102 added · 111 removed between the two most recent 10-Ks. The risks a company starts — or stops — disclosing are often the story.
Newly disclosed
For example, we manufactured a full batch of ZEVASKYN ® following patient biopsy collection in August 2025 that, despite being a bonafide drug product, could not be released because a rapid sterility assay, mandated by the FDA as a release assay during the final stage of the BLA review, initially yielded a false positive result for sterility.
Failure to comply with current and future laws and regulations could result in government enforcement actions (including the imposition of significant penalties), criminal and/or civil liability for us and our officers and directors, private litigation and/or adverse publicity that negatively affects our business. 36 Trends toward managed health care, health technology assessment, and downward price pressures on medical products and services may limit our ability to profitably sell any drugs that we may develop.
Foreign governments may also adopt non-tariff measures, such as procurement preferences or informal disincentives to engage with, purchase from or invest in U.S. entities, which may limit our ability to compete internationally and attract non-U.S. investment, employees, customers and suppliers.
Disruptions at FDA and other agencies may also increase the time necessary to meet with and provide feedback to entities developing drug products, review and/or approve our submissions, conduct inspections, issue regulatory guidance, or otherwise authorize our actions requiring regulatory approval, which would adversely affect our business.
The commercialization of ZEVASKYN ® exposes us to the risk of product liability claims and other litigation, including claims related to adverse events, off-label promotion, false advertising, pricing, or reimbursement practices.
Although we resumed biopsy collection in November 2025 upon completion of assay optimization and the necessary regulatory submission for its implementation, this false positive caused a manufacturing rejection, which caused a delay in our launch of ZEVASKYN ® .
Even if we are successful in defending ourselves against such claims, litigation could be costly, time-consuming, and damaging to our reputation.
Our commercialization efforts may expose us to increased risk of product liability and other litigation.
Moreover, California adopted the California Consumer Privacy Act of 2018 (“CCPA”), which went into effect in January 2020.
RISK FACTOR SUMMARY Our business is subject to numerous risks and uncertainties, including those described in Item 1A “Risk Factors.” These risks include, but are not limited to the following: ● We may not be able to successfully manufacture or commercialize ZEVASKYN ® and the revenue that we generate from its sales, if any, may be limited. ● Our financial performance depends on the commercial success of ZEVASKYN ® and we have limited experience as a commercial-stage company. ● We may encounter challenges with engaging or coordinating with qualified treatment centers needed for the ongoing commercialization of ZEVASKYN ® . ● Our cell and gene therapy product candidates are based on proprietary methodologies, which makes it difficult to predict the time and cost of product candidate development and regulatory approval.
Additionally, regulatory requirements governing cell and gene therapy products have evolved and may continue to change in the future. ● We may encounter substantial delays in our clinical studies, or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities.
Risks Related to the Commercialization of ZEVASKYN ® and our Ability to Generate Revenue We are in the early stages of commercializing ZEVASKYN ® and our limited operating history as a commercial-stage company makes it difficult to predict the long-term success of our business.
No longer disclosed
For example, in September 2019, we received a clinical hold letter in connection with our phase 3 clinical trial for pz-cel stating that the FDA would not provide approval for us to begin our planned phase 3 clinical trial for pz-cel until we submitted additional data points on transport stability of pz-cel to clinical sites.
For example, for product candidates that we develop and commercialize on our own, we remain responsible for ensuring that each of our IND-enabling studies and clinical studies are conducted in accordance with the study plan and protocols, and that our viral vectors and drug products are manufactured in accordance with GMP as applied in the relevant jurisdictions.
Despite our efforts to protect our trade secrets, our competitors may discover our trade secrets, either through breach of these agreements, independent development or publication of information including our trade secrets in cases where we do not have proprietary or otherwise protected rights at the time of publication.
Nearly all clinical trials involve the processing of these “special categories” of personal data, and thus processing of personal data collected during the course of clinical trials is subject to heightened protections under GDPR. 44 Moreover, California adopted the California Consumer Privacy Act of 2018 (“CCPA”), which went into effect in January 2020.
Although the FDA removed the clinical hold in December 2019 and provided clearance for us to proceed with our planned phase 3 clinical trial, we may encounter similar delays in our clinical studies in the future.
For example, our product candidates may compete with other products and product candidates for access to manufacturing facilities.
In August 2024, we completed a Type A Meeting with the FDA to discuss our forthcoming resubmission of our BLA and in October 2024, we resubmitted our BLA.
The FDA notified the Company in November 2024 that the BLA was accepted for review, with an assigned PDUFA target action date of April 29, 2025.
RISK FACTOR SUMMARY Our business is subject to numerous risks and uncertainties, including those described in Item 1A “Risk Factors.” These risks include, but are not limited to the following: ● Our cell and gene therapy product candidates are based on proprietary methodologies, which makes it difficult to predict the time and cost of product candidate development and regulatory approval.
Additionally, regulatory requirements governing cell and gene therapy products have evolved and may continue to change in the future. ● If we do not obtain the necessary U.S. or worldwide regulatory approvals to commercialize pz-cel, we will not be able to sell pz-cel. ● Even if we receive regulatory approval for pz-cel, our lead drug candidate, we may not be able to successfully manufacture or commercialize the product and the revenue that we generate from its sales, if any, may be limited. ● We may encounter substantial delays in our clinical studies, or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities.
We may also experience delays in developing a sustainable, reproducible and commercial-scale manufacturing process or transferring that process to commercial partners, which may prevent us from completing our clinical studies or commercializing our products on a timely or profitable basis, if at all. 28 In addition, the clinical study requirements of the FDA, the EMA, and other regulatory agencies and the criteria these regulators use to determine the safety and efficacy of a product candidate vary substantially according to the type, complexity, novelty and intended use and market of the potential products.
Further, the cost of compliance with post-approval regulations may have a negative effect on our operating results and financial condition. 34 The Complete Response Letter related to our Biologics License Application for pz-cel for the treatment of patients with recessive dystrophic epidermolysis bullosa may impair our ability to successfully commercialize pz-cel.