By Claude Leguilloux Published on 02/08/2024 at 07:01
Photo credit © Cellectis – Ramon Martinez
(Boursier.com) — Cellectis a clinical-stage biotechnology company using its pioneering TALEN genome editing technology to develop potential innovative therapies for serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Cellectis’ CLLS52 (alemtuzumab), an investigational medicinal product (IMP) used in the lymphodepletion regimen in combination with its UCART22 product candidate, being evaluated in the BALLI-01 clinical trial in relapsed or refractory B-cell acute lymphoblastic leukemia (ALL).
“We are pleased that the FDA has granted CLLS52 (alemtuzumab) ODD designation. As previously highlighted, the importance of alemtuzumab in the lymphodepletion regimen was demonstrated in our BALLI-01 clinical trial, where the addition of this lymphodepleting agent to the fludarabine and cyclophosphamide regimen was associated with sustained lymphodepletion and significantly greater UCART22 cell expansion, leading to greater clinical activity,” said Mark Frattini, MD, PhD, Chief Medical Officer of Cellectis.
Cellectis is the inventor of the combination of CD52 knockout UCART with a lymphodepletion regimen including an anti-CD52 antibody such as alemtuzumab. The CD52 knockout renders the UCART product candidate resistant to alemtuzumab. Lymphodepletion of patients, in turn, reduces host immune cells and is expected to improve the expansion and persistence of allogeneic CAR T cells. CD52 knockout in the UCART22 product candidate is achieved using TALEN genome editing technology.
The FDA grants ODD status to drugs intended for the treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. Obtaining ODD status can help expedite and reduce the cost of developing, approving, and commercializing a therapeutic agent.
©2024 Boursier.com
