Latest / The BioHub - by Avetix / Episode 152 - Nazli Azimi - Co-Founder & CEO - Therna Bio
Transcript
- 0:06Welcome to another episode of the Bio Hub podcast.
- 0:09Today I'm joined by Nazli Azimi, Co Founder and CEO of Therna
- 0:14Biosciences, a company building AI driven platforms to design
- 0:18programmable RNA medicines. Nazli has previously founded and
- 0:23scaled 2 biotech companies through to acquisition and is
- 0:26now focused on combining deep biological expertise with
- 0:29artificial intelligence to rethink how RNA therapeutics are
- 0:33designed. In today's conversation, we'll
- 0:36explore the evolution of AI in drug discovery, what
- 0:39differentiates donors approach, and where the future of RNA
- 0:42therapeutics is heading. Naslie, welcome to the podcast.
- 0:46Thank you, Amber. I'm excited to be here.
- 0:49Amazing. And to start, I'd love to give
- 0:51the listeners a bit of context on your background.
- 0:54And you've built and exited 2 biotech companies already.
- 0:57What led you to start Thona and move into the AIRNA space?
- 1:02Yes, maybe I'd start by giving a little background about myself.
- 1:06I'm a scientist by training. I did my postdoc at the NIH and
- 1:11worked on immunology. I was fortunate enough that as
- 1:15part of the lab that I was working on at doing my postdoc,
- 1:19we discovered a very important cytokine at that time.
- 1:23So it created a lot of opportunities for all the
- 1:27members of the lab, including myself.
- 1:30So we delved into characterizing this newly discovered cytokine
- 1:36and figuring out what what are its function.
- 1:39And you know, a lot of publication and good discoveries
- 1:43came out of it. And that cytokine Illinois 15
- 1:46turned out to be a really important regulator of the
- 1:50immune system. So that as part of that journey
- 1:55I started, I started questioning what are the diseases that could
- 2:02potentially be affected by regulation, by dysregulation of
- 2:05this cytokine. And I published on that.
- 2:09And then it turned out that it's really important cytokine, one
- 2:14of the master regulators of the immune system.
- 2:17And in certain diseases there is a dysregulation in production of
- 2:22the cytokine. So the obvious next question in
- 2:25my mind was what, what do we do? You know, if I want to block
- 2:29that cytokine, it's overproduction so I can have so
- 2:34it would have some therapeutic effect in these diseases that
- 2:39are driven by the overproduction of the cytokine.
- 2:42Why do I do that? And that was the beginning of
- 2:45how did my previous biotech companies started.
- 2:49I came up with a very novel way of targeting and blocking these
- 2:55cytokines in the context of these diseases.
- 2:59And then I started, you know, developing that 1st, the concept
- 3:03of it that day, NIH and later on when I transitioned to
- 3:06entrepreneurship. So the, the thing that's was
- 3:11interesting in my journey, when I reflect back now, is that why
- 3:15I felt compelled to do that. It was, you know, I, I was
- 3:20never, I was never brought up to think about entrepreneurship,
- 3:25never had any desire. I just, all I wanted to do is
- 3:28just to do research and publish paper and give lectures.
- 3:33And I did pretty good good at that while I did my postdoc at
- 3:38the NIH. But one thing that became clear
- 3:40to me is that my, my brain worked in a very direct and goal
- 3:47oriented fashion. I had, I was focused when I was
- 3:51setting up an experiment. This is the experiment I want to
- 3:53do. This is the answer I want to get
- 3:56and I was always able to bring it into a conclusion, to
- 4:01meaningful conclusion. Yes or no or you know, cannot
- 4:04continue on this. A lot of brilliant scientists,
- 4:10they follow their desire different paths and they just
- 4:15wander around wherever their curiosity takes in, which is
- 4:18amazing. We definitely need those
- 4:20peoples. But my brain was more, I want to
- 4:23see a conclusion in the work that I'm doing.
- 4:27And then I realized that this is this mentality is very well
- 4:30suited for taking this concept, scientific concept and turn it
- 4:38into an application like this is what it means, the discovery.
- 4:42So what I'm going to do with that, that type of question,
- 4:46that type of transition. So I realized that in order for
- 4:50me to do that, maybe I need to pursue entrepreneurship.
- 4:54After a night, I I got a job at Fritz Hutchinson as a faculty
- 5:00member and I was there. But, you know, it was a moment
- 5:06for me to realize, do I want to be in academia or is it time for
- 5:10me to move on to something else? And I chose the latter.
- 5:15It was a really hard decision for me to leave academia, but I
- 5:20I felt that I can have a better impact.
- 5:23I can finish the job that I started at the NIH of I
- 5:27discovered this. We or our group discovered this
- 5:30cytokine. I discovered the connection of
- 5:33the cytokine to some diseases. Now I want to see if I block
- 5:37that cytokine, can I see some therapeutic effect?
- 5:40So I started the companies around that very interesting
- 5:45learning curve, loved it, loved the whole experience.
- 5:49I'm, I'm, I'm happy that we managed to take that the, the,
- 5:56the, the, the strategy, the medicines that we created into
- 5:59clinical trial and show efficacy and, and all that good stuff.
- 6:03When you were first thinking about Ferna, what was the key
- 6:07problem in RNA therapeutics or drug discovery that you felt
- 6:11wasn't being solved properly? Yeah, when when I met the
- 6:14scientific Co founders of of Therna, what became clear to me
- 6:20was the potential that an approach that we are pursuing at
- 6:26Therna could solve. Because if you think about it,
- 6:30RNA has been overlooked. Understanding the RNA biology
- 6:35has been overlooked as part of the central dogma being genetic
- 6:41material in the DNA, transferring the information
- 6:45through the RNA into protein. The focus has always been let's
- 6:50figure out the genetic material, the DNA, the efforts on the
- 6:54sequencing in the human genome progress, Human Genome Project
- 6:59and many other efforts figuring out what is the genetic
- 7:04component of of life is made of. The focus has been a lot on that
- 7:10and also on the protein because life, the functionality of life
- 7:15is exerted through protein. So a lot of effort has been done
- 7:19of what the proteins are, how do they behave, what's destruction,
- 7:22structure, function, relationship of the protein, but
- 7:26not much effort on what is this transition transitional molecule
- 7:33called mRNA is doing. And in the past few decades that
- 7:36scientists started figuring out and just asking this question,
- 7:41what's what's in it? You know, are there any
- 7:43uniqueness about properties in there on the RNA are the ways
- 7:48that the expression of any given gene has been controlled through
- 7:53the through the mRNA. And my conversation with with
- 7:59Honey and the focus on the RNA resonated with me because when I
- 8:04was at the NIH, one of the project that we heavily focused
- 8:08on when we discovered the cytokine R15 was on relational
- 8:15control on this cytokines mRNA. Because this particular mRNA is
- 8:21heavily controlled not at the chain level surprisingly, but at
- 8:25the mRNA level. So I knew there are many levers
- 8:29that one can pull and push in order to control the expression
- 8:34level at the RNA. So this is this was something
- 8:39that I realized that has been missing just because of the lack
- 8:42of understanding. The whole industry doesn't think
- 8:46about it that that well that deeply.
- 8:49And I hope that with the work that you're doing, implementing
- 8:54some AI, we could shed some light into our new biology in
- 9:00more systematic way so we can leverage it for further drug
- 9:04development and discovery. And there has been a huge wave
- 9:09of AI drug discovery companies over the past few years.
- 9:12Where do you think that first generation fell short?
- 9:16Yes, very good question. It goes back to how much do you
- 9:23know about the biology of the RNA?
- 9:27Because when you talk about the AI and you talk about the
- 9:32morally, it all comes down to data.
- 9:36If you have enough data, you can build any model.
- 9:41The question is, is that model good enough?
- 9:44Is that model is doing something appropriately?
- 9:47Can it have the right prediction?
- 9:49If you don't, if you don't train your model or if you don't build
- 9:55your model based on appropriate data, it's going to be garbage
- 9:59in garbage ads. So the core advantage to be the
- 10:05main advantage or the OR the fundamental advantage that we
- 10:09have at Turner is that the root of the company goes back into
- 10:15biology and AI. And I cannot emphasize it enough
- 10:20because if you don't understand the biology of the RNA, you
- 10:24don't ask the right question, you don't set up the proper
- 10:28experiments, you don't generate the right data.
- 10:32And then again, you have a model that it's a model which is not a
- 10:35good model. And the benefit, our advantage
- 10:39is that the legacy of the company goes for many, many
- 10:43decades on the fundamental work that our founders, our
- 10:47scientific founders have conducted an understanding what
- 10:52mRNA is, what are the elements that control its regulation, in
- 10:58what context, in what tissue, in what organ, what are the
- 11:04elements that are affecting its three-dimensional structure.
- 11:09Any possible scenario that could affect the RNA regulation, we
- 11:17have taken into consideration when we set up our own
- 11:20experiments and these experiments are different set of
- 11:26readouts, different set of outputs with tremendous amount
- 11:31of data that are proprietary to us.
- 11:35So we can interrogate mRNA, you can interrogate RNA at every
- 11:41potential angle, every potential, every potential
- 11:46question that one can have, what could possibly have to
- 11:51understand the RNA and its relationship within the cell
- 11:56with other proteins with the DNA that it's get translated from.
- 12:01So all of that collectively makes a tremendous amount of
- 12:06proprietary data that it's unique to us and it's relevant,
- 12:10it's biologically relevant because now you can build your
- 12:14model based on your data with the confidence that the good
- 12:20with the with the proper ask input, you can have a reliable
- 12:26output, whatever that output is going to be, that you can be
- 12:30reliable. The first generation of the AI
- 12:34companies that came out, probably very likely it lacked
- 12:40this fundamental biological knowledge.
- 12:45Therefore, there are models, there are robust models, but the
- 12:49output is not necessarily reflective of the biology or
- 12:54grounded in biology. So not not relevant.
- 12:58And this is the problem that you're trying to solve by
- 13:01building a comprehensive set of datas that are rooted in
- 13:09biological system, validated in biological system.
- 13:13So we are actually our lab is mostly our company is mostly wet
- 13:18lab, meaning people are actually doing experiments, taking those
- 13:22data, giving it to their AI scientists who are doing the
- 13:26data build out training, remodeling and building and
- 13:32training the models. So we have confidence that the
- 13:36output is going to be meaningful and biologically relevant.
- 13:41That's a great insight into how Ferner is building things
- 13:44differently when it comes to that biological setting.
- 13:48And so you are building a platform that can design those
- 13:51RNA molecules with very specific properties.
- 13:54What does that actually look like in practice?
- 13:57Yes. So when we have this different
- 14:02models and each of them have a different functionality based on
- 14:06the questions that we had asked during setting up the
- 14:09experiments, getting that lab data, laboratory data and
- 14:14feeding them into the Model 1 of the questions.
- 14:17Is that, for example, is there a way that you can make an RNA to
- 14:24be on in certain cells or tissue and off in others?
- 14:30In other words, is it possible, for example, for us to make an
- 14:35RNA that is tissue specific? You can say, you know, I want, I
- 14:39want my RNA to be only on or expressed in my in muscles, but
- 14:46not in anywhere else. Is that possible?
- 14:49So we have built models to address that specifically.
- 14:55And it's a process where you set up this experiments because you
- 14:58understand the biology, you set up this experiments and then you
- 15:04come and collect this data and you feed them into the into your
- 15:11models in a setting that we call it lab in the loop.
- 15:15So, generation of the data goes into the AI model, trains it,
- 15:20send us some output, that output get tested and then data goes
- 15:25back into the model, retrain it and this thing can go on and on.
- 15:31So you can have that those asks, you can have those proper
- 15:38characteristic as part of the model and then build this
- 15:44different models together. And what we have done at Derna
- 15:49is that we built different models on tissue specificity.
- 15:54We have different model to control the longevity of the
- 15:59RNARNA is short lived by nature because it's job is to naturally
- 16:07take the information from DNA to protein and then disappear.
- 16:12That's, that's what it was intended to do.
- 16:14But if you're thinking about use RNA as a, as a medicine, you
- 16:20want it to be long lasting. So how can you do that?
- 16:23So a tremendous amount of work work has gone into doing
- 16:28experiments to address that. So we can leverage specific
- 16:32properties to make the OM RNA long lasting.
- 16:35So there's a model for that. There's a model that can address
- 16:39the splicing within the RNA. It's a property that different
- 16:43part of RNA can be spliced up and stitched together.
- 16:46You can have a prediction on that.
- 16:48A model that talks about, you know, how the the
- 16:53three-dimensional structure of the RNA could affect its, it's
- 16:57transcription, translation, and regulation.
- 17:00So all of that stacked into on top of each other.
- 17:04And then at the end you can ask basically to have an mRNA that
- 17:13is bespoke. You can ask an mRNA and say I
- 17:19want to have an mRNA to be only specific and expressed or on in
- 17:23T cells but nowhere else in the body.
- 17:27So it can be cell or tissue specific.
- 17:30And I want it to last for three days and you can incorporate it
- 17:34that and I want it to be expressed at such and such
- 17:37efficiency. So I want it to be translated
- 17:41into protein at this level you can find to that.
- 17:44So all of these are properties that you can demand the model,
- 17:48our model to make that for you. And the outcome, the output is
- 17:54actually is an mRNA with this specific properties that you can
- 17:59take and you can test it in the lab and to see whether to see
- 18:03whether the the mRNA has this programmable components in it.
- 18:09And that's something that we can think about.
- 18:12Could it be an medicine to treat such and such diseases or not.
- 18:17So that's, that's what part of the equation.
- 18:22On the other side again because we have built this model that we
- 18:27call it RNA intelligence model, it's an AI system.
- 18:31We have this RNA intelligence knowledge.
- 18:34We can also leverage it to target the mRNA with modalities,
- 18:42well established modalities, oligonochlorides, antisense
- 18:47oligonochloride, Asos or SIRNAS that can that can control the
- 18:53expression at the mRNA level. You, they can target it to
- 18:58silence the mRNA expression or you can use them to up regulate
- 19:03or fine tune the mRNA expression.
- 19:06Again, that insight of where and how where to target the mRNA and
- 19:12how to design this ASO or SIRNAS also is derived from our RNA
- 19:18intelligence model. So we can do both.
- 19:22We can use our RNA intelligence model as a way to as a
- 19:29generative model to make programmable mRNA with the
- 19:33properties that we want. We can also design ASO and SI
- 19:38RNA that are identified and designed based on our RNA
- 19:45intelligence model. So it's a very, it's a the most
- 19:50comprehensive a model out there that allows to make any kind of
- 19:56RNA medicine for any purpose that is needed based on based on
- 20:02patient's need. And I think the most interesting
- 20:06thing about the model and your platform is that you're already
- 20:09having some real world impact. Can you tell the listeners about
- 20:12the example that you shared with me about designing that RNA
- 20:16molecule for a pulmonary fibrosis patient?
- 20:19Yes, you know the the objective, the whole purpose of this
- 20:25building, the platform, this RNA intelligence platform is to
- 20:28apply towards making new medicines.
- 20:32And the way that we believe we at Ferno are uniquely positioned
- 20:37to do that is because we have all the capabilities that are
- 20:42needed for making these medicines.
- 20:48It starts with the biological understanding of the RNA, the AI
- 20:53capabilities to build these models and having the right
- 20:58talents and experience in how drug development works and the
- 21:03drug developments are all these three components.
- 21:07The biological understanding, the AI modelings and the drug
- 21:11developments, they're all hand in hand, work hand in hand, work
- 21:16hand in hand from the beginning, from the day one in order for us
- 21:22to think about how we can make these new medicines.
- 21:26So at Eternal we, we are doing, we are making medicines for the
- 21:33large populations and also for patients who are most in needs.
- 21:40And this is the patients that you're referring to who had the
- 21:43long fibrosis. But let me spend a little time
- 21:46talk about the most standard drug development part and that
- 21:54is figuring out where the need is, where is the unmet medical
- 22:00need is. So for that we look at some
- 22:05diseases for the larger populations and whether the
- 22:11current medicines are serving them.
- 22:15And if not, is there something that they can do with our
- 22:19technology to address those unmet medical needs?
- 22:23The population is usually large enough that we can think that if
- 22:28you make this medicine, this many people could benefit from
- 22:32that. And that's how we think about it
- 22:36from day one. There is an unmet medical need.
- 22:39We need a medicine to have these properties.
- 22:42OK. Let's go and see whether we can
- 22:43make that medicine or not because there are enough people,
- 22:48there are enough patients suffering from that disease that
- 22:53once the medicine is made, we can actually run the clinical
- 22:57trials. And the clinical trial is
- 23:00basically is an experiment where you take your medicine and you
- 23:04give it to some patients and you ask the question, are you
- 23:07benefiting from taking that medicine?
- 23:10If does this medicine have any therapeutic benefit for you, for
- 23:15this patient population or not, That's the purpose of the
- 23:18clinical trial. The size and the design of it
- 23:22totally depends on the disease and how big is the population.
- 23:26So we are doing that for some diseases that we're going to,
- 23:32we're going to disclose hopefully later this year.
- 23:35But then you come across some patients that have this very
- 23:42rare genetic or genetic problem that causes the disease.
- 23:52We refer to them as ultra rare patients.
- 23:55They have ultra rare diseases, ultra rare mutations or for
- 24:00short. And A1A patient has a
- 24:03dysfunction in a gene that is very unique to that patient.
- 24:09So what do we do with that? If these patient population are
- 24:15not part of the larger group, does it mean that we cannot make
- 24:20any medicine for that for them? So far it has been the case
- 24:26because there has to be some commercial monetary benefit for
- 24:33making bed, for making medicines.
- 24:35All these pharmaceutical companies, they're really doing,
- 24:39they're doing an amazing job working for the patients.
- 24:43But at the same time it's a business that needs to, that
- 24:47needs to make sense. And for the patients who are
- 24:51suffering from these ultra rare diseases, that monetary benefit
- 24:56and reward is just not there. So it has been literally
- 25:00impossible to serve them, to help them.
- 25:05So what our platform can offer to them and did offer to this
- 25:11patient who had lung fibrosis is that they they came to us and
- 25:16the patient is suffering from the severe form of lung
- 25:19fibrosis, very, very critical condition and they want a
- 25:24medicine as fast as possible for this patient.
- 25:29And what we have to offer is that this RNA intelligence AI
- 25:35platform that allows us to make medicine for this individual,
- 25:41for a patient in a very time efficient manner.
- 25:47Usually traditionally the time that takes to make this
- 25:52medicines for large population are in the years like 3 years,
- 25:57two to three years until you have a molecule that you can
- 26:00test it in in animals and then take it into human to see
- 26:04whether this thing is work or not.
- 26:07So our model allows us to do that, allows us to make medicine
- 26:13that were not even possible before in an unprecedented
- 26:18speed. So for this particular patient,
- 26:21it was less than the actual molecule design was in 2-3 days
- 26:27and the whole testing it in the context of the lab in the loop
- 26:31was completed in less than three months.
- 26:33So this is very, very important because a patient who comes and
- 26:38this patient is suffering from a serious severe ailment, how do
- 26:43we help that patient? You need to make a medicine that
- 26:46works for that patient but also in a very time efficient manner
- 26:52as fast as possible. And this is something that we
- 26:55were able to do for this patient.
- 26:57And now that that medicine is going through the pre clinical
- 27:02testing as fast as possible and we're hopeful that the patient
- 27:06can be dosed and benefit from this medicine in, in few months.
- 27:13And we could see the impact of our work in real time in, in a
- 27:19matter of months to save a patient's life.
- 27:24And it's extremely rewarding for me for everyone at Derna to be
- 27:29able to do that, not only in a like more traditional drug
- 27:34development that we're doing for the larger population, but also
- 27:38for these patients, for this and one individuals who are
- 27:42suffering from this ultra rare diseases.
- 27:46Because if we don't, who would? Who would serve these patients?
- 27:53And how do you think that that approach could be scaled to
- 27:56reach a broader group of patients who need personalized
- 27:59treatments? I I think about it Amber as like
- 28:03how is it? How, how we can make this
- 28:06accessible to a larger group of individuals, each of them
- 28:12suffering from a very unique and ultra rare genetic disease.
- 28:18And we have been approached especially since we had that
- 28:21announcement almost every week with different groups, with
- 28:26different individuals, parents who have their baby diagnosed
- 28:32with the lethal, with the, with the deadly genetic disease.
- 28:38And I ask that question, how, how we can help them because
- 28:43some they have the resources, majority don't.
- 28:46So who, who is, who is going to come up with the, with the
- 28:52system, with the mechanism that we can, we can serve these
- 28:56patients. Our view at Thermal is that we
- 29:00have these unique capabilities that can make these medicines
- 29:05that were not even possible few years ago and we can do it
- 29:11really fast. And again, time is of the
- 29:13essence for these patients and we can offer this, this life
- 29:19saving treatment for these patients.
- 29:23But the fundamental question is that how the how it can be
- 29:29supported, how this work can be supported because there are
- 29:32costs associated with it. And although our view at Thermal
- 29:37is that we're not looking at this opportunity as commercial
- 29:40opportunities because it's just they're not, this is just for a
- 29:44patient saving their lives. A medicine for one patient just
- 29:49cannot be a commercial opportunity.
- 29:51And that's not our intention. But we need to cover our cost.
- 29:55So we are willing to do that as part of our commitment to our
- 30:01community to serve them and we continue to do that.
- 30:06But how can we think about in a system where we can reach these
- 30:15patients who do not have the resources, maybe a minority of
- 30:19have the resources to provide them with this, with this life
- 30:23saving treatments. And I don't have an answer for
- 30:26that. And I think this is something
- 30:28that we as the community of the life science researcher, as a
- 30:35community of the drug developer need to answer it and think
- 30:40about it as whether we can come up with the mechanism to do it.
- 30:45Should it be a government, private public relationship
- 30:52support? Could it be a nonprofit system
- 30:56that supports it? I don't know.
- 30:59But I intend to explore it to see whether we can come up with
- 31:04a strategy that next time when a patient comes to us, a dad or
- 31:08mom comes to us and they say their child have this disease.
- 31:13And if we know that our platform can help that child, I can say
- 31:18yes, in spite the fact that they don't have the resources to
- 31:23support the cost associated with making this medicines.
- 31:27And this is something that we as a community of within the life
- 31:33science research ecosystem and also drug development, biotech,
- 31:38pharma industry, we have to think about it because now we're
- 31:43at the stage that the technology can answer that question.
- 31:47We just have the capability now it comes down to how to come up
- 31:52with the resources to make it a reality.
- 31:54Yeah. And such an important topic
- 31:56across across the market as well.
- 31:59But maybe thinking about further forward.
- 32:03Yeah, thinking further forward with RNA therapeutics.
- 32:07You know, they had a huge moment with RNA within vaccines.
- 32:11But where do you see the field moving over the next 5 to 10
- 32:15years? Yeah, RNA definitely is just
- 32:20finding its place in the spectrum of the modalities for
- 32:26for drugs from small molecules to biologics.
- 32:32Now gene therapies, they're finding, finding their footing.
- 32:35So is the RNA medicines and RNA medicine.
- 32:40If you categorize them into mrnas, we have to successfully
- 32:44the mRNA vaccine and also ASOS and SI RNAs which target the RNA
- 32:50and control the gene expression at the RNA level.
- 32:53They're definitely they're having, they're having the
- 32:55moment and then maybe you start with the ASOSIRNA side which
- 33:01target the RNA. I think the next generation of
- 33:04those molecule are going to be the ones that are going to up
- 33:09regulate the expression of the gene at the RNA level and also
- 33:14the capabilities of fine tuning that we've gotten really good at
- 33:19knocking down the expression, basically shutting down the
- 33:23expression at the RNA level. Number of them has been approved
- 33:27in the market, they're doing really well.
- 33:30But for capabilities of these Asos and srna to up regulate
- 33:36their, the gene expression is something that you're working on
- 33:40and there are like amazing opportunities that you're trying
- 33:43to explore. I think that would be the next
- 33:45generation on that front on the mRNA level, still a lot to go.
- 33:51It's the it's still in this in my view, it's still in their
- 33:55infancy. We certainly saw the potential
- 33:58in the mRNA vaccine. But the next frontier will be
- 34:02how you're going to treat the chronic diseases, not, you know,
- 34:07targeting broader group of the tissues, broader group of cells.
- 34:13Because of that, it is very much dependent on the delivery
- 34:17component, which is a big challenge.
- 34:21But there's tremendous amount of resources on time and talent is
- 34:25going into that. So I'm very hopeful that finally
- 34:29there will be a breakthrough on what would be a delivery system
- 34:33that could target the cargo, cargo being the mRNA to the the
- 34:39tissue or the cells that that we need this mrnas at a lot of
- 34:49acquisition and MNA happened. If you have few, few of them in
- 34:53the past, past year around the targeting of the immune cells
- 35:00for the in vivo gene editing where the cargo is the mRNA,
- 35:07there are there are benefits of doing that because the mRNA
- 35:11doesn't integrate permanently into the host genome.
- 35:15It does its job and it just disappears.
- 35:18So there's less chance of the side effect.
- 35:21It could be programmed as we discussed with technologies like
- 35:26ours to where it needs to go. For example, you want it to go
- 35:30only to be in the T cells, in the immune cells, in the muscle
- 35:34cells. You can we can program the mRNA
- 35:38to be active only in that, in that tissue or cells that that
- 35:43matters. These are the technologies that
- 35:45we have. What we think it's going to be
- 35:48the future at the at Therna is that we add a targeted delivery,
- 35:55which we are working with, with the technology that in a
- 36:01collaboration with Upenn, a targeted delivery with the mRNA
- 36:07that is programmed. These two combine together, they
- 36:12can provide an elegant and exquisite way to deliver the
- 36:17medicine to where it matters and where it intend to be.
- 36:21I don't think that the targeted delivery alone would be the
- 36:24answer. I don't think the programmable
- 36:28mRNA would be the answer. But these two together, they
- 36:32would be the in vivo editing or the in vivo therapies, the 2.0
- 36:39that there's so much hope and excitement for it.
- 36:43And I think that's going to be the future.
- 36:45And we certainly working towards that our focus our technologies
- 36:50on the cargo by making the programmable mRNA.
- 36:57But at the same time, we're partnering with groups such as
- 37:01Upenn or Expert in this targeted delivery.
- 37:04Combining these two together, I think you can achieve amazing
- 37:08things with the next generations of the RNA medicines.
- 37:12Yeah. No, that's a really great
- 37:13insight into the future of RNA medicines there.
- 37:16And to round off our episode then with everything that we we
- 37:19ask every guest, if you could have dinner with any scientist
- 37:23or innovator, past or present, who would that be and why?
- 37:27You know, amongst all your questions, all the questions to
- 37:31think about, this one took most of my time because there's so
- 37:35many people that I want to meet and chat with them.
- 37:38But I finally landed on Leonardo da Vinci.
- 37:44And the reason for that is that to me, he seemed like the a
- 37:50complete man, a true, I mean, he was the Renaissance man, but
- 37:54true in every sense because he was a thinker, he was an artist,
- 38:02he was a scientist and innovator.
- 38:06And I admire that. I, I, I don't call myself an
- 38:11artist, but I've painted for many, many years is one of my
- 38:15joys and loves in my life. And I see how that artistic
- 38:23brain of mine can help impact my scientific mind and vice versa.
- 38:31So that comprehensive and well-rounded individuality that
- 38:41Leonardo da Vinci present, it's something that I admire, that I
- 38:46admire and I, I personally inspire it to be.
- 38:49It's, it's ultimate man, it's ultimate human being.
- 38:53And I would have loved the opportunity to sit down and chat
- 38:57with him and get an insight into how does he bring operates and
- 39:02how does he see the world and just learn from him.
- 39:08I would have loved that. Nice.
- 39:11Well, thank you so much for taking the time today, Naslie,
- 39:15and joining us on the Biohub podcast.
- 39:17Thank you. It was great fun.
- 39:19A lot of good question and I enjoyed it.
- 39:22Thank you. And for all of our listeners,
- 39:25please like and subscribe.