Rocket Pharmaceuticals

What Rocket Pharmaceuticals does

Rocket Pharmaceuticals is a clinical-to-commercial-stage biotechnology company headquartered in Cranbury, New Jersey, that develops gene therapies for rare pediatric genetic disorders using two proprietary technology platforms: an in vivo adeno-associated viral (AAV) platform using AAV9 and AAVrh74 serotype vectors targeting cardiac, hepatic, ocular, and CNS tissues, and an ex vivo lentiviral platform that modifies patient-derived hematopoietic stem cells (HSCs) to address bone marrow-derived disorders. Its commercial product KRESLADI (marnetegragene autotemcel) received FDA accelerated approval in March 2026 for severe leukocyte adhesion deficiency-I (LAD-I) in pediatric patients, representing the company's first approved gene therapy; commercial launch is planned for late 2026 through a controlled rollout to specialized treatment centers. The clinical pipeline includes RP-A501 (AAV9, Danon disease, Phase 2 pivotal), RP-A601 (AAVrh74, PKP2-arrhythmogenic cardiomyopathy, Phase 1 with RMAT designation), RP-A701 (AAVrh74, BAG3-associated dilated cardiomyopathy, Phase 1 IND-cleared August 2025), plus lentiviral programs RP-L102 (Fanconi Anemia, BLA withdrawn October 2025) and RP-L301 (Pyruvate Kinase Deficiency).

The company operates its own ~103,720 sq ft cGMP manufacturing facility in Cranbury, NJ (constructed 2022), with roughly half dedicated to AAV manufacturing, providing direct control over a critical bottleneck in gene therapy supply. Revenue mechanics are built around ultra-rare, one-time gene therapy treatments administered at a limited set of qualified treatment centers (UC San Diego Health, Children's Hospital of Philadelphia, Great Ormond Street Hospital, German Heart Center Munich, Duke, Mayo, Medical University of South Carolina), supplemented by a one-time $180M sale of a Rare Pediatric Disease Priority Review Voucher in June 2026, a $100M at-the-market (ATM) equity facility with Cantor Fitzgerald, and additional public equity offerings ($100.3M in October 2022, $175M in September 2023). Pro forma cash stands at approximately $322.6M, supporting operations into Q2 2028. Following a 30% workforce reduction in 2025 to extend runway, the company is led by CEO Gaurav Shah (former Novartis Cell & Gene Therapies Unit Global Program Head) and is publicly traded on NASDAQ under ticker RCKT with a market capitalization of approximately $290.45M as of June 2026.

Name

Rocket Pharmaceuticals

Legal name

Rocket Pharmaceuticals, Inc.

Website

http://www.rocketpharma.com/

Company type

Public

Founded year

2015

Operating status

Operating

Headcount range

101–250 employees

Short description

Rocket Pharmaceuticals is a clinical-to-commercial biotech developing AAV and lentiviral gene therapies for rare pediatric genetic disorders, with FDA-approved KRESLADI for severe LAD-I and a cardiovascular pipeline targeting Danon disease, PKP2-ACM, and BAG3-DCM, administered through specialized treatment centers.

Ownership category

akta.pro rank

Product category

Gene Therapy for Rare Genetic Diseases

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714), Pharmaceutical and Medicine Manufacturing (3254)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro primary industry

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

akta.pro secondary industries

Rare Cardiovascular & Vascular Disorder Therapies (HLAIAIAH), Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN)

Keywords

• Gene therapy
• Rare disease treatments
• Adeno-associated viral vectors
• Lentiviral gene therapy
• Cardiac gene therapy

Headquarters

HQ city

Cranbury

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Infrastructure, Marketing or Sales, Supply Chain

Revenue model

1. KRESLADI Product Sales: Rocket Pharmaceuticals launched its first commercial product KRESLADI (marnetegragene autotemcel) in March 2026 for severe leukocyte adhesion deficiency-I (LAD-I) in pediatric patients. The company is implementing a measured commercial launch beginning in late 2026, with revenue expected to begin in 2027. The therapy is a one-time gene therapy treatment for an ultra-rare genetic immunodeficiency.
2. At-the-Market (ATM) Equity Offering: The company entered into a $100 million ATM equity offering agreement with Cantor Fitzgerald, allowing it to sell shares of common stock at its discretion through various trading venues. This provides ongoing access to equity capital markets.
3. Priority Review Voucher Monetization: Rocket Pharmaceuticals received a Rare Pediatric Disease Priority Review Voucher upon FDA approval of KRESLADI in March 2026, which was subsequently sold for $180 million in non-dilutive capital in June 2026. This represents a one-time non-dilutive revenue stream from the FDA's rare pediatric disease incentive program.
4. Public Equity Offerings: The company has raised capital through multiple public offerings of common stock and pre-funded warrants. In September 2023, a $175 million public offering closed. In October 2022, a $100.3 million offering (6,800,000 shares at $14.75/share) was completed.

Pricing tiers

Go-to-market motion3 records

Distribution channels2 records

Marketing channels5 records

Core offering

Rocket Pharmaceuticals develops and commercializes one-time, potentially curative gene therapies for rare pediatric genetic disorders using two proprietary platforms: an in vivo AAV platform (AAV9, AAVrh74 serotypes) targeting cardiac diseases and an ex vivo lentiviral platform modifying hematopoietic stem cells to address bone marrow-derived disorders. Its commercial product KRESLADI (marnetegragene autotemcel) received FDA accelerated approval in March 2026 for severe leukocyte adhesion deficiency-I (LAD-I), with a clinical pipeline spanning Danon disease, PKP2-ACM, BAG3-DCM, Fanconi Anemia, and Pyruvate Kinase Deficiency.

Product overview

Rocket Pharmaceuticals is a late-stage biopharmaceutical company developing gene therapies for rare genetic diseases using two technology platforms: in vivo adeno-associated viral (AAV) vectors and ex vivo lentiviral (LV) vectors. The company's commercial product is KRESLADI (marnetegragene autotemcel), FDA-approved in March 2026 for severe leukocyte adhesion deficiency-I (LAD-I), representing Rocket's first approved gene therapy. The clinical pipeline includes: RP-A501 (AAV9-based for Danon disease), RP-A601 (AAVrh74-based for PKP2-arrhythmogenic cardiomyopathy), and RP-A701 (AAVrh74-based for BAG3-associated dilated cardiomyopathy) on the AAV platform; and RP-L102 (for Fanconi Anemia) and RP-L301 (for Pyruvate Kinase Deficiency) on the lentiviral platform. The company operates its own manufacturing facility in Cranbury, New Jersey. Beyond therapy development, Rocket supports patients through the Mission: Genome no-charge genetic testing and counseling program.

Differentiator

Problem solved

Functional benefit

Brands

• KRESLADI: FDA-approved gene therapy product (marnetegragene autotemcel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I) in pediatric patients.

Products and services

• KRESLADI (marnetegragene autotemcel) FDA-approved ex vivo lentiviral gene therapy consisting of autologous hematopoietic stem cells genetically modified with a lentiviral vector to contain a functional copy of the ITGB2 gene encoding CD18. Indicated for severe leukocyte adhesion deficiency-I (LAD-I) in pediatric patients; first FDA-approved gene therapy for severe LAD-I, with clinical trial data showing 100% overall survival at 12 months post-infusion across 9 treated patients.
• RP-A501 Investigational in vivo AAV9-based gene therapy for Danon disease, a rare genetic disorder characterized by severe hypertrophic cardiomyopathy, skeletal muscle weakness, and cognitive impairment. Delivers a functional version of the human LAMP2B transgene as a single IV infusion; AAV9 capsid has demonstrated particular propensity for heart muscle cells. Rocket is the first company to demonstrate safety and efficacy data in clinical studies for gene therapy targeting the heart.
• RP-A601 Investigational AAVrh74-based gene therapy for PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM/ARVC/ARVD), an inherited heart disease caused by PKP2 gene mutations associated with life-threatening arrhythmias and sudden cardiac death. Delivers a functional PKP2 transgene using AAVrh74 capsid technology with cardiac tropism.
• RP-A701 Investigational AAVrh74-based gene therapy for BAG3-associated Dilated Cardiomyopathy (BAG3-DCM), an inherited heart disease caused by mutations in the BAG3 gene. Delivers a functional BAG3 transgene using AAVrh74 capsid technology; targeting a severe heart failure condition.
• RP-L102 Investigational ex vivo lentiviral gene therapy for Fanconi Anemia (FA) subtype A (FA-A), a rare genetic disorder affecting DNA repair and characterized by bone marrow failure and cancer predisposition. Contains autologous patient-derived hematopoietic stem cells modified with a lentiviral vector carrying the FANCA gene.
• RP-L301 Investigational ex vivo lentiviral gene therapy for Pyruvate Kinase Deficiency (PKD), a rare genetic blood disorder characterized by excessive rupture of red blood cells. Contains autologous patient-derived hematopoietic stem cells modified with a lentiviral vector carrying the PKLR gene.

Quantifiable outcome

• 100% overall survival at 12 months post-infusion for LAD-I patients treated with KRESLADI in Phase 1/2 trials (all 9 patients survived without requiring subsequent allogeneic HSCT)
• +4 more outcomes

Named customers1 record

Segments3 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

Ten partnerships are on record, tiered minor and core.

• American Biotech Innovation Alliance (ABIA)minorStrategic or Co-development Partner · 1 May 2026ABIA is a Washington D.C.-based industry organization founded to address concerns about China's growing prominence in biotech innovation and to create a coordinated national strategy for America's biotech sector. Rocket Pharmaceuticals is among approximately 30 companies (including Moderna) that have joined the alliance, with the goal of growing membership to 50 and developing a 'Biotech Vision 2030' strategy.
• RenovacorcoreStrategic or Co-development Partner · 1 December 2022Rocket Pharmaceuticals completed the acquisition of Renovacor on December 1, 2022, gaining access to an advanced program targeting BAG3-associated dilated cardiomyopathy (a severe heart failure condition) along with other scientific assets. The all-stock acquisition strengthened Rocket's leadership in AAV-based gene therapy for cardiac diseases and expanded its clinical asset portfolio.
• UC San Diego HealthcoreStrategic or Co-development PartnerClinical trial site for multiple Rocket programs including the global Phase 2 pivotal trial of RP-A501 for Danon disease, the Phase 1 trial of RP-A601 for PKP2-ACM, and the Phase 1 trial of RP-A701 for BAG3-DCM. UC San Diego Health is a leading academic medical center in gene therapy research.
• Children's Hospital of Philadelphia (CHOP)coreStrategic or Co-development PartnerClinical trial site for the Phase 2 pivotal trial of RP-A501 for Danon disease and Phase 1 trial of RP-A601 for PKP2-ACM. CHOP is a leading pediatric hospital with a prominent gene therapy program.
• Great Ormond Street Hospital & UCL Institute of Cardiovascular Science (London)coreStrategic or Co-development PartnerInternational clinical trial site for the global Phase 2 pivotal trial of RP-A501 for Danon disease, providing European patient enrollment capability.
• German Heart Center MunichcoreStrategic or Co-development PartnerInternational clinical trial site for the Phase 2 pivotal trial of RP-A501 for Danon disease and natural history study, providing European patient enrollment capability.
• InvitaecoreStrategic or Co-development PartnerPartnership for the Invitae Unlock Cardiomyopathy and Arrhythmia testing program, providing no-charge genetic testing for patients with suspected genetic heart conditions. Rocket sponsors the program as part of its commitment to raising awareness of genetic testing programs that can shorten the diagnostic journey for rare disease patients.
• Rady Children's Institute for Genomic MedicinecoreStrategic or Co-development PartnerPartnership as founding member of the BeginNGS consortium, facilitating newborn screenings using rapid Whole Genome Sequencing (rWGS) to drive early diagnosis of rare genetic diseases. Rocket plays a leading role in advocating for early diagnosis of rare genetic diseases through this initiative.
• California Institute for Regenerative Medicine (CIRM)coreStrategic or Co-development PartnerCIRM invested nearly $5.9 million in the clinical trial for KRESLADI (LAD-I gene therapy) at UCLA, making KRESLADI the first therapy emerging from California taxpayer funding through CIRM to receive FDA approval. CIRM is California's state stem cell agency.
• Citizen HealthminorStrategic or Co-development PartnerPartnership to support drug development and long-term patient outcomes in PKP2 cardiomyopathy. Citizen Health is a patient advocacy and support organization for those affected by genetic heart conditions.

Scale indicators12 records

Recent moves10 records

Expansion highlights6 records

Emerging players

• Lexeo Therapeutics: Lexeo Therapeutics is a clinical-stage gene therapy company developing AAV-based therapies for cardiac diseases (including a Danon disease program) and other genetic conditions. Highly comparable to Rocket given overlapping cardiac gene therapy focus and similar platform technology.
• Tenaya Therapeutics: Tenaya Therapeutics develops gene therapies for genetic cardiomyopathies and heart failure, including PKP2-related arrhythmogenic cardiomyopathy. Directly comparable as another cardiac-focused gene therapy developer addressing overlapping indications.
• Regenxbio: Regenxbio is developing AAV gene therapies using its proprietary NAV platform for rare diseases including MPS I/II, Duchenne, and wet AMD. Comparable as an AAV platform gene therapy company with similar rare disease focus and platform licensing model.

Direct peers

• bluebird bio: bluebird bio commercializes lentiviral-based gene therapies (Lyfgenia, Skysona, Zynteglo) for ultra-rare genetic diseases. Comparable given the shared lentiviral ex vivo gene therapy platform and ultra-rare disease commercial model.
• uniQure: uniQure develops AAV-based gene therapies for rare diseases including hemophilia B (HEMGENIX, first FDA-approved gene therapy for hemophilia) and Huntington's disease. Comparable as a direct AAV gene therapy peer focused on rare monogenic disorders.
• Krystal Biotech: Krystal Biotech developed and commercializes VYJUVEK, the first FDA-approved topical gene therapy for a rare skin disease (DEB). Highly comparable given shared AAV-based platform, commercial-stage rare disease gene therapy focus, and similar headcount/scale.
• Sarepta Therapeutics: Sarepta Therapeutics develops and commercializes AAV-based gene therapies for Duchenne muscular dystrophy (Elevidys) and other rare neuromuscular diseases. Comparable as an AAV gene therapy leader with approved products for rare genetic disorders.
• Spark Therapeutics: Spark Therapeutics (acquired by Roche in 2019) pioneered FDA-approved AAV gene therapy (LUXTURNA for RPE65-mediated inherited retinal dystrophy). Comparable as an AAV gene therapy platform company targeting ultra-rare inherited disorders, now operating within Roche.

Broad incumbents

• BioMarin Pharmaceutical: BioMarin is a larger rare disease biopharma with AAV gene therapy (ROCTAVIAN for hemophilia A) and a broader enzyme replacement portfolio. Comparable as an established rare disease player with both gene therapy and traditional biologic offerings.
• CRISPR Therapeutics: CRISPR Therapeutics developed CASGEVY (with Vertex), the first FDA-approved CRISPR/Cas9 gene-edited therapy, for sickle cell disease and beta-thalassemia. Comparable as a gene therapy/ editing leader addressing rare genetic blood disorders through ex vivo cell modification.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• YouTubeyoutube.com/channel/UCfCtYzVk6MTCZoDV5A93Kmg
• Xx.com/RocketPharma
• LinkedInlinkedin.com/company/rocket-pharmaceuticals-ltd

Revenue estimate

Valuation estimate

Number of profiles

Profiles13 records

Subsidiaries1 record

Funding overview

Funding rounds14 records

Investors7 records

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M&A3 records

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Rocket Pharmaceuticals