Gene therapy and pharmacoeconomics
For gene therapy, production has become an unsolvable task
Alexander Noskov, Hi-tech+
Promising medical startups promising therapy for incurable diseases have attracted about $13 billion. In laboratories, they show amazing results, but literally everyone is at risk of burning out because of an insurmountable obstacle - production.
Gene therapy is associated with progress in the treatment of many incurable diseases, including Alzheimer's disease. The first drugs cost hundreds of thousands of dollars, and the industry itself has attracted about $13 billion. However, according to Business Insider, the entire industry could turn into a bubble. Now scaling production is an unsolvable task.
Gene therapy is a set of techniques for "tuning" body cells to resist certain diseases. The first drug approved in the United States, Onpattro, is designed to combat transtiretin amyloidosis. It works on the basis of the RNA interference technique, and a one-year course will cost $450,000.
Unfortunately, this price reflects not only the unique nature of the offer, but also the difficulties in scaling.
Tony Khuri from the consulting company Project Farma describes the problems of the industry, resorting to analogy. Imagine that you are baking delicious pies for relatives – stirring the dough with your hands and in the kitchen oven. If an order for 10,000 buns arrives, all these processes will have to be changed, but this is at least possible for most recipes. However, in gene therapy, a way to "scale the recipe" has not yet been invented. As well as a way to accelerate the synthesis of targeted viruses.
This is the main reason that startups are more often looking for ways to cure rare diseases. And also the fact that gene therapy risks never becoming a "weapon of choice". In the meantime, the approach remains unusual, regulators impose the most stringent requirements. This means a ten–year development cycle - and a corresponding price tag in case of success, which prevents offering therapy to more patients. The circle closes.
However, while deals worth hundreds of millions and billions of dollars are being concluded on the market – and, apparently, the pharmaceutical giants hope to break the vicious circle and bring the technology to light.
BI provides the most illustrative examples in its material. London-based MeiraGTx has entered into a $440 million partnership with Johnson & Johnson, intending to explore new production methods. The Swiss pharmaceutical giant Roche bought Spark Therapeutics for $5 billion. And the creators of the company Paragon Bioservices received $ 1.2 billion from the drug manufacturer Catalent.
Such amounts also reflect not only prospects, but also prices in this industry. For example, it costs about $7.4 million to rent facilities on the side and synthesize drugs to start clinical trials. However, as Mustang Bio's top manager Knut Niss points out, it's still half as cheap as organizing your own production.
Optimists believe that a turning point will definitely come – it is only necessary to confirm the effectiveness of therapy. They remind of the difficult road to the medical market of monoclonal antibodies (MAB). Initially, manufacturers encountered more and more barriers in an attempt to establish a stable release, and now MAB is the basis of a number of cancer drugs that are sold worldwide.
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