The rapid development of milasen, a bespoke therapy for a single patient with a rare genetic disease described in a landmark New England Journal of Medicine article, suggests how the US Food and Drug Administration’s regulatory framework for new drugs will need to stretch to accommodate the challenge of individualized genomic medicine.
The splice-modulating antisense oligonucleotide (ASO) milasen offers a “possible template for the rapid development of patient-customized treatments,” study authors Jinkuk...