Human Stem Cell Use: Adult or Tissue Stem Cell
Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells
Therapeutic Candidate or Device Bone marrow stem cells that have been treated by inserting a normal Artemis gene into the DNA using a modified virus called a lentivirus. Indication Children with severe combined immunodeficiency or "bubble baby disease" due to a defective gene that makes a protein called Artemis Therapeutic Mechanism Stem cells from the […]
A phase I trial of intratumoral administration of CCL21-gene modified dendritic cell (DC) combined with intravenous pembrolizumab for advanced NSCLC
Therapeutic Candidate or Device Combination therapy with adenoviral CCL21 gene-modified DC and pembrolizumab Indication Patients with confirmed and measurable stage IV NSCLC expressing PD-L1 in less than 50% of cells who are naïve to systemic treatment for NSCLC. Therapeutic Mechanism The central rationale this approach is to utilize in situ vaccination with intratumoral injection of […]
Cellular Immunotherapy for Induction of Immune Tolerance in HLA Matched Living Donor Kidney Transplant Recipients
Therapeutic Candidate or Device MDR-101 is cellular therapy consisting of kidney donor-derived CD34+ HSCs and CD3+ T-cells. Indication Maintenance of kidney allograft function after withdrawal of post-transplant immunosuppressant (IS) drugs in HLA matched kidney transplants recipients Therapeutic Mechanism Following infusion and engraftment of MDR-101, the progeny cells establish a state of mixed lympo-hematopoetic chimerism. This […]
Clinical Study of T stem cell memory (Tscm)-based CAR-T cells in Patients with Multiple Myeloma
Therapeutic Candidate or Device Genetically engineered, Centyrin-based, stem cell memory CAR-T cells (CARTyrin T cells) Indication Multiple Myeloma Therapeutic Mechanism The Centyrin-based chimeric antigen receptor (CARTyrin) cells are cells that are removed from a myeloma patient's body and genetically engineered to express a receptor that binds to BCMA that is selectively found on myeloma cells, […]
Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation (ACES)
Therapeutic Candidate or Device Partially HLA-matched virus-specific T-cell therapy targeting cytomegalovirus, Epstein-Barr virus, and adenovirus. Indication This study will treat persistent viral infections with CMV, EBV, and/or adenovirus in patients with immunodeficiency. Therapeutic Mechanism The goal of this study is to use banked virus-specific T-cell therapy in A) patients who have persistent viral infections after […]
Evaluation of the Safety and Tolerability of KA34 in a Phase 1, Double-Blind, Dose Escalation Trial in Patients with Knee Osteoarthritis
Therapeutic Candidate or Device KA34 is an intra-articularly delivered small molecule therapeutic candidate which directs the differentiation of endogenous stem and progenitor cells Indication Osteoarthritis Therapeutic Mechanism KA34 promotes the differentiation of cartilage endogenous stem cells through increased chondrogenic gene expression to generate healthy chondrocytes. KA34 will potentially limit the progression of and / or […]
AB-110-001 Phase 1b Trial and Related Activities to Support Clinical Development of AB-110
Therapeutic Candidate or Device AB-110 consists of cord blood derived hematopoietic stem and progenitor cells co-cultured and expanded with E-CEL UVEC cells Indication Hematologic and immune reconstitution in patients who have received myeloablation conditioning Therapeutic Mechanism Stem and progenitor cells (active ingredient) of AB-110 engraft into the bone marrow of patients, rebuilding a new blood […]
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 […]