Therapeutic/Technology: Therapeutic Approach
Identifying a lead compound for COVID19 using high throughput screening with lung stem cell organoids
Research Objective We propose to use a lung stem cell based organoid to identify a new compound for COVID-19 by screening a library of FDA approved compounds that could be repurposed for COVID-19 infection. Impact If successful, we will find a therapy to treat COVID-19 infection and prevent the lung complications that are so deadly. […]
Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell Disease
Therapeutic Candidate or Device Autologous CD34+ cells transduced ex vivo with the BCH_BB694 LCR(-HS4) bGp D12shmiR lentiviral vector Indication Sickle cell disease with severe phenotype. Therapeutic Mechanism Silencing of beta-sickle globin and induction of anti-sickling fetal hemoglobin Unmet Medical Need Sickle cell is a severe disease with protean manifestations. The only curative therapy is an […]
Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease
Therapeutic Candidate or Device CRISPR corrected blood stem cells are manufactured from persons with severe sickle cell disease and returned by transplant to the same person Indication Persons with sickle cell disease (adults and adolescents) with repeated, severe painful and lung events are eligible for the clinical trial Therapeutic Mechanism Blood stem cells are harvested […]
A Phase 1 Study of ECT-001 Expanded Cord Blood and Myeloablative Regimen with Reduced Toxicity in Patients with Severe Sickle Cell Disease.
Therapeutic Candidate or Device ECT-001 graft contains more stem and immune cells than conventional cord blood graft, leading to prompt recovery and better outcomes for patients. Indication Severe Sickle Cell Disease Therapeutic Mechanism Hematopoietic stem cell transplantation is the only cure for severe sickle cell disease. The ECT-001 expanded cord blood cells will replace the […]
Simplification of Excipient Solution for Implanting Candidate Human H9-scNSC Cell Line for Spinal Cord Injury
Stimulating endogenous muscle stem cells to counter muscle wasting
Human neural stem cell (hNSC) derived exosomes vs CSC14 hNSCs for the treatment of traumatic brain injury (TBI)
Evaluation of Gene Therapy Approaches for Autosomal Recessive Hyper IgE Syndrome Due to Mutations in DOCK8
Global gene expression profiling of single inhibitory neurons derived from human stem cells
University of California, San Francisco (UCSF) CIRM Alpha Stem Cell Clinic
We propose a cellular trial operation at UCSF that will focus on childhood hereditary hematological and immunologic disorders and in anti-inflammatory lung cellular therapies. This alpha clinic operation at UCSF will expand participation by California citizens in these novel clinical themes. The objective of this proposal from the Univ. of Calif., San Francisco is to […]