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SCIENTIFIC FOUNDERS

David A. Williams

Dr. Williams is a practicing Pediatric Hematologist/Oncologist and Chief Scientific Officer/Senior Vice-President at Boston Children’s Hospital. An internationally renowned researcher, Dr. Williams' work focuses on blood stem cell biology, leukemia, and gene therapy to correct genetic blood disorders. He has won numerous prestigious awards for his research, including: the E. Mead Johnson Award for Research in Pediatrics; the William Dameshek Award of the American Society of Hematology; the Samuel Rosenthal Prize for Excellence in Pediatrics; the Frank Oski Award of the Society of Hematology; the Donald Metcalf Award from the International Society of Experimental Hematology.  He is the recipient of the American Society of Gene and Cell Therapy’s Outstanding Achievement Award for his work in gene therapy in 2012. Dr. Williams has recently served as President of the American Society of Hematology.  Dr. Williams was a Howard Hughes Medical Institute Investigator for 16 years, is currently a member of the National Academy of Medicine, and is a Fellow of the American Association for the Advancement of Science.  His laboratory has been continuously funded by the NIH since 1986. During his fellowship research at the MIT Cancer Center and the Whitehead Institute, he developed techniques that allowed for the introduction of genes into murine and human hematopoietic stem and progenitor cells. Those techniques are still commonly utilized today. Dr. Williams has multiple patents, several of which have been licensed to pharmaceutical and biotechnology firms. 

Axel Schambach

Axel Schambach, MD, PhD, is Director of the Institute of Experimental Hematology and Professor for Gene Modification of Somatic Cells (W3) at Hannover Medical School (MHH). In addition, he holds a 2nd appointment as adjunct faculty and lecturer at Boston Children’s Hospital at Harvard Medical School. He studied medicine in Hamburg, San Diego, San Francisco, Dallas and Zürich received his PhD in Molecular Medicine. He is a group leader in the Excellence cluster REBIRTH since 2007 and led (together with Prof. Duanqing Pei) a German-Chinese research group funded by DAAD and BMBF. His main research interest is to understand the molecular pathophysiology of inborn and acquired diseases to develop tailor-made gene therapy and molecular medicine strategies. Here he focuses on novel gene delivery tools, including state-of-the-art integrating vectors and designer nucleases as well as new reprogramming and transdifferentiation strategies and stem cell-based disease modeling. He has authored and co-authored more than 220 PubMed listed articles in respected journals, such as New England Journal of Medicine, Nature, Nature Medicine, Cell and Cell Stem Cells.