There's no doubt that the genetics industry is full of investment opportunities. In this Fool Live video clip, recorded on May 21, Fool.com contributor Brian Orelli, PhD, and healthcare bureau chief Corinne Cardina discuss why Intellia Therapeutics (NTLA 0.62%) should be on investors' radar.
Corinne Cardina: All right, so Intellia, in the clinic, has an in vivo program. It is called, let's see if I can say this, transthyretin amyloidosis, it is shortened as ATTR. This is caused by the accumulation of misfolded protein, and that's what TTR is, that's the protein. The disease affects your nerves, your heart, your kidneys, and your eyes. There is a current treatment, but it requires chronic dosing. Of course, as with most of these in vivo programs, it has the potential to be a one-and-done dose, cure with one. Again, we're going to talk more about partnerships later on, but this particular program is in partnership with Regeneron (REGN 0.15%). They also are testing in the clinic in ex vivo program for sickle cell, and that is in partnership with Novartis (NVS 0.30%). Further out, Intellia is also testing an in vivo program for hereditary angioedema, which is the swelling. They're also testing hemophilia A and B in vivo. Then ex vivo, they are testing acute myeloid leukemia as well as solid tumors. They are also looking at immuno-oncology, although of course these are much earlier on than their in-the-clinic programs. The ATTR market for that main in vivo candidate. There are 50,000 patients who have the hATTR indication.
Brian Orelli: H is for hereditary.
Cardina: Thank you. What is wATTR, Brian?
Orelli: I'm not actually sure what wA is, but it happens spontaneously. Presumably, the other that's more common is that it happens spontaneously.
Cardina: Okay.
Orelli: Tends to be in older patients, but I don't actually know what wA is.
Cardina: I got this from the company site, 200,000 to 500,000, which tracks what they're saying much more common wATTR patients, and that is a worldwide market. It's very interesting, it's not a disease that I have really heard of before, but Brian, did you have anything to add on the indications Intellia's targeting?
Orelli: No. There are a couple of treatments for ATTR. Pfizer (PFE 0.63%) has one and then so does Alnylam (ALNY 1.53%) and Honest Pharmaceuticals all have treatments. Then it comes in two forms, so it comes in a narrow form, where the TTR protein tends to barge into the nerve cells and that causes those neurological side effects. Then it also comes in the heart form, where it lodges in the heart and then that causes problems with heart. Some people have both types of symptoms.
