Your unwavering support has been instrumental in advancing therapeutic research for VCP disease. Thanks to the generosity of our partners and the CZI Rare As One initiative, we are creating Induced Pluripotent Stem Cells (iPSCs) from VCP disease patients. iPSCs are created from patients' blood and can be transformed into neurons and muscle cells. This exciting technology will allow us to study the disease and test potential drugs with greater speed and precision.
In January, the Cure VCP Disease organization signed a contract with iXCells Biotechnologies for the creation of these models for our pre-clinical toolbox. We are pleased to report that several academic labs have already expressed an interest in utilizing these models for research. Our unique approach includes developing two distinct models: the R155H variant and R159C variant. Both models incorporate custom motor neuron differentiation and the generation of skeletal myoblasts. If you have questions about our research or utilizing these models, please feel free to reach out to me via Calendly or our Scientific Director, Armelle Pindon. We welcome the chance to connect with you. We are grateful for your continued support.