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.
Sanjeev Luther, president and chief executive officer (CEO) of Rafael Pharmaceuticals talks about his company’s lead compound, PI-613 (devimistat).
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.
Angelman syndrome is a rare neurodevelopmental disorder caused by a genetic mutation – usually a mutation in the ubiquitin protein ligase E3a (UBE3A) gene – that leads to a reduction in UBE3A protein.
Mark Rothera, CEO of Orchard Therapeutics, discusses his company, a fairly new gene therapy biotech that has a pipeline more appropriate for a bigger, more established company.