Autolus Theraputics has announced that the
The drug, AUTO3, is “autologous enriched T-cells which are genetically modified with a retroviral vector to express two chimeric antigen receptors targeting CD19 and CD22 proteins.”
“We are pleased to receive orphan drug designation for AUTO3 for acute lymphoblastic leukaemia,” said Dr
He continued, “Earlier this year, we presented encouraging updated data from the AMELIA phase 1/2 trial of AUTO3 in paediatric ALL patients. We believe that AUTO3 has the potential to be a best in class therapy in paediatric ALL by addressing antigen escape, a common cause of relapse in these patients. AUTO3 may also provide an improved safety profile over currently marketed CART therapies with low levels of severe CRS and neurotoxicity observed in clinical studies.”
It is currently being tested in paediatric ALL in the AMELIA clinical trial, and in diffuse large B cell lymphoma in the ALEXANDER clinical trial.
Orphan drug designation is granted by the
There will be an estimated 5,930 new cases of ALL and an estimated 1,500 related deaths in 2019, with approximately 60% of cases occurring in patients younger than 20.