Therapeutic/Technology: Therapeutic Approach
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 […]
Stem cell-derived islet cell replacement therapy with immunosuppression for high-risk type 1 diabetes
Therapeutic Candidate or Device hESC-derived pancreatic progenitor cells delivered in a device that allows direct vascularization of the cell therapy Indication high-risk type 1 diabetes including "brittle" diabetes and hypoglycemia unawareness Therapeutic Mechanism People with type 1 diabetes have lost their pancreatic cells that make insulin, and therefore have to self-administer insulin. It is very […]
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 […]
Development of a Chondrogenic Drug Candidate Targeting Resident Mesenchymal Stem Cells for the Treatment of Osteoarthritis
Therapeutic Candidate or Device The therapeutic candidate is a drug-like small molecule that promotes cartilage resident mesenchymal stem cell differentiation into chondrocytes. Indication Osteoarthritis and cartilage injury Therapeutic Mechanism The therapeutic candidate, through intra-articular administration, promotes cartilage resident mesenchymal stem cell differentiation into chondrocytes. The newly formed chondrocytes replace the dead chondrocytes, synthesize and secret […]
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 […]