A new indication-by-indication analysis is reshaping how the gene therapy field thinks about recombinant AAV (rAAV) manufacturing costs and capacity. The study assesses six leading targets—RPE65 retinal dystrophy, age-related macular degeneration (AMD), hemophilia A and B, spinal muscular atrophy (SMA), and Duchenne muscular dystrophy (DMD)—using prevalence-based patient estimates and dosing assumptions tied to each indication. The report’s key output is a cost curve that varies by disease category and assumed dose intensity, alongside capacity implications for how suppliers may need to scale. For manufacturers and payers, the framework reframes “one-size-fits-all” budgeting by anchoring planning to real-world indication profiles rather than average utilization. The work also points to cost-cutting levers embedded in the manufacturing supply chain, including strategies to reduce waste and improve throughput—pressing issues as the field expands beyond first-wave approvals and toward broader patient populations.
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