Regenerative medicine is still in its early stages. But the science, many analysts agree, has the potential to change the course of healthcare. Instead of constantly treating a disease, regenerative medicine zeroes in on the underlying cause of an ailment and fixes it for good.
"That's why people are excited about it and why investors are flocking to it," said Matt Phipps, a William Blair analyst, who covers the biotech sector. "If you have the ability to cure a patient, that's going to make other drugs obsolete. And that changes a lot of those markets."
Take hemophilia. A hemophilia patient may need two to three infusions a week of a protein they lack to prevent them from bleeding. Those drugs can cost $300,000 to $400,000 a year, or millions over a lifetime; a one-time regenerative cure could cost a million dollars, Phipps says.
Researchers are currently testing therapies for hemophiliacs to fix a faulty gene with such one-time cure-all remedy. Biotech companies Spark Therapeutics and BioMarin are each conducting clinical trials in hemophilia with impressive results seen to date, including little to no infusions needed for the patients treated over the course of one to two years.
New therapy for vision loss
Spark also received FDA approval in late 2017 for a new gene therapy, Luxturna, to treat children and adults with an inherited form of vision loss that may cause blindness. Luxturna is the first gene therapy approved in the United States that targets a disease caused by mutations in a specific gene.
"What's impressive is the therapeutics getting approved now are seen as late-stage, first-generation products and part of a larger kind of growth and expansion in this field," says Raju Prasad, an analyst and member of our biotech research team.
"What's next? What's coming? That's what gets me excited," Prasad says. "There are tons of private companies, some public companies that are already working on bigger, better versions of these products."
A year ago we expanded our biotech research coverage to provide clients with a deeper level of investment insights on companies leading such regenerative innovation.
"We are committed to providing our clients best-in-class service and insights into this vital sector," said John Moore, head of institutional equities and research.
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