Orchard Therapeutics, a global gene therapy leader, announced that six presentations from its hematopoietic stem cell (HSC) gene therapy platform will be featured at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles from May 16-20, 2023.
The presentations will include updated results from the company’s ongoing proof-of-concept (PoC) studies of OTL-201 in mucopolysaccharidosis type IIIA (MPS-IIIA) and OTL-203 in mucopolysaccharidosis type I Hurler (MPS-IH), as well as abstracts demonstrating the therapeutic potential of HSC gene therapy for genetic sub-types of frontotemporal dementia (FTD) and Crohn’s disease, and chronic autoimmune disorders.
Dr. Leslie Meltzer, chief medical officer of Orchard Therapeutics, expressed optimism in the transformative impact and broad applicability of their platform. The data from their MPS programs continue to show potential for a one-time HSC gene therapy to address severe manifestations of these multi-system diseases.
The company is also applying its differentiated delivery mechanism to advance research in difficult-to-treat diseases like NOD2-Crohn’s disease.
Orchard Therapeutics aims to end the devastation caused by genetic and other severe diseases by discovering, developing, and commercializing new treatments using HSC gene therapy.
In this approach, a patient’s own blood stem cells are genetically modified outside of the body and reinserted to correct the underlying cause of the disease in a single treatment.