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.
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.