Final Operational Milestone #4
Under the CLIN1 award, we have performed all necessary pre-clinical studies to develop cultivated limbal stem cells (cLSC) produced by using an animal-free culture system on amniotic membrane for the treatment of both unilateral and bilateral limbal stem cell deficiency (LSCD) . Specifically, we have established a GMP compliant process to manufacture the final cLSC product and a set of in-process controls (IPCs) and release assays to assess the quality of the cLSC product. An imaging technology that evaluates limbal stem cell function in patients has been developed. We have investigated the stability of cLSC.
The cLSC manufacturing was transferred and qualified in a Good Manufacturing Practice (GMP) facility under standard quality controls. The intermediate-term stability of the cLSC product has been optimized. We have started to develop a potency assay that will correlate the quality of the graft with the outcome success.
All these activities allowed a successful IND application to the FDA for a Phase I clinical trial.