Rocket Pharma partners with Stanford University to advance FA and PKD gene therapy research

Rocket Pharmaceuticals, Inc. ("Rocket"), a leading U.S.-based multi-platform gene therapy company, and the Stanford University School of Medicine today announced a strategic collaboration to support the advancement of Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD) gene therapy research. Rocket's lentiviral vector (LVV)-based gene therapy program for FA is currently in clinical trials with academic partners in the U.S. and Europe. The LVV-based gene therapy program for PKD is currently in preclinical development in Europe.

Under the terms of the agreement, Stanford will serve as a lead clinical trial research center in the United States for a planned upcoming registrational trial for FA, and would also be the lead site for PKD clinical trials. Maria Grazia Roncarolo, M.D., director of the Stanford Center for Definitive and Curative Medicine and co-director of the school's Institute for Stem Cell Biology and Regenerative Medicine, will lead the school's effort. The center is a joint initiative of the School of Medicine, Stanford Health Care and Stanford Children's Health and is focused on bench to bedside development of innovative cell- and gene-based therapies.

Gaurav Shah, M.D., Chief Executive Officer and President of Rocket, commented, "Rocket is delighted to expand the reach of our gene therapy program in the U.S. and prepare for our registrational trial. We are committed to developing FA and PKD programs in collaboration with outstanding gene therapy centers and pioneers in the field. This collaboration with the Stanford Center for Definitive and Curative Medicine is a critical step within our overall strategy of building relationships with gene therapy experts, with investigators who have dedicated their careers to improving the care of patients afflicted with these disorders, and within the broader FA and PKD communities."

"This project will also evaluate the introduction of conditioning regimens for both FA and PKD, where we hope to develop best-in-class gene therapy approaches for both clinical indications. The regulatory design and preparation for our registrational trial for FA is ongoing and we remain on track to advance this program to a registration study in 2019. Our PKD program continues to advance in preclinical studies with an Investigational Medicinal Product Dossier (IMPD) expected to be filed in early 2019," continued Dr. Shah.

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