Atla-cel, an autologous CD19-directed CAR-T cell therapy developed by BioOra, is moving into pediatric clinical development after BioOra entered a strategic collaboration with Cincinnati Children's Hospital Medical Center to evaluate the therapy in children and adolescents with relapsed or refractory B-cell acute lymphoblastic leukemia. Cincinnati Children's will lead and manage a global clinical programme enrolling patients across the US, New Zealand, and potentially Australia. The collaboration builds on adult clinical data from BioOra's ENABLE trial program, conducted with the Malaghan Institute of Medical Research in New Zealand.
Financial terms were not disclosed, consistent with an academic-industry clinical collaboration in which BioOra retains IP, manufacturing, and commercial rights while Cincinnati Children's contributes clinical infrastructure and trial management.
Deal context
Atla-cel (atlacabtagene autoleucel; pINN List 134) is a third-generation, autologous CAR-T therapy directed at CD19, a surface antigen expressed on malignant B cells. CD19-targeted CAR-T therapies are an established therapeutic class — Novartis's tisagenlecleucel was the first approved in paediatric B-ALL — but BioOra describes Atla-cel as incorporating design advances intended to improve anti-tumour activity and tolerability relative to earlier-generation constructs. That characterization is sourced from company materials and has not been independently validated in a randomized comparison.
Results from the Phase I ENABLE-1 study in adults with relapsed or refractory lymphoma, presented at the 2024 American Society of Hematology Annual Meeting, showed complete response rates alongside low rates of serious cytokine release syndrome and markedly reduced immune effector cell-associated neurotoxicity syndrome, or ICANS. In paediatric patients, ICANS carries heightened concern given the vulnerability of the developing brain to immune-mediated injury, and the observed adult safety signals form the primary scientific rationale for pediatric investigation.
Atla-cel is manufactured using the Cocoon automated closed-system platform, developed by Lonza and deployed at BioOra's purpose-built GMP facility in Christchurch, New Zealand. The Cocoon system integrates T cell enrichment, activation, viral transduction, and expansion within a single-use, functionally closed cassette, reducing operator handling and enabling operation in lower-grade cleanroom environments. BioOra's strategic positioning centres on this decentralised manufacturing model as a logistical and accessibility advantage over the centralised approaches used by first-generation approved CAR-T products.