Gene Therapy for Sensory Disorders



David R. Liu David R. Liu, PhD is the Richard Merkin Professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of MIT and Harvard, and vice chair of the faculty at Broad Institute. He is also Professor of Chemistry and Chemical Biology at Harvard University and a Howard Hughes Medical Institute investigator. He has received the Ronald Breslow Award for Biomimetic Chemistry, the American Chemical Society Pure Chemistry Award and the Arthur C. Cope Young Scholar Award. Dr. Liu was named one of the ten most influential scientists of 2017 by Nature. His major research interests include the engineering and delivery of genome editing proteins such as base editors to study and treat genetic diseases, the evolution of proteins with novel therapeutic potential using phage-assisted continuous evolution (PACE), and the discovery of bioactive synthetic small molecules and synthetic polymers through DNA-templated organic synthesis. He is the scientific founder or co-founder of Ensemble Therapeutics, Permeon Biologics, Editas Medicine, Pairwise Plants, and Beam Therapeutics.
Botond Roska Botond Roska, MD, Phd, received an MD in ​Budapest, Hungary, and a PhD at the University of California, Berkeley. He was a Junior Fellow in the Department of Genetics at Harvard Medical School and in the Department of Molecular and Cellular Biology​at Harvard University before starting his own research group at the Friedrich Miescher Institute for Biomedical Research in Basel, Switzerland. He has recently been named codirector of the new Institute of Molecular and Clinical Ophthalmology in Basel. Dr. Roska investigates neuronal circuits in the retina, thalamus and visual cortex; he is interested in using the knowledge gained to understand the circuit basis of neurological diseases and to design new therapies.
Katherine High Katherine High, MD, received her A.B. from Harvard University and an M.D. from the University of North Carolina School of Medicine. She has had a long-term interest in hereditary disorders and their treatment, which began with gene therapy for hemophilia and evolved to encompass potential gene therapies for a range of genetic diseases. She has been Director of the Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia and president of the American Society of Gene & Cell Therapy, and she served a term on the FDA Advisory Committee on Cell, Tissue and Gene Therapies. Dr. High was a member of the faculty at the University of Pennsylvania and of the medical staff at CHOP, and was an Investigator of the Howard Hughes Medical Institute. She now is President and Head of Research and Development at Spark Therapeutics, which developed Luxturna for treating blindness. In December, this became the first FDAapproved gene therapy for genetic disease, and the first AAV therapeutic approved in the United States.