Stage of Program: Clinical Stage Programs (2.0 and relevant 1.0 projects)


Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy

Capricor is using donor cells derived from the heart to treat patients suffering from Duchenne Muscular Dystrophy (DMD), a genetic disorder that leads to progressive muscle degeneration, including heart muscle. One of the leading causes of death for children with DMD is heart failure and the aim of this treatment is to help improve heart […]

A Human Acellular Vessel in Patients Needing Renal Replacement Therapy: A Comparison with ePTFE Grafts as Conduits for Hemodialysis (HUMANITY)

Humacyte is using donor cells to create a bioengineered vein needed by people with end-stage kidney failure undergoing hemodialysis, the most common form of dialysis. In dialysis a person is connected to a machine that removes waste from their blood. The bioengineered vein is called a Human Acellular Vessel (HAV) and is made of extracellular matrix from […]

Pulmonary Arterial Hypertension Treated with Cardiosphere-Derived Allogeneic Stem Cells

Pulmonary arterial hypertension (PAH) is a progressive condition with no cure. Scientists at Cedars-Sinai Medical Center are using donor cells derived from the heart to reduce two hallmark symptoms of pulmonary hypertension: inflammation and high blood pressure in the blood vessels within the lungs. These conditions make the heart struggle to pump blood to the heart […]

Treatment of non-traumatic osteonecrosis with endogenous Mesenchymal stem cells

Femoral head osteonecrosis (aka avascular necrosis) is a disease caused by loss of blood supply to the bone, leading to bone cell death, end stage hip arthritis and total hip replacement. There is an unmet need for treatment of this disease, that affects individuals at prime of life (peak age 35 years). This small molecule […]

Allogeneic Cardiac-Derived Stem Cells for Patients Following a Myocardial Infarction

In this trial, Capricor Therapeutics used donor cells derived from the heart to treat patients at risk for developing heart failure after a heart attack. In previous clinical studies, the cells appeared to reduce scar tissue, promote blood vessel growth and improve heart function. This trial is ending after failing to achieve its primary goal […]

A Phase III randomized double-blind, controlled study of ICT 107 with maintenance temozolomide (TMZ) in newly diagnosed glioblastoma following resection and concomitant TMZ chemoradiotherapy

ImmunoCellular Therapeutics is targeting six proteins that are found on the surface of cancer stem cells in glioblastoma, a brain cancer. Immune cells from the patient’s own immune system are exposed to fragments of these cancer cell proteins in the lab. When returned to the patient’s body, these immune system cells can now help the […]

A Phase I dose escalation and expansion clinical trial of the novel first-in-class Polo-like Kinase 4 (PLK4) inhibitor, CFI-400945 in patients with advanced solid tumors

Solid tumors are the most prevalent form of cancer, and are a major cause of death worldwide.  A team at UCLA is testing a drug for the treatment of cancer that works by blocking PLK4, a protein that is important in regulating cell growth, division and death. This protein is important for the survival of […]

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

Forty Seven, Inc. has developed an antibody therapy to block a protein called CD47 that is found on the surface of cancer cells. CD47 acts as a ‘don’t eat me’ signal that tells immune cells not to eliminate the cancer cells. When this ‘don’t eat me’ signal is blocked by the antibody, the cancer cells […]

A Phase I, Open-Label Study To Assess The Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells (SB-728MR-HSPC) with Escalating Doses of Busulfan In HIV-1 (R5) Infected Subjects with Suboptimal CD4 Levels on cART

A team at City of Hope and Sangamo Therapeutics is genetically modifying patients’ blood forming stem cells to functionally cure people with HIV. The team is using a technology called zinc finger nucleases – a kind of molecular scissors – to snip out the target gene that codes for the CCR5 receptor. This receptor is the gateway for […]

Therapeutic Eradication of Cancer Stem Cells with UC-961 (Cirmtuzumab)

Cancer is a leading cause of death in California. Many cancers resist current therapies due to therapy-resistant cancer stem cells (CSCs). Scientists at UCSD have discovered a protein, ROR1, that is present on the surface of CSCs, but not on normal, healthy cells. The team has developed an antibody called cirmtuzumab, that specifically targets and attaches to ROR1. […]