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Researchers at FDA workshop highlight advances and manufacturing, potency challenges for cell therapies
Summary
Keynote and technical sessions showcased rapid advances — pluripotent stem cells, organoids, extracellular vesicles — and stressed deep product characterization, robust potency assays and manufacturing scale‑up as prerequisites for safe clinical translation.
Speakers at an FDA workshop on March 3, 2025, framed recent scientific progress in cell and tissue‑based therapies alongside practical manufacturing and assay challenges that can slow patient access.
"Cell products have great potential to replace cells and restore function," said Dr. Sally Temple, scientific director of the Neural Stem Cell Institute, while reviewing a decade of progress in differentiation protocols, product characterization and manufacturing automation. She emphasized that products span a broad spectrum — some requiring minimal data, others extensive clinical and manufacturing evidence — and that funding remains a major barrier to moving candidates through clinical trials. (Dr. Sally Temple)
Presentations ranged from amniotic suspension allografts for osteoarthritis (Katie Mowry) to cord blood and mesenchymal stromal cell clinical uses (Dr. Amanda Olson) and neural stem cell‑derived extracellular vesicles (Steven). Industry and academic presenters converged on several technical challenges:
- Potency assays: Katie Mowry described selecting and validating a single predictive in‑vitro target ("target A") for lot release, then discovering its limitations outside the nominal dose range and adopting pooled positive controls to stabilize comparisons across lots.
- Characterization depth and assay sustainability: Panelists and CMC experts urged broad early characterization followed by a pragmatic set of validated lot‑release assays. "Early and extensive product characterization is prudent and pays off in the end," said Deb, a cell therapy consultant, stressing the need for reference materials and multiple release assays that reflect mechanism of action.
- Manufacturing and CMC: Dr. Jane Lebkowski used an RPE implant program to illustrate issues from starting‑material selection, substrate effects on cell phenotype, to cryopreservation and scaling of implant manufacture.
- Extracellular vesicles and novel modalities: Steven described AB126, a neural stem cell‑derived EV product with preclinical efficacy in rodent and pig stroke models and an IND‑enabling safety package; he also highlighted CMC challenges including impurity removal and up‑/downstream scaling.
Why this matters: Translational success depends on reproducible manufacturing and on potency and safety assays that predict clinical performance. Workshop speakers repeatedly warned that insufficient assay depth or sole reliance on a single potency readout can create downstream delays or comparability problems if a reagent becomes unavailable or the assay proves invalid for a broader dose range.
Panelists recommended maintaining assay 'depth' (a broad pool of characterization assays retained for comparability), early engagement with FDA on potency strategies, and investment in manufacturing process controls and qualification tools such as process analytical technologies and single‑cell/spatial analytics.
The workshop reinforced a recurring theme: scientific advances are outpacing some regulatory and manufacturing practices, and closing that gap — through robust assays, validated reference materials and scalable production — is critical to moving safe, effective cell therapies into broader clinical use.
