Rocket Pharmaceuticals
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2015-01-01
- Employees
- 268
- Market Cap
- $1.7B
- Website
- http://www.rocketpharma.com
- Introduction
Rocket Pharmaceuticals, Inc. is a clinical-stage biotechnology company, which engages in the development of gene therapy treatment options for rare and devastating pediatric diseases. Its multi-platform development approach applies the lentiviral vector and adeno-associated viral vector gene therapy platforms. Its clinical program is an LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. The company was founded on July 7, 1999 and is headquartered in Cranbury, NJ.
Clinical Trials
29
Trial Phases
3 Phases
Drug Approvals
0
Drug Approvals
No drug approvals found
This company may not have drug approvals in our database
Clinical Trials
Distribution across different clinical trial phases (24 trials with phase data)• Click on a phase to view related trials
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
- Conditions
- Dilated Cardiomyopathy (DCM)
- First Posted Date
- 2025-08-22
- Last Posted Date
- 2025-08-22
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Target Recruit Count
- 8
- Registration Number
- NCT07137338
PKP2-ACM Natural History Study
- Conditions
- CardiomyopathiesHeart DiseasesCardiovascular DiseasesGenetic Diseases
- First Posted Date
- 2024-10-16
- Last Posted Date
- 2025-09-22
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Target Recruit Count
- 36
- Registration Number
- NCT06644742
- Locations
- 🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸Duke University, Durham, North Carolina, United States
🇳🇱Amsterdam UMC, Amsterdam, Netherlands
Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency
- Conditions
- Pyruvate Kinase Deficiency
- First Posted Date
- 2024-05-21
- Last Posted Date
- 2024-05-22
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Target Recruit Count
- 10
- Registration Number
- NCT06422351
- Locations
- 🇺🇸
Stanford University, Palo Alto, California, United States
🇪🇸Hospital Infantil Universitario Niño Jesús, Madrid, Spain
🇪🇸Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain
Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)
- Conditions
- Leukocyte Adhesion Deficiency
- First Posted Date
- 2024-02-28
- Last Posted Date
- 2024-02-28
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Target Recruit Count
- 9
- Registration Number
- NCT06282432
- Locations
- 🇺🇸
University of California, Los Angeles (UCLA), Los Angeles, California, United States
🇪🇸Hospital Infantil Universitario Niño Jesús, Madrid, Spain
🇬🇧University College London Great Ormond Street Institute of Child Health (GOSH), London, United Kingdom
Danon Disease Natural History Study
- Conditions
- Danon Disease
- First Posted Date
- 2024-01-19
- Last Posted Date
- 2025-10-10
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Target Recruit Count
- 46
- Registration Number
- NCT06214507
- Locations
- 🇺🇸
Phoenix Children's Hospital, Phoenix, Arizona, United States
🇺🇸University of California, San Diego, La Jolla, California, United States
🇺🇸Children's Hospital Colorado, Aurora, Colorado, United States
- Prev
- 1
- 2
- 3
- 4
- Next
News
Rocket Pharmaceuticals Withdraws FDA Application for Fanconi Anemia Gene Therapy RP-L102
Rocket Pharmaceuticals voluntarily withdrew its FDA Biologics License Application for RP-L102 (mozafancogene autotemcel), a gene therapy for Fanconi anemia, following a similar withdrawal in the EU in July 2025.
Rocket Pharmaceuticals Cuts 30% of Workforce, Refocuses on Cardiovascular Gene Therapies
Rocket Pharmaceuticals is laying off 80 employees, representing 30% of its workforce, to reduce cash burn by almost 25% and extend its runway into 2027.
AAV Gene Therapy Pipeline Expands with 180+ Companies Developing 250+ Treatments Despite Recent Setbacks
Over 180 companies are actively developing more than 250 AAV-based gene therapies, representing a robust pipeline for treating genetic disorders including hemophilia, spinal muscular atrophy, and inherited retinal diseases.
NodThera Appoints Former AstraZeneca Executive Elisabeth Björk to Board as Phase 2 Obesity Trial Advances
NodThera has appointed Elisabeth Björk, M.D., Ph.D., former Senior Vice President at AstraZeneca's obesity franchise, to its Board of Directors.
FDA Places Clinical Hold on Rocket Pharmaceuticals' Gene Therapy Trial After Patient Death
The FDA placed a clinical hold on Rocket Pharmaceuticals' Phase 2 pivotal trial for RP-A501, a gene therapy for Danon disease, following a patient death from a serious adverse event.
FDA Places Clinical Hold on Rocket Pharmaceuticals' Danon Disease Gene Therapy After Patient Death
The FDA has placed a clinical hold on Rocket Pharmaceuticals' Phase II gene therapy trial for RP-A501 after a patient developed capillary leak syndrome and subsequently died.
REGENXBIO Secures $250 Million Royalty Monetization Deal to Advance Gene Therapy Pipeline
• REGENXBIO has closed a non-dilutive royalty bond agreement with Healthcare Royalty for up to $250 million, receiving $150 million upfront that extends its cash runway into early 2027. • The deal monetizes select royalties from ZOLGENSMA for SMA and payments from gene therapies for MPS disorders, while REGENXBIO retains other funding opportunities including a potential Priority Review Voucher. • This strategic financing supports REGENXBIO's late-stage pipeline development, including RGX-121 for MPS II, RGX-202 for Duchenne muscular dystrophy, and ABBV-RGX-314 for wet AMD.
Rocket Pharmaceuticals to Present First Phase 1 Data for Gene Therapy in Rare Heart Condition at ASGCT
Rocket Pharmaceuticals will present preliminary data from its Phase 1 trial of RP-A601 for plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM) at the upcoming ASGCT Annual Meeting on May 15, 2025.
Rocket Pharmaceuticals Advances Gene Therapy Pipeline with Key Updates on Danon Disease and PKP2-ACM Programs
Rocket Pharmaceuticals continues its Phase 2 pivotal study of RP-A501 for Danon disease, with a program update expected mid-year 2025 and clinical data readout anticipated by mid-2026.
Gene Therapy Shows Promising Results in Children with Rare Immune Disorder LAD-I
An investigational gene therapy has successfully restored immune function in all nine children treated for severe leukocyte adhesion deficiency-I (LAD-I) in an international clinical trial co-led by UCLA.