Research Objective The goal of this grant is to develop a gene therapy for a rare painful disorder, Inherited Erythromelalgia (IEM). Impact There are currently no FDA approved drugs for…
Research Objective Modified synthetic noncoding RNA molecule Impact Heart failure with preserved ejection fraction Major Proposed Activities Lead optimizationPerform extensive preclinical testing and select a therapeutic candidate.Develop and test preliminary…
Research Objective A drug-stem cell combination therapy wherein the drug will direct and promote the delivery and distribution of stem cells to the disease site for the optimal therapeutic effect…
Research Objective These studies will produce a new CAR-targeted iPSC-derived macrophage-based cell therapy product for treatment of refractory malignancies such as ovarian cancer. Impact These studies eliminate a bottleneck in…
Research Objective We will develop a cell therapy for a rare skin disease. Patient-derived iPS cells will be genetically corrected and differentiated into epithelial sheets to be grafted on skin…
Research Objective AAV9-Cas13 gene therapy for Angelman syndrome using a first-in-kind mechanism of action that will safely and permanently restore expression of endogenous UBE3A that is deficient in CNS neurons.…
Research Objective A lead therapeutic candidate will be selected: 1) exosomes from hypoxia-injured iPSC-derived cardiomyocytes (iCMs), 2) exosomal miRNA cluster, and 3) siRNA inhibition of exosomal target gene, Notch3. Impact…
Research Objective We propose to study small molecules that inhibit Nemo-like Kinase, to improve the production of red blood cells in bone marrow stem cells of children with Diamond Blackfan…
Research Objective These preclinical studies will discover the efficacy of stem cell-derived, nanoscale, extracellular vesicles (candidate) to treat adverse effects of cancer therapy on brain function and cognition. Impact Stem…
Research Objective We will develop a small molecule inhibitor that blocks the growth of human pancreatic cancer and AML cancer stem cells in vitro and in vivo. Impact This work…
Get the latest CIRM news, information on upcoming meetings, and funding opportunities delivered to your inbox.
