Therapeutic/Technology: Therapeutic Approach
Allogeneic mesenchymal stem cells loaded with oncolytic virus for cancer treatment
Therapeutic Candidate or Device Supernova-1 (SNV1): Allogeneic adipose-derived culture expanded mesenchymal stem cells (AD-MSC) loaded with oncolytic vaccinia virus Indication -Metastatic melanoma -Triple negative breast cancer -Advanced Head & Neck Squamous Cell Carcinoma Therapeutic Mechanism SNV1 will induce direct killing of cancer cells and will modify the tumor microenvironment, converting immunologically "cold" umors into "hot" […]
IND-Enabling activities for the masked immunocytokine, QXL138AM
Therapeutic Candidate or Device QXL138AM: A Masked Immunocytokine comprising an anti-CD138 antibody fused to interferon alpha 2b, with a protease cleavable peptide mask Indication QXL138AM is for the treatment of advanced or metastatic solid tumors with CD138 expression and multiple myeloma Therapeutic Mechanism The antibody targets the tumor antigen, CD138, on the surface of tumor […]
Development of OSSM-007, cryopreserved interferon-gamma primed allogeneic MSCs, for treatment of steroid refractory acute graft versus host disease
Therapeutic Candidate or Device OSSM007: cryopreserved, interferon-gamma-primed bone marrow mesenchymal stem cells (BM-MSC) Indication acute Graft versus host disease (aGVHD) resulting from hematopoietic cell transplantation Therapeutic Mechanism Immunomodulation of host-reactive T cells to induce operational tolerance of donor HSC-derived lymphocytes through direct cell-to-cell contact and secreted paracrine factors. Interferon-gamma priming of MSCs enhances therapeutic effects […]
Hematopoetic stem cell gene therapy for the treatment of Tay-Sachs disease
Therapeutic Candidate or Device Autologous hematopoietic stem cells transduced with a HexA/HexB expressing lentiviral vector Indication Tay-Sachs disease Therapeutic Mechanism The transplanted gene modified autologous hematopoietic stem cells will engraft in the bone marrow and start producing HexA/HexB expressing immune progeny. Microglia, which establish residence in the brain, will deliver functional beta-hexosaminidase enzyme to affected […]
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 […]
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 […]
Skin regeneration and wound healing with a topical BRAF inhibitor
Therapeutic Candidate or Device LUT017 gel is a small molecule inhibitor of BRAF Indication Venus leg non-healing ulcerous wounds Therapeutic Mechanism The administration LUT017 gel will regenerate cutaneous stem cells and induce keratinocyte proliferation resulting in an improvement of VLU non-healing wounds. Unmet Medical Need There are no previously approved FDA drugs for this condition […]
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 […]
Plurocart: a novel stem cell-based implant for articular cartilage restoration
Therapeutic Candidate or Device Plurocart consists of pluripotent stem cell-derived chondrocytes, seeded onto a scaffold; it is intended to treat damaged cartilage in the knee joint. Indication Plurocart is intended to be surgically implanted in the knee and regenerate injured cartilage – relieving pain and improving function of the joint. Therapeutic Mechanism Plurocart is surgically […]
IND-enabling activities for a Phase 1 Study of Autologous CD4LVFOXP3 T Cells in Subjects with IPEX Syndrome
Therapeutic Candidate or Device CD4+ T cells that have undergone lentiviral -mediated gene transfer of Forkhead Box P3 (FOXP3) and acquired regulatory T cell function. Indication Immune dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) syndrome Therapeutic Mechanism Administration of autologous CD4LVFOXP3 that constitutively and stably express wild-type FOXP3 gene will replace the lack of functional regulatory T […]