In the reported quarter, general and administrative (G&A) expenses rose 40% year over year to $22.1 million. The upside was driven by an increase in the company's commercial preparation expenses in anticipation of a potential FDA approval for Kresladi in severe LAD-I. A month has gone by since the last earnings report for Rocket Pharmaceuticals (RCKT) . Shares have lost about 4.8% in that time frame, underperforming the S&P 500. Will the recent negative trend continue leading up to its next earnings release ...

Regulatory Approvals and Clinical Trials - The FDA accepted the Biologics License Application for RP-L201 for severe LAD-I, with regulatory filings for FA anticipated in 2024[102]. - The company has initiated a Phase 1 study for Plakophilin-2 Arrhythmogenic Cardiomyopathy after receiving FDA clearance[104]. - The global Phase 2 pivotal trial for RP-A501 has been aligned with the FDA, targeting 12 patients with a dose level of 6.7 x 10^13 GC/kg[125]. - The company received regenerative medicine advanced therapy designation from the FDA and priority medicines designation from the EMA for RP-A501 in 2023[127]. - A BLA filing for RP-L201 was accepted by the FDA with priority review in October 2023, with an initial Prescription Drug User Fee Act date of March 31, 2024[154]. - The Phase 1/2 trial of RP-L201 showed 100% overall survival at 12 months post-infusion for all nine LAD-I patients, with significant reductions in hospitalizations and infections[152]. Financial Performance and Funding - The company has sold 4.2 million shares under the at-the-market offering program for gross proceeds of $65.8 million, resulting in net proceeds of $63.8 million[104]. - The company raised approximately $1.0 billion from investors through equity and convertible debt financing from inception through March 31, 2024[165]. - The company has not generated any revenue from product sales to date and does not expect to do so in the near future[166]. - The net loss for the three months ended March 31, 2024, was $62.1 million, compared to a net loss of $58.3 million for the same period in 2023[180]. - Total operating expenses increased by $5.2 million to $67.4 million for the three months ended March 31, 2024, compared to $62.2 million in the same period of 2023[180]. - Cash used in operating activities was $56.9 million for the three months ended March 31, 2024, compared to $57.6 million for the same period in 2023[188]. - The company had an accumulated deficit of $1.02 billion as of March 31, 2024[186]. - The company expects R&D expenses to increase for the foreseeable future as it continues to invest in product candidates[172]. Research and Development - RP-A501 is in clinical trials for Danon Disease, with an estimated prevalence of 15,000 to 30,000 patients in the U.S. and EU[111]. - In the Phase 1 trial of RP-A501, a low-dose cohort showed a 98% reduction in high sensitivity troponin I and an 83% reduction in BNP after 36 months[119]. - The company has produced 2 cGMP RP-A501 batches with superior specifications compared to Phase I material, potentially optimizing the safety profile[121]. - The company is focusing on a low dose of 6.7e13 gc/kg for RP-A501 to mitigate safety concerns observed in higher doses[115]. - The NYHA Class assessment indicated that patients in the low-dose cohort exhibited improvements, with some achieving Class I status[116]. - RP-A501 showed durable treatment activity with improvements in biomarkers and NYHA class in pediatric and adult patients over follow-up periods of 6 to 36 months[124]. - The ongoing Phase 2 study of RP-L102 has treated 14 patients, achieving phenotypic correction in at least 6 of 10 evaluable patients with ≥12 months of follow-up[145]. - The primary endpoint for the RP-L102 study is resistance to mitomycin-C in bone marrow stem cells, with a threshold of 10% for marketing application support[144]. - As of April 17, 2023, RP-L102 demonstrated sustained genetic correction in 8 of 12 evaluable patients and comprehensive phenotypic correction in 7 of 12 evaluable patients with ≥12 months of follow-up[147]. - The safety profile of RP-L102 remains highly favorable, with no signs of bone marrow dysplasia or insertional mutagenesis observed[147]. - RP-L301 has shown robust and sustained efficacy in adult patients, including hemoglobin normalization and transfusion independence, with no serious adverse events reported[158]. - The company is initiating a 10-patient, single-arm Phase 2 pivotal trial for RP-L301, with a primary endpoint of ≥1.5 point hemoglobin improvement at 12 months[161]. - The manufacturing facility in Cranbury, New Jersey, has been scaled up to produce AAV drug products for the Phase 2 pivotal study[162]. - The BAG3-DCM program aims to address a patient population of approximately 30,000 individuals in the U.S. with no current therapies targeting the underlying cause[135]. - The company completed the acquisition of Renovacor, enhancing its gene therapy capabilities for BAG3-DCM[137]. - The prevalence of PKP2-ACM is estimated at 50,000 patients in the U.S. and EU, highlighting a significant unmet medical need[130]. - The company has initiated a multi-center Phase 1 study for RP-A601, targeting PKP2-ACM patients with a starting dose of 8 x 10^13 GC/kg[133]. Cash and Investments - The company had $330.3 million in cash, cash equivalents, and investments as of March 31, 2024, which is expected to fund operations into 2026[186]. - Cash provided by investing activities was $35.0 million for the three months ended March 31, 2024, primarily from proceeds of $101.0 million from the maturities of investments[191]. - As of March 31, 2024, the company’s cash, cash equivalents, and marketable securities are primarily invested in U.S. treasury securities and corporate bonds, classified as available-for-sale securities[196]. - If market interest rates increase by 100 basis points, the net fair value of the company's interest-sensitive marketable securities would hypothetically decline by $1.3 million[197]. - The company maintains significant amounts of cash and marketable securities exceeding federally insured limits, which poses potential risks of loss due to financial institution instability[197]. Internal Controls and Legal Matters - The company's management evaluated the effectiveness of disclosure controls and procedures as of March 31, 2024, concluding they were effective at a reasonable assurance level[199]. - There were no changes in internal control over financial reporting during the reporting period that materially affected the company's internal controls[202]. - The company does not believe it is party to any legal claims that would materially adversely affect its business, although litigation can still impact due to costs and resource diversion[204]. Expenses - R&D expenses decreased by $1.1 million to $45.2 million for the three months ended March 31, 2024, compared to $46.4 million in the same period of 2023[181]. - General and administrative expenses increased by $6.3 million to $22.1 million for the three months ended March 31, 2024, compared to $15.8 million in the same period of 2023[182]. - Other income increased by $1.5 million to $5.3 million for the three months ended March 31, 2024, compared to $3.9 million in the same period of 2023[183].

Exhibit 99.1 Rocket Pharmaceuticals Reports First Quarter 2024 Financial Results and Highlights Recent Progress Advanced RP-L102 for Fanconi Anemia towards regulatory reviews; EMA accepted MAA for review and BLA submission anticipated in the first half of 2024 Continued preparations to launch LV portfolio beginning with KRESLADI™ (marnetegragene autotemcel) for severe LAD-I; PDUFA date of June 30, 2024 Appointed Aaron Ondrey as Chief Financial Of icer, bringing seasoned leadership experience in commercial-s ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR □ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-36829 Rocket Pharmaceuticals, Inc. (Exact Name of Registrant as Specified in Its Charter) (Registrant's Telephone Number, including Ar ...

Exhibit 99.1 Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Progress Advancing leading pipeline of six disclosed programs across AAV cardiovascular and LV hematology portfolios; all milestones remain on track for 2024 Expanding commercial capabilities to support launch of LV portfolio beginning with KRESLADITM (marnetegragene autotemcel) for severe LAD-I; PDUFA date of June 30, 2024 Cash, cash equivalents and investments of approximately $407.5M; exp ...

Form 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Commission File Number: 001-36829 Rocket Pharmaceuticals, Inc. (Exact name of registrant as specified in its charter) (State or other jurisdicti ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ________ to ________ Commission File Number: 001-36829 Rocket Pharmaceuticals, Inc. (Exact name of registrant as specified in its charter) (State or other jurisdiction of ...

[PART I - FINANCIAL INFORMATION](index=2&type=section&id=PART%20I%20-%20FINANCIAL%20INFORMATION) [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) The company reported no revenue in Q1 2023, with net loss increasing to $58.3 million and total assets decreasing [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) Total assets decreased to $514.8 million as of March 31, 2023, primarily due to reduced cash and cash equivalents Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2023 | December 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $64,579 | $140,517 | | Investments | $295,462 | $259,153 | | Total current assets | $338,033 | $364,060 | | Total assets | $514,757 | $551,807 | | **Liabilities & Stockholders' Equity** | | | | Total current liabilities | $31,206 | $39,169 | | Total liabilities | $55,881 | $62,121 | | Total stockholders' equity | $458,876 | $489,686 | | Accumulated deficit | $(772,110) | $(713,775) | [Consolidated Statements of Operations](index=6&type=section&id=Consolidated%20Statements%20of%20Operations) The company reported no revenue and a net loss of $58.3 million for Q1 2023, driven by increased operating expenses Statement of Operations Summary (in thousands, except per share data) | Metric | Q1 2023 | Q1 2022 | | :--- | :--- | :--- | | Revenue | $0 | $0 | | Research and development expenses | $46,371 | $30,794 | | General and administrative expenses | $15,823 | $11,770 | | Total operating expenses | $62,194 | $42,564 | | Loss from operations | $(62,194) | $(42,564) | | Net loss | $(58,335) | $(42,982) | | Net loss per share - basic and diluted | $(0.73) | $(0.67) | [Consolidated Statements of Cash Flows](index=10&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities increased to $57.6 million in Q1 2023, with overall cash decreasing by $75.9 million Cash Flow Summary (in thousands) | Activity | Q1 2023 | Q1 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | $(57,560) | $(39,223) | | Net cash used in investing activities | $(36,721) | $(62,995) | | Net cash provided by financing activities | $18,343 | $76 | | Net change in cash, cash equivalents and restricted cash | $(75,938) | $(102,142) | | Cash, cash equivalents and restricted cash at end of period | $65,919 | $131,895 | [Notes to Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) Notes detail the company's gene therapy business, financial risks, accounting policies, cash runway, and acquisition details - The company is a clinical-stage biotechnology firm focused on gene therapies for rare diseases, with three clinical-stage ex vivo LV programs (FA, LAD-I, PKD) and an in vivo AAV program for Danon disease[32](index=32&type=chunk) - The company has incurred losses since inception, with an accumulated deficit of **$772.1 million** as of March 31, 2023. Existing cash, cash equivalents, and investments of **$360.0 million** are expected to fund operations into the first half of 2025[35](index=35&type=chunk) - Through its at-the-market (ATM) offering program, the company sold **0.9 million** shares for net proceeds of **$17.2 million** during Q1 2023. Cumulatively, the program has raised **$63.8 million** in net proceeds[36](index=36&type=chunk)[64](index=64&type=chunk) - The acquisition of Renovacor on December 1, 2022, was accounted for as a business combination with a total consideration of **$72.3 million**, resulting in **$25.2 million** of IPR&D intangible assets and **$8.3 million** of goodwill[90](index=90&type=chunk)[91](index=91&type=chunk)[95](index=95&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=28&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses gene therapy pipeline advancements, Q1 2023 financial results with a **$58.3 million** net loss, and a cash runway into H1 2025 [Pipeline Overview](index=30&type=section&id=Pipeline%20Overview) The company's gene therapy pipeline shows progress with RMAT designation for Danon Disease, IND filings, and BLA submissions targeted for 2023 - **Danon Disease (RP-A501):** Received RMAT designation from the FDA. The company anticipates initiating the initial component of a global pivotal study in Q2 2023[129](index=129&type=chunk) - **PKP2-ACM (RP-A601):** The company anticipates filing an Investigational New Drug (IND) application in the second quarter of 2023[134](index=134&type=chunk) - **BAG3-DCM:** The company plans to submit an IND for its BAG3-DCM program in the first half of 2024[139](index=139&type=chunk) - **Fanconi Anemia (RP-L102):** The primary endpoint in the pivotal Phase 2 study has been achieved. The company anticipates filing a Biologics License Application (BLA) in the fourth quarter of 2023[147](index=147&type=chunk)[148](index=148&type=chunk) - **LAD-I (RP-L201):** Data showed **100%** overall survival at 12 months post-infusion. The company anticipates filing a BLA in the second quarter of 2023[154](index=154&type=chunk)[155](index=155&type=chunk) - **PKD (RP-L301):** Enrollment in the Phase 1 study is complete. Initiation of the Phase 2 pivotal trial is anticipated in the fourth quarter of 2023[161](index=161&type=chunk) [Results of Operations](index=46&type=section&id=Results%20of%20Operations) Q1 2023 R&D expenses increased by **$15.6 million** to **$46.4 million**, and G&A expenses rose by **$4.1 million** to **$15.8 million** Comparison of Operations (in thousands) | Item | Q1 2023 | Q1 2022 | Change | | :--- | :--- | :--- | :--- | | Research and development | $46,371 | $30,794 | $15,577 | | General and administrative | $15,823 | $11,770 | $4,053 | | Loss from operations | $(62,194) | $(42,564) | $(19,630) | | Net loss | $(58,335) | $(42,982) | $(15,353) | - The **$15.6 million** increase in R&D expenses was driven by higher manufacturing and development costs (**$2.8 million**), compensation and benefits from increased headcount (**$6.6 million**), direct materials (**$0.9 million**), and laboratory supplies (**$0.9 million**)[184](index=184&type=chunk) - The **$4.1 million** increase in G&A expenses was primarily due to increases in commercial preparation expenses (**$1.1 million**), compensation and benefits (**$0.7 million**), and non-cash stock compensation (**$1.1 million**)[185](index=185&type=chunk) [Liquidity and Capital Resources](index=46&type=section&id=Liquidity%20and%20Capital%20Resources) The company had **$360.0 million** in cash and investments as of March 31, 2023, expected to fund operations into H1 2025 - As of March 31, 2023, the company had **$360.0 million** in cash, cash equivalents, and investments. These resources are expected to fund operating expenses and capital requirements into the first half of 2025[189](index=189&type=chunk) Cash Flow Summary (in thousands) | Activity | Q1 2023 | Q1 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | $(57,560) | $(39,223) | | Net cash used in investing activities | $(36,721) | $(62,995) | | Net cash provided by financing activities | $18,343 | $76 | - Cash used in operations in Q1 2023 was **$57.6 million**, resulting from a net loss of **$58.3 million**, adjusted for non-cash items like stock-based compensation (**$8.9 million**) and changes in operating assets and liabilities[192](index=192&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=49&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) Market risk is primarily from cash and marketable securities, invested in high-quality instruments, with immaterial interest rate risk - The company's market risk is mainly confined to its cash and marketable securities, which are invested in high-quality instruments like U.S. treasury securities and corporate bonds[200](index=200&type=chunk) - The company maintains cash balances at financial institutions that may exceed federally insured limits, posing a potential risk of loss given the instability of financial institutions[201](index=201&type=chunk) [Controls and Procedures](index=49&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of March 31, 2023, with no material changes - Management concluded that as of March 31, 2023, the company's disclosure controls and procedures were effective at a reasonable assurance level[202](index=202&type=chunk) - No changes in internal control over financial reporting occurred during the quarter that have materially affected, or are reasonably likely to materially affect, these controls[204](index=204&type=chunk) [PART II - OTHER INFORMATION](index=50&type=section&id=PART%20II%20-%20OTHER%20INFORMATION) [Legal Proceedings](index=50&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently party to any legal proceedings expected to have a material adverse effect - The Company does not believe it is party to any claim or litigation that would have a material adverse effect on its business[205](index=205&type=chunk) [Risk Factors](index=50&type=section&id=Item%201A.%20Risk%20Factors) No material changes to the risk factors previously disclosed in the company's 2022 Form 10-K - There have been no material changes from the risk factors previously disclosed in the company's 2022 Form 10-K[206](index=206&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=50&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) No unregistered sales of equity securities or use of proceeds to report - None[207](index=207&type=chunk) [Exhibits](index=51&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Quarterly Report on Form 10-Q, including agreements and certifications

FORM 10-K Filing Information This section outlines Rocket Pharmaceuticals' 2022 Form 10-K filing details, including registrant information and incorporated documents [Registrant Information](index=1&type=section&id=Registrant%20Information) Details Rocket Pharmaceuticals' 2022 Form 10-K registrant information, including incorporation state and principal offices - Rocket Pharmaceuticals, Inc. is a Delaware corporation with its principal executive offices in Cranbury, NJ, and its common stock is registered on the NASDAQ Global Market under the symbol RCKT[2](index=2&type=chunk)[3](index=3&type=chunk) [Filer Status and Market Value](index=1&type=section&id=Filer%20Status%20and%20Market%20Value) Large accelerated filer with $644.3 million non-affiliate common stock market value as of June 30, 2022 - The registrant is a large accelerated filer and has filed all required reports during the preceding 12 months[4](index=4&type=chunk)[5](index=5&type=chunk) Filer Status and Market Value | Metric | Value | | :----- | :---- | | Aggregate Market Value of Common Stock (non-affiliates, as of June 30, 2022) | $644.3 million | | Common Stock Outstanding (as of February 22, 2023) | 79,347,760 shares | [Documents Incorporated by Reference](index=2&type=section&id=Documents%20Incorporated%20by%20Reference) Part III of this 10-K incorporates information from the 2023 Proxy Statement, to be filed within 120 days - Information for Part III of the 10-K is incorporated by reference from the 2023 Annual Meeting of Stockholders Proxy Statement, to be filed within 120 days[10](index=10&type=chunk) [Table of Contents](index=3&type=section&id=Table%20of%20Contents) Comprehensive listing of all chapters and items included in the Annual Report on Form 10-K [Forward-Looking Statements](index=4&type=section&id=FORWARD-LOOKING%20STATEMENTS) Identifies forward-looking statements subject to risks that could cause actual results to differ materially - The report contains forward-looking statements, identifiable by words like 'anticipate,' 'expect,' 'will,' and 'may,' which are subject to risks and uncertainties[14](index=14&type=chunk) - Key factors that could cause actual results to differ include clinical study timing and results, regulatory approvals, competitor activities, funding availability, manufacturing capabilities, and global economic/political developments[16](index=16&type=chunk)[17](index=17&type=chunk) [Summary of Material Business Risks](index=6&type=section&id=SUMMARY%20OF%20THE%20MATERIAL%20RISKS%20ASSOCIATED%20WITH%20OUR%20BUSINESS) Summarizes key business risks: COVID-19 impacts, commercialization, funding, clinical trials, manufacturing, reimbursement, and IP - The business faces numerous risks, including potential adverse impacts from COVID-19 on preclinical and clinical studies[19](index=19&type=chunk) - Key risks include inability to establish sales/marketing, failure to obtain additional funding, lack of product sales revenue, delays in clinical trials, and challenges in demonstrating safety and efficacy[19](index=19&type=chunk) - Other significant risks involve production problems, limited manufacturing experience, regulatory compliance, reimbursement availability, pipeline expansion, reliance on third parties, loss of key personnel, and intellectual property protection[19](index=19&type=chunk) PART I Detailed overview of the company's business, risk factors, properties, and legal proceedings [Item 1. Business](index=8&type=section&id=Item%201.%20Business) Outlines Rocket Pharmaceuticals' business: gene therapy platforms, product pipeline, manufacturing, IP, and regulatory environment [Overview](index=8&type=section&id=Overview) Clinical-stage biotech developing gene therapies for rare diseases using LV and AAV platforms, with multiple clinical programs - Rocket Pharmaceuticals is a clinical-stage, multi-platform biotechnology company focused on first, only and best-in-class gene therapies for rare and devastating diseases[23](index=23&type=chunk) - The company has three clinical-stage ex vivo lentiviral vector (LV) programs for Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD)[23](index=23&type=chunk) - Clinical-stage in vivo adeno-associated virus (AAV) programs include Danon disease, Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), and BAG3 Dilated Cardiomyopathy (DCM) (acquired via Renovacor)[23](index=23&type=chunk) [Gene Therapy Overview](index=8&type=section&id=Gene%20Therapy%20Overview) Gene therapy treats genetic diseases by delivering functional gene copies using viral vectors (LV, AAV) for potential cures - Gene therapy addresses disease-causing effects of absent or dysfunctional genes by delivering functional copies directly into patient cells, aiming for a cure[26](index=26&type=chunk) - Rocket Pharmaceuticals utilizes modified non-pathogenic viruses, called viral vectors (LV and AAV), to deliver therapeutic genes either ex vivo (outside the body) or in vivo (inside the body)[27](index=27&type=chunk)[28](index=28&type=chunk) - Recent FDA approvals for gene therapies indicate a promising regulatory pathway for these products[30](index=30&type=chunk) [Essential Terminology](index=10&type=section&id=Essential%20Terminology) Defines key terminology for LV and AAV gene therapies, including CD34+ cells and Vector Copy Number, with optimal ranges Gene Therapy Terminology and Optimal Ranges | Term | Definition | Optimal Ranges | | :--- | :--- | :--- | | CD34+ cell(s) (LV Therapy) | Hematopoietic Stem Cell (clinically useful measure) | > 1 million CD34+ cells/kg | | Vector copy number (VCN) [product] (LV Therapy) | Average gene copies per infused stem cell | 0.5 to 2 (target in some studies, 5.0 generally maximum) | | Vector copy number (VCN) [in vivo, post-treatment] (LV Therapy) | Average gene copies per peripheral blood or bone marrow cell | Many disorders correctable with VCNs << 1.0 | | Vector copy number (VCN) [in vivo, post-treatment] (AAV Therapy) | Average gene copies per cell in the organ of interest | Vivo VCNs << 1.0 | [Pipeline Overview](index=10&type=section&id=Pipeline%20Overview) Pipeline features LV and AAV gene therapy programs for cardiovascular and hematological rare diseases, advancing towards regulatory filings [Cardiovascular Programs](index=10&type=section&id=Cardiovascular%20Programs) Develops AAV gene therapies for severe cardiac conditions: Danon Disease, PKP2-ACM, and BAG3 DCM, with ongoing clinical and preclinical progress [Danon Disease (RP-A501)](index=10&type=section&id=Danon%20Disease) RP-A501, an in vivo AAV therapy for Danon Disease, showed promising Phase 1 safety/efficacy, RMAT designation, pivotal study Q2 2023 - RP-A501 is an in vivo AAV therapy for Danon Disease, a multi-organ lysosomal-associated disorder with an estimated prevalence of **15,000-30,000 patients** in the U.S. and EU[33](index=33&type=chunk)[34](index=34&type=chunk) - Phase 1 trial (n=7) showed RP-A501 was generally well-tolerated at the low dose (6.7e13 gc/kg), with no drug product-related SAEs in the low-dose adult/older adolescent cohort[36](index=36&type=chunk)[39](index=39&type=chunk) - Efficacy assessments demonstrated sustained cardiac LAMP2B expression, reduced autophagic vacuoles, stabilized/decreased cardiac wall thickness, improved/stabilized ejection fraction, and improvements in BNP, NYHA class, and KCCQ scores in compliant patients[40](index=40&type=chunk)[42](index=42&type=chunk)[43](index=43&type=chunk)[49](index=49&type=chunk) - The FDA granted RMAT designation for RP-A501, and a single-arm open-label pivotal study with a natural history comparator is anticipated to initiate in Q2 2023[44](index=44&type=chunk)[50](index=50&type=chunk) Phase 1 RP-A501 Study: Improvement or Stabilization Across Key Biomarker, Echo Findings and Functional Measures (Most Recent Visit) | Cohort | Patient ID | Most recent visit (months) | Δ hsTnI | Δ BNP | Δ LV mass | Δ LV max wall thickness | Δ NYHA class | Δ KCCQ score | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | Low dose pediatric | 1008 | 12 | ↓ 86% | ↓ 83% | ↓ 29% | ↓ 15% | II -> I | +32.3 | | Low dose pediatric | 1009 | 6 | ↓ 90% | ↓ 62% | ↓ 21% | ↑ 3% | II -> I | +26 | | Low dose adult/ adolescent | 1001 | 36 | ↓ 98% | ↓ 8% | ↓ 32% | ↓ 9% | II -> II | +5.3 | | Low dose adult/ adolescent | 1002 | 36 | ↓ 96% | ↓ 94% | ↓ 48% | ↓ 40% | II -> I | +17.8 | | Low dose adult/ adolescent | 1005 | 30 | ↓ 46% | ↑ 6% | ↓ 14% | ↓ 27% | II -> I | +8.3 | | High dose adult/ adolescent | 1006 | 24 | ↓ 63% | ↓ 69% | ↓ 27% | ↓ 15% | II -> I | +3.1 | [Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) (RP-A601)](index=16&type=section&id=Plakophilin-2%20Arrhythmogenic%20Cardiomyopathy%20%28PKP2-ACM%29) RP-A601, an AAVrh.74 gene therapy for PKP2-ACM, showed preclinical efficacy; IND filing anticipated Q2 2023 - PKP2-ACM is an inheritable cardiac disorder characterized by arrhythmias and sudden death, with an estimated prevalence of **50,000 patients** in the US and EU[51](index=51&type=chunk)[52](index=52&type=chunk) - RP-A601, an AAVrh.74 vector expressing PKP2a, aims to address the root cause of PKP2 deficiency[53](index=53&type=chunk) - Preclinical studies showed RP-A601 extended survival, reduced cardiac dilation, preserved LV function, and mitigated arrhythmic phenotype in animal models[54](index=54&type=chunk) - An Investigational New Drug (IND) application for RP-A601 is anticipated to be filed in Q2 2023[55](index=55&type=chunk) [BAG3 Dilated Cardiomyopathy (AAV9-BAG3)](index=16&type=section&id=BAG3%20Dilated%20Cardiomyopathy) Acquired Renovacor's AAV9-BAG3 gene therapy for BAG3 DCM, showing preclinical proof-of-concept; IND submission planned H1 2024 - BAG3 Dilated Cardiomyopathy (DCM) is a highly penetrant genetic variant causing early-onset, rapidly progressing heart failure, estimated to affect up to **30,000 individuals** in the United States[56](index=56&type=chunk) - Rocket acquired Renovacor's AAV9-based gene therapy program, designed to augment BAG3 protein levels in cardiomyocytes[59](index=59&type=chunk) - Initial proof-of-concept for AAV9-BAG3 was demonstrated in BAG3-knockout mouse models, showing improved ejection fraction[59](index=59&type=chunk) - An IND submission for BAG3-DCM is planned for the first half of 2024[60](index=60&type=chunk) [Hematology Programs](index=18&type=section&id=Hematology%20Programs) Develops ex vivo LV gene therapies for Fanconi Anemia, LAD-I, and PKD, with positive clinical data and anticipated BLA/pivotal trials in 2023 [Fanconi Anemia Complementation Group A (FANCA) (RP-L102)](index=18&type=section&id=Fanconi%20Anemia%20Complementation%20Group%20A%20%28FANCA%29) RP-L102, an ex vivo LV gene therapy for Fanconi Anemia, showed Phase 2 phenotypic correction; BLA filing anticipated Q4 2023 - FA is a rare, life-threatening DNA-repair disorder, with **60-70%** of cases from FANCA gene mutations, leading to bone marrow failure and malignancies. Prevalence is ~**4,000 patients** in U.S. and EU[61](index=61&type=chunk)[63](index=63&type=chunk) - RP-L102 is an ex vivo LV-based program in Phase 2 registrational-enabling clinical trials, administered without cytotoxic conditioning[64](index=64&type=chunk)[69](index=69&type=chunk) - Phase 2 data showed phenotypic correction in at least **six of ten** evaluable patients, with increased resistance to mitomycin-C (primary endpoint achieved) and concomitant genetic correction and hematologic stabilization[67](index=67&type=chunk)[69](index=69&type=chunk) - The safety profile of RP-L102 has been highly favorable, with no signs of bone marrow dysplasia, clonal dominance, or insertional mutagenesis[69](index=69&type=chunk) - Rocket has initiated FDA dialogue for BLA filing plans for RP-L102, anticipated in Q4 2023[70](index=70&type=chunk) [Leukocyte Adhesion Deficiency-I (LAD-I) (RP-L201)](index=20&type=section&id=Leukocyte%20Adhesion%20Deficiency-I%20%28LAD-I%29) RP-L201, an ex vivo LV gene therapy for severe LAD-I, showed sustained CD18 restoration and 100% survival; BLA filing anticipated Q2 2023 - LAD-I is a rare autosomal recessive disorder causing severe, recurrent, life-threatening infections and substantial infant mortality (**60-75%** by age two without HSCT)[71](index=71&type=chunk) - RP-L201 is an ex vivo LV-based program in a Phase 1/2 registration-enabling clinical trial, with **nine severe LAD-I patients** treated[72](index=72&type=chunk)[73](index=73&type=chunk) - All **nine patients** demonstrated sustained CD18 restoration (**20%-87%** expression) and **100%** overall survival at **12 months** post-infusion[75](index=75&type=chunk) - Patients showed statistically significant reductions in all-cause hospitalizations and severe infections, with resolution of LAD-I-related skin rash and restoration of wound repair capabilities[75](index=75&type=chunk)[76](index=76&type=chunk) - Rocket has initiated FDA discussions for BLA filing plans for RP-L201, anticipated in Q2 2023[77](index=77&type=chunk) [Pyruvate Kinase Deficiency (PKD) (RP-L301)](index=21&type=section&id=Pyruvate%20Kinase%20Deficiency%20%28PKD%29) RP-L301, an ex vivo LV gene therapy for PKD, showed robust efficacy in Phase 1; Phase 2 pivotal trial anticipated Q4 2023 - PKD is a rare autosomal recessive disorder causing chronic non-spherocytic hemolytic anemia, with an estimated **2,500 diagnosed cases** in the U.S. and EU[78](index=78&type=chunk) - RP-L301 is an ex vivo LV-based program in a global Phase 1 open-label, single-arm clinical trial, with adult and pediatric cohorts[79](index=79&type=chunk)[80](index=80&type=chunk) - Phase 1 data from **two adult patients** showed normalized hemoglobin levels (from **7.0-7.5 g/dL to 13.3-14.8 g/dL**), improved hemolysis, transfusion independence, and improved quality of life at **24 months** post-infusion[81](index=81&type=chunk)[83](index=83&type=chunk) - The safety profile of RP-L301 appears highly favorable, with no RP-L301-related serious adverse events and no evidence of insertional mutagenesis[83](index=83&type=chunk) - Enrollment in the Phase 1 study is complete, and initiation of the Phase 2 pivotal trial is anticipated in Q4 2023[84](index=84&type=chunk) [cGMP Manufacturing](index=22&type=section&id=cGMP%20Manufacturing) Operates a 103,720 sq ft cGMP manufacturing facility in Cranbury, NJ, for AAV drug product, with FDA agreement on CMC - Rocket Pharmaceuticals operates a **103,720 square foot** cGMP manufacturing facility in Cranbury, New Jersey, for AAV drug product[85](index=85&type=chunk) - The facility is scaled to manufacture AAV drug product for a planned Phase 2 pivotal study in Danon disease[85](index=85&type=chunk) - The company has an agreement with the FDA on chemistry, manufacturing, and controls requirements and potency assay plans for its in-house AAV cGMP manufacturing[85](index=85&type=chunk) [Strategy](index=22&type=section&id=Strategy) Aims to be a fully-integrated biotech, developing and commercializing first-in-class gene therapies for rare pediatric diseases - The company aims to become a fully-integrated biotechnology company, developing and commercializing potentially curative first-in-class gene therapies for rare pediatric diseases[86](index=86&type=chunk) - Near-term objectives include advancing product candidates, developing proprietary in-house analytics and manufacturing, and commencing registration trials[86](index=86&type=chunk) - Medium- to long-term goals involve submitting BLAs for clinical programs, establishing its gene therapy platform, and expanding the pipeline to additional indications[86](index=86&type=chunk) - The company believes its competitive advantage lies in its disease-based selection approach, providing a potential first-mover advantage[87](index=87&type=chunk) [Intellectual Property](index=22&type=section&id=Intellectual%20Property) Protects technology via patents, trade secrets, and in-licensing, with expirations from 2037-2041, leveraging regulatory protections - Rocket Pharmaceuticals protects its technology through patents, trade secrets, know-how, and in-licensing, focusing on gene expression vectors and methods for gene therapy[88](index=88&type=chunk)[90](index=90&type=chunk) - The company's patent portfolio includes both owned and in-licensed patent families for its product candidates[90](index=90&type=chunk) - Expected patent expiration dates for current programs range from **2037 to 2041**, absent extensions[91](index=91&type=chunk)[92](index=92&type=chunk)[93](index=93&type=chunk)[94](index=94&type=chunk) - The company also relies on regulatory protection through orphan drug designations, data exclusivity, market exclusivity, and patent term extensions[88](index=88&type=chunk)[96](index=96&type=chunk) [Material Contracts](index=24&type=section&id=Material%20Contracts) Holds key license agreements with CIEMAT, UCSD, and REGENXBIO for product candidates, involving upfront fees, milestones, and royalties - Rocket has exclusive worldwide license agreements with CIEMAT for lentiviral vectors for PKD, FA-A gene therapy, and LAD-I, involving upfront fees, milestone payments, and low to mid-single digit percentage royalties on net sales[98](index=98&type=chunk)[99](index=99&type=chunk)[101](index=101&type=chunk)[102](index=102&type=chunk)[104](index=104&type=chunk)[107](index=107&type=chunk) - A license agreement with UCSD grants exclusive worldwide rights for Danon disease treatment, requiring an upfront payment, clinical/commercial milestone payments (up to **$1.5 million**), and low single-digit percentage royalties[109](index=109&type=chunk) - An exclusive license with REGENXBIO Inc. for NAV AAV-9 vector for Danon disease involved a **$7.0 million** upfront payment, high-single digit to low-teens royalties on net sales, and up to **$13.0 million** in clinical/regulatory milestone payments per Licensed Product[111](index=111&type=chunk)[112](index=112&type=chunk) - The company also has an at-the-market offering program with Cowen, through which it sold **3.3 million shares** for net proceeds of **$46.6 million** in 2022[113](index=113&type=chunk) [Competition](index=27&type=section&id=Competition) Operates in a highly competitive biotech industry, differentiating with single-treatment curative therapies for high-mortality/morbidity - The biotechnology and pharmaceutical industries are characterized by rapidly advancing technologies and intense competition from larger companies, new entrants, and research institutions[114](index=114&type=chunk) - Key competitive factors for product candidates include efficacy, safety, convenience, price, pharmaco-economic value, tolerability, and reimbursement[114](index=114&type=chunk) - Rocket aims to differentiate with single-treatment curative therapies for clinical indications addressing mortality or high morbidity[114](index=114&type=chunk) [Manufacturing](index=27&type=section&id=Manufacturing) Gene therapy manufacturing involves in-house cGMP for AAV and third parties for LV, planning a hybrid approach for commercial production - Rocket's gene therapy platform involves LV and AAV vector production and target cell transduction[117](index=117&type=chunk) - GMP manufacturing for AAV drug product commenced at the Cranbury, NJ facility in 2022, supplemented by third-party manufacturers[117](index=117&type=chunk) - LV programs currently rely on third-party manufacturers for all components (plasmids, vectors, cell banks, final drug product)[117](index=117&type=chunk) - The company plans to use a combination of direct manufacturing and contract manufacturing organizations for future commercial production[117](index=117&type=chunk) [Government Regulation](index=27&type=section&id=Government%20Regulation) Gene therapy development is subject to extensive FDA and foreign regulatory oversight, including preclinical, clinical, and BLA requirements - FDA regulates drugs under FDCA and biologics (including gene therapies) under the Public Health Service Act, with CBER overseeing gene therapy products[119](index=119&type=chunk)[120](index=120&type=chunk)[121](index=121&type=chunk) - The approval process involves preclinical studies (GLP), IND submission, multi-phase clinical trials (GCP), BLA submission, manufacturing facility inspections (cGMP, cGTP), and FDA approval[122](index=122&type=chunk)[123](index=123&type=chunk)[124](index=124&type=chunk)[128](index=128&type=chunk)[135](index=135&type=chunk) - Gene therapy products are subject to specific guidance, including recommendations for long-term follow-up studies (**15 years** for lentiviral, **5 years** for AAV)[239](index=239&type=chunk) - Expedited programs like Fast Track, RMAT, Priority Review, and Accelerated Approval are available for serious or life-threatening conditions with unmet medical needs, potentially shortening review times but not altering approval standards[147](index=147&type=chunk)[148](index=148&type=chunk)[149](index=149&type=chunk)[150](index=150&type=chunk)[151](index=151&type=chunk) - Post-marketing requirements include ongoing monitoring, adverse event reporting, compliance with cGMP, and potential REMS, with non-compliance leading to significant sanctions[152](index=152&type=chunk)[154](index=154&type=chunk)[155](index=155&type=chunk) [Coverage and Reimbursement](index=35&type=section&id=Coverage%20and%20Reimbursement) Commercial success depends on adequate reimbursement from third-party payors, with foreign pricing often subject to government controls - Sales of approved products depend on reimbursement from third-party payors (government, managed care, private insurers)[156](index=156&type=chunk) - Payors determine coverage based on medical necessity, cost-effectiveness, and may limit products to formularies, potentially excluding FDA-approved drugs[157](index=157&type=chunk)[158](index=158&type=chunk) - Adequate reimbursement is essential for market acceptance and profitability, and the process is time-consuming and varies by payor[157](index=157&type=chunk) - Foreign countries, particularly in the EU, often have government price controls, leading to significantly lower prices than in the U.S[160](index=160&type=chunk) [Anti-Kickback and False Claims Laws and Other Regulatory Matters](index=36&type=section&id=Anti-Kickback%20and%20False%20Claims%20Laws%20and%20Other%20Regulatory%20Matters) Business activities are subject to federal and state healthcare fraud, abuse, and data privacy laws, with non-compliance risking severe penalties - Business activities are subject to federal and state anti-kickback, fraud and abuse, and data privacy laws (e.g., Federal Anti-Kickback Statute, False Claims Act, HIPAA)[161](index=161&type=chunk)[163](index=163&type=chunk)[164](index=164&type=chunk)[165](index=165&type=chunk)[167](index=167&type=chunk) - Violations can result in criminal sanctions, civil penalties, exclusion from federal healthcare programs, contractual damages, and reputational harm[163](index=163&type=chunk)[164](index=164&type=chunk)[169](index=169&type=chunk) - The intent standard for the Federal Anti-Kickback Statute was amended by the ACA to a stricter standard, no longer requiring actual knowledge or specific intent to violate[163](index=163&type=chunk) - Evolving data privacy laws (e.g., GDPR, UK GDPR, CCPA, VCDPA, CPA, CTDPA, UCPA) impose complex compliance burdens and potential liabilities[167](index=167&type=chunk)[280](index=280&type=chunk)[281](index=281&type=chunk)[283](index=283&type=chunk)[284](index=284&type=chunk) [Healthcare Legislative Reform](index=39&type=section&id=Healthcare%20Legislative%20Reform) U.S. healthcare reforms like ACA and IRA aim to control costs and drug pricing, potentially reducing revenues and impacting profitability - The ACA subjected biologic products to biosimilar competition, increased Medicaid rebates, and created Medicare Part D coverage gap discounts[172](index=172&type=chunk) - The Inflation Reduction Act (IRA) allows government negotiation of prices for select high-cost Medicare Part D and B drugs, and requires manufacturers to pay rebates if prices increase faster than inflation[175](index=175&type=chunk) - These reforms are expected to result in more rigorous coverage criteria and downward pressure on drug prices, potentially harming future revenues and profitability[177](index=177&type=chunk)[178](index=178&type=chunk) - State-level legislation also increasingly targets pharmaceutical pricing, including price controls, discounts, and transparency measures[176](index=176&type=chunk) [European Union Drug Review and Approval](index=40&type=section&id=European%20Union%20Drug%20Review%20and%20Approval) EU drug approval involves national/centralized procedures for ATMPs, offering data/market exclusivity, and accelerated assessment via PRIME - EU clinical trial approval requires national competent authority and ethics committee approval, now streamlined by the Clinical Trials Regulation (EU) No 536/2014[179](index=179&type=chunk)[180](index=180&type=chunk) - Marketing authorization can be centralized (mandatory for ATMPs like gene therapies, evaluated by CAT and CHMP) or national (Decentralized/Mutual Recognition Procedures)[181](index=181&type=chunk)[182](index=182&type=chunk)[183](index=183&type=chunk) - Innovative products receive **8 years** data exclusivity and **2 years** market exclusivity (extendable to **11 years**). Orphan medicinal products get **10 years** market exclusivity, which can be reduced to **6 years** or revoked under certain conditions[186](index=186&type=chunk)[187](index=187&type=chunk)[188](index=188&type=chunk) - PRIME designation facilitates accelerated assessment and early regulatory dialogue for products addressing unmet medical needs[189](index=189&type=chunk) [Brexit and the Regulatory Framework in the United Kingdom](index=44&type=section&id=Brexit%20and%20the%20Regulatory%20Framework%20in%20the%20United%20Kingdom) Brexit created separate UK and EU regulatory regimes, increasing complexity and potential divergence, disrupting clinical trials and business - Brexit resulted in separate UK and EU regulatory regimes for pharmaceuticals, potentially increasing regulatory complexity[190](index=190&type=chunk) - Great Britain's regulatory regime currently aligns with EU regulations but may diverge in the future, impacting clinical trials and approvals[190](index=190&type=chunk) - The cumulative effects of regulatory disruption could add to development lead times and create legal uncertainty[277](index=277&type=chunk) [Human Capital](index=44&type=section&id=Human%20Capital) As of December 31, 2022, Rocket Pharmaceuticals had 240 global full-time employees, focusing on talent attraction, retention, and inclusive culture Human Capital Statistics (as of December 31, 2022) | Metric | Value | | :----- | :---- | | Full-time Employees | 240 | | Employees in United States | 230 | | Employees in Spain | 5 | | Employees in Switzerland | 2 | | Employees in UK, Sweden, Portugal | 1 each | | Labor Union Representation | None | - Objectives include identifying, attracting, recruiting, retaining, incentivizing, developing, and integrating employees, advisors, and consultants[192](index=192&type=chunk) - The company is investing in a diverse, equitable, and inclusive culture, with all employees upholding Rocket Behaviors and Code of Conduct[193](index=193&type=chunk) [Corporate Information](index=44&type=section&id=Corporate%20Information) Rocket Pharmaceuticals, Inc., incorporated in Delaware in 1999 and listed on NASDAQ, uses its website for SEC filings - Rocket Pharmaceuticals, Inc. was incorporated in Delaware in 1999 as Inotek Pharmaceuticals Corporation and changed its name in January 2018 after a merger[194](index=194&type=chunk) - The company's common stock is listed on the NASDAQ Global Market under the symbol 'RCKT'[194](index=194&type=chunk) - The company uses its website (www.rocketpharma.com) for disclosing material non-public information and SEC filings[194](index=194&type=chunk) [Item 1A. Risk Factors](index=44&type=section&id=Item%201A.%20Risk%20Factors) Details significant risks impacting Rocket Pharmaceuticals' business: pandemics, financial stability, clinical development, regulatory compliance, and market [Risks Related to COVID-19 Pandemic](index=44&type=section&id=Risks%20Related%20to%20COVID-19%20Pandemic) COVID-19 pandemic may adversely impact business, causing disruptions in preclinical/clinical studies, manufacturing, and regulatory timelines - COVID-19 and similar pandemics can adversely impact preclinical and clinical studies, causing disruptions[197](index=197&type=chunk)[199](index=199&type=chunk) - Potential disruptions include delays in patient enrollment, clinical site initiation, diversion of healthcare resources, interruption of trial activities, manufacturing delays, and impacts on FDA/regulatory agency operations[199](index=199&type=chunk) [Risks Related to Our Financial Condition and Capital Needs](index=46&type=section&id=Risks%20Related%20to%20Our%20Financial%20Condition%20and%20Capital%20Needs) History of operating losses and requires substantial additional funding; future profitability uncertain, with potential adverse tax law impacts - The company has incurred net losses since inception (**$221.9 million** in 2022, **$169.1 million** in 2021, **$139.7 million** in 2020) and has an accumulated deficit of **$713.8 million** as of December 31, 2022[202](index=202&type=chunk) - No revenue has been generated from product sales, and profitability is not anticipated in the foreseeable future[201](index=201&type=chunk)[216](index=216&type=chunk) - Substantial additional funding will be required to expand gene therapy platforms, advance product candidates, and potentially commercialize them[214](index=214&type=chunk) - Failure to obtain necessary capital could force delays, reductions, or elimination of product development or commercialization efforts[213](index=213&type=chunk)[214](index=214&type=chunk) - Changes in tax law and limitations on the ability to use net operating losses (NOLs) and research and development credits could adversely affect the business[210](index=210&type=chunk)[211](index=211&type=chunk)[212](index=212&type=chunk) [Risks Related to Clinical Development and Product Regulatory Matters](index=51&type=section&id=Risks%20Related%20to%20Clinical%20Development%20and%20Product%20Regulatory%20Matters) Clinical trials are expensive and uncertain, with risks of delays, failures, unforeseen side effects, and unpredictable regulatory approval - Clinical trials are expensive, time-consuming, and uncertain, with risks of delays or failures in demonstrating safety and efficacy[222](index=222&type=chunk)[223](index=223&type=chunk) - Novel gene therapy technology makes predicting development time and cost, and obtaining regulatory approval, difficult and potentially longer/more expensive[237](index=237&type=chunk)[238](index=238&type=chunk) - Product candidates may cause undesirable or unforeseen side effects, potentially delaying or preventing clinical advancement or regulatory approval[231](index=231&type=chunk)[233](index=233&type=chunk)[234](index=234&type=chunk) - Orphan drug designation, Fast Track, RMAT, or PRIME designations do not guarantee faster development, review, or approval, nor do they increase the likelihood of marketing approval[244](index=244&type=chunk)[249](index=249&type=chunk)[250](index=250&type=chunk)[251](index=251&type=chunk)[252](index=252&type=chunk) - Even if approved, products remain subject to ongoing regulatory obligations, scrutiny, and potential restrictions, including long-term follow-up studies[262](index=262&type=chunk) - Biosimilar competition and healthcare legislative reforms (e.g., ACA, IRA) could limit market potential and reduce reimbursement for approved products[269](index=269&type=chunk)[272](index=272&type=chunk)[273](index=273&type=chunk) [Risks Related to Noncompliance with Applicable Laws or Regulations](index=64&type=section&id=Risks%20Related%20to%20Noncompliance%20with%20Applicable%20Laws%20or%20Regulations) Business activities are subject to federal/state healthcare fraud, abuse, and data privacy laws, with non-compliance risking severe penalties - Relationships with customers and third-party payors are subject to anti-kickback, fraud and abuse, and other healthcare laws, with non-compliance risking criminal sanctions, civil penalties, and exclusion from government programs[279](index=279&type=chunk) - Failure to comply with U.S. and foreign privacy and data protection laws (e.g., GDPR, UK GDPR, CCPA) can lead to significant liabilities, fines (up to **20 million euros** or **4% of global revenue** for GDPR), and adverse impacts on business[280](index=280&type=chunk)[281](index=281&type=chunk)[283](index=283&type=chunk) - The global legislative and regulatory landscape for privacy and data protection is evolving, creating uncertainty and increasing compliance costs[288](index=288&type=chunk) - The company is also subject to environmental, health, and safety laws, with potential for liability and substantial expenses for non-compliance or remediation[292](index=292&type=chunk)[293](index=293&type=chunk) [Risks Related to Manufacturing, Commercialization and Development of Our Product Candidates](index=67&type=section&id=Risks%20Related%20to%20Manufacturing%2C%20Commercialization%20and%20Development%20of%20Our%20Product%20Candidates) Manufacturing gene therapies is complex, risking production problems and supply limitations; commercialization success depends on market acceptance and reimbursement - Gene therapy products are complex to manufacture, risking production problems, delays, or supply limitations due to equipment malfunctions, contamination, or raw material shortages[294](index=294&type=chunk)[295](index=295&type=chunk) - The company has limited manufacturing experience and relies on third parties for LV programs, with risks of non-compliance with cGMPs or termination of agreements[295](index=295&type=chunk)[297](index=297&type=chunk)[298](index=298&type=chunk)[299](index=299&type=chunk) - Commercial success depends on market acceptance by physicians, patients, and payors, influenced by efficacy, safety, cost, and reimbursement availability[300](index=300&type=chunk)[302](index=302&type=chunk)[310](index=310&type=chunk) - The success of R&D activities is uncertain, with potential for cost overruns, regulatory delays, unexpected side effects, or insufficient efficacy[315](index=315&type=chunk) - Failure to expand the pipeline of additional product candidates or secure strategic collaborations could significantly harm financial position and results of operations[312](index=312&type=chunk)[314](index=314&type=chunk)[327](index=327&type=chunk)[328](index=328&type=chunk) [Risks Related to Third Parties](index=74&type=section&id=Risks%20Related%20to%20Third%20Parties) Heavily relies on third parties for preclinical, clinical, and manufacturing, risking non-performance, delays, or regulatory disruptions - Reliance on third parties (CROs, medical institutions, contract laboratories) for preclinical and clinical programs carries risks of non-compliance, delays, or termination of studies[317](index=317&type=chunk)[318](index=318&type=chunk)[319](index=319&type=chunk)[320](index=320&type=chunk)[321](index=321&type=chunk) - Third-party manufacturers for gene therapy production (especially LV product candidates) introduce risks like inability to negotiate agreements, reduced control, and supply disruptions[325](index=325&type=chunk) - Disruptions at the FDA and other government agencies (e.g., due to funding shortages, pandemics) could hinder their ability to review and approve products, causing delays[329](index=329&type=chunk)[330](index=330&type=chunk) [Risks Related to Our Intellectual Property](index=76&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) Success depends on robust patent protection and trade secrets, with reliance on third-party licenses, risking invalidation or infringement - Reliance on third-party licenses for patent rights and proprietary technology is critical, with risks of non-exclusive rights, unavailability of future licenses, or loss of rights if licensors fail to maintain patents[332](index=332&type=chunk)[333](index=333&type=chunk)[334](index=334&type=chunk) - Failure to obtain and maintain broad patent protection, or if patents are found invalid/unenforceable, could allow competitors to commercialize similar products[336](index=336&type=chunk)[340](index=340&type=chunk)[342](index=342&type=chunk)[355](index=355&type=chunk) - Breach of license agreements could result in loss of license rights essential for commercialization efforts[344](index=344&type=chunk) - Inability to protect trade secrets or obtain necessary rights to gene therapy components through acquisitions/in-licenses could harm competitive position and business growth[348](index=348&type=chunk)[350](index=350&type=chunk) - Changes in U.S. or foreign patent law, challenges to inventorship/ownership, or third-party claims of infringement could prevent or delay development and commercialization[356](index=356&type=chunk)[364](index=364&type=chunk)[365](index=365&type=chunk) [Risks Related to Personnel and Expansion of our Company](index=85&type=section&id=Risks%20Related%20to%20Personnel%20and%20Expansion%20of%20our%20Company) Depends on senior management and qualified personnel, facing risks from misconduct, expansion, acquisition integration, and international operations - The company is highly dependent on senior management and its ability to attract and retain qualified scientific and technical personnel[369](index=369&type=chunk) - Risks include employee/partner misconduct (e.g., non-compliance with regulations, fraud, insider trading) and claims of wrongful use/disclosure of third-party confidential information[370](index=370&type=chunk)[371](index=371&type=chunk) - Organizational expansion may lead to management difficulties, operational setbacks, loss of business opportunities, and increased capital expenditures[373](index=373&type=chunk) - Potential acquisitions (e.g., Renovacor) carry integration risks, such as loss of key employees, business disruption, and failure to realize anticipated benefits[374](index=374&type=chunk) - Strategic alliances or joint ventures could disrupt business, lead to conflicts with collaborators, or divert management time and focus[376](index=376&type=chunk)[378](index=378&type=chunk)[383](index=383&type=chunk) - International operations expose the company to risks like different regulatory requirements, reduced IP protection, economic/political instability, and foreign currency fluctuations[381](index=381&type=chunk)[382](index=382&type=chunk) [Risks Related to Ownership of our Common Stock](index=88&type=section&id=Risks%20Related%20to%20Ownership%20of%20our%20Common%20Stock) Common stock price is volatile; future sales may cause dilution; capital appreciation is sole gain source as no cash dividends are anticipated - The market price of common stock is volatile and can fluctuate due to clinical trial results, competition, regulatory developments, and macroeconomic conditions[390](index=390&type=chunk) - Future sales of common stock, including by RTW Investments, LP (**25.2% beneficial ownership**), or through equity/convertible debt issuances, could cause significant dilution and stock price decline[385](index=385&type=chunk)[393](index=393&type=chunk) - The company does not anticipate paying cash dividends, so capital appreciation is the sole source of gain for stockholders[394](index=394&type=chunk)[395](index=395&type=chunk) - Provisions in corporate charter documents and Delaware law could make an acquisition of the company more difficult[399](index=399&type=chunk) [Other Risks Related to Our Business](index=91&type=section&id=Other%20Risks%20Related%20to%20Our%20Business) Faces risks from internal control over financial reporting, computer system failures, social media challenges, and unfavorable economic/political developments - Failure to maintain proper and effective internal control over financial reporting could impair financial statement accuracy, harm operating results, and negatively impact investor confidence[396](index=396&type=chunk)[398](index=398&type=chunk) - Internal computer systems or those of third-party collaborators are vulnerable to failures or security breaches, risking disruption of development programs and loss of proprietary information[401](index=401&type=chunk)[402](index=402&type=chunk) - The increasing use of social media platforms presents risks related to regulatory compliance (e.g., adverse event reporting) and managing negative or inaccurate public information[403](index=403&type=chunk) - Unfavorable national or global economic conditions or political developments (e.g., inflation, global conflicts) could adversely affect business, financial condition, or results of operations[404](index=404&type=chunk) [Item 1B. Unresolved SEC Comments](index=93&type=section&id=Item%201B.%20Unresolved%20SEC%20Comments) No unresolved comments from the SEC as of the filing date of this report - No unresolved SEC comments[405](index=405&type=chunk) [Item 2. Properties](index=93&type=section&id=Item%202.%20Properties) Primary facilities include Cranbury, NJ headquarters/manufacturing, a New York office, and assumed Renovacor leases - Corporate headquarters, R&D, and cGMP manufacturing facility in Cranbury, NJ: **103,720 sq ft**, lease until **2034**[406](index=406&type=chunk) - Office space in New York, NY: **6,600 sq ft**, lease until July 2024[406](index=406&type=chunk) - Additional storage facility in Dayton, NJ: **4,666 sq ft**[406](index=406&type=chunk) - Assumed leases from Renovacor acquisition for facilities in Hopewell, NJ (**15,463 sq ft**, lease until March 2033) and Cambridge, MA (**5,945 sq ft**, sublease until April 2024), with intent to sublease[408](index=408&type=chunk)[409](index=409&type=chunk) [Item 3. Legal Proceedings](index=94&type=section&id=Item%203.%20Legal%20Proceedings) Not party to any legal proceedings expected to have a material adverse effect, though litigation can incur significant costs - The company is not currently involved in any legal proceedings expected to have a material adverse effect on its business[410](index=410&type=chunk) - Litigation can incur substantial defense and settlement costs and divert management resources[410](index=410&type=chunk) [Item 4. Mine Safety Disclosures](index=94&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) Mine Safety Disclosures are not applicable to the company - Mine Safety Disclosures are not applicable to the company[411](index=411&type=chunk) PART II Covers market information for common equity, management's discussion and analysis, and controls and procedures [Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=94&type=section&id=Item%205.%20Market%20for%20Registrant%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) Provides information on Rocket Pharmaceuticals' common stock market, including trading symbol, stockholder count, and dividend policy - Common stock is traded on the NASDAQ Global Market under the symbol 'RCKT'[412](index=412&type=chunk) Common Stock Information | Metric | Value | | :----- | :---- | | Last Reported Sale Price (Feb 22, 2023) | $19.20 per share | | Stockholders of Record (Feb 22, 2023) | 45 | - The company has never declared or paid cash dividends and intends to retain all future earnings for business growth, making capital appreciation the sole source of gain for stockholders[417](index=417&type=chunk) - No repurchases of common stock were made during the year ended December 31, 2022[419](index=419&type=chunk) [Item 6. Reserved](index=95&type=section&id=Item%206.%20Reserved) This item is reserved and contains no information [Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=95&type=section&id=Item%207.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management's perspective on financial condition and results of operations, covering business overview, financial developments, and liquidity [Introduction](index=95&type=section&id=Introduction) Clinical-stage biotech focused on developing gene therapies for rare diseases using LV and AAV platforms, with a pipeline of clinical programs - Rocket Pharmaceuticals is a clinical-stage, multi-platform biotechnology company developing gene therapies for rare and devastating diseases[423](index=423&type=chunk) - The pipeline includes LV programs for FA, LAD-I, PKD, and AAV programs for Danon disease, PKP2-ACM, and BAG3 DCM[423](index=423&type=chunk) - The RP-L401 program was discontinued in December 2021 to focus resources on other advanced candidates[425](index=425&type=chunk) [Recent Developments](index=97&type=section&id=Recent%20Developments) In 2022, Rocket Pharmaceuticals engaged in significant financial activities: an at-the-market offering, Renovacor acquisition, and public offering - Through December 31, 2022, the company sold **3.3 million shares** via an at-the-market offering program, generating **$46.6 million** in net proceeds[426](index=426&type=chunk) - Acquired Renovacor, Inc. on December 1, 2022, issuing **3,391,976 shares** of common stock as part of the **$72.3 million** total consideration[427](index=427&type=chunk)[672](index=672&type=chunk)[673](index=673&type=chunk) - Completed a public offering on October 6, 2022, selling **7,820,000 shares** for **$108.1 million** in net proceeds[428](index=428&type=chunk) [Financial Overview](index=97&type=section&id=Financial%20Overview) No product sales revenue generated or anticipated; operations funded primarily through equity and convertible debt - No revenue from product sales has been generated to date, and none is expected in the near future[430](index=430&type=chunk) - From inception through December 31, 2022, the company raised approximately **$835.6 million** in net cash proceeds from equity and convertible debt financing[429](index=429&type=chunk) - R&D expenses primarily consist of external costs for preclinical, clinical, and manufacturing activities, as well as internal personnel and facility costs[431](index=431&type=chunk)[434](index=434&type=chunk) - R&D expenses are expected to increase as programs advance and additional clinical trials are conducted[439](index=439&type=chunk)[440](index=440&type=chunk) - General and administrative expenses are expected to increase due to anticipated headcount growth and public company operating costs[442](index=442&type=chunk) [Results of Operations](index=101&type=section&id=Results%20of%20Operations) Increased net losses in 2022, driven by higher R&D and G&A expenses, with improved other income due to decreased interest expense Consolidated Statements of Operations Summary (in thousands) | Metric | 2022 | 2021 | 2020 | | :--- | :--- | :--- | :--- | | Research and development expenses | $165,570 | $125,476 | $105,438 | | General and administrative expenses | $58,773 | $41,772 | $28,865 | | Total operating expenses | $224,343 | $167,248 | $134,303 | | Net loss | $(221,863) | $(169,069) | $(139,700) | - R&D expenses increased by **$40.1 million** in 2022 (to **$165.6 million**) primarily due to higher manufacturing and development costs (**$26.3 million**), laboratory supplies (**$6.6 million**), and compensation/benefits (**$11.5 million**), partially offset by decreased license fees[447](index=447&type=chunk) - G&A expenses increased by **$17.0 million** in 2022 (to **$58.8 million**) mainly due to commercial preparation expenses (**$4.9 million**), increased headcount compensation (**$4.4 million**), and acquisition-related expenses (**$3.2 million**)[448](index=448&type=chunk) - Other income (expense), net, increased by **$4.3 million** in 2022 (to **$2.5 million**) due to decreased interest expense from convertible notes and increased interest income[449](index=449&type=chunk) [Liquidity and Capital Resources](index=103&type=section&id=Liquidity%20and%20Capital%20Resources) History of net losses and negative cash flows; $399.7 million in cash and investments expected to fund operations through 2024 - The company has incurred net losses and negative cash flows from operations since inception, with an accumulated deficit of **$713.8 million** as of December 31, 2022[455](index=455&type=chunk)[457](index=457&type=chunk) Cash, Cash Equivalents and Investments (as of December 31) | Year | Amount (in millions) | | :--- | :------------------- | | 2022 | $399.7 | | 2021 | $388.7 | | 2020 | $302.0 | - Current resources are expected to fund operating expenses and capital expenditure requirements through 2024[457](index=457&type=chunk) - Future viability depends on generating cash from operations or raising additional capital, with risks of stockholder dilution or restrictive debt covenants[459](index=459&type=chunk) Net Cash Flows (in thousands) | Activity | 2022 | 2021 | 2020 | | :--- | :--- | :--- | :--- | | Operating activities | $(178,142) | $(121,163) | $(74,640) | | Investing activities | $(69,326) | $18,853 | $(96,591) | | Financing activities | $155,288 | $37,681 | $282,989 | | Net change in cash, cash equivalents and restricted cash | $(92,180) | $(64,629) | $111,758 | [Critical Accounting Policies and Estimates](index=106&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) Financial statements rely on significant estimates for R&D expenses, stock-based compensation, goodwill, and intangible assets - Critical accounting policies include Accrued Research and Development Expenses, Stock-Based Compensation, Goodwill, and Intangible Assets[474](index=474&type=chunk) - Goodwill is tested for impairment annually or more frequently if circumstances indicate impairment, using a qualitative assessment[475](index=475&type=chunk) - Intangible assets (IPR&D) are indefinite-lived until development completion/abandonment and are tested for impairment annually[478](index=478&type=chunk) - Accrued R&D expenses are estimated based on contractual payment schedules, invoices, and assessment of services performed by third parties (CROs, CMOs, investigative sites)[479](index=479&type=chunk) - Stock-based compensation is recognized based on fair value using the Black-Scholes option pricing model, expensed over the service period[480](index=480&type=chunk) [Recent Accounting Pronouncements](index=109&type=section&id=Recent%20Accounting%20Pronouncements) No recent accounting pronouncements significantly impacted or are expected to affect consolidated financial statements - No recent accounting pronouncements had a significant effect on the consolidated financial statements[482](index=482&type=chunk) [Item 7A. Quantitative and Qualitative Disclosures About Market Risk](index=109&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) Quantitative and Qualitative Disclosures About Market Risk are not applicable - Quantitative and Qualitative Disclosures About Market Risk are not applicable[483](index=483&type=chunk) [Item 8. Financial Statements and Supplementary Data](index=109&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) Financial statements required by this item are included in Item 15 of this Annual Report - Financial statements are included in Item 15 of the Annual Report[483](index=483&type=chunk) [Item 9. Changes in and Disagreements with Accountants on Accounting and Financial Disclosure](index=109&type=section&id=Item%209.%20Changes%20in%20and%20Disagreements%20with%20Accountants%20on%20Accounting%20and%20Financial%20Disclosure) No changes in or disagreements with accountants on accounting and financial disclosure - No changes in or disagreements with accountants on accounting and financial disclosure[484](index=484&type=chunk) [Item 9A. Controls and Procedures](index=109&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded disclosure controls and internal control over financial reporting were effective as of December 31, 2022 - Disclosure controls and procedures were effective at a reasonable assurance level as of December 31, 2022[485](index=485&type=chunk) - Management maintained effective internal control over financial reporting as of December 31, 2022, based on the COSO 2013 framework[487](index=487&type=chunk) - The effectiveness of internal control over financial reporting was audited by EisnerAmper LLP, who expressed an unqualified opinion[488](index=488&type=chunk) - Internal controls have inherent limitations and may not prevent or detect all misstatements[489](index=489&type=chunk) - No material changes in internal control over financial reporting occurred during the period[490](index=490&type=chunk) [Item 9B. Other Information](index=110&type=section&id=Item%209B.%20Other%20Information) No other information is reported under this item - No other information is reported under this item[491](index=491&type=chunk) [Item 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections](index=110&type=section&id=Item%209C.%20Disclosure%20Regarding%20Foreign%20Jurisdictions%20that%20Prevent%20Inspections) Disclosure Regarding Foreign Jurisdictions that Prevent Inspections is not applicable - Disclosure Regarding Foreign Jurisdictions that Prevent Inspections is not applicable[492](index=492&type=chunk) PART III Incorporates information on directors, executive compensation, security ownership, related transactions, and accountant fees by reference [Item 10. Directors, Executive Officers, and Corporate Governance](index=110&type=section&id=Item%2010.%20Directors%2C%20Executive%20Officers%2C%20and%20Corporate%20Governance) Information on directors, executive officers, and corporate governance is incorporated by reference from the 2023 Proxy Statement - Information for this item is incorporated by reference from the 2023 Proxy Statement[494](index=494&type=chunk) [Item 11. Executive Compensation](index=110&type=section&id=Item%2011.%20Executive%20Compensation) Executive compensation information is incorporated by reference from the company's 2023 Proxy Statement - Information for this item is incorporated by reference from the 2023 Proxy Statement[495](index=495&type=chunk) [Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=110&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) Security ownership information for beneficial owners and management is incorporated by reference from the 2023 Proxy Statement - Information for this item is incorporated by reference from the 2023 Proxy Statement[496](index=496&type=chunk) [Item 13. Certain Relationships and Related Transactions, and Director Independence](index=110&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions%2C%20and%20Director%20Independence) Information on certain relationships, related party transactions, and director independence is incorporated by reference from the 2023 Proxy Statement - Information for this item is incorporated by reference from the 2023 Proxy Statement[497](index=497&type=chunk) [Item 14. Principal Accountant Fees and Services](index=110&type=section&id=Item%2014.%20Principal%20Accountant%20Fees%20and%20Services) Principal accountant fees and services information is incorporated by reference from the 2023 Proxy Statement; EisnerAmper LLP is the independent firm - EisnerAmper LLP is the independent public accounting firm[498](index=498&type=chunk) - Information for this item is incorporated by reference from the 2023 Proxy Statement[498](index=498&type=chunk) PART IV Details exhibits, financial statements, schedules, and the Form 10-K summary [Item 15. Exhibits, Financial Statements and Schedules](index=111&type=section&id=Item%2015.%20Exhibits%2C%20Financial%20Statements%20and%20Schedules) Lists all financial statements, schedules, and exhibits filed as part of the Annual Report on Form 10-K, including consolidated financial statements - The section includes consolidated financial statements: Balance Sheets, Statements of Operations, Comprehensive Loss, Changes in Stockholders' Equity, and Cash Flows, along with their notes[500](index=500&type=chunk) - All financial statement schedules have been omitted as not applicable or information is included in financial statements/notes[499](index=499&type=chunk) - A comprehensive list of exhibits, including merger agreements, corporate governance documents, stock plans, license agreements, and certifications, is provided[501](index=501&type=chunk)[502](index=502&type=chunk)[503](index=503&type=chunk) [Item 16. Form 10-K Summary](index=114&type=section&id=Item%2016.%20Form%2010-K%20Summary) Form 10-K Summary is not applicable to the company - Form 10-K Summary is not applicable[506](index=506&type=chunk) [Signatures](index=115&type=section&id=SIGNATURES) Contains the required signatures for the Annual Report on Form 10-K [Index to Consolidated Financial Statements](index=116&type=section&id=Index%20to%20Consolidated%20Financial%20Statements) Provides an index to the consolidated financial statements included in the Annual Report [Report of Independent Registered Public Accounting Firm](index=117&type=section&id=REPORT%20OF%20INDEPENDENT%20REGISTERED%20PUBLIC%20ACCOUNTING%20FIRM) Presents the independent auditor's report on the company's financial statements and critical audit matters [Opinion on the Financial Statements](index=117&type=section&id=Opinion%20on%20the%20Financial%20Statements) EisnerAmper LLP issued an unqualified opinion on Rocket Pharmaceuticals' consolidated financial statements for 2020-2022 - EisnerAmper LLP issued an unqualified opinion on the consolidated financial statements for 2020, 2021, and 2022[517](index=517&type=chunk) - The financial statements present fairly, in all material respects, the financial position and results of operations in conformity with U.S. GAAP[517](index=517&type=chunk) [Critical Audit Matters](index=117&type=section&id=Critical%20Audit%20Matters) Accruals for R&D expenses were a critical audit matter due to complex cost estimation for third-party contracts where invoicing may not align - Accruals for research and development expenses were identified as a critical audit matter[522](index=522&type=chunk) - The complexity arises from estimating progress and costs incurred under third-party contracts (CROs, CMOs, investigative sites) where invoicing may not match service timing[523](index=523&type=chunk) - Audit procedures included inspecting contracts, invoices, payments, confirming amounts, and comparing company estimates to actual progress and data[524](index=524&type=chunk) [Report of Independent Registered Public Accounting Firm (Internal Control)](index=119&type=section&id=REPORT%20OF%20INDEPENDENT%20REGISTERED%20PUBLIC%20ACCOUNTING%20FIRM%20%28Internal%20Control%29) Presents the independent auditor's opinion on the effectiveness of the company's internal control over financial reporting [Opinion on Internal Control over Financial Reporting](index=119&type=section&id=Opinion%20on%20Internal%20Control%20over%20Financial%20Reporting) EisnerAmper LLP issued an unqualified opinion on Rocket Pharmaceuticals' internal control over financial reporting as of December 31, 2022 - EisnerAmper LLP issued an unqualified opinion on the effectiveness of internal control over financial reporting as of December 31, 2022[528](index=528&type=chunk) - The assessment was based on criteria established in the Internal Control – Integrated Framework (2013) issued by COSO[528](index=528&type=chunk) - Internal control over financial reporting has inherent limitations and may not prevent or detect all misstatements[532](index=532&type=chunk)[533](index=533&type=chunk) [Consolidated Balance Sheets](index=120&type=section&id=Consolidated%20Balance%20Sheets) Presents Rocket Pharmaceuticals' financial position as of December 31, 2022, and 2021, detailing total assets, liabilities, and equity Consolidated Balance Sheet Summary (in thousands) | Metric | December 31, 2022 | December 31, 2021 | | :--- | :--- | :--- | | Cash and cash equivalents | $140,517 | $232,694 | | Investments | $259,153 | $156,046 | | Total current assets | $364,060 | $392,059 | | Property and equipment, net | $29,009 | $22,299 | | Goodwill | $39,154 | $30,815 | | Intangible assets | $25,724 | $- | | Total assets | $551,807 | $497,020 | | Accounts payable and accrued expenses | $36,660 | $19,615 | | Total current liabilities | $39,169 | $22,167 | | Total liabilities | $62,121 | $42,296 | | Total stockholders' equity | $489,686 | $454,724 | - Total assets increased from **$497.0 million** in 2021 to **$551.8 million** in 2022, driven by increases in investments, property and equipment, goodwill, and intangible assets[536](index=536&type=chunk) - Total liabilities increased from **$42.3 million** in 2021 to **$62.1 million** in 2022, primarily due to higher accounts payable and accrued expenses[536](index=536&type=chunk) - Total stockholders' equity increased from **$454.7 million** in 2021 to **$489.7 million** in 2022[536](index=536&type=chunk) [Consolidated Statements of Operations](index=121&type=section&id=Consolidated%20Statements%20of%20Operations) Shows Rocket Pharmaceuticals' financial performance for 2020-2022, reporting net losses with no revenue, primarily driven by operating expenses Consolidated Statements of Operations Summary (in thousands) | Metric | 2022 | 2021 | 2020 | | :--- | :--- | :--- | :--- | | Revenue | $- | $- | $- | | Research and development | $165,570 | $125,476 | $105,438 | | General and administrative | $58,773 | $41,772 | $28,865 | | Total operating expenses | $224,343 | $167,248 | $134,303 | | Loss from operations | $(224,343) | $(167,248) | $(134,303) | | Net loss | $(221,863) | $(169,069) | $(139,700) | | Net loss per share (basic and diluted) | $(3.26) | $(2.67) | $(2.52) | - The company generated no revenue in 2022, 2021, or 2020[538](index=538&type=chunk) - Net loss increased from **$139.7 million** in 2020 to **$221.9 million** in 2022[538](index=538&type=chunk) - R&D expenses increased by **$40.1 million** (**32%**) from 2021 to 2022, and G&A expenses increased by **$17.0 million** (**41%**) in the same period[447](index=447&type=chunk)[448](index=448&type=chunk) [Consolidated Statements of Comprehensive Loss](index=122&type=section&id=Consolidated%20Statements%20of%20Comprehensive%20Loss) Shows Rocket Pharmaceuticals' total comprehensive loss for 2020-2022, driven by net loss and minor unrealized losses on investments Consolidated Statements of Comprehensive Loss Summary (in thousands) | Metric | 2022 | 2021 | 2020 | | :--- | :--- | :--- | :--- | | Net loss | $(221,863) | $(169,069) | $(139,700) | | Net unrealized loss on investments | $(196) | $(119) | $(62) | | Total comprehensive loss | $(222,059) | $(169,188) | $(139,762) | - Total comprehensive loss increased from **$139.8 million** in 2020 to **$222.1 million** in 2022[541](index=541&type=chunk) - Net unrealized losses on investments were minor, contributing **$(0.2) million** in 2022[541](index=541&type=chunk) [Consolidated Statements of Changes in Stockholders' Equity](index=123&type=section&id=Consolidated%20Statements%20of%20Changes%20in%20Stockholders%27%20Equity) Details movements in common stock, additional paid-in capital, and accumulated deficit for 2020-2022 Key Changes in Stockholders' Equity (in thousands, except share amounts) | Metric | December 31, 2022 | December 31, 2021 | December 31, 2020 | | :--- | :--- | :--- | :--- | | Common Stock Shares | 79,123,312 | 64,505,889 | 60,996,367 | | Common Stock Amount | $791 | $645 | $610 | | Additional Paid-in Capital | $1,203,074 | $946,152 | $825,794 | | Accumulated Deficit | $(713,775) | $(491,912) | $(322,843) | | Total Stockholders' Equity | $489,686 | $454,724 | $503,519 | - Issuance of common stock (net of costs) from public offerings and at-the-market program contributed **$108.1 million** and **$46.6 million**, respecti

Months post gene therapy 23 For 5 patients, increased BM CFC MMC resistance ranging from 51% to 94% was observed at 18 to 24 months post– RP-L102 administration 25 Disease etiology 26 24 Months post gene therapy RP-L201: LAD-I INVESTOR DECK INVESTOR DECK INVESTOR DECK 41st Annual J.P. Morgan Healthcare Conference Company Presentation Gaurav Shah, MD Chief Executive Officer January 9, 2023 DISCLAIMER Various statements in this presentation concerning Rocket's future expectations, plans and prospects, includi ...