Program Type: Translation
A CIRM Disease Team for the Repair of Traumatically Injured and Arthritic Cartilage
Arthritis is a disabling condition that afflicts 6 million Californians, costing our state nearly $32 billion annually for health care and lost wages. Given the growth of an aging population encouraged to maintain an active lifestyle due to the overall health benefits, the impact of arthritis is expected to increase significantly. For example, the Center […]
A CIRM Disease Team for the Treatment and Cure of Diabetes
This proposal is for the establishment of a group of faculty, staff and industrial partners to develop a proposal for a Diabetes Disease Team. Diabetes is one of the most devastating diseases. Inadequate blood glucose control results on many long term complications including: kidney disease, blindness, amputation and nerve damage. The diabetes epidemic affects almost […]
Clinical Regenerative Wound Healing With Stem Cells
Chronic skin wounds affect a large number of patients every year who suffer from diabetes, burns, venous disease leg ulcers, arterial disease leg ulcers, pressure ulcers, and ulcers from spinal diseases. The annual health care costs for wound care in the United States is $15 billion. The purpose of our CIRM Disease Team Planning Award […]
COVID-19 Antiviral Therapy to Block Direct Cell Injury and Associated Tissue Damage
Translational Candidate Berzosertib (VE-822), a safe drug candidate for treatment against COVID-19, will be investigated. Area of Impact The outcome of the proposed studies will have a significant health benefit to COVID-19 affected patients. Mechanism of Action Our drug candidate, Berzosertib, works as a treatment against COVID-19 by blocking a critical step in virus replication. […]
Purification of Human Hematopoietic Stem Cells (HSCs) for Clinical Stem Cell Transplantation
Translational Candidate Two new monoclonal antibodies anti-CD34 and anti-CD90, and protocols to purify cancer-free and/or T cell free human HSC for clinical transplantation. Area of Impact The development of hematopoietic stem cell-based therapies, starting with rescue of metastatic breast cancer patients with their own cancer-free HSC Mechanism of Action The mechanism is using highly purified […]
Development of a low-cost, clinical-grade iPS maintenance medium for enabling stem cell therapy manufacturing
Translational Candidate HiDef-B8, an iPS cell maintenance medium designed to reduce stem cell GMP manufacturing costs and risk. Area of Impact HiDef-B8 addresses scale-up manufacturing, by being lower-cost ($300/L) and requiring fewer passages per week (1-2 as opposed to 3-5). Mechanism of Action HiDef-B8 underwent extensive empirical optimization and alternative component screening, focused on reducing […]
Generation of human universal donor iPS cells
Translational Candidate Universal donor cell that is a genetically-engineered iPSC clone and is equipped with a safety switch. Area of Impact Regenerative medicine including replacement therapies affected by immune rejection by host immune cells. Mechanism of Action The universal donor cell mitigates immune rejection by host immune cells and is equipped with a suicide gene […]
T-Pure: Peripheral Blood Processing Tool for Point of Care CAR-T Manufacturing
Translational Candidate Tool kit added directly to peripheral blood allowing for purification of T cell enriched product suitable for (CAR)-T cell generation. Area of Impact The goal is to generate a tool that can address one of the most expensive and rate-limiting steps in the production of genetically engineered cells Mechanism of Action Our application […]
Cell Villages and Clinical Trial in a Dish with Pooled iPSC-CMs for Drug Discovery
Translational Candidate Human stem cells in a dish engineered into heart cells to supplement, refine, reduce, and/or ultimately replace human clinical trials. Area of Impact Increase genetic diversity of preclinical studies in human samples to derisk clinical trials and save time and costs. Mechanism of Action We will have several non-invasive human-derived stem cells collected […]
Human iPSC-derived micro-heart muscles for high-throughput cardiac drug discovery
Translational Candidate In vitro miniaturized array of heart muscle amenable for use in efficient high-throughput drug discovery and screening campaigns. Area of Impact Effective high-throughput screening of drugs on human heart muscles does not exist, hindering the discovery of therapeutics to treat heart failure. Mechanism of Action Current approaches for drug discovery often miss a […]