Vectorized biologics: are patients and payers ready to swap biologics for gene therapies?

Gene therapy can be more than a niche solution to a rare genetic disease. That’s the belief of a set of gene therapy developers repositioning the modality as a vehicle for once-and-done administration of an array of biologic drugs. The clinical data are promising. The next hurdles will be gaining patient, physician and payer buy-in.

All gene therapies marketed in the U.S. align with the initial vision for the modality, which centered on treating genetic diseases by delivering a functional, or even optimized, version of a patient’s mutated gene. That straightforward biological rationale — replace a broken gene with a good one — combined with the high risk tolerances of patients with severely debilitating rare diseases, made genetic disorders a natural fit for the new modality. ...