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HuCNS-SC (Human Central Nervous System Stem Cells) is a cell therapy product consisting of purified, fetal-derived human neural stem cells. Developed by StemCells, Inc., these cells are isolated using fluorescence-activated cell sorting (FACS) to select for CD133+ and CD24-/lo populations. The therapeutic mechanism involves the cells' ability to engraft, migrate, and differentiate into functional neural cells—specifically oligodendrocytes for remyelination in Pelizaeus-Merzbacher disease (PMD), or to provide lysosomal enzymes in neuronal ceroid lipofuscinosis (Batten disease). In spinal cord injury (SCI) and age-related macular degeneration (AMD), the cells were intended to provide neurotrophic support and cellular replacement. Although early trials in PMD showed evidence of donor-derived myelination, the development program was terminated in 2016 after the Phase 2 Pathway study in chronic spinal cord injury failed to meet its primary endpoint, leading to the dissolution of StemCells, Inc.
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