A groundbreaking milestone in genetic medicine has been reached at the Children’s Hospital of Philadelphia, where a baby with a devastating genetic condition received the world’s first personalized CRISPR-based gene therapy. This therapy was uniquely tailored to correct the child’s specific disease-causing mutation.
Crucially, the treatment was delivered using lipid nanoparticles (LNPs) developed by Acuitas Therapeutics—a company co-founded in 2009 by Dr. Pieter Cullis, a professor in our Department of Biochemistry and Molecular Biology (BMB). Dr. Cullis is internationally recognized as a pioneer in LNP research and development. His foundational and translational work in this field laid the essential groundwork for this medical breakthrough.
This landmark success marks a pivotal moment in precision medicine, opening the door to the treatment of many other monogenic rare diseases. The Department proudly congratulates Dr. Cullis for his visionary contributions that continue to shape the future of genetic therapies.
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Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease
