Therapeutic Candidate or Device Blood stem cells collected from individuals with sickle cell disease will have the sickle gene corrected and then given back to the same individual. Indication Sickle…
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…
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)…
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 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 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…
Severe combined immunodeficiency caused by mutations in the IL2RG gene on the x-chromosome (SCID-X1 or "bubble boy disease") is a devastating genetic disease that results in boys not being able…
The HIV-1 virus enters cells by binding to a protein called CCR5 on the cell surface. A naturally occurring mutation in CCR5, CCR5d32, has been shown to provide protection from…
Sickle cell disease (SCD)results from an inherited mutation in the hemoglobin gene that causes red blood cells to "sickle" under conditions of low oxygen. It occurs with a frequency of…
β-thalassemia is a genetic disease caused by diverse mutations of the β-globin gene that lead to profoundly reduced red blood cell (RBC) development. The unmet medical need in transfusion-dependent β-thalassemia…
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