Disease Focus: Heart Disease

A Novel, Robust and Comprehensive Predictive Tool Using Human Disease-Specific Induced Pluripotent Stem Cells for Preclinical Drug Screening

Translational Candidate A library of induced pluripotent stem cell-derived cardiomyocytes from healthy subjects as well as patients with common hereditary cardiac disorders Area of Impact Preclinical toxicity screening and drug discovery Mechanism of Action Patients with pre-existing cardiac conditions are more susceptible to drug-induced cardiotoxicity than general population. Including iPSCs derived from this subset of […]

Autologous MPO Knock-Out Hematopoietic Stem and Progenitor Cells for Pulmonary Arterial Hypertension

Translational Candidate Autologous MPO Knock-Out Hematopoietic Stem and Progenitor Cells Area of Impact Pulmonary Arterial Hypertension (PAH), initially associated with Scleroderma (Systemic Sclerosis -SSC), and then applied to other causes of PAH Mechanism of Action Myeloperoxidase (MPO) protein produced by neutrophils plays a critical role in the development of PAH. Disrupting the MPO gene in […]

IND-enabling Studies of Wearable Evolve-FSTL1 for Cardiac Regeneration after MI

Translational Candidate The therapeutic candidate is the Regencor’s proprietary Cardio-Regenerative Factor (FSTL1.37) formulated in the Wearable Injector Evolve-FSTL1. Area of Impact The targeted area of impact is to restore cardiac function and reduce progression to heart failure in patients after myocardial Infarction Mechanism of Action FSTL1.37 activates the controlled proliferation of progenitor heart cells within […]

Targeting stromal progenitors to prevent the development of heart failure

Translational Candidate Monoclonal antibody targeting Ectonucleotide phosphodiesterase/pyrophosphatase 1 (ENPP1) Area of Impact Heart disease: To prevent the development of heart failure after heart attacks Mechanism of Action After myocardial infarction, myofibroblast progenitors express ENPP1. ENPP1 is a type II transmembrane protein that hydrolyzes extracellular ATP and hydrolytic products generated by ENPP1 initiate an inflammatory cascade […]

Drug Discovery & Stem Cell Models for Cardiovascular Disease Conference

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 of heart failure (HF) and for preventing progression to HF in patients with chronic ischemic cardiomyopathy. Therapeutic Mechanism There are two commonly accepted mechanisms by […]

Pulmonary Arterial Hypertension Treated with Cardiosphere-Derived Allogeneic Stem Cells

Therapeutic Candidate or Device CAP-1002 Indication Patients with Pulmonary Arterial Hypertension (PAH) Therapeutic Mechanism CAP-1002's mechanism of action may result in reduced wall thickening of small blood vessels in the lung that are markedly narrowed/obstructed in PAH. The latter results in high resistance against which the right ventricle (RV) must pump in order to drive […]

Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy

Therapeutic Candidate or Device Allogeneic Cardiosphere-Derived Cells (CDCs, CAP-1002) Indication Duchenne Muscular Dystrophy Cardiomyopathy Therapeutic Mechanism Secretion of identified bioactive elements will reduce myocardial fibrosis and improve cardiac function. Unmet Medical Need Heart failure is the leading cause of death among young men with Duchenne Muscular Dystrophy. No specific therapies exist to treat this element […]

Development of 3D Bioprinting Techniques using Human Embryonic Stem Cells Derived Cardiomyocytes for Cardiac Tissue Engineering

Heart, stroke and other cardiovascular diseases are responsible for ~17 million deaths per year globally and this number is predicted to reach 23.3 million by 2030. Cardiovascular diseases impose a staggering annual cost of $300 billion on the U.S. health care system. Heart transplantation is the ultimate solution to end-stage heart failure. However, a major […]

Development of a scalable, practical, and transferable GMP-compliant suspension culture-based differentiation process for cardiomyocyte production from human embryonic stem cells.

As ongoing CIRM-funded development of regenerative medicine (RM) progresses, the demand for increasing numbers of pluripotent stem cells and their differentiated derivatives has also increased. We have established a scalable suspension culture system for the production of large quantities of hESC for banking and to seed production of a number of regenerative medicine cell types, […]