Will business stand in the way of gene therapy? Photo : Penn Medicine

Recent advancements in science and medicine have put cures within reach for diseases that have long plagued humankind.


In a recent report, though, Goldman Sachs analysts posed an uncomfortable question that quickly sparked criticism.

“Is curing patients a sustainable business model?” analysts asked in an April 10 report entitled “The Genome Revolution,” which CNBC first reported.


One-and-done cures enabled by gene editing, analyst Salveen Richter wrote in the note to clients this week, are near-miraculous innovations that stand to benefit patients immensely.

But they also present a challenge to business.

“While this proposition carries tremendous value for patients and society, it could represent a challenge for genome medicine developers looking for sustained cash flow,” she wrote.

The trouble is, it’s hard to reap long-term profits when you’re actually curing the patients who would buy your treatment.

The report points to Gilead’s treatments for hepatitis C, which cure more than 90 percent of patients. In 2015, at the height of sales, US sales of the drug hit $12.5 billion. But as people have been cured, sales have fallen. Goldman estimates US sales for Gilead’s hepatitis C treatments will be less than $4 billion this year.


“[Gilead] is a case in point, where the success of its hepatitis C franchise has gradually exhausted the available pool of treatable patients,” the analyst wrote. “In the case of infectious diseases such as hepatitis C, curing existing patients also decreases the number of carriers able to transmit the virus to new patients, thus the incident pool also declines… Where an incident pool remains stable (eg, in cancer) the potential for a cure poses less risk to the sustainability of a franchise.”

Cutting-edge gene therapies, which are often pitched as one-time cures and on top of that often target small groups of patients, present the same risk.


The MIT Tech Review points out that that this may have been the impetus behind GlaxoSmithKline’s announcement today that it will sell off its pipeline of gene therapies for rare disease to London gene-therapy upstart Orchard Therapeutics for a 20 percent stake in the company. One of those therapies is Strimvelis, a cure for a rare immune deficiency often called “bubble boy” disease. But even with the hefty price of $665,000, the small patient population means that the cure didn’t look like it would amount to much of a business.

It’s a callous calculation, and it shows that bringing to fruition the promises of the gene-editing revolution will take a lot more than mere scientific breakthroughs.