients withdrawing from clinical trials or failing to return for post-treatment follow-up; • difficulty collaborating with patient groups and investigators; • failure by our CROs, other third parties, or us to adhere to clinical trial protocols; • failure to perform in accordance with the FDA’s or any other regulatory authority’s good clinical practice (“GCP”) requirements or applicable regulatory guidelines in other countries; • occurrence of adverse events associated with the product candidate that are viewed to outweigh its potential benefits; • changes in regulatory requirements and guidance that require amending or submitting new clinical protocols; • changes in the standard of care on which a clinical development plan was based, which may require new or additional trials; • the cost of clinical trials of our product candidates being greater than we anticipate; • clinical trials of our product candidates producing negative or inconclusive results, which may result in our deciding, or regulators requiring us, to conduct additional clinical trials or abandon development of such product candidates; • transfer of manufacturing processes to larger-scale facilities operated by a CMO or by us, and delays or failure by our CMOs or us to make any necessary changes to such manufacturing process; • third parties being unwilling or unable to satisfy their contractual obligations to us; and • adverse public perception or regulatory scrutiny of genetic medicine technology may negatively impact the developmental progress or commercial success of products that we develop alone or with collaborators.
Patient enrollment, a determinative factor in the timing of clinical trials, is affected by many factors including the severity of and difficulty of diagnosing the disease under investigation, knowledge of the disease in the medical community and availability of effective diagnostic methods, size and distribution of the patient population and process for identifying subjects, access of patients to medical professionals experienced in their disease, our ability to effectively disseminate information about our clinical trials to the patient population and access of patients to such information, eligibility and exclusion criteria for the trial in question, design of the trial protocol, availability, efficacy of, and our ability to compete with approved and standard of care therapies or other clinical trials for the disease or condition under investigation, perceived risks and benefits of the product candidate under trial or testing, availability of genetic testing for potential patients, efforts to facilitate timely enrollment in clinical trials, patient referral practices of physicians, ability to obtain and maintain subject consent, the risk that enrolled subjects will drop out before completion of the trial, the ability to monitor patients adequately during and after treatment, the time and financial commitments required of patients to enroll in our trials beyond the costs covered by the company, and the proximity and availability of and access to clinical trial sites for prospective patients.
We are at an early stage of development of our product candidates.
Failure of a product candidate may occur at any stage of preclinical or clinical development, and, because our product candidates and our Therapeutic Vector Evolution platform technology are in an early stage of development, there is a relatively higher risk of failure, and we may never succeed in developing marketable products or generating product revenue.
Each of our product candidates is in the early stages of development and will require significant additional clinical development, management of preclinical, clinical, and manufacturing activities, regulatory approval, adequate manufacturing supply, a commercial organization, and significant marketing efforts before we generate any revenue from product sales, if at all.
If any of our product candidates successfully completes clinical trials, we generally plan to seek regulatory approval to market our product candidates in the United States, the EU, and in additional foreign countries where we believe there is a viable commercial opportunity.
We have never commenced, compiled or submitted an application seeking regulatory approval to market any product candidate.
Disruptions at the FDA and other government agencies caused by funding shortages or global health concerns could hinder their ability to hire, retain or deploy key leadership and other personnel, or otherwise prevent new or modified products from being developed, approved or commercialized in a timely manner or at all.
Separately, in response to the novel coronavirus ("COVID-19") pandemic, the FDA postponed most inspections of domestic and foreign manufacturing facilities at various points.
If a prolonged government shutdown occurs, or if global health concerns prevent the FDA or other regulatory authorities from conducting their regular inspections, reviews, or other regulatory activities, it could significantly impact the ability of the FDA or other regulatory authorities to timely review and process our regulatory submissions, which could seriously harm our business.
For example, the BIOSECURE Act (H.R. 7085) implicates U.S. government contracts, grants, and loans to entities that use equipment and services from certain named Chinese biotech companies, and authorizes the U.S. government to name additional Chinese biotechnology companies “of concern.” The BIOSECURE Act was passed by the House of Representatives in September 2024 and a substantially similar bill is pending in the Senate (S.3558).
If the BIOSECURE Act becomes law, or similar laws or restrictions are passed, they would have the potential to severely restrict the ability of U.S. biopharmaceutical companies like us to purchase services or products from, collaborate with, or otherwise work with certain Chinese biotechnology companies “of concern” without losing the ability to contract with, or otherwise receive funding from, the U.S. government.
Compliance with the CTR requirements by us and our third-party service providers, such as CROs, may impact our developments plans in the EU.
There are a number of large pharmaceutical and biotechnology companies that are currently pursuing the development of products for the treatment of the indications for which we have product candidates, including wet AMD, DME, GA, cystic fibrosis, and Fabry disease.

and 72 more.

Sentence-level comparison of Item 1A in the two most recent 10-Ks (FY2024 ↗, FY2025 ↗). A reworded sentence counts as one dropped and one added, so heavy edits read as low “kept”. Headings and page furniture are stripped; nothing is summarised by a model.

Watch FDMT. Know when insiders buy.

One email the day a filing lands. Free.