Stage of Program: IND enabling
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 […]
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 […]
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 […]
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. […]
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 […]
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 […]
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 […]