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Genetic therapies are starting to enter the market to cure previously incurable diseases: a great hope for patients, but a huge challenge for health systems because of their costs, especially if their use becomes widespread
These therapies usually consist of replacing directly in the body (in vivo) a defective gene with a functional gene or taking cells for genetically modifying them in the laboratory before returning them to the patient (ex vivo). [19659002] This biotechnology segment could generate $ 363 million in revenue by 2022 globally, according to a recent study by Research and Markets.
This would still be a drop in the colossal global pharmaceutical market. prescription drug sales could exceed $ 1 trillion in 2022, according to Evaluate Pharma.
But if the first gene therapy treatments have targeted rare and monogenic diseases (involving a single gene), biopharmaceutical research is bubbling in this area, with more than 2,200 clinical trials in progress worldwide, including in mbad pathologies such as cancer , diabetes and neurodegenerative diseases
It is particularly in cancer that an indirect strategy of gene therapy, CAR-T cells, is currently making great strides.
– "Like a missile" – [19659002] This technology consists in genetically reprogramming a category of immune cells, T cells, to "arm them like a missile" to enable them to detect and kill cancer cells, summarizes AFP Antoine Papiernik, president of Sofinnova , a venture capital firm specializing in the life sciences
Swiss biopharmaceutical giant Novartis led the way in August 2017 by winning the approval of a first CAR-T product, Kymriah, targeting a rare form of leukemia
Major maneuvers in the pharmaceutical industry have not been slow: just after the approval of Kymriah, the American Gilead put in hand for nearly $ 12 billion on the Kite Pharma T CAR specialist, imitated a few months later by Celgene with the acquisition of Juno Therapeutics for $ 9 billion.
At the end of June, Kymriah and Yescarta, Gilead's CAR-T drug targeting lymphatic cancer, have also been approved in Europe. In the United States, their list prices are dizzying: $ 475,000 per patient for Kymriah, $ 373,000 for Yescarta.
Because the current complexity of their manufacture, from autologous cells (taken from the patient himself), swells their costs and also cast doubt on their use on a large scale.
"For now these treatments target only blood cancers, but they give impressive results.The next stage of the CAR-T, targeting solid tumors, looks more complicated ", according to M. Papiernik.
– A model of groping activity –
To industrialize their use, it would also be necessary to find a way to use healthy donor cells (cells so-called "allogeneic"), an obstacle on which many biotechs work, including the French Cellectis or the American Allogene Therapeutics.
The power of CAR-T is also double-edged, because "the enormous resurgence the immune system they trigger can kill the patient, "still warns Mr. Papiernik.
Used as a last resort, gene therapies have struggled so far to find a viable business model, because of price
Thus the pioneer Glybera, launched in Europe in 2012 to treat a genetic disease of the pancreas and sold a million dollars per patient, was a resounding commercial failure. To the point that its manufacturer, the Dutch biotech UniQure, removed it from the sale last year.
As for the British GSK, he preferred to transfer in April his gene therapy portfolio (including the Strimvelis for "baby-bubbles") to the specialized biotech Orchard Therapeutics.
To pre-empt criticism of the prices of these Some manufacturers use atypical business practices for the industry.
Thus, in early 2018, US biotech Spark Therapeutics is committed to reimbursing payers for non-responders at Luxturna, its gene therapy targeting degeneration hereditary retina and sold $ 850,000 for both eyes in the United States. An approach inspired by Novartis' similar commitment to Kymriah.
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