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 physician, I was looking after children with very severe immune deficiencies and actually what we found was the children would either die of their disease or if there was a bone-marrow transplant then that could have side effects and they would die from some of the complications of that,” noted Dr. Gaspar. Understanding that using a bone marrow transplant on a patient who is immune compromised was a problem, Dr. Gaspar gravitated toward the concept of using a patient’s own stem cells to develop a gene therapy that would be a safer and more effective means to treat patients with severe immune deficiencies.
“With a colleague at UCL Great Ormond Street Hospital, we conducted some of the first trials to show that gene therapy could actually correct a monogenic disease and that was in these forms of severe combined immunodeficiency,” noted Dr. Gaspar. “That was a very powerful moment for us to see that effect and to see patients recovering from their disease through this gene therapy approach.”
Further studies confirmed that this approach was a safe and effective treatment option and the next step was to make it available to other conditions.
”We really wanted to take this out of the academic setting and to make it into something that could be used by children worldwide as an approved and standard medicine,” stated Dr. Gaspar. And to do that, they needed a commercial entity and that lead to the development of Orchard Therapeutics.
The company is currently focused on a few medical conditions, including primary immune deficiencies [e.g., adenosine deaminase severe combined immunodeficiency (ADA-SCID), Wiskott-Aldrich syndrome], neurometabolic disorders [e.g., metachromatic leukodystrophy (MLD)], and hemoglobinopathies [e.g., transfusion-dependent beta-thalassemia].
For more information, visit www.orchard-tx.com