Our Pipeline

Current Portfolio

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.