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

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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…

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A Phase I, Pilot Study of Human Embryonic Stem Cell-Derived Cardiomyocytes in PaTients with ChrOnic Ischemic Left VentRicular Dysfunction (HECTOR)

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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…

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Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy

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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…

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Novel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy

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Therapeutic Candidate or Device RJB-0402 is a liver-targeting adeno-associated virus vector-based gene therapy that drives over expression of FGF21. Indication Desmoplakin-related arrhythmogenic cardiomyopathy (DSP ACM) at high risk of life-threatening…

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Development of a therapeutic monoclonal antibody for the treatment of myocardial infarction and heart failure

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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…

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