Human Stem Cell Use: Adult or Tissue Stem Cell
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
Therapeutic Candidate or Device CD34+CD90+ hematopoietic stem cells (HSC) in combination with AMG 191, a humanized anti-CD117 monoclonal antibody Indication Severe Combined Immunodeficiency Therapeutic Mechanism AMG 191 is being utilized as a conditioning agent for selectively eliminating endogenous stem cells in pediatric SCID patients prior to CD34+CD90+ hematopoietic stem cell transplantation for repopulation of the […]
Genetic Modification of Stem Cells and T cells to Activate the Immune System to Target Solid Tumors
Therapeutic Candidate or Device Autologous Peripheral Blood Stem Cells expressing the NY-ESO-1 TCR and a suicide/reporter gene combined with T cells expressing the same TCR Indication Locally advanced (unresectable stage IIIc) or metastatic malignancies (stage IV) that are HLA A2.1 +, NY-ESO-1 +, solid tumors, including sarcomas Therapeutic Mechanism The administration of TCR transduced mature […]
AB-205-001 Phase 1b Trial and Related Activities to Support Clinical Development of AB-205
Therapeutic Candidate or Device AB-205 consists of genetically engineered CD31+ cells derived from Human Umbilical Vein tissue. Indication To ameliorate or accelerate recovery from toxicities related to high-dose chemo followed by HDT-ASCT for the treatment of lymphoma and other cancers. Therapeutic Mechanism E-CEL UVEC cells (the active ingredient in AB-205) work both via the secretion […]
A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia
Therapeutic Candidate or Device ST-400 is a gene-edited cell therapy candidate for patients with transfusion-dependent beta-thalassemia Indication Transfusion-dependent beta-thalassemia Therapeutic Mechanism ST-400 is intended to disrupt BCL11A erythroid enhancer in CD34+ HSPC resulting in an increase in fetal hemoglobin which can substitute for reduced or absent adult Hb. Therefore, treatment with ST-400 may potentially reduce […]
Treatment of sickle cell disease by induction of mixed chimerism and immune tolerance using CD4+ T-depleted haploidentical blood stem cell transplant
Therapeutic Candidate or Device Haploidentical (half-match) T cell depleted blood stem cell transplant with a low-toxic conditioning regimen Indication Adult patients with severe sickle cell disease who are excluded from the potentially curative current standard stem cell transplant. Therapeutic Mechanism The proposed therapy is intended to achieve mixed chimerism and immune tolerance. Mixed chimerism is […]
Phase 1 Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Adults with Recurrent or Refractory B Cell Malignancies
Therapeutic Candidate or Device T cells genetically engineered to express as bispecific Chimeric Antigen Receptor (CAR) targeting CD19 and/or CD22 Indication Patients with relapsed and refractory B cell malignancies Therapeutic Mechanism T cells expressing the bispecific CAR will recognize cancer cells expressing one of both of the target antigens. Upon recognition, the T cells will […]
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, […]
Cellular Immunotherapy for Induction of Immune Tolerance in HLA Matched Living Donor Kidney Transplant Recipients
Therapeutic Candidate or Device MDR-101 is cellular therapy consisting of kidney donor-derived CD34+ HSCs and CD3+ T-cells. Indication Maintenance of kidney allograft function after withdrawal of post-transplant immunosuppressant (IS) drugs in HLA matched kidney transplants recipients Therapeutic Mechanism Following infusion and engraftment of MDR-101, the progeny cells establish a state of mixed lympo-hematopoetic chimerism. This […]