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Rocket Ventures
Cranbury, New Jersey, United Statesrocketventures1.com ↗
- Ownership
- Private
- Employees
- ~80
- Development stage
- Other
01
Overview
Rocket Pharmaceuticals is a late-stage biotechnology company developing first-in-class gene therapies for rare genetic diseases. Its lead programs include AAV and LVV-based therapies targeting cardiovascular and hematologic disorders. The company has multiple programs in pivotal or late-stage trials, including RP-A501 for Danon disease, which recently experienced an FDA clinical hold following a patient death in Phase 2. Other programs remain on track, with regulatory milestones achieved in the US and Europe[5][8][9].
- Therapeutic areas
- ImmunologyCardiovascularRare DiseasesHematology
- Modalities
- Gene therapyAdeno-associated virus-based gene therapyLentiviral vector-based gene therapyBiologic (as a general category)
- Industry
- Biotech
02
Partnerships
RTW Investments [major investor/supporter]Multiple academic institutions via licensing agreements[6]
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