© ARTHIMEDES/SHUTTERSTOCK.COMIt’s a compelling narrative: A parent learns that his or her child has a fatal disease with no cure, and, though not a scientist, embarks on a quest to find some treatment. Such stories have played out in the plotlines of films such as Lorenzo’s Oil and Extraordinary Measures, on national morning shows and local news segments, and on crowdfunding pages to drum up support for the cause.
Parent-led funding campaigns to develop gene therapies for rare diseases are especially prevalent, and for good reason. Rather than finding a drug that can fill the void left by a protein lost to a single-gene disorder, gene therapy holds the promise of replacing the defective gene itself—of a cure. Just one of the thousands of single-gene disorders has an FDA-approved gene therapy, but through hard work and determination, some parents hope to change that.
Some crowdfunding campaigns have been astoundingly successful: they’ve raised millions of dollars to fund basic research and, later, clinical trials that have likely saved children’s lives. Donations can, however, only carry a therapy so far before a pharmaceutical company must grab the baton—an outcome that’s not guaranteed, even when a gene ...
