RCKT Stock — Rocket Pharmaceuticals Inc.: Cash, EV, Pipeline

What they do

We are a fully integrated, late-stage biotechnology company focused on the development, manufacturing, and potential commercialization of genetic therapies for rare and often fatal diseases with high unmet medical need. Our innovative multi-platform approach is designed to create best-in-class gene therapy product candidates aimed at correcting the underlying genetic causes of complex inherited cardiomyopathies and related disorders, with the potential to deliver transformative and durable clinical benefits.

Source: 10-K FY2025 via SEC EDGAR

Share price

$2.48

Market cap

$272.2M

Cash

$283.7M

Enterprise value

-$11.5M

Balance sheet

Cash	$283.7M
Debt	$0.0M
Net cash	$283.7M
Enterprise value	-$11.5M
Basic shares	109.8M shares
Fully diluted shares	115.3M shares
Fully diluted market cap	$285.9M
Cash per share	$2.58
Quarterly burn	$39.6M
Cash runway	7.2 quarters

Cash figures as of 2026-06-30. Database snapshot: 2026-11-11.

Share structure

Common

109.8M

Pre-funded warrants

0.0M

Options

—

RSUs

—

Warrants

5.5M

Converts

—

Conv preferred

—

ESPP reserve

—

FDSO

115.3M

Cap table as of 2026-07-31.

Price history

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Next catalyst

RP-A501 · May 13 - Jun 24 in 18 mo · Phase 2 · Danon Disease

Upcoming catalysts

Expected	Drug	Phase	Indication	Mgmt guide
May 13 - Jun 24 in 18 mo	RP-A501	Phase 2	Danon Disease	—

Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.

Full readout calendar →

Pipeline assets

KRESLADI

Phase 3 · Active Modality: ex vivo gene therapy (lentiviral-modified autologous hematopoietic stem cells)

Aliases RP-L201, marnetegragene autotemcel

Target ITGB2 (CD18)

Partnerships In-licensed from CIEMAT, CIBERER and Instituto de Investigación Sanitaria Fundación Jiménez Díaz; LV vector developed in collaboration with University College London and CIEMAT

Indications

Leukocyte adhesion deficiency-I (LAD-I) (Phase 3)

Catalysts

— March 28, 2026

Status note Phase 1/2 registration-enabling trial treated 9 severe LAD-I patients (published NEJM May 2025). BLA accepted with priority review (Sept 2023); review extended to June 2024; FDA issued CRL June 2024 (limited additional CMC). BLA resubmission accepted October 2025 with PDUFA March 28, 2026. RMAT, Rare Pediatric, Fast Track, PRIME, ATMP, Orphan designations.

RP-A501

Phase 2 · Active Modality: gene therapy (AAV9 capsid, human LAMP2B transgene, single IV infusion)

Target LAMP2

Partnerships CIRM: up to $5.8M CLIN2 grant (Aug 2024) supporting clinical development

Indications

Danon disease (DD) (Phase 2)

Status note Global single-arm Phase 2 pivotal trial (12 patients). Enrollment completed Sep 2024. Dosing paused after two SAEs (capillary leak syndrome, one death) in May 2025; FDA clinical hold May 23, 2025 lifted August 2025. Resuming at recalibrated dose 3.8e13 GC/kg with sequential dosing. RMAT, Fast Track, Rare Pediatric, Orphan, ATMP, PRIME designations.

RP-A601

Phase 1 · Active Modality: gene therapy (AAVrh74 capsid, human PKP2 transgene, single IV infusion)

Target PKP2

Indications

Plakophilin-2 arrhythmogenic cardiomyopathy (PKP2-ACM) (Phase 1)

Status note Single-arm open-label Phase 1 ongoing in adults with ICDs (3 treated; 8e13 GC/kg selected as development dose; generally well tolerated; PKP2 protein expression increased in all 3). RMAT, Fast Track, Orphan designations.

RP-A701

Preclinical · Active Modality: gene therapy (AAVrh74 capsid, human BAG3 transgene)

Target BAG3

Partnerships Acquired via Renovacor acquisition (December 2022)

Indications

BAG3 dilated cardiomyopathy (BAG3-DCM) (Preclinical)

Status note IND cleared June 2025; Fast Track granted July 2025. First-in-human multi-center dose-escalation Phase 1 in adults with BAG3-DCM planned.

RP-L102

Phase 2 · Active Modality: ex vivo gene therapy (lentiviral-modified autologous hematopoietic stem cells)

Target FANCA

Indications

Fanconi anemia (FA) (Phase 2)

Status note Phase 2 study treated 14 FA type A patients without myeloablative conditioning. As of July 2025, no internal resources allocated to regulatory filings/commercialization; company exploring external partnership options (decision based on business strategy, not safety/efficacy).

RP-L301

Phase 1 · Active Modality: ex vivo gene therapy (lentiviral-modified autologous hematopoietic stem cells)

Target PKLR

Partnerships In-licensed from CIEMAT, CIBERER and Instituto de Investigación Sanitaria Fundación Jiménez Díaz

Indications

Pyruvate kinase deficiency (PKD) (Phase 1)

Status note Phase 1 study treated 2 adult and 2 pediatric patients. FDA alignment on 10-patient pivotal Phase 2 for accelerated approval, but no internal resources allocated and no enrollment planned; exploring external partnerships.

Per-asset detail extracted from the 10-Q filed 2026-08-10. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.

Pipeline

Lead drug	RP-L102
Lead indication	Fanconi Anemia Complementation Group A
Lead phase	Phase 2
Active trials	10

Active clinical trials

Trial	Phase	Status	Role	Sponsor	BP	Condition(s)	Mgmt guide
NCT06092034	Phase 2	Recruiting	Sponsor	RCKT	—	Danon Disease	—
NCT04248439	Phase 2	Active, not recruiting	Sponsor	RCKT	—	Fanconi Anemia Complementation Group A	—
NCT05885412	Phase 1	Recruiting	Sponsor	RCKT	—	PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)	—
NCT07137338	Phase 1	Recruiting	Sponsor	RCKT	—	Dilated Cardiomyopathy (DCM)	—
NCT06214507	Not disclosed	Recruiting	Sponsor	RCKT	—	Danon Disease	—
NCT06644742	Not disclosed	Recruiting	Sponsor	RCKT	—	Cardiomyopathies; Heart Diseases; Cardiovascular Diseases	—
NCT07486752	Not disclosed	Not yet recruiting	Sponsor	RCKT	—	Dilated Cardiomyopathy (DCM); Cardiovascular Diseases; Heart Diseases	—
NCT04437771	Not disclosed	Active, not recruiting	Sponsor	RCKT	—	Fanconi Anemia Complementation Group A; Fanconi Anemia	—
NCT06282432	Not disclosed	Active, not recruiting	Sponsor	RCKT	—	Leukocyte Adhesion Deficiency	—
NCT07527975	Not disclosed	Enrolling by invitation	Sponsor	RCKT	—	Fanconi Anemia	—

Sponsor-matched active trials from ClinicalTrials.gov (10 as sponsor, 0 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.

View all 10 active trials for Rocket Pharmaceuticals Inc. on ClinicalTrials.gov

Specialist fund holders

Tracked specialist funds: ADAR1 Capital Management, Aisling Capital, Ally Bridge Group, Avoro Capital Advisors, BVF Partners, Baker Bros. Advisors, Boxer Capital, Braidwell LP, Casdin Capital, Cormorant Asset Management, DAFNA Capital, Deerfield Management, EcoR1 Capital, Frazier Life Sciences, Great Point Partners, Ikarian Capital, Logos Global Management, OrbiMed Advisors, Perceptive Advisors, RA Capital Management, RTW Investments, Sphera Biotech, Stonepine Capital, Tang Capital Partners, Vivo Capital.

Fund	Shares	Q/Q change	% of portfolio	Filing
RTW Investments	18,037,772	no change	0.52%	2026-08-14
ADAR1 Capital Management	435,500	+3,000	0.07%	2026-08-14
Tang Capital Partners	100,000	no change	0.02%	2026-08-14

Specialist-fund positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.

Latest news

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RCKT — Rocket Pharmaceuticals Inc. ☆

What they do

Balance sheet

Share structure

Price history

Next catalyst

Upcoming catalysts

Pipeline assets

Pipeline

Active clinical trials

Specialist fund holders

Latest news

RCKT — Rocket Pharmaceuticals Inc.