In this episode of Raising Biotech, Surani explores the untapped world of tRNA therapeutics with Flagship Pioneering-founded company Alltrna. The company raised $109 million in a series B in August 2023. Surani talks to CEO Michelle Werner about the inception of Alltrna, the company's tRNA therapeutic thesis and her journey to the role. She also talks about the company's plans to study its technology in rare genetic liver diseases and the potential to accelerate clinical trials with the use of basket trials. Dr David Weinstein, a rare diseases pediatrician and owner of Weinstein Rare Disease and Clinical Development Consulting also joins the podcast to give his take on the potential of tRNA therapeutics to serve thousands of diseases. He gives his thoughts on the company's mission, potential clinical trial challenges ahead and how the therapeutics might fit into the treatment paradigm.
Timestamps:
01:58 - Background of Alltrna and tRNA therapeutics for rare genetic diseases
06:00 - Company formation under Flagship and Michelle's personal backstory
11:35 - Focussing on stop-codon diseases of the liver for first clinical trials
12:55 - Dr Weinstein talks about tRNA therapies for rare genetic diseases
16:55 - The use of basket trials to accelerate clinical trials
18:30 - Efficacy and safety considerations & regulatory pathway
21:45 - How tRNA therapies would fit into the treatment paradigm
24:05 - Potential for tRNA in other genetic diseases beyond the liver
26:00 - Alltrna's future pipeline and business strategy
For any comments, questions, feedback or suggestions you can connect directly with Surani Fernando on LinkedIn or email: raisingbiotech@gmail.com
Music composed by: Yrii Semchyshyn (Coma Media)
[00:00:00] Welcome to the Raising Biotech Podcast, I'm your host, Surani Fernando and thanks for tuning in.
[00:00:09] This podcast has a mission of exploring biotechs raising impressive funds to develop ambitious medical breakthroughs.
[00:00:16] I speak with CEOs and founders to get origin stories, missions and future visions for the company.
[00:00:21] And I also talk with relevant medical and industry experts to get more context on the company's potential
[00:00:27] to really make a difference in healthcare.
[00:00:30] Today I'm talking to Old Turner, a company all about tapping the potential of TRNA
[00:00:36] to unlock precision medicines for hundreds, if not thousands of rare genetic diseases.
[00:00:41] This is another company originating out of multi-billion dollar venture capital firm, flagship pioneering
[00:00:47] and perhaps the future little sister of mRNA-focused powerhouse Moderna.
[00:00:52] The company was unveiled in 2021 with a 50 million dollar series A
[00:00:56] and recently raised $109 million in a series B to help get its novel therapeutics closer to the clinic.
[00:01:03] Right now, the company is still pretty early in its journey but the strong potential for accelerated
[00:01:08] and abbreviated clinical trials could see it with marketed products a lot sooner than we think.
[00:01:15] Joining me on the podcast is CEO Michelle Werner, a veteran big farmer executive
[00:01:20] with years of oncology experience up her sleeve, who came to the rare diseases world for personal reasons.
[00:01:26] We hear about her own journey to Old Turner, the company's scientific thesis and mission,
[00:01:31] and plans to take its therapeutics into first clinical trials.
[00:01:35] Also joining me is Dr. David Weinstein, a rare disease's pediatrician
[00:01:39] who now heads up his own rare diseases and clinical development consulting firm,
[00:01:43] an Old Turner is tapping him for clinical advice.
[00:01:46] He'll give us more context on the current rare diseases unmet need
[00:01:50] and potential for TRNA based therapeutics to shift the treatment paradigm.
[00:01:55] I hope you enjoy the episode.
[00:01:57] Hi Michelle, thanks for coming onto the podcast.
[00:02:00] Hi, well thanks for inviting me, I'm looking forward to it.
[00:02:03] So just to briefly start off, can you explain what Old Turner does,
[00:02:08] what it's developing and the significance of what you guys are doing?
[00:02:14] Sure, so Old Turner is a really novel platform company that was founded in 2021
[00:02:22] through flagship pioneering and we are all focused on really unlocking the exciting biology of TRNA,
[00:02:30] which is transfer RNA into a new modality for patients.
[00:02:35] And transfer RNA is a really exciting space because with this modality,
[00:02:40] we have the potential to use a single medicine to treat perhaps hundreds,
[00:02:44] if not thousands of different diseases because we, I'd say,
[00:02:49] simplify the challenge associated with the 6000 plus genetic diseases
[00:02:56] by identifying patients that have those common mutations across all those different diseases.
[00:03:02] And the TRNA can play a very unique role in addressing those individuals
[00:03:07] with these common mutations in a much more universal way than we have the ability to do so today.
[00:03:13] So let's take a step back here and talk about the role of TRNA,
[00:03:17] a bit of high school biology but when we talk about RNA which is ribonucleic acid,
[00:03:22] it's a molecule that takes instructions from DNA to build proteins that make up our body.
[00:03:28] There are three major types of RNA, we have messenger RNA or mRNA,
[00:03:33] which provides the genetic code template for the protein that's to be made,
[00:03:38] then there's transfer RNA or TRNA, which reads that genetic code and brings amino acids together.
[00:03:45] And finally there's ribosomal RNA or RRNA,
[00:03:48] which plays more of a structural role to build the protein.
[00:03:52] So, Alterna is focusing on that middle step of transfer RNA.
[00:03:57] Not many people have really paid attention to the TRNA in the protein translation process.
[00:04:03] And so, the transfer RNA plays this fundamentally important role because what its role is
[00:04:11] is to read the instructions for the amino acids that are coded in the mRNA
[00:04:18] in the individual triplet codes within each mRNA.
[00:04:22] It reads those instructions, understands which amino acid comes next in the growing polypeptide chain
[00:04:27] and then transfers that corresponding amino acid to that growing polypeptide.
[00:04:32] And that really demonstrates the real importance of the TRNA because without a TRNA
[00:04:38] there just would never be any proteins.
[00:04:40] As brilliant as the human body is with these protein building processes,
[00:04:44] the codes for those amino acids are not always 100% correct and mutations do occur.
[00:04:50] Those mutations are the basis of many different genetic diseases
[00:04:54] and can either mean there's a missing or incorrectly coded protein
[00:04:58] or even a toxic protein.
[00:05:00] And each mutation translates to a different cause of genetic disease.
[00:05:05] What we're tackling first at Alterna is looking at a specific type of mutation
[00:05:10] that's called a premature termination codon or a nonsense mutation.
[00:05:14] And what ends up happening in that particular instance is that a code for amino acid
[00:05:19] in the mRNA strand instead of coding for an amino acid actually codes for a stop instead.
[00:05:25] And when you have a code for a stop, it means the translation process terminates too soon
[00:05:31] and the result is a missing or a truncated protein
[00:05:35] and that is what causes disease in that instance.
[00:05:39] And when your TRNAs are designed to specifically read through these premature termination codons
[00:05:45] and so what ends up happening in that instance is that we can read through these premature termination codons
[00:05:51] and restore that protein translation process and the result is that functional, full-length protein.
[00:05:58] So we'll come back to the technology and clinical trial plans
[00:06:01] but before that I wanted to dive into how this idea came about at Flagship.
[00:06:06] If you listen to episode one, you'll recall we talked to another Flagship company, Empress There Are Pudigs.
[00:06:11] So we got a good sense of Flagship's unique business model
[00:06:15] and I was curious to know how it all started at Alterna
[00:06:18] and how Michelle came into the picture.
[00:06:21] The whole premise behind Alterna actually dates back to 2017
[00:06:26] where our founding scientific team asked the question
[00:06:30] well what if we could alter the protein translation process by leveraging the TRNA?
[00:06:39] And that led to a series of what we call killer experiments
[00:06:44] where we try to throw hurdles in the way of this idea
[00:06:48] and ultimately having generated the initial proof of concept data during those early couple of years,
[00:06:56] it actually led to the formation of Alterna as I mentioned,
[00:06:59] which has launched as part of the Flagship pioneering firm in 2021.
[00:07:05] Now I joined Alterna as CEO in April of 2022 and also a CEO partner at Flagship pioneering.
[00:07:15] So after the founding CEO and the initial founding scientific team,
[00:07:21] I joined as the first external CEO to the company.
[00:07:24] Female CEOs in this sector are pretty rare,
[00:07:27] but on paper Michelle's experience and background makes her no surprise as a choice for CEO.
[00:07:32] She came to Alterna from an illustrious career in Big Pharma.
[00:07:36] I've been at a number of different firms, so BMS and then AstraZeneca and Novartis most recently
[00:07:42] where I've held a number of different roles mostly in, I'd say, late stage development
[00:07:48] but commercialization roles. I'd say predominantly focused in the oncology space,
[00:07:53] but in a few other therapeutic areas as well.
[00:07:56] And I've been really fortunate over this 20 plus year career
[00:08:00] to have the opportunity to launch a number of different medicines,
[00:08:05] many of which have now become mega blockbusters in their own right.
[00:08:09] You know, if we just go back to the beginning of my career,
[00:08:11] which again, as I mentioned has been focused in the oncology space,
[00:08:14] the standard of care across most cancers at that time was chemotherapy
[00:08:19] or chemotherapy combination regimens. That was pretty much it.
[00:08:24] It was before the time of targeted therapies and before the time of immunotherapies
[00:08:30] and antibody drug conjugates, which now have become the mainstay of treatment for patients
[00:08:37] across a number of different tumor types.
[00:08:40] And I like to look back on my 20 year career, really feeling as though
[00:08:46] I've had the opportunity to make a small impact on the advancement of treatment
[00:08:53] for patients battling some really tough diagnoses.
[00:08:56] But you know, I'd say very importantly what drew me to alternate is also something
[00:09:03] that I'd say is quite personal.
[00:09:05] And you know, over the last three and a half years,
[00:09:09] I've been thrown into the rare genetic disease community on a personal level
[00:09:16] when my son who is now 13 but on his 10th birthday was diagnosed with douché
[00:09:23] and muscular dystrophy.
[00:09:25] And at that point in time, I had never worked in rare diseases.
[00:09:28] Of course, I had some familiarity with them, but not douché and specifically
[00:09:33] just to jump in here, Douché's muscular dystrophy or DMD is a debilitating genetic disease
[00:09:39] that ravages the muscles in the body and leads to a loss of movement coordination
[00:09:44] and eventually the inability to walk.
[00:09:46] You know, as somebody who has essentially spent my entire career in drug development
[00:09:52] and advancing novel therapies, I was really disheartened to learn about how little innovation
[00:10:00] there is or was in the rare genetic disease space.
[00:10:05] And it had a very specific impact on our family because the standard of care for him
[00:10:11] and his condition has been the same for 10 years plus and hasn't changed.
[00:10:17] And that felt unacceptable to me.
[00:10:21] And so for me, what really propelled me to alternate was a lot of reflection on how could I take the
[00:10:29] decades worth of experience that I have in drug development
[00:10:34] and being in the life science sector focused in therapeutic areas that I feel passionate about
[00:10:40] but then use that experience and then channel it in a way where hopefully in the next 20 years,
[00:10:47] I can look back on the rare genetic disease setting and feel as though I've had
[00:10:51] the opportunity to make again a small impact if not a significant impact similar
[00:10:57] to the one that I've had in the oncology space.
[00:11:00] So the team at Ultrana had this idea which was good enough for flagship
[00:11:04] to unveil the company with a 50 million dollar series A financing in 2021.
[00:11:09] Then two years later, in August 2023, the company announced a 109 million dollar series B
[00:11:16] with top tier undisclosed investors.
[00:11:19] That capital will help the company to get its therapeutics closer to the clinic
[00:11:24] and since TRNA therapeutics can theoretically be applied to thousands of diseases,
[00:11:29] I asked Michelle if the company had any priority diseases to focus on in its first clinical trials.
[00:11:36] We actually call our first patient population those that have stopped Codon disease
[00:11:43] which is any number of those patients who have a genetic disease
[00:11:47] and that genetic disease stems from a premature stop codon.
[00:11:52] So right now stop codon disease doesn't have an official nomenclature
[00:11:57] because there are no medicines that really have this universal ability to be applied
[00:12:02] across patients across a number of different premature stop codons unlike what we have the potential
[00:12:08] to do at Ultrana.
[00:12:09] So when we think about the patient population, it's in the first instance stop codon disease
[00:12:16] and that by the way, stop codon disease affects over 30 million people around the world.
[00:12:21] So all of the patients that have a genetic disease that stems from a stop codon
[00:12:25] which occurs in about 10% of cases, that brings us to about 30 million or so patients around the world.
[00:12:31] Now of course we have to start somewhere.
[00:12:33] We know that we can't tackle stop codon disease in one bite where we need to get there in a stepwise fashion.
[00:12:40] So we're going to be looking specifically at rare genetic liver diseases
[00:12:45] where we can target those conditions, but being able to address a number of different rare genetic liver diseases
[00:12:52] of which there are 400 that have a stop codon disease.
[00:12:56] To give us more context on Ultrana's very ambitious plans
[00:12:59] I spoke to David Weinstein, a pediatric enter chronologist by training
[00:13:03] who now runs his own consulting firm.
[00:13:06] He spent most of his 30 year academic career devoted to glycogen storage disease
[00:13:11] and 25 of those years were devoted to gene therapy.
[00:13:14] David switched to industry after the pandemic, worked for some gene therapy companies
[00:13:19] including Passage Bio and Grace Science,
[00:13:21] and now he's an independent consultant for rare disease trials.
[00:13:26] My involvement with Ultrana has been in the capacity of serving on the clinical advisory board.
[00:13:32] I do not work for the company, but it's my pleasure to try to help talk about the therapy that they're doing.
[00:13:39] We spoke about the general unmet need for rare genetic diseases,
[00:13:42] the state of drug development in the space and where Ultrana's TRNA approach fits in.
[00:13:48] There are over 6,000 monogenic diseases
[00:13:52] and less than 5% of these conditions have a treatment.
[00:13:56] The technologies such as gene therapy, mRNA and gene editing are now showing so much potential
[00:14:02] and children and adults with previously fatal diseases now have the potential to improve.
[00:14:09] There's one major challenge with all these new technologies.
[00:14:13] Because of the need for an individualized trial for each indication,
[00:14:18] most of these diseases will never have a chance to benefit from other technologies.
[00:14:23] The TRNA approach is unique because it allows a single therapy to be used across multiple diseases.
[00:14:30] I think the approaches being used with the TRNA and determination codons
[00:14:36] is particularly useful because those mutations are common across all diseases,
[00:14:42] and this should allow a single medication to be used to treat hundreds or maybe even thousands of diseases
[00:14:48] without having to do a trial for each disease.
[00:14:51] So that's what's unique about this. This type of approach can't be used for any of the other techniques.
[00:14:57] I asked him what he thought about Ultrana's decision to go into genetic liver diseases as a first priority.
[00:15:03] I completely support the approach for going after the monogenic diseases of the liver first.
[00:15:12] There are over 400 monogenic diseases of the liver and most of these have no trials.
[00:15:18] I think the liver is attractive because we have the most experience with this targeting it with both AAV vectors
[00:15:28] and with lipinata particles, which is what's being used by Ultrana.
[00:15:33] There's increasing experience with these lipinata particles
[00:15:38] and the approach is proving to be quite safe and efficacious at delivering to the liver.
[00:15:45] So I think the liver is an ideal target to go after first.
[00:15:49] I did ask David to clarify why he thought the liver was a good first organ to target
[00:15:54] just because if you listen to episode two with Acrobio,
[00:15:57] there was a pretty strong theme of the liver being one of the most difficult organs to tackle with novel therapeutics.
[00:16:03] Well, it's easy to deliver the organucleotides and the treatment to the liver.
[00:16:08] It's much easier to deliver to the liver than any other organ in the body.
[00:16:13] Whether you're using viral vectors or lipinata particles or like we used to do back 20 years ago, light-person.
[00:16:22] I think the challenge with the liver has been the toxicity with gene therapy.
[00:16:27] I think the challenge with the liver is that the liver regenerates.
[00:16:31] So when you do gene therapy and it's a one-time treatment in the liver's constantly turning over,
[00:16:37] that has posed some of the difficulties.
[00:16:41] But I think for TRNA, where we don't have to worry about the liver turnover or the cells regenerating
[00:16:49] because we know that this is going to be a repeated treatment.
[00:16:53] When I spoke to Michelle about alternate plans for clinical trials
[00:16:57] I asked her about how they might be set up if they were trying to study a number of diseases in parallel.
[00:17:03] And this is where Michelle's oncology background comes in handy.
[00:17:07] One of the development and regulatory strategies that's used fairly commonly in oncology is what's called basket trials.
[00:17:14] And this is a type of clinical trial design where we look at patients across a number of different diseases
[00:17:20] but all have the same exact mutation.
[00:17:22] And we bring them into one individual clinical trial.
[00:17:26] We give them the same therapeutic,
[00:17:28] and then we measure a similar or the same outcome across all of those patients
[00:17:32] despite the fact that they have technically a different diagnosis.
[00:17:36] Now this type of strategy has actually led to the approval of well over half a dozen different medicines in the oncology space,
[00:17:44] including a couple of programs that I've had the opportunity to work on myself.
[00:17:48] And it's the same type of strategy that we're looking at from a development perspective for our engineered TRNAs.
[00:17:56] So looking at patients across a number of different liver diseases,
[00:17:59] but they all have the same premature termination code on looking at endpoints that can be consistent
[00:18:05] to cross those different patients and then giving them the same engineered TRNA formulated it in the same lipid nanoparticle
[00:18:11] in order to address their condition.
[00:18:13] So the TRNA basket trial strategy aims to include patients across many rare genetic diseases
[00:18:19] that would have otherwise been left out in a traditional clinical trial setting.
[00:18:24] But designing and conducting these abbreviated trials is a lot easier said than done
[00:18:30] and it won't come without its unique challenges.
[00:18:33] I think the basket trial approach is ideal,
[00:18:37] and that approach involves eroding people with different diseases in the trial using the same treatment,
[00:18:44] but it's something that is somewhat novel.
[00:18:47] The FDA has accepted a few trials using this approach,
[00:18:53] but it's still relatively new so we just have to continue to educate why this approach is being used.
[00:18:59] And I think one of the challenges with this approach is finding ways to assess efficacy across different indications
[00:19:07] when the diseases may be somewhat different.
[00:19:10] So while efficacy will be its own challenge, safety is what's paramount in the beginning.
[00:19:15] And I asked Michelle if there was any indication or hypothesis on how safety RNAs can be at this stage,
[00:19:22] perhaps also comparing them with mRNA therapeutics.
[00:19:26] So far we're encouraged by what we've seen in the INVIVO setting,
[00:19:31] but of course we'll be paying close attention to that.
[00:19:33] What's really good about the strategy that we have in place with formulating
[00:19:38] in a lipid nanoparticle is the fact that similar to mRNAs,
[00:19:42] lipid nanoparticles now have been in millions and millions of patients thanks to the COVID vaccines.
[00:19:49] So we have a very good idea about what the profile is associated with the lipid nanoparticles,
[00:19:57] but of course we need to be diligent in making sure that we understand the safety profile associated with the TRNAs,
[00:20:04] and that's not something that's been done before.
[00:20:07] We will be doing, of course, all of the preclinical and toxicology work that's really important to help ensure that we have a good idea about that before heading to the clinic.
[00:20:17] With alternatives intended abbreviated approach to clinical trials,
[00:20:21] the hope for the companies that typical trial timelines like the 10 to 15 years that are standard for traditional drug development might be dramatically shortened.
[00:20:29] But that will be up to regulators like the FDA and the M.A., and the discussions they have with the company.
[00:20:35] So I was curious to know if Oltena had any plans to apply for official breakthrough status that would lend to accelerating regulatory timelines.
[00:20:43] We haven't disclosed anything like that, but certainly I would believe,
[00:20:48] you know, if you think about the patient populations that we're talking about here,
[00:20:52] especially where the standard of care is really lacking,
[00:20:58] we do believe that there is going to be support from the health authorities in order to try to accelerate these types of evaluation of TRNAs moving forward.
[00:21:10] I think that the health authorities both in the US and elsewhere,
[00:21:14] that are focused on rare diseases are already having their own narrative around needing to tackle the challenge of rare diseases using a many diseases at a time strategy.
[00:21:24] And we think that our technology complements what we're hearing from the health authorities and that there will be a willingness to partner with us and define those paths forward in order to be able to bring these clinical trials and these investigations to patients.
[00:21:40] So we're looking forward to advancing that.
[00:21:43] As we know, we're a little far off from these being readily available on the market for patients,
[00:21:48] but the company has to be thinking about practicalities and implementation considerations in a clinical setting from the beginning.
[00:21:56] David spoke a little bit about how he thought this type of therapeutic would fit into the treatment paradigm.
[00:22:02] The biggest challenge with TRNA from an implementation standpoint is that it's going to require almost certainly repeated doses.
[00:22:11] We know that whether it's TRNA or NMRNA, the treatment still lasts very long.
[00:22:18] And so, we chronic dosing or repeated dosing is almost certainly going to be required.
[00:22:25] We just at this point don't know how long it's going to last because it hasn't been done yet.
[00:22:30] This is going in a different direction to advanced gene therapies which are intended to be a one and done dose,
[00:22:36] which is attractive to patients, but it's also very expensive.
[00:22:40] But David said that one potential consolation with TRNA repeated dosing is that it gives the opportunity to adjust the dose if that initial dose isn't correct,
[00:22:49] which you can't do with gene therapy.
[00:22:51] The other potential advantage is a lower risk of possible long term side effects that may come with gene editing or gene therapy.
[00:22:59] So there's some advantages and disadvantages of that.
[00:23:03] When you're dealing with a treatment that's going to require chronic dosing, we have to find a way to deliver it to all different sites.
[00:23:11] Gene therapy can be done at just a few sites in the world, but for this to work, it's going to have to be something that can be distributed worldwide because of the need for repeated dosing.
[00:23:22] To me the biggest challenge we have is that in the United States, we don't genotype diseases as frequently as they do in other countries.
[00:23:32] The US is particularly lagging because insurance companies often won't cover genome typing if the patient already has a clinical diagnosis via other means.
[00:23:41] For example, a liver biopsy for glycogen storage disease and without routine genome typing it will be hard to match patients with potentially effective TRNA based therapies.
[00:23:51] In my mind, we have to change the mentality in the United States so that everybody gets genotype because that's going to be critical for the alternative TRNA approach.
[00:24:02] So once Alterna has established itself as a viable therapeutic, I was wondering what other common diseases fall into its treatment bag.
[00:24:10] I knew that stop codons in cystic fibrosis and DMD have previously been explored by the industry, so I asked David where he felt the therapy could have the most potential.
[00:24:20] The diseases you mentioned, cystic fibrosis and Tushandamus who just be our priority, these are just because they're so common.
[00:24:28] I think there are challenges with those, you know, getting it to the organs and that's why I think starting with diseases like glycogen storage diseases or PKU or other metabolic diseases of the liver are much easier even though they may not be quite as common.
[00:24:45] But there are millions of people being affected by metabolic liver diseases so I think that their ideal even though they may not be on the radar of every other company.
[00:24:55] I would love for this technique to one day be used for the genetic diseases of the brain.
[00:25:01] There's a whole slew of lice, especially lice is almost storage diseases, I think those are devastating diseases but that's much more challenging because we have to get any treatment across the blood brain barrier.
[00:25:12] So something that we're going to have to see how they can deliver the TRNA all the nucleotides to the brain but let's start with the liver first.
[00:25:23] I mean, honestly, I think you can almost name the genetic disease and stop codons exist in all of those. So yes, cystic fibrosis and Tushandmoscular dystrophy are two.
[00:25:34] There's also sickle cell disease, there's PKU, there's the hemophilia, there's retsyndrome, I mean there are thousands of these different diseases.
[00:25:45] And because in 10% of those genetic diseases start with a premature termination codon, we actually see the same mutations across all of those different diseases.
[00:25:57] As mentioned earlier, Ulterna raised $109 million in August 2023 from a group of top tier investors. Michelle said they were a very diverse group of investors but I asked her why Ulterna hadn't disclosed them.
[00:26:11] In terms of disclosing the investors, we have to go through a process of getting the requisite approvals by everybody in order to be able to make those disclosures but given the timing in which we announced the series B, this seemed to be the you know, the best way to move forward at that at that moment.
[00:26:29] So Ulterna may reveal its investors at a later date but overall the company seems to be keeping pretty tight, lipped at the moment including where the 109 million will exactly take them and when they hope to enter the clinic.
[00:26:40] But I asked Michelle where she sold the general direction for the company's pipeline and business strategy.
[00:26:47] You know today we're talking about the application of our engineered tyrannies and premature termination codon mutations but we do think that there's broad applicability across different types of mutations and perhaps frame shift and missense mutations are also areas that we could explore in the future.
[00:27:04] We also think that there's a role for tyranny pools and tyranny fragments to even to address an even greater or broader number of patient populations in the future.
[00:27:16] You know but that being said, I also think we want to make sure that we're not getting ahead of ourselves and because we're doing a looking at a new modality for the very first time it's also important to have focus and that's why we are very much focused on demonstrating the potential of our platform from a clinical level.
[00:27:34] We're also looking at a whole perspective with our engineered tyrannies and premature termination codons as are starting place.
[00:27:40] Michelle said the broader long term vision is to build a pipeline with a number of different assets but also to develop and enhance the platform's capabilities so that it has the opportunities to expand and unlock the entirety of tyranny biology.
[00:27:53] You know when it comes to the nancings and IPOs right of course these are things that we will be taking step by step and you know in deep conversation with our investors but really primarily we're very much focused on continuing to invest in the platform and continuing to invest in our ability to demonstrate potential over a platform with an initial clinical proof of concept.
[00:28:19] Ten years down the line Michelle sees antenna doing some pretty big things.
[00:28:24] Yeah so I hope that in 10 years time that we have a number of these engineered tyrannies that have now become available to patients to address a number of different mutations and you know hopefully we have the challenge of stop codon disease resolved or at least have a completely new modality
[00:28:46] in order to be able to treat patients with that condition which hasn't been done before.
[00:28:51] I also hope that in 10 years time in addition to stop codon disease we're also starting to see the potential of how tyranny biology can be used as a new modality for other types of mutations as well.
[00:29:04] I think that's also insight too and I'm really excited about that possibility.
[00:29:09] Well that's it for another episode of Raising Biotech.
[00:29:13] While this is very sophisticated science and Ultrana has a steep road ahead navigating its clinical path, if successful this will be a massive leap for the industry
[00:29:22] and the often forgotten rare disease community so we'll be watching Ultrana's next steps pretty closely.
[00:29:30] Thanks to my guests Michelle Werner and Dr David Weinstein for giving up their time and valuable insight and thanks to you for listening in.
[00:29:38] That's the last episode for season one so thanks to everyone for their support and feedback so far.
[00:29:43] If you want to support this podcast for future episodes please subscribe if you haven't already, share it around, join the LinkedIn page for updates and give the podcast a rating or review on Apple or Spotify.
[00:29:55] I'll be back for another season in 2024 but until then I'm Serrani Fernando and stay tuned for the next installment of Raising Biotech.