Gene Therapy for Rare Disorders
Prof Bobby Gaspar
CEO, Orchard Therapeutics
Prof Bobby Gaspar is a world-renowned scientist and physician and accomplished strategic and organizational leader with more than 25 years of experience in medicine and biotechnology. As one of Orchard Therapeutics’ principal scientific founders, Bobby serves as Chief Executive Officer of the company, and also sits on Orchard’s Board of Directors. Bobby has been a pioneer in gene therapy and the evolution of hematopoietic stem cell (HSC) technology – including some of the first studies in patients with severe combined immunodeficiency (SCID) – bringing it from some of the first studies in patients to potential regulatory approvals. His unparalleled expertise and deep relationships with key physicians and treatment centers around the world are integral to Orchard’s efforts to identify patients with metachromatic leukodystrophy (MLD) and other diseases through targeted disease education, early diagnosis and comprehensive newborn screening.
Bobby is also an Honorary Professor of paediatrics and immunology at the UCL Great Ormond Street Institute of Child Health and has led multiple clinical trials that have shown that gene therapy can successfully correct the genetic defect in immune deficiencies. He studied medicine and surgery at Kings College in London before completing his Ph.D. at the UCL Institute of Child Health.
Prof Gaspar’s talk
The use of haematopoietic stem cell gene therapy for correction of specific monogenic rare disorders has made a long journey from early academic endeavours to licensed medicines. Lentiviral vector mediated gene therapy trials for ADA deficiency, Wiskott-Aldrich syndrome and Chronic Granulomatous disease for primary immunodeficiencies (PIDs) and for neurometabolic disorders such as metachromatic leukodystrophy (MLD), Hurlers syndrome (MPSI) and San Filippo A (MPSIIIA) are showing encouraging data and a favourable safety and tolerability profile. These are now progressing through clinical development pathways and most recently, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending authorisation for an investigational gene therapy for the treatment of MLD. The ongoing development of gene therapies, together with improvements in cell transduction techniques and optimised conditioning regimes, could mean that once approved, they may be amenable for a greater number of diseases to potentially become a widely used, standard treatment option.