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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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