Helen Heslop, MD
Helen Heslop is Professor of Medicine and Pediatrics at Baylor College of Medicine, and also is the Interim Director of the Center for Cell and Gene Therapy at Baylor College of Medicine, Houston Methodist Hospital and Texas Children’s Hospital. She is also Associate Director for Clinical Research at the Dan L Duncan Cancer Center. Dr Heslop is a physician scientist engaged in translational research focusing on adoptive immunotherapy with gene-modified effector cells, to improve hemopoietic stem cell transplantation and cancer therapy. Her initial studies were the first to demonstrate that antigen-specific cytotoxic T cells could eradicate an established malignancy and because the cells were genetically marked, she and her collaborators obtained definitive evidence of cell expansion, trafficking to tumor sites and decade-long persistence. Subsequent protocols have extended this approach to Hodgkin’s Disease, NHL and nasopharyngeal cancer. An additional focus in reconstituting antiviral immunity post transplant and she has led an NHLBI-funded multicenter trial of allogeneic multivirus specific T cells. She therefore has extensive experience in developing and conducting transplant studies and cell and gene therapy studies and currently holds 10 INDs and has also obtained orphan drug designation for her initial study.
She was a Doris Duke distinguished clinical research scientist and serves as Principal Investigator on several peer-reviewed research programs, including an NCI-funded program project grant (Enhancing T-Cell Therapy of Cancer) a Leukemia and Lymphoma Society Specialized Center of Research (SCOR) award (Immunotherapy of Lymphoma) and a SPORE in lymphoma from the NCI. She is also the principal investigator on an NHLBI-funded training grant in Cell and Gene Therapy. She is the current President of the Foundation for Accreditation of Cell Therapy (FACT) and a past president of the American Society for Blood and Marrow Transplantation.
Michele P. Calos, PhD
Michele Calos is currently Professor in the Department of Genetics at Stanford University School of Medicine, where she has focused on creating and utilizing innovative genetic engineering and gene delivery systems. Dr. Calos pursued undergraduate studies in Zoology at Oxford University, followed by a Ph.D. in Biochemistry and Molecular Biology at Harvard University, working with Nobel laureate Dr. Walter Gilbert. Dr. Calos did postdoctoral research at the Universite de Geneve in Geneva, Switzerland with Dr. Jeffrey H. Miller. Dr. Calos then moved to Stanford, where she became involved in gene therapy research. Dr. Calos served on the Cellular, Tissue, and Gene Therapies Advisory Committee of the U.S. Food and Drug Administration from 2004 – 2008 and has been active in the American Society of Gene and Cell Therapy, with continual committee assignments from 2003 to the present, including serving on the Board of Directors from 2007 – 2010 and chairing the Musculo-Skeletal Gene and Cell Therapy Committee and election to Vice President in 2016.
The Calos laboratory has developed a succession of vectors and technologies that are in widespread use for the safe and easy modification of mammalian genomes, including extrachromosomal replicating vectors and vectors for homologous and site-specific recombination. The lab created the 293T cell line that is widely used for production of viral vectors, as well as technologies that have been applied to genome editing in vitro and in vivo, construction of transgenic animals, and gene and stem cell therapy. The laboratory developed the phiC31 integrase system, one of the first methodologies for sequence-specific modification of mammalian genomes, and applied it for in vivo and in vitro modification of many tissues and organs. The Calos lab has also applied the power of its genome engineering systems to the creation and modification of stem cells for therapeutic uses, demonstrated the use of fC31 integrase for reprogramming adult cells into induced pluripotent stem cells and the use of site-specific recombinase, TALEN, and CRISPR/Cas9 systems for precise correction of patient-derived iPSC. The lab is currently creating novel non-viral cell and gene therapies for genetic disorders such as Duchenne muscular dystrophy and limb girdle muscular dystrophies 2B and 2D, with the mission to create effective, safe, convenient, and reasonably priced therapeutic options for patients.
Guangping Gao, PhD
During his 30 plus years of his scientific research career, Dr. Gao has primarily focused on molecular genetics and viral vector gene therapy of human diseases, encompassing disease gene cloning, causative mutation identification, pathomechanisms, animal modeling, novel viral vector discovery and engineering for in vivo gene delivery, vector biology, preclinical and clinical gene therapy product development, viral vector manufacturing for preclinical and clinical gene therapy applications as well as technology platforms development as novel approaches for gene therapy. More specifically, Dr. Gao’s current research areas include molecular mechanisms of AAV evolution and diversity, AAV capsid structure-tissue tropism-host response correlations, AAV capsid discovery, modifications and vector genome engineering for novel vector development, next generation recombinant AAV (rAAV) manufacturing and Quality Control testing pipeline development, gene silencing and gene editing for in vivo somatic gene therapy, molecular and immunological interactions between endogenous AAV, rAAV and host, rAAV vectored antibody and other therapeutic biologics production in vivo and minimization of anti-transgene responses, microRNA functional genomics and therapeutic development, nonhuman primate animal modeling for human genetic diseases, preclinical, translational and clinical development of rAAV gene therapy for CNS disorders such as Canavan disease, an inherited lethal white matter degenerative disorder.
Cynthia E. Dunbar, MD
Dr. Dunbar has pursued a career encompassing clinical investigation and patient care, basic science, and education/administration. As a translational research scientist, she has made important contributions to the science and translational development of gene therapy, stem cell biology, leukemogenesis and transplantation, focusing on large animals to provide insights that are not possible using murine or in vitro models. She has also led pioneering clinical trials in gene therapy, transplantation, autoimmune disease, and bone marrow failure.
Dr. Dunbar graduated from Harvard University with a degree in History of Science, and received her Doctor of Medicine from Harvard Medical School. Following internal medicine training at Boston City Hospital, she came to the National Institutes of Health (NIH) as a postdoctoral research fellow in the laboratory of Dr. Arthur Nienhuis, and following clinical training in hematology at University of California, San Francisco, she returned to the NIH to set up her own research program. She has been elected to the American Society for Clinical Investigation and the American College of Physicians. She is active in major professional societies, including service on the Executive Committee of the American Society of Hematology, and was a founding member and three-time Board member of the American Society for Cell and Gene Therapy (ASGCT) prior to her election as Vice-President in 2014. She served for ten years as an Associate Editor of the premier hematology journal BLOOD, and became the first woman to serve as BLOOD’s Editor-in-Chief 2007-2013. She has served on the Editorial Board of Molecular Therapy, and as head of the Publications Committee for ASGCT.
Dr. Dunbar is focused on the education and career development of physician-scientists, running the NIH fellowship program for 17 years and serving as the chair of the NIH ACGME committee. She is a faculty member for an international training program in translational medicine sponsored by the American and European gene therapy societies, and on the board of the NIH Clinical Research Training Program. She has been a leader in efforts to improve the NIH Intramural Research Program through her service on various committees and as a founding member and Co-Chair of the NIH Assembly of Scientists. She recently participated in the Executive Leadership in Academic Medicine program, and is active in efforts to improve representation of women and under-represented minorities in the top levels of academia. She has received numerous teaching and mentoring awards from NHLBI, NIH and her professional societies. She lives in Washington, DC with her husband and two daughters, and relaxes by performing and listening to classical music, hiking, and rowing.