Stage of Program: Clinical Stage Programs (2.0 and relevant 1.0 projects)
Development of a Gene Therapy for the Treatment of WWOX related epileptic encephalopathy (WOREE)
Therapeutic Candidate or Device MZ-9138 is an AAV9 gene therapy Indication WWOX-related epileptic encephalopathy Therapeutic Mechanism MZ-9138 delivered to the brain may transduce neurons to create functioning WWOX protein and ultimately improve the phenotype of patients with WOREE Unmet Medical Need WOREE is a severe epileptic disorder resulting in dramatically shortened survival of patients. There […]
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) […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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" […]