Building the Infrastructure for Made to Order Gene Therapies

The case of baby KJ Muldoon, an infant born with a lethal genetic metabolic disorder, demonstrates the potential to compress years of therapeutic development into months using an in vivo base editing approach. Jeff Coller, director of the Johns Hopkins RNA Innovation Center, wrote about the case in a New York Times op-ed, arguing that CRISPR-based base editors—delivered via lipid nanoparticles as mRNA molecular surgery payloads—could be generalized to thousands of monogenic disorders. He further explains that realizing this promise will be constrained less by scientific limitations than by regulatory, manufacturing, and reimbursement challenges. Coller also outlines what it will take to make bespoke therapies economically and operationally viable, and argues that failure to scale this approach would reflect not a failure of science, but of imagination.