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

Induction of Tolerance by Combinatorial Therapy w/ Donor Stem Cells and Expanded Recipient Treg cells in HLA-mismatched Kidney Transplant Recipients

Therapeutic Candidate or Device Combined hematopoietic stem cell graft and recipient T regulatory cells Indication Kidney disease requiring kidney transplantation Therapeutic Mechanism The study will determine whether patients treated with TLI and rATG, and given a haploidentical living donor hematopoietic progenitor cell transplant (HSCT) , along with in vitro expanded recipient Treg cells (what we […]

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 […]

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 […]

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, […]

Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation (ACES)

Therapeutic Candidate or Device Partially HLA-matched virus-specific T-cell therapy targeting cytomegalovirus, Epstein-Barr virus, and adenovirus. Indication This study will treat persistent viral infections with CMV, EBV, and/or adenovirus in patients with immunodeficiency. Therapeutic Mechanism The goal of this study is to use banked virus-specific T-cell therapy in A) patients who have persistent viral infections after […]