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…
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…
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…
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…
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)…
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…
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…
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…
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…
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…
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