Building the Infrastructure for Made to Order Gene Therapies

The case of baby KJ Muldoon, an infant born with a deadly genetic metabolic disorder, demonstrates the potential to compress what would normally take years of development into months, using an in vivo base editing therapy to save a child. Jeff Coller, director of the Johns Hopkins RNA Innovation Center, who wrote about the case in an op-ed in The New York Times, argues that CRISPR-based base editors—delivered via lipid nanoparticles as an mRNA molecular surgery payload—could be generalized to thousands of monogenic disorders. Coller discusses why that promise will be constrained less by science than by regulatory, manufacturing, and reimbursement challenges; what it will take to make bespoke therapies economically and operationally viable; and why failing to scale this approach would represent a failure not of science, but of imagination.