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GABAeron is developing a first-in-class cell replacement therapy utilizing human induced pluripotent stem cell (iPSC)-derived GABAergic interneuron progenitor cells. The therapy specifically targets the restoration of somatostatin-positive (SST) interneurons within the dentate gyrus of the hippocampus. This approach is designed to address GABAergic interneuron dysfunction, particularly in individuals carrying the apoE4 allele, which is a major genetic risk factor for Alzheimer's disease. By transplanting these progenitor cells, the therapy aims to integrate into existing neural circuits, restore inhibitory signaling, and improve cognitive functions. Beyond Alzheimer's disease, the company is exploring applications in Down's syndrome and epilepsy, where similar inhibitory deficits occur. Preclinical studies in mouse models have demonstrated that the transplanted cells mature, integrate, and restore normal hippocampal activity and memory function.
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