In today’s episode, Chad sits down with guest scientist, Dr. Laura Sepp-Lorenzino the Chief Scientific Officer at Intellia Therapeutics. Laura is an innovative leader in drug discovery and the development of small molecules and nucleotide therapeutics. Pharma is in Laura’s blood as her mother, a pharmacist, encouraged her to pursue a career in the science. She shares how she went from studying biochemistry in Argentina and her pursuit of a Ph.D., then pivoting in a life-changing move to the United States. She also talks about the importance of paying it forward as a mentor, how gene therapy can prevent diseases, gene editing at Intellia Therapeutics, debunking stigmas behind vaccines, revisiting the Rubik’s Cube, and joining a debate club!

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Molecular Moments Episode 6: Dr. Laura Sepp-Lorenzino Talks Gene Editing, Mentorships, and Making Big Moves!: Audio automatically transcribed by Sonix

Molecular Moments Episode 6: Dr. Laura Sepp-Lorenzino Talks Gene Editing, Mentorships, and Making Big Moves!: this mp4 audio file was automatically transcribed by Sonix with the best speech-to-text algorithms. This transcript may contain errors.

Announcer:
Welcome to the Molecular Moments podcast.

Dr. Chad Briscoe:
In today's episode, we sat down with our guest, Dr. Laura Sept, Lorenzino chief scientific officer at Telia Therapeutics. Laura is an innovative leader in drug discovery and development of small molecule and nucleotide therapeutics. She's also led vaccine development programs, and more recently, she has made her mark in the exciting field of gene therapy and gene editing. She has over 30 years of experience in academic and industrial settings. I first met Laura several years ago when she was the keynote speaker at the Land O'Lakes bio analytical meeting. And I've been a fan ever since. I hope you'll enjoy our conversation as much as I did. We were talking scientists as scientists do. So without further ado, here is the sixth episode of Molecular Moments. Welcome to the podcast, Laura. I'm delighted to have you join me today. Can we start with you giving us a few highlights from your career?

Dr. Laura Sepp-Lorenzino:
Chad, thank you so much for having me today. I've been listening to your Molecular Moments podcast. And, you know, it's really, really very engaging and informative. So, you know, I hope that this episode will mean that, Mark. So I'm a biochemist by training.

Dr. Laura Sepp-Lorenzino:
I was born, went to school in Argentina. Then I came to the U.S. to do my PhD the and postgraduate work.

Dr. Laura Sepp-Lorenzino:
And then I've been working in industry since 1999, so long time in industry, first at Merck, then I went to Alnylam Vertex and now the chief scientific officer at Intaglio Therapeutics really have in each of these experiences. You know, Bill, you know, my my professional experience. But, you know, it's all of them were different than it gave me the opportunity to to learn different things from, you know, the different companies. And, you know, I'm still learning. This is what makes it fun.

Dr. Chad Briscoe:
I'm really excited to explore that with you. And one of the topics that I'm curious about is how you made this transition coming from Argentina to the United States for graduate school and what kept you here. And tell me about what brings a girl then, I guess pretty young here to the U.S. So I studied biochemistry, pharmacy and biochemistry at the University of Buenos Aires.

Dr. Laura Sepp-Lorenzino:
And although my mom is a pharmacist and her wish was that I was going to take over the family business, I was always drawn towards research and I started doing my PhD the in Argentina, and my boyfriend at that time was going to start his speech in the U.S. So she sister, who is smart and very practical person, suggested that maybe we should do the the in the same geographical location. And, you know, and I quit the program in Argentina and I joined him in coming to the US. So we were both newlyweds. And, you know, I think we were two weeks after our marriage when we both started graduate school. So it was, you know, quite an awesome experience. Right. And, you know, starting our life in a different country, different language, and both of us pursuing our dreams. You know, at that time, he was a chemist. I was in biochemistry. He was really great to have each other to help, you know, support. Then he pushes forward. Right. And I can tell you that, you know, anybody who has immigrated can share this, that, you know, as you go to a different country with different language, different culture, you know, you encounter, you know, a number of challenges.

Dr. Laura Sepp-Lorenzino:
And, you know, it's good to have someone as your partner to help you figure out how to navigate those new challenges together.

Dr. Chad Briscoe:
Well, you may not have ended up as a pharmacist, but I have to believe your mother would be proud of you for what you've achieved in drug development. So your mother was a pharmacist and then you wanted to do research and move forward in that direction.

Dr. Chad Briscoe:
It's a little bit a little bit different direction. But are there any other personal connections or anything else that really drove you into science or or maybe why research versus taking up the family business?

Dr. Laura Sepp-Lorenzino:
Yeah, and I think that mom is responsible for that as well. So I grew up going to her pharmacy and, you know, she had books and magazines. So, you know, even as a young child, I got like flip through all of that and make believe, you know, that I was contributing to those journals. Right. But then one day she took me to a seminar, and you'll appreciate that they were showing a bio distribution of of a drug. Right. And they were doing this whole by the order of radiography and, you know, the distribution and disposition of the drug and seeing how that, you know, label drug, you know, was taken in the animal, distributed, how it made it to the target audience. Right. And then and I was nine. I was really young. But, you know, the guy who explain it, you know, somehow made it really understandable.

Dr. Laura Sepp-Lorenzino:
And that's when it clicked. It was like, this is so awesome. Nothing compares to this. I really got lucky because I had people who helped me, you know, with opportunities and mentoring. And these has been invaluable to me, you know, and it's so important for for us now to pay it forward. Right. To do the same for the. Or generations, without a doubt.

Dr. Chad Briscoe:
So I want to jump right into some of the science and I'm going to go right into what you're working on now in the space of gene therapy and gene editing. And maybe you can give a three to five minute primer on what's gene therapy, gene therapy versus gene editing and kind of how those play in with the modern science. Because I'm thinking when you were in that pharmacy in your mom's pharmacy, you probably weren't thinking about, wow, if there is a drug that could change your genes, you know. So tell us about that. Yeah, I know that that time.

Dr. Laura Sepp-Lorenzino:
Yeah. So gene therapy and genetic DNA are a new class of therapeutic modalities. And here we're aiming to treat diseases at the genetic level. That is, you know, the DNA level, which is the source of instructions that tells the cell how to behave in the context of the organism. What happens is that when those instructions are missing or they're wrong or incomplete, that's what leads to disease with gene therapy and gene editing, we are aiming to reset those instructions. And, you know, there are a number of analogies of, you know, using a word Edita or writing computer code that, you know, I think that they are really good illustrations to make the point. So with gene therapy, you're not changing the mustard code, but what you're doing is you're adding, you know, a small program that, you know, runs in parallel and sends new or overriding commands to address these disease causing event. What we're doing with gene editing and this is what we're doing at Intaglio Therapeutics and other companies in this space, is that we're actually going to that master code and we're changing the information in the appropriate line.

Dr. Laura Sepp-Lorenzino:
So we're either deleting an entire command line or you could add a new one, or in some cases, you know, if there's is a typo or. Right, we could just go precisely, specifically make that, you know, small change that at the end. These, you know, the driver of of disease, you know, now moving on to the language of, you know, genetics. So the you know, the deletion, it's called in knockout. So you eliminate the gene that you're not functioning or hyperactive. You could do an insertion or Enochian where you're adding a gene that has been missing. And in some cases we can do correction. And this is, you know, particularly when they are very they find mutations that we can use some of the new tools to go and and fix that. So, you know, with these approaches in particular with gene editing, the because we're changing this master code, you know, the goal that we're aiming is that this is going to be a permanent change in that cell. And we're looking to have potentially one and then curative treatments.

Dr. Laura Sepp-Lorenzino:
And we can discuss a bit more about, you know, that.

Dr. Chad Briscoe:
Yeah. And I'm going to jump to something that scares a lot of people about this. Right. Earlier when we were talking before the podcast, you heard a reference to the superheros. I was talking with previous guest with Jim McNally and said, can we create superheroes or they're gene therapy. But in reality, what really does make a lot of people nervous about gene therapy is either side effects or misusing the technology in some. Could you talk about the potential side effects and what we're doing and why this really is a safe tool for us to use to cure people? I said it's a it's a one and done cure. So, yeah.

Dr. Laura Sepp-Lorenzino:
So I'm going to I think that there are two layers in your questions. One, you know, and one is the ethical aspects of this. Are we going to be creating superheroes with, you know, special powers? You know, this is not where we're going, you know, as companies, as a field, you know, as the scientific community. Although, you know, we heard in twenty eighteen. Right. About the scientists in China in eighteen embryos. Right. And there is a moratorium on doing that then, you know, we all ascribe to that. Right. I think it's right now we're doing somatic everything, not germline editing. And we're addressing this is nothing beyond that. So then the second part of the question is, you know, how safe is this rain? Because this is, you know, Gene. So we're modifying the genes, right? So there are a number of molecular tools that allow us to precisely design the effectors, you know, and. Talking more about, you know, crispier, how the crispier machinery is going to find the exact region in the genome where we want to introduce the evidence and nowhere else. Right. So you could use a number of tools in silico and in the lab to figure that out. But also we have a number of robust, very well characterized and accepted by regulatory agencies on how we are experimentally evaluating the on target versus the potential of target defects. And the goal, as you know, we are developing human therapeutics, is to have these therapeutics present know of targets at doses that are going to be used in the humans, in the patients.

Dr. Laura Sepp-Lorenzino:
And to that you add, you know, safety margin. Right. So it's our goal is to find the safest, you know, more efficacious candidates as possible and say we'll say no regulatory agencies. Right. So, you know, the regulatory agencies in the US and Europe and, you know, other countries, they had been learning and partnering with the scientific community to understand how to do these and what to require from companies that are developing therapeutics to ensure that the highest standards are being met. So there is, you know, data now coming from, for example, sickle cell disease and beta thalassemia using CRISPR hematopoietic stem cells that have really transformed the lives of, you know, the patients that have received them. Right. And there is no ill effects. We at INTAGLIO, we have a large number of preclinical studies we have done and now we're in the clinic with our first systemically administered CRISPR drug. So, you know, as more companies and ourselves continue to advance these therapies to the clinic, you know, I believe that, you know, the data is robust and we're going to appease some of these potential concerns that, you know, people may have.

Dr. Chad Briscoe:
Yeah, and I think you touched on exactly what I wanted to mention next is that when you think about the diseases that were really and truly curing a few years ago, these seem like diseases, often without a cure. And typically when I when I think of gene therapies, gene editing, we see a lot of treatments. And in the oncology space, of course, rare diseases and also in eye diseases. So maybe you can touch again, there's a probably a multilayer answer there, but maybe you could just explain a little bit why those are the spaces where we see gene therapies being so effective for people.

Dr. Laura Sepp-Lorenzino:
So when we're thinking about, you know, how to deploy these new technologies. Right. So one of the considerations is, you know, first, what can the technology do to fix a particular biological problem?

Dr. Laura Sepp-Lorenzino:
And are there other therapies available today that are, you know, providing benefit to those patients? If the answer to that is no right. There is these unmet medical need. Right. So and how can you help those patients for new technologies? You look at opportunities where there is high and medical need and where the potential risk of taking a new therapeutic modality are outweighed by that potential benefit that those patients are going to receive. You know, and I'll draw from, you know, one example from gene therapy, right. For example, for spinal muscular atrophy. This is a genetic defect in which for Type A, you know, the patients will die by, you know, a year of age or, you know, very young through their work. Then they have access. And now by Novartis, you know, these kids are living they're riding bicycles.

Dr. Laura Sepp-Lorenzino:
You know, it's it's really game changing. And this is, you know, for me, a good example of, you know, huge unmet medical need and how in novel therapy was really able to transform the lives of these patients.

Dr. Chad Briscoe:
You can see it as the next step to a Star Trek type world where it seems that nobody is sick. Right. I mean, that's a big Star Trek fan. So I am, too. Oh, good. Well, we're good. We're going to hit that later, then. So you touched on something that I'm actually curious about. Is it because my background is not extremely strong in the gene therapy space is preclinical so traditional? The preclinical programs supporting a pharmaceutical development, you you're giving the same drug to a couple of different species, large and small, and you look for toxic effects and you know that for the most part, the animals are going to metabolize it in a similar way to to humans. And we've we've got it worked out how to translate those effects. And it's and it's pretty straightforward.

Dr. Chad Briscoe:
But in a gene therapy program, what does a gene therapy preclinical program look like?

Dr. Laura Sepp-Lorenzino:
So for any of the and this is a big difference with small molecules. Right. So, you know, in spite of differences and similarities. Right. Because in a small molecule for your species, you want to make sure that, you know, your small molecules will have activity against, you know, the row then gene, the rolling protein.

Dr. Laura Sepp-Lorenzino:
Right. So for for gene therapy and for Jeanneret in particular, more of what we're doing.

Dr. Laura Sepp-Lorenzino:
So, you know, this standard preclinical packages. Right. So you do need to look at toxicity, understand the sources of toxicity that come from the drug form and all the elements that make up the drug. And we can talk about delivery in a few moments. And the only thing that's different for for example, for gene editing, as we were just discussing, is these in-depth characterization of the target and potential of targets and showing that, you know, there is nothing that could lead to potential toxicity because the sequences, the DNA sequence is often not conserved between species. If they're conserved, no problem, because you could use your clinical candidate in non-human primates are rodents, and that will be perfect. In some other cases you may want, depending on the regulatory agencies advice. Right. You may want to have a surrogate to at least to understand exacerbated pharmacology. Right.

Dr. Laura Sepp-Lorenzino:
And that's that's really helpful, you know, but there is nothing very different from what you would do from other therapeutic modalities.

Dr. Laura Sepp-Lorenzino:
The other aspect of this is that for platform technologies in which the components are basically the same from program to program, you know, there is a lot of information that you can draw from the past.

Dr. Laura Sepp-Lorenzino:
And let me explain this a bit better. So for delivering or, you know, these nucleic acid therapies, whether it's gene therapy or gene editing, in the majority of the cases, you require what's called a delivery vehicle. And this is either in non-viable formulation, like a lipid nanoparticle or a polymer or a viral formulation. And the thing that changes is what goes inside brain. So once you understand the bio attribution, the atme properties of that delivery vehicle. Right. That's going to be the same independent of the cargo that they have. You know, and that characterization, you know, translates from program to program.

Dr. Laura Sepp-Lorenzino:
And that's one of the, you know, really nice aspects of these modular platforms. Right. Because you understand what to expect. And then you what's different is obviously the pharmacology of the gene or you're trying to elicit.

Dr. Chad Briscoe:
But it sounds like it's a little bit of plug and play in a sense, you know, that the real simplification of it.

Dr. Laura Sepp-Lorenzino:
But yes. Yeah, I you know, and that translates also on the CMC. Right. On the technical operations and, you know, all the analytical tools that you need to develop to characterize all the components, you know, the drug substance and the drug product.

Dr. Laura Sepp-Lorenzino:
So there is a lot of synergies that are gained for from, you know, some of these modular platform technologies.

Dr. Chad Briscoe:
Yeah, without a doubt. So you touched a little bit on the on the delivery and it sounded like you have more to say on that. I think I think the delivery of these therapies is one of the most exciting aspects of how they function. And it's something that that we get the opportunity, you know, talking to different companies within, you know, within biogenetics, how they're going about it and why they're going about it using different adenoviruses and things like that for delivery. So maybe want to touch on that a little more.

Dr. Laura Sepp-Lorenzino:
Sure. Yeah. So I'll start with how we're approaching it at Intaglio. So, you know, using gene editing at first, there are two major classes of uses. One is to take cells, put in them on a dish and in.

Dr. Laura Sepp-Lorenzino:
Have you seen the evidence that rewiring of the instructions that you want to have in your cell product and then you would introduce that into the patient? And, you know, for that, there is a number of stablish and more noble delivery technologies.

Dr. Laura Sepp-Lorenzino:
But there is this cell that is the therapy. Right. And the applications for that is for immuno oncology, for our immune diseases, for hematopoietic stem cell diseases like psychosocial disease and regenerative medicine.

Dr. Laura Sepp-Lorenzino:
The other big category is the use of, you know, CRISPR can sign or, you know, gene therapy, genes, replacement, inviable where now you're administering these, you know, in our case, the CRISPR machinery to a patient. And you could do that in several ways. One, he said it could be local administration, for example, separating out injection in the eye. Right. For some of these inherited retinal dystrophies. Or you could give it by inhalation to go to the lung or in the brain or, you know, intrathecal delivery for local administration to CNS. We can also give it by systemic administration, and that's by an infusion, intravenous infusion where you're delivering aid to the bloodstream. And then you need to make sure that, you know, that delivery vehicle makes it to the Oregon, the target, Oregon, and then to the cells in that target, Oregon. And these last approaches, would we are using that intaglio for our inviable portfolio for that we have chosen to use in non viral delivery system that's called a lipid nanoparticle or LMP delivery, kind of like a ball of fat if you really want to.

Dr. Chad Briscoe:
You really want to really make it simple. Lipids are fat. So it's like it's like you're delivering a gene therapy with a little ball of fat. Yeah. Yeah.

Dr. Laura Sepp-Lorenzino:
So this is like eating a big hamburger with a gift and only it's healthy. The gift that comes into those little, you know, globules is in our case, the CRISPR machinery that will make the edit.

Dr. Laura Sepp-Lorenzino:
And, you know, the body recognizes these fat globules as dietary fat. So it will process them the same way. So it's a really great means of delivery, nucleic acids to the hepatocytes in the liver. And this is the technology we're exploiting. And there are a number of advantages. You know, the delivery, you know, Liben nanoparticles are not immunogenic. So that is, you know, old patients would qualify to get them, you know, say if it's a clinically validated approach.

Dr. Laura Sepp-Lorenzino:
There is a drug called On Patrol developed by my former company alum. That's you know, it's is a commercial product because it's a chemical synthesis. You know, it doesn't have the complexities that making viruses do. So you could make tons of very homogeneous particles. And, you know, and we can there is a lot of chemistry that we can apply to make them do different things and take different cargos, potentially go to different organs. And that's what we're working on. Other companies are exploring viral delivery as their main, you know, for their, you know, first products most of them have for gene, for systemic or local administration use.

Dr. Laura Sepp-Lorenzino:
People use adenovirus associated vectors or AVY. They have different tropism. So there is a number of serotype, you know, a class of viruses with small differences on their capsid. And those differences result in the viruses being able to go to different cell types in the body. So, you know, some go grade to liver, others go to muscle. Others, you know, worked really well in the eye or in CNS. So that that is a great advantage of that technology. Some of the disadvantages is, you know, there is pre-existing immunity to the avy. So there is a percentage of the population that will not be able to receive the therapy because they have neutralizing antibodies. Fortunately, after people get a dose, they will. If they didn't have antibodies before, they will. Now, Sellery, those he needs. This is an issue.

Dr. Laura Sepp-Lorenzino:
You know, needs is effective, but, you know, could be a lot better, right? So, for example, with Liben nanoparticles, we can get to every hepatocyte in the liver with HIV, that number is much, much lower. And then the complexities of making a biologic right. So they're the the product is the process you follow. And, you know, that's that's an art.

Dr. Chad Briscoe:
And making the DNA is fairly straightforward, but making the complete drug and assembling it into a deliverable product is well, it's a whole new different science in itself, I guess.

Dr. Chad Briscoe:
I want to talk a little bit about vaccines with you, since that's obviously a hot topic right now. But I have one one more question. I just want you to think into your crystal ball quite often with the well, always with these gene therapies were repairing a very small piece of your DNA, you know, a single point mutation or word, you know, repairing a deletion. But there's so many more complex genetic diseases. I'm curious, how far do you think we can take this DNA repair or how far should we take this to solve more complex chromosomal disorders?

Dr. Laura Sepp-Lorenzino:
So I think I'm really excited about our insertion capabilities in the liver. So there we can put entire genes that have been either missing or, you know, are not functioning in the liver. And we're doing that for hemophilia and B, with our partner Regeneron and then Alpha one antitrypsin deficiency. And we can get really unprecedented levels of expression in non-human primates.

Dr. Laura Sepp-Lorenzino:
So you could put really large pieces of DNA, you know, as we're building the proof that this technology works.

Dr. Laura Sepp-Lorenzino:
There are lots of advances, you know, new ideas and new technologies and how to introduce different edits or do gene therapy more efficiently. You know, part of my job as the CSO is to have a good pulse on what are the tools that we need to have in our toolbox for us to be able to do the right type of ebeid genetic change for the right type of disease. So, you know, it's not that, you know, if you have a hammer, that's the only thing you need. You need to have, you know, a complete toolbox. And and I think that through that tool box development, we will be able to make more sophisticated and, you know, and really expand the utility of these technologies, even to the point of going after complex genetic diseases that go beyond, you know, one gene. Ray, these monogenic diseases, if they are, you know, chromosomal, no abnormalities, you know, can you fix that?

Dr. Laura Sepp-Lorenzino:
Do they? Not yet. But yeah, I would think in the near future, you know, five to 10 years, I'm confident we're going to be making progress on that as well.

Dr. Chad Briscoe:
All the diseases you mentioned that we are able to cure or close to a cure on the curing hemophilia right at a base level where it's just gone. Right.

Dr. Chad Briscoe:
I mean, imagine that in the many other diseases. So I'm really excited about that. You have a background in vaccines.

Dr. Chad Briscoe:
Honestly, the vaccines haven't been very exciting for a long time other than immuno oncology. Right. Immuno oncology aside. But vaccines haven't been all that exciting for a long, long time. Right. And it's programs that a lot of companies have just kind of they kind of kept them along because it was it was a good thing to do.

Dr. Chad Briscoe:
And, you know, and they probably made some money, but that all changed with the pandemic. So tell me a little bit from what you've seen, how how vaccines have changed over the last 15 years or so?

Dr. Laura Sepp-Lorenzino:
Yeah, and let me qualify my experience here. So when I was at Tomato, I had the opportunity to be part of the Vaccines Research Group. So, you know, I learned from a really talented, you know, team, you know, John Shavar, who's now at Sanofi Pasteur, was, you know, my my boss and the leader of the group, you know, and Catherine Janssen, who is leading the Pfizer, she was a marketer. So it was a really great, knowledgeable group of people. So, yeah. So vaccines people have taken vaccines for granted to what you were saying.

Dr. Laura Sepp-Lorenzino:
Right. You know, for me is, you know, vaccine without that is the most impactful achievement in medicine. Right. So if you're looking at a world has really saved lives and eradicated vaccines, that's vaccines. You know, and take it from Jenners, you know, vaccine for smallpox to, you know, now that was eradicated to all the, you know, childhood diseases.

Dr. Laura Sepp-Lorenzino:
You know, when even when I was a little kid, you know, people get measles, you know. It's gone, right?

Dr. Chad Briscoe:
I was actually telling my son just yesterday about my mom sent me out to catch chickenpox when I was maybe four or five years old. Go get chickenpox now because it's convenient, right? And he's like, well, nobody gets chickenpox. That's because we all get vaccinated, Jack.

Dr. Laura Sepp-Lorenzino:
Exactly. Like, you know, you haven't entered kindergarten, so go get it because we don't want you to miss school. Exactly. Exactly right. How things have changed so particular.

Dr. Laura Sepp-Lorenzino:
I'm very excited about the irony. You know, Lippitt Nanoparticle vaccines for obvious reasons, right? I mean, this is what I'm working on, the example of how, you know, biotech and Mother Nature. Right.

Dr. Laura Sepp-Lorenzino:
Have rapidly, you know, identify potent vaccines for covid-19, you know, the scalability, the speed and the efficacy and how now that's also being applied.

Dr. Laura Sepp-Lorenzino:
As you know, these variants of the virus are emerging, how quickly they can react. Right. And modify, you know, the vaccine, you know, as needed.

Dr. Laura Sepp-Lorenzino:
This is you know, it's a leap, you know, close to to my colleagues doing that, doing that.

Dr. Chad Briscoe:
And neither of my parents were scientists. So when they were fortunate enough to get the first shot of the vaccine and I think my I think my mom gets her next shot on Saturday. She's a week ahead of my dad because she got the Fizer. So so she's excited about that. But I was telling her when she got the vaccine, she's like, OK, so how long will it take before I have antibodies and how does it work? I said, well, first get your vaccine and then visualize your ribosomes working hard, right. Cranking, creating the RNA and making the proteins. So then your immune system can kick in and do their job. And so I was trying to give her a little a little push. Right. Like visualize what your immune system is doing to to fight this virus. So I like that you qualified your experience a little bit in your in your expertise, because I've seen that with a number of the scientists I talked to who who I absolutely consider experts. But but then as soon as you see vaccine, they say, oh, Laura's definitely an expert. And you are right that, you know, there's different levels. But I get asked all the time also those questions because they know I'm a scientist, but for Pete's sake, I'm a chemist. Right. So, so so, you know, we all become virus and vaccine experts.

Dr. Chad Briscoe:
One of the things that's really exciting with the vaccine development and you touched on is how rapidly those were developed.

Dr. Chad Briscoe:
The groundwork was there, but the disease didn't exist a year and a half ago. Now we have a cure, if you will, in the form or in the form of a vaccine or prevention, I guess you would say. But do you think we can take that lesson of developing a drug in nine months or developing a vaccine in nine months and apply it to other drugs and other therapies with critical unmet needs?

Dr. Laura Sepp-Lorenzino:
Yes, I think so. And for Olympias and vaccines. Right. So big difference with developing biologics or small molecules, right. Where you see an enormous space for nucleic acid technologies. You know, the code is written. You just need to make it right. So that cuts years of, you know, drug design. You know, it's you already know what the instructions are. You know, in the case of, you know, this spike protein for sars-cov-2, you know, once that that was done, you just make it right and so forth. This is in which, you know, you can apply these modular solutions. I think that you can move fast, you know, and examples. You know, some of my colleagues in the Olegas Therapeutic Society meeting, you know, we you know, as you know, there are multiple nucleotide type of drugs being developed, whether it's short interfere in irony's or Sarina's or a single strand, you know, antisense and splice switchin type. Olegas, you know, those have been clinically validated. You can make it, you know, the synthesis, the characterization, all of that excess. So people are now beginning to think about using that platform that exists for enough of one type of, you know, therapies. Right. So for these hyper personalized therapies will need to figure out, as you know, as a field how to really execute through that because, you know, it's today the cost and, you know, it's not there yet.

Dr. Laura Sepp-Lorenzino:
But, you know, that is a really great example. For that was the lead was Teemu at Boston Children's Hospital, right where he there was a patient with a rare disease. He identified a mutation and developed a single strand of the nucleotide and started treating that patient. That was then that he said. All of this is published. So I don't know exactly the time, but it was months, right, between the sequence and being able to treat that patient. So is it possible? Yes, it's possible. How much of that we're going to see being implemented will need to wait and see?

Dr. Chad Briscoe:
Well, my head's exploding with this is amazing. Really, really have enjoyed listening to all the science. I want to talk about mentors a little bit. That's something I try to hit on in all of my molecular moments episodes.

Dr. Chad Briscoe:
And you I have to believe that as a CSO, mentoring is a big part of that. And I feel like you've taught me so much just sitting here today. So and you mentioned it actually earlier, the mentoring that you receive. Can you just talk a little bit about that in your experience, sir?

Dr. Laura Sepp-Lorenzino:
Yeah.

Dr. Laura Sepp-Lorenzino:
So I think that mentoring is one of the great gifts that you can give someone and that you can receive, you know, because here, you know, people are sharing with you the wisdom of their knowledge and their experiences.

Dr. Laura Sepp-Lorenzino:
And and for me, you know, it's one of the core components, as you know, as a leader and as a people manager. So I didn't tell you. We are very keen in, you know, in working with our teams, with mentoring and also really elucidating different career paths.

Dr. Laura Sepp-Lorenzino:
Right. Because, you know, sometimes, you know, when you go to grad school or even in college. Right. It's kind of, you know, very limited. What, you know, the opportunities are. Right. So for me, I had formal and informal mentors. You know, throughout my career, I have a very close group of friends. We call each other the Wonderwoman. We work together at Merck. You know, we all come from different backgrounds and we help each other, you know, and this is like a sounding board. One of the things I really enjoy with them, he said, you know, you can be completely open and vulnerable because you know that, you know, they really want to be there for you and provide, you know, these actionable feedback. Right. So here's a problem. This is how I approach it. What did you do not do? So, you know, I encourage people to to have these, you know, group of people that are going to be with them throughout their career. So I think that's really, really important. The other is the misconception that in a mentoring, you know, relationships, someone has to be much higher than, you know, the mentor needs to be much higher than the mentee. And I don't think that applies. Right. So I have gained a lot of knowledge from people I mentor. Right. Perhaps more than what they've gotten from me. So, you know, and you could have, you know, peer mentoring, which is, you know, very important to us as well. The other is I mentioned before about this concept of paying it forward. So as people have help, you make sure you do that to to others, you know, and at the end, it all is a big circle. Right. Because, you know, it comes back to you at one point, karma, good karma, bad karma.

Dr. Chad Briscoe:
To just want to touch on one last point with you. Boy, we're going on about a year now of covid in lockdown, started in March. Anything you took on in twenty twenty or, you know, over the course of covid, anything you focused on? What's been your outlet here over the last year? Almost a year.

Dr. Laura Sepp-Lorenzino:
So the silver lining of covid times was that I could spend more time with my family. And, you know, I'm really grateful about that hobbies. I spend a lot of time in meetings. I'm in my office. So, you know, besides learning how to solve the Rubik's Cube, which was something that was pending from my high school years, I guess I've been doing, you know, growing succulence, which is it's so fun.

Dr. Laura Sepp-Lorenzino:
I mean, it's slow. I wish I could speed it up a bit, but, you know, it's it's fun, you know, and the other is using, you know, sume and Skype to connect with people.

Dr. Laura Sepp-Lorenzino:
I mean, a lot more intentional about, you know, doing that. So, you know, I've re-engage with even many of my friends from high school. Now, we have a debate club every month where we talk about different topics and it's really fun.

Dr. Chad Briscoe:
That's fantastic. Laura, thank you so much for coming on and being a guest a.

Dr. Chad Briscoe:
I feel like I have a new mentor in the gene therapy space. I got so much out of this. Thank you so much and I would love to have you back maybe in a year or two and we'll see where things have gone. We can get an update on intelligence research.

Dr. Chad Briscoe:
There's so much we could have clearly, we could have talked for two or three hours. I think I could listen to you. So thank you so much for joining us today.

Dr. Laura Sepp-Lorenzino:
Yeah. And thank you again, Chad, for the opportunity.

Dr. Laura Sepp-Lorenzino:
And I hope to see you in Boston sometime soon. Absolutely. And I have a nice dinner.

Dr. Chad Briscoe:
Well, that's all for Episode six, if you enjoyed today's episode. Be sure to subscribe on Apple podcast Spotify or your favorite podcast app. So you never miss a conversation if you'd like to hang out with us outside the podcast. We have many webinars and other presentations available for your enjoyment and education. Visit Biogenetics Dotcom to see what's coming up and how you can stay in touch. And don't forget to keep an eye out for more episodes coming soon. We're looking forward to some great guests will have world renowned experts talking about rare diseases, diversity in the pharmaceutical industry, new and exciting technologies, and a conversation with a patient who has benefited from the recent tremendous developments in our industry.

Dr. Chad Briscoe:
Molecular moments would not be possible without the support of our sponsor, Biogenetics Labs Biogenetics is a global contract research organization specializing in large molecule bio analysis based in Durham, North Carolina, with labs in Hamburg, Germany, and Boston, Massachusetts. Biogenetics provides high quality bio analytical services to leading pharma and biotech companies around the world. They offer assay development, validation and sample analysis under Nagbe, JLP and GCP, as well as GMP quality control testing. If you are looking to work with a team of highly experienced scientific and kuai professionals through all phases of clinical development, look no further than biogenetics. For more information or to speak with their scientists today, visit their website at Biogenetics Dotcom.

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