Therapeutic/Technology: Therapeutic Approach
Treatment of the TMJ disc complex
Therapeutic Candidate or Device Tissue implant engineered using expanded, allogeneic chondroprogenitor cells Indication Defects of the temporomandibular joint disc complex Therapeutic Mechanism Though the mechanism has not been established, Hyaleon®️ may initially act as a load bearing structure that fills defects of the temporomandibular joint (TMJ) disc complex. The implant is slowly remodeled allowing for […]
Cancer Stem Cell Interception with Rebecsinib: A First-in-Class ADAR1 Inhibitor
Therapeutic Candidate or Device Rebecsinib is a novel small molecule inhibitor of ADAR1 splicing that selectively eradicates therapy-resistant cancer stem cells in blood cancers. Indication The target indication is relapsed/refractory secondary acute myeloid leukemia (sAML), or int-2 or high-risk myelofibrosis (MF). Therapeutic Mechanism Rebecsinib therapy for patients with relapsed/refractory sAML, or int-2 or HR MF […]
Clinical Translation of Autologous Regenerative Pluripotent Stem Cell Therapy for Blindness
Therapeutic Candidate or Device The therapeutic candidate is a patient specific (autologous) induced pluripotent stem cell derived retinal pigment epithelium (AiPSC-RPE) product. Indication AiPSC-RPE cell product will be indicated for the treatment of maculopathies related to RPE atrophy. Therapeutic Mechanism The proposed therapy is an autlogous cell product and intended to be used as a […]
Allogeneic iPSC derived Dopaminergic Drug Product for Parkinson’s disease
Therapeutic Candidate or Device Allogeneic iPSC derived dopamine progenitors delivered to the brain of Parkinson's disease patients Indication Idiopathic Parkinson's disease Therapeutic Mechanism The cellular product reconstitutes dopaminergic neuron circuits lost owing to the disease Unmet Medical Need There are currently disease modifying therapies. This approach is intended to be disease modifying. Project Objective To […]
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 […]
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 […]
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" […]
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 […]
Genome Editing of Autologous Hematopoietic Stem Cells to Treat Severe Mucopolysaccharidosis type 1 (Hurler Syndrome)
Therapeutic Candidate or Device Autologous blood stem cells edited to restore iduronidase expression Indication Severe Mucopolysaccharidosis Type 1 (MPS1/ Hurler's syndrome) Therapeutic Mechanism Autologous blood stem cells undergo genome editing to restore the production of the missing enzyme. These cells are returned to the patient to replace their bone marrow, where they can secrete functional […]