[jfarlow]

Part of the beauty of these kinds of gene therapies is that the cost can ultimately be lowered to near-zero levels. These are not drugs that must be taken over a lifetime, but can potentially provide 1-time delivery of the instructions to your own body about how to cure itself. Certainly in the near term the R&D must be paid for (and the marketing, and regulatory burden, and everything else). But in the long term the actual material used to change someone's life forever can cost pennies and a one-time delivery.