At the presentation of the financial results for the quarter and year ended 31 December 2021, Orchard Therapeutics CEO Bobby Gaspar, M.D., Ph.D., says that a promising early-stage research program that apply a hematopoietic stem cell (HSC) gene therapy approach in HAE will remain an important part of the portfolio going forward given the promise in larger indications and as a possible source of future partnerships.

In the HSC approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

“In light of our experiences and knowledge gained in this current and rapidly evolving market environment for gene therapy, our plan is to concentrate resources on programs that have the potential to make a remarkable difference to patients while also providing sustainable value to the business to enable the achievement our long-term vision,” says Bobby Gaspar.
(Source: Orchard)