REGENXBIO Inc.

We believe that our facilities are adequate to meet our operating needs for the foreseeable future.

• Because we are developing certain product candidates for the treatment of diseases in which there is little clinical experience and we are using new endpoints and methodologies, there is increased risk that the FDA, the EMA or other regulatory authorities may not consider the endpoints of our clinical trials to provide clinically meaningful results and that these results may be difficult to analyze.
• The results from our preclinical studies or clinical trials for our product candidates may not support as broad a marketing approval as we seek, and the FDA, the EMA or other regulatory authorities may require us to conduct additional clinical trials or evaluate subjects for an additional follow-up period.
• Our planned clinical trials may be substantially delayed, or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities.
• The FDA’s clinical holds on RGX-111 and RGX-121 may delay development, impose additional regulatory requirements, increase costs and adversely impact our business, financial condition and results of operations, or lead to the termination of one or both programs.
• We received a Complete Response Letter from the FDA on our BLA for RGX-121, and if the resubmission of our BLA is not approved in accordance with our expected timeframe, our business could be materially and adversely affected.
• We may be negatively impacted if the results of our planned clinical trials are inconclusive or if there are safety concerns or serious adverse events associated with our product candidates.
• Our NAV Technology Platform, our product candidates or our licensees’ or collaborators’ product candidates, and the process for administering such product candidates, may cause undesirable side effects or have other properties that could delay or prevent regulatory approval of product candidates, limit the commercial potential or result in significant negative consequences following any potential marketing approval.
• Even if we complete the necessary preclinical studies and clinical trials, we cannot predict when, or if, we will obtain regulatory approval to commercialize a product candidate in the United States or elsewhere, and the approval may be for a narrower indication than we seek.
• We face significant competition in an environment of rapid technological change and there is a possibility that our competitors may achieve regulatory approval before us or develop products that are safer, less expensive or more convenient or effective than ours, any of which may harm our financial condition and our ability to successfully market or commercialize our product candidates.
• We have incurred cumulative net losses since inception and expect to regularly incur losses until we have successfully commercialized one or more product candidates and may never achieve or maintain profitability in the future.
• Our existing cash resources may not be sufficient to fund our operations for the next 12 months.
• We will need to raise additional funding, which may not be available on acceptable terms, or at all, and failure to do so may force us to delay, limit or terminate certain of our licensing activities, product development and commercialization efforts or other operations.
• Although we have generated significant revenues from licensing our NAV Technology Platform and our other intellectual property, such as our licensing pursuant to the AbbVie Collaboration and License Agreement and Nippon Shinyaku Collaboration and License Agreement, we have never generated revenue from sales of our product candidates and may never do so in the future.
• If the third parties we rely on to conduct aspects of our clinical trials and certain preclinical research development activities do not meet our deadlines or otherwise conduct the preclinical research and development activities and trials as required, our clinical and preclinical development programs could be delayed or unsuccessful.
• We have in the past, and in the future may, enter into licensing agreements or collaborations with third parties licensing parts of our NAV Technology Platform for the development of product candidates which, if unsuccessful, could harm our business.
• Products intended for use in gene therapies are novel, complex and difficult to manufacture, which could lead to production problems that result in delays in our development or commercialization programs, limit the supply of our products or otherwise harm our business.
• Delays in obtaining regulatory approval of our manufacturing process or disruptions in our manufacturing process, including the development of our cGMP production facility, may delay or disrupt our commercialization efforts.
• We currently rely and expect to continue to rely on third parties to conduct parts of our product manufacturing including key components, and these third parties may not perform satisfactorily.
• Failure to comply with ongoing manufacturing regulatory requirements could cause us to suspend production or put in place costly or time-consuming remedial measures, and shortages of resources or raw materials could result in delays in our research studies, preclinical and clinical development or marketing schedules.
• If we are unable to adequately establish sales and marketing capabilities or enter into agreements with third parties to market and sell our product candidates, if approved, we may be unable to generate any product revenue.
• If we do not achieve our projected development goals in the timeframes we announce and expect, the commercialization of our products may be delayed and, as a result, our stock price may decline.
• Even if we receive regulatory approval, we still may not be able to successfully commercialize our lead product candidates or any future product candidate, and the revenue that we generate from any approved product’s sales, if any, could be limited.
• The insurance coverage and reimbursement status of newly approved products is uncertain. Failure to obtain or maintain adequate coverage and reimbursement for our products, if approved, or the imposition of price controls or other forms of pricing regulation could limit our ability to market those products and decrease our ability to generate product revenue.
• We may not be successful in our efforts to identify or discover additional product candidates and may fail to capitalize on programs or product candidates that may be a greater commercial opportunity or for which there is a greater likelihood of success.
• Our future success depends on our ability to retain key employees, consultants and advisors and to attract, retain and motivate qualified personnel.
• If our employees, principal investigators, consultants or commercial partners engage in misconduct, or if we are unable to comply with federal and state healthcare fraud and abuse laws, false claims laws, health information privacy and security laws or other applicable laws or regulations, then we could face substantial penalties.
• Product liability lawsuits against us could cause us to incur substantial liabilities and could limit licensing of our NAV Technology Platform or commercialization of any product candidates that we may develop.
• If we, our development partners, including our licensees and collaborators, or our third-party manufacturers or suppliers fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could materially harm the success of our business.
• Our information technology systems, or those of our vendors, collaborators or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our business or financial operations, including our licensing and product development programs.
• Our customers are concentrated and therefore the loss of a significant customer may harm our business.
• Changes to tax legislation may adversely affect our business.
• Our rights to license our NAV Technology Platform and to develop and commercialize our product candidates are subject, in part, to the terms and conditions of licenses granted to us by others.
• If we are unable to obtain and maintain patent protection for our products and technology, or if the scope of the patent protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and our ability to successfully license our NAV Technology Platform and commercialize our products and technology may be harmed.
• Our reliance on third parties requires us to share our trade secrets, which increases the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed.
• Our intellectual property licenses with third parties may be subject to disagreements over contract interpretation, which could narrow the scope of our rights to the relevant intellectual property or technology, increase our financial or other obligations to our licensors or other parties, or decrease financial or other obligations of our licensees and collaborators.
• If we fail to comply with our obligations in the agreements under which we license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.
• We may not be successful in obtaining necessary rights to our product candidates through acquisitions and in-licenses.
• We may not be able to protect our intellectual property rights in the United States and throughout the world.
• Issued patents covering our NAV Technology Platform or our product candidates could be found invalid or unenforceable if challenged in court and we may be unable to protect our trade secrets in court.
• Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could materially harm our business.
• We may be subject to claims asserting that our employees, consultants or advisors have wrongfully used or disclosed alleged trade secrets of their current or former employers or claims asserting ownership of what we regard as our own intellectual property.
• Changes in U.S. patent law could diminish the value of patents in general, thereby impairing our ability to protect our products.
• If we do not obtain patent term extension and data exclusivity for our product candidates, our business may be materially harmed.
• Our operating results may fluctuate substantially, which makes our future operating results difficult to predict and could cause the price of our common stock to fluctuate substantially.
• Raising additional capital may cause dilution to our existing stockholders, restrict our operations or require us to relinquish proprietary rights.
• If we engage in future acquisitions or strategic partnerships, this may increase our capital requirements, dilute our stockholders, cause us to incur debt or assume contingent liabilities and subject us to other risks.
• Provisions in our certificate of incorporation and bylaws and under Delaware law might discourage, delay or prevent a change in control of our company or changes in our board of directors and, therefore, depress our stock price.
• Our restated certificate of incorporation includes an exclusive forum clause for certain litigation that may be initiated by our stockholders, which could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with us or our directors, officers or employees.
• Our business could be negatively affected as a result of the actions of activist stockholders or stockholder litigation.
• If we are unable to maintain effective internal control over financial reporting, investors may lose confidence in the accuracy of our financial reports.