Since Orange Julius Caesar is the illegitimate love child of Ayn Rand and Benito Mussolini, we've seen (I'm talkin Pruitt, Ryan, et al) the rise of Social Darwinism to never attained heights. Which brings us to this "study".
When curing a disease with gene therapy is bad business
The answer, naturally, is that such science has no business being a business. Especially in the cited cases where the "cures" are for truly rare diseases, and not the faux types which Pharma is jumping into with both feet.
So, where's the moolah going to come from?
Novartis's projections that the treatment could bring in several billion a year in revenue also suggest that the company might charge unheard-of prices, perhaps $2.25 million according to Wall Street bankers.
Society as a whole, through whatever damn gummint insurance exists and "taxing" the privately insured, should shift mountains of money for some basic science?? Time to wake up and smell the coffee. There are public goods, and there's no benefit to piling on gobs of empty profit to the price. The science will out, no matter.