Microgel encapsulated iPSC-derived notochordal cells to treat intervertebral disc degeneration and low back pain

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Research Objective We aim to discover an injectable, rejuvenating treatment for painful intervertebral disc degeneration using microtissue-encapsulated iPSC-derived notochordal cells (iNCs) using large animal model Impact Our treatment candidate may…

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Toward a Cure for Gaucher Disease Type 1: Autologous Transplantation of Genome Edited Hematopoietic Stem Cells

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Translational Candidate Autologous blood stem cells edited to restore glucocerebrosidase expression Area of Impact Gaucher disease type 1 (non-neuronopathic) Mechanism of Action To treat Gaucher disease, autologous blood stem cells…

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Therapeutic development of Oxy200, an oxysterol with bone anabolic and anti-resorptive properties for intervention in osteoporosis

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Translational Candidate A novel oxysterol with bone anabolic and anti-resorptive activity that will effectively and safely treat osteoporosis better than current options. Area of Impact Osteoporosis that results in bone…

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