Grant Award Details
Conduct a phase 1 clinical trial to assess the safety and feasibility of cultivated autologous limbal stem cells for limbal stem cell deficiency.
Grant Application Details
- Safety and Feasibility of Cultivated Autologous Limbal Stem Cells for Limbal Stem Cell Deficiency
Therapeutic Candidate or Device
Autologous cultivated limbal stem cells (cLSC)
Limbal stem cell deficiency
Restoration of a normal corneal surface using cLSC might be achieved by replenishing the LSC population and/or providing trophic factors to stimulate residual LSCs.
Unmet Medical Need
Therapy using cultivated LSC, which achieves the best clinical outcomes is a standard of care in Europe. The lack of this therapy in the US is an unmet medical needs . Our therapy has the potential to safely, adequately and successfully treat LSCD and become the standard of care in the US.
Complete a phase I clinical trial
Major Proposed Activities
- Manufacture and transplant 15 patient-specific cLSC in 15 subjects with LSCD and provide scleral lens treatment to 5 control subjects
- Refine and validate the new diagnostic assays for LSCD and quantify LSC function after transplantation.
- Complete the 1 year follow-up in patients treated with the cLSC to assess clinical safety of the cLSC and scleral lens.
The LSCD patient population in California differs from that outside of the US and the efficacy of all currently available LSC treatments have not been demonstrated in our patient population.. Our cLSC product has the potential to treat LSCD by increasing safety in a larger population including those in California. The new diagnostic tools will increase the accuracy of LSCD diagnosis and enable LSC function assessment in patients. Our cLSC therapy will raise the standard of care for LSCD.
- Prog Retin Eye Res (2021) Human limbal epithelial stem cell regulation, bioengineering and function. (PubMed: 33676006)
- Exp Eye Res (2021) Limbal stem cell diseases. (PubMed: 33571530)
- Biofabrication (2021) Bioprinting of dual ECM scaffolds encapsulating limbal stem/progenitor cells in active and quiescent statuses. (PubMed: 34330126)
- Sci Rep (2021) Wnt6 plays a complex role in maintaining human limbal stem/progenitor cells. (PubMed: 34686698)
- Cornea (2020) Global Consensus on the Management of Limbal Stem Cell Deficiency. (PubMed: 32639314)
- Am J Ophthalmol (2020) Long-term outcomes of Descemet membrane endothelial keratoplasty in eyes with prior glaucoma surgery. (PubMed: 32619607)
- Am J Ophthalmol (2020) Corneal Epithelial Thickness Measured Using Anterior Segment Optical Coherence Tomography as a Diagnostic Parameter for Limbal Stem Cell Deficiency. (PubMed: 32283095)