Cellectis Achieves 100% ORR with Lasme-cel in Phase 1 BALLI-01 Trial, Advances to Pivotal Phase 2

Cellectis's BALLI-01 Phase 1 data, presented at EHA 2026, signal an imminent scale-up demand for allogeneic CAR-T manufacturing infrastructure, with a Pivotal Phase 2 now open for recruitment and a first interim analysis targeted for Q4 2026. For QA directors and manufacturing leads at cell therapy facilities, the transition from Phase 1 to a pivotal study compresses the timeline for GMP readiness, batch consistency validation, and release testing protocols.

In the target Phase 2 population of the BALLI-01 study, lasme-cel, a CD22-directed allogeneic CAR-T, achieved an overall response rate (ORR) of 100% (7/7) in patients with relapsed/refractory B-cell acute lymphoblastic leukemia. Complete remission or complete remission with incomplete count recovery (CR/CRi) was recorded in 57% of patients, with 75% of those achieving MRD-negative status. All seven patients subsequently proceeded to hematopoietic stem cell transplantation (HSCT). The 45-patient cohort was heavily pretreated, with the target Phase 2 population carrying a median of five prior lines of therapy.

The safety profile carries direct relevance for clinical manufacturing and pharmacovigilance teams. Cytokine release syndrome (CRS) grade 3 or above occurred in 4% of patients; ICANS grade 3 or above in 4%; and IEC-HS grade 3 or above in 2%. All events resolved. For process development teams, these low severe-toxicity rates will inform the product's comparability criteria as manufacturing scales from Phase 1 lot sizes to pivotal-study volumes, a transition where 21 CFR Part 211 and ICH Q10 quality system requirements apply with full force.

Separately, Cellectis presented preliminary data from the NATHALI-01 study evaluating eti-cel, a dual CD20/CD22 allogeneic CAR-T, in relapsed/refractory B-cell non-Hodgkin lymphoma. As of the February 2026 data cutoff, 14 patients had been treated. The poster focused on the role of alemtuzumab in optimizing responses, a lymphodepletion variable that manufacturing and clinical operations teams will need to account for in process characterization and comparability assessments as the program matures.

The off-the-shelf nature of both products underscores the manufacturing differentiation Cellectis is pursuing: allogeneic platforms require robust donor-derived starting material controls, cryopreservation validation, and supply-chain redundancy that autologous programs do not. As BALLI-01 moves into its pivotal phase, the facility and quality systems supporting lasme-cel production will face the same scrutiny applied to any product approaching a BLA or MAA submission.

The first interim analysis of the Pivotal Phase 2 BALLI-01 trial (NCT04150497) is expected in Q4 2026, providing the earliest dataset against which manufacturing consistency and clinical outcomes can be jointly assessed.

Source: Cellectis press release via cellectis.com, June 11, 2026.