Individual rare diseases are... rare. But taken together, they affect a significant fraction of the population. We need to develop a sustainable economic model for gene therapy.

"As a result, while the number of people eligible for any single gene therapy might be small, the total number of people eligible across all of them could soon be significant. Annual spending on gene therapies could increase from $5.15 billion 2020 to $25.3 billion in 2026."

https://www.bloomberg.com/opinion/features/2023-10-15/sickle-cell-gene-therapy-will-test-how-us-pays-for-million-dollar-cures?srnd=undefined