Until 5-10 years ago, pharma companies were vertically integrated players doing everything from early stage research to commercialization. Then they realized they were no good at research and stopped doing it, instead buying companies like avexis to do research, and then using their commercial and clin reg engines to get products to patients

No one knows whether the gene therapies will actually be one shot cures. we know they'll be one shot, because the immune system will reject the viral vectors after the first dose. We just don't know if they'll be curative. If not, then the investments in gene therapy platforms will look quite different