Welcome to the Age of Programmable Medicine

A recent clinical breakthrough has me more excited than ever about the future of medicine. In a world-first, physicians at the Children’s Hospital of Philadelphia (CHOP) used CRISPR gene editing to treat an infant with carbamoyl phosphate synthetase 1 (CPS1) deficiency, a rare and typically fatal disorder that disrupts the urea cycle and causes toxic ammonia buildup. The team designed a bespoke guide RNA targeting the child’s specific mutation in the CPS1 gene and delivered the CRISPR components directly to the liver using lipid nanoparticles. The result: a corrected gene, a restored metabolic pathway, and a life saved. Voilà, gene therapy  CRISPR-style.

This milestone is not an isolated event, it’s part of a broader revolution underway in medicine. Just over a decade ago, the first patient was successfully treated with CAR T-cell therapy (also at CHOP). More recently, mRNA vaccines famously transformed the global response to COVID-19. While gene therapy, cell therapy, and RNA-based medicine are often treated as distinct fields, they are rapidly converging into a single, powerful paradigm: reprogramming cells within the body to perform therapeutic functions on demand.

To understand how this new approach differs, it helps to consider what today’s technologies typically do. Gene therapies deliver functional copies of genes to correct rare mutations. CAR T-cell therapies involve removing immune cells, engineering them outside the body to target disease (usually cancer) and reinfusing them. mRNA vaccines hijack one's own body and turn it into a bioreactor to produce specific, designed proteins such as viral antigens.

Now that we’ve demonstrated each of these capabilities independently, the real question is: what happens when we start combining them—using mRNA to generate CAR T cells directly in vivo, CRISPR to reprogram B cells to produce designer antibodies, and advanced delivery systems to target cells wherever you’d like them to go in the body?

Today, cell therapies like CAR T, stem cell transplants, and even a recent case of Type 1 diabetes remission all rely on ex vivo manipulation which involve complex, expensive, and logistically intensive procedures. But the future of in vivo cell engineering is arriving quickly. New research has shown that gene therapy can reprogram blood stem cells inside the body. Companies founded by Weissman and Jennifer Doudna are pioneering CRISPR- and mRNA-based approaches to engineer immune cells in vivo. Personalized cancer vaccines are using mRNA to train the immune system to recognize patient-specific tumor mutations.

What excites me about the news of a successful CRISPR treatment is what it signals for the future of all medicine. These advances are not isolated; they are additive. CRISPR, mRNA, cell reprogramming, and next-generation delivery technologies are becoming part of a shared toolkit. The most transformative therapies of the next decade will not come from any single field. They will be built at the intersection. We are entering an era where medicines are no longer just administered; they are programmed.