Overview

• The Chan Zuckerberg Initiative and Innovative Genomics Institute have funded the Center for Pediatric CRISPR Cures to advance in vivo treatments for ultra-rare childhood genetic disorders.
• The initiative unites CRISPR design at UC Berkeley’s Innovative Genomics Institute with clinical administration at UCSF to build an end-to-end therapy pipeline.
• Multidisciplinary teams at UC Berkeley, UCSF and industry partners including Danaher’s IDT, Aldevron and Cytiva are developing, manufacturing and delivering bespoke CRISPR therapies.
• Building on last month’s success treating Baby KJ Muldoon with personalized base editing, researchers will treat a first cohort of eight pediatric patients.
• Trial protocols, manufacturing workflows and regulatory submissions for a platform-based designation are being finalized to reduce repetitive animal testing and speed approvals.