Cranbury, New Jersey-based gene therapy company Rocket Pharmaceuticals made considerable headway in 2023, advancing multiple first-in-class rare-disease treatments through promising clinical trials.
Its RP-A501 investigational gene therapy aims to treat patients with Danon Disease, a rare and fatal genetic heart disorder affecting roughly 45,000 people in the U.S. and Europe whose only current treatment is a heart transplant. In October 2023, Rocket launched a global Phase 2 clinical study in males with the disease.
Also in October, the FDA granted Priority Review (with a March 2024 decision date) for Rocket’s RP-L201, a treatment for severe LAD-1—an immunodeficiency that results in recurrent infections—after reporting 100% overall survival at 12 months post-infusion in Phase 2 trials and significant decreases in the incidence of infections.
Those efforts followed the May 2023 FDA clearance it received to start Phase 1 trials of the first clinical gene therapy program for PKP2-ACM—an inherited heart disease affecting approximately 50,000 people in the U.S. and E.U.—as well as Regenerative Medicine Advanced Therapy (RMAT) designation for an investigational gene therapy for rare blood disorder pyruvate kinase deficiency.
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