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


Phase 2 Safety and Efficacy Study of CLBS03 Autologous T-Regulatory Cells in Adolescents with Recent Onset Type 1 Diabetes Mellitus

Children with type 1 diabetes (T1D) face lifelong struggles with controlling their blood sugar levels and, despite careful management, an increased risk of severe complications. Currently, there is no approved therapy that maintains or restores the insulin-producing pancreatic beta cells that are destroyed by this disease. Researchers at Caladrius Biosciences will take cells, called regulatory T cells […]

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 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 […]

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 […]

Clinical Investigation of a Humanized Anti-CD47 Antibody in Targeting Cancer Stem Cells in Hematologic Malignancies and Solid Tumors

A team at Stanford University is using a molecule known as an antibody to target cancer stem cells. This antibody can recognize and bind to CD47, a protein the cancer stem cells carry on their cell surface. The cancer cells use that protein to evade the component of our immune system that routinely destroys tumors. By disabling […]

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 […]

Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients

A team at UC Davis is taking a patient’s blood forming stem cells and inserting three anti-HIV genes into them and then returning them to the individual to help rebuild their immune system. The anti-HIV genes are then passed on to all new immune system cells, which makes them resistant to HIV. Because AIDS-related lymphoma is linked […]

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 […]