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…
Therapeutic Candidate or Device blood stem cells and T cells from organ transplant donors will be studied under this proposal to prevent rejection of kidney transplants Indication to withdraw immunosuppressant…
Therapeutic Candidate or Device Autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding the human ADA gene (or "OTL-101") Indication Adenosine Deaminase - Severe Combined Immunodeficiency (or…
Therapeutic Candidate or Device Autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding the human ADA gene (or "OTL-101") Indication Adenosine Deaminase - Severe Combined Immunodeficiency (or…
Therapeutic Candidate or Device Autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding the human ADA gene (or "OTL-101") Indication Adenosine Deaminase - Severe Combined Immunodeficiency (or…
Therapeutic Candidate or Device CNS10-NPC-GDNF - a neural progenitor cell secreting GDNF Indication ALS Therapeutic Mechanism This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As…
Therapeutic Candidate or Device Maternal bone marrow-derived CD34+ hematopoietic stem cells. Indication Fetal alpha thalassemia major. Therapeutic Mechanism This strategy that takes advantage of existing tolerance between the mother and…
Therapeutic Candidate or Device Human Acellular Vessel (HAV) Indication Conduit for Vascular Access for Hemodialysis Therapeutic Mechanism Mechanism of action: the HAV is comprised of intact extracellular matrix constructed by…
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…
Therapeutic Candidate or Device HSC gene modified by a lentiviral vector which encodes a triple combination of HIV-resistance genes and a pre-selective marker. Indication HIV in AIDS-lymphoma patients. Therapeutic Mechanism…
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