Innovation Showcases
UniXell has developed an autologous iPSC-derived midbrain dopaminergic progenitors (mDAPs) for Parkinson’s disease, UX-DA001, which have been cleared for FIH trial by the National Medical Products Administration in China and FDA. Here the preclinical data will be presented, including establishment of clinical-grade induced pluripotent stem cells (iPSCs) from patients, manufacturing of mDAPs, GLP-compliant toxicity study, biodistribution and tumorigenicity evaluation.Notably, we achieved high in vivo mDA neuron yields across batches derived from multiple patients, with more than 50% of the human cells in the graft positive for TH (DA neuron marker), accounting for more than 20% of the number of transplanted cells, at 6 months post-grafting. Most of these DA neurons are positive for EN1, a classical midbrain marker. These in vivo outcomes demonstrate the high efficiency and robustness of our new differentiation protocol, bolstering the feasibility of personalized cell therapy for numerous patients with PD. An efficacy study confirmed that the transplanted cells mediated full dopamine level restoration in the grafted striatum (by microdialysis coupled with HPLC) and behavioral recovery in a mouse model of PD. Furthermore, the parkinsonian non-human primates receiving mDAP transplantation exhibited behavioral improvements accompanied with strong DA activity in positron emission tomography.