NEW YORK, June 9, 2026
Cellectis announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to lasmecabtagene timgedleucel (lasme-cel), its CD22-targeting allogeneic CAR-T cell therapy, for the treatment of patients with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). The designation recognizes the therapy’s potential to address a significant unmet medical need and supports accelerated interactions with the FDA as the program advances through pivotal clinical development.
RMAT Designation Supported by Promising Phase 1 BALLI-01 Results
The RMAT designation was granted based on encouraging data from the Phase 1 BALLI-01 clinical trial, which demonstrated promising anti-leukemia activity alongside a manageable safety profile in patients with relapsed or refractory B-ALL. Final Phase 1 results will be presented during an oral session at the 2026 European Hematology Association (EHA) Congress, highlighting the growing clinical evidence supporting lasme-cel as a potential treatment option for patients with limited therapeutic alternatives.
First Allogeneic CAR-T Therapy Advancing Through a Pivotal Trial in B-ALL
Lasme-cel is a CD22-targeting allogeneic CAR-T therapy developed using Cellectis’ gene-editing platform and represents one of the most advanced off-the-shelf CAR-T programs in hematologic malignancies. Unlike autologous CAR-T therapies that require patient-specific manufacturing, allogeneic CAR-T therapies are designed to be readily available for immediate treatment. The ongoing Phase 2 pivotal portion of the BALLI-01 study is currently enrolling patients and is intended to further evaluate the efficacy and safety of lasme-cel in relapsed or refractory B-ALL.
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Off-the-Shelf CAR-T Approach Aims to Address Urgent Treatment Needs
Cellectis believes the RMAT designation validates the potential value of readily available allogeneic CAR-T therapies for patients who cannot afford delays associated with individualized cell therapy manufacturing. The company stated that the designation strengthens regulatory engagement as it advances lasme-cel through late-stage development. If successful, lasme-cel could become a novel treatment option for patients with relapsed or refractory B-cell acute lymphoblastic leukemia, a life-threatening blood cancer with substantial unmet medical need.
Source: Cellectis press release
