Company intelligence / Profile preview

Rocket Pharmaceuticals, Inc.

Cranbury, New Jersey, United Statesrocketpharma.com ↗
Ownership
Public · RCKT
Employees
~150
Development stage
Phase 2
01

Overview

Advancing multiple gene therapy programs with positive Phase 1 results for RP-A501 for Danon Disease; initiated rolling BLA for RP-L102 for Fanconi Anemia; FDA review ongoing for KRESLADI for LAD-I; Phase 1 trial of RP-A601 for PKP2-ACM ongoing with data expected in first half of 2025

Therapeutic areas
HematologyImmunologyRare DiseasesCardiovascular
Modalities
Gene therapy (ex vivo lentiviral vector and in vivo adeno-associated viral vector)
Industry
Biotech
02

Drug pipeline

17 assets

marnetegragene autotemcel

Approved

16 more assets in the full pipeline.

03

Partnerships

Children's Hospital of Philadelphia (CHOP)Temple UniversityREGENXBIOMemorial Sloan Kettering Cancer CenterNYUCIRMUniversity of MinnesotaStanford Medical SchoolForty Seven (a Gilead company)University of ColoradoUCLAUCSDFred Hutchinson Cancer Research CenterCIBERCIBERERCIEMATUniversity College London Great Ormond Street Hospital for ChildrenLund UniversityMolMedUniversity of Hannover
04

Financials

Market cap
$267.3M
Total revenue
$0
Enterprise value
$8.4M

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