01 — Basic research
Define the mechanism
Laboratory studies identify mechanisms of development, inflammation, fibrosis, angiogenesis and repair, establishing a rationale and measurable biological targets.1
02 — Preclinical research
Test activity and risk
Candidate interventions are evaluated for mechanism, biodistribution, dose, persistence, toxicity and biological activity. Models should reflect the proposed clinical indication and acknowledge their limits.2
03 — Product development
Make biology reproducible
Translation requires controlled sourcing, manufacturing, analytical characterisation, sterility, identity, purity, potency and stability. Batch variability is a central challenge for complex cell and vesicle products.3
04 — Clinical research
Evaluate people, prospectively
Early trials usually focus on safety, feasibility and dose. Later phases test efficacy against appropriate controls and clinically meaningful outcomes under ethical and regulatory oversight.1
05 — Implementation
Use only where evidence supports use
Clinical adoption should follow regulatory review and an evidence base appropriate to the specific product and indication. Trial registration alone does not establish that a therapy works.2
Quality by design
Critical quality attributes and potency
A translational programme defines the product before testing it: identity, purity, viability, sterility, genomic stability and relevant biological activity. Potency assays should reflect the proposed mechanism and predict consistent performance across manufacturing lots.3
Clinical protocols then connect dose, route, biodistribution and persistence to safety and outcome measures. Long-term follow-up may be required when cells can proliferate, persist or have been genetically modified.