Rocket Pharmaceuticals, Inc.
NASDAQ Global MarketCapital Structure
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in mil. unless spec.Quarterly Revenue
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in mil. unless spec.Quarterly Earnings Per Share
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in mil. unless spec.Company Description
- CEO
- Gaurav D. Shah
- Full Time Employees
- 202
- Sector
- Healthcare
- Industry
- Biotechnology
- Address
- 9 Cedarbrook Drive Cranbury NJ United States of America 08512
- IPO Date
- Feb 18, 2015
- CIK
- 0001281895
- Website
- rocketpharma.com
- Similar Companies
- Business
- Rocket Pharmaceuticals, Inc. (RCKT) is a fully integrated, late-stage biotechnology company that develops gene therapies for rare and devastating pediatric diseases, employing lentiviral vector (LVV) and adeno-associated viral (AAV) platforms to target genetic disorders in hematopoietic stem cells, heart, liver, and other tissues. The company's pipeline includes late-stage LVV programs for KRESLADI (RP-L201) in severe leukocyte adhesion deficiency-I (LAD-I), Fanconi anemia (FA), and pyruvate kinase deficiency (PKD); and AAV programs for Danon disease (RP-A501), plakophilin-2 arrhythmogenic cardiomyopathy (PKP2-ACM, RP-A601), and BAG3 dilated cardiomyopathy (BAG3-DCM). It operates primarily in the United States, with research, development, and manufacturing facilities in Cranbury, New Jersey, where it was founded in 2015. In July 2025, Rocket announced a strategic corporate reorganization to prioritize its AAV cardiovascular platform, including Danon disease, PKP2-ACM, and BAG3-DCM, alongside regulatory efforts for KRESLADI following FDA acceptance of its BLA resubmission in October 2025; this refocuses resources on late-stage assets after a strategy outlined in January 2025. The company targets patient populations with high unmet needs in inherited cardiomyopathies and hematologic disorders, affecting over 100,000 patients in the U.S. and EU, and maintains partnerships with institutions like the University of California and Temple University to advance its therapies.
Company News
RCKT Aligns With FDA on Completing Phase II Study in Danon Disease
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Rocket Pharmaceuticals, Inc. (RCKT) Presents at Morgan Stanley 24th Annual Global Healthcare Conference Transcript
Rocket Pharmaceuticals, Inc. (RCKT) Presents at 12th Annual Cantor Fitzgerald Global Healthcare Conference Transcript
Rocket Pharmaceuticals Announces FDA Alignment on Path to Complete Pivotal Phase 2 Trial of RP-A501 in Danon Disease
Rocket Pharmaceuticals Announces FDA Alignment on Path to Complete Pivotal Phase 2 Trial of RP-A501 in Danon Disease
Rocket Pharmaceuticals (RCKT) Up 6% Since Last Earnings Report: Can It Continue?
Rocket Pharmaceuticals to Participate in Upcoming Investor Conferences
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) Given Average Recommendation of “Hold” by Analysts
Rocket Pharmaceuticals Q2 Earnings Rise Y/Y, Kresladi Launch in Focus
Rocket Pharmaceuticals Reports Second Quarter 2026 Financial Results and Highlights Recent Progress
Rocket Pharmaceuticals Touts Positive Early Safety Data from Revised Genetic Therapy Trial
Rocket Pharmaceuticals Announces Positive Clinical Safety Update from Initial Three Patients Treated with RP-A501 Under Modified Phase 2 Protocol for Danon Disease
Rocket Pharmaceuticals Announces Positive Clinical Safety Update from Initial Three Patients Treated with RP-A501 Under Modified Phase 2 Protocol for Danon Disease
Rocket Pharmaceuticals Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $180 Million
Rocket Pharmaceuticals, Inc. (RCKT) Presents at Goldman Sachs 47th Annual Global Healthcare Conference 2026 Transcript
Rocket Pharmaceuticals, Inc. (RCKT) Presents at Jefferies Global Healthcare Conference 2026 Transcript
Rocket Pharmaceuticals to Participate in Upcoming Investor Conferences
RCKT Stock Down on Q1 Earnings Miss, Kresladi Launch in Focus
Rocket Pharmaceuticals Reports First Quarter 2026 Financial Results and Highlights Recent Progress