Human Stem Cell Use: Adult or Tissue Stem Cell
Autologous Gene Corrected Sinus Basal Cells to Treat Serious Cystic Fibrosis Sinus Disease
Therapeutic Candidate or Device Gene corrected autologous sinus airway basal stem cells from patients with Cystic Fibrosis. Indication The proposed studies provide an innovative stem cell based approach with gene correction to treat chronic sinusitis in CF. Therapeutic Mechanism Corrected upper airway cells will produce differentiated epithelium with restored Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) […]
Ex vivo Engineering of Autologous Hematopoietic Stem Cells for the Treatment of Hypophosphatasia
Therapeutic Candidate or Device Hematopoietic stem/progenitor cells collected from patients with hypophosphatasia and genetically modified with a lentiviral vector to release TNALP Indication Hypophosphatasia (HPP) Therapeutic Mechanism We are proposing a cell-based enzyme replacement therapy for HPP: autologous gene-modified hematopoietic stem/progenitor cells (HSPCs) will be infused into the patient and will engraft into the bone […]
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 […]
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 […]
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 […]
MRI Guided Delivery of Neural Progenitor Cells Secreting GDNF for the Treatment of Parkinson’s disease
Therapeutic Candidate or Device CNS10-NPC-GDNF is a neural progenitor cell line transfected with glial cell line derived neurotrophic factor (GDNF) Indication Mid-stage Parkinson's disease (UPDRS stage III or lower) Therapeutic Mechanism Degeneration of dopaminergic neurons that project from the substantia nigra to the striatum causes the primary motor symptoms of Parkinson's disease. CNS10-NPC-GDNF cells will […]