by Peter Ciszewski | Sep 16, 2019
Bobby Gaspar, MD, chief scientific officer at Orchard Therapeutics describes the origins of the company and his involvement. Dr..Gaspar is a professor of paediatrics and immunology at the UCL Great Ormond Street Institute of Child Health in London, UK. “As an academic...
by Peter Ciszewski | Sep 16, 2019
Sangamo is a genomic medicine company that is starting to develop gene therapies for a number of conditions. Sandy Macrae, PhD, Chief Executive Officer of Sangamo talks about the work they are currently doing to develop a gene therapy for hemophilia A. While great...
by Peter Ciszewski | Sep 13, 2019
Heather A. Lau, MD, Assistant Professor, Department of Neurology; Associate Director, Division of Neurogenetics; Director, Lysosomal Storage Disease Program at NYU Langone Health discusses the differential diagnosis of Mucopolysaccharidosis type I (MPS I), a...
by Peter Ciszewski | Sep 12, 2019
Sanjeev Luther, president and chief executive officer (CEO) of Rafael Pharmaceuticals talks about his company’s lead compound, PI-613 (devimistat). PI-613 targets enzymes involved in cancer cell energy metabolism located in the mitochondria of cancer cells....
by Peter Ciszewski | Sep 10, 2019
Nick Kenny, PhD, Chief Scientific Officer at Syntheos Health discusses his company’s efforts to streamline data management for clinical trials, including the need to determine the value that a new drug will have on the health care system. Dr. Kenny said,...
