Therapeutic/Technology: Therapeutic Approach
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 […]
Phase 2b Clinical Study of Safety and Efficacy of Intravitreal Injection of Retinal Progenitor Cells (jCell) for Treatment of Retinitis Pigmentosa
Therapeutic Candidate or Device Allogeneic human retinal progenitor cells (hRPC) Indication Retinitis Pigmentosa (RP) Therapeutic Mechanism The cells are intended to remain suspended in the vitreous cavity of the eye and exert a beneficial neurotrophic effect on the degenerating retina. Unmet Medical Need RP is an incurable orphan disease. There are no treatments currently available […]
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 […]
Clinical trial of directly vascularized islet cell replacement therapy for high-risk type 1 diabetes
Therapeutic Candidate or Device pancreatic progenitor cells in a delivery device that allows direct vascularization Indication high-risk type 1 diabetes including "brittle" diabetes and hypoglycemia unawareness Therapeutic Mechanism People with type 1 diabetes have lost their pancreatic cells that make insulin, and therefore have to self-administer insulin. It is very difficult to manage blood sugar […]
A Phase 1b/2 Trial of the Anti-CD47 Antibody Hu5F9-G4 in Combination with Cetuximab in Patients with Solid Tumors and Advanced Colorectal Cancer
Therapeutic Candidate or Device The treatment is two drugs: Hu5F9-G4 and cetuximab. These are antibodies, which are engineered drugs that eliminate cancers using the immune system. Indication Patients with advanced colon cancer that have a genetic mutation in the KRAS gene and those without the KRAS genetic mutation. Therapeutic Mechanism This treatment targets cancer stem […]
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 […]