Stage of Program: Clinical Stage Programs (2.0 and relevant 1.0 projects)
Phase 1 Safety Assessment of CPCB-RPE1, hESC-derived RPE Cell Coated Parylene Membrane Implants, in Patients with Advanced Dry Age Related Macular Degeneration
Age-related macular degeneration is a progressive disease resulting in death of the retinal pigment epithelium (RPE) causing distortion to central vision and eventually to legal blindness. Regenerative Patch Technologies and scientists at the University of Southern California and UC Santa Barbara, are growing specialized cells of the retina (called retinal pigment epithelium) from embryonic stem […]
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 […]
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 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 […]
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.
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 […]
Clinical trial of directly vascularized islet cell replacement therapy for high-risk type 1 diabetes
ViaCyte is developing cell therapies to replace lost beta cells for people with type 1 diabetes (T1D). The therapies are derived from human embryonic stem cells, which are partially matured into becoming pancreatic tissues (the type destroyed in T1D). The cells are inserted into a small pouch that is transplanted under the patient’s skin. The […]
A Phase 1b Trial of Hu5F9-G4 Monotherapy or Hu5F9-G4 in Combination with Azacitidine in Patients with Acute Myeloid Leukemia
Forty Seven Inc. is conducting a Phase 1b clinical trial for acute myeloid leukemia patients. Leukemia stem cells have a protein on their surface that enable them to evade being identified and destroyed by the patient’s own immune system. This protein also helps these leukemia stem cells survive traditional therapies such as chemotherapy, enabling the […]