Gene therapy and rare diseases: ‘Is curing patients a sustainable business model?’

Child with ADA-SCID, or "bubble boy" disease.

An analyst at Goldman Sachs asked a troubling question…about gene therapy.

“Is curing patients a sustainable business model?”

In social media, reactions were quick and sharp. “Cold and immoral.” “Capitalism at its finest.”

But the Goldman analyst has a point. It’s tricky to make a sustained profit from one-shot cures. For at least one big drug maker, the answer to Goldman’s question is no.


We just saw GlaxoSmithKline sell off its pipeline of gene therapies for rare disease to a London startup called Orchard Therapeutics for a 20 percent stake in the young company. The treatments Glaxo didn’t want were bona fide miracles: one-and-done cures.

The economic problem is that companies can run out of patients as they’re cured. Or they may not have enough of them in the first place, if the gene therapies treat exceedingly rare diseases.

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For instance, only a couple of dozen kids each year in the US and Europe are diagnosed with the type of “bubble boy” disease that Strimvelis treats, called ADA-SCID. So even at a price of $665,000, Glaxo didn’t see that the drug was going to be much of a business.


“I think society has to find a balance between the extraordinary value we are bringing and a sustainable business model,” [Orchard CEO Mark Rothera] said. “There is a balancing act.”

Read full, original post: When curing a disease with gene therapy is bad business

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