IND-enabling study of subretinal delivery of human neural progenitor cells for the treatment of retinitis pigmentosa

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Therapeutic Candidate or Device human fetal cortex derived neural progenitor cells (CNS10-NPC) Indication To stabilize disease progression and maintain ocular integrity and vision for RP patients. This approach can be…

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Development of a Chondrogenic Drug Candidate Targeting Cartilage-residing Mesenchymal Stem Cells for the Treatment of Osteoarthritis

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Osteoarthritis (OA) is the most prevalent musculoskeletal disease affecting nearly 27 million people in the United States, and is the leading cause of chronic disability in the United States. Current…

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A Treatment For Beta-thalassemia via High-Efficiency Targeted Genome Editing of Hematopoietic Stem Cells

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β-thalassemia is a genetic disease caused by diverse mutations of the β-globin gene that lead to profoundly reduced red blood cell (RBC) development. The unmet medical need in transfusion-dependent β-thalassemia…

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