Orphan Status for Cellectis Leukemia Therapy

FDA has granted Cellectis an orphan drug designation (ODD) for CLLS52 (alemtuzumab), an investigational therapy for treating relapsed/refractory B-cell acute lymphoblastic leukemia (ALL). “The importance of adding alemtuzumab to a lymphodepletion regimen has been demonstrated in Cellectis’ BALLI-01 study, where the addition of this lymphodepletion agent to the fludarabine and cyclophosphamide regimen was associated with sustained lymphodepletion and significantly higher UCART22 cell expansion allowing for greater clinical activity,” the company says.

Cellectis describes itself as the “inventor” of the CD52 knockout UCART cells combination with a lymphodepleting regimen containing an anti-CD52 antibody such as alemtuzumab. “The CD52 knockout aims to render the UCART product candidates resistant to alemtuzumab as part of the lymphodepleting regimen,” it says. “Cellectis’ UCART22 product candidate has the CD52 gene inactivated by TALEN gene editing technology.”