Shekerah Primus 0:04
What can you do with your love of science we'll tell you?
Hello, hello everyone. Welcome back to your favorite podcast. We love science. We are your hosts, I'm Shekerah,
Fatu Badiane-Markey 0:43
and I'm Fatu and today we're back on the CRISPR train
Shekerah Primus 0:49
Yes we are, the CRISPR train, the CRISPR train
Fatu Badiane-Markey 0:52
come and ride the train choo choo. Do you remember that song?
Shekerah Primus 1:01
Okay, so today's episode is called changing lives by the million a bold claim. Can we do it?
Fatu Badiane-Markey 1:10
It is a bold claim but I think we can do it. And on top of that, I think we're already doing it. There have been so many I feel like advances in sciences, you know that like you and I, and I'm sure even other students, you're just hearing about them now. But in the next 10 years, they're going to be reality and it's so amazing, you know?
Shekerah Primus 1:28
Yeah. Yeah, we're gonna do it. I agree. So we started with the sickle cell disease CRISPR treatment that made history as the very first CRISPR based gene therapy treatment to be approved for use. It's approved in the US it's approved in the UK, in the EU, and it's under review or planned for submission in other countries, including Canada and Saudi Arabia. So we did a full episode focusing only on sickle cell disease last time. So what causes it? What are the symptoms? The number of infected individuals around the world and how the CRISPR gene therapy works? So we'll put a link in the show notes so that you can find that episode. But in today's episode, we're talking about some of the other diseases that are currently in clinical trials for a CRISPR based treatment.
Fatu Badiane-Markey 2:23
So what you're saying is that we're already on the way to changing millions of lives Shekerah, and I was super excited to learn all about this. So how many clinical trials are we going into today?
Shekerah Primus 2:37
Yeah, yeah so I was hoping that we could do a whole lot cause there are many CRISPR clinical trials ongoing. They make up about eight different major disease categories, like cancers, for example, another category is blood disorders and sickle cell disease, fits into that category. And we discussed that last time as I said, But you know, we won't be able to talk about all of them unfortunately, today, you know, just because of timefor this episode. I did try as you know, as much as, as best we could to get as much in there as possible, but I wanted to be clear about the ones that we did decide to talk about. Yeah, so today, we'll touch on a few of them. And I made sure to select at least one from each like clinical trial phase.
Fatu Badiane-Markey 3:34
Okay, no, that sounds doable. And honestly, I know that you especially I've been putting so much passion and soul into this series. So you know, I feel like we need a whole separate like mini podcast to go into all of that. So time around less is more and yeah, and we'll get to it. So before we jump in, though, can you explain clinical trials a bit more? Even myself? I don't know very much about them. I've never participated really? So what are you know, like the different phases? What exactly does that mean? You know, how do they work?
Shekerah Primus 4:08
Yeah, yeah, yeah. No, you know what I realized? That, you know, going through grad school and doing research, you're in the lab, as a scientist, there's a lot of these things that we don't learn about, that they do in biotechs and pharmaceutical companies, where they are actually turning the basic research that we do in the lab into therapies for people to use and so yeah, a lot of this a lot of scientists don't really know about. So yeah, before a new drug or medical treatment is approved or medical device is approved, to be released to the general public. It's tested on volunteers In clinical trials to determine the safety and effectiveness of the treatment. So in the US, the regulatory body that evaluates clinical trial results, and either approves or denies new drug applications is the FDA the Food and Drug Administration. And there are similar regulatory bodies in the UK and EU and other parts of the world, right. So clinical trials are very highly regulated. So, clinical trials typically advance in a standard series of phases from one to three usually and sometimes there's also phase four as well, but we won't talk about that so much today. Each trial phase has different goals that need to be met, and questions that they're trying to answer to determine whether or not to proceed to the next phase. So the goal of the earlier part of the clinical trial so that's phase one, is to determine the safety of the new treatment. That is the main goal of the phase one, is this safe to put into people's bodies right is this treatment safe. So safety and that includes any side effects from the treatment right to to log any side effects that the trial participants are exhibiting. And usually they start with a small number of people, again, just to make sure that you're not giving this drug to hundreds of people and they're going to be really detrimental effects. So they start with a small number of people before expanding it into more people. So you'll sort of hear that theme as we go through the rest today. So then the later phases phases two and three, are used to determine how effective the new treatments as well as how it compares to the treatments for that particular disease that are already available on the market. Right. So for example, if your new treatment works better than what's already on the market, then there'll be a lot of interest in getting that drug to market right especially if it has side effects are very minimal. But if your drug provides a similar therapeutic effect, to what's already on the market, then it becomes a bit more of a tricky business decision. And it's more of looking at things from a manufacturing cost versus expected profit margin based on market share that type of like thing right like businessy things right. So that situation tends to be more nuanced. And lastly, it results for your drug or not. Even as good as what's currently on the market. Well, that's not ideal, right that's the first response It's not ideal. However, this can also be kind of a nuanced situation, because I'll tell you why. The company first of all, might not want to go into the expense of even trying to get the drug approved. However, it may still be beneficial to get the drug approved, especially if there are no side effects or minimal side effects. For example, if a disease has a mild form and a severe form, like many diseases do, right, we've talked about sickle cell disease last time there is a mild form and a severe form. Right, then it might be better for patients suffering from the mild form of the disease to take a weaker drug that has fewer side effects, compared to a stronger drug that has a long list of more severe side effects. I mean, we've heard some of the side effects from drug commercials on on TV, right? I mean, they go on and on. Sometimes they are long sometimes. So it may be more beneficial for certain types of patients to take a weaker drug if it has fewer side effects. So in that type of situation, then they might decide to go forward with it. So like I said it's a bit more nuanced. But yeah, for clinical trials, you've got phase one, used to evaluate safety and side effects. And then phases two and three are more about effective dosage and how well is this treatment working? And sometimes the phases will get combined for different reasons. So you'll hear things like combined phase one-two clinical trials. And of course, there are a lot of rules and regulations, as we said that they have to follow that we won't get into too much today. But yeah, that's kind of in a nutshell how clinical trials work.
Fatu Badiane-Markey 9:13
Okay, that actually makes a lot of sense. And I like how there's room for nuance also in that, you know, I wouldn't have thought of that. I would have thought it would have been very black and white. You know, like, yeah, so but I think I think that's good, because, you know, biology is complicated. So when we're trying to make treatments that makes a lot of sense to me. All right. So today we're going to touch on clinical trials in all three phases, right? So phase one, two, and three. So let's move into how CRISPR is being used in disease treatments that are currently in trials. So which one is first Shekerah? I'm all strapped in and ready to go.
Shekerah Primus 9:50
Ready to go, alright Take off. So now we know all about clinical trials. Let's start at the beginning. And first talk about a couple of phase one or combined phase one phase two clinical trials. And then we'll end with a trial that has already advanced to phase three Okay, so we know that CRISPR-cas9 gene therapy is primarily used to treat genetic diseases, because it cuts DNA. And if you missed our episode, where we discussed how CRISPR works as a gene therapy, go back and listen to our secrets about CRISPR episode and we'll put a link to that episode in the show notes so that you can find it. But yeah, CRISPR is typically used to treat genetic diseases. However, the first clinical trial that we'll talk about is not a genetic disease, surprise. It's an infectious disease. Yeah, specifically HIV/AIDS. And as we know, HIV/AIDS is caused by a virus, specifically it's a type of retrovirus. And a special characteristic of retroviruses is that when they infect a host, they can add their viral DNA into the genome of that person they infected. So, in effect, the DNA of the virus becomes a part of that person's genome. Right. And that is one of the main reasons why it's been so difficult to cure a person who has an HIV infection. Even though at this point, we have really effective drugs now for HIV, right. HIV infection is treated with antiretroviral therapy, which is usually a combination of three different drugs that can clear the viral load in the body down to undetectable levels. And so while that situation is enough to cure many, many infectious diseases, it's not enough to cure HIV. Because even when there's no active virus detected in the person's body, that person is not cured. Because all the while the viral DNA is hiding in that person's genome. And at any time the viral DNA can reactivate and start producing more virus. Yeah, So in order to completely cure HIV, you have to also get rid of the viral DNA that's hiding in the patient's genome. And that's exactly what this CRISPR treatment is designed to do, to find and cut out the viral DNA that's hiding in the patient's genome.
Fatu Badiane-Markey 12:36
What? You know, like, in my mind, like, my logic was like going there, but hearing you say it, I'm just like, wow, oh, my gosh, that's incredible. I'm really excited to hear more about that. I mean, what you're, what you're saying is totally right. HIV has definitely been a really tricky one to cure. You know, yes. Patients have been living longer Fuller, I would say even just, you know, normal lives these days, with a lot of the treatments that are available, but ultimately, yeah, you want to cure it because unfortunately, they do have to take those retroviral therapies for their entire lives. Yeah, wow. And, you know, another I think, really big caveat also to think about is if a patient doesn't stick to their treatment regimen, right? So taking these drugs every day for the rest of their lives. Anytime, you know, there is an opportunity for the virus to sort of come back that also is an opportunity for resistance to treatment, and then that again, leads into more complications. So that would again be so life-changing for so many, so many millions, millions of individuals. So yeah, all right. So tell us more about this clinical trial. Now. Shekerah I am, I'm now committed.
Shekerah Primus 13:57
We're all Committed girl. Okay, so this clinical trial is being run by excision Biotherapeutics. I really love that name I feel like that's the perfect name for this type of company. Excision, We're going to take that DNA out that bad DNA excision Biotherapeutics the trial started in 2022. And last year, 2023, The therapy was granted Fast Track status by the FDA. For obvious reasons, right? HIV infects millions of people worldwide as you said, and even though it can be controlled by these antiretrovirals these drugs are expensive, They have serious side effects, and for HIV positive individuals, they have to take these drugs for the rest of their lives, right to make sure that they keep the virus in check. So, yea, it's been granted Fast Track status. And so if all goes well with the clinical trials, we may have an actual HIV cure on the market within the next three to five years. Right, which is amazing to think about right now, I know some of you may be saying wow, it's on the fast track and it could still take five years. That sounds more like the slow track. I get that really just shows you how long clinical trials take normally, right. So, but yeah, it's currently in combined phase one, phase two of clinical trials. They started dosing volunteer patients in July of 2022. And I believe they've only dosed three people at this point, which is a pretty small number right in the grand scheme of clinical trials. But as we just discussed, the purpose of phase one in a clinical trial is to determine safety of the treatment, and CRISPR is still a new technology. This is a brand new type of treatment that they're, you know that they're undergoing here to cut out a piece of DNA from the person's genome within the body of the person, and so they want to go carefully and operate with an abundance of caution. Right so the data that has been reported is that there have been no serious side effects or toxicities reported in any of those trial participants. So that's really good news to hear. So now that they've established minimum safety criteria, that there have been no serious adverse reactions in the treated patients. They're more confident now to expand the trial. Right, so they're going to expand the trial to include more patients, they'll continue tracking side effects of course throughout the entire life of the trial. This is normal. But now they'll also be trying to establish dosage as well as efficacy of this treatment, is it actually going to be able to cure HIV?
So that's why it's called a combined phase one phase two trial because they've got both phases going at the same time. So they're looking at safety and efficacy at the same time.
Fatu Badiane-Markey 17:08
That is really, really interesting. Yeah, that makes sense. That's so cool. Oh my gosh, yeah. That's so cool. And you know, I also really appreciate how structured and methodical these clinical trials are. You know, this abundance of caution is just also so greatly appreciated, you know, it's like they actually they really care, you know, if there are adverse events, you know, they really care. Yeah. So what else should we know about this trial? And what are people watching for specifically with this one?
Shekerah Primus 17:38
Yeah, yeah excellent question. So they've got a lot of eyes on them right for sure. And you know, more patients, any scientist will tell you, more people mean more diversity. bigger sample size, means more fluctuations in your data right more variety in your data. And more variation in your data rather. So with a larger sample size, so everyone's watching and hoping that the data from the new patients that they're enrolling in the trial is going to echo the data from these initial phase one patients. And that is going to be you know, no adverse events, that type of thing, especially since this is sort of a new thing that they're trying right. Now, there are, of course, also caveats and challenges with this treatment, which we won't get into too deeply here just because of time, but one big challenge with this type of treatment is that for this to truly be curative, the viral DNA needs to be removed from everywhere where it is in the genome right, removed from all the cells that it's hiding in immune cells, right. So HIV infects T cells, right? Yeah. From every spot in the genome where it secreted away is a little reservoir to reactivate. Right. So it needs to be removed. And so you know, in order to say that, you know, the patient is cured. So the bar for cure is super, super steep way steeper than what was needed for sickle cell disease. We talked about sickle cell disease. The last time the bar was not as steep. Right. So definitely, there's some skepticism about how successful this therapy is going to be just because of what is needed for it to be successful in eliminating all the viral DNA that's hiding in the patient's body. But there's also a lot of hope, you know, there's also obviously a lot of hope for this treatment. And so yeah, needless to say, This trial is being closely watched by everyone.
Fatu Badiane-Markey 19:41
Yeah no, and I definitely understand the skepticism because it's literally like if one little viral genome is left, right, then that means you're you can end up with reactivated virus and then the person will get sick again.
Shekerah Primus 19:56
Yeah, maybe you know, we're not we're not sure exactly how much we will have to get rid of, but we're assuming that we got to get rid of it. All right. because Yeah. That that one might be the one that's like, Oh, I'm super active. I'm going to reactivate right now You know you never know
Fatu Badiane-Markey 20:11
You Never know.
Shekerah Primus 20:13
Yeah, so this one's tricky, for sure.
Fatu Badiane-Markey 20:17
Definitely, definitely. I can definitely understand why there's so many eyes on this. So what other clinical trials have you looked at Shekerah?
Shekerah Primus 20:25
Yeah, so a couple of other interesting phase one trials that I'll just quickly mention. The first one is lupus, which is a chronic autoimmune disease, and so on autoimmune disease, that's when the body's own immune system is attacking itself. And that can cause damage, right, almost, almost anywhere in the body. Right. So you see damage to the heart, the kidneys, the joints, and it often shows up on the skin, of the of the patient. In severe cases, it can result in heart failure, and kidney failure. So it is a life threatening condition as well. And the last one I'll mention that's also in phase one is cardiovascular disease, which is caused basically by high levels of bad cholesterol, right. And that results in a buildup of fatty deposits called plaque on the walls of the blood vessels. And so these plaque deposits, they partially block the blood vessels, leading to heart attack strokes and premature death. And there are genetic mutations. That cause high cholesterol. So you might think that, oh cholesterol that's basically caused by your diet and whether or not you're exercising right and while that's true. There are also genetic mutations that can lead to high cholesterol just like there are genetic mutations that predispose you to like breast cancer, right? The BRCA mutations, so they're, they're targeting this genetic mutation that causes high cholesterol. And so yeah, cardiovascular disease is one of the leading causes of death world wide. So this is another big one to watch out for.
Fatu Badiane-Markey 22:12
Yeah. Wow. That's so amazing. And I think also just looking at kind of like the diversity of different diseases, right, that can really be impacted by this, I think is also just really amazing. Because it gives you an understanding of sort of like what is the promise of this technology, right? It's like, I almost feel like we can use it to treat anything under the sun. We hope Yeah. I mean, in a way, it's exciting, but I think in a way it also makes me a little cautious to be like, are we putting all our eggs in one basket? Or is this just the it biotechnology right now but five years down the line, we'll have something even better, right? That could also be the so I just really think this is like the cornerstone of just like an amazing new era in biotechnology, in medicine, in understanding diseases and how to treat them. So yeah.
Shekerah Primus 23:11
I agree with that. You know, but I don't think we're putting all our eggs in one basket. You know, I think that this is a newest basket right. This is this is the shiny new basket, that we're trying to figure out how to get it to incorporate into all the systems we already have going. And we do see that happening. You know, because you have researchers combining CAR T cells for a while with CRISPR technology and getting CAR T to be able to attack cancer even more effectively. And so, there are of course some caveats and problems that're having with that and we're not, we're not going to get into cancer this episode because it's way too much but just to like point out you know, we're definitely combining this new technology with our existing technology, and just just upping the ante and in our fight against all these diseases that are trying to take us out.
Fatu Badiane-Markey 24:12
Yeah, that's a good point. That's a really good point. All right. So what's next
Shekerah Primus 24:18
so we've touched on a couple of phase one and a combined phase one phase two trial. Now, let's talk about a trial that has already advanced to phase three. And this one is interesting, because it's a disease that I really didn't know much about before I started doing this research, and you kind of just mentioned how, you know, there's so much variety in what people are targeting, and it's so true. We're doing things that are very prevalent in the population causing a lot of strife and we're also doing things that are more rare diseases and it's also causing it's also causing hardship to people. It's just not as prevalent in population so there's definitely a large variety of things that are in clinical trials. And so this one is a protein folding disease. It's called hereditary trans. I don't know if I could say this. Sorry
Fatu Badiane-Markey 25:13
Sound it out, sound it out you got it.
Shekerah Primus 25:15
It's called Hereditary Transthyretin. amyloidosis. whoo Or hereditary ATTR. That's what I'm gonna be saying from now on. It's caused by a mutation in a gene called TTR. And similar to what happens with like sickle cell disease, this mutation results in the production of a faulty protein. So this mutant protein then accumulates in tissues and organs, including the heart and the nervous system causing a whole lot of problems. And Also, similar to sickle cell disease, the symptoms can vary, but the most severe lead to heart failure and affect the nervous system so patients can experience nerve pain, loss of control of their bodily movements. That's so scary, right? vision loss and dementia, right? Also scary. And so because these mutant misfolded proteins tend to accumulate in the nervous system as well. The neurological symptoms are similar to what you'd see with Alzheimer's and Parkinson's disease right that also have these accumulations of plaque bodies in the nervous system. And this is one of those rare genetic diseases, only affecting about 50,000 people worldwide.
Fatu Badiane-Markey 26:40
Okay, wow. I hadn't actually heard about hereditary ATTR before. Yeah, I you know, but yeah, that's just another I think, really good target because it is really impacting people and their lives. And if it's hereditary, like the name suggests that that also means families, right? Yeah. Wow. Goodness. So can you tell us more about the clinical trial?
Shekerah Primus 27:05
Yeah. So this clinical trial is being done by Intellia Therapeutics or being sponsored by Intellia Therapeutics is the right terminology here. And using patient volunteers with either nervous systems symptoms from this disease or with heart issues caused by the buildup of that mutant protein and those organs, so kind of like two cohorts of patients. It started in 2020. So the first patient was dosed in November of 2020 in the UK. And this is another study where they use a pretty clever strategy, I think. So instead of fixing the mutant gene that is responsible for creating this bad protein that's causing all these problems, they decided to break the gene even more so that it can't produce any protein at all. Right. Because if the buildup of this bad protein is what's causing problems in your organs. You want to make sure that that protein can't build up right so you just completely turn off production of that protein and so that's what they're doing with this study. So the goal is to prevent the production of the bad, mutant protein. It's not a really good terminology prevent production of the mutant protein that's accumulating in the body and causing all these problems. Another thing they did that was pretty clever was the way that they delivered the gene editing components. So one thing to note is that this is the first clinical trial to deliver genome editing components so that's the CRISPR-Cas9 components systemically to the entire body just like through an IV, right, so compare this to say the sickle cell disease trial that has already been approved. They took the cells out of the patient's body, edited them in the lab, right, did some quality control to those cells in the lab, and then put those edited cells back into the patient's body. In this trial, the genome editing happens live, so to speak, right inside the patient's body. So this is a more risky approach. Because all those CRISPR components that cuts up DNA, it's floating around in the patient's body, right. So this is a more risky approach than what they did. For the sickle cell disease trial. But what they did though, was they put the CRISPR components into lipid nanoparticles. And you might have heard this term you know, when people were talking about the COVID vaccine and all that, right, so lipid nanoparticles and they injected them into the body so just through an IV.
Shekerah Primus 29:08
So the lipid nanoparticles are basically the packaging system that carry the CRISPR components through the body. And the thing that makes us so clever is that lipid nanoparticles have a tendency to accumulate in the liver. And this TTR protein that is causing all these problems with this disease is also made in the liver. So instead of having the CRISPR treatment just go all throughout the body willy nilly wherever it wants. they packaged it into a system that will take it straight to the organ where it needed to go to the liver. So that was a really clever strategy, right?
Fatu Badiane-Markey 29:46
So clever. So clever, oh my gosh, I love it. That is that's just like putting together like so many different pieces. I love it.
Shekerah Primus 29:57
Yeah.
Fatu Badiane-Markey 29:58
So what was the data? What have they reported from this clinical trial?
Shekerah Primus 30:03
Yeah, so as I said, they're already in phase three. And this is the first CRISPR clinical trial to deliver components systemically to the body, and also the first to use lipid nanoparticles as a delivery system. So they they were a lot of firsts as well. So you know, everyone was watching, everyone was watching with bated breath just like with sickle cell disease, right? Everyone was watching with bated breath to see what would happen. And I'm happy to say that they got great results right? So they wouldn't they wouldn't be in phase three if they weren't getting good results. Right. After phase one of the trial, they had 27 participants, and those people only showed mild side effects from the treatment right? That were reported from phase one of the trial. Now as you just said, once you increase your number of participants, you increase your sample size, your data starts to fluctuate. So after phase two of the clinical trial, still most side effects are mild. However, seven of the now 65 patients experienced some serious side effects. There were no deaths, so all the participants were still able to move on to phase three of the clinical trial. And so what they're seeing is that even at the lowest treatment dose that they tested, there is a very large more than 85% reduction in the amount of that mutant protein that is in the patient's bloodstream. Right? And there is a greater than 90% reduction of that protein with patients receiving the highest dose of treatment. So imagine that even the lowest dose that they tried 85% of that protein that's causing all that problems. It's, it's gone. So that's a really amazing result. They also saw that all the patients, regardless of whatever dose they received, are showing sustained reduction of that mutant protein over time. So they have followed patients up to two years and released data to here's post treatment, right? And released that data and they're still showing reduction in that in that the amount of that protein that's in their bloodstream. So this is what they want to see in clinical trials. Right. This is a kind of thing that just makes you just let go yay yay yay yay. So phase two has completed and they received approval from the FDA to move on to phase three. And so based on their phase two results, they selected a treatment dose in the middle of the range of all the different doses that they tried. So as I said, even the lowest dose they saw more than 85% reduction, so they selected a dose in the middle. And so that's what they're moving on to phase three of the trial. So yeah, this is a pretty exciting one as well to keep an eye on and you never know we might have another approved CRISPR treatment very shortly.
Fatu Badiane-Markey 33:05
Yeah, you'll have to keep us posted Shekerah, since you're like the CRISPR Queen officially.This is just so phenomenal. It's like you know, so many different types of diseases. And when you think about the impacts, you know, it's like yes, okay, you know, maybe for this, the hATTR you know, that only affects about like 50,000 people worldwide. But when you now take like all these diseases together and think about treatments for all of them cures even right. I feel like that just compounds, right? The number of people who are going to be impacted because now it's like 50,000 For this one, plus, however many 1000s Because other one plus how many millions, right? Like HIV/AIDS. Yeah, that's just the scale of that impact is just I don't even think we can imagine what that is going to be like you know, and what that's going to mean for the future. So this is just
Shekerah Primus 34:00
Changing live by the millions.
Fatu Badiane-Markey 34:01
Yeah literally, literally. Now I see it now I see it. So it's like sickle cell disease, started it all off. And now here we are. Yeah, it's just, it's incredible. So last time, we talked about sickle cell disease that started it all off. So what's the update now for that? Where are we with that treatment?
Shekerah Primus 34:25
Yeah, so the sickle cell disease treatment is approved, as I said in several places and still undergoing approval and other countries. The official name is Casgevy. But yeah, we got some feedback after that last episode and a few questions. One from Jesse from North Carolina, Jesse, thank you for listening. And for reaching out but Jessie commented that it doesn't seem that the treatment will be affordable. And that's definitely a valid point. So yeah, currently the sickle cell disease treatment is priced at about 2 million per patient. So yeah, I mean, that's that's pretty pricey, right? And there, there are, you know, other challenges with administering this treatment as well as we discussed in the last episode. Part of the treatment is that the patient has to go through a chemotherapy procedure to kill patients blood stem cells. But you know, we do expect that the price will go down with time and I think we talked about this a little bit. I believe it was the episode with Irina. New medicines and technologies usually start out with this steep price tag. Right but it becomes more affordable over time. And part of what drives the price reduction is yes, improvement in technology and resources but also competition. Right? And don't worry, Casgevy is gonna get competition right. So currently, it is only CRISPR sickle cell disease treatment that has been approved, but there are several others that are still in clinical trials and doing well. So beam therapeutics and Editas both have sickle cell disease clinical trials in phase one, phase two. And the molecular techniques that they're using is a little bit different. So So yeah, they're both CRISPR. They're both CRISPR clinical trials, but the molecular techniques they're using is a bit different than what CRISPR therapeutics did, with this Casgevy treatment, but the ultimate goal is the same it's to increase fetal hemoglobin. But the clinical trial for sickle cell disease that is most interesting that I think everyone is looking at, is being done by a nonprofit consortium, including a few universities in California. And this one is taking a completely different tactic as they're aiming to actually fix the mutation in the adult hemoglobin instead of doing the fetal hemoglobin workaround that all the other trials have used. So that's, that's more tricky, obviously. But really exciting. And as I said, it's a not-for-profit group. So if that works, it will be a game changer in treatment availability, for sure. So do not worry, Jesse, we'll get there we're getting there. We're getting there. So that trial is still in very early stages and has had some delays and challenges as you might expect, but they do plan to start enrolling participants for phase one early next year. So 2025. So that's the update.
Fatu Badiane-Markey 37:30
Yeah, no, I love it. There's a lot of optimism there. And I also love the question from a listener. Yeah, and I think, you know, I think that's a very valid point. And I definitely think you know, yeah, everything that you said makes a lot of sense as well. These things start off really expensive, because they're new, but then Yeah, over time, there's competition, the technology changes, and I think this price tag is going to come down a lot. So yeah, I think there's a lot of optimism here. Definitely. For the future. So super exciting. .
Shekerah Primus 38:07
Yay, so Yeah, that is it, folks for this episode of The CRISPR Chronicles. We hope you enjoyed it. We hope you learned a lot about all the clinical trials that are ongoing to use CRISPR gene therapy for treatment to treat all of these different diseases. Thank you, as always for tuning in. On the next installment of the CRISPR Chronicles, we are going to talk all about ethics and safety. So we touched on safety a little bit here. There's been a lot of talk and pushback. About how safe is this was the ethical, you know conundrums of using gene therapy, changing our DNA playing God all kinds of things like that. So in the next episode, we're going to tackle some of these really deep some, some philosophical but also some very practical scientific questions. To talk about the ethics of CRISPR gene therapy. So with that, thank you all for listening. And if you would like to reach out to us, you always can reach us at lovesciencepodcast@gmail.com So until next time, bye everyone.
Fatu Badiane-Markey 39:19
bye everyone. Last stop for the train, almost.
Shekerah Primus 39:33
Just upping the ante in our fight against all these genetic diseases that are trying to take us out
Transcribed by https://otter.ai