Stage of Program: Clinical Stage Programs (2.0 and relevant 1.0 projects)
Retinal progenitor cells for treatment of retinitis pigmentosa
Retinitis pigmentosa (RP) is a severe form of blindness that runs in families with an incidence of 1:4000. A team at UC Irvine, is using cells called retinal progenitor cells to repair the damage caused by this vision destroying disease. The cells are injected into the back of the eye and it’s hoped they will […]
Phase 2b Clinical Study of Safety and Efficacy of Intravitreal Injection of Retinal Progenitor Cells (jCell) for Treatment of Retinitis Pigmentosa
Retinitis pigmentosa (RP) is a severe form of blindness that runs in families with an incidence of 1:4000. A team at UC Irvine, is using cells called retinal progenitor cells to repair the damage caused by this vision destroying disease. The cells are injected into the back of the eye and it’s hoped they will help […]
Progenitor Cells Secreting GDNF for the Treatment of ALS
ALS is a devastating neurodegenerative disease with no cure that specifically affect a patient’s motor neurons in the brain. A team at Cedars-Sinai is transplanting millions of genetically engineered stem cells into patients with ALS. When transplanted into the patient spinal cord, these cells become astrocytes, the support cells that keep nerve cells functioning. Due to […]
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
Stroke is a major cause of long-term disability and there are no proven medical treatments for chronic stroke. SanBio is carrying out a Phase 2 clinical trial using mesenchymal stem cells (MSCs) to help people suffering from chronic disability following a stroke. The MSCs are isolated from the bone marrow of healthy adult donors, and […]
Evaluation of Safety and Preliminary Efficacy of Escalating Doses of GRNOPC1 in Subacute Spinal Cord Injury
Geron carried out a Phase 1 clinical trial to assess the safety and preliminary activity of escalating doses of human embryonic stem cell (hESC) derived oligodendrocyte progenitor cells for treatment of spinal cord injury. Five patients were treated, all safely and without any serious side effects, before the trial was halted by Geron for financial […]
MSC engineered to produce BDNF for the treatment of Huntington’s disease
A team at UC Davis plans to use bone marrow derived mesenchymal stem cells to deliver a growth factor, called BDNF, to patients’ brains in order to reduce the death of nerve cells that occurs in Huntington’s Disease (HD). In preparation for such a clinical trial study, the team completed a CIRM-funded observational trial in […]
A Phase I/IIa Dose Escalation Safety Study of AST-OPC1 in Patients with Cervical Sensorimotor Complete Spinal Cord Injury
Up to 12,000 Americans suffer a spinal cord injury (SCI) each year. This condition leads to a high level of permanent disability and decreased life expectancy. Currently, there are no approved therapies for patients with SCI. Asterias Biotherapeutics uses cells derived from embryonic stem cells to heal the spinal cord at the site of injury. They […]
A Phase 3, Randomized, Placebo-controlled Multicenter Study to Evaluate Efficacy & Safety of Repeated Administrations of NurOwn® in Patients with ALS
BrainStorm Therapeutics is using mesenchymal stem cells that are taken from the patient’s own bone marrow. These stem cells are then modified in the lab to boost their production of neurotrophic factors, which are known to help support and protect neurons, the cells destroyed by ALS. The modified cells are then returned to the patients […]
Genetic Re-programming of Stem Cells to Fight Cancer
There are few options for patients whose cancers have metastasized due to resistance to current therapies. This team is using gene editing technology to modify a patient’s own immune system cells and blood-forming stem cells with the aim of creating a continuous supply of immune cells that can recognize and attack hard-to-treat cancers.
AB-110-001 Phase 1b Trial and Related Activities to Support Clinical Development of AB-110
Angiocrine is developing a cell therapy aimed to improve the availability and engraftment of blood stem cell transplants for cancer patients who have had their cancerous bone marrow removed by chemotherapy. The cell therapy is made of blood stem cells and endothelial cells, which line blood vessels and are thought to improve the engraftment of the […]