Reduced CAR T expansion post infusion is associated with poor survival in patients with large B cell lymphoma after two or more therapies.

CD19 directed chimeric antigen receptor (CAR) T-cell therapy is now standard of care for relapsed/refractory large B-cell non-Hodgkin lymphoma. Despite good overall response rates, many patients still experience disease progression and therefore it is important to predict those at risk of relapse following CAR T-cell therapy. We performed a prospective study using a flow cytometric assay at a single treatment centre to assess early CAR T-cell expansion in vivo 6 - 9 days after CAR-T cell infusion.

Delphi-driven consensus definition for mesenchymal stromal cells and clinical reporting guidelines for mesenchymal stromal cell-based therapeutics.

Background Aims: Despite promising results in pre-clinical studies, mesenchymal stromal cells (MSCs) face significant challenges in clinical translation. A scoping review by our group highlighted two key issues contributing to this gap: (i) lack of a clear and consensus definition for MSCs and (ii) under-reporting of critical parameters in MSC clinical studies. To address these issues, we conducted a modified Delphi study to establish and implement a consensus definition for MSCs and develop reporting guidelines for MSC clinical studies.

Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies.

Adeno-associated virus (AAV) vectors are promising gene therapy candidates, but pre-existing anti-AAV neutralizing antibodies (NAbs) pose a significant challenge to successful gene delivery. Knowledge of NAb seroprevalence remains limited and inconsistent. We measured activity of NAbs against six clinically relevant AAV serotypes across 10 countries in adults ( = 502) and children ( = 50) using a highly sensitive transduction inhibition assay.