Stage of Program: Clinical Trial, Phase 1


Evaluation of the Safety and Tolerability of KA34 in a Phase 1, Double-Blind, Dose Escalation Trial in Patients with Knee Osteoarthritis

Therapeutic Candidate or Device KA34 is an intra-articularly delivered small molecule therapeutic candidate which directs the differentiation of endogenous stem and progenitor cells Indication Osteoarthritis Therapeutic Mechanism KA34 promotes the differentiation of cartilage endogenous stem cells through increased chondrogenic gene expression to generate healthy chondrocytes. KA34 will potentially limit the progression of and / or […]

AB-110-001 Phase 1b Trial and Related Activities to Support Clinical Development of AB-110

Therapeutic Candidate or Device AB-110 consists of cord blood derived hematopoietic stem and progenitor cells co-cultured and expanded with E-CEL UVEC cells Indication Hematologic and immune reconstitution in patients who have received myeloablation conditioning Therapeutic Mechanism Stem and progenitor cells (active ingredient) of AB-110 engraft into the bone marrow of patients, rebuilding a new blood […]

Phase I Study of Chimeric Antigen Receptor Engineered Central Memory T cells for the Treatment of Malignant Glioma

Therapeutic Candidate or Device A promising immunotherapy utilizing a patient’s memory T cells engineered to express chimeric antigen receptors for targeted tumor killing. Indication Malignant glioma (WHO III and IV), including glioblastoma (WHO IV), that express the tumor-associated antigen IL-13 receptor alpha 2 (IL13Rα2). Therapeutic Mechanism A promising immunotherapy utilizing a patient’s memory T cells […]

Phase 1b Trial of Hu5F9-G4 Monotherapy or Hu5F9-G4 in Combination with Azacitidine in Patients with Acute Myeloid Leukemia and Myelodysplastic Syndrome

Therapeutic Candidate or Device Hu5F9-G4 is a drug called an antibody that is designed to mobilize the body's immune system to eliminate cancer and cancer stem cells. Indication Patients with relapsed and/or refractory AML or newly diagnosed AML who cannot receive standard high dose chemotherapy. Therapeutic Mechanism This treatment targets cancer stem cells, which are […]

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 […]

In Utero Hematopoietic Stem Cell Transplantation For The Treatment Of Fetuses With Alpha Thalassemia Major

Therapeutic Candidate or Device Maternal bone marrow-derived CD34+ hematopoietic stem cells. Indication Fetal alpha thalassemia major. Therapeutic Mechanism This strategy that takes advantage of existing tolerance between the mother and fetus during pregnancy, so that maternal cells can be transplanted into a fetus without conditioning or immunosuppression. Survivors of alpha thalassemia need chronic blood transfusions […]

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 Sub…

The HIV-1 virus enters cells by binding to a protein called CCR5 on the cell surface. A naturally occurring mutation in CCR5, CCR5d32, has been shown to provide protection from HIV-1 infection and AIDS. All individuals carry two copies of the CCR5 gene, and those with both copies of CCR5 mutated are highly resistant to […]

Phase 1 Safety Assessment of CPCB-RPE1, hESC-derived RPE Cell Coated Parylene Membrane Implants, in Patients with Advanced Dry Age Related Macular Degeneration

It is estimated that by 2020, over 450,000 Californians will suffer from vision loss or blindness due to the age-related macular degeneration (AMD), the most common cause of retinal degeneration in the elderly. AMD is a progressive ocular disease of the part of the retina, called the macula, which enables people to read, visualize faces, […]

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

Cancer is a major cause of morbidity and mortality worldwide. Many believe that progress in drug development has not been as rapid as one would have predicted based on the significant technological advancements that have led to improved molecular understanding of this disease. There are numerous explanations for the lag in clinical success with new […]

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

Most normal tissues are maintained by a small number of stem cells that can both self-renew to maintain stem cell numbers, and also give rise to progenitors that make mature cells. We have shown that normal stem cells can accumulate mutations that cause progenitors to self-renew out of control, forming cancer stem cells (CSC). CSC […]