Slide 1: Investor Deck: Seeking Cures, Genetic Medicines
INVESTOR DECK INVESTOR SEEKING DECK CURES GENETIC MEDICINES INVESTOR DECK August 2026
Slide 2: Disclaimer
ABOUT ROCKET PHARMACEUTICALS DISCLAIMER This presentation contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to diff
Slide 3: Vision: Seeking Genetic Medicines Cures
ABOUT ROCKET PHARMACEUTICALS Vision: Seeking Genetic Medicines Cures Values Mission To develop first-in-class and best-in-class curative v Curiosity genetic medicines for Trust Elevate patients with devastating diseases Generosity 3
Slide 4: A Fully Integrated Cardiovascular Rare Disease Company
ABOUT ROCKET PHARMACEUTICALS A Fully Integrated Cardiovascular Rare Disease Company Promising clinical data designed to Late-stage First company with safety and facilitate US and European registration science and efficacy data for genetic medicines and launch with potential???? for expansion innovat
Slide 5: Strong Science, Carefully-selected Assets and Smart Execution
ABOUT ROCKET PHARMACEUTICALS Strong Science, Carefully-selected Assets and Smart Execution Criteria used to THERAPEUTIC PHASE 2 DISCOVERY PRECLINICAL PHASE 1 APPROVED DESIGNATIONS select programs AREA (Pivotal) RMAT, ATMP, Fast Track, AAV RP-A501 Danon disease Orphan Drug (US), Rare First-, best- Pe
Slide 6: Rocket Offers Multi-platform Gene Therapy Expertise
ABOUT ROCKET PHARMACEUTICALS Rocket Offers Multi-platform Gene Therapy Expertise IN VIVO platform EX VIVO platform Laboratory- Remove cells Laboratory- produced AAV and isolate produced LV patient HSCs Therapeutic AAV Therapeutic LV Infusion Direct intravenous of modified injection HSCs Gene-modifie
Slide 7: Well-Positioned as the Leader in Cardiovascular Genetic Medicines
ABOUT ROCKET PHARMACEUTICALS Well-Positioned as the Leader in Cardiovascular Genetic Medicines Why Cardiomyopathy? Arrhythmogenic • High unmet medical need, current standard of care focuses Cardiomyopathy on symptom palliation and does not address root cause • One-time IV therapy with the potential
Slide 8: Our Commitment to the Rare Disease Community
ABOUT ROCKET PHARMACEUTICALS Our Commitment to the Rare Disease Community Allies and Partners Global Focus Mission: in Patient Advocacy GENOME No-Cost Genetic Investigational Counseling and Raising Gene Therapies Testing Awareness Market Research Inclusion of & Patient Epidemiology Perspectives Stud
Slide 9: Our R&D and Manufacturing Capabilities
ABOUT ROCKET PHARMACEUTICALS Our R&D and Manufacturing Capabilities • Total Lab Space: ~30,000 sq. ft. dedicated to R&D, process and analytical development, MS&T, and QC. • GMP Manufacturing Space: ~11,000 sq. ft. for multi-product clinical manufacturing with 2X expansion capability. • Manufacturing
Slide 10: Danon Disease is a Highly Aggressive Rare Genetic Cardiomyopathy
RP-A501: DANON DISEASE Danon Disease is a Highly Aggressive Rare Genetic Cardiomyopathy Danon Disease Clinical Manifestations Disease Etiology Impaired autophagy • X-linked, dominant, monogenic disease • Prominent • Myocardial disarray • Loss-of-function mutations in LAMP2 autophagic vacuoles Severe
Slide 11: Danon Disease – Cumulative Epidemiology
RP-A501: DANON DISEASE Danon Disease – Cumulative Epidemiology • Anchor Population: US HCM Prevalence: 700,000 patients1,2 • Age-Adjustment: Age distribution of prevalent HCM patients Hypertrophic likely older relative to DD patients Cardiomyopathy US HCM Population (HCM) • Weighted, Age-Adjusted Av
Slide 12: Danon Disease can be Recognized Through Multiple Pathways
RP-A501: DANON DISEASE Danon Disease can be Recognized Through Multiple Pathways Cardiac, multisystem, and family-based entry points can converge on a shared path to diagnosis and coordinated care MULTIPLE ENTRY POINTS RECOGNITION & REFERRAL GENETIC CONFIRMATION COORDINATED CARE Cardiac findings • M
Slide 13: RP-A501 Phase 1 Study: Sustained LAMP2 Expression in Cardiomyocytes
RP-A501: DANON DISEASE RP-A501 Phase 1 Study: Sustained LAMP2 Expression in Cardiomyocytes Durable myocardial LAMP2 protein expression seen in all patients Myocardial LAMP2 Protein Expression Representative LAMP2 IHC Images Cohort Patient BL M6 M12 M18 M24 M30 M36 M60 Pre-infusion Post-infusion Post
Slide 14: RP-A501 Phase 1 Study: Benefit Observed Across All Key Parameters
RP-A501: DANON DISEASE RP-A501 Phase 1 Study: Benefit Observed Across All Key Parameters Early LAMP2, BNP, cTnI changes associated with sustained clinical improvement and guided Phase 2 endpoint selection Age at Most LVEF Δ LVMI,* Δ IVSd, Δ LVPWd, Δ NT-proBNP, Δ cTnI,† Δ KCCQ-12 Δ NYHA Cohort Patien
Slide 15: RP-A501 Phase 1 Study: Sustained Improvements in LV Mass Index
RP-A501: DANON DISEASE RP-A501 Phase 1 Study: Sustained Improvements in LV Mass Index LVMI % Change from Baseline LVMI % Change: Baseline to 12M† Patient 1001 1* Patient 1002 2 Patient 1005 3 Patient 1006 4* Patient 1008 6 Patient 1009 7* M12 M18† M9† M12 M12 M12 30% -10% -11% -16% -14% -15% 10% -32
Slide 16: Insights from Danon Disease Patients Treated on the Phase 1 Trial
RP-A501: DANON DISEASE Insights from Danon Disease Patients Treated on the Phase 1 Trial He can walk upstairs without being short of breath or having to stop half-way. He doesn’t have chest pain Prior to therapy, he would say "my wish is not or fast heart rates like he used to. Another amazing to di
Slide 17: Primary Endpoint Reasonably Likely to Predict Clinical Benefit
RP-A501: DANON DISEASE Primary Endpoint Reasonably Likely to Predict Clinical Benefit Justification for use of LAMP2 protein expression and LV Mass Index WT Full Length LAMP2 Protein Expression • Epidemiologic support: Mutation of LAMP2 is the root cause of Danon disease and even Support for Co-Prim
Slide 18: Phase 2 Trial Design of RP-A501 in Male Patients with DD
RP-A501: DANON DISEASE Phase 2 Trial Design of RP-A501 in Male Patients with DD Pivotal, global, single-arm, open-label trial1 Key eligibility criteria Co-Primary Endpoint Current Status Males age ≥8 years, LAMP2 To support accelerated approval, co-primary endpoint Actively engaging with FDA to muta
Slide 19: Pivotal Phase 2 Trial of RP-A501 is Ongoing
RP-A501: DANON DISEASE Pivotal Phase 2 Trial of RP-A501 is Ongoing Phase 2/ Submission/ Preclinical Phase 1 Pivotal Approval Phase 1 Safety & Efficacy Pivotal Phase 2 trial NEXT STEPS • Phase 1 study of RP-A501 demonstrated • Pivotal, global, multicenter, • Regulatory update expected in the second d
Slide 20: A Cardiomyopathy with High-Risk of Arrhythmias and Sudden Cardiac Death
RP-A601: PKP2-ACM A Cardiomyopathy with High-Risk of Arrhythmias and Sudden Cardiac Death PKP2-Arrhythmogenic Cardiomyopathy Clinical Manifestations (PKP2-ACM) Disease Etiology Susceptibility2 • Autosomal dominant mutations in PKP2 • Frequent and/or endurance exercise • Patients are often advised to
Slide 21: PKP2-ACM Prevalence in the US and EU
RP-A601: PKP2-ACM PKP2-ACM Prevalence in the US and EU ACM prevalence 1:1000 to 1:5000 1,2 PKP2 variants 2,572 ACM patients assessed from 13 publications an aggregated 32.9% mean of 32.9% had PKP2 mutations3 Utilizing the conservative ACM ACM-PKP2 US & EU Prevalence prevalence (1:5000) and the ~50,0
Slide 22: Phase 1 Trial Design of RP-A601 in Adult Patients with PKP2-ACM
RP-A601: PKP2-ACM Phase 1 Trial Design of RP-A601 in Adult Patients with PKP2-ACM First-in-human, multi-center, open-label, dose escalation trial1 Starting Dose (RP-A601): Safety and Efficacy Long-Term Follow-Up 8 x 1013 GC/kg (IV) Assessments Yearly Safety Monitoring Month 0 Month 3 Month 6 Month 9
Slide 23: Preliminary Indications of Improvement or Stabilization with RP-A601
RP-A601: PKP2-ACM Preliminary Indications of Improvement or Stabilization with RP-A601 in Clinical Markers of Arrhythmia Burden, Heart Function and QoL Heart Function & QoL Arrhythmia Burden RV Systolic T-wave Age at Most Recent Function KCCQ-12 Score NYHA Class PVCs per 24h NSVTs per 24h inversions
Slide 24: Ongoing FDA Engagement for RP-A601 in PKP2-ACM
RP-A601: PKP2-ACM Ongoing FDA Engagement for RP-A601 in PKP2-ACM Phase 2/ Submission/ Preclinical Phase 1 Pivotal Approval Phase 1 Safety Phase 1 Efficacy NEXT STEPS • RP-A601 was generally well- • All 3 patients demonstrated cardiac transduction with localized • With no further dose escalation plan
Slide 25: Cardiovascular Health is Dependent on Functional BAG3 Protein
RP-A701: BAG3-DCM Cardiovascular Health is Dependent on Functional BAG3 Protein BAG3-Dilated Cardiomyopathy Disease Etiology (BAG3-DCM) • Autosomal dominant mutations in BAG3 gene • Loss of BAG3 leads to an accumulation of misfolded and damaged proteins, which impairs the heart’s ability to contract
Slide 26: BAG3-DCM Patients are at High Risk of Progression to End Stage Disease
RP-A701: BAG3-DCM BAG3-DCM Patients are at High Risk of Progression to End Stage Disease Clinical Manifestations Dilated cardiomyopathy (DCM) • Characterized by progressive thinning of • Impairment of the heart’s ability to contract, the walls of the heart, causing dysfunction of leads to impaired c
Slide 27: RP-A701 Restores Cardiac Function Dose-Dependently in a BAG3-KO Mouse Model
RP-A701: BAG3-DCM RP-A701 Restores Cardiac Function Dose-Dependently in a BAG3-KO Mouse Model1 Restoration of Cardiac Protein Expression Human BAG3 Protein Expression HSPB8 Protein Expression Profibrotic Marker Col1a1 15000 120 **** Arbitrary Unit (Western Blot) 2.0 ✱✱ ✱ • AAVrh.74-BAG3 produced dos
Slide 28: Phase 1 Trial Design of RP-A701 in Adult Patients with BAG3-DCM
RP-A701: BAG3-DCM Phase 1 Trial Design of RP-A701 in Adult Patients with BAG3-DCM First-in-human, open-label, dose escalation trial1 Safety and Efficacy Dose of RP-A701 (IV) Assessments Long-Term Follow-Up Yearly Safety Monitoring Month 0 Month 3 Month 6 Month 9 Month 12 Month 18 Month 24 ≥60 Months
Slide 29: Severe LAD-I: A Life-Threatening Pediatric Genetic Immune Disorder
KRESLADI : Severe LAD-I Severe LAD-I: A Life-Threatening Pediatric Genetic Immune Disorder Severe Leukocyte Adhesion Deficiency-I Clinical Manifestations (LAD-I) Disease Etiology Patients suffer from severe, recurrent infections; fatal in majority1,2 • ITGB2 gene mutations (21q22.3), encoding • An u
Slide 30: KRESLADI FDA accelerated approval
30 *KRESLADI received FDA accelerated approval based on CD18 and CD11a surface expression. Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Slide 31: Commercial Launch Preparation Activities Ongoing
KRESLADI : Severe LAD-I Commercial Launch Preparation Activities Ongoing Commercial Availability Anticipated by Year-End 2026 • Coordinating product supply and readiness with external manufacturing partners Approximately 25 children • QTC engagement and activation in progress estimated to be born wi
Slide 32: Wave 2: Future Cardiovascular Gene Therapies
FUTURE DIRECTIONS Wave 2: Future Cardiovascular Gene Therapies Current programs reflect only the tip of the iceberg – Disciplined focus today for scalable innovation tomorrow. Focused R&D Strategy Above the Surface: for Sustainable Innovation Current Clinical Pipeline First-, Best- and/or Only-in-cl
Slide 33: Expert Leadership With Proven Track Record
ABOUT ROCKET PHARMACEUTICALS Expert Leadership With Proven Track Record Gaurav Shah, M.D. Sarbani Chaudhuri Syed Rizvi, M.D. Chief Executive Officer Chief Commercial & Medical Chief Medical Officer 20+ years of experience across Spearheaded Kymriah Affairs Officer all stages of drug development. dev
Slide 34: Thank You!
THANK YOU! 34