The healing power of gene therapy

One of the things that is tricky about treating these diseases is that they're so unique to each patient, and yet there are so many people affected by them.

400 million people worldwide, half of whom are children, have one of these rare diseases.

How close are we to being able to help patients like that on an individual basis?

Yeah, that's really a major hurdle and I would argue a major responsibility that societies have.

Now that we have technologies like the ones we just summarized, we should figure out how to maximize the ability of the science to reach as many patients as possible.

And in some cases, there are still scientific limitations.

But in many cases, and increasingly, the barriers are less scientific and more economic or regulatory.

Or is there a reimbursement mechanism that society has established that can make sustainable efforts to treat patients like baby KJ over and over again? So your question, I think, is really important because there are many patients like baby KJ who are facing life-threatening diseases right now, for which we probably know how to correct the mutation that causes their disease and even how to deliver the gene editing agent to the right cells and how to manufacture it in a way that has a good risk-benefit ratio.

But that doesn't necessarily mean, as tragic as this sounds,

that there's a path to connect all of that science and all of that technology with the patients.

Because it still takes a lot of resources to produce and test and characterize to the extent that FDA requires or other regulatory agencies in other countries require.

And for gene editing agents, that can still cost millions of dollars or even tens of millions of dollars.

So the conundrum is, if you have a disease as rare or a mutation that's as rare as baby KJs, it's not practical to spend millions or even tens of millions of dollars to develop that treatment if it can only treat one or five or 10 or 20 patients.

It's not sustainable.

So all of this is really what motivated us to found the Center for Therapeutic Genetics, CTG.

The first decision we made was that this would be a nonprofit effort.

So it's a collaboration between three nonprofit entities, the Broad Institute, Boston Children's Hospital, and Jackson Labs.

And the concept is that for the rarest of genetic diseases where industry isn't really able to develop treatments simply for this economic reason among others, it makes more sense to try to take on those diseases and help those patients when the science is already well-established in a non-profit setting.

That's the kind of change that I think ultimately will allow this newfound ability to take some control of our genetic futures to be much more widely applied than it currently is.