Rocket Pharma’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 the patient’s own hematopoietic cells. Each disease-program is intended to be transformative, enabling not only the reversal of the disorder at molecular and cellular levels but sustained relief from debilitating and potentially life-threatening symptoms.
