It is a privilege to lead the center and to leverage my previous experience to build Stanfords preeminence in stem cell and gene therapies, said Roncarolo, who is also chief of pediatric stem cell transplantation and regenerative medicine, co-director of the Bass Center for Childhood Cancer and Blood Diseases and co-director of the Stanford Institute for Stem Cell Biology and Regenerative Medicine. Stanford Medicines unique environment brings together scientific discovery, translational medicine and clinical treatment. We will accelerate Stanfords fundamental discoveries toward novel stem cell and gene therapies to transform the field and to bring cures to hundreds of diseases affecting millions of children worldwide.

The center consists of several innovative pieces designed to allow the rapid development of early scientific discoveries into the clinic that in the past have languished. This includes an interdisciplinary team of basic and clinical scientists to shepherd nascent therapies developed at Stanford. The team will be headed by associate directors Matthew Porteus, PhD, associate professor of pediatrics, and Anthony Oro, MD, the Eugene and Gloria Bauer Professor and professor of dermatology.

To help with clinical development, the center boasts a dedicated stem cell clinical trial office with Sandeep Soni, MD, clinical associate professor of pediatrics, as medical director. In addition, the center has dedicated clinical trial hospital beds in the Bass Center for Childhood Cancer and Blood Diseases located on the top floor of the soon-to-open LucilePackardChildrensHospital. From work performed by scientists over the past decade, the center already has a backlog of nearly two dozen early stage therapies whose development the center will accelerate.

The center will provide novel therapies that can prevent irreversible damage in children, and allow them to live normal, healthy lives, said Mary Leonard, MD, professor and chair of pediatrics and physician-in-chief at Stanford Childrens Health. The stem cell and gene therapy efforts within the center are aligned with the strategic vision of the Department of Pediatrics and Stanfords precision health vision, where we go beyond simply providing treatment for children to instead cure them definitively for their entire lives.

One of the unique features of the center is its close association with the recently opened $35 million Stanford Laboratory for Cell and Gene Medicine, a 23,000-square-foot manufacturing facility located on California Avenue in Palo Alto. One of the first of its kind in the world, the laboratory has the ability to produce newly developed cell and gene therapy therapies according to the Good Manufacturing Practice standards as required for patient treatment.

Headed by executive director David DiGiusto, PhD, the lab can produce diverse cellular products for patient use, such as genetically corrected bone marrow cells for sickle cell anemia, genetically-engineered skin grafts for children with the genetic disease epidermolysis bullosa or genetically-engineered lymphocytes to fight rejection and leukemia.

We are fortunate that Stanford researchers have created such a strong portfolio of innovative candidate therapeutics to develop, said DiGusto. The capabilities of the laboratory will bridge the gap between research and clinical investigation so that the curative potential of these exciting cell and gene therapies can be realized.

For more information about the center, or for information about trials associated with the center, please see https://med.stanford.edu/ptrm/faculty.html, or contact Jennifer Howard at jmhoward@stanford.edu.

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Stanford announces new Center for Definitive and Curative Medicine - Stanford Medical Center Report