What’s next for mRNA?
April 13, 2021
While regular people may not have been familiar with mRNA, the platform has been in development for decades. It may be having a spotlight moment due to its use in two of the three major COVID-19 vaccines, but that does not mean it’s new or that it’s unproven technology. Our newest lead advisor, Dana Abramovitz actually worked with mRNA while she was in college. She joins Dan Kline on 7investing Now to discuss where the technology might be used next.
A full transcript follows the video.
Dan Kline: And as is our tradition on Seven Investing Now we’re gonna throw data right into the deep end. It’s her first show, and we are going to lead off, she wants to talk about what’s next for mRNA. When it comes to drug development, I will point out that I learned on this show that it’s mRNA not Mr. Na. So this is a new tech for many of us who’ve I’ve taken the Pfizer vaccine. So I’ve had an mRNA based vaccine. And this is not a technology that the general public really knows about. But it could be a major platform going forward. Dana, what’s next for mRNA? technology?
Dana Abramovitz: Thanks, Dan. Yeah, no. So the M stands for message. So it’s messenger RNA. And that’s the cool thing, right, is that that message is translating what’s in our genes, to what our cells are going to turn into a protein. And so mRNA technology, and just the ability to, you know, inject or, you know, give a new cell that that new message for the cellular structure to create is just a new tool for scientists and drug developers. So it’s just, you know, just another new tool in their toolbox, which is great.
The thing, you know, the interesting thing, and I know, there are a lot of people that are, you know, talking about the vaccines and the worried about the vaccines, and that drives me crazy. It’s just, you know, like, this is not new, you know, messenger RNA, it’s just part of our, you know, the central dogma of biology and how information that’s stored in our genes in the form of DNA gets translated into protein, which is the cellular machinery. And it’s, you know, something that people have been studying for decades now.
And, you know, that’s how we were able to turn, you know, all of that knowledge, including knowledge that I contributed to, right. So no, I’m from the RNA field. You’re turning that into actionable tool, right? We were able to do it so quickly, because you know, people have been studying it for so long. So it’s, you know, it’s not new. It’s just that it’s now a new tool, because we’ve figured out how to deliver it to a cell. And so the cool thing is right, and so now You can use it to treat diseases of mis-splicing. So. The process of I know, I’m getting deep into the science, I
Dan Kline: No, no I’m just gonna say you’re gonna have to
Simon Erickson: I got my coffee, I’m ready Dana
Dan Kline: You’re gonna have to explain mis-splicing, that is not a term I’ve ever heard before.
Dana Abramovitz: Right? Okay, so So, um, biology so awesome, right? So you know, how can, our cells are really efficient, right? So we have in our DNA, the ability to have multiple types of information in one thing, right and one piece in one gene, right, but we can make it into different proteins based on different cells, different needs, by removing parts of it, and splicing it together. And that’s all done as the RNA, right, so the DNA gets transcribed, it becomes a pre messenger RNA. And then it’s splices and we remove bits. So introns, splice it all together. And then now you have the mature mRNA, that mature message that gets translated into protein. So this is basic biology.
Dan Kline: So from a practical point of view, from, from what I’ve read, the COVID vaccine was a relatively easy application of this, because of how it’s delivered. It’s not so simple to say that it’s the road to other uses of mRNA. It’s not necessarily going to go as quickly. And also, we’re not going to have half the medical companies in the world pursuing it with billions of dollars. But realistically, what do you think is next in the pipeline that we’ll be using mRNA for?
Dana Abramovitz: Right, so, um, so, you know, my guess so there are diseases where, you know, that maturation of the message fails. Right? So neurological diseases, like ALS, or Parkinson’s is one of those right? And then there are a lot of diseases where a protein drug or biologic is used, right? You know, so insulin, for example, you can, you know, like, you know, people with diabetes can use insulin, right. And so sometimes that protein can’t be made into a drug, right? Because making that protein in vitro, right. So outside of the cellular machinery doesn’t work, because the protein has to fold into a three dimensional shape in order for it to be active.
And if you do it outside of the cellular structure, it may not fold into the proper shape, or the delivery. So if you have to lyophilize it, and reconstitute. So dry it down, remake it with liquid to inject, it may lose its shape, right. But, um, with this mRNA technology, you can, you know, give it to a cell, and then the cell will make the proper correct protein. And it can it’ll do its own folding. Right. So, you know, that’s a possibility. That the caveat, so So again, it’s a tool, and I’m not saying that everybody’s going to be able to do it, right? These are possible uses for the technology, you know, there, there are caveats, right. So, you know, making sure that the mRNA gets to the right cell type that needs to produce that protein, right. So all cells go through that same, you know, process of maturing and making a protein, but not every cell needs to make that protein. Right.
So how do you deliver it to the right cell? So that’s going to be a delivery problem, we, you know, we see with, you know, cancer drugs and using antibodies to deliver. So, you know, there’s that possibility it hasn’t been tested, right. So, you know, we have that one thing, and then the next thing is, you know, we’re not actually fixing the gene. So that would be a gene editing tool, right. And so, you know, mRNA, degrades, you know, a patient who would be using this as a treatment would need to, you know, continually make it right.
And so, you know, like with the vaccine, you know, with the Pfizer’s in the journal, you get two doses, your cell makes the, the antibody or the antigen spike protein so that your system can make the, the antibodies for it. That’s a great technology, you know, using it for drug treatment, you know, we’re gonna have to figure that out and just see if it’s something that patients are really going to want to, to utilize. And then you know, the last one, you know, is just titrating and making sure the right amount of RNA is producing the right protein, the amount of protein for the event, the treatment.
So, you know, there’s there’s still a lot to be done. But the cool thing is, and you know, like I said, you know, people since, you know, Tom Tech won the Nobel Prize for, you know, looking at RNA splicing and seeing cell splicing in 1993, you know, people have been studying new RNA, like, what can we do with it? Right? So this is just, you know, it’s a tool that we can do that we can use. Um, you know, it’s really exciting. And to Yeah, let’s, let’s, let’s see, there’s, there’s lots of potential and possibility.
Dan Kline: Yet another year, I did not win the Nobel Prize. I didn’t do anything worthy of it. But I kind of feel like I should get one. Anyway. Simon Erickson, you wanted to weigh in here?
Simon Erickson: Well, Dan, first of all, I hope that you make your coffee extra strong, we start talking about messenger RNA with Dana on the show here, good luck keeping up for both of us, right.
Dan Kline: She actually sent in our notes that as a grad student, she made messenger RNA. And she said that to illustrate that this is relatively cheap and easy to do. And it’s, of course gotten more efficient. I’m not sure in college, I could make coffee. So this is a, this is an unbelievably it’s weird enough to say like, what, when I was in college coffee wasn’t as big a thing as it is now. Like, there wasn’t really Starbucks, so but I am not sure if you gave me a pot in the recipe and stuff and you’re making mRNA Maxx is making who knows what, you know, in his in his basement. So I feel a little bit left behind that said, I now make an excellent risotto. Simon, you want it to weigh in with some comments here.
Simon Erickson: But this is innovation. I mean, this is I think why I’m so excited to work with Dana and you know, and take notes every time Dana says these things because this is where the field is heading. This is where the science wants to go. Dana, one thing that I just wanted to add, that you mentioned was the delivery mechanism for software. So it should be just so important, right? We talk about kind of upstream versus downstream, right?
Do we want to edit the genome and change the genes themselves, or do we want to, you know, change how they get expressed, and change the RNA and you make sure that you know, the proteins that we do not want to be created or not being created, I really liked the approach of RNA in the in the fact that it’s kind of tissue and organ specific, right, you don’t have to have it go to your ears, and your feet and your toes and your liver all at the same time, you can actually pick where the problems are occurring. And I think that that might be one of the most promising areas of this field of research.
Dana Abramovitz: Yeah, and then and that’s going to be good delivery, and to finding that mechanism to target those individual cells. And, you know, like I mentioned, we are doing it with, you know, some cancer technologies and the antibodies, and just, you know, we know enough about different cell types, and you like, what types of receptors that has so that we can kind of, you know, target it. But you know, like, you know, I’m not saying that this is happening tomorrow, I’m just saying that this is now a new tool, and we can explore it. And it was just so exciting that we were able to, you know, find a use for this so quickly. You know, just because of the years, the decades long, basic research into this problem.
Dan Kline: And I’ll just jump in and say thank you science, like, as a lay person, it tends to feel like science is standing still, because there aren’t generally news articles, you know, that normal people are reading about, you know, advancements in mRNA. And it takes an awful lot behind the scenes that you don’t see before something happens. That’s why there’s some vaccines skepticism, because to the average person, they go, Wait a minute, did they just spin this vaccine up in six months, and here’s the reality, there’s 20 plus years of research going into this, and then a need came along. So it’s sort of like you know, if you’re really really good at, you know, starting a fire because you like to camp, and then all of a sudden you find yourself without power in the middle of nowhere. those skills come in, you practice them, you you learn them. That is what is happening here.