In the pharmaceutical space, there is clearly a lot of potential when it comes to genetics. In this Fool Live video clip, recorded on May 24, Fool.com healthcare and cannabis bureau chief Corinne Cardina, along with Fool.com contributor Brian Orelli, PhD, and chief growth officer Anand Chokkavelu, discuss why CRISPR Therapeutics (NASDAQ:CRSP) could be such an interesting opportunity for investors.
Anand Chokkavelu: Anyway, let's talk about these three. We've kind of talked about the space in general, but each one is targeting different applications or diseases. Which ones are each of these companies going after?
Corinne Cardina: I can start and then Brian can add on and give a more scientific answer. We'll keep going alphabetically. We'll start with CRISPR. I'm going to mostly focus on what these companies have in the clinic and just briefly mention anything a little bit further out in their pipeline that's either pre-clinical or in R&D still, but in the clinic, CRISPR Therapeutics is focused on genetic blood disorders, that is sickle cell disease as well as transfusion-dependent beta-thalassemia, and they also have several candidates in immuno-oncology. Further out, CRISPR is exploring type 1 diabetes, it is also exploring in vivo approaches for cystic fibrosis, Duchenne muscular dystrophy, and some other rare diseases. For the most part, CRISPR is not doing in vivo, they are doing ex vivo, where the patient stem cells are removed from their body, reengineered in the lab, and then reintroduced into the patient's body. Sickle cell disease is a large market in the U.S., 100,000 people in the U.S. are estimated to have it. Brian can tell us a little bit more about sickle cell disease, but beta-thalassemia is a slightly smaller market. If you zoom out and look worldwide, 300,000 cases of sickle cell disease and about 60,000 cases of beta-thalassemia. So I'll let Brian jump in on CRISPR here.
Brian Orelli: What's interesting here is that both of these diseases, sickle cell disease and beta-thalassemia are caused by the lack of hemoglobin. Instead of putting hemoglobin back into the cells, what they're actually doing is knocking out a gene that is responsible for turning off the expression of fetal hemoglobin. When we're born, we go from expressing fetal hemoglobin to expressing adult hemoglobin. What they're doing is they're knocking out the gene that does that control and by knocking it out now you get expression of the fetal hemoglobin and then that results in the cells being functional where they weren't before.
Cardina: Brian already touched on this, but sickle cell disease, it doesn't really have an approved treatment. Most of the medical care for patients who have sickle cell disease is focused on symptom management and those symptoms are really severe in terms of impacting quality of life. It can result in anemia, infection, pain, fatigue, it can stunt your development, it also can result in early death. A lot of these patients, they need frequent blood transfusions and they are also hospitalized frequently because of vaso-occlusive crisis, which I believe is like a blockage in the blood. Like Brian said, these companies are targeting where there's basically a vacuum, there's a really need for treatment and ideally a cure, which is what CRISPR is going after here.
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