Science, Startups and Drugs: Translating Groundbreaking Research into Transformative Therapeutics
Hello and welcome. Thank you so much for joining us this evening or afternoon or morning, wherever you are. We are delighted to have more than 60 of you already on the call, and I understand there's about 178 folks who have called in. So, I'd really like to welcome all of you, our alumni, parents, Brandeis National Committee members and friends from around the world. My name is Vipin Suri. I'm an alumnus of the Graduate School of Arts and Sciences, Class
of '01. And I represent the graduate school on the Brandeis Alumni Association Board of Directors. I'm a scientist by training, and I'm the chief scientific officer at Catamaran Bio, which is a one year old startup in Cambridge that is developing new drugs for cancer. We have a fantastic panel plan for today. And what we will be talking about is how groundbreaking basic research discoveries lead to startup biotechs that discover and develop effective drugs. And we're seeing how important this is right now, like the last 13 or 14 months have been a very difficult time for much of the world due to COVID-19, but there is light at the end of the tunnel. And a major reason for optimism is the vaccines that are proven
to be highly effective in preventing serious disease. Vaccinated grandparents can hug their grandkids without the fear of getting sick, and we are hopefully on the road to recovery. What's enabled this speedy development of vaccines was new technology that was developed over the past two decades... over the past decade or so, actually, by two companies, Moderna, which is based in Cambridge, and by BioNTech, which is based in Germany.
And what enabled will this startups that started about 2008, 2010, was basic research done in part by professor Drew Weissman at the University of Pennsylvania, Brandeis alum, Class of '81, and Katalin Kariko, currently at BioNTech. And they were basically asking why cells react in a certain way to messenger RNA. And those basic research questions ended up in the discovery and development of these vaccines that are now saving lives and helping us get back to work. And so, there are two key points I want to make here with this example.
One is that going from basic research to these dramatic outcomes requires a lot of folks and not just scientists, a lot of different folks to come together and work together. And secondly, by accelerating these interactions and getting more of these folks to talk to one another, we can really increase the pace of these innovations and developments. To learn about how these interactions spark innovation and how to accelerate the innovations, we have to offer all superstars from the Brandeis family that have effectively translated basic research into startups that are developing new drugs. On the basic research side, we
have professor Dorothee Kern, professor of biochemistry and an investigator of the Howard Hughes Medical Institute. Dorothy has been at Brandeis for about 22 years and has led a very successful research program, looking at how large molecules called proteins move and sway. In addition to being a standard researcher, Dorothee is also quite an accomplished basketball player.
She was on the German national team, and you can still find her frequently at the Brandeis gym playing basketball. On the startup side, we have Kevin Gillis from the class of '89. Kevin is a partner and chief operating officer at Third Rock Ventures, where he leads operations, finance, legal, and a number of other functions. Kevin and Third Rock are a force behind many biotech startups in the area. They've been around for about 15 years and have developed a number of drugs for cancer from neuroscience, metabolic and other diseases. Kevin is also a sports aficionado, and he played on men's soccer team during his time at Brandeis. In addition to his Brandeis bachelor's degree in history, Kevin also has an MBA from Bentley University. And together, Dorothee and Kevin have launched two startups, Relay Therapeutics
and MOMA Therapeutics. With that, let's dive in. How are you, Dorothee? Excellent. Just waiting for your first questions. We'd love to hear about your journey from growing up in Germany to becoming a professor of biochemistry at Brandeis. So, I thought I'd give you a little story because all of these things, what I'm doing now was totally non-planned. It started really following my passion and that's what I would give the advice to all the young scientists from a love of basketball, via dancing proteins. So over here on the left, I'm playing basketball for East Germany because I grew up behind the Iron Curtain and practiced macroscopic protein dynamics. I love dancing. And so,
what I realized that not only macroscopic, the definition of life is actually motion, but that's also the secrets of the functional proteins. So I show you the protein here is three-dimensional structure. So microscopic, in vitro, but you might know proteins are our muscles or an egg, or what we just heard actually the happy defense proteins our body is making if you get the vaccine right now. So it's rigorous that we are making this foreign proteins, which defense the virus. But of course, back in East Germany, we couldn't get those proteins made, so we had no loopholes, no equipment. So we went to the brewery over here and got out of the brewery yeast purified
our proteins. And then we measured them by hand, right, fast forward, and how did I get to Brandeis? The world came down, East Germany became the United Germany. I played for the United national team. And I had this victory to come to America to play basketball and study science. So here we fast forward all the way to Brandeis. The big tank is not a
brewery tank anymore, but it's a big magnet. It's actually the lensman facility. It costs a little bit more money, as well, three million dollars and thanks to God to many, he actually build as a building. And so, what we can do in this big magnet now can actually look at proteins at atomic resolution, right? I leave you with that, macroscopically, we have to look at the dynamics.
[Playing Lady Gaga's song "Just Dance"] Love it. Thank you, thank you. I just leave you that without much words, I practice macroscopic dynamics in the gym, pick up basketball at Brandeis, and then my team of students and postdocs helps me to make real time movies at atomic resolution. The only other thing I want to tell you, what is most important for life is ATP. You're consuming ATP here in this enzyme. What I'm studying in the lab is actually making ATP. So it's one of the most essential protein. So for that, I give it over to Kevin.
Thank you, Dorothee. Hey, Kevin. How are you doing? I'm doing well, Vipin. How are you doing tonight? Great. So Kevin, what is a partner at a venture capital firm do and how did you become one? Yeah. So let me pull up something as a backgrounder here. Yeah. So a little bit about Third Rock and how I got there, Vipin. So it wasn't really a destination that I had in my mind as I was navigating my business career. I had actually started in high tech, went to Atlanta, Georgia,
worked in the Coca-Cola company's headquarters for numerous years. And when I made the decision to move back to the Boston area, I was really intrigued by what biotech was doing. This is in the late 90s. And had an opportunity to join a company called Millennium Pharmaceuticals. That was a high flyer, and had a good career there, went to another company that went public and three of the senior leaders at this company, Millennium got Third Rock Ventures up and running in '07. And they started to recruit some of their colleagues from Millennium. And it was a unique moment in time. And I was really intrigued by their model. The basic premise behind Third Rock was it
was at a moment in time where the ecosystem of biotech and drug discovery was really changing. Some of the amazing academic breakthroughs were taking places at universities like Brandeis, and at the same time, the pharma industry was really struggling internally. And so, what Third Rock was trying to do was take some of those incredible discoveries and translate them into the next generation of new companies with an idea that the pharmaceutical industry would be there as a recipient. And so, that was the idea. And over the years we've had, as you can see with some of these stats, tremendous amount of success, working with folks like Dorothee, we now have 55 companies in the funded portfolio. We're operating out of five funds with nearly three billion in assets, under management. On the capital markets in
the United States have been very receptive to the type of companies that we've been building. You see here on the slide, 25 of our companies have become public entities. And that's based on all the idea behind our ability to get the companies quickly in a capital efficient manner into the clinic and start to really move the needle in terms of drug approvals. So I spent most of my activities on all of the operating activities that you mentioned on the initial part of the call today, Vipin. Outstanding. Thank you. Dorothee, going back to something you mentioned earlier, which
is the moving protein and the ATP metabolizing enzyme that you've had, what are some of the research questions that is trying to address? Yeah. So I gave you one example and I told you already from the movies that we want to understand is the absolute principles how proteins work in your body, right? The proteins control almost everything. So you see on the left side, singling, right? So that you can see me and transmit that signal membrane transport. So this one, I show you here that drugs are
experts out of the body. That's actually a bad thing, right? Catalysis, anything that can digest your food, all the way that you make ATP is dependent on biocatalysts. Anything in your brain actually is controlled by proteins like the long-term memory. In your vision that you can see, right? So these are unhappy natural functions. And then what we actually wanted to say is we need to understand those proteins in those functions.
So we have to go beyond those static structures and look at these movies, right? So that is how those proteins function. And so, what we really did is edit the fourth dimension to start the real protein side. So now you see them making those happy proteins doing hopefully good functions in your body, but what we want to do in the second part, and that's really sort of the panel discussion to use that natural building property of proteins that are happily dancing to rationally designed, very good medication. And so, this is actually
what we do starting from what nature built to now take advantage of that to make much better medication with no side effects in curing diseases. To understand the natural working functional your proteins, actually understand how they evolved. So what I show you here, what we actually do in our lab, we create the evolution from speed 3.5 billion years ago, where the first creatures, the first living creatures arose on earth all the way to today.
And one of the fundamental things in biology is actually where the energy comes from and the energy comes actually from thermal energy from temperature. Now, when we think about... when Earth rose, we actually had a volcano on Earth. It was so hot that we had a lot of energy. Now we have very little energy because the earth's cooled down. The second part, these, all the organisms were very primitive. And I hope that we are a little bit more sophisticated today, right? And so, we're recreating that evolution starting from old ancestors and building them and our test you all the way to you today. So these are the few questions
we actually answering in other lab. Great. Dancing proteins. I love that. Where others see statues, you see jello, right? Kevin, a question for you. You mentioned Third Rock has been involved in companies that have discovered a lot of drugs. Can you explain a little bit more where venture capital firms set in the discovery of new drugs? How do biotech start, and the role that you play and venture capital firms play in the formation of new biotechs? Yeah, absolutely. So we've put this process together over the years that we call Discover,
Launch, Build and Transform. And it really reflects a strategic shift that Third Rock made over the years to focus our efforts on a hundred percent company creation. So maybe unlike another investment firm or another VC that may be looking at existing companies, we felt that we had a unique ability to partner with professors like Dorothee, on the early science. And I'll just show you the next slide briefly here. It's this concept we call Group
Genius, Vipin, where we're constantly scouring the world. And in trying to find those next generation breakthroughs, really where is the science going? And we're really looking for the boulder initiatives. We're really not looking for a slight improvement on an existing technology. Our belief is to really go big and we are cautious in terms of our process as you get a sense here on the slide. We have about eight or nine of
our team members working on these various projects from a management standpoint. And then we set them up into separate projects, and over a couple year period working closely with folks like Dorothee and other founders, we map out in essence, the strategy for the initial investment. And so, the initial investment in the Third Rock portfolio in today's fund are averaging anywhere from 60 to 80 million. We think that should take the company somewhere between two and three years forward. And ideally, the product engine or initial science is starting to develop the first couple of development candidates that we can start thinking about from a clinical development standpoint. And then the company at that time, will go more now into what we call the build stage. So looking to raise another round of capital,
what we call the series B stage, probably in the 60 to a hundred million is often what you're seeing there in build. And then one last financial transaction is called the crossover round. It's an essence of the last private round before the company thinks about going public. And so, Dorothee, in Third Rock, we showed one of the brands, the name of the company is called Relay, really had just an incredible run through those private financings. And as just a beautiful technology that has a lot of investors, super excited about it, we were able to take that company public just this past summer. And the company is sitting there today with a market capitalization
North of three billion, which is just really remarkable, but it speaks to the vision that Dorothee and the other founders had at the beginning. And I think Third Rock had a unique ability to take that technology and really prosecuted from a clinical development standpoint. That's very helpful. And it reflects the enormous work that goes into these companies. So two and a half to five years just to get to the build phase. And that's when you start your actual drug discovery programs. So that's pretty amazing. Congratulations on the success of Relay. And now that you mentioned Relay and your other company MOMA as well, let me
ask Dorothee a question about how these companies came together. So Dorothee, was there a specific experimental result or an aha moment when somebody walked in with a picture of a gel or something, and you said, "Wow, we got to make a drug out of this." So the best moments are these aha moments, right? But for sure not the gel. I just told you that you're making atomic resolution movies, right? So the aha moment was first, I'll show you that. The aha moment was that we cannot rational design blocks, which is very frustrating. And the reason is, is that rigid-body docking problem I just showed you, right? Which all the biotech companies, all the farmer had been doing, and what is the problem? If you look where the drug is, it's actually an HIV drug, it's inside of the protein. If you look basically you can't even see it. So in other words, you know if something goes inside,
it can only go inside if the protein moves. Okay. So clearly, my passion, my dancing proteins should be the answer, right? That we have to understand the dynamic nature, how they can actually move in space and time. And we had the new vision, put protein dynamics of the heart of product design, but that's not enough to convince anybody to give you $65 million. You have to have a proof of concept. And the proof of concept was really that we looked at one of the best drugs on the markets in cancer, which is called the wonder drug Gleevec. And what we see for Gleevec actually is that it binds to the drug targets, right? But it also binds... From the structure, it
also looks to things that shouldn't inhibit, which are they happy proteins, and it doesn't. So the reason why it's really inhibits very well, the targets, the bad protein is because of the dynamic nature, and we characterized that in very much detailed and found completely new principles, how to make very selective individuals nominal people in 2015, but then we went one step further. And the big step further is a secret I tell you, right? Is how can we even make better drugs where you really cure diseases, and you're not having any side effects, so you're not losing your hair and so on. It's going to this called allosteric sites. You'll learn the new words. allosteric means away from the active site. So here's where all the other
drug companies go in, but the question is, why are we more sophisticated than a bacteria cell? It's because we actually have deregulation, whereas these other sites. So in other words, these other sites evolved that we're more sophisticated, but that's why we should target them there. And so, we had a very second big break, so to show that if you make drugs against those regulatory sites, that they are way better. So these were the two aha effects, which actually got... We teamed up with Kevin and started
this amazing company. It an amazing team at Relay Therapeutics. Right? And you will see the website and what are the secrets, Relay was awarded #1 workers place in the Massachusetts area. So you have to get the dream team together. You want to have a great environment. And the other thing which popped up you fuse teams, so you have to have one of the most innovative methods and approaches to actually be successful. Many thanks to the Relay team, that they made my dream become reality.
Fantastic. Well, thank you for that new addition to the vocabulary. So just to bring it together, my new dream and passion is you need to between teams, here's my dream team at Brandeis, my own lab, right? Here's my Relay team, here's my MOMA team, so that the success to go to health avenue and happiness avenue is to have amazing teams. The comments in between academics and tech is really there was moving proteins. But most importantly, I think to me is my dream team, my family, is my two daughters, Nadia and Julia, my husband. In fact, I published with both my phone and my husband and future son in-law. So they are kind of a stable point for the happiness. Thank you. That's just great. And I couldn't agree more. I mean, teamwork is the key to success. I mean, drug discovery above all is a team sport. There's just so much effort that goes into
it. Now, Kevin, what happened at the first meeting? So clearly, Dorothee has got fantastic research going. She is full of energy and she shows you these dancing proteins and the allosteric sites. And, how did you decide this is what you wanted to invest in? Yeah. So, it's a great question, but then it is... Part of this process where are more scientific oriented members of our team. So similarly, scientifically trained PhDs and
MDs are out meeting with folks like Dorothee on a consistent basis. We do a lot of vetting internally. We're also checking with some of our industry peers to really get a sense of what the opportunity could be, and then working closely with the founders and our extended team. We're trying to get a sense of where we could to take the technology and on what type of dollars, because it is at the end of the day, a business. And we have to be mindful that if we're putting large sums of capital in, we need to be able to move from a point in time today to a point in time in three years where we can actually now attract another investor to come in.
And there've been many technologies over the years that we continue to be excited about, but they're probably still a little too early. And we actually think in those instances, it makes more sense for them to stay in the academic lab and stay in contact, and stay in touch. And with the idea that that could be a company for us down the road. And I think it's that approach that is, I think, led to the success that we've had in essence, very few, results turn out to be zeros where the company just hasn't had success. And I think
it's because we're asking a lot of these very critical questions while the dollars are small in the intention we can give to it as a very important one here. Thank you, Kevin. So a question for both of you, and maybe we can start with Dorothee first and then you Kevin, which is that the theme of sports runs in a fairly significant way to both your careers. Dorothee played on the German national team, you played soccer at Brandeis. Did your share passion of sports help in the formation of these companies that helped bring you guys together? Okay.
There you are. There you go. Sorry. It just crashed for a second here. Okay. I'm back. Yeah. So, Dorothee, I was just mentioning your love of sports. Yeah. So I think it's very simple. I think there are so many parallels between sports and running an academic lab, doing research discovery or studying companies. It's really teamwork, I mentioned that before. Right? And I just feel like I'm the point guard.
I was always the point guard. I'm the point guard now at Brandeis, right? Or with the companies. What the key really is that you make everybody around you a battle. You have to find a secret how to... Everybody's different, everybody's individual. How do you get the best out of every person? And it's kind of simple. You make them happy, you make them
really be proud of what they're doing and being part of it. And everybody contributes with their own personality. Right? Definitely, other credentials is you have to have a lot of passion, you have to have lots of energy. Speed is important. Right? I mean, Kevin can speak to it. I mean, it
is competitive, right? We beat that virus with a vaccine in 10 months. Right? So speed is everything. Right? And then I would really say that you want to shoot big, right? So analogy is the basketball again, right? And Kevin mentioned that again, not doing something incremental, but what I'm really dreaming of, to win the championship. Right? Which
I did on the right. So these are my teammates, my German teammates, we are defending world champions. But many of the championship means not just prolonging the life of a cancer patient for a few months, but actually curing them, that they are living really unhealthy life. So these are the few credentials, I would say a lot of parallels, which I learned early on from sports. Right? And finally, you can't be a perfectionist. You have to keep trying, working hard and it's a long road to the journey. Right?
Thank you, Dorothee, that's wise words for startups and wise words for life. And is that you playing basketball in heels? Yeah, these are my guys. Every day I play pickup basketball at lunchtime, unfortunately not right now, but they prepare me always for the world championships. So these are my best friends. Kevin.
Yeah. Let me pull something up here to share. Okay. So similar, was a soccer player, growing up and being a part of a team was really in my DNA that been, and I think as I went into my business career, I naturally gravitated to settings where I could be part of product development teams in high-tech initially, down at the Coca-Cola company, worked on their bottled water project. And when I got to Millennium, I had the opportunity to be part of the Velcade development, a drug that they got approved for multiple myeloma that became a huge success and the impact it had on the patients. And they just solidified for me that biotech is where I want to be. And from day one at Third Rock, we have instilled that type of sentiment into our culture, because drug development is a very difficult business. The odds of failure are incredibly high and the resiliency, the teamwork, how you communicate, how you lean on each other in the early stages of a company being formed, and then how you navigate that as teammates is really critical. And we talk a lot about at Third Rock about
the science is super important, the capital is super important, but often the company's success is often determined by the first 20 to 30 people that get hired into these companies and how they set the culture and the tone and how they think about Dorothee's point going forward. Like we want to be the best. They want to develop that. And that mentality that I will do anything to make the company successful, this technology successful because the person that's waiting for the success is ultimately the patient. And if that doesn't motivate you, as a member of our industry, then you're in the wrong industry and you should go out and sell insurance or something like that. And you shouldn't be in biotech or drug development.
Great. So it's the passion and keeping the eye on the ball. Exactly. That's the recipe for success. Excellent. Well, thank you for the background questions
and answers. Now for the fun part, where we open this up to the audience to ask any questions for any three of us, frankly. And please put your questions in the chat box or the Q&A box. I see that some questions are ready. So let me pull those up. Okay. This one is
for you Dorothee. And it's for Sam Brower. I'm assuming one of the advantages of targeting an allosteric site rather than an active site is that undruggable targets can now be accessed. Has anyone been looking at potential allosteric sites for COVID-19 proteases? Excellent question. Of course, one of... Besides that the allosteric sites give you the selectivity,
we have now made a hundred percent selective inhibitors. The other thing that you said, we can go to... Which are doomed undruggable in the industry because the normal approach of going in the active side has failed. So one example are phosphatases, which are as important as... In cancer treatment. And the reason is because the actives of undruggable
were charged. So for the proteases, I mean now for COVID of course everybody's screening, and trying to find drugs against these different proteins. Of course, well, have... the important thing that we're actually having immunization, I think that's a way better thing than trying to treat after the fact. For treatment, I think it's a very good question you ask. I think it's really the principle
understanding, which will help us for future. So the way drug development really works that, will probably be delayed for this COVID thing, but what we're learning will help us for future outbreaks. And I'm not aware of... I mean, there's not a single new developed drug for any of the proteins for COVID right now, but it's a very interesting question you raised there and definitely these are the sites you want to go after because they are very selective. Thank you, Dorothee. Great question, Sam. A question from Jose Perez, how are you Jose? On average, approximately how much does it cost to take a drug from Michelin investment to the patient. Kevin, this one's for you, I think. Yeah. I'm happy to share some of the stats. I remember a study that Forbes did probably
six years ago. So somewhere in that 2015 timeframe where they had looked at the last 14 to 15 years for the large pharma industry and they divided their R&D spend per year by drugs approved. And it was often in the four to sometimes eight billion per approved drug, which is really unsustainable longer-term I think, for our industry. And so, in our own experience, the first company in our portfolio that was able to develop a drug, a company called Agios, which is in the cancer metabolism space. They ended up with a $1.1 billion cumulative spend to their first approval. And that I think is indicative of what we had hoped that biotech may be moving a little bit more nimbly, maybe a little bit more focused on what they're trying to do would lead to some efficiency and span versus some of the larger pharma companies. There are a lot of
layers of management, maybe not as risk tolerant. And I think that bodes well for the industry, if we can start to drive the cost into the low, it's still a lot of money. I mean, a billion dollars is a billion dollars, but spending six to eight per approved drug, is a tough model for the large pharma companies to maintain. Thank you, Kevin. Thank you, Jose, for the question.
Maybe just to add to that, right. I mean, Kevin knows that even better, but maybe just to add to that, the research to discovery is still the smaller fraction of the investment. It's really the clinical trials. I mean, not being really in that field, I mean, I'm learning
but I really think also changes need to be made in the FDA, to make actually those clinical trials more efficient. And so, it's really multidimensional all the pieces that have to come together to finally have a safe, and very good drug on the market. Yeah. That's an excellent point. It's a very important current discussion given that FDA has gone above and beyond with the COVID vaccines to improve them as fast as they could and review them. And what can we learn that we can apply to other drugs to make sure they're available to the patients faster? So that is a very good point, Dorothee. Thank you for that. A question from David Stern for Kevin. As a VC, do you feel that there is
a higher rate of failure when investing in the discovery phase and having your own discovery versus investing in a more proven company in a series A round? Another excellent question. Yeah. I think in our experience, we have used our discovery process in a way that we think is a good use of capital. And it allows us to ask some of the really difficult questions while the cash burn is very low. And so, often before you see a Third Rock company come out with a series A presentation, and we just raised 60 million, we may have already spent five or six million over a couple of years working with Dorothee and other founders, and really asking the tough questions to give that company the best chance of success. And then in addition to that, from a risk management standpoint, we traunch all of our capital into our companies. And by that, I mean the 60 million doesn't go all in on day
one, 20 million may go in and we work with the founders, the management team of these companies to say, these are the things we expect to get executed on over the next 18, 24 months. And a lot of that are progress that we want to see in the scientific setting, business development, hiring the management team. And there have been instances where we're 30 million into a $45 million A round, and we've had to shut the company down because the science hasn't progressed. We're hopeful that those are few and far between because
as COO, zeros in my portfolio, aren't going to lead to a good outcome. So that's just a little bit about how Third Rock is going about it. The last part of the question, for sure. I think to look at opportunities and we periodically will, if there's an existing
company with a novel technology that one could argue is maybe been a little bit de-risk, we have an appetite to do some of those investments as well. Yeah. Maybe just to add to that, because it's perfect for this round table discussion. I really think the interaction between academic research and early biotech, and even that scouting phase, what Kevin just explained, this is really the model for success. And
it's a win-win situation, right? Academics wants to work on things which are highly relevant for the public health. Funding by agencies and the NIH, the NSF and governmental funding is not sufficient to run our labs anymore. On the other side for early biotech is actually way cheaper and more rigorous to work with academics. So I think this new partner model is really the future of science. And I would finally say you saw it right now, again, coming back to COVID, what really can happen and how fast the academics, but also different industries, different companies finally get together and say, "We you have one goal and one called only to help people." And so, I'm really hoping out of this pandemics that we never go back to have closed doors and do something in competition, but rather together to cure diseases.
Great point. And the point you make about companies coming together with COVID is really key because the Pfizer vaccine was developed by BioNTech- In Germany. By Pfizer and similarly J&J and Moderna are working together. And that's again, to make
sure that people are healthy and get past these current unusual situation. So that was very good question from David. And Dorothee, you started touching on this, the question that comes to us from Dan, how do academic institutions profit from these activities? Yeah. So, I mean, I think there's many avenues, I just touched on it, right? That university is... the main focus of a university of course, is to train the next generation. And I think
that's what is really amazing what we do at Brandeis, right? So Brandeis gives us opportunities. We have cutting edge technology, right? We have great facilities and a full support of research so that we can really create these amazing young scientists. We then go out in the world and do those discoveries and help. Right? But what is Brandeis getting back? I think what Brandeis is getting back is of course, what I just said, these collaboration, these early seed funding, where we can actually explore with exploratory research. Of course,
the next thing is we're recruiting the best scientists, we're recruiting the best undergrads, because they want to be part of it. I want to remind you that, we have an undergrad from Brandeis who won the Nobel prize. We have the scientist who was a grad student in our department, actually in biochemistry, who was the key player at Moderna to do the key foundation for that, we can do this vaccine. So these stories, enable Brandeis to really be absolutely cutting edge in science. And
therefore, an internationally valuable, recognized organization, which we need. I mean, we're smaller, we're smaller than Harvard and our big brothers down the road. But I would say Brandeis is a jewel, right? It's excellence a little bit comparable to biotech, right? Not big pharma. We are excellent and notable. It's about quality and not quantity and efficiency. But it's a really good question because yeah, the question, is what is Brandeis profiting from those interactions? Excellent. Yeah, Brandeis may be small, but it packs a punch for several star researchers and also stellar programs for students. Thank you for the question. The next question is from Randy Hampton. With early stage therapeutics, what first round of funding options are available?
Are VC funds like yours and the only, and or the best option and why? A assume this is for you, Kevin. Yeah. Another excellent question. And obviously being in the seat for 13 years, I think a lot about what we do and maybe why we are a value add. A lot of the... why I feel that way, it does come back to the original thesis of Third Rock. There was a belief that our three founders share that the venture model had lost its way. That venture was really
a little bit risk averse and really not leaning in and often backing in investment, but challenging an entrepreneur who maybe had no real experience building a biotech company to go and try and navigate what is often a very difficult path. And so, for us that I think the pitch that we make to folks like Dorothee and others is you've got within the partnership folks that have often spent 20 to 30 years building some of the most well-known biotech companies and that ability to work closely with the founding science team to translate those ideas into companies and then navigate those companies, we think is the right way to do it. And we were pioneering that a bit in the early of Third Rock, but if you look at the landscape today, people have been paying attention. And so, folks in town like Flagship, like Atlas on the West coast, like Arch, like Versus, like 5AM. The space that we were competing in in the early days has gotten incredibly more crowded, but it's back on that fundamental belief that this marriage of venture in academia is the right way to go about building the next generation of companies and ultimately drugs. Thank you. Question from Oliver, can you speak about some of the ethical issues when academics
work with companies? Dorothee, you want to take this one? Yeah. I mean, ethics is a broad word, right? It's I'm not quite sure Oliver where you're ongoing, so maybe you can type in which direction you go. So let me start with, of course academic research has to be available to everybody, right? And I mean, the whole... That is also the big advantage. So the one thing which is hard for me now as founder, advice of the companies that we can talk about the exciting new research. And so, in academics, of course, we talk about all the results we brainstorm about it, which we can with companies like Relay, MOMA, can only do internally, right? So ethically, maybe you mean that one cannot give an advantage financially to one company versus the other. I mean, there are really
very good mechanisms in place anyway. So I'm also a Hughes Medical Institute investigator. So that's actually the people who fund my lab and so on and a lot of equipment. And they do not allow me to directly collaborate
with the company founder. It's automatically that there's a nice separation between academic research and then once a company starts, they just also better anyway. So I think it is very good that when you found the company that you give the company, the CEO, the CFO, the director, the... all the employees the full power, it's their baby and they make it happen. So the way I'm advising those companies is really that I believe in their excellence and that they are taking over to crack the problem, so that they are... And are already working in an academic lab on the next high-risk question. So coming to high risk, we can take
high risk. We don't have to make a drug. So I think it's a good separation between high-risk and proof of concept in academics. And then all of the ever made drugs, you have to have VC funding and millions of dollars in the company. So I don't know whether that answers your question, Oliver.
You did. And Oliver clarified that he was asking financially, so you did answer that question. Next question is from Alan Bergman, investors are interested in key customers or vendors as well as the market size, without those two factors in an early startup, what other factors do you consider in an early stage five tech startup? I think it comes back to people in working with the rock stars of the industry. I think it's really important for us to keep a very high scientific bar, because at the stage we're talking about at discovery even into early launch, we really aren't at that point where we're knowing even the type of indication that a company may be going into. I look at oncology, for instance, we may have an excellent technology that we're developing, but until the technology matures, we may not necessarily know what type of cancer the company wants to go treat. So it really does come back to what's the academic institution, what is the pedigree of that scientist? Is there a group of scientists working together in a similar area that we could put together and build a spectacular company? Those are a lot of the ingredients that we put into our analysis is we're trying to ask those tough questions during the discovery phase.
Thank you. That is the list of questions that's on there. I would encourage the 85 participants, if you have additional questions, please do submit them all. Oh, there is some question from Sam again for Dorothee. I don't understand how the mRNA of the mRNA COVID vaccines leave the exosomes and gets into the cytosome since the liquid membrane excludes molecules greater than 500,000. Typically, it takes months for a biologic drug to be fully metabolized, but the mRNA vaccine seem to getting very quickly, luckily for hours. The assumption is that there is some disruption of the membrane to allow the mRNA through but has anyone looked at this? Okay, excellent question. Very tellingly. So in order to not lose the other 85, I was
just going to summarize basically the messenger RNA is the thing which actually in codes that you'll finally make those proteins, the antibodies, which will fight off COVID. But of course, it has to get into the cell. And messenger is getting charged, so it would not go through the membrane. And so, the second ingredient in the COVID vaccine is actually cationic lipids. And that is a difference between Moderna and the Pfizer vaccination, is the details of this cationic lipid. So basically, you have a negative charged mRNA, you cover it
up as a positive charge lipids and it goes into cells within actually seconds or minutes. Now, why is it so important to explain that? First of all, it answers your question. That's why it goes into no time. Second, lipids, of course, biological natural materials, so there is absolutely no risks, so there is no side effects, right? And that's the difference for instance, with the AstraZeneca vaccine that just delivered by adenovirus. So they're the biggest breaks through, they're two big break throughs with this mRNA vaccines, which makes it revolutionizing and no risk what's so ever, mRNA cannot do anything except making that happy protein, which fights off the COVID.
And it gets degraded and it's gone. Lipids, it's basically when you eat some butter, right? It gets degraded. So there is no component in these vaccines, which has any, any artificial thing in there, right? Basically. So this mRNA delivery that was developed many years ago, that's why we
could develop the vaccines within 10 months, because all this methodology was developed. We are using their technology to deliver DNA for research in ourselves. We even hijack the delivery to put our allosteric protein inhibitors into cells. So when you use these cation lipids, just to put anything in the cell, but this is really a very good question.
I hope it explains to the non-experts why the Moderna Pfizer vaccination is so cool and so safe. Excellent question and excellent answer. We're getting some fantastic questions. The next question is from Chris Doyle, could you comment on current controversies around ownership of IP related to the COVID vaccines. IP around COVID vaccines, any information there, Kevin
or Dorothee? I'm the wrong guy on that one, unfortunately, Vipin. Yeah. Sorry, Chris. I don't know either. But what I'm very encouraged by is that people are getting the vaccinations at very low costs or free worldwide. The vaccinations are going to meet us out of this pandemic. You ask a good question and I apologize.
There's always so much in the media, so sometimes I'm not even sure whether there's actually that much controversy. But I agree with both of your answers, I'm really happy as a scientist to see that the real treatments supersedes or super overrides any IP issues, if there is, so I'm very happy about that. That's right. A fantastic question from Melissa. It's for you Dorothee, which is a question that I want to ask you as well. How do you balance all your different roles? Teaching, research, starting companies, basketball, family, and finding time to sleep. What does
a typical day or week for you? So maybe let me start with a nickname. The basketball teams where you go to these world championships gave me, so my nickname is actually... My usual name, people call me Doro and they call me Dorocell from Duracell.
So they asked the same question. Do you just stick two fingers in the outlet and then recharge in the morning. So my nickname Dorocell. I think it's genetics. So, that's would be a joking answer, but... The way I approach things that... I do things I love and things which
are harder to do, I try to get it done efficiently. My great grandmother actually gave me some very good advice, she said, we have to do a lot of things that you don't like, but if you try enjoying them, you will get them done much quicker. But really the balance, what I would say is you have to always set priorities and the priorities early on was professional basketball. Eventually you had to make the hard call and when it came to the states, I had to give that up. So priorities change. Second, you
cannot be a perfectionist. I would call myself, I wing a lot of things. And finally, it's all about quality over quantity. And I think that's really, really important that you say, okay, where can you focus on something right now and do you want to do it to really excellent.
And for excellence, it's actually quality and not quantity. So I quite often think first about a little long ago, what I want to do and how I want to approach it. So for instance, science, don't do the experiment from A to Z but maybe you can only miss two or three experiments. You get the full answer. So you can get it done in a much quicker way. So it's really about quality versus quantity and not try to be a perfectionist. But finally, I would
say sports, family, these other things are actually giving you the energy to be better in science and in my job as well. So the balance is actually a strength and not a dragging time away, I would say. So, a really good question. Everybody has to find their own style and it's a constant learning. It's juggling. You want to do 50 things, but you can only get 10 things done a day and you have to be okay with that. Love it. Next question is from Lakshme Miller Baden, for Kevin. How do you decide on the breadth of focus of a new biotech company? More specifically, how do you balance focusing on our, a.k.a, fascinating problems while still being able to convince investors it's
doable? That's something we wrestle with all the time and it goes into that process I referenced as discovery where we're starting to map out what we think the size of the series A can be and where we think it's going to get us. And it's very important in that phase where we are driving the initial progress on maybe the first program and or programs and all at the same time developing what we call our, these product engines, these platform of technologies that we hope would allow the company to have multiple opportunities as it gets closer to the clinic. Because I think anyone who's been in our space understands if a company, unfortunately, is pioneering, maybe just one drug, you are often setting yourself up for a binary outcome clinically. And I think we've all unfortunately been in those instances or seen those instances where the card gets turned over and you're in a tough spot. So that balance by which you're prosecuting the early programs, keeping enough capital going toward the engine is a fine balance that we spend a lot of time at Third Rock thinking about. I think for that next
round of financing beyond the A, in order to see that investor appetite, I think demonstrating that you are on that trajectory, that you have met your series A deliverables, you're at that point in time that you expected to, and then to be able to show the investor, what are you going to do with that next 60 to 80 millions is part of the equation. Thank you, Kevin. Great question, Lakshme. Next question is from Patrick Devin. How close are we to having truly personalized medicine that's customized to each person's genetic profile? Dorothee, you want to take that? I take it, I don't care. Why don't you take it out? I'll add one comment that comes to mind, but I'd love to hear what you have to say.
Yeah. So of course, what's really great that we can now very cheaply sequence the genome of every patient. And I think that's really the key where would say across most medicine, meaning we can find easier... I mean, it's still a complex because of complex signaling
pathways, likely reasons, likely targets, which we have to target. And so, we're getting much closer because of that. Of course, financially, you cannot say, you can go for each single person gets a different drug, but you don't need that because these are common mutations, common alterations, which then need to be fixed. Of course, the second part of the equation is eventually CRISPR. Whether we could actually take the wrong nucleotide out and put the right one in. So I would say we're getting
much to that, particularly the diagnostics, what we want to target. And that's what I was going to add. One of the successes that we've had at Third Rock was a company in the cancer space known as Foundation Medicine. And it was an essence to develop that when a poor patient who progressed with cancer, when their tumor was assayed, our testing was often enlightening the oncologist to look at potential therapies that they may not have known that that patient would respond to. And that became a very successful product... actually two products that are on the marketplace and Roche, the large pharma company, ultimately bought that company because of the success that they were having.
Thank you, Kevin. Good question, Patrick. And the next question is from Pascal Porten, to both Kevin and Doro, what planting therapeutic modalities or technologies most excites you and could have the potential to significantly change human health in the next decade or so? That is, what are you excited to start up next? It had to come from Pascal. You want to get the secrets out? So I would give two answers. I'm very excited about the possibility what we did as a proof of concept in our lab to use small proteins as drugs because they are very selective and they're very easy to develop and they are biological materials. And so far, they have been very successful as extracellular drugs. Antibodies and so on. So the technology I'm most excited about, and we'll have to
find companies is delivering of small proteins or inform of mRNA for this protein in the cell so that we actually can use biologics, small proteins as intracellular as drugs. So that's number one. And number two is to be able to big scale rationally designed enzymes for all kinds of biotechnological applications, vitamin, including plastic degrading, enzymes and so on, that we actually can rationally design it, based on the knowledge we have about enzyme catalysis and then machine learning. So really that we are completely revolutionalizing all aspects of life by biocatalysts. So I would say these are the two most exciting things I would like to correct next.
Great. Kevin. Yeah. I'm going to reference one of the companies that we got up and running. It'll be two years old in May. And it's a company was another diagnostic, but this one was in the early cancer detection space, a company called Thrive. That's using a technology that was developed at John Hopkins University using a liquid biopsy. And the hope there, the company was recently acquired by Exact Sciences at 18 months old. Is that on a standard of care
basis, every year when you go in to have your blood pressure checked and your cholesterol checked, you would be administered the thrive test, and the hope there is that it would have the ability to predict that five or 10 years from now, you have a tendency to develop various cancers. And as everyone knows, the cancer space, if an oncologist is getting that news much sooner, and not waiting to pancreatic cancer to stop showing up at stage four or ovarian at stage four is really nothing much the doctor could do versus if they have line of sight to that, I think the treatment options become abundant. And so, that company now will start a very large phase three study. And in the next two to three years, we'll start to see the data. And I think it could be a game changer if everything were to work.
Outstanding. So many proteins, designer enzymes and diagnostics. That's the bet. So this is way too much fun, but we are at the hour. There's one last question from none of us attendee. And as I read that question, I'm also going to ask you for closing comments,
Kevin and Dorothee. And the question is what have you been most impressed with about Brandeis science students and getting to work with them to do this incredible research? These are so amazing questions. I would just hire some of those people ask these questions. So what is very important in science that you're always humbled and that you're hungry to learn, that you want to work as a team, and you understand how little we understand. So there's this combination between hunger to learn and being humbled, but at the same time, confident that you can make a big impact. And what I see in Brandeis is we tend to attract students in this category. So, I did my postdoc at Berkley and of course I go around the world
giving talks and some of these very high name universities, sometimes you have the tendency that people really full of themselves. And so, students who think they know everything and they are overachievers. And I think that is a death penalty for research and discovery. And so, what I really like with Brandeis students is that they are not like that. But at the same time, I think we
have an environment at Brandeis, where there is not this cooker pressure environment. We allow them to explore and to exploit and to look into different directions. And that's the other thing you need to do in science, you cannot say, in two months, you'll have to have this problem solved. It's a journey, and you have to be able to breathe free, to think free, to exploit your ideas. And I think it's the environment of Brandeis, which allows that, and what I have seen with my grad students and undergrads is amazing growth from where they started and where they ended up and then they graduated. And they made it happen. So they took their own journey in their own hands, and they were
proud of all the struggles and all puzzling together in their growth. And I would say that's the secret of Brandeis of our students, but also about our professors, our environment. This distinguishes us from many other universities. That is very well said. Kevin, any closing comments from you? Yeah, no. I look back on my time at Brandeis very fondly. It wasn't really sure where I
was going to end up in my business career. But I think the ability for the university to develop an ability for me to communicate, I think, as a finance professional, I've used that along with just the sense of teamwork to really dig in and be that a solid teammate on various projects over the years. A couple of things that Dorothee said, I think of the university is a very scrappy university as well. My mom has often characterized me as a bit of an overachiever and actually that's one of my most favorite compliments because I really do think that people shouldn't be resting on your laurels every day, get out of bed and work as hard as you possibly can for what you're excited and passionate about. And that's how I've lived my life and I really do appreciate everything that the university gave me from a solid footing to embark on this very exciting career.
Thank you, Kevin. I would close to thanking everybody in my closing remarks. You could be sitting in front of Netflix that everybody dialed in. I loved the questions. I think really helping to make the world a better place, whether it's training the next generation, that's big translation that I had the privilege to now work with Relay, with MOMA, with amazing people. I owe
them everything. My teammates in basketball, my family. So I really think it's the people you surround yourself, and they are really the answer. And so, I'm just really glad that people dialed in and ask some questions. Please reach out to us, you'll find us on the internet.
The best part is really the communication. I hope we can meet each other in person very soon, because I am a very vocal person and this Zoom is really hard. It was really fun to have people interested in this kind of geeky question. We're hoping to help a little bit as much as we can do the world to make it a better place.
That's great. It's the people that you surround yourself with. And I am blessed that I was surrounded for the past hour and a half with Dorothee and Kevin, and the 80 plus people who joined in the conversation, thank you so much for your time. Thanks to Alyson and Loren for organizing the event and for the tech support. This has been a fantastic event. And I appreciate all the questions as well.