Therapeutic/Technology: Gene-modified, personalized cell therapy
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 […]
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 […]
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 […]
Pre-clinical development of gene correction therapy of hematopoietic stem cells for SCID-X1
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 to form an immune system. If they are exposed to the environment for even a short period of time they can get infections that a […]
A Phase I, Open-Label Study To Assess The Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells (SB-728MR-HSPC) with Escalating Doses of Busulfan In HIV-1 (R5) Infected Sub…
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 HIV-1 infection and AIDS. All individuals carry two copies of the CCR5 gene, and those with both copies of CCR5 mutated are highly resistant to […]
Clinical Trial of Stem Cell Gene Therapy for Sickle Cell Disease
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 1/500 African-Americans, and is also common in Hispanic-Americans, who comprise up to 5% of SCD patients in California. The median survival based on 1991 national […]
A Treatment For Beta-thalassemia via High-Efficiency Targeted Genome Editing of Hematopoietic Stem Cells
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 is significant, with life expectancy of only ~30-50 years despite standard of care treatment of chronic blood transfusions and iron chelation therapy. Cardiomyopathy due to […]
Stem Cell Programming With Chimeric Antigen Receptors to Eradicate HIV Infection
The AIDS virus infects and destroys cells of the immune system such that the bodies of infected individuals cannot fight infections or some cancers. If untreated HIV infection leads to death. Current therapies to stop virus replication in the body are expensive and can have side effects. They also do not eliminate the virus from […]
Gene therapy-corrected autologous hepatocyte-like cells from induced pluripotent stem cells for the treatment of pediatric single enzyme disorders
Liver transplantation (LT) has been used to treat a variety of liver diseases. Within hours after birth, neonates can present with disorders of the urea cycle (UCDs), the critical metabolic liver pathway needed to detoxify waste nitrogen from the diet and cellular turnover. The overall incidence of UCDs is estimated to be 1 in 8200 […]