The approvals of Roche’s Luxturna (2017) for inherited retinal disease and Novartis’ Zolgensma (2019) for spinal muscular atrophy are indicative of a resurgence in the gene therapy field after a decline due to the death of a patient in 1999. The growth is reinforced by a 2019 US Food and Drug Administration (FDA) statement indicating that starting in 2020, the agency was expecting to see greater than 200 Investigational New Drug (IND) applications per year and projecting ten to twenty cell and gene therapies would be approved annually by 2025. Due to the “promise” of possibly being curative, gene therapies often receive expedited designations from regulators, including accelerated approval.
