Fresh Or Frozen? Orchard Aims For Cryopreserved Gene Therapy Approvals Using Pivotal Trials With Fresh Cell Formulations

Orchard aims to submit three ex vivo autologous gene therapies for approval by the end of 2021, helped by a new regenerative medicine advanced therapy designation (for a rare primary immunodeficiency syndrome) and a breakthrough therapy designation (for another rare primary immunodeficiency).