Autolus Therapeutics ALL treatment gets FDA orphan drug designation


Autolus Therapeutics’ (NASDAQ:AUTL) AUTO3 has received FDA orphan drug designation for the treatment of acute lymphoblastic leukemia (ALL).

AUTO3 is a T-cell therapy produced by genetically modifying a patient’s own T-cells to express two antigen receptors targeting B-cell malignancies such as ALL.

“We believe that AUTO3 has the potential to be a best in class therapy in pediatric ALL by addressing antigen escape, a common cause of relapse in these patients,” Dr. Christian Itin, Autolus’ chairman and CEO, said in a statement.

“AUTO3 may also provide an improved safety profile over currently marketed CAR T therapies with low levels of severe CRS [cytokine release syndrome] and neurotoxicity observed in clinical studies,” he added.

AUTO3 is being tested in pediatric ALL and diffuse large B-cell lymphoma in ongoing Phase 1/2 trials.

Michelle Carr