There are some big opportunities in the gene editing space over the coming decades. In this Fool Live video clip, recorded on May 24, Fool.com Healthcare Bureau Chief Corrine Cardina, contributor Brian Orelli, PhD, and Chief Growth Officer Anand Chokkavelu discuss Editas Medicine (EDIT 3.76%) and why it could be a good stock to put on your radar.
Corrine Cardina: So, Editas. In the clinic, they are actually testing in in-vivo approach for, let's see if I can say this, Leber congenital amaurosis, or also known as LCA. This is a group of inherited retinal degenerative disorders and it's in the eyes. Brian will tell us more about why that's important for the in vivo approach. But they are also testing in the clinics in ex vivo sickle cell program, and they're actually using CRISPR-Cas12a, which is a little bit of a different technique than CRISPR-Cas9, which is what we've been mostly talking about. Editas has a strong pipeline further out. They are also testing ex vivo for beta-thalassemia. They have some other in vivo programs earlier on, including one for Usher Syndrome, which is a rare genetic disease that affects vision and hearing. They are really focused on ocular. They have another autosomal dominant retinitis pigmentosa four program and then they're also exploring oncology, and that is not yet in the clinic, but they have some partnerships with Bristol-Myers Squibb (BMY 1.49%), which is of course brings a lot of expertise in the oncology space, and BlueRock Therapeutics. We'll talk more about why these partnerships are important, but they are definitely exploring the oncology side as well, which so as CRISPR. Let's see if Brian wants to add anything and tell us a little bit more about why the eye is so interesting here.
Brian Orelli: I mean, I think they definitely made the right choice to go for the eye, and we've seen gene therapies work really well in the eye, and the main reason is it basically like a test tube. Your eye stock is all contained, and so you don't have to put that much drug into it to get a decent response, whereas you try to go through the liver or the kidney or something, got to inject it into the bloodstream and then now it's getting diluted out. I think that's really the reason why they went after the eye. First it's because it's a lot easier to treat the eye compared to other organs if you're going to do in vivo versus taking the cells out and manipulating them in the laboratory. Then on their sickle cell treatment that's edit, the real one, I think just because they are actually taking a different approach than CRISPR Therapeutics, they're editing the actual fetal hemoglobin gene. DNA strand before genes, there's something called the promoter, and other proteins bind to that part of the DNA, and that regulates the expression. They're making mutations in the promoter region of hemoglobin gene versus CRISPR Therapeutics, which is editing a completely different gene that regulates expression of fetal hemoglobin, but in both cases, they're trying to get expression of fetal hemoglobin to take care of the fact that the patients aren't making enough adult hemoglobin.
Anand Chokkavelu: Sorry. Regardless of what you-all say from now on, that visual of the eye as a test tube, I think is my takeaway from this whole thing, I love that. Never thought of it like that. I'm sorry, go ahead, Corinne.
Cardina: Yeah, I just wanted to add about the market for LCA, that in vivo approach that they're taking with that eye disease, it's actually the most common cause of inherited childhood blindness effecting three out of 100,000 children in the world. There are different kinds of this disease, and Editas is targeting the most common form, which is called LCA10, and that accounts for 20% to 30% of all LCA patients, and again, this is an indication for which there is no current treatment, follows along with they're really trying to cure something that there aren't a lot of options for patients out there. They are targeting quite a chunk of that audience.