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…
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…
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…
Research Objective This DISC2 tests a strategy to target survival and self-renewal in glioma stem cells (GSC), and will identify a therapeutic candidate for safety, dosing and efficacy testing. Impact…
Research Objective This proposal will develop a cell-based therapy that can restore vision in retinal degeneration associated blindness such as Stargardt disease and age-related macular degeneration. Impact Chemically induced method…
Research Objective Development of a CRISPR genome editing therapy for ALS caused by C9orf72 and the delivery vehicle required to bring this novel therapy to patients. Impact A cure for…
Research Objective With a single administration to an individual, lentiviral vectors that selectively transduce B cells in vivo and express highly potent anti-HIV-1 proteins to suppress HIV-1 replication throughout life…
Research Objective The therapeutic candidate we aim to discover under this award is clomipramine, an antidepressant that might help reduce a harmful protein in the nerve cells of patients with…
Research Objective This personalized chimeric antigen receptor (CAR) T cell therapy will attack a wider range of cancer cells, and block cancer's defense mechanisms to empower the immune system to…
Research Objective To discover CRISPR-based genetic strategies that will directly and therapeutically modify cardiac myosin heavy chain isoform expression to treat HF and cardiomyopathies. Impact Heart Failure and Cardiomyopathies Major…
