Programmable therapeutics

Technological breakthroughs are ushering in the era of programmable therapeutics—a new class of modular, gene- and cell-based medicines that can be designed, adapted, and redeployed to address a wide range of conditions. Rather than simply treating disease symptoms, programmable therapeutics aim to correct disease-causing changes in DNA, direct cells to produce therapeutic proteins, or rewire cellular pathways that drive disease.

Many of the technologies enabling this new era were developed by Broad scientists, including Feng Zhang, a pioneer in CRISPR technologies, and David Liu, who developed base editing and prime editing. They and other Broad researchers, including Xiao Wang, are continuing to expand the toolbox of gene- and cell-based technologies to improve scalability, precision, and accessibility across a wide range of conditions, ranging from genetic disorders to aging-related illnesses. Broad researchers are also pioneering new systems to deliver these medicines—one of the major hurdles for these therapeutics, especially in regards to overcoming the blood–brain barrier.