Human Stem Cell Use: Adult or Tissue Stem Cell

A Double-Blind, Controlled Ph 2b Study of the Safety and Efficacy of Modified Stem Cells in Patients with Chronic Motor Deficit from Ischemic Stroke

Therapeutic Candidate or Device Modified adult donor bone marrow-derived mesenchymal stem cells (Modified MSC) Indication Chronic motor deficit secondary to ischemic stroke Therapeutic Mechanism Local intracerebral delivery of Modified MSC adjacent to motor pathways stimulate via a paracrine mechanism neuropoiesis & angiogenesis by the release of FGF-2, other trophic factors & ECM proteins. The net […]

Phase I Study of Chimeric Antigen Receptor Engineered Central Memory T cells for the Treatment of Malignant Glioma

Therapeutic Candidate or Device A promising immunotherapy utilizing a patient’s memory T cells engineered to express chimeric antigen receptors for targeted tumor killing. Indication Malignant glioma (WHO III and IV), including glioblastoma (WHO IV), that express the tumor-associated antigen IL-13 receptor alpha 2 (IL13Rα2). Therapeutic Mechanism A promising immunotherapy utilizing a patient’s memory T cells […]

A Phase 3, Randomized, Placebo-controlled Multicenter Study to Evaluate Efficacy & Safety of Repeated Administrations of NurOwn® in Patients with ALS

Therapeutic Candidate or Device A cell therapy that delivers high levels of neurotrophic factors to the CNS Indication Amyotrophic lateral sclerosis (ALS) or Lou Gehrig Disease Therapeutic Mechanism The Cell therapy is aimed at providing high levels of neurotrophic factors directly to the CNS, to support the dying neurons Unmet Medical Need Amyotrophic lateral sclerosis […]

Phase 2 Safety and Efficacy Study of CLBS03 Autologous T-Regulatory Cells in Adolescents with Recent Onset Type 1 Diabetes Mellitus

Therapeutic Candidate or Device Autologous Ex Vivo Expanded Polyclonal CD4+CD25+CD127lo/-FOXP3+ Regulatory T-cells (CLBS03) Indication Early Onset Type 1 Diabetes Mellitus with Residual Beta Cell Function Therapeutic Mechanism It must be acknowledged that the mechanism(s) by which the effector arm of the immune system becomes unrestrained in the setting of T1D, resulting in the immune destruction […]

A Phase 3 Study Comparing the Utility of Human Acellular Vessels to Arteriovenous Fistula in Subjects with End-Stage Renal Disease (California Sites)

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 human smooth muscle cells (SMC) in a biomimetic bioreactor system. The manufacturing process is designed to create a biologic matrix similar in protein composition and […]

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 […]

Lentiviral Gene Therapy for Infants with X-linked Severe Combined Immunodeficiency using Autologous Bone Marrow Stem Cells and Busulfan Conditioning

Therapeutic Candidate or Device Bone marrow stem cells will be transduced with a lentiviral vector to deliver a normal copy of the gamma-chain gene to treat X-linked SCID. Indication x-linked severe combined immunodeficiency, a severe pediatric disorder in which children have multiple defects in their immune system. Therapeutic Mechanism The gene therapy will correct the […]

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 […]

Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects

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 ADA-SCID) Therapeutic Mechanism This project will lead to a License Application for OTL-101 as a treatment for ADA-SCID. The patient’s own stem cells ("autologous") are […]