Human Stem Cell Use: Adult or Tissue Stem Cell

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 […]

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 […]

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. […]

Ex vivo transduced autologous human CD34+ hematopoietic stem cells for treatment of cystinosis

Therapeutic Candidate or Device Transduced Hematopoietic Stem Cells from Peripheral Blood Stem Cells of adults and pediatric patients with cystinosis Indication Autologous hematopoietic stem cell gene therapy for patients with cystinosis Therapeutic Mechanism Direct transfer of proteins from interstitial macrophages to host cells via long tubular protrusions called tunneling nanotubes, transplantion of autologous HSC modified […]

Regeneration of a Normal Corneal Surface by Limbal Stem Cell Therapy

Therapeutic Candidate or Device cultivated patient-specific corneal epithelial stem cells (limblal stem cells, LSC) Indication Corneal blindness from inability to heal due to corneal epithelial stem cell deficiency as a results of injury Therapeutic Mechanism Limbal stem cell deficiency (LSCD) leads to inability to heal. The most desired treatment is to replace the necessary amount […]

Ex Vivo Transduction of the Human Artemis (DCLRE1C) cDNA by Lentiviral Vector AProArt into CD34+ Hematopoietic Cells for Artemis (ART)-Deficient Severe Combined Immunodeficiency (SCID)

Therapeutic Candidate or Device Blood-forming stem cells harboring a SCID gene defect, modified to become normal by addition of a correct copy of the Artemis/DCLRE1C DNA repair gene. Indication Treatment of severe combined immunodeficiency due to defects in the Artemis/DCLRE1C gene. Therapeutic Mechanism Severe combined immunodeficiency (SCID) is characterized by absence of T and B […]

Development of AB-110: genetically-modified endothelial cells plus expanded cord blood hematopoietic stem cells as a transplantation therapy

Therapeutic Candidate or Device CD34+ Cord-Blood Derived Hematopoietic Stem and Progenitor Cells Co-Cultured and Co-Infused with Genetically Modified Endothelial Cells Indication Life-threatening high-risk hematologic malignancies including leukemia and lymphoma Therapeutic Mechanism Appropriately matched cord-blood derived stem and progenitor cells which has a significant population of both short- and long-term stem cells (expanded by genetically modified […]

IND-enabling study of subretinal delivery of human neural progenitor cells for the treatment of retinitis pigmentosa

Therapeutic Candidate or Device human fetal cortex derived neural progenitor cells (CNS10-NPC) Indication To stabilize disease progression and maintain ocular integrity and vision for RP patients. This approach can be applied to more prevalent AMD. Therapeutic Mechanism A single subretinal injection of Human neural progenitor cells (CNS10-NPC) offers dramatic preservation of vision. CNS10-NPC engraft and […]