Periodic Reporting for period 1 - SightLeaders (StemSight: curing blindness with stem cells)
Reporting period: 2023-06-01 to 2024-05-31
StemSight develops off-the-shelf tissue products using induced pluripotent stem cells (iPSCs) and biomaterials. StemSight's first target indication limbal stem cell deficiency (LSCD). LSCD is a rare (incidence 1 per 10,000 in Europe), but painful and debilitating form of corneal blindness, that can be caused by traumatic injuries to the eye (such as thermal or chemical burns), infections or genetic conditions. In LSCD, the native limbal stem cells (LSCs) located on the edges of the cornea are destroyed, which disrupts the normal healing capacity of the corneal surface epithelium and leads to overgrowth of surrounding opaque conjunctiva and blood vessels. LSCD affects especially young and working-age population. Currently there is no therapy that could cure bilateral LSCD, leaving patients on expensive supportive care. StemSight’s high-purity regenerative iPSC-derived limbal stem cells aim to permanently restore sight for LSCD patients. In the future, StemSight’s platform can be expanded to other forms of vision loss.
In the WomenTechEU project, we have focused on regulatory and project management for advancing our novel regenerative medicine products towards first-in-human clinical trials, as well as exploring new market opportunities for our pipeline. We have established a regulatory framework for our lead asset, which will allow us to enter clinical trials in the EU. As part of this regulatory pathway, we received a regulatory classification from the EMA Committee of Advanced Therapies (CAT) as a non-combined tissue-engineered product. Further, our plans for preclinical proof-of-concept and safety studies have been defined, with positive regulatory agency feedback. We now have a second therapy in the pipeline, with iPSC-derived corneal endothelial cells. We have assessed the freedom-to-operate and health economic potential of this asset, encouraging us to pursue further development of this asset.
The results of this WomenTechEU project are an important step towards the creation of a new class of iPSC-based regenerative medicine products. While further execution and resources are needed to realize the preclinical plans for these products, informative regulatory feedback has enabled the refinement of the plans to streamline the path to clinical development. In the next phase, careful clinical trial planning will be crucial to ensure that the trial will be able to also address the needs of European joint Health Technology Assessment for successful market adoption. Access to further capital will be needed to support the preclinical and clinical developments of our lead product, and to realize the ambitious growth plans to become the leading ocular stem cell therapy company in the world.