Stage of Program: Clinical Trial, Phase 1
Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells
Therapeutic Candidate or Device Bone marrow stem cells that have been treated by inserting a normal Artemis gene into the DNA using a modified virus called a lentivirus. Indication Children with severe combined immunodeficiency or "bubble baby disease" due to a defective gene that makes a protein called Artemis Therapeutic Mechanism Stem cells from the […]
A phase I trial of intratumoral administration of CCL21-gene modified dendritic cell (DC) combined with intravenous pembrolizumab for advanced NSCLC
Therapeutic Candidate or Device Combination therapy with adenoviral CCL21 gene-modified DC and pembrolizumab Indication Patients with confirmed and measurable stage IV NSCLC expressing PD-L1 in less than 50% of cells who are naïve to systemic treatment for NSCLC. Therapeutic Mechanism The central rationale this approach is to utilize in situ vaccination with intratumoral injection of […]
Clinical Study of T stem cell memory (Tscm)-based CAR-T cells in Patients with Multiple Myeloma
Therapeutic Candidate or Device Genetically engineered, Centyrin-based, stem cell memory CAR-T cells (CARTyrin T cells) Indication Multiple Myeloma Therapeutic Mechanism The Centyrin-based chimeric antigen receptor (CARTyrin) cells are cells that are removed from a myeloma patient's body and genetically engineered to express a receptor that binds to BCMA that is selectively found on myeloma cells, […]
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 […]