Phase 1 Study of Base Edited CAR T Cells Against AML: Deep Conditioning Ahead of Allogeneic Stem Cell Transplantation
The BE CAR-33 asset represents a novel approach in the treatment of relapsed acute myeloid leukaemia (AML) in pediatric patients, utilizing base editing technology to enhance CAR T cell efficacy while minimizing off-target effects. The market for AML therapies is growing, driven by the increasing incidence of the disease and the demand for innovative treatments. Competitive implications include potential differentiation from existing CAR T therapies through its unique mechanism of action and safety profile. Diligence considerations should focus on the long-term follow-up data and the scalability of manufacturing the base-edited CAR T cells, as well as regulatory pathways given the innovative nature of the technology.
Indication: Relapsed Acute Myeloid Leukaemia
Modality: protein therapy
Target: CD33+ leukaemia cells
Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust
Source URL: ClinicalTrials.gov
Source updated: Detailed source ingestion pending
Ingested: Jun 24, 2026
Model: trialsignal-ai-v1
Validation: validated
Matched by target_normalized: CD33+ leukaemia cells
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Condition raw: Relapsed Acute Myeloid Leukaemia
Condition normalized: Relapsed Acute Myeloid Leukaemia
Modality raw: protein therapy
Modality normalized: protein therapy
Target raw: CD33+ leukaemia cells
Target normalized: CD33+ leukaemia cells