New CRISPR Center brings hope for rare and deadly genetic diseases

January 22, 2024

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(link is external), MD, a pediatrics professor who directs the UCSF Jeffrey Modell Diagnostic Center for Primary Immunodeficiencies. “All patients deserve a sense of urgency – including those with rare diseases, many of whom are children.”

Since the CRISPR platform being created at IGI could, in theory, be reprogrammed to address any gene mutation, the goal is to use treatments for HLH and ART-SCID as models to develop a scalable approach from which new medicine for other genetic diseases can be rapidly developed.

“The unique nature of CRISPR makes it ideal for developing and deploying a platform capability for CRISPR cures on demand,” said Fyodor Urnov, IGI’s Director of Technology and Translation, who is overseeing the project along with Jennifer Doudna and IGI Executive Director Brad Ringeisen. “Danaher and the IGI are in a unique position to potentially create a first-of-its-kind CRISPR cures ‘cookbook’ that could be used by any team wishing to take on other diseases.”

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