Best-in-class curative gene & cell therapies for patients with devastating diseases. Rocket’s pipeline is comprised of first-in-class gene therapies for rare & 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 th...
Best-in-class curative gene & cell therapies for patients with devastating diseases. Rocket’s pipeline is comprised of first-in-class gene therapies for rare & 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 & 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.
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