YouSaid · the spoken record

Dinakar Singh

lines on the record: 41
first: 2025-06-15
most recent: 2025-06-15
sittings or episodes: 1
sources: podcast

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“After a daughter is diagnosed, people just give me hugs. Lizabeth the best feeling on earth. And there's nothing more powerful than just compassion from friends, strangers, just to feel like you're all human together. I think in life, at our worst moments, just getting some of people's strength and energy imbued in you a little bit gives you the ability to go and stand up and move forward the next day.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“The family had gone around through supermarket stuff, meaning cans and signs in town raised $400,000 to get her treated. And they couldn't find someone to take the money or find a practical way to treat her on stuff. And so she was going to die or suffer unnecessarily because of crazy regulatory bureaucratic stuff between countries. We got to figure out something on the cost. And maybe it's with these drugs having some amount of money get set aside when there are these windfall profits to go and help charity care or things like that. But the notion that we develop these drugs and their kids that are dying because they can't afford them is pretty insane.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“CFO. Because since Columbia was a major centrist, it could fill in Harvard or Stanford or whatever as well. The SMA drug cost $800,000 a year, the first one. And so to go and actually buy the doses to treat the kids would have cost tens of millions of dollars that they didn't have their budget. It's crazy. It's a real issue. And I still get calls and we get calls all the time from people in other countries because, as an example, there's Goldman Sachs analyst who gives from Uzbekistan and they called asking for help because their family knows someone close to them whose daughter is diagnosed. And if they get treatment, they'll be saved. But Uzbekistan, there's no place to get treated in Uzbekistan. You can't just come to the US and get treated because all sorts of legal and other issues and loopholes. And of course, then you've got to cough up the money to do it as well, cost you a couple of million bucks. You can't just go to London and do it because, again, the whole regulatory framework is crazy. And so there are still kids dying of SMA. Even in cases, in this case, for example,”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“And companies really don't like collaborating with each other because it's an IP problem. And companies and universities actually now are having more difficulty collaborating because universities want IP because they've seen the money you can get from royalties and stuff like that. So having a third party that's sort of Switzerland and can be a bit of an honest broker on this stuff is actually pretty important. So that's my hope. finding a way to have a more thoughtful process so that people don't have to go and try and figure out how to go and see if there's something there. Now, on the back end, boy, I mean, it's good that people care about disease, but the cost of this stuff is insane. I mean, what companies are charging is just nuts. I wish when we had put all the money in, we had put in restrictions on what companies could eventually charge because a million to two million bucks a year is bonkers. Give you a funny example of it. I remember talking to the people at Columbia when the first drug was coming out. And it actually was a working capital problem that had to bring their...”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“And so, whether it's places like Jackson Labs, whether it's NIH, having these amped up translator research efforts where people can set aside a little bit of money, because honestly, that early seeds doesn't cost that much either. It's later on that you really start going down many rabbit holes. But for a relatively small amount of money, you could actually go and get people a good chunk down the field to where companies would care more. Or you think about obviously the flavor of the day is terror governed down. Government did a pretty good job in some of this stuff. But certainly we can make government smarter. And I think whether it's government or whether it's institutions like AX, there is a role there to be played that could be pretty thoughtful where a few diseases are picked at a time and a modest amount of money is put in or coordinated so that you actually end up having a gameplank because what a lot of people just need is a game plan as opposed to handing money out randomly. And also a third party who that's viewed as being somewhat neutral because academics are great people, but man, they don't like collaborating that much.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Let's say parallel processing or ruling things out. You don't get a grant by saying, I don't think this is going to work, but I just want to prove it doesn't work so I can then move on. That doesn't get you grant money on stuff. But a lot of those things and the tools and things like that are very much the nitty gritty that actually get you through that early stage where you say, whoa, I've got something.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Because scientists writing papers and grants is really not a very effective way to get this done. And there's still too big a gap between a good scientific idea and when a biotech company is actually going to go and spend the money and do it. You don't have to get into the 10-yard line anymore, but you still have to go to the midfield. And so I think more novel structures would actually be great. And I actually come back to that and I translational research program from way back when it didn't end up making that much of a difference in SMA because we just end up throwing the money out, going it ourselves. It's a pretty cool idea. And if one could do that, again, it's that Ghostbusters idea that I'm doing at Jackson Labs. Because the problem is we founded this world that companies mean well, but they're not trying to solve a disease. They're trying to attach value to an IP that they have, meaning if you solve SMA with something that they don't have IP on, it isn't very useful. Scientists are in the business of getting grants and you don't get grants for.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Some good things and some bad things. I guess that have happened leading to what I hope will happen next on the good side. You don't have to bash someone over the head to get them to understand the urgencies can be lucrative for them. I mean, SMA has been, there haven't been that many, but when people have seen the money that's sort of been made on this stuff, people care. But the challenge remains, which is that if you're a company working on oncology, even if there isn't a drug tomorrow, you're going to be working on ecology for the next hundred years and that information can be useful in other things. But the challenge with her disease remains that a lot of these are pretty bespoke. So the difference from 20 years ago is that you don't need to convince someone that it can eventually be very profitable, but you still have to convince them that there's a good chance that they'll be able to go and cure it. Because that early stage investment is tricky. There needs to be a much more thoughtful effort working with NIH FDA and disease groups to find ways to go and have a much more thoughtful, practical early stage.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“And we'll actually get some scientists to work on it to go and help you put together a plan, not caring whether it's with us or not, but essentially help be the brain trust for people who want to go and think about game plans for trying to solve disease that affects someone that they care about. How do you hope?”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“They essentially been to mouse models and are still the leader in that. It's an incredible institution. No drug has ever been developed without using their mice. So I joke it's the most important biotech institute that no one has heard of. Early on, we had gone to them to work on building mouse models and making them free so people could call them up and they'd send the mice overall. But working with them and setting up actually a Respute, we're going to have a squad of people to do a bit of what we did in SMA because I hear all these heartbreaking stories and they're the same things that I had 20 years ago. People that want to make a difference don't just want to go and make random donations that I think will just be like throwing water on sand. They don't know how. Like in our case, we had a lot of resources and a lot of connections and money and could essentially go reinvent or invent the wheel. But for other people, it's a waste of money. It's a waste of time, both of which are incredibly scarce when you're a parent with a child who's suffering. And so our goal is to actually have, I call it the Ghostbusters, a rare disease, where if you have a disease, you call us.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Think it captures the process that one goes through. I think for every parent it's part sorrow and it's part rage. And depending on the moment, sometimes it's rage than sorrow. You just want to go and break everything. But I do think that you do eventually figure out that that is, well, very consuming. It's not going to get verified under any circumstance. This is where knowing other people and talking to people that have gone through things like this actually is hugely helpful because it just helps you recognize that there's a journey on all this stuff. And so I think every parent I know whose child has an issue was unbelievably emotionally supportive to us. And as well, almost every couple weeks, I'll get a call from someone saying, look, I've got a friend or some and etc who has a disease. They want to make an impact on it. They've heard your story. Can you help? And that's to me the greatest joy I have speaking to other parents and helping them think about what they can do, what can be done, what can't be done, etc. I joined the board of”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Sorrow into productive channels so as to perhaps have some good, some purpose, some meaning in all this. And so joined his other parents of children of SMA in efforts to improve care and further research that this disease may one day be eradicated. In doing so, we found a productive outlay for our sorrow and our anger, and also a real reason for hope. And finally, let me just say how grateful have been part of such a wonderful community. The strength and comfort offered by many of you has helped us focus on life, not on sorrow. After all, in life, there's no doers, no attractions, there are no appeals. Either find joy in the life you have or live a life without joy. And while the circumstances of your life might not be up to you, whether you choose to find joy and purpose in them is very much ultimately up to you. So that was his speech.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Slowly realized that if we just hold our heads in our hands, we're accomplishing nothing. A daughter or a sales for that matter. And first, it's hard not to be consumed by the shattered dreams and lost notions of what could have been. Yet over time, we've come to focus on the many good things that can be, and realize that there is joy in everyday, if anything, we're now much more focused on appreciating the little things in life. And today, despite all the issues, our daughter is as happy and joyful as any child we know, and we are as lucky as any parent we know to have a child as sweet and adorable as she is. So have learned from our daughter that while the sources of joy and happiness might be different than what previously imagined, that doesn't mean that the magnitude of the joy is any less. And third, we realize the importance of channeling the network anger.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Even still by yourself. And yeah, three realizations have helped us slowly face the future. First, we've truly learned and appreciated the importance of friends and community, the comfort and love of friends has been a source of incredible stride for us. Getting other parents with issues and learning from them and the incredible challenge many of them deal with has been an incredible source of inspiration for us. We've realized that there are no good diseases. There are many, many strong people, and their courage is truly infectious, far more than they need seize. More than ever, we've come to realize how much deeper our reserves of strength are as part of community than as individuals. Second, the strength, comfort, spatial of our friends, have gained some perspective and been able to focus on finding the joy in the life that we have.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Most of you probably know of SMA. We've certainly hadn't, and we'll horrify by what you learned. It is the most common genetic killer of infants and is untreatable, incurable, and eventually fatal. It's described as a children's version of ALS or a genetic version of polio. Simply put, motor neurons die, muscles break down, leading to eventually fatal complications. In most cases, SMA kills when the first two years of life, less severe cases, such as our daughters, depression can take many years, so as the mind grows, the body steadily weakens, and eventually betrays it. Obviously, we are devastated and shattered, and over the past year, our hearts have broken every day as we've watched her become irresistibly adorable, and learned to sing, tell jokes, talk endlessly. But also lose the ability to stand up, the booty crawl, silt and walk.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Consider. I mean, guys, you know, that's absolutely illegal, but you had to do it because it might be legal. But unless I tried, I mean, so when she would start in school, we would send up write-ups to other parents, basically saying, here's what SMA is, here's what to make of it. But in any event, for nursery school, she went to all souls, which is the Ontario Church School. And initially, they didn't want to take her and they said, look, there's great surgical policy schools. You can send her to. Yeah, that'd be great, except she doesn't have surgical policy, guys. This is a totally different disease, but eventually I think they felt badly and they took her and she had a great experience, but you're supposed to go and volunteer to give sermons as members of the congregation. And so I did. I'll read you that quickly. Speak to a good”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Is like two or three. We're getting into nursery school. And by the way, another saga and another crusade that I'm going to be on more aggressively going forward, the getting schools to care or take her was unbelievably hard. When she was going to private school, I think she was the only kid and major in New York City private school in a wheelchair. And I kind of know this as well because I'm on lots of boards, public library, lots of instructional boards, lots of eventual friends, lots of charity relationships. I couldn't find a single school that would take her. Many are perfectly accessible, but I'd get calls from friends on the board saying, Lieutenant, it's just not going to happen. The ostensible reason, I think one school said she wasn't bright enough crazy. Turned out to be maybe not bright enough for them, but bright enough to be straight as at Yale and all that kind of stuff.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Funny how it's ended up being a circular process, but it costs a lot of money. I think we spent probably $150 million on our own money on this. But in the scheme of things, The commitment that we made at the beginning to ourselves was that if there was a way, we were going to find it. And no matter what, make sure that we were able to go and drive through it and winding back to where we started in some of this. I guess that's a bit of a stubbornness, but also maybe preferable word. It's the termination I saw in my parents. So in that sense, I think really nothing less than one that I would do for my daughter.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“And Roche makes the drug that my daughter's on, and my father got a job at Roche as a Roche scholarship that paid for my college.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“10,000 kids and their families. Many of the people that we've gotten know the best over the years are people whose kids are affected by disease because there's a commonality and it's devastating for everyone. Parents lose their jobs because they keep taking time off and they can't afford an insurance. I mean, it's just this horrible spiral. There's a family, the father worked at Walmart. They had twins with SNA and he just wanted to cry hearing their story. That is the funny irony with all this stuff. My parents were both scientists and they would joke that it was such a tragedy that both their kids became partners Goldman Sachs. In some ways this all came the full circle because having some of the science background than having the good fortune and the good luck and privilege of working and investing in finance helped us go and actually put that science to work. The further amazing irony Columbia was our leading center in all this and Oman had worked on faculty Columbia.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Till I submit none of them have been solved. And so it's a bit like that boat that you haven't fixed the hole in in the first place. SMA is now becoming this really fascinating area because suddenly if you have an idea for a muscle drug, you can actually test an SMA and actually see whether it actually does something because now you've fixed that boat, if you will. And so it is funny, quite a difference from 20 years ago when no one cared. Now suddenly we're calling out people saying, look, we think there's an idea your group can really work here. Let's go test it out. They're doing it. So fingers crossed. I think my hope here is that we'll end up with a handful of drugs in the next handful of years that could really help bring back some strength for kids with SMA and other things as well. That would be amazing because as amazing life as a daughter has now, she is still fragile. And when you're fragile, as you get older, things happen. And so if we can bring back some strength, that will be even more transformative and even more factual. So it's exciting stuff.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Assume that there would be tons of people and experts that knew all about muscle development from muscle stem cells called satellite cells into muscles, et cetera. There may be six people in this country that know what they're talking about when it comes to muscle. It's crazy. I assume, well, gosh, you know, if you've got ARD and steroids and all that kind of stuff, I mean, there must be big money in it, but the reality is muscle has been a really tough field for a couple of interesting reasons. There are two types of people that have muscle problems. People with degeneral diseases like SMA, musculosphrophy, et cetera. But on the other side, old weak people. Old weak people are a disaster to go and do trials on and you're trying to see what a moderate impact can make when there's such decline happening. And so clinical trials, and this is the problem with Alzheimer's, are very, very challenging in elderly generating parents. And in that first category, people with muscular disease,”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Describe to people if you have a vote and you have a poll in it, trying a bit of water out of the boat is not really going to be a useful strategy. You're going to sink just maybe a tiny bit slower. Once you fix that pole, and now there's water on a boat, now bailing water to boat could actually be a really good strategy. Mission stage two now is that we're working on a ton of drugs that we think can regenerate muscle, node is harder to do, but muscle is a little bit easier and also help existing nerves get a little bit stronger on stuff. We have one already in the clinic. It's not the best one, but we expect to have three or four things coming forward that could be really impactful. And what's actually pretty cool about this is that the SMA drugs are SMA specific, 20,000 kids, so it's a lot of people, but they're SMA specific. These regeneration drugs will actually help lots of people. And it's ironic because SMA is actually becoming a poster child for this because cake muscle is an example.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“So they keep getting weaker and weaker on top of the original poem in the first place. And that creates this devastating cycle. And so here between diet, between exercise, between therapy, the goal was to go and keep her as strong as possible to hold off that damage from all these things. And it worked and it didn't work. She did end up meeting his final fusion and having rods put on her back, etc. But fortunately, those were late enough that she had already grown and so didn't create the deformity you normally seen in someone else. She had promised their hip that blew out and things like that. When she was turning 10, 11 every year, there became a major surgery. And even aside from those major surgeries, she was in the operating room, or she was in the hospital probably three times a year with severe pneumonia at risk of dying each time. And with all these things, it is bit humpty dumpty like. Once it breaks, you can't just put it back together again. On one hand, if it had been a year earlier, that would have been all the better. But the good news is that compared to what we expect.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“And then you start getting pneumonia and all sorts of stuff. And so you end up, I mean, in the old days, you have enormous deformities with children. And like many degenerate diseases, ALS, Parkinson's, even Alzheimer's, no one dies of SMA. It just makes you really weak and then something gets you. Whether it's cessation or it's choking or it's pneumonia more often than not. Val Kilmer just destroy of pneumonia, but pneumonia was really just a side effect of throat cancer. In her case, our goal was to manically keep her strong as possible until the drugs can come along. And so physical therapists coming to see her every day, we built a pool in our apartment building for her because you need warm water and it's a special therapy pool. But that was important because when you're a person who's weak, part of the problem is it's circular. You're weak. You can't exercise and stretch your muscles and bones.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Which is pretty cool. The good news, bad news, of course, my daughter ended up being on the first strike since she was 12. So it's been stable since then, and has ended up being just the rock star of the family, went to Yale, won three of the 11 awards for most outstanding student class speaker, you name it. And during COVID, amazingly, went on hinge and met up with this unbelievably amazing 64 fantastic Princeton kid. They fell in love and they got married last fall. She's now getting her PhD at Cornell. So I guess we'll end up with our doctor and the family. But just watching her brief has been remarkable. That said, the clock was the clock. Every year she got weaker until the drugs came along. And SNA, the progression typically would be kids get weaker, stuff starts falling apart like your spine and you need spinal fusions and that means your spine is constricted. You can't grow. That creates lung damage.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Their approval to essentially run a natural history site, meaning while the drugs were being developed, we paid for people's care for a handful of years so that we could have a log of what the natural progression of the disease was and use that as our placebo. And so when it came time to the trials, the trials didn't have to be placebo on trials, essentially because the FDA was willing to accept the fact that the whole compare, the results in the trial to what were the natural history study results of kids normally with SMA. That saved a lot of lives and a lot of money because otherwise half the kids would have died in those trials, like potentially our daughter. With all the persistence and with a few little help from our friends as well, the first estimated drug, I think, was then maybe still now the fastest approval by the FD ever in history of any drug.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Because it might be fixable. And I think that became a powerful combination. It took some work. For example, got 50 top scientists who Nobel Prize winners to sign a letter talking about how there was a really good chance of successor. We then started literally taking out full page Colorads in some of the political newspapers in Washington, roll call, Daily Hill, all that sort of stuff, so that all the staffers would see this very striking roll color ad every day on this stuff. Lots of stuff like that to really go and essentially create a real sense of momentum on the side because it did occur to us that while at the end of the day developing the drug mattered and getting companies involved would matter, everyone would move faster if they thought that the other guy was there too. And with the FDA as an example, that really helped. They worked with us and we said, look guys, we need this to move fast. And so what can we do? And by the way, kids are dying. We can't afford to run a placebo trial if it doesn't make any sense. And so we work with them and got.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Approve drugs. So the first was nebiogen cells, the second was gene therapy with the lexis, and the third is Roche, which is just a synergies you take every day. That was incredible luck, I suppose. But part of what we did from a time perspective, because we're sort of desperately worried about the clock running out, was try and make sure that we could have everything in place so that when these drugs were ready, they could go 90 miles an hour. And so, for example, we went to the FDA through lots of relationships on the political side, both to get NIH moving, by the way, in the early days, and to help later on, we got to know, if you will, all the relevant senators and congressmen that mattered when it came to drug development, NIH, and things like that. And look, our view was just apparent with tears on their face. It doesn't get very far. But we would say, look, we want to be really supportive of you. There's a chance there to actually achieve a success. And that's why you should focus on this, not just because it's sad, but”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Go and test everything under the sun. They can imagine with different screens and ended up finding one that really, really worked. And so we basically just paid to develop that and I actually got Rochan to go and take it from there. That's actually dragging my daughters on now. In between was gene therapy, which a company called the Vexus did and then Novartis bought in for $8 billion. And I guess this is really the first gene therapy drug ever approved. Here we didn't support it as directly from a financial and other perspective, though our board members were active investors and we helped them with the network and the library and the clinical trial networks are focused just being selfish to some degree was that gene therapy is great but only works for very little kids essentially gene therapy you're taking viruses you're bringing protein in your body and when you're large that amount of virus will kill you basically the toxicity becomes an issue but we wanted all three shots on gold to be explored look we got lucky today we have all three is”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“We basically had to go put in a box with a bow on it and give it to people for free because otherwise they'll never touch it. And that's really what we did. I mean, we spent a fortune on models testing everything. It was clear after testing everything that existing FDA-proof drugs, some would have some impact when none would be good enough and you'd have toxicity issues, so it didn't work. So we then pursued all three shots on goal that you thought were relevant. Cole String Harbor Labs and a great scientist that had what's called antisense oligose with the first biologics essentially the viral vector takes approaching the body and it was designed to patch that defect in that backup gene and help that backup gene become a fully functioning regular gene which should be magic presto that became the first drive when the biogen ended up buying in was good old fashioned small molecules syridges and pills a bit like novartis for example had found something that worked we basically paid a company called pTC therapeutics lots and lots of money to”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“In the world is Vastin, which was a mycology drug, at $50,000 a year. No one's ever going to pay that kind of money for a small rare disease for children. It became a running joke in the end because this person, Alessandrock, was an incredible advisor helper to us, joined RSAB and really helped us really immensely. At the end, the first drug that we helped develop through Culture Marbor Labs and then a company called Ionis, we then had them partner up with biogen. So Biogen essentially bought in the drug and became the first market with an SMA drug. And when they did, they charged, I think, $730,000 a year for it. I think they're a top three drug pulling in, I think, four or five billion dollars in revenue. And so amazingly today, the SMA drug market, I think it's high single digit billions and it's a top three drug for erosion of artists and biogen. But the notion that a pediatric neurology drug would be anything of value back then was a hard cell.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Finally, we got Novartis to bite and they ended up finding a drug that had failed for tox reasons for anorexia or something back in the 70s that was off the charts good. And so suddenly like, oh, this is interesting to give an example of just how different the world is now versus then. Early on, I'd use relationships to go drag some of our scientists and some of our advisors, et cetera, to go meet with heads of research at different companies and try and pitch them and say, look, guys, I know this is a small disease, but there's a chance to do something. It won't cost a whole lot to find out. You don't have to spend 40% on marketing, all that kind of stuff. And so one of my slides showed that if you charge five to fifty thousand a year, you could actually end up with a drug worth hundreds of millions of dollars in profit. And so it wouldn't just be a charity project. It could actually make a little bit of money from it. And the head of one of the largest biotech companies actually, it'll have to be in the meeting and said, look, Jennifer, most expensive.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Some scientists have done some work suggesting that some existing drugs could actually modify the disease in mice and things like that. So we basically created all these mouse models and some models of different types to test different versions and each has pros and cons and set up facilities to essentially give them away for free. So essentially what we said to everyone around the world, guys, send your drug libraries in, your compound libraries in, test them for free. We will pay for it. You keep the IP. We just want you to know if there's something interesting here on stuff.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“For Olvin, what was extraordinary was how much of their time and energy they're going to commit. Obviously, you weren't paying anyone, but we'd get a dozen and a half of the smartest drug development people in the entire world coming together for a couple weekends a year and just taking time away from their families and their work and just sitting with us and brainstorming about our plan of action. And I think part of it was because it sounds crazy to think about this, but there had not been a single meaningful drug for a neurological disease developed in decades. crazy, right? So the idea that there was a chance to do something here was really cool because neurology was kind of a graveyard. I think everyone seemed to have this sense of excitement that, okay, there's actually an interesting plan here. Maybe we'll work, maybe it won't. And there's someone going to write the check and fund this. So it's not about going and begging for money. We're excited with the idea of joining in to go and figure out whether we could actually go and solve a disease. One first stop was, look, there were”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Rounding back to the long answer, the very short question was really just there to remind me of what the mission was. And so it was always amusing because people would sort of wonder and I explain, well, you know, Axon is the kinx muscles and nerves and brain. And so kiking thought to action. And they thought that was a cool analogy from a just general perspective. Every now and then you get a scientist or a former company who would see our logo, which is actually the shape of an axon and would get the fact that this was actually about medical research on stuff, which is always kind of cute. It's a bit of an Easter egg that we sort of hid in there. But look, so that was what we did. I mean, functionally then my day job was really there to pay for my night job, if you will. We got teams together. TPG was my partner in starting up axon. They were really remarkably helpful through my relationships and through some of their network. We were able to really go and connect with some of the absolute rock stars in drug development and people that were not just smart scientists but actually were biotech guys that really understood this stuff.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“Would move this ball down the field, and then go and do it, whether we pay someone at a company or we give a grant or whatever, but give money, get it done, move on every front at the same time, and then keep coming back and saying, okay, what do we learn? What do we learn? Let's go on to the next thing. Now, all that takes a lot of money. My father would joke that for the straight hour kid, I developed the world's most expensive drug habits. I was a partner Goldman. And look, at some point, I would have left anyway. In reality, at some point, if you love investing, you want to go and just do it in its own pure form. But it certainly occurred to me at that point that given all this, it was time to get going. One, because of course I'm passionate about investing and I wanted to build a great firm. But second, because the value doing that right now and the dollars that could come from it right now could save my daughter in a way that if I waited five years or three years or two years it wouldn't. The axon name.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“It struck me that all these things take time. You could fund some scientists, get some basic research, and then go to the next one and the next one to the next one. And that will eventually work, but it'll take forever. It struck me that we need to do things in parallel processing, not zero processing. If there are five interesting ideas, let's do all five at the same time and see which ones work, not do one at a time and go A, B, C, D, E, FG. So basically, what we drew up was a pretty aggressive vision. Early on, it was giving money to people like Columbia, et cetera. And we did set up Motor Neur Institute there with someone who wanted to fund ALS as well and quite similar diseases in terms of some of the impact. But it occurred to us that academia, while very important, was never going to go and develop a drug. And so we really had to create a virtual company. So the thought then was hire some scientists, get some amazing advisors together to really help BR brain trust, and then sit down every so often with Evere and say, what do we need to do to?”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“But we might be waiting a couple decades or so for this to work by that time, she's going to be gone. It occurred to me, and I tell people this now, the second worst thing that can happen to a parent is to see their child suffer and die. The single worst thing, though, is to see your child suffer and die only to find out that you could have done something about it, but it wasn't done in time. Once we got the idea in our head that there's a glimmer of healthcare, it really almost draines you, if you will, because then any second you're spending doing anything that isn't max speed is a wasted moment. And so that led to just, we're going to take a shot of this. I'm not going to go and take a shot, only to find out we went slowly. So this has to be max turbo, plug all the stops. Let's go for it. Part of that was money.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“There were a bunch of changes. The head of NIH stepped out and the head of NINDS, the neurological institute, stepped down. And so you had acting directors in both places. And I joke to people that, but if acting directors, that means there is no action on stuff. And so nothing is really happening. So there's pitiful grant money from NIH for this disease, even though other diseases of similar size and a lot less potential probability success were getting tons of funding. Because, I mean, there is just a latency bias in things if you think about this if you're a student in medical school and a disease is well known, you might want to focus on it afterwards. So you're doing more research. If a disease no one's ever heard of until recently, there aren't many people working on it, so there aren't many people apply for grants. And when you apply for grants, there aren't many people who understand it to know whether it's a good grant or not. And so there's a lot of stuff that just takes a long time for the cycle to work. And as you stared at this and said, look, there's a chance here.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source
“As it happened, there was an interesting coincidence, which is that NIH at that time, for the first time ever, the world is very different today, but rare disease was not something people focused on back then. No one cared. There had been one rare disease drug really ever, and companies were focused on big things at that time, statin, BDs, drugs, oncology. No one wanted to work and look at rare disease. NIH was also funding all these big diseases, but they actually came up with a novel program at that time to have the first ever grants awarded for translational research. And so they actually picked SMA because of this cool nifty new disease and said, we think this is the best target. We'd like to have a small program where we can actually go and provide some grants to work not just on basic science, but work on actual drug discovery. Again, today, this seems completely obvious. Back then, that was a really novel thing.”
2025-06-15 · Invest Like the Best · Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428] · IDENTIFIED FROM THE TRANSCRIPT · source