Therapeutic/Technology: Gene-modified, personalized cell therapy
Development of TriLeukeVax, an Engineered Autologous Leukemia Vaccine for Stimulating Cytolytic Immune Responses to Residual Leukemic Stem Cells
Therapeutic Candidate or Device TriLeukeVax, an autologous AML vaccine designed to stimulate induction of anti-leukemic cytolytic activity and improve relapse free survival (RFS). Indication Older leukemia patients who achieve remission with chemotherapy and are at high risk of relapse, but are not eligible for allogeneic transplantation Therapeutic Mechanism Most older patients with acute myelogenous leukemia […]
Hematopoietic Stem Cell Gene Therapy for XCGD
Therapeutic Candidate or Device Hematopoietic stem and progenitor cells collected from X-CGD patients modified with a highly regulated lentiviral vector Indication X-linked Chronic Granulomatous Disease Therapeutic Mechanism Lentiviral vector (LV) modification of autologous hematopoietic stem and progenitor cells (HSPCs) to restore physiologic gp91phox expression. We have developed a next-generation LV designed by bioinformatic-guided screening of […]
Late Stage Pre-Clinical Development of a Cirmtuzumab Based CAR T-cell for the Treatment of ROR1+ Hematological Malignancies
Therapeutic Candidate or Device We are developing ROR1 Chimeric Antigen Receptor (CAR) modified T-cells for the treatment of hematological and solid tumor cancers. Indication The target for our therapy are patients with chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL) and acute lymphoblastic leukemia (ALL). Therapeutic Mechanism Our lead candidate ROR1 CAR-T cell therapy modifies […]
Curing Sickle cell Disease with CRISPR-Cas9 genome editing
Therapeutic Candidate or Device Blood stem cells collected from individuals with sickle cell disease will have the sickle gene corrected and then given back to the same individual. Indication Sickle cell disease is a hereditary blood disorder associated with pain and other serious medical complications including a shortened life-span Therapeutic Mechanism It is possible to […]
Late-Stage Preclinical Study of CAR-T Memory Stem Cells Targeting PSMA (P-PSMA-101) for the Treatment of Castrate-Resistant Metastatic Prostate Cancer
Therapeutic Candidate or Device Genetically engineered, Centyrin-based, CAR- or CARTyrin-T memory stem cells Indication Castrate-resistant metastatic prostate cancer Therapeutic Mechanism The Centyrin-based chimeric antigen receptor (CARTyrin) cells are cells that are removed from a patient's body and genetically engineered to express a receptor that binds to PSMA that is selectively found on prostate cancer cells, […]
Ex Vivo Gene Engineering of Blood Stem Cells for Enhanced Chemotherapy Efficacy in Glioblastoma Patients
Therapeutic Candidate or Device Blood stem cells will be genetically engineered to protect them from chemotherapy in glioblastoma patients, producing better patient survival. Indication Patients with newly diagnosed glioblastoma (GBM) multiforme, or any grade IV newly diagnosed glioma, will be eligible to receive this therapy. Therapeutic Mechanism Chemotherapy is the first-line treatment for GBM, but […]
Genome Editing of Autologous Hematopoietic Stem Cells to Treat Sickle Cell Disease
Therapeutic Candidate or Device Autologous blood stem cells edited to correct the sickle cell disease mutation to be given back to the patient as an autologous stem cell transplant Indication Severe sickle cell disease Therapeutic Mechanism The mechanism of the proposed therapy for sickle cell disease is that the genetically engineered autologous HSCs (pathologic S […]
Ex vivo transduced autologous human CD34+ hematopoietic stem cells for treatment of cystinosis
Therapeutic Candidate or Device Transduced Hematopoietic Stem Cells from Peripheral Blood Stem Cells of adults and pediatric patients with cystinosis Indication Autologous hematopoietic stem cell gene therapy for patients with cystinosis Therapeutic Mechanism Direct transfer of proteins from interstitial macrophages to host cells via long tubular protrusions called tunneling nanotubes, transplantion of autologous HSC modified […]
Ex Vivo Transduction of the Human Artemis (DCLRE1C) cDNA by Lentiviral Vector AProArt into CD34+ Hematopoietic Cells for Artemis (ART)-Deficient Severe Combined Immunodeficiency (SCID)
Therapeutic Candidate or Device Blood-forming stem cells harboring a SCID gene defect, modified to become normal by addition of a correct copy of the Artemis/DCLRE1C DNA repair gene. Indication Treatment of severe combined immunodeficiency due to defects in the Artemis/DCLRE1C gene. Therapeutic Mechanism Severe combined immunodeficiency (SCID) is characterized by absence of T and B […]