Engineering Cardiac Tissue for Regeneration and Drug Development
The ‘valley of death’ in scientific parlance refers to the time period where biomedical discovery in the laboratory fails to result in a cure for a disease. While the last…
Modulating cardiac myosin heavy chain isoform expression for treating cardiomyopathies
Research Objective To discover CRISPR-based genetic strategies that will directly and therapeutically modify cardiac myosin heavy chain isoform expression to treat HF and cardiomyopathies. Impact Heart Failure and Cardiomyopathies Major…
Novel Lipid Nanoparticles for Enhancing eNOS Synthesis for Cardioprotection Post Myocardial Infarction
Research Objective Our therapeutic candidate is a lipid nanoparticle that delivers a therapeutic dose of mRNA to the human heart, which transiently transfects of cells within the heart to improve…
Modified RNA-Based Gene Therapy for Cardiac Regeneration Through Cardiomyocyte Proliferation
Research Objective Efficacious and safe intramyocardial delivery of modified mRNA encoding cell cycle regulators as a gene therapy for cardiac regeneration through resident cardiomyocyte proliferation. Impact This project would provide…
Cardiac Reprogramming Gene Therapy for Post-Myocardial Infarction Heart Failure
Research Objective The candidate is a gene therapy that delivers cardiac reprogramming factors to convert resident cardiac fibroblasts into functioning cardiac muscle. Thus, it is a regenerative cardiac gene therapy.…
New noncoding RNA chemical entity for heart failure with preserved ejection fraction.
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…
Hypoxia-specific Production of Exosomes from iPSC-derivatives for Myocardial Repair
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…
A Phase I, Pilot Study of Human Embryonic Stem Cell-Derived Cardiomyocytes in PaTients with ChrOnic Ischemic Left VentRicular Dysfunction (HECTOR)
Therapeutic Candidate or Device The therapeutic candidate is human embryonic stem cell-derived cardiomyocytes (hESC-CMs) as a new therapy for chronic ischemic cardiomyopathy patients Indication hESC-CMs will be indicated for treatment…
Development of a therapeutic monoclonal antibody for the treatment of myocardial infarction and heart failure
Therapeutic Candidate or Device Fully Humanized monoclonal antibody targeting human ectonucleotide pyrophosphatase/phosphodiesterase (ENPP1) Indication Heart Disease: To prevent the development of heart failure after heart attacks Therapeutic Mechanism After myocardial…