ResearchFocus on the pharmaceutical and biotech industry
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.
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...
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.
Accelerating the Road to Treatment: The Global Commission to End the Diagnostic Odyssey for Children
The Global Commission to End the Diagnostic Odyssey for Children, co-chaired by Takeda, Microsoft and EURORDIS-Rare Diseases Europe, is a multidisciplinary group of patient advocates, physicians and other experts working together to help solve the complex challenges impacting the rare disease community.
Daniel Barber is chief operating officer at Aquestive Therapeutics, a specialty pharmacy company that is highly focused on providing treatment options for persons unable to properly swallow medicine (e.g., people with epilepsy, ALS, Parkinson’s, etc).
Spark Therapeutics is developing a gene therapy to treat one of the most devastating genetic disease known – Huntington’s disease. A disease that robs people of their motor skills and psychological stability in the prime of their life.
Hideki Garren, MD, PhD, global head of neuroimmunology at Roche discusses neuromyelitis optica spectrum disease (NMOSD).