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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, DC 20549

FORM 8-K

CURRENT REPORT
Pursuant to Section 13 OR 15(d) of The Securities Exchange Act of 1934

Date of Report (Date of earliest event reported):  January 9, 2023


Rocket Pharmaceuticals, Inc.
(Exact name of registrant as specified in its charter)


Delaware
001-36829
04-3475813
(State or other jurisdiction of incorporation)
(Commission File Number)
(IRS Employer Identification No.)


9 Cedarbrook Drive, Cranbury, NJ
  
08512
(Address of principal executive offices)
  
(Zip Code)


Registrant’s telephone number, including area code:  (646) 440-9100


Not applicable
(Former name or former address, if changed since last report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2):

☐
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

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

Title of each class
  
Trading
Symbol(s)
  
Name of each exchange on which
registered
Common stock, $0.01 par value
  
RCKT
  
The Nasdaq Global Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company ☐

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. ☐

Item 8.01.
Other Events.

On January 9, 2023, Rocket Pharmaceuticals, Inc. (the “Company”) updated information reflected in a slide presentation and a press release, which are attached as Exhibit 99.1 and Exhibit 99.2 to this Current Report on Form 8-K, respectively, and are incorporated herein by reference. Representatives of the Company intend to use the updated presentation and information contained in the press release in meetings with investors from time to time.

Item 9.01.
Financial Statements and Exhibits.

(d)
Exhibits.

Investor Presentation of Rocket Pharmaceuticals, Inc.
Press Release of Rocket Pharmaceuticals, Inc.
104
Cover Page Interactive Data File (embedded within the Inline XBRL document).

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 
Rocket Pharmaceuticals, Inc.
     
Date: January 9, 2023
By:
  /s/ Gaurav Shah
   
Gaurav Shah, MD
   
Chief Executive Officer and Director


Exhibit 99.1

 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, including without limitation, Rocket's expectations regarding its guidance for 2023 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD), and other diseases, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials and related data readouts, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2021, filed February 28, 2022 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise. 
 
 v  To develop first-in-class and best-in-class curative gene therapies for patients with devastating diseases  Generosity  Trust  Elevate  Values  Mission  Curiosity  ABOUT ROCKET PHARMACEUTICALS  Vision: Seeking Gene Therapy Cures 
 
 Multi-platform,   first-and-best in-class approach to treating complex and life-threatening childhood disorders   ????  Late-stage science and innovation  Strong capabilities and financials  Collaboration and expertise  Promising top-line clinical data designed to facilitate US and European registration and launch with potential for expansion into Asian markets and beyond  Therapeutic area focus:   Heart and bone marrow  Only company with safety and efficacy data for gene therapy targeting the heart  US-based in-house facility dedicated to AAV cGMP manufacturing  ~100,000 sq ft  Well capitalized to develop full pipeline of assets with  $401M1  in cash and cash equivalents; sufficient to fund operations through 2024           Leadership team with proven track record of  drug approvals and launches   20+   World-class scientific experts and partners learning from and collaborating with patient communities  Generating Value-based Gene Therapies  ABOUT ROCKET PHARMACEUTICALS  1Preliminary, unaudited cash balance as of December 31, 2022. 
 
 On-target MOA; clear endpoints   Sizeable market to maximize patient impact  First-, best- and/or only-in-class  Criteria used to select programs  6+ programs with 2 programs fast approaching regulatory filing and launch  AAV, adeno-associated virus; ATMP, advanced therapy medicinal product; BLA, Biologics License Application; LV, lentiviral vector; MAA, Marketing Authorisation Application; MOA, mechanism of action; PRIME, PRIority MEdicines; RMAT, regenerative medicine advanced therapy. PKP2: plakophilin 2; ACM: Arrhythmogenic Cardiomyopathy; BAG3: BLC2-associated athanogene 3 DCM: Dilated Cardiomyopathy  Data on file. Rocket Pharmaceuticals. 2023.  Wave 2  programs   Fast Track, Orphan Drug (US/EU)  DISCOVERY  PRECLINICAL  PHASE 1  PHASE 2 (Pivotal)  Submission and Approval  DESIGNATIONS  AAV RP-A501 Danon Disease  LV RP-L102 Fanconi Anemia  LV RP-L201 Leukocyte Adhesion Deficiency-I  LV RP-L301 Pyruvate Kinase Deficiency  Multiple Undisclosed  Candidates  AAV BAG3-DCM  THERAPEUTIC AREA  AAV RP-A601 PKP2-ACM  CARDIOVASCULAR  HEMATOLOGY  RMAT, ATMP, Fast Track, Rare Pediatric, Orphan Drug (US/EU), PRIME  Fast Track, Orphan Drug (US), Rare Pediatric Designation  RMAT, ATMP, Fast Track, Rare Pediatric, Orphan Drug (US/EU), PRIME  Strong Science, Carefully-selected Assets and Smart Execution: 6 Disclosed programs with compelling clinical and/or pre-clinical proof of concept   ABOUT ROCKET PHARMACEUTICALS 
 
 IN VIVO platform  EX VIVO platform  RP-A501: Danon Disease​  RP-A601: PKP2-ACM ​  AAV BAG3-DCM  RP-L102: Fanconi Anemia   RP-L201: Leukocyte Adhesion Deficiency-I   RP-L301: Pyruvate Kinase Deficiency   AAV, adeno-associated virus; HSC, hematopoietic stem cell; LV, lentiviral vector.   Data on file. Rocket Pharmaceuticals. 2023  Laboratory- produced AAV  Direct intravenous injection  Therapeutic AAV  Remove cells and isolate patient HSCs  Infusion of modified HSCs  Therapeutic LV  Laboratory-produced LV  Gene-modified HSCs  All Rocket therapies transfer full (non-truncated) coding sequence to target tissue   ABOUT ROCKET PHARMACEUTICALS  Rocket Offers Multi-platform Gene Therapy Expertise 
 
 Looking Forward to a Catalyst-Rich 2023  Q1  Completed 2 ​In-house cGMP Danon Batches  2023  2024  cGMP, current Good Manufacturing Processes; FA, Fanconi anemia; H1, first half of the year; 2H, second half of the year; LAD-I, leukocyte adhesion deficiency-I; PKD, pyruvate kinase deficiency; Q1, first quarter of the yearQ2, second quarter of the year; Q3, third quarter of the year; Q4, fourth quarter of the year.  Data on file. Rocket Pharmaceuticals. 2023.  BAG3 IND Filing  FA (C & G) IND Submission   Non-Genotoxic Conditioning for LV  Additional Wave 2 Assets Disclosed   Q2  Q3  Q4  Planned Danon Phase 2 Study Initiation   LAD-I Product Filing   PKP2-ACM IND Filing  Danon EU IMPD Filing  FA Product Filing  PKD Phase 2 Pivotal Study Initiation  Danon Female Study Initiation  LAD-I Moderate Study Initiation  ABOUT ROCKET PHARMACEUTICALS  Transition from Clinical to Commercial Stage 
 
 Standard of care:   Heart transplant (HTx)  Limitations:  Considerable morbidity and mortality   Only ~20% of patients receive HTx  Not curative of extracardiac disease  Therapeutic Challenges  X-linked, dominant, monogenic disease   Loss-of-function mutations in LAMP2​  Disease Etiology  Impaired autophagy  Prominent ​autophagic vacuoles  Myocardial disarray  Clinical Manifestations  Addressable Market – US and EU   Prevalence of 15,000 to 30,000 individuals  Annual incidence of 800 to 1,200 individuals  Severe cardiomyopathy  Mortality secondary to heart failure or arrhythmia  Males: Aggressive disease course, median overall survival: 19 years  Females: Delayed median presentation (~20 years later) due to additional X chromosome, highly morbid and fatal disorder  Other clinical manifestations  Skeletal myopathy  CNS manifestations  Ophthalmologic manifestations  Danon Disease (DD): Serious Condition with Unmet Medical Need   RP-A501: Danon Disease  CNS, central nervous system; LAMP-2B, lysosome-associated membrane protein 2B.  Boucek D, Jirikowic J, Taylor M. Natural history of Danon disease. Genet Med. 2011;13(6):563-568.   Brambatti M, Caspi O, Maolo A, et al. Danon disease: Gender differences in presentation and outcomes. Int J Cardiol. 2019;286:92-98.  
 
 Non-randomized open label study in male DD patients  PRIMARY OUTCOMES  6 to 36 months  **Enrollment Complete**  Data Reporting Details  Pre-dose (baseline) value defined as the mean values from all visits prior to infusion  Core lab data presented for echocardiographic parameters, cardiac serologies and cardiac histology  Phase 1 Study: Treatment Completed  RP-A501: Danon Disease  Pediatric 8 to 14 years  n=2​ at CHOP  Adults (and Adolescents) >15 years  n=5​ at UCSD  Single intravenous dose of RP-A501 (AAV9.LAMP2B) delivering full coding sequence of the LAMP-2B gene  CHOP, Children’s Hospital of Philadelphia; DD, Danon disease; LAMP-2B, lysosome-associated membrane protein 2B; UCSD, University of California San Diego.  *No further enrollment at this dose. †Due to advanced heart failure at the time of dosing (LVEF <40%), patient 1007 received a heart transplant 5 months following infusion of RP-A501. Patient is currently stable  Cohort​  Patient ID​​  Age at infusion​  Time of follow-up (months)​  Low dose  (6.7x1013 GC/kg)  Pediatric​  1008​  12.3​  12​  1009​  11.7​  6​  Low dose   (6.7x1013 GC/kg)  Adult and older​adolescent​  1001  17.4 ​  36​  1002​​  20.3​  36​  1005​​  18.3​  30​  High dose*   (1.1x1014 GC/kg)  Adult and older​adolescent  1006​​  21.1​  24​  1007  20.7  N/A†  Early and long-term safety   Target tissue transduction and LAMP2B protein expression​  Improved myocardial histology  Clinical improvement or stabilization 
 
 RP-A501: Danon Disease  ADULT COHORT   PEDIATRIC COHORT (Low dose)   Revised Immunomodulatory Protocol:   More rigorous daily monitoring of labs in initial days following infusion with independent clinical review team   Reduced steroid dose with earlier taper  Administration of sirolimus and rituximab  Low Dose  No SAEs related to drug product: 2 steroid related SAEs (myopathy)  High Dose  One instance of reversible TMA; led to enhanced RMP  One instance of steroid myopathy   Platelets remained within normal range  No reported skeletal myopathy or late transaminitis with initial steroid dose reduction and more rapid taper, and introduction of sirolimus  Minimal complement activation  No complement-related clinical or laboratory AEs  All AEs were transient and reversible  No treatment-related SAEs  AE, adverse event; SAE, serious AE; TMA, thrombotic microangiopathy.  Data cut-off September 27, 2022, with source data verification through July 11, 2022.  RP-A501 Demonstrates Favorable Safety Profile With Enhanced Immunomodulation Protocol  All SAEs observed within initial 2-4 months following dosing; reversible with supportive care  Both high and low-doses continue to be well tolerated at 2-3 years post treatment   No additional SAEs observed following initial 2-4 months   No RP-A501–related SAEsAll AEs were transient and reversible, with 8 and 13 months of follow up in 1008 and 1009, respectively 
 
 Patient ID   Predose  Month 6  Month 12  Month 36  1001†  0  0.384  0.197  0.120  1002  0  ND  0.575  0.590§  1005  0  0.583  ND  1.228§  1006  0  2.693  1.131  -  1008  0  0.492  -  -  1009  0  Data pending  -  -  LAMP2, lysosome associated membrane protein 2; M, month. IHC = immunohistochemistry; qPCR = quantitative polymerase chain reaction; ND = not done.   The 2nd Cohort 2 pt (1007) underwent heart transplant due to DD progression five months post RP-A501 infusion; data post not included. Pt is currently clinically stable.   1Derks W, Bergmann O. Polyploidy in Cardiomyocytes: Roadblock to Heart Regeneration?. Circ Res. 2020;126(4):552-565.   Note: Cardiomyocytes frequently multinucleated and/or have polyploid nuclei (several genome copies per cell); however, VCN is calculated assuming one diploid nucleus per cell. As a result, presented VCNs likely underestimated by factor of 2-41; ND.  not done, -, visit pending.   † Corticosteroid compliance uncertain. § Month 30 visit.  Pediatric LAMP2 Protein and DNA Suggests Durable Expression As Demonstrated in Adult Cohort  RP-A501: Danon Disease  LAMP2 Protein Expression (VENDOR)   Cardiac LAMP2 DNA by qPCR   (vector copies per diploid nucleus)  *LAMP2 protein expression assessed (relative to normal human controls) by core lab in a blinded fashion of entire tissue sample; Percentages reflect estimated extent of LAMP2 staining:    Grade 0=negative staining; Grade 1 ≤25%; Grade 2 =26%-50%; Grade 3 =51%-75%; Grade 4 >75%.  1001  1002  1005  1006  1008  1009  4  3  2  1  0  Pre  M3  M6  M12  M24  Pre  M3  M6  M12  M24  Pre  M3  M6  M12  M24  Pre  M3  M6  M12  M24  Pre  M3  M6  M12  M24  Pre  M3  M6  M12  M24  M36  Grade 0  Grade 0  Grade 0  Grade 0  No Data  Grade 0  No Data  Grade 0  Grade 0  Oct 2023  Grade 0  March 2023  March 2024  LAMP2 Grade* by IHC  LAMP2 Protein Expression   M36 
 
 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%​1  ↓15%​1  II -> I ​  +32.3​  1009  6  ↓90%​  ↓62%​  ↓21%​  ↑3%​  II -> I​  +26​  Low dose adult/ adolescent  1001  36  ↓98%​  ↑8%​  ↓32%​  ↓9%​  II -> II  +5.3​  1002​  36  ↓96%​  ↓94%​  ↓48%​  ↓40%​  II -> I2​  +17.8​  1005​  30  ↓46%​  ↑6%​  ↓14%​  ↓27%​  II -> I​  +8.33  High dose adult/ adolescent  1006​  24  ↓63%​  ↓69%​  ↓27%​  ↓15%​  II -> I​  +3.1​  BNP, brain natriuretic peptide; DD, Danon disease; hsTnI, high-sensitivity troponin I; KCCQ, Kansas City Cardiomyopathy Questionnaire; LAMP2, lysosome-associated membrane protein 2; LV, left ventricle; NYHA, New York Heart Association. Does not include pt 1007 in Ph1 trial who had advanced HF with EF<40% at enrollment and received HTx 5M following tx due to pre-existing advanced HF. Patient is currently stable.  11008 echocardiographic parameters are M9 visit (M12 pending).  21002 NYHA class depicted for M30 visit (M36 pending).  31005 KCCQ score depicted for M24 visit (M30 pending).  Darker Green = improved; Lighter Green = minimal change (stabilization)   Improvement or Stabilization Observed Across Key Biomarker, Echo Findings and Functional Measures in Phase 1 RP-A501 Study  RP-A501: Danon Disease 
 
 Note: Graphs do not depict serologic values during initial months after therapy; BL, baseline; M, month. Does not include pt 1007 in Ph1 trial who had advanced HF with EF<40% at enrollment and received HTx 5M following tx due to pre-existing advanced HF. Patient is currently stable.     Indicates normal range (>100pg/mL BNP; >0.04ng/mL Troponin-I)  Improvement or Stabilization Observed Across Key Cardiac Biomarkers  RP-A501: Danon Disease 
 
 RP-A501 Phase 1 Patients: Marked Divergence from Natural History in Key Biomarkers  RP-A501: Danon Disease  Note: aNT-proBNP tends to be higher than corresponding bBNP values given longer plasma half-life. External comparator data from prospective natural history (#s 3,4,6,7,8,9), natural history (DD53) and screen failure from Phase I study (1010). Phase I graph only reflects BNP levels and does not depict serologic values prior to initial 6 months after therapy. Does not include pt 1007 in Ph1 trial who had advanced Hf with EF<40% at enrollment and received HTx 5M following tx due to pre-existing advanced HF. Patient is currently stable.  RP-A501 Phase 1Baseline & Timepoints ≥6 months  ULN NT-proBNP (125pg/mL)  ULN BNP (100pg/mL)  17.5y  20.4y  18.3y  21.1y  12.3y  11.7y  1001  1002  1005  1006  1008  1009  Note: All values for RP-A501 Phase I patients are BNP levels  Sample Pediatric and Adolescent External Comparators  Pt#4a  8.8y  Pt#6a  10.6y  Pt#8a  13.7y  DD53b  12.1y  1010b  13.8y  Pt#7a  17.8y  Pt#9a  19.5y  Pt#3a   9.8y  #8: Received HTx 3M following this timepoint  #6: Placed on HTx list 11M following this timepoint;  M36 & pre-transplant BNP pending (likely increased)  DD53: Placed on HTx list ~3.5yrs following this timepoint  #7: Entered Ph1 Trial (1005)  #3: Entered Ph1 Trial 8M following this visit (1008)  1010: Received HTx ~2M following this timepoint  #4: Progressed NYHA Class II to III & Significant decrease in KCCQ (28pts)  #9: Entered Ph1 Trial (1007*) 
 
 Improvement≥ 1 NYHA Class  No changein NYHA Class  Deterioration≥1 NYHA Class  Prospective Natural History Cohort1  RP-A501 Phase 1 Treated Patients (LVEF>40%)  n=6 8-36m f/u  n=6 6-24m f/u  Age 11.7-21.1y2  Age 8.1-19.5y  Indicates pts with LVEF ≥40% at enrollment inprospective Nat Hx study or RP-A501 Phase 1  Indicates pts with LVEF ≥40% at enrollment inRP-A501 Phase 1; unmonitored immunomodulation  Prospective Natural History:  No patients had improved NYHA Class  RP-A501:  All patients with baseline LVEF ≥40% and monitored immunomodulation had improved NYHA Class (from Class II at baseline to Class I)  Note: Does not include pt 1007 in Ph1 trial who had advanced HF with EF<40% at enrollment and received HTx 5M following tx due to pre-existing advanced HF. Patient is currently stable.  1Prospective natural history cohort: Sequential NYHA information was available for 6 of 9 patients in cohort. For remaining 3 patients, 1 was lost to follow-up, one received heart transplant (presumably deteriorated), and one was enrolled in the Phase 1 study.  2RP-A501: Pediatric patients age 11.7 and 12.3 years at rx; all other pts age 17.4-21.1 years at rx.  NYHA Class in Danon Disease Male Patients: Natural History versus RP-A501 Phase 1   RP-A501: Danon Disease 
 
 Insights from Danon Disease Patients Treated on the Phase 1 Trial  RP-A501: Danon Disease  He went to overnight summer camp on his own for the first time and is no longer out of breath walking up stairs. -Pt 1008  Prior to therapy, he was afraid of dying and wanted a chance at life......After gene therapy, we see him smile more now, he bought his own place and working a couple of days a week, he has started to open up for meeting more friends in real life and has gotten a whole new peace of mind now ...he feels better, and he didn’t think that would ever happen –Pt 1006  He walked a 10K with his father following treatment.  He is exercise training twice a week for an hour. -Pt 1009  He can walk upstairs without being short of breath or having to stop half-way. He doesn’t have chest pain or fast heart rates like he used to. Another amazing thing we have seen is about 4 months after his therapy trial he started working and stopped using his motorized scooter altogether. -Pt 1005 
 
 Based on End of Phase I Regulatory Discussion and ongoing dialogue with FDA.   Elements in Discussion  Key Agreements Reached with FDA  6.7x1013 GC/kg dose   Single-arm, open-label study (randomization not appropriate)  Support for use of natural history as external comparator information  Potential for accelerated approval based on a composite biomarker-driven endpoint  6MWT, CPET are not appropriate endpoints in DD   Specific components of composite endpoint including LAMP2 expression  Trial duration and time to endpoint  2 patient run-in for pediatric enrollment (age 8-14 years)  Confirmation pending submission of Phase II protocol and FDA review  Will utilize revised Phase I eligibility criteria (i.e. LVEF >50%)  Age 8 years and older   Optimized immunomodulatory regimen used in Phase I pediatric cohort   All drug product will be produced in-house at Cranbury, NJ facility  Additional Study Elements   Projected Pivotal Study Design  RP-A501: Danon Disease 
 
 HTx, heart transplant; LVEF, LVEF, left ventricle ejection fraction.   *Brambatti M, Caspi O, Maolo A, et al. Danon disease: Gender differences in presentation and outcomes. Int J Cardiol. 2019;286:92-98.   RP-A501: Danon Disease  US Retrospective Natural History  9   Males  1   Female  51   Males   Males (goal 60)   Females (goal 60)  Initiated in 2019, expansion planned  Initiated in 2006  Initiated in 2022  5 in follow-up  4 referred to  RP-A501-0219  2 underwent HTx  3 enrolled  1 not enrolled   (Phase 1 enrollment closed)  13 HTx  Literature review  (Brambatti 2019 publication*)  1 with low LVEF at enrollment progressed rapidly to end stage  7 died  9 HTx  1 died  67   Females  Robust Ongoing Natural History Efforts to Support External Comparator Sample  Prospective Natural History Study  EU Retrospective Natural History Study 
 
 2 Successful Danon AAV cGMP batches produced in Q4 2022  Superior specifications to Phase I material; allow for full dosing with lower total viral particles, potentially further improving safety profile  Productivity: ~3X increase in number of patient treatments per batch   Product Quality: Significant increase in full versus empty viral particles  Product Comparability: All attributes tested to date are comparable or improved  Regulatory progress and production capacity can support pivotal study and commercialization   FDA clearance on continued utilization of HEK-293 cell-based process through commercial  FDA alignment on comparability approach  Potency assay developed in-house  Overall, in-house cGMP manufacturing delivers commercial-ready product with higher yield, improved quality, and likely enhances safety profile  In-House Manufacturing to Support Danon Pivotal Study and Commercial Production  RP-A501: Danon Disease 
 
 Total Lab Space: ~30,000 sq. ft. for process development, analytical development, MS&T and QC  Manufacturing capability from small-scale to toxicology-scale material   Streamlined tech transfer timeline for pipeline assets from plasmid selection to IND in <15 months  Manufacturing expansion to add media and buffer production capability  Incorporating fully automated in-house vial filler suite  Anticipated 2X capacity increase in 2023  Enables rapid, robust and cost-efficient internal development capability for new and existing programs in addition to full-scale commercial manufacturing   facility in Cranbury, NJ  ~100,000 ft2  MS&T, manufacturing, science and technology. QC, quality control. R&D, research and development. IND, Investigational new drug.  Cranbury R&D and Manufacturing Facility Overview   RP-A501: Danon Disease 
 
 Moving toward pivotal global Phase 2 study  FDA, Food and Drug Administration; PRV, priority review voucher.  Data on file. Rocket Pharmaceuticals. 2023.  RP-A501: Danon Disease  Development Plan  GLOBAL  REGISTRATIONAL PHASE 2 STUDY  Final Phase 2 Study Design and Endpoints   Initiate Phase 2 Global Pivotal Study Activities  Expanded natural history study  Ongoing Activities  Phase 1 treatment completed in males  Orphan Drug, Rare Pediatric and Fast Track designations in the US (eligible for PRV)  Completed 2 in-house cGMP batches   End of Phase 1 Regulatory Meeting held with FDA  Study Milestones 
 
 FA-A is an autosomal recessive disease caused by FANCA gene mutations  FA proteins enable DNA repair  FA-A accounts for 60% to 70% of FA cases  Disease etiology  BMF, bone marrow failure; FA, Fanconi Anemia; FA-A, FA, group A; FANC, FA complementation group; GvHD, graft-versus-host disease; HLA, human leukocyte antigen; HSCT, hematopoietic stem cell transplantation.   Alter BP, Giri N, Savage SA, et al. Malignancies and survival patterns in the National Cancer Institute inherited bone marrow failure syndromes cohort study. Br J Haematol. 2010;150(2):179-188.   Disorder of DNA repair characterized by:  Progressive BMF; 80% of patients experience BMF within first decade of life  Predisposition to hematologic malignancies and solid tumors  Clinical manifestations  Standard of care:   Allogeneic HSCT   Limitations:  Significant toxicities, especially for patients who do not have an HLA-identical sibling donor (~80%)  100-day mortality  GvHD  Increased long-term cancer risk  Therapeutic challenges  Market Opportunity – US and EU  Prevalence of 5,500 to 7,000 individuals  Annual incidence of 200 to 275 individuals  Fanconi Anemia (A, C, and G)  RP-L102: Fanconi Anemia  RP-L102 for Fanconi Anemia Complementation Group A (FA-A)  Gene therapy approach: Selective advantage of corrected cells allows for ex-vivo LV therapy without conditioning; highly favorable benefit risk profile  
 
 Progressive increases in gene markings in PB and BM in 7 patients   BM, bone marrow; CD34, cluster of differentiation 34; PB, peripheral blood; PBMC, PB mononuclear cells; VCN, vector copy number.   Not shown: PB and BM VCN in Patient 2 (1002), who was withdrawn from the study at 18 months post–RP-L102 infusion.   BM VCN not available at some stipulated time points due to insufficient sample to run assay.  PBMC VCN  BM VCN  VCN in bone marrow mononuclear cells  VCN in bone marrow CD34+ cells  VCN in peripheral blood mononuclear cells  RP-L102: Fanconi Anemia  Progressively Increasing and Sustained Genetic Correction in 7 of 10 Patients ≥1 Year Post–RP-L102 
 
 For 5 patients, increased BM CFC MMC resistance ranging from 51% to 94% was observed at 18 to 24 months post–RP-L102 administration  MMC resistance of >20% achieved at 2 consecutive timepoints ≥12 months for n=5  12m post gene therapy  20% resistance   to MMC  BM, bone marrow; CFC, colony-forming cells; MMC, mitomycin-C.  Not shown: MMC resistance in Patient 2 (1002), who was withdrawn from the study at 18 months post–RP-L102 infusion.  *One additional patient (Patient 1: 1001) was noted to have BM MMC resistance of 49% at ~40 months post–RP-L102 infusion.  Sustained BM CFC MMC resistance observed in at least 6 patients*  RP-L102: Fanconi Anemia  Increasing Phenotypic Correction (MMC-resistance) over 1 to 3 Years Post–RP-L102 
 
 Additional life-cycle management activities:  Expansion to FANC C and G   Exploration of non-genotoxic conditioning and HSC expansion  Rejection of null hypothesis with minimum of 5 patients with increased MMC resistance >10% at 2 timepoints between 12 and 36 months  Initial comprehensive efficacy in 6/10 evaluable patients (≥12-month follow-up)   No cytotoxic conditioning, only 1 transient RP-L102 related SAE (Grade 2)  INITIAL EFFICACY AND HIGHLY FAVORABLE SAFETY PROFILE  Update: CMC and clinical FDA discussions support BLA activities  2 patients to be treated with product from commercial cell processing site in preparation for US launch   NEXT STEPS  TOP-LINE DATA READOUT ACHIEVED  Anticipated simultaneous BLA/MAA filings   ATMP, advanced therapy medicinal product; BLA, Biologics License Application; CMC, chemistry, manufacturing, and controls; FANC, Fanconi Anemia complementation group; FDA, Food and Drug Administration; HSC, hematopoietic stem cell; MAA, Market Authorization Application; MMC, mitomycin-C; PRIME, PRIority MEdicines; PRV, priority review voucher; RMAT, Regenerative Medicine Advanced Therapy; SAE, severe adverse event.  Data on file. Rocket Pharmaceuticals. 2023.  REGULATORY DESIGNATIONS:   RMAT, PRIME  Orphan Drug designation in the US/EU  Rare Pediatric Disease designation (eligible for PRV)  Fast Track (US), ATMP  Moving toward BLA/MAA filing  Development Plan  RP-L102: Fanconi Anemia 
 
 Patients suffer from recurrent infections; fatal in majority  Severe LAD-I: Death prior to age 2 in 60% to 75% of patients, infrequent survival >5 years in absence of allogeneic HSCT  Moderate LAD-I: Death prior to age 40 in >50% of patients, extensive morbidity with recurrent infections and inflammatory lesions  Clinical manifestations  ITGB2 gene mutations (21q22.3), encoding the beta-2-integrin, CD18; essential for leukocyte adhesion to endothelium  CD18 absent or reduced on neutrophils  Disease etiology  Standard of care:  Allogeneic HSCT  Limitations:  Donor availability   Infections  Frequent GvHD  Graft failure  Therapeutic challenges  CD18, cluster of differentiation 18; GvHD, graft-versus-host disease; HSCT, hematopoietic stem cell transplant; ITGB2, integrin subunit beta 2; LAD-I, leukocyte adhesion deficiency-I.  Almarza NE, Kasbekar S, Thrasher AJ, et al. Leukocyte adhesion deficiency-I: A comprehensive review of all published cases. J Allergy Clin Immunol Pract. 2018;6(4):1418-1420.e10.   Market Opportunity – US and EU  Prevalence of 800 to 1,000 individuals  Annual incidence of 50 to 75 individuals  RP-L201 for LAD-I: ITGB2 Gene Mutation   RP-L201: LAD-I 
 
 CD18 Expression in PB Polymorphonuclear Cells (PMNs)  At 3 to 24 months after infusion, 9/9 patients sustained stable CD18 expression (median: 56%) with no therapy-related serious adverse events  *Dim/weak CD18 expression reported at baseline for Subject L201-003-1004 in ~63% of cells in conjunction with <2% CD11a/CD11b expression, likely indicating abnormal/unstable protein.   LAD-I, Leukocyte Adhesion Deficiency-I; PB, peripheral blood; PMN, polymorphonuclear neutrophil.  Data on file. Rocket Pharmaceuticals. 2022. Data Cut-Off: April 6, 2022; Preliminary interim results are presented from the ongoing clinical study.  RP-L201: LAD-I  * 
 
 Survival without allogeneic HSCT  Primary outcomes  ≥2 years of age AND  ≥1-year post–RP-L201 infusion  100% overall survival Kaplan–Meier estimate  Significant reduction in incidence of hospitalizations  Post engraftment  EOS, end of study; HSCT, hematopoietic stem cell transplantation; LAD-I, leukocyte adhesion deficiency-I.  Data cut-off: March 9, 2022.  Data on file. Rocket Pharmaceuticals. 2022.   Significant Reduction in Hospitalizations and 100% Overall Survival  RP-L201: LAD-I  All patients have been able to stop prophylactic antibiotics  
 
 Life-cycle management  Potential label expansion to include moderate LAD-I population  Potential study initiation in 2023  ATMP, advanced therapy medicinal product; BLA, Biologics License Application; GvHD, graft-versus-host disease; LAD-I, leukocyte adhesion deficiency-I; MAA, Market Authorization Application; PRIME, PRIority MEdicines; PRV, priority review voucher; RMAT, Regenerative Medicine Advanced Therapy; SAE, severe adverse event.  Data cut-off: March 9, 2022.  Data on file. Rocket Pharmaceuticals. 2022.   Survival for 9/9 patients, ≥2 years age and ≥1 year post-treatment  No graft failure, GvHD  No RP-L201 related SAEs  Enrollment completed; 9/9 patients treated  Efficacy observed in 9/9 patients with 3 to 24 months follow-up  Efficacy is comprehensive, across all efficacy parameters including CD18 expression and survival  ENROLLMENT AND INITIAL EFFICACY  Progression to regulatory filing activities   NEXT STEPS  TOP-LINE DATA READOUT Q2 2022  Guiding Q2 2023 regulatory filing  REGULATORY DESIGNATIONS:   RMAT, PRIME  Orphan Drug designation in the US/EU  Rare Pediatric Disease designation (eligible for PRV)  Fast Track (US), ATMP  Moving toward product filing  Development Plan  RP-L201: LAD-I 
 
 Lifelong chronic hemolysis  Other clinical manifestations:   Anemia  Jaundice  Iron overload  Clinical manifestations  Standard of care: Chronic blood transfusions and splenectomy  Limitations:  Iron overload   Extensive end-organ damage  Splenectomy confers lifelong infection and thrombotic risk  Therapeutic challenges  Autosomal recessive inheritance  Pyruvate kinase deficient RBCs cannot synthesize ATP, resulting in hemolytic anemia  Disease etiology  ATP, adenosine triphosphate; PKD, pyruvate kinase deficiency; PKLR, pyruvate kinase L/R; RBC, red blood cell.  Zanella A, Fermo E, Bianchi P, Valentini G. Red cell pyruvate kinase deficiency: molecular and clinical aspects. Br J Haematol. 2005;130(1):11-25.   Market Opportunity – US and EU  Prevalence of 4,000 to 8,000 individuals  Annual incidence of 75 to 125 individuals  RP-L301 for PKD: PKLR Gene Mutation  RP-L301: PKD 
 
 Sustained hemoglobin normalization from ~7.4 g/dL to 13.2 g/dL 24 months post–RP-L301 infusion  No red blood cell transfusions required following engraftment  PATIENT 1001  PATIENT 1002  Sustained hemoglobin normalization from ~7.0 to 14.7 g/dL 24 months post–RP-L301 infusion  No red blood cell transfusions required following engraftment  Hemoglobin improvement to normal range (from baselines in severe (<8 g/dL range)Transfusion independence (extensive transfusion requirements prior to RP-L301)   Sustained improvement of hemolysis markers (LDH, bilirubin) and PB VCNs in 1.0 to 3.0 range  LDH, lactate dehydrogenase; PB, peripheral blood; VCN, vector copy number.  Data cut-off: October 26, 2022; preliminary interim results are presented from the ongoing clinical study.  Data on file. Rocket Pharmaceuticals. 2022.   Δ = 5.8 g/dL  Δ = 7.7 g/dL  RP-L102 Infusion  Dotted lines indicate average hemoglobin for each patient prior to gene therapy  Preliminary Efficacy Results for Patients L301-006-1001 and L301-001-1002   RP-L301: PKD 
 
 Development Plan  Moving toward pivotal Phase 2 study  Key endpoints selected  Hemoglobin increase  ↓ 50% in transfusions or transfusion independence  Well-delineated natural history in recent PKD NHS publications  Complete Phase 1 pediatric cohort dosing (N=2 to 3)  End of Phase 1 regulatory meeting with FDA in 2023  Approve and launch RP-L301; seek regulatory approval in the US and EU  PLAN FOR PHASE 2 AND LAUNCH  Anticipated expansion study to pre-splenectomy patients  Exploration of non-genotoxic conditioning  LIFE-CYCLE MANAGEMENT  FDA, Food and Drug Administration; NHS, National Health Service; PKD, pyruvate kinase deficiency; PRV, priority review voucher.  Data on file. Rocket Pharmaceuticals. 2022.  REGULATORY DESIGNATIONS  Fast Track, Orphan Drug (US/EU), Rare Pediatric Disease (eligible for PRV)  RP-L301: PKD 
 
 Future Therapies: Wave 2 (AAV)  We continue to build our pipeline based on our core R&D strategy, identifying the “most impactful” indications for the most efficient development path.  AAV, adeno-associated virus; MOA, mechanism of action; R&D, research and development.  Data on file. Rocket Pharmaceuticals. 2022.  On-target MOA; clear endpoints   Sizeable market to maximize patient impact  First-, best- and/or only-in-class  Current Clinical Pipeline  Focused R&D Strategy for Sustainable Innovation  3 therapeutic areas   (CV, heme and undisclosed)  FUTURE DIRECTIONS 
 
 We believe that a gene therapy approach is best positioned to restore the broad biological functions of BAG3 in the heart  BAG3, BLC2-associated athanogene 3; BCL2, B-cell lymphoma 2.  Knezevic T, Myers VD, Su F, et al. Adeno-associated Virus Serotype 9 - Driven Expression of BAG3 Improves Left Ventricular Function in Murine Hearts with Left Ventricular Dysfunction Secondary to a Myocardial Infarction. JACC Basic Transl Sci. 2016;1(7):647-656.   Myers VD, Gerhard GS, McNamara DM, et al. Association of Variants in BAG3 With Cardiomyopathy Outcomes in African American Individuals JAMA Cardiol. 2018;3(10):929-938.   4  Cardiac contractility  Enhances contractility by linking the β-adrenergic receptor and L-type Ca2+ channel  Provides support for the sarcomere by linking actin myofibrils with the Z-disc  Structural support  Facilitates autophagy as a co- chaperone with heat shock proteins, recycling misfolded proteins  Protein quality control  Inhibits apoptosis (programmed cell death) through binding of BCL2  Anti-apoptosis  BAG3 Dilated Cardiomyopathy  BAG3 Regulates Critical Functions in Cardiomyocytes 
 
 AAV, adeno-associated virus; BAG3, BCL2-associated athanogene 3; DCM, dilated cardiomyopathy; EF, ejection fraction; GFP, green fluorescent protein; H1, first half of the year; IND, investigational new drug; WT, wild type. Haploinsufficiency data published in Myers VD et. al., J Cell Physiol. 2018; AAV-BAG3 administration adapted from data published in Myers VD et. Al., JAMA Cardiol. 2018; and unpublished data from the Feldman lab.  Dilated cardiomyopathy (DCM) is the most common form of cardiomyopathy  20% to 50% of DCM patients have familial DCM; up to 40% of whom have an identifiable genetic cause(1)  Scientific societies recently endorsed clinical genetic testing for DCM patients and families(2,3)  Prevalence of BAG3 DCM in US is estimated to be as high as 30,000 patients(4) and is expected to grow with increasing genetic testing and disease awareness  Initial proof of concept for AAV9-BAG3 demonstrated in BAG3-knockout mouse model  Evaluating optimal development pathway; IND planned H1 2024   BAG3-DCM Represents a Significant Market with Unmet Need   Initial Proof-of-Concept for AAV9-BAG3 Supports Further Development  WT mice: AAV-GFP or  AAV-BAG3  BAG3 +/- mice: AAV9 BAG3  treatment group  BAG3 +/- mice: Control group  (AAV-GFP)  Ejection fraction in WT and BAG3 +/- mice treated at age 6 to 8 weeks with AAV9-GFP or AAV9-BAG3  BAG3 Dilated Cardiomyopathy  BAG3-DCM Opportunity and Next Steps  
 
 Project Pegasus (PKP2-ACM) 
 
 Current standard of care includes beta-blockers, anti-arrhythmic agents, and ablation   Available treatments do not modify disease progression; no curative therapeutic options  Therapeutic Challenges  Autosomal dominant mutations in PKP2 gene, which encodes for Plakophilin-2, a component of the desmosome localized to cardiac intercalated discs  Disease Etiology  Clinical Manifestations  Estimated Prevalence (US+EU): ~50,000  Advanced ACM Heart with  fibrofatty replacement in right ventricle   ACM: Diminished Myocardial PKP2   Normal   patient  PKP2-ACM  patient  N-Cadherin  PKP2  PKP2-Arrhythmogenic Cardiomyopathy (ACM)*:   A high-risk disease with no curative options  RP-A601: PKP2-ACM  Kaplan-Meier Incidence of ICD Firing​  Event free survival in ACM patients with ICDs based on VT inducibility on pre-ICD EPS study  >50% of patients who were inducible on EP study had an ICD firing over 2 year follow-up  Mean age at presentation: 35y (±18) 1  5-10% annual risk of sustained ventricular arrhythmias (VA), with higher risk in patients who present with disease symptoms (index patients)2-3   Lifetime VA risk approximately 100% in index patients 4  ICD placement in >80% of index pts 5  For pts with ICDs:   45-75% will have ICD firing (shock) over 3-5 years   ≥50% 2 year incidence of firing in subgroups:• male; • EPS-induced VT; • history of VT; • ≥3 ECG leads with TWI; • >1000 PVC/24h 5-6  Biopsy figure adapted from: Asimaki et al. NEJM, 2009; Table adapted from Dalal et al. Circulation 2006. SOC: standard of care; CM: cardiomyopathy; HF: heart failure: HTx: heart transplantation; RV: Right ventricular; SD: Standard Deviation; VT: ventricular tachycardia; LBBB: left bundle branch block; ICD: implantable cardioverter defibrillator; RVEF: right ventricular ejection fraction; LV: left ventricle; SVA: sustained ventricular arrhythmia.  * This cardiomyopathy initially manifests in the right ventricular free wall, so the disease was termed arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/ARVC). However, since left dominant and biventricular forms have also been observed, this has led more recently to the use of the term “ACM”.  1. Bhonsale. EHJ 2015; 36: 847-55. 2. Towbin JA. Heart Rhythm 2019;16(11). 3. Cadrin-Tourigny J. Eur Heart J 2022;43. 4. Groenweg. Circ Cardiovasc Genet 2015; 8: 437-46. 5. Calkins. Circ 2017; 136: 2068-82. 6. Orgeron. J Am Heart Assoc 2017: e006242.  Electrical manifestations can precede structural abnormalities 
 
 Peters S, Trümmel M, Meyners W. Prevalence of right ventricular dysplasia-cardiomyopathy in a non-referral hospital. Int J Cardiol. 2004;97(3):499-501.   McKenna WJ, Judge DP. Epidemiology of the inherited cardiomyopathies. Nat Rev Cardiol. 2021;18(1):22-36.   1 Data on file  2,572 ACM patients assessed from 13 publications an aggregated mean of 32.9% had PKP2 mutations1   Utilizing the conservative ACM prevalence (1:5000) and the 32.9% PKP2 mutation frequency in ACM  PKP2-ACM Prevalence in the US and EU  RP-A601: PKP2-ACM  1:1000 to 1:5000  ACM prevalence  32.9%  PKP2 variants  ~50,000  ACM-PKP2 US & EU Prevalence  Peters 2004, McKenna 2021 
 
 Proof of Concept in Translationally Relevant Animal Model  PROGRAM 4: RP-L301 for PKD  RP-A601: PKP2-ACM  Initial POC evaluated 4 AAV Vectors: Cardiac Functional & Structural Analyses  Dose-related effects evaluated with 2 AAV vectors: Cardiac Functional & Structural Analyses   Evaluated Survival, Functional, and Anatomic Benefit in ‘Arrest Progression’ Models  Including delivery of AAV +7 or +14 Days after induction of PKP2 knockout and subsequent disease onset  Completed RCKT Studies with Cardiomyocyte-specific PKP2 Knockout Mouse Model of ACM  Analyses Include:  Survival  Echocardiography and ECG  PKP2 expression (IF and WB)  Cardiac pathology & fibrosis  Vector DNA, transgene mRNA  General safety including pathology  Academic Partner:  Mario Delmar, MD, PhD  Patricia and Robert Martinsen Professor of Cardiology,   Department of Medicine; Division of Cardiology,  NYU Grossman School of Medicine  Marina Cerrone, MD  Research Associate Professor,   Co-Director, Inherited Arrhythmia Clinic,  Department of Medicine; Division of Cardiology,   NYU Grossman School of Medicine  Ongoing sponsored research. No future royalty obligations   NYU Grossman School of Medicine 
 
 Tamoxifen-induced ACM in the PKP2-cKO Mouse Model        The PKP2-cKO mouse model recapitulates ACM following induction of PKP2 KO by tamoxifen (TAM) injection  Progression of cardiomyopathy evidenced by Masson’s trichrome staining of heart sections in PKP2-cKO mice from 14 to 42 days post-TAM (dpi)   100% mortality by day ~50 following TAM injection  Left ventricular ejection fraction (LVEF) diminishes significantly across time  Right ventricular (RV) enlargement occurs across time   Premature Ventricular Contractions (PVCs) are a clinical hallmark of ACM and emerge in the animal model because of Pkp2 loss     Cerrone M, Montnach J, Lin X, et al. Plakophilin-2 is required for transcription of genes that control calcium cycling and cardiac rhythm. Nat Commun. 2017;8(1):106. Published 2017 Jul 24.   ISO, isoproterenol; cKO: conditional knockout        RP-A601: PKP2-ACM 
 
 1Data not illustrated in Figures  Increased Survival & Preserved Cardiac Function in the PKP2-cKO Model  RP-A601: PKP2-ACM  AAVrh.74-PKP2 delivered 7 days post-TAM:  100% survival to 5 months, compared to 100% mortality by day ~50 in PKP2-cKO control animals   Preserved Ejection Fraction and Right Ventricular Area at 28 Days, sustained to 5 months  AAVrh.74-PKP2 delivered 14 days post-TAM:  Mitigated isoproterenol-induced PVCs and arrhythmia, disease-related characteristics of ACM  Robust benefit on survival, cardiac function & structure to 5 months1  *p <0.0001 vs PKP2-cKO FB  *p <0.0001 vs PKP2-cKO FB  *p <0.05 vs PKP2-cKO FBISO = isoproterenol; TAM = tamoxifen; ECG = Electrocardiography   ISO-Induced Arrhythmia  AAV+14D Post-TAM; ECG at 21D post-TAM  AAV +7D post-TAM  AAVrh.74-PKP2a Dose 1 (n = 10)  Control-FB (n = 9)  PKP2-cKO-FB (n = 10)  - 
 
 cDNA/isoform:  PKP2a: full wild type coding sequence of therapeutic gene, protein loss drives ACM   AAV Serotype:   AAV.rh74 serotype associated with favorable safety profile in DMD/LGMD2E1-2; potential for safe administration at optimal doses for adult ACM patients   Cardiac-Specific Promoter:  Effectively drives expression of therapeutic transgene in cardiomyocytes; minimizes off-target effects  Route of Administration:   Intravenous (IV) Pharmacology studies demonstrate efficient cardiac transduction with IV administration   Robust Proof of Concept in Disease Relevant Animal Model:  NYU Cardiac-specific cKO-PKP2 mouse (biologically relevant translational model)  Optimal Gene Therapy for PKP2-ACM, Expected to be First-and Best-In-Class  RP-A601: PKP2-ACM  1Rodino-Klapac et. al. Safety, β-Sarcoglycan Expression, and Functional Outcomes From Systemic Gene Transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4. Presented at the Muscular Dystrophy Association (MDA) Conference. Nashville, TN, March 13−16, 2022.​  2Mendell et. al. A Phase 2 clinical trial evaluating the safety and efficacy of delandistrogene moxeparvovec (SRP-9001) in patients with Duchenne muscular dystrophy. Presented at the 2022 Muscular Dystrophy Association (MDA) Conference Nashville, TN, March 13–16, 2022. 
 
 Clinical Development Plan  Moving Toward Phase 1 Dose Escalation Study  FDA, Food and Drug Administration; 1H, first half of the year. FIH, first in human.   a potential for up to 3 additional patients per cohort in setting of DLTs in 1st 3 pts  Study design:   FIH, multi-center, 3+3 dose escalationa study  Two dose levels planned   Target population:  High risk adult PKP2-ACM patients   Primary endpoint:  Safety events related to RP-A601  Secondary and exploratory endpoints: TBD  High Level Phase 1 Proposed Trial Design   GMP drug product manufacturing completed  Pharmacology and GLP toxicology studies  Potency assay  Upcoming Scientific Advisory Board   Clinical trial planning activities, including site selection, underway  Submit Orphan Disease Designation   FDA IND submission anticipated Q2 2023  Completed or Ongoing Activities  To provide context for the Phase 1 study results, we will leverage data from existing ACM registries as well as longitudinal and population-level data from published case series  Natural History  RP-A601: PKP2-ACM 
 
 Pre-eminent maturing gene therapy pipeline in which each program is First- and Best-in-Class   Experienced management team with a history of delivering transformative and curative therapies to patients with devastating diseases  Well-capitalized and poised to elevate from a clinical-stage to a commercial-stage company  Rocket – The Leader in Rare Disease Gene Therapy 
 
 THANK YOU! 
 

Exhibit 99.2

Rocket Pharmaceuticals Expands Cardiac Gene Therapy Portfolio with Addition of RP-A601 for PKP2-ACM
and Announces Positive Updated Phase 1 Data for RP-A501 in Danon Disease

RP-A601 for Arrhythmogenic Cardiomyopathy due to PKP2 pathogenic variants (PKP2-ACM) represents meaningful commercial opportunity with estimated prevalence of 50,000 adults and children in the U.S. and EU; IND filing anticipated in Q2 2023

In support of Phase 2 pivotal study, latest positive Phase 1 data in Danon Disease recently shared with FDA demonstrate improvements in all biomarker and clinical endpoints across pediatric and adult patients with marked difference vs. natural history; two cGMP manufacturing runs completed with high product quality at in-house facility

BLA filings for LAD-I and FA on track for Q2 2023 and Q4 2023, respectively

Well-capitalized to develop full pipeline of assets with $401M (preliminary, unaudited) in cash and cash equivalents; operational runway now expected through 2024 (inclusive of PKP2-ACM program)

CRANBURY, N.J. – Jan. 9, 2023 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces the addition of RP-A601 to Rocket’s cardiac gene therapy portfolio as well as anticipated highlights for the year ahead across the Company’s world-class pipeline of lentiviral and AAV gene therapy programs targeting rare hematologic and cardiovascular diseases. These announcements and anticipated highlights will be presented at the 41st Annual J.P. Morgan Healthcare Conference today at 2:15 p.m. PT by Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.

“I am extremely excited to build on our significant progress in 2022 as we advance six programs with compelling clinical and/or preclinical proof of concept, including the addition of RP-A601 for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM), expanding our industry-leading cardiovascular AAV gene therapy portfolio. Equally, we look forward to submitting our first regulatory filings, for Fanconi Anemia and Leukocyte Adhesion Deficiency (LAD-I), this year,” said Dr. Shah. “RP-A601 for ACM aims to address the unmet needs of approximately 50,000 adults and children in the U.S. and EU who face this devastating genetic heart disease marked by arrhythmias and heart failure that often results in sudden cardiac death. We are moving this program forward into the clinic having demonstrated robust preclinical proof of concept using an rh74 serotype that has been associated with a favorable safety profile in the clinic for other diseases. We anticipate being first to clinic with a submission of an IND for RP-A601 in the second quarter of 2023 for a multi-center, dose-escalation study for the treatment of PKP2-ACM.”


Dr. Shah continued, “Additionally, positive data updates today in Danon Disease include several months of additional Phase 1 results that demonstrate further robust improvements in all biomarkers, and in how both adult and pediatric patients function and feel, that diverge meaningfully from natural history patients of similar age and disease characteristics. We have shared these updates with the FDA as part of our pivotal Phase 2 study design discussions. Additionally, we have successfully produced two Danon AAV cGMP batches at our Cranbury, N.J. facility with improved product specifications versus Phase 1 thus taking another essential step towards commercial readiness.”

“Clinical programs from our hematology portfolio, represented by our Phase 2 pivotal studies in Fanconi Anemia and LAD-I, remain on track. We are laser focused on regulatory submissions, with BLA filings anticipated for LAD-I and FA in the second quarter and fourth quarter of 2023, respectively,” said Dr. Shah. “Lastly, I am pleased to note an extension of our cash runway, which we expect to fund operations through 2024. I am incredibly pleased with our progress, and excited about the catalyst-rich year ahead as we continue on our path to becoming a fully integrated rare disease and gene therapy company with capabilities extending from discovery through manufacturing and commercialization.”

RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy on Track for IND Submission in Q2 2023

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ACM, or arrhythmogenic right ventricular dysplasia (ARVC), due to plakophilin 2 pathogenic variants (PKP2-ACM), is a high-risk cardiomyopathy caused by autosomal dominant mutations in the PKP2 gene. ACM is characterized by frequent and life-threatening ventricular arrhythmias and structural ventricular myopathy. Available treatments fail to address the underlying genetics and disease biology and do not alter disease progression. PKP2-ACM affects approximately 50,000 people in the U.S. and EU.

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Preclinical proof of concept from a translationally relevant animal model has been demonstrated following Rocket-sponsored studies with academic partners at NYU Grossman School of Medicine. The preclinical studies with a cardiomyocyte-specific PKP2 knockout mouse model of ACM evaluated initial proof of concept and dose-related effects of AAV vectors, including survival, functional and anatomic benefits. Notably, studies evaluated the delivery of AAV at seven and 14 days following induction of PKP2 knockout and subsequent disease onset.

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Results demonstrated increased survival and preserved cardiac function in the PKP2 knockout mouse model.

o
100% of adult PKP2 knockout mice receiving RP-A601 seven days after knockout induction demonstrated survival to the five-month duration of the evaluation compared to 100% mortality by approximately day 50 in PKP2 knockout mice receiving formulation control. PKP2 knockout mice receiving RP-A601 were observed with preserved ejection fraction and right ventricular area at 28 days, sustained to five months.

o
Fourteen days following RP-A601 administration, PKP2 knockout mice demonstrated robust survival with a similar degree of cardiac benefit to five months. RP-A601 was also associated with mitigation of isoproterenol-induced PVCs and arrhythmias, which are major morbidity components of ACM.

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GMP drug product manufacturing is completed, and a potency assay has been developed. Based on the strength of the pharmacology and toxicology data, Rocket anticipates filing an IND in the second quarter of 2023 for a Phase 1 multi-center, dose-escalation study evaluating two doses.


RP-A501 for Danon Disease Moving Toward Global Phase 2 Pivotal Study

 •
Efficacy results from the Phase 1 study continue to demonstrate durable improvement or stabilization of clinical parameters in the Danon Disease patients treated to date. Notably, these patients’ improvements and stabilization of brain natriuretic peptide (BNP) and New York Heart Association (NYHA) class are in stark contrast to BNP increases and NYHA class deterioration observed in a representative sample of pediatric and adolescent natural history patients. These data have been presented recently to the FDA.

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The Phase 2 pivotal trial remains on track for initiation in the second quarter of 2023 based on ongoing and productive FDA interactions.

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Robust Technical Operations capabilities are highlighted by the advancement of in-house AAV cGMP manufacturing at Rocket’s state-of-the-art facility in Cranbury, N.J. Completion of two in-house production runs resulted in high-quality drug substance enabling an approximately threefold increase in the number of patients treatable per batch, a significantly improved full versus empty particle ratio, and promising product comparability data generated to date compared to the Phase 1 material manufactured externally.

J.P. Morgan Healthcare Conference Webcast
Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma, will be presenting at the 41st Annual J.P. Morgan Healthcare Conference today at 2:15 p.m. PT. A live audio webcast of the presentation is available under “Events” in the Investors section of the Company’s website at https://ir.rocketpharma.com. The webcast replay will be available on the Rocket website following the conference.

Anticipated 2023 Milestones
Hematology (LV)
RP-L102 for Fanconi Anemia

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Product filing – Q4 2023

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Complementation Groups C & G Investigational New Drug (IND) submission – 2024

RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I)

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Product filing – Q2 2023

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LAD-I moderate study initiation – Q4 2023

RP-L301 for Pyruvate Kinase Deficiency (PKD)

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Phase 2 pivotal study initiation – Q4 2023

LV Platform Enhancements

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Non-genotoxic conditioning for LV​ – 2024

Cardiovascular (AAV)
RP-A501 for Danon Disease

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Completed two in-house cGMP batches – Q1 2023



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Planned Phase 2 pivotal study initiation – Q2 2023

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EU Investigational Medicinal Product Dossier (IMPD) filing – Q3 2023

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Danon female study initiation – Q4 2023

RP-A601 for PKP2-ACM

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IND filing – Q2 2023

BAG3-Associated DCM

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IND filing – 2024

Undisclosed Candidates

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Disclosure of additional Wave 2 assets – 2024

About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also has preclinical AAV-based gene therapy programs in PKP2-arrhythmogenic cardiomyopathy (ACM) and BAG3-associated dilated cardiomyopathy (DCM). For more information about Rocket, please visit www.rocketpharma.com.


Rocket Cautionary Statement Regarding Forward-Looking Statements
Various statements in this release concerning Rocket’s future expectations, plans and prospects, including without limitation, Rocket’s expectations regarding its anticipated 2023 and 2024 milestones, goals and target, ability to develop its full pipeline of assets with its current cash and cash equivalents, the sufficiency of its cash and cash equivalents to fund operations through 2024, guidance for 2023 and 2024 in light of COVID-19,  the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, Rocket’s plans for the advancement of its Danon Disease program, including its planned pivotal trial, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2021, filed February 28, 2022 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q.  Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Media
Kevin Giordano
kgiordano@rocketpharma.com

Investors
Brooks Rahmer
investors@rocketpharma.com