News Article

CRISPR cures: The path to patient impact

April 3, 2025
A little child plays with their dog in the desert

One year into our collaboration with the Innovative Genomics Institute we’re making strides towards delivering on our promise to create transformative solutions to speed potentially curative CRISPR-based treatments for children with rare inherited immune deficiencies.

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A healthy immune system is essential for survival, but a single genetic mutation can cause serious – and often deadly – disease. One of the most well-known of these diseases is ARTEMIS-SCID (ART-SCID), also known as “Bubble Boy Disease,” where mutations in the gene encoding the Artemis protein result in babies born with no T and B cells, two essential components of the immune system. ART-SCID is just one of over 500 Inborn Errors of Immunity, or IEIs. While each individual IEI is rare, when combined they impact over 112,000 patients.

Danaher and IGI collaborators Dr. Morton Cowan, MD, and Dr. Jennifer Puck, MD, both Pediatrics Professors at UCSF, have successfully used gene therapy to treat ART-SCID.

The question now is: How can we use CRISPR to create a replicable, scalable treatment to help all of the children born with IEIs?

The collaborators at Danaher and the IGI have spent the last year laying the groundwork to update the current development, manufacturing and regulatory systems – which are far too slow and costly – with a standardized CRISPR platform that could theoretically be reprogrammed to treat nearly any gene mutation responsible IEIs. Work has commenced to address the multifaceted challenges of developing such a platform, including optimizing workflows, connecting to patient communities and engaging with regulatory bodies.

Now moving into its second year, the collaboration continues progressing towards our goal of creating a new model for future development of a wide range of genomic medicines. 

Stay tuned for more updates on delivering these impactful medicines to these deserving young patients.