Small molecule gene therapy

Ariad Pharmaceuticals Inc. last week treated its first patient with a small molecule compound, AP1903, that is part of its ARGENT (Ariad Regulated Gene Expression Technology) system targeting graft-versus-host disease (GvHD) following bone marrow transplantation.

While many cell-based gene therapies require treatment of autologous cells and are more of a service than a product, ARIA is developing its gene therapy as a one-time injection that enables subsequent oral treatment with small molecules...