ROCKET PHARMACEUTICALS INC

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, DC 20549

FORM 10-K

(Mark One)

For the fiscal year ended December 31,2024

OR

For the transition period from to

Commission File Number:001-36829

(Exact Name of Registrant as Specified in Its Charter)

(609)659-8001

(Registrant’s Telephone Number, including Area Code)

Securities registered pursuant to Section 12(b) of the Act:

Securities registered pursuant to Section 12(g) of the Act: None

Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes☒No□

Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Exchange Act. Yes□No☒

Indicate by check mark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒No□

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒No□

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company or an emerging growth company. See the definitions of“large accelerated filer,” “accelerated filer,” “smaller reporting company” and“emerging growth company” in Rule 12b-2 of the Exchange Act.

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.□

Indicate by check mark whether the registrant has filed a report on and attestation to its management’s assessment of the effectiveness of its internal control over financial reporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the registered public accounting firm that prepared or issued its audit report.☒

If securities are registered pursuant to Section 12(b) of the Act, indicate by check mark whether the financial statements of the registrant included in the filing reflect the correction of an error to previously issued financial statements.□

Indicate by check mark whether any of those error corrections are restatements that required a recovery analysis of incentive-based compensation received by any of the registrant’s executive officers during the relevant recovery period pursuant to §240.10D-1(b).□

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes□No☒

The aggregate market value of the common stock held by non-affiliates of the registrant as of June 30, 2024 (the last business day of the registrant’s most recently completed second fiscal quarter) was approximately $1.4 billion, based upon the closing price on the NASDAQ Global Market reported for such date.

As of February 21, 2025, there were 106,629,701 shares of common stock, $0.01 par value per share, outstanding.

Documents Incorporated by Reference

Part III of this annual report on Form 10-K incorporates by reference information (to the extent specific sections are referred to herein) from the registrant’s definitive proxy statement for its 2025 Annual Meeting of Stockholders (the“Proxy Statement”). The Proxy Statement will be filed with the United States Securities and Exchange Commission within 120 days of the end of the period covered by this Annual Report on Form 10-K.

Table of Contents

Summary of Abbreviated Terms

Rocket Pharmaceuticals, Inc. may be referred to as Rocket, the Company, we, our or us, in this Annual Report, unless the context otherwise indicates. Throughout this Annual Report, we have used terms which are defined below:

FORWARD-LOOKING STATEMENTS

This Annual Report on Form 10-K (“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:

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

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

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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;

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

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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;

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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;

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

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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;

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

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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;

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

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

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

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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;

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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;

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anticipated trends and challenges in our business and the markets in which we operate;

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our estimates regarding our capital requirements; and

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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.

SUMMARY OF THE MATERIAL RISKS ASSOCIATED WITH OUR BUSINESS

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:

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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.

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If we fail to obtain necessary 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.

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We have never generated any revenue from product sales and may never be profitable.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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

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

pathway.

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Healthcare legislative reform measures may have a material adverse effect on our business and results of operations.

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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.

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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.

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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.

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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.

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We have limited experience in manufacturing, and there can be no assurance that we will be able to successfully manufacture products.

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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.

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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.

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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.

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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.

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

approval.

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

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The success of our research and development activities, clinical testing, and commercialization, upon which we primarily focus, is uncertain.

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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.

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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.

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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.

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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.

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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.

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If we breach our license agreements, it could have a material adverse effect on our commercialization efforts for our

product candidates.

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We may be subject to claims challenging the inventorship or ownership of our patents and other intellectual property.

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

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

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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.

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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.

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

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

financial condition and operating results.

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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.

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

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Future sales of our common stock in the public market could cause the market price of our common stock to drop

significantly, even if our business is performing well.

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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.

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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.

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Unfavorable national or global economic conditions or political developments could adversely affect our business,

financial condition or results of operations.

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.