Wither Gene Editing Blockbusters To Come?

SometimesASequenceIsJustASequence

WhoseCorrectionMayBeTooSmallToProfitFromVille

On Monday we talked a little bit about a new gene editing technology that has the potential to efficiently treat a devastating inherited immunodeficiency disorder.

The work was funded by a biotech spin-off company called Prime Medicine that has already signaled that it will have a hard time funding the further development of the technology as a viable therapeutic. This was reported by Hedi Ledford writing in Nature back in May when the initial results of the work were first announced (sorry 'bout the paywall):

"...Despite these early signs of success, Prime Medicine also announced that it will not develop the therapy, called PM359, any further on its own. “Prime Medicine is exploring options for the continued clinical development of PM359 external to the company,” it said in a statement.

That decision reflects the harsh realities of developing gene-editing therapies for very rare diseases, says David Liu, a chemical biologist at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts, and a co-founder of Prime Medicine. “The science has moved far enough that many patients would benefit from these gene-editing treatments,” he says. “But it boils down to an issue not just of science and technology, but of economics.”...

Of course, some of this is little more than biotech posturing and, to be clear, the company has raised significant amounts of money despite the fact that its stock price has fallen significantly since its IPO three years ago.

The thing is, the disease concerned is very rare which, as an editorial in the Guardian noted recently, makes developing the technology any further really difficult given the costs involved and the commercial incentives that drive our current system of drug development:

"...Novel gene-editing breakthroughs are making headlines. But therapies are expensive and complex to develop. The cost of bringing any new drug to patients is now around $2bn, in part because, as Brian David Smith notes in New Drugs, Fair Prices, the “success rate, from discovery to market, is tiny” and there are approved treatments for “less than 10% of the 8,000 diseases that affect humans”. Commercial incentives, he argues, skew innovation towards lucrative cancer drugs and long-term treatments for large populations. Complex gene therapies for very rare conditions are seen as too costly to develop and too small to profit from..."

In other words, life science geeks who are trying to develop new therapeutics are constantly pushed to work on indications that exclusively serve large numbers of patients because they are told that they will never be able to garner funding to move their discoveries from the lab bench to anywhere near the bedside if they don't.

So.

Are there other models out there that might give us a better chance of moving all this exciting universe-denting research forward towards the clinic?

Stay tuned...

______

Image at the top of the post is from a prime editing explainer from the good folks at MIT.



.