You know how I keep saying it’s time to learn the basics of gene therapy, because it’s coming at us fast? Well, I was wrong. It’s not coming; it’s here.
Not for XLH yet, but I’ve gotten the impression recently that a lot of people don’t know that gene therapy is indeed happening now for other genetic conditions. And if it can be done for other conditions, it can be done for XLH (with a lot of money and hard work). So I thought I’d share a little bit about the current state of gene therapy.
The FDA now has a whole page at its website dedicated to already-approved “Cellular and Gene Therapy.” I was expecting to see maybe three or four entries, but there are actually thirty-two! Most of them are cell therapy, not gene therapy, but still, it’s an impressive number.
As I understand it, cell therapy involves introducing already-functioning cells into the patient, while gene therapy involves introducing DNA that then is absorbed into a patient’s existing cells to cause them to function in ways the DNA tells them to do. You can read more about it from real experts at the website of the American Society of Gene and Cell Therapy. Both types of therapy are way more advanced than my understanding of science, but I believe gene therapy is somewhat more tricky than cell therapy, so it’s taking a bit longer to get approvals.
You can tell which products are gene therapy by looking at their generic name. Just like the generic name burosumab ends in “mab” to show it’s a monoclonal antibody, gene therapies signal what they are by including “gene” at the end of the generic name.
I counted ten approved gene therapies on the FDA list. A little over half appear to be for treatment of specific cancers, while the others are for a variety of extremely disabling rare disorders. And there are many, many more in the pipeline — tiny pharma company Ultragenyx has six gene therapies in its pipeline! Sickle cell anemia has long been considered a good target for gene therapy, and I believe there are multiple studies going on, some fairly far along in the process. (For example, check out this site, sponsored by a pharmaceutical company, not just for the sickle cell information, but for some really good explanations of gene therapy in general).
Gene therapy is also already here for the pharmaceutical industry, not in terms of what they’re selling right now, but in terms of the research they’re focused on for selling in five to ten years. You can see it in the pipeline of one pharma company we’re all familiar with: Ultragenyx. When Crysvita was approved in 2018, it was a cutting-edge treatment (antibody rather than a chemical compound), and Ultragenyx was working on a few other monoclonal antibodies. Now, their pipeline, beyond the ones left over from 2018, consists almost entirely of gene therapies, and they just built a new gene therapy manufacturing facility not far from me in Massachusetts!
I know gene therapy still feels like science fiction, and it is admittedly still early days, but we’re on the very cusp of a very different health care future already and need to be prepared for it!
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Please note that the author is a well-read patient, not a doctor, and is not offering medical or legal advice.
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