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
Last week Novartis placed a $8.7B bid on AveXis. ($218 per share, an 88% premium). Manufacturer of a miracle MS therapy for young children. A move that could increase the price to over $2.5M per treatment.
When curing a disease with gene therapy is bad business
https://www.technologyreview.com/s/610873/the-gene-therapy-t...
Amazingly, as outlined in Goldman's note, that lifetime cash flow a pharma developer relies on could be cut short. By "cured" patients no longer requiring long term pharmaceutical therapy!
It makes the space very interesting to watch right now. Particularly as much of the regulation centers upon health or geopolitical concerns. And not on markets or affordability.
My guess is we will see the rise of behemoth-sized global vertically integrated players. Controlling every step of the process. From Research to therapy development. Through patient acquisition. And including the clinical administration of one-shot "cures" in a futuristic branded setting.
Very much resembling something akin to Weyland Industries or Tyrell Corporation. From hollywood sci-fi dreams.