Therapeutic/Technology: Donor cell therapy
A Phase 2 Open-Label, Multi-Center, Randomized, Controlled, Optimal Dose-Finding Study of DCC-UCB in Adults Receiving High Dose Chemotherapy for AML
Therapeutic Candidate or Device Cryopreserved, universal donor hematopoietic stem cell therapy that restores blood cell function and protects against infection after chemotherapy Indication Neutropenia arising from high-dose chemotherapy for treatment of Acute Myeloid Leukemia Therapeutic Mechanism The primary treatment for patients with AML is chemotherapy. Most chemotherapy results in a period of neutropenia (very low […]
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 […]
Induction of Tolerance to Combined Kidney and Hematopoietic Progenitor Cell Transplants from HLA Haplotype Matched Living Donors
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 drugs from kidney transplant recipients Therapeutic Mechanism Injection of the donor blood stem cells into recipients will prevent recipient immune cells from rejecting the donor […]
In Utero Hematopoietic Stem Cell Transplantation For The Treatment Of Fetuses With Alpha Thalassemia Major
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 fetus during pregnancy, so that maternal cells can be transplanted into a fetus without conditioning or immunosuppression. Survivors of alpha thalassemia need chronic blood transfusions […]
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 […]
Human Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI)
Therapeutic Candidate or Device Human embryonic stem cell-derived neural stem cells. Indication Spinal cord injury Therapeutic Mechanism The proposed neural stem cell therapy will be transplanted into a spinal cord injury site, providing cell replacement and a new neuronal relay across the injury site in an effort to promote significant functional improvement. Unmet Medical Need […]
Treatment of the TMJ disc complex
Therapeutic Candidate or Device Tissue implant engineered using expanded, allogeneic chondroprogenitor cells Indication Defects of the temporomandibular joint disc complex Therapeutic Mechanism Though the mechanism has not been established, Hyaleon®️ may initially act as a load bearing structure that fills defects of the temporomandibular joint (TMJ) disc complex. The implant is slowly remodeled allowing for […]
Allogeneic iPSC derived Dopaminergic Drug Product for Parkinson’s disease
Therapeutic Candidate or Device Allogeneic iPSC derived dopamine progenitors delivered to the brain of Parkinson's disease patients Indication Idiopathic Parkinson's disease Therapeutic Mechanism The cellular product reconstitutes dopaminergic neuron circuits lost owing to the disease Unmet Medical Need There are currently disease modifying therapies. This approach is intended to be disease modifying. Project Objective To […]
Allogeneic mesenchymal stem cells loaded with oncolytic virus for cancer treatment
Therapeutic Candidate or Device Supernova-1 (SNV1): Allogeneic adipose-derived culture expanded mesenchymal stem cells (AD-MSC) loaded with oncolytic vaccinia virus Indication -Metastatic melanoma -Triple negative breast cancer -Advanced Head & Neck Squamous Cell Carcinoma Therapeutic Mechanism SNV1 will induce direct killing of cancer cells and will modify the tumor microenvironment, converting immunologically "cold" umors into "hot" […]
Development of OSSM-007, cryopreserved interferon-gamma primed allogeneic MSCs, for treatment of steroid refractory acute graft versus host disease
Therapeutic Candidate or Device OSSM007: cryopreserved, interferon-gamma-primed bone marrow mesenchymal stem cells (BM-MSC) Indication acute Graft versus host disease (aGVHD) resulting from hematopoietic cell transplantation Therapeutic Mechanism Immunomodulation of host-reactive T cells to induce operational tolerance of donor HSC-derived lymphocytes through direct cell-to-cell contact and secreted paracrine factors. Interferon-gamma priming of MSCs enhances therapeutic effects […]