Project Objective: Tool/Resources/Bottleneck
Laboratory for Cell and Gene Medicine: A partner in the California Cell and Gene Therapy Manufacturing Network
The Stanford LCGM supports process development and manufacturing for investigator initiated and select industry partner clinical trials. The objective of our proposal is to complete our project plan and enhance the LCGM value proposition positioning us competitively for the phase two funding period. A major roadblock in stem cell therapies is confirming efficacy in patients. […]
A comprehensive biomanufacturing center solving bottlenecks in cell and gene therapy manufacturing to accelerate new therapies for California patients
We aim to considerably expand access for all Californians to outstanding cell and gene therapy treatments for multiple patients. With operational enhancements to develop diverse talent from underserved communities, our GMP facility will advance all aspects of biomanufacturing within the network. As part of the Cell and Gene Therapy (CGT) Manufacturing Network, we will bring […]
The UCI GMP Cell and Gene Therapy Manufacturing Facility
To contribute to a network that will de-risk pathways to commercialization for cell and gene therapies and develop a diverse and skilled manufacturing workforce in California, we propose to develop and implement several key activities to meet these goals over the two years of this project proposal. This project will have a significant public impact […]
UCSD Advanced Cell Therapy Laboratory
The project plan outlines key operational enhancements, development of specialization areas, and support for new workforce development initiatives that will enable our GMP cell manufacturing facility be a key component of the new CIRM Cell and Gene Therapy Manufacturing Network. Support for this project will enable our facility to expand our capabilities and workforce development […]
Mapping and modeling endothelial cell fate decisions for pulmonary arterial hypertension
Research Objective We will build a foundational model and experimental platform to catalog all genes that promote and protect against PAH, with the potential to extend to many other developmental and disease processes. Impact The studies aim to identify genetic targets for PAH therapy and develop a predictive model to accelerate stem cell research and […]
An interactive data resource for hypothesis testing in stem cell single-cell gene expression and validation of the results with brain organoids
Research Objective We are building a "virtual molecular microscope" where anyone can quickly visualize a very recent, high-throughput molecular assay, single-cell RNA-seq and spatial gene expression studies Impact Currently, a lot of data has been published, hundreds of datasets on the cerebral cortex alone, but it takes hours to convert the datasets and look at […]
Characterization and applications of human blastoids for understanding early human embryogenesis
Research Objective Our work will yield an improved stem-cell based embryo model that we will explore with various omics approaches and genetic screens to gain insights into the pathways that control human embryos. Impact An improved stem cell-based embryo model is a crucial step for in-depth studies of human development and will enhance our ability […]
Overcoming barriers for airway stem cell gene therapy for Cystic Fibrosis
Research Objective This research will allow the targeting of airway stem cells for long lived gene therapy for Cystic Fibrosis and for other airway diseases Impact We will overcome the barriers to accessing airway basal stem cells for gene correction for Cystic Fibrosis (CF) and use a new gene correction strategy to correct >99% of […]
Modeling Retinitis Pigmentosa using patient-derived human iPSC organoids
Research Objective The objective of this proposal is to develop a human retinal organoid model of adRP to gain insights in pathogenesis and assess clinically relevant approaches to restore RHO protein function. Impact Upon successful completion of this study, we will have established a disease-in-a-dish model and a novel therapeutic approach towards management of the […]
Engineering pluripotent stem cells for universally available, off-the-shelf T cell therapies
Research Objective Our goal is to develop new gene editing methods for producing universal, off-the-shelf, therapeutic T cells from induced pluripotent stem cells (iPSC) that can be applied to a range of diseases. Impact We will develop new gene editing approaches to overcome the block to T cell development from iPSC that occurs when key […]