A new center in San Francisco will offer tailor-made CRISPR therapies to cure children with rare diseases. The Center for Pediatric CRISPR Cures, announced in June, brings together pediatrician Priscilla Chan, co-founder of the Chan Zuckerberg Initiative (CZI), and Nobel laureate Jennifer Doudna of the University of California, Berkeley. The new center builds on the successful treatment whereby baby KJ was cured of a rare and life-threatening metabolic disorder caused by carbamoyl phosphate synthetase 1 deficiency using a personalized CRISPR therapy. With $20 million in support from the CZI, the new center aims to extend CRISPR’s therapeutic impact to thousands of rare Mendelian disorders.
As a first step towards establishing a standardized process for in vivo gene editing therapies, eight children will enroll in a clinical trial to receive a custom-made CRISPR therapy. Each treatment will require US Food and Drug Administration (FDA) approval. However, as more information on the safety and potential risks of CRISPR for different indications is gathered, the FDA could recognize CRISPR as a platform technology that can be applied across multiple drug candidates, greatly reducing the time and cost associated with developing these therapies.
