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ALD-601
- Development stage
- Discontinued
- Lead developer
- Aldagen
- Modality
- Stem Cell Therapies → Cell Therapies
- Administration
- Intrauterine
01
Overview
ALD-601 is a cell therapy comprising aldehyde dehydrogenase-bright (ALDHbr) stem and progenitor cells isolated from human umbilical cord blood. Developed by Aldagen (later acquired by Cytomedix and Nuo Therapeutics), the product utilizes a proprietary flow cytometry-based sorting process to select for cells expressing high levels of the enzyme ALDH, a marker for primitive, highly potent hematopoietic and mesenchymal stem cells. ALD-601 was specifically investigated for in utero transplantation to treat fetuses diagnosed with lethal lysosomal storage diseases (LSDs), such as Krabbe disease, metachromatic leukodystrophy (MLD), and Tay-Sachs disease. The therapeutic goal of this fetal stem cell therapy is to establish early donor chimerism and provide functional enzyme replacement and cellular repair before irreversible neurological damage occurs in infancy. Clinical development was discontinued after the withdrawal of its primary Phase 1 trial.
- Other names
- ALDH-bright umbilical cord blood cellsALDHbr UCB cells
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