ROCKET PHARMACEUTICALS, INC.

This Annual Report contains forward-looking statements that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this Annual Report are forward-looking statements. In some cases, you can identify forward-looking statements by words such as “aim,” “anticipate,” “believe,” “can,” “contemplate,” “continue,” “could,” “design,” “develop,” “estimate,” “expect,” “expand,” “future,” “hope,” “intend,” “likely,” “may,” “plan,” “potential,” “predict,” “project,” “pursue,” “seek,” “should,” “strategy,” “target,” “will,” “would,” or the negative of these words or other comparable terminology. These forward-looking statements include, but are not limited to, statements about:

•

our ability to obtain additional funding to conduct our planned research and development efforts;

•

our ability to meet our anticipated milestones for our various drug candidates with respect to the initiation and timing of clinical studies;

•

federal, state, and non-U.S. regulatory requirements, including regulation of our current or any other future product candidates by the FDA;

•

the timing of and our ability to submit regulatory filings, including filings with the FDA, and to obtain and maintain FDA or other regulatory authority approval of, or other action with respect to, our product candidates;

•

our competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting;

•

whether safety and efficacy results of our clinical trials and other required tests for approval of our product candidates provide data to warrant progression of clinical trials, potential regulatory approval or further development of any of our product candidates;

•

our ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, and our ability to apply for and obtain regulatory approval for such product candidates, within currently anticipated timeframes, or at all;

•

our ability to establish key collaborations and vendor relationships for our product candidates and any other future product candidates;

•

our ability to develop our sales and marketing capabilities or enter into agreements with third parties to sell and market any of our product candidates;

•

our ability to acquire additional businesses, form strategic alliances or create joint ventures and our ability to realize the benefit of such acquisitions, alliances or joint ventures;

•

our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire;

•

the development of our direct manufacturing capabilities for our AAV programs;

•

our ability to expand our pipeline to target additional indications that are compatible with our gene therapy technologies;

•

our ability to achieve the expected benefits of our portfolio prioritization and strategic restructuring, including extending our cash runway, and our estimates related to the costs and timing of implementing such initiatives;

•

our ability to successfully operate in non-U.S. jurisdictions in which we currently or in the future do business, including compliance with applicable regulatory requirements and laws;

•

our ability to obtain and enforce patents to protect our product candidates, and our ability to successfully defend ourselves against unforeseen third-party infringement claims;

•

•

our estimates regarding our capital requirements; and

•

our ability to obtain additional financing and raise capital as necessary to fund operations or pursue business opportunities.

We caution you that the foregoing list may not contain all of the forward-looking statements made in this Annual Report.

Any forward-looking statements in this Annual Report reflect our current views with respect to future events or to our future financial performance and involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by these forward-looking statements. Factors that may cause actual results to differ materially from current expectations include, among other things, those listed under Part I, Item 1A. Risk Factors and elsewhere in this Annual Report. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Except as required by law, we assume no obligation to update or revise these forward-looking statements for any reason, even if new information becomes available in the future.

This Annual Report also contains estimates, projections and other information concerning our industry, our business, and the markets for certain diseases, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events, or circumstances may differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, we obtained this industry, business, market and other data from reports, research surveys, studies and similar data prepared by market research firms and other third parties, industry, medical and general publications, government data and similar sources. Unless stated otherwise, references in this Annual Report to “us,” “we,” “our,” or our “Company” and similar terms refer to Rocket Pharmaceuticals, Inc.

Our business is subject to numerous risks and uncertainties that you should be aware of in evaluating our business. These risks and uncertainties include, but are not limited to, the following:

•

Our strategic restructuring may not result in the savings we anticipate, could result in total costs and expenses that are greater than expected and could disrupt our operations.

•

We are and may continue to be targets of securities-related class action and derivative lawsuits and defending against these claims could result in substantial costs and divert management time and resources and have a material adverse effect on our results of operations.

•

We have a history of operating losses, and we may not achieve or sustain profitability. We anticipate that we will continue to incur losses for the foreseeable future. If we fail to obtain additional funding to conduct our planned research and development efforts, we could be forced to delay, reduce, or eliminate our product development programs or commercial development efforts.

•

Our limited operating history may make it difficult for us to evaluate the success of our business to date and to assess our future viability.

•

If, in the future, we are unable to establish sales and marketing capabilities or enter into agreements with third parties to sell and market any of our product candidates, we may not be successful in commercializing those product candidates if and when they are approved.

•

We may need to raise additional funding, which may not be available on acceptable terms, or at all. Failure to obtain this necessary capital when needed may force us to delay, limit or terminate certain of our licensing activities, product development efforts or other operations.

•

We may encounter substantial delays in commencement, enrollment or completion of our clinical trials or may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities, which could prevent us from commercializing our current and future product candidates on a timely basis, if at all.

•

If we have difficulty enrolling a sufficient number of patients to conduct our clinical trials as planned, we may need to delay, limit, or terminate planned clinical trials, the occurrence of any of which would harm our business, financial condition, results of operations and prospects.

•

Preliminary, interim or topline results in our ongoing clinical studies may not be indicative of results obtained when these studies are completed and success in early clinical studies may not be indicative of results obtained in later studies.

•

Our product candidates may cause undesirable and unforeseen side effects or be perceived by the public as unsafe, which

could delay or prevent their advancement into clinical trials or regulatory approval, limit the commercial potential or result in significant negative consequences.

•

Our gene therapy product candidates are based on novel technology, which makes it difficult to predict the time and cost

of product candidate development and subsequently obtaining regulatory approval.

•

Even though we have obtained orphan designation for certain of our product candidates, we may not be able to realize the benefits of such designation, including potential marketing exclusivity of our product candidates, if approved.

•

Even if we successfully complete the necessary preclinical studies and clinical trials, we cannot predict when, or if, we will obtain regulatory approval to commercialize a product candidate and the approval may be for a narrower indication than we seek.

•

We may never obtain approval for any of our product candidates in the U.S. or the EU, or other jurisdictions, which would limit our ability to realize our full market potential.

•

Even if we obtain regulatory approval for a product candidate, we will remain subject to ongoing regulatory obligations and continued regulatory scrutiny.

•

If approved, our product candidates may face competition from biosimilars approved through an abbreviated regulatory

pathway.

•

•

If we are successful in commercializing any product, our relationships with customers and third-party payors will be

subject to applicable anti-kickback, fraud and abuse and other healthcare laws and regulations, which could expose us to

criminal sanctions, civil penalties, exclusion from government healthcare programs, contractual damages, reputational

harm and diminished profits and future earnings.

•

We are subject to stringent laws, rules, regulations, policies, industry standards and contractual obligations regarding data

privacy and security and may be subject to additional related laws and regulations in jurisdictions into which we expand.

•

We are subject to environmental, health and safety laws and regulations, and we may become exposed to liability and

substantial expenses in connection with environmental compliance or remediation activities.

•

We could experience production problems that result in delays in our development or commercialization programs, limit the supply of our products or otherwise harm our business.

•

Our manufacturing facilities are subject to significant government regulations and approvals, which are often costly and could result in adverse consequences to our business if we fail to comply with the regulations or maintain the approvals.

•

Product liability lawsuits against us could cause us to incur substantial liabilities and could limit the potential

commercialization of any products that we may develop.

•

Our ability to successfully develop and commercialize our product candidates will substantially depend upon the availability of reimbursement funds for the costs of the resulting drugs and related treatments.

•

We face intense competition and rapid technological change and the possibility that our competitors may develop therapies that are more advanced or effective than ours.

•

The commercial success of any of our product candidates will depend upon the degree of market acceptance of gene

therapy by physicians, patients, third-party payors and others in the medical community.

•

Ethical, legal, and social issues may reduce demand for any gene therapy products for which we obtain marketing

approval.

•

We may not be successful in our efforts to expand our pipeline of additional product candidates.

•

The success of our R&D activities, clinical testing, and commercialization, upon which we primarily focus, is uncertain.

•

We expect to rely on third parties to conduct some or all aspects of our drug product manufacturing, research, and preclinical and clinical testing, and these third parties may not perform satisfactorily.

•

Disruptions at the FDA and other government agencies caused by funding shortages, Executive Orders, 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, which could negatively impact our business.

•

Our rights to intellectual property for the development and commercialization of our product candidates are subject 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 related technology or are unable to

otherwise protect our intellectual property rights and trade secrets related to our product candidates, we may not be able to

compete effectively in our markets.

•

•

We may not be successful in obtaining or maintaining necessary rights to gene therapy product components and processes for our development pipeline through acquisitions and in-licenses.

•

Changes in U.S. patent law or the patent law of other countries or jurisdictions could diminish the value of patents in

general, thereby impairing our ability to protect our products.

•

•

•

Our business could suffer if it loses the services of, or fails to attract, key personnel.

•

Our employees, principal investigators, consultants, and commercial partners may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading which could harm our business.

•

We may need to expand our organization and may experience difficulties in managing this growth, which could disrupt our operations.

•

Given our commercial relationships outside of the U.S., in particular the EU, a variety of risks associated with international operations could harm our business.

•

We may fail to realize the anticipated benefits of potential acquisitions or business combinations.

•

Future formations of strategic alliances or joint ventures with third parties could disrupt our business and harm our

financial condition and operating results.

•

If conflicts arise between us and our collaborators or strategic partners, these parties may act in a manner adverse to us

and could limit our ability to implement our strategies.

•

significantly, even if our business is performing well.

•

RTW, our largest stockholder, may have the ability to significantly influence matters submitted to stockholders for approval.

•

If we fail to maintain proper and effective internal control over financial reporting, our ability to produce accurate and

timely financial statements could be impaired, which could harm our operating results, investors’ views of us and, as a

result, the value of our common stock.

•

Our internal computer systems, or those of our third-party collaborators or other contractors, may fail or suffer security

breaches, which could result in a material disruption of our development programs.

The summary risk factors described above should be read together with the text of the full risk factors below and in the other information set forth in this Annual Report, including our consolidated financial statements and the related notes, as well as in other documents that we file with the SEC. If any such risks and uncertainties actually occur, our business, prospects, financial condition, and results of operations could be materially and adversely affected. The risks summarized above or described in full elsewhere in this Annual Report are not the only risks that we face. Additional risks and uncertainties not currently known to us, or that we currently deem to be immaterial may also materially adversely affect our business, prospects, financial condition, and results of operations.