Gene therapy’s next frontier lies beyond rare, monogenic diseases

As in vivo gene therapies continue notching approvals for rare monogenic diseases, the next frontier for the modality will be in treating complex indications without obvious genetic drivers. At least seven companies have clinical or preclinical gene therapies for non-monogenic diseases, with a flurry of data expected this year.

Two in vivo gene therapies have been approved since 2017, Luxturna voretigene neparvovec-rzyl from Spark Therapeutics Inc. and Zolgensma onasemnogene abeparvovec-xioi from Novartis AG (NYSE:NVS; SIX:NOVN). Another eight are in Phase III or Phase II/III testing. All address rare monogenic diseases where a single gene is mutated, and the goal is to deliver a functional copy of the gene to compensate for the defective one (see “AAVs on the Brink”)...