Children and adults with rare, deadly genetic diseases have fresh hope for curative therapies, thanks to a new collaboration between the Innovative Genomics Institute (IGI) and Danaher Corporation, a global life sciences and diagnostics innovator.
The new Danaher-IGI Beacon for CRISPR Cures center will use genome editing to address potentially hundreds of diseases, including rare genetic disorders that have no cure. The goal is to ensure treatments can be developed and brought to patients more quickly and efficiently.
The IGI comprises genetics researchers and clinician experts from three University of California campuses: UCSF, UCLA and UC Berkeley, where the institute is housed, as well as other research institutions. Danaher will provide tools, reagents, resources and expertise to accelerate preclinical and clinical development and establish new standards for safety and efficacy.
The center will work first on CRISPR treatments for two genetic defects of the immune system: familial hemophagocytic lymphohistiocytosis (HLH), which causes immune cells to become overactive, damaging tissues and organs throughout the body; and Artemis-deficient severe combined immunodeficiency (ART-SCID), in which T and B lymphocytes fail to mature, making infants vulnerable to fatal infections.
The standard treatment for both conditions, a bone marrow transplant, is inadequate due to frequent complications.
“With CRISPR, we can speed up the development of improved therapies that can reach all the patients who need them,” said Jennifer Puck, MD, a pediatrics professor who directs the UCSF Jeffrey Modell Diagnostic Center for Primary Immunodeficiencies and is a faculty member at UCSF’s Institute for Human Genetics. “All patients deserve a sense of urgency – including those with rare diseases, many of whom are children.”