The UCSF Alpha Clinic has focused its efforts on three important areas over the past year: bolstering the pre-clinical regulatory support provided by Alpha Clinic staff for investigator-initiated cell and gene therapy projects at UCSF, promoting the CRISPR gene editing toolbox as an Alpha Clinic network-wide resource for investigator-initiated research and clinical trial development through a partnership with the Innovative Genomics Institute at UC Berkeley, and to improve access to clinical trial participation through a collaboration with the Clovis Community Medical Center in establishing a fellowship/junior faculty training opportunity that would embed a clinician with cell and gene therapy expertise in that community setting. Together these activities should advance scientific progress and also improve access to these new therapies in California.
Reporting Period:
Year 2
The UCSF Alpha Clinic continues to focus its efforts on three important areas: establishing a Regulatory Affairs Unit that combines clinical, pre-clinical and manufacturing support for product development journey for investigator-initiated cell and gene therapy projects at UCSF, promoting patient outreach and education strategies using social media, and to improve access to clinical trial participation by collaborating with the Clovis Community Medical Center to establish a fellowship/junior faculty training opportunity that would provide a clinician with cell and gene therapy expertise in this community setting. Together, these activities should advance scientific progress and improve access to cell and gene therapies in California.
The UCSF Alpha Clinic has also expanded the scope of disorders represented in its clinical trial portfolio including new investigator-initiated studies that target multiple sclerosis, rheumatological disorders, Type-I diabetes, Artemus-SCID, and alpha thalassemia major. In addition, new industry-sponsored clinical trials in Parkinson's Disease, mucopolysaccharidoses, inflammatory bowel disease, and seizure disorders have been supported in the past year. Thus, the robust foundation of support in malignant disorders by the UCSF Alpha Clinic has been successfully broadened to include a broad array of non-malignant disorders.
Grant Application Details
Application Title:
University of California, San Francisco (UCSF) Alpha Stem Cell Clinic (ASCC)
Public Abstract:
We propose cell and gene therapy trial infrastructure that will focus on hereditary hematological and immunologic disorders and on anti-inflammatory lung disorders. This alpha stem cell clinic operation at UCSF will expand access to these novel cell and gene therapies to California citizens .
Statement of Benefit to California:
The objective of this proposal from the Univ. of Calif., San Francisco is to expand the opportunity for California citizens to receive cellular therapies that have curative potential, especially in under-represented populations and diseases. We will accomplish this by giving therapy that can cure hereditary blood disorders, highlighted by a first-in-human, CIRM-funded CRISPR gene editing trial in sickle cell disease, and in treating lung inflammation from trauma and infections such as COVID-19.
