CDCM Announces Strategic Research Collaboration


The Center for Definitive and Curative Medicine (CDCM) at Stanford Medicine has entered into a collaborative research agreement with Rocket Pharmaceuticals, Inc. ( to further the CDCM’s mission of curing the seemingly incurable. The agreement outlines how the two entities will collaborate in their shared mission to advance gene therapy research, with Stanford named as the lead US-based clinical trial research center for two programs targeting rare orphan diseases: Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD).

The CDCM is a joint initiative of the Stanford University School of Medicine, Stanford Health Care and Stanford Children’s Health. It is co-directed by Drs. Maria Grazia Roncarolo, Anthony Oro, and Matthew Porteus. The CDCM brings together a consortium of high-impact principal investigators across therapeutic areas with translational capabilities to meaningfully change the paradigm of translational research in personalized cell and gene therapy. The CDCM is focused on bench to bedside development of innovative therapies for children and adults with genetic and other complex devastating diseases.

Previous work led by Maria Grazia Roncarolo’s team in Milan showed that lentiviral vector stem cell gene therapy is effective in curing genetic diseases of the blood and metabolism. The Stanford Medicine and Rocket collaboration aims to use Rocket’s lentiviral vector platforms to develop lentiviral vector-based gene therapy for the treatment of FA, a genetic disease that leads to bone marrow failure and potentially cancer, and PKD, an inherited metabolic disorder with high unmet medical need.