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Rocket Pharmaceuticals, Inc.

Developer of gene-modified cell therapies designed to treat devastating orphan diseases

Rocket’s pipeline is comprised of first-in-class gene replacement strategies for rare and devastating inherited diseases. Designed in collaboration with leading academic and industry partners, Rocket gene-therapy programs aim to enable transduction of patients’ stem cells by means of 3rd-generation, self-inactivating lentiviral vectors to optimize the potential for gene-correction and stem cell engraftment such that the functional deficits of each disorder are corrected, with sufficient quantities of healthy protein manufactured by patients’ own hematopoietic cells. Each disease-program is intended to be transformative, enabling not only reversal of the disorder at molecular and cellular levels, but sustained relief from debilitating and potentially life-threatening symptoms.