Research Objective AAV-SPL 2.0 is a gene therapy cure for SPLIS, a lethal childhood disorder of metabolism that causes kidney failure. Our gene therapy may also work in more common…
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 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 will develop an antisense oligonucleotide, or DNA therapy for diverse forms of amyotrophic lateral sclerosis (ALS). Impact ALS is fatal and incurable, and if successful, we will…
Research Objective Therapeutic candidate to cure hemophilia A is AAV-based genome editing vector that corrects the disease-causing mutation in the factor VIII gene in patient stem cells to develop a…
Research Objective An intravenously injectable virus that converts the scar cells responsible for liver cirrhosis into the cells that provide most of the liver’s function, thereby preventing or reversing liver…
Research Objective Discovery of a novel therapeutic candidate for Parkinson’s disease which modifies gene expression using human stem cell-derived neurons to halt the neurodegenerative disease process. Impact Stopping the neurodegenerative…
Research Objective To develop a gene therapy product to deliver cardiac reprogramming factors into the heart for regeneration of new heart muscle. Impact The proposed candidate would regenerate heart muscle…
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