Making Personalized Crispr Cures to Save More Babies
New Center Aims to Scale Personalized CRISPR Cures for Children with Rare Genetic Diseases
Bloomberg covers the launch of the Center for Pediatric CRISPR Cures – a $20M initiative that could transform how rare genetic diseases are treated in infants and children. The center builds directly on breakthrough work by Dr. Jennifer Doudna’s Innovative Genomics Institute (IGI).
The IGI played a critical role in developing and ensuring the safety of the first-ever personalized CRISPR gene-editing therapy—the treatment that saved baby KJ Muldoon, born with a fatal metabolic disorder. The IGI CRISPR Cures team at UC Berkeley, led by Dr. Fyodor Urnov, co-developed the therapy, which Bloomberg notes could normally cost $15 million per patient and take three years to develop.
The new center will bridge Doudna’s IGI team with clinical treatment at UCSF. The goal: create a standardized “cookbook” approach to personalized CRISPR treatments – reducing development time to 4-5 months and dramatically cutting costs.
With appropriate confidentiality protections, the Center will make data from investigative new drug studies, regulatory documentation, and clinical trials available to other U.S. academic centers developing similar CRISPR-based therapies.
Eight children with severe metabolic disorders and inborn errors of immunity will be the first patients. If successful, the model could scale to address hundreds of rare genetic diseases – each with treatments precisely customized to a child’s unique genetic mutation.
For parents who’ve received desperate emails asking if their child’s condition might be treatable, the center offers something previously out of reach: a realistic pathway to personalized cures.
