California Institute for Regenerative Medicine (CIRM)
- The Alpha Stem Cell Clinic – A Program for the Development and Delivery of Innovative Cell-based Treatments and Cures for Life-Threatening Diseases.
The goal of this 5-year program is to sustain an Alpha Stem Cell Clinic and to accelerate and complete stem cell-based regenerative medicine trials.
- 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 subjects with suboptimal CD4 levels on cART.
The goal of this project is to develop a treatment for HIV-1/AIDS by rendering blood stem cells permanently resistant to HIV-1 infection.
- Treatment of sickle cell disease by induction of mixed chimerism and immune tolerance using CD4+ T-depleted haploidentical blood stem cell transplant
The aim of this project is to assess the efficacy of hematopoietic stem cell transplantation comprising a CD4+ T-cell-depleted haploidentical hematopoietic progenitor cell product in combination with a radiation-free, non-myeloablative regimen to cure severe sickle cell disease. The goal is to induce mixed chimerism and immune tolerance to donor stem cells in a safe manner.
- Ex Vivo Gene Engineering of Blood Stem Cells for Enhanced Chemotherapy Efficacy in Glioblastoma Patients
The goal of this project is to use genetic engineering to protect blood stem cells from the toxic side effects of chemotherapy in glioblastoma patients, while sensitizing the brain tumor cells to chemotherapy by using a more potent drug regimen.
- CMV-specific T cells expressing anti-HIV CAR and CMV vaccine boost as immunotherapy for HIV/AIDS
The goal of this project is to engineer T cells expressing anti-HIV chimeric antigen receptors (CARs) that will kill reactivated HIV-infected cells in people living with HIV/AIDS. These cells are also engineered to proliferate in response to cytomegalovirus (CMV) exposure. We will use a CMV vaccine to maintain these CAR T cells in the body, even when HIV viremia is low. The goal is to achieve long-term remission in the absence of antiretroviral therapy (ART).
- Production Assistance for Cellular Therapies (PACT) – Cell Processing Facilities
The goals of this project are 1) the development of gene modification and product characterization standard operating procedures (SOPs) to be used in the production of gene-modified CD34+ hematopoietic cells modified via a) self-inactivating lentiviral vectors; b) zinc finger nucleases; ZFN; and c) CRISPR/Cas gene editing; CRISPR); and 2) establishment of Quality Control (QC) test methods for final product release criteria.
- Targeted inhibition of HIV-1 latency
The goal of this project is to develop a cell-targeted approach that delivers small noncoding RNAs (ncRNAs) that can mediate sustained activation of HIV-1 in latently infected cultures.
- Activating cystic fibrosis transmembrane conductance regulator (CFTR): the therapeutic potential of RNA directed gene activation
The goal of this project is to mechanistically determine the function of long noncoding RNAs (lncRNAs) in controlling CFTR and to target particular lncRNAs to activate CFTR expression as a therapeutic.
- Transcriptional control, targeted modification, and excision of HIV in the brain.
The major goal of this project is to develop compounds that transit the blood brain barrier and instill stable silencing and/or excision of HIV.