Second shot for shRNA

Having long been demoted to tool compound status, shRNA is gaining renewed attention as a therapeutic modality by offering a simpler path to creating next-generation cell therapies than gene editing. Celyad has jumped in, using Horizon Discovery’s shRNA platform to launch a new arm of its CAR T cell pipeline.

While siRNA has yielded an approved drug and a growing stack of deal activity, short hairpin RNA (shRNA) has become the poor cousin form of RNAi, virtually absent from the rally for oligonucleotide therapies (see “Amplifying Oligos”). ...