Healthcare companies are at work on potentially groundbreaking therapies in trending areas that are being overlooked by many investors. In this segment from "This Week in Healthcare," recorded on Jan. 31, Motley Fool contributors Keith Speights and Brian Orelli explain some of the science behind this work, and name the companies that are at the forefront.
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Keith Speights: A trend that's overlooked or underappreciated.
Brian Orelli: I have one if you want to take a second to think.
Keith Speights: One that comes to the top of my mind is, I'm not sure exactly how underappreciated it is, but I do think that the next generation of CRISPR gene editing is a trend that certainly could really blossom. Obviously, we've got CRISPR Therapeutics, we mentioned earlier in the program that CRISPR therapeutics and Vertex Pharmaceuticals hope to file for approval of their gene editing therapy and treating sickle cell disease and Beta-thalassemia later this year. But that's a first-generation type CRISPR therapy, but some other CRISPR therapies for example, from base editing approach from Beam Therapeutics, I think that might come to the forefront over the next few years, so that's one trend that I think I would especially watch.
Brian Orelli: Yeah, and there's a private company right there too. Is it Mammoth?
Keith Speights: Yeah.
Brian Orelli: I'm looking forward to that company coming public because I think that'll be an interesting story too. The one I would go with is protein degraders. I've been looking into them recently. The cells have a natural way of degrading proteins that are misfolded, and so these drugs are taking advantage of that and the advantage here is that you don't necessarily have to inactivate the proteins, so there's a lot proteins where they call them undruggable because it's really hard to get a molecule into the active site that's very specific to that protein that you're trying to inactivate.
But these protein degraders can bind anywhere on the protein, so all you have to find as a specific area somewhere on the protein that you can bind and then you trigger the activation of the ubiquity and ligase system in the cells to degrade the protein and by degrading the protein, you're essentially doing the same thing as inactivating it, or the same thing that RNAi drugs or antisense drugs do by inactivating the mRNA, which then therefore results in no protein or antibodies do by binding up the proteins.
But generally speaking, antibody drugs only work for proteins that are on the outside of the cell and these protein degraders can work on for proteins on the inside of the cell. Arvinas is one that I've been looking at. Kymera which I bought at some very small stake in when I started researching that one, that seems like a good option. Then C4 Therapeutics is a third one that I have been looking at. All three of those are something to look forward to, especially when we move to our hour format I'll probably do deep dive on at least one of them.
