Our Pipeline

Current Portfolio

Rocket’s pipeline is comprised of first-in-class gene therapies for rare and devastating inherited diseases.

Designed in collaboration with leading academic and industry partners, Rocket lentiviral-based 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. The AAV-based program involves direct injection of the therapeutic vector, which has displayed substantial tropism for the organs most afflicted by the underlying disorder, ultimately enabling sufficient quantities of healthy protein to restore homeostasis.

Each 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.

pipeline-graph-201801-v4