Therapeutic/Technology: Therapeutic Approach


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 […]

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 […]

Late-Stage Preclinical Study of CAR-T Memory Stem Cells Targeting PSMA (P-PSMA-101) for the Treatment of Castrate-Resistant Metastatic Prostate Cancer

Therapeutic Candidate or Device Genetically engineered, Centyrin-based, CAR- or CARTyrin-T memory stem cells Indication Castrate-resistant metastatic prostate cancer Therapeutic Mechanism The Centyrin-based chimeric antigen receptor (CARTyrin) cells are cells that are removed from a patient's body and genetically engineered to express a receptor that binds to PSMA that is selectively found on prostate cancer cells, […]

Ex Vivo Gene Engineering of Blood Stem Cells for Enhanced Chemotherapy Efficacy in Glioblastoma Patients

Therapeutic Candidate or Device Blood stem cells will be genetically engineered to protect them from chemotherapy in glioblastoma patients, producing better patient survival. Indication Patients with newly diagnosed glioblastoma (GBM) multiforme, or any grade IV newly diagnosed glioma, will be eligible to receive this therapy. Therapeutic Mechanism Chemotherapy is the first-line treatment for GBM, but […]

An hESC-derived hNSC Therapeutic for Huntington’s Disease

Therapeutic Candidate or Device The therapeutic candidate is a human Neural Stem Cell product to prevent or delay disease symptoms for treatment of Huntington's disease (HD). Indication Huntington’s disease, a progressive, degenerative brain disease, typically strikes in midlife with no disease modifying treatment treatments exist. Therapeutic Mechanism Based on our pre-clinical studies, the human neural […]

IND enabling development of FT516: A Natural Killer Cell Immunotherapy for Cancer Derived from a Human Inducible Pluripotent Stem Cell

Therapeutic Candidate or Device FT516: A Natural Killer Cell Immunotherapy for Cancer Derived from a Human Inducible Pluripotent Stem Cell Line Indication FT516 monotherapy for patients with advanced cancer and in combination with approved ADCC-competent monoclonal antibodies Therapeutic Mechanism FT516 drug product is comprised of natural killer (NK) cells derived from a clonal human induced […]

Genome Editing of Autologous Hematopoietic Stem Cells to Treat Sickle Cell Disease

Therapeutic Candidate or Device Autologous blood stem cells edited to correct the sickle cell disease mutation to be given back to the patient as an autologous stem cell transplant Indication Severe sickle cell disease Therapeutic Mechanism The mechanism of the proposed therapy for sickle cell disease is that the genetically engineered autologous HSCs (pathologic S […]

Development of CLT030-ADC, a Leukemic Stem Cell Targeting Antibody-Drug-Conjugate, for Treatment of Acute Myeloid Leukemia

Therapeutic Candidate or Device CLT030-ADC, a novel drug targeting a leukemic stem cell surface protein CLL1, is intended to improve therapeutic outcome for AML patients. Indication CLT030-ADC is intended to improve remission rates in AML patients. Therapeutic Mechanism CLT030-ADC is an antibody-drug conjugate targeting leukemic stem cell surface protein CLL1. Leukemic stem cells (LSC) are […]

Intraparenchymal NR1 Stem Cell Therapy for Chronic Subcortical Ischemic Stroke

Therapeutic Candidate or Device A human embryonic derived, non-genetically modified neural stem cell (NR1), originally derived from the Wi-Cell H-9 line. Indication Patients with chronic motor deficits, from 6 months to 5 years after stroke. NR1 cells will be transplanted into the brain near the stroke. Therapeutic Mechanism The proposed therapeutic mechanism of action of […]

Allogenic human adipose-derived mesenchymal stem cells for the treatment of knee osteoarthritis

Therapeutic Candidate or Device Intra-articularly injected allogeneic culture-expanded human adipose derived mesenchymal progenitor cells Indication Knee osteoarthritis Therapeutic Mechanism Cartilage regeneration (as determined by cartilage volume increase), immunomodulatory effects Unmet Medical Need There is no approved disease modification therapy for osteoarthritis (OA), and OA is a leading cause of both hospitalization and joint replacement surgery. […]