Therapeutic/Technology: Therapeutic Approach
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
Placental Mesenchymal Stem Cell Augmentation of Fetal Myelomeningocele Repair
Therapeutic Candidate or Device Allogeneic Placenta-derived Mesenchymal Stem Cells Seeded on Cook Biodesign® Dural Graft Extracellular Matrix (PMSC-ECM) Indication Myelomeningocele (MMC) -or Spina Bifida -diagnosed prenataly Therapeutic Mechanism Placenta-derived mesenchymal stem cells (PMSCs) act by a paracrine mechanism, secreting a variety of growth factors, cytokines, and extracellular vesicles. This secretory profile is unique to PMSCs […]
IND-enabling development of ART352-L, an endogenous stem cell reactivation therapy to enhance bone healing in the elderly
Therapeutic Candidate or Device ART352-L, a liposomal formulation of recombinant human WNT3A protein that is intended to enhance the osteogenic properties of autografts in elderly Indication Patients with Degenerative Spondylolisthesis (DS) undergoing a spinal fusion surgery Therapeutic Mechanism WNT proteins are potent pro-osteogenic signals. L-WNT3A is the investigative prototype material of ART352-L. L-WNT3A treated autografts […]
CMV-specific T cells expressing anti-HIV CAR and CMV vaccine boost as immunotherapy for HIV/AIDS
Therapeutic Candidate or Device Cytomegalovirus (CMV)-reactive T cells that express chimeric antibody receptors (CARs) to recognize and kill HIV-infected cells Indication HIV/AIDS Therapeutic Mechanism Antiretroviral drug therapy (ART) suppresses HIV to undetectable levels but does not eradicate the cellular reservoirs of the virus. We will engineer HIV-specific CAR T cells that will kill reactivated HIV-infected […]