Rocket Pharmaceuticals, Inc. (RCKT) — Company Research

Last Updated: 19 April 2026

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Rocket Pharmaceuticals is a US clinical-stage gene therapy company focused on rare diseases, with no approved commercial products. Its lead programme, KRESLADI (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I, had a PDUFA date of 28 March 2026 that is overdue as of this report with no public FDA action yet disclosed. The pipeline is pivoting to cardiovascular AAV gene therapies for Danon disease, BAG3-associated dilated cardiomyopathy and PKP2-arrhythmogenic cardiomyopathy after a mid-2025 restructuring that cut headcount by ~30% and pushed hematology programmes (Fanconi anemia, pyruvate kinase deficiency) back beyond 2026.

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1. Company Snapshot

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Item	Detail
Full Name	Rocket Pharmaceuticals, Inc.
Ticker	NASDAQ: RCKT
Sector / Industry	Biopharmaceutical — Gene Therapy (rare disease)
Headquarters	Cranbury, New Jersey, USA
CEO & Co-founder	Gaurav Shah, MD
Market Cap (mid-April 2026)	~$378–393 million
FY2025 Revenue	~$0 (pre-commercial)
FY2025 Net Loss	$(223.1) million
Cash & investments (31 Dec 2025)	$188.9 million
Shares outstanding (31 Dec 2025)	108.3 million
Employees	~202 (post mid-2025 restructuring)
Exchange	Nasdaq Global Select
Website	rocketpharma.com

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2. Bull Case vs Bear Case

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Bull Case

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• KRESLADI (LAD-I) BLA resubmission was accepted by FDA in October 2025 with PDUFA 28 March 2026; pivotal Phase 1/2 data showed 100% overall survival at 12 months across all treated patients with no treatment-related serious adverse events.
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• Approval would trigger eligibility for a Rare Pediatric Disease Priority Review Voucher, which have recently transacted in the ~$100m range.
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• RP-A501 clinical hold was lifted by the FDA in August 2025; Phase 2 dosing resumption expected in H1 2026 at a recalibrated 3.8 × 10^13 GC/kg dose with sequential 4-week patient intervals; RCKT remains the only clinical-stage gene therapy in Danon disease.
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• RP-A601 Phase 1 data (May 2025) in PKP2-arrhythmogenic cardiomyopathy showed 110–398% increase in PKP2 protein expression from baseline across three patients and no dose-limiting toxicities at 12-month follow-up; Fast Track (US) and Orphan Drug (US/EU) designations in place.
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• Q2 2027 cash runway is guided after ~25% expected operating-expense reduction from the mid-2025 strategic reorganisation; Baker Bros. increased its position in Q1 2026.
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Bear Case

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• The KRESLADI PDUFA date of 28 March 2026 has passed without a public decision as of the date of this report; the drug has previously received one CRL in June 2024 for CMC concerns.
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• A patient death from capillary leak syndrome was reported in the Phase 2 Danon trial in May 2025, linked to the novel C3 inhibitor immunosuppression agent; FDA placed and then lifted the clinical hold in August 2025, but the safety overhang remains.
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• Stock is down roughly 56% year-on-year, with a 52-week range of $2.19–$8.26 reflecting the June 2024 LAD-I CRL and May 2025 Danon safety event.
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• Short interest of 18.6m shares as of 31 March 2026 represents ~22.6% of the float; short position rose 26.5% in the second half of March ahead of the PDUFA.
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• Recent CEO Form 4 activity is net-selling: Gaurav Shah sold shares on 13 and 18 February 2026 and again on 18 November 2025, against a single discretionary purchase of 20,000 shares at $5.08 in April 2025.
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3. What Does This Company Actually Do?

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Rocket develops ex-vivo and in-vivo gene therapies for rare genetic diseases using two platforms: autologous lentiviral vector (LVV) therapies manufactured in-house at the Cranbury, NJ site, and adeno-associated virus (AAV) therapies primarily for cardiovascular indications. None of the products are approved — the company has essentially no product revenue; operations are funded by equity issuance and partnerships.

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LAD-I (KRESLADI, marnetegragene autotemcel). Lentiviral autologous gene therapy for severe Leukocyte Adhesion Deficiency-I, an ultra-rare paediatric immunodeficiency. BLA accepted October 2025; PDUFA 28 March 2026. Previous CRL June 2024 cited CMC issues.

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Danon disease (RP-A501). AAV9 gene therapy for a fatal X-linked lysosomal storage disorder affecting the heart. FDA clinical hold imposed May 2025 after a patient death from capillary leak syndrome; hold lifted August 2025 with a modified immunosuppression regimen and recalibrated dose of 3.8 × 10^13 GC/kg.

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BAG3-associated dilated cardiomyopathy (RP-A701). AAV gene therapy. IND clearance achieved; first-in-human dosing expected mid-2026.

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PKP2-arrhythmogenic cardiomyopathy (RP-A601). AAVrh74 gene therapy; Phase 1 ongoing with three patients at 12-month follow-up. Fast Track (US), Orphan Drug (US/EU).

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Deprioritised programmes. RP-L102 (Fanconi anemia, LVV) and RP-L301 (pyruvate kinase deficiency, LVV) remain in Phase 1/2 development but FDA approval is no longer anticipated in 2026 following the mid-2025 portfolio reshuffle.

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Note: Rocket Pharmaceuticals is a pre-revenue clinical-stage gene therapy company. The table below reflects the pipeline-value distribution rather than commercial revenue.

Segment	% of revenue	What it is
LAD-I / KRESLADI (LVV)	0% (PDUFA 28 Mar 2026)	Lentiviral autologous gene therapy for severe Leukocyte Adhesion Deficiency-I. BLA accepted October 2025 following a June 2024 CRL over CMC issues. Lead commercial programme.
Danon disease / RP-A501 (AAV)	0%	AAV9 gene therapy for a fatal X-linked lysosomal storage disorder of the heart. FDA clinical hold lifted August 2025 after a patient death; modified immunosuppression regimen at a recalibrated 3.8×10^13 GC/kg dose.
BAG3 cardiomyopathy / RP-A701 (AAV)	0%	AAV gene therapy for BAG3-associated dilated cardiomyopathy. IND clear; first-in-human dosing expected mid-2026.
PKP2-ACM / RP-A601 (AAV)	0%	AAVrh74 gene therapy for PKP2-arrhythmogenic cardiomyopathy. Phase 1 ongoing with three patients at 12-month follow-up; Fast Track (US), Orphan Drug (US/EU).
Deprioritised LVV programmes	0%	RP-L102 (Fanconi anemia) and RP-L301 (pyruvate kinase deficiency) — remain in Phase 1/2 but no longer expected to file for approval in 2026 following the mid-2025 portfolio reshuffle.

4. The Business Model

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Rocket is a pre-revenue biotech. Operating expenses are R&D-heavy with commercial infrastructure being built out in anticipation of a potential KRESLADI launch. Q3 2025 R&D expense was $34.1m and SG&A $18.4m (three-month); this is materially lower than Q2 2025 pre-restructuring levels of $42.7m R&D and $25.0m SG&A. The mid-2025 30% workforce reduction is expected to deliver ~25% operating-expense reduction over 12 months.

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Capital is raised through equity issuance, including a recent At-the-Market (ATM) programme. In late March 2026 Rocket sold approximately 19.6m shares via the ATM at $5.11, raising $100m gross proceeds. There is no material debt on the balance sheet.

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The eventual commercial model is premium-priced autologous and AAV gene therapies in ultra-rare indications with small but highly concentrated patient populations, supported by direct-to-patient patient-finding and centres-of-excellence treatment networks. Reimbursement in gene therapy remains challenging; sector precedents (BioMarin's Roctavian, CSL's Hemgenix) show lower-than-expected commercial uptake in spite of approval.

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5. Financial Health

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Metric	FY2024	FY2025
Revenue	~$0	~$0
Net loss	$(258.7)m	$(223.1)m
Net loss per share	$(2.73)	$(2.01)
Cash, cash equivalents & investments (year-end)	$372.3m	$188.9m
Shares outstanding (year-end)	n/a	108.3m

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FY2025 net loss narrowed to $223.1m from $258.7m in FY2024, reflecting the mid-2025 restructuring. Cash fell from $372.3m at end-2024 to $188.9m at end-2025 as the company funded operations from the balance sheet before the late-March 2026 ATM programme. Management has guided to a runway extending into Q2 2027 after the restructuring and capital raise. Institutional ownership is approximately 98.4% of shares outstanding with holders including RTW Investments, Vanguard, BlackRock, Morgan Stanley, Citigroup, Suvretta, Newtyn and Baker Bros. Advisors.

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6. Valuation & Market Data

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Metric	Value	As of
Share price (trading range)	$3.44–$3.64	Mid-April 2026
Market capitalisation	~$378–$393 million	Mid-April 2026
Enterprise value	~$190 million (cash-adjusted)	Mid-April 2026
Price-to-sales	N/A (no revenue)	—
EV/EBITDA	N/A (negative EBITDA)	—
52-week high	$8.26	April 2025
52-week low	$2.19	April 2026
YoY price change	-55.86%	As at mid-April 2026
Short interest (shares)	18.6 million	31 Mar 2026
Short interest (% float)	~22.6%	31 Mar 2026
Days to cover	~2.9 days	31 Mar 2026

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Short interest increased 26.5% between 15 March and 31 March 2026, consistent with positioning into the PDUFA date. Stock is cash-adjusted to roughly $190m EV — a market that is pricing high regulatory risk into the name.

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7. What Are They Building / What's Coming?

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KRESLADI FDA action. PDUFA date was 28 March 2026; as of this report no public FDA decision has been disclosed. Outcomes are binary: approval, CRL, or further delay.

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Danon Phase 2 resumption. The company expects to dose additional patients in the Phase 2 of RP-A501 in H1 2026 at 3.8 × 10^13 GC/kg with a minimum four-week interval between patients. Data presentations are expected at ASGCT in May 2026.

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RP-A701 first-in-human dosing. First patient dosing expected mid-2026 in BAG3-DCM Phase 1.

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RP-A601 ongoing. Additional patients and longer follow-up expected through 2026 in PKP2-ACM Phase 1.

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Management statements from Q4 2025 call (26 February 2026). Gaurav Shah described Rocket as "positioned at the intersection of cardiovascular disease and gene therapy" with "more than five years of clinical experience in Danon disease". Strategic focus is now on three cardiovascular programmes; hematology programmes (Fanconi anemia, PKD) have been explicitly deprioritised and approvals for both are no longer expected in 2026.

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8. Competitive Landscape

Rocket's lead indications sit in niches with few direct rivals, making platform peers more relevant than same-indication competitors.

Same-indication competition. LAD-I — Rocket's KRESLADI would be the first approved gene therapy; no approved competitor exists, but the market is very small (hundreds of patients globally). Danon disease — no approved competitor; Rocket is first-in-class. BAG3-DCM and PKP2-ACM — no approved gene therapies in cardiovascular indications; field is emerging.

Platform / sector peers. Sarepta Therapeutics (Elevidys for DMD) is pivoting after a 2025 safety crisis that halted several AAV programmes following three patient deaths from liver failure. BioMarin (Roctavian for haemophilia A) has seen commercially disappointing uptake. CSL / uniQure (Hemgenix for haemophilia B) — similar slow commercial trajectory. bluebird bio — effectively wound down. Beam Therapeutics and Prime Medicine compete at the next generation (base / prime editing) rather than classical AAV/LVV.

Peer	Market cap (Apr 2026)	Notable capex / KPI	Positioning vs Rocket
Sarepta Therapeutics (SRPT)	~$4bn	Elevidys ~$900m FY25 revenue; 2025 AAV safety crisis	Largest rare-disease gene therapy peer; AAV liver-toxicity pause is a read-across risk to Rocket's AAV programmes
BioMarin Pharmaceutical (BMRN)	~$12bn	Roctavian uptake <$50m annualised	Category warning on commercial realisation for gene-therapy launches
CSL Ltd / uniQure (CSL.AX / QURE)	~$80bn / ~$600m	Hemgenix (haemophilia B) revenue ramp slow	Approved AAV gene therapy in a related indication; demonstrates reimbursement friction
Beam Therapeutics (BEAM)	~$2bn	Multiple base-editing INDs filed 2024–25	Next-generation editing platform; competes for the same development capital but different mechanism
Prime Medicine (PRME)	~$300m	Prime-editing preclinical pipeline	Early-stage competitor for next-gen genome editing capital; not a near-term commercial rival

9. Leadership and Ownership

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Gaurav Shah, MD — Co-founder and CEO. Previously Global Program Head, Cell & Gene Therapies, Novartis; hematology/oncology fellowship at Memorial Sloan Kettering; MD from Columbia (AOA); undergraduate at Harvard (summa cum laude).
\ Aaron Ondrey — Chief Financial Officer. Previously CFO at Mirati Therapeutics.
\ Kinnari Patel, PharmD, MBA — President, Head of R&D and COO.
\ Sarbani Chaudhuri — Chief Commercial & Medical Affairs Officer. Previously VP Hematology at J&J Innovative Medicine.
\ Roderick Wong, MD — Chair of the Board (founder/managing partner of RTW Investments).

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Ownership. Institutional holders own approximately 98.4% of shares. Top holders include RTW Investments LP, Vanguard, BlackRock, Morgan Stanley, Citigroup, Suvretta Capital, Newtyn Management and Baker Bros. Advisors. Baker Bros. raised its position in Q1 2026 to approximately 1.1m shares, adding roughly 1m shares at ~$7.30.

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Recent insider transactions (SEC Form 4).

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Name	Date	Type	Shares	Price	Value	Plan
Gaurav Shah (CEO)	18 Feb 2026	Sell (tax withholding)	5,990	$3.34	~$20.0k	RSU-related
Gaurav Shah (CEO)	13 Feb 2026	Sell	12,279	$3.31	~$40.6k	Discretionary
Gaurav Shah (CEO)	18 Nov 2025	Sell	6,276	$2.98	~$18.7k	Discretionary
Gaurav Shah (CEO)	20 May 2025	Sell	2,253	$6.45	~$14.5k	Discretionary
Gaurav Shah (CEO)	10 Apr 2025	Buy	20,000	$5.08	~$101.6k	Discretionary (open-market)

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On a 12-month view the CEO has been a net seller, with one notable open-market discretionary purchase in April 2025 at $5.08 — close to current market levels.

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10. Risks and Challenges

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Regulatory. KRESLADI's PDUFA date of 28 March 2026 has passed with no public outcome, exposing shareholders to a possible second CRL or further delay. Any adverse outcome on RP-A501 (Danon) safety monitoring could result in another clinical hold.

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Clinical. The May 2025 patient death tied to capillary leak syndrome with a novel C3 inhibitor immunosuppression agent is a material historical safety signal. Gene-therapy field-wide, the 2025 Sarepta events have raised the FDA bar for AAV product safety.

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Cash burn and dilution. Runway into Q2 2027 is sensitive to approval timing and commercial ramp assumptions; ATM issuance and potential future offerings imply further dilution. Share count and net loss per share trends need monitoring through Q1 2026 earnings.

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Commercial execution. Rocket has no commercial experience; gene-therapy pricing and reimbursement remain difficult. Addressable markets for LAD-I and Danon are small, amplifying the importance of every patient identified and reimbursed.

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Competitive and technological. AAV platform sentiment is impaired following Sarepta events; CRISPR-based competitors could reach some of Rocket's target indications.

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Concentration. Near-term value is highly concentrated in KRESLADI approval and RP-A501 execution. Operational disruption at the Cranbury LVV facility would be disproportionate given in-house manufacturing dependence.

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11. Recent Developments

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Last 48 hours (17–19 April 2026): No material company-specific announcements detected. Market remains focused on the overdue KRESLADI PDUFA decision.

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April 2026. ATM offering of ~19.6m shares at $5.11 for $100m gross proceeds completed in late March / early April per prospectus filing. Short interest updated 16 April showing 22.6% of float short as of 31 March 2026. No FDA decision on KRESLADI has been publicly disclosed as at the date of this report.

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February 2026. Q4 2025 and full-year 2025 earnings released 26 February: FY2025 net loss $(223.1)m, cash $188.9m, runway guided into Q2 2027. Management confirmed focus on Danon, BAG3-DCM and PKP2-ACM; Fanconi anemia and PKD programmes deprioritised. CEO sold small amounts of stock on 13 and 18 February.

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October 2025. FDA accepted resubmission of the KRESLADI BLA; PDUFA date set to 28 March 2026.

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August 2025. FDA lifted the clinical hold on the RP-A501 Phase 2 Danon trial (imposed in May 2025 following a patient death) with a modified immunosuppression protocol and recalibrated dose (3.8 × 10^13 GC/kg) with four-week intervals between sequential patient dosings.

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May 2025. One patient in the RP-A501 Phase 2 died following capillary leak syndrome associated with a novel C3 inhibitor immunosuppression agent; a second patient showed early capillary leak signs but recovered after a reduced course. FDA placed a clinical hold.

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June 2024. FDA issued a CRL for the first KRESLADI BLA submission citing CMC concerns.

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12. Key Dates Coming Up

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• Imminent: KRESLADI (LAD-I) FDA action — PDUFA was 28 March 2026 and remains outstanding as at the date of this report.
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• H1 2026: RP-A501 Phase 2 additional patient dosing resumption.
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• May 2026: ASGCT 28th Annual Meeting — gene therapy data presentations (RP-A501, pipeline updates).
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• Mid-2026: RP-A701 (BAG3-DCM) Phase 1 first-in-human dosing.
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• May 2026 (approx): Q1 2026 earnings release — refreshed cash burn and runway update.
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• Q2 2027: Guided cash runway — financing required beyond this point absent a material monetising event.
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• December 2026: ASH Annual Meeting — potential hematology programme data.
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Disclaimer

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This research is produced by ChartsView for educational and informational purposes only. It does not constitute financial advice or a recommendation to buy or sell any security. All information is sourced from publicly available company filings, press releases and official data. ChartsView does not use analyst opinions or third-party ratings. Always conduct your own due diligence and consider your personal financial situation before making investment decisions. Past performance is not indicative of future results.

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