Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428]

Transcript from https://podmenti.com/t/0adcdbc7074f22cf

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Hello and welcome, everyone. I'm Patrick O'Shaughnessy and this is Invest Like the Best. This show is an open-ended exploration of markets, ideas, stories, and strategies that will help you better invest both your time and your money.
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My guest today is Dinnikar Sync.
Dinikar is the founder and CEO of Axon, the family office successor to TPG Axon, which was a successful global long short hedge fund.
We wanted to share his story on Father's Day to honor the person and the dad that Dinnar is.
He shares one of the most extraordinary stories at the intersection of finance and medicine that I've ever encountered.
This conversation explores the highest stakes investment themes, timing, concentrated conviction, exceptional team building.
and deploying resources towards outcomes that mattered most.
I wanna let him tell the story of the remarkable journey that he's been on to cure a disease called SMA that so deeply affected his family. Please enjoy my conversation with Dinnar Singh.

So Dinekar, I'm so excited to tell your entire story. It's one of the most unique.
moving and incredible stories I've encountered in investing and I guess in life more generally.
I was thinking about how to ask about it.
To kick us off.
And maybe the appropriate way is just to ask you why you named your firm TPG Axon.
The axon is the nerve that connects your brain to your muscle.
And it
Happens to be
the core part of what degenerates in spinal muster atrophy, which is the disease my daughter's diagnosed with.
And so from my perspective
an important factor in starting the firm and leaving Goldman at that time.
Was that
On one hand, why I loved investing.
I also
just resolved that there was a glimmer of hope to save my daughter and other kids who had her condition.
But that glimmer of hope was not gonna last very long, and time was not our friend.
And so I needed to go as quickly as possible.
To drive.
Success as quickly as possible.
obviously for its own sake, but more importantly, honestly so that I can go and throw
A terrible fortune.
into the research we needed to do to make sure we did it as fast as possible in time to save her.
Because she was diagnosed in two thousand one.
I just come back from Hong Kong literally a month or two months before. I've become global head of the group while there.
I was in Hong Kong for four years, setting up the Asian business and becoming global head at that time.
Came back.
We were on top of the world. Our group was historically profitable even during the coup years of the firm.
Came back and a month later, nine eleven happened.
And a month later.
Our daughter was diagnosed with
SMA is spinal muscle atrophy.
And she was year and half the time we've been sort of shifting back and forth to Hong Kong and New York and it's your first child.
It's ironic because you actually don't know what
is normal in terms of development.
And so we thought she was developing normally, but
We were at a party and one of our friends' mother was a very good the trician and said, Look, you want to go and have her checked out because
Just go have her checked out.
And that started the saga getting test after test after test and eventually
Literally a month after nine eleven.
sitting in my office watching the smoldering ruin of uh the world trade center, which was right in front of my window.
And getting this all from the doctor basically saying that she has SMA.
For a bunch of reasons it was a shock.
Well, she technically he called my wife.
Thirty seconds. Sorry, H is SA.
Gotta go click.
And she called me
In tears.
We called him back and it was maybe sixty seconds this time, but
At that time.
No one knew much about this disease.
The gene for it was just discovered a few years before.
And until then.
People thought it was just a variant of muscular dystrophy or some other muscle disease.
So in SMA.
You're missing the key gene.
That makes a key protein.
That protein is like a fertilizer.
For muscles and nerves.
And like fertilized road plants, without it, they wither and die.
All of us.
have a backup gene because it's such a critical protein your body has some degunding.
Some people have one, some people have two or three. That backup gene doesn't work very well. It makes a protein that falls apart because it's got a defect in it.
But it has some.
And so
depending on how many backup genes you have and how little or more than little protein you have.
You might
Die within a few weeks.
Or he might die within ten, fifteen years.
Our daughter was in the middle, and so
The diagnosis of the time is that she'd probably live to be in her mid teens or so.
But getting weaker and suffering along the way.
So
My parents had been scientists as it happened.
probably a source of my strange thinking in life, but after school in India, I'd go hang out their lab and do mass dissection as I was six years old.
Yeah, it's a difficult actual activity.
But I mean, I grew up in the science world and so had
Enough scientific knowledge to be dangerous.
And so she stared at this, I mean it seems
Super
Intriguing to me. I mean in the first few months we was just devastation.
When you're told there's no treatment.
There's no cure.
It's just a question of when and with how much pain.
You're mentally clobbered.
We first start just giving some money to some groups, and as I started digging into the research, it became clear that
Huh, there isn't as much known about this as actually one would think.
given the gene was just found, for example, why did that you know just diagnose their
Hang up in sixty seconds.
Honestly because she didn't know anything about this.
When he went to medical school, there was no spinal atrophy because no one knew that it was a separate disease.
Let alone one that actually affected twenty thousand kids.
So technically actually at that time
the most common genetic colour of kids, which
Is a
Pretty incredible thing when you think about it.
Intrigue starts setting in. And so at first my wife and I started
Giving money to some
causes. There are a few groups that were involved and so we give a bit of money.
As we dug in more, we then started talking to scientist friends, Columbia, et cetera.
And
got more intrigued that there was a chance here because there's some really good work had been done.
And in your body.
If you're missing a gene and it's completely gone.
Replacing it when it's totally gone missing. I mean true gene therapy.
It's still really, really hard to do.
On the other hand, here
Do you had this back up, Gene?
That was defective.
But
having a backup factory in place that you can try and crank up and fix to make a bit better.
Is a
Wholly easier challenge.
Then
having to replace something that simply went missing in the first place.
And so
That struck me as being pretty interesting.
There was some work being done. You could create mice that had SMA, which is also a pretty big powerful factor.
You had a intriguing thing to tackle.
you had some tools that actually were pretty nifty tools in these cell models and mouse models.
So you could test things in them that had to see whether they had an impact or not.
And this is getting a little nitty gritty, but for a lot of drugs
One of the biggest problems is
What they call the third vic window, meaning
With almost anything in your body.
Too much or too little.
It's pretty bad for you.
And I joke to people that
Take water as an example.
Water has a very wide therapeutic window.
But by the way, if you drink enough of it, you actually get kidney damage to die.
And most other things are much, much narrower. And so there are many drugs that
Actually might work, but we just don't know exactly what the precise amount is and where where to get it as a result.
they end up having toxicity problems or undershoot or et cetera.
With SMA, the difference between some of these kids with multiple backups versus not.
And somebody who is a carrier versus having a disease or someone like you presumably doesn't have it all.
There's such a massive wide range of this protein in the body.
It's pretty clear that
You almost can't have too much.
There's no real worry about that.
And that really was a pretty treating thing from developing a drug as well. And so again, not to get too much in the weeds, but
Brand in these things and
It really seemed like
There was at least something to be tried here.
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 B these 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 it's this cool nifty new disease.
And so we think this is the best target. Would 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, to me this seems completely obvious.
Back then that was a really novel thing.
Unfortunately.
There were a bunch of changes to
Head of an age.
step down and the head of N I N D S, 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 was 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 um
Latency bias and things. If you think about this.
If you're a student in medical school
And a disease is well known.
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.
I was just curious and said, Look.
There's a chance there.
But
We might be waiting.
Couple decades or so for this to work.
By that time.
She's gonna 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.
So
is to
See your child suffer or die.
We need to find out.
Right.
You could have done something about it.
But it wasn't done in time.
And so
Once we got the idea in our head that there's a glimmer of filter.
It really almost stranges you, if you will.
Because then.
Any second year spending doing
Anyway.
That isn't max speed.
is a wasted moment.
And so that led to just
We're gonna take a shot of this.
I'm not gonna go.
And take a shot.
Only to find out we went slowly.
So this has to be
Max Turbo.
Pull all stops, let's go for it.
Part of that was money.
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, to the next one, 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, F, G.
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 was sort of motor neural institute there with someone who wanted to fund
A LS as well, and they're quite similar to diseases in terms of some of the impact.
But it occurred to us that
Academia, while very important, was never gonna go and develop a drug.
And so we really had to create a virtual company.
So thought then was hire some scientists.
get some amazing advisors together to really help be our brain trust.
And then sit down every so often with ever and say, What do we need to do to go and 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 didn't 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 hope.
the world's most expensive drug habit. 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 dollar.
In a way that if I waited five years or three years or two years, it wouldn't.
The axon name routing back to the long answer, very short question.
Was really just there to remind me.
Um
what the mission was. And so it was always amusing because people would sort of wonder and I explain, Well, you know, the axon is the connects muscles and
Nerves and brain. And so
Cutting thought to action.
And I thought that was a cool analogy.
from a just general perspective. Every now and then you get a scientist or a former company going in and he would see our logo, which is actually the shape of an axon.
And would get the fact that this was actually about medical research and stuff, which is always kind of cute. It's a bit of an Easter egg that we sort of hit in there.
But look, so that was so 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.
T P G was my partner.
And 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.
And I think for all of them, what was extraordinary was how much of their time and energy they're willing to commit. Obviously we weren't paying anyone.
So we'd get
A dozen and a half of the smartest drug development people in the entire world.
coming together for a couple of 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 in your logical 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 interesting plan here.
Maybe it will work, maybe won't.
And there's someone willing to write the check and fund this. So it's not about going and begging for money.
People I think were excited by the idea of joining in.
to go and figure out whether we could actually go and solve disease. One first stop was look.
There were some scientists that 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 cell models of different types to test different versions and each has pros and cons.
And so 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 library is in.
Test'em for free.
We'll pay for it.
You keep the IP.
We just want you to know if there's something interesting here on stuff.
And
Again, because no one cared about rare disease, let alone a pediatric one at that time.
It took years before any company
even for free was willing to do that, which is crazy.
Finally we've got Navartis to bite.
And they ended up finding a drug that had
failed for talk reasons for anorexia or something back in the seventies, that was off the charts good.
And so suddenly like, oh, this is interesting.
Do you have 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, What 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.
Don't have to spend 40% on marketing, all that kind of stuff. And so the one of my slides show that if you charge five to 15,000 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, you can 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 drug in the world.
is Vastin, which was an ecology drug.
at fifty thousand dollars a year.
No one's ever gonna pay that kind of money.
Or
A small rare disease for children.
became a running joke in the end because
This person outside Iraq was an incredible advisor and helper to us.
joined our S C B and really helped us really immensely.
At the end, the first drug that we helped develop
through culture mover 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 to market with an SA drug. And when they did, they charge I think seven hundred and thirty thousand dollars a year for it. I think that's one of the top three drugs pulling in I think four or five million dollars in revenue.
And so
Amazingly, today the SMA drug market, I think it's highest in relation billions.
And it's a top three drug for rotion of artists and biasen.
But
The notion that a pediatric neurology drug
Would be
Anything of value back then was a hard sell.
And so 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 the existing F J approved drugs
Some would have some impact, but none would be good enough.
And you have toxicity issues, so it didn't work.
So we then pursued all three shots on goal that we thought were relevant.
Close from Harbor Labs and a great scientist.
that had what's called an anti sense oligos of bio with the first biologics, essentially
The viral vector takes a protein to body.
And it was designed to patch that defect in their backup gene.
And help that backup gene become a fully functioning regular gene, which should be magic. Presto.
That became the first drive the when the Bay ended up buying in.
Second was
Good old fashioned small molecules.
Sewages and pills.
A bit like Navarda's, for example.
And found something that works.
We basically paid a company called PTC Therapeutics.
Lots and lots of money to 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 pay to develop that.
And then actually got Roshan to go and take it from there.
That's actually the drug my daughter's on now.
In between was gene therapy, which a company called The Vexis did and then Navartis bought in for eight billion dollars.
And I guess this would be the first gene therapy drug ever approved.
Here we
didn't support it as directly from a financial and other perspective.
So
Our board members were active investors and we help them with the network and the library and the local trial networks.
our focus just being selfish to some degree was that
Gene therapy is great, but only works for very little kids.
Essentially in gene therapy, you're taking viruses from bringing protein in your body.
And when you're large
that amount of virus will kill you, basically. Toxicity becomes an issue.
But we wanted all three shots on goal to be explored.
Look, we got lucky.
Today we have
All three is approved drugs.
So the first was libogen cells.
The second was gene therapy with the Mexicans, and the third is Roche.
Which is just a sort of she take every day.
That was incredible.
Luck, I suppose.
But part of what we did from a time perspective because 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 ninety miles an hour.
And so, for example
We went to FDA through lots of relationships on the political side, both to get NH moving, by the way, in the early days.
And to help later on.
Yeah.
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 where he 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 because it might be fixable. And I think that became a powerful combination.
It took some work.
We, for example.
Got fifty top scientists who are 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 color ads 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 roomer add 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
Well
End of the day develop the drug.
matter and getting companies involved would matter.
Everyone move faster if they thought that the other guy was there too.
And with the FDA as an example, that really helps.
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. It doesn't make any sense.
And so we work with them and got their approval to essentially run a natural history study, meaning while the drugs were being developed.
We basically paid for people's care.
For 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 for the trials
The trials didn't have to be proceedable on trials, essentially because the FDA was willing to accept the fact that they'll compare the results in the trial.
to what were the natural history study results of kids normally.
With us on that.
That saved a lot of lives and a lot of money because otherwise half the kids would have died in those trials.
We could 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 and maybe still now the fastest approval by the FD ever in history of any drug.
Wow.
Which is pretty cool.
The good news, bad news, of course.
My daughter.
And
Being on the first strike since you swallowed.
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 eleven awards for most outstanding student.
Class speaker.
You name it.
And during Covid, amazingly.
Went on hinge and
Met up.
With this unbelievably amazing
Sixty four fantastic princely 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 in the family, but just watching her
Right.
Has been remarkable.
That said the clock.
Was the clock.
Every year she got weaker.
Until the drugs came along.
And that's say 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. 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 dissociation or it's choking.
Or it's
Pneumonia more often than not.
Valcomer just yesterday of pneumonia, but
pneumonia was really just a side effect of throw cancer.
In her case
Our goal was to manically
Keep her as 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 just 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. They're weak, you can't exercise and stretch your muscles and bones, 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 needing his final fusion.
And having rods put on her back, et cetera. But fortunately those
What
Late enough that she had already grown and so didn't create the deformity in really seen someone else. She had problems with her hip that blew out and things like that.
When she who was turning ten eleven 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 a 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 expected at the outset.
To see her.
Here and a rock star.
I'm truly happy with amazing friends.
And how it's just So touching.
I describe to people if you have a vote
And you have a corner.
Trying to build water out of the boat is not really gonna be a useful strategy.
You're gonna sink just maybe a tiny bit slower.
Once you fix that holes.
And now there's water in a boat.
Now being on 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.
Nerve 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.
twenty thousand kids, so it's a lot of people, but it the recipe 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
Take muscle as an example.
I assume that we'd be tons of people and experts that knew all about muscle development from muscle, stem cells, pulse satellite cells, into muscles, et cetera.
There are maybe 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 got A rod 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
Most of all.
People with degenerative diseases like SMA.
Muscle dystrophy, etc. On the other side.
Old weekly.
Oh week people.
Or 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 degenerating parents.
And in that first category, people with muscular disease.
Until SMA, 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.
S is now becoming this really fascinating
Yeah, because suddenly if you have an idea for a muscle drug
You can actually
Test an SNA 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 twenty years ago when no one cared.
Now suddenly
We're calling out people saying, Look, we think there's an idea.
Your drug 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
Cute S May and other things as well.
That would be amazing because as amazing life as the 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 impactful.
So it's exciting stuff.
the third time I'll say it, one of the most remarkable, inspiring, incredible
stories I've ever heard of this type.
And I'd love just to put a bow on it by asking what the
prognosis is now for a new child.
Born.
with this issue.
with these drugs available from a very early age versus from the age of twelve when your daughter started on them.
So a child born and getting
The drug.
close diagnosis will never show symptoms.
Unbelievable.
Twenty thousand kids.
Yeah.
Two thousand 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 who lose their jobs because they keep taking time off and they can't afford insurance. I mean, it's just this horrible spiral.
There is a family of
The father worked at Walmart.
They had twins with S and A and we just wanted to cry hearing the 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 of Goldman Sachs.
In some ways.
This all came the full circle.
having some of the science background.
Than having the good fortune.
Good luck and privilege.
Of working in investing in finance.
helped us to go and actually
Put that science to work.
A further amazing irony.
Columbia was our leading center in all this and my mom had worked on faculty Columbia.
And Roche.
makes the drug that my daughter's on and my father
got a job at Roche and as a Roche scholarship that paid for my dollars.
Unbelievable.
Funny how it's ended up being a circular process, but cost a lot of money. I think we spent probably 150 million dollars our own money on this, but in the scheme of things.
The commitment.
That we made as a beginning to ourselves.
Was that?
If there was a way.
We're gonna 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.
When I step back.
I've now had the luck to hear the story.
twice.
When you told it to me the first time
It seems almost absurd.
If you submitted a screenplay for this
you'd get laughed out of the room by the producer. You're gonna start one of the fastest growing
investment firms, one of the most successful investment firms.
Save your daughter, save twenty thousand kids.
develop a drug which just economically is very valuable.
to say which is a footnote compared to the impact it has on the lives and the families. It's almost not
believable. It's so incredible. It's such an incredible story.
What you didn't say the first time, which makes it even more magical is the tie back to your parents and their persistence.
And honestly, it's just one of the most incredible inspirational
Stories I've heard.
And a follow up question that I have is
What you learned, maybe that you've taught other parents about.
Persisting through
Years of
This kind of effort.
And
What advice you might give to people that
Have to dig deep.
Every day for so long.
to be able to accomplish the thing that they're
trying to do for a loved one or
For a similarly powerful reason.
Because it's not hard for me to imagine the love.
For the child.
But it's hard for me to imagine the ability
With my body.
To get through it for so long.
And I'm curious how you did that.
Like two or three. We're getting into nursery school and
By the way, another saga and another crusade that I'm gonna
beyond more aggressively going forward.
Yeah, the
Getting schools to care or taker was
Unbelievably hard.
When she was going to private school, I think she was
The only kid in a major New York City private school in a wheelchair.
And I kinda know this as well because
I'm on lots of boards.
Public library, lots of inspection boards, lots of eventual friends.
Lots of charity relationships.
I could find a single school.
That would take her.
Many
Or perfectly accessible.
But I get calls from friends on the board saying with Jenny, it's just not gonna happen.
The stensible reason I think
One school said she
Wasn't bright enough.
Crazy.
Turn out to be maybe not bright enough for them, but bright enough to be straight as at yellow and all that kind of stuff. We had one school actually asked.
To see her medical records and talk to her doctor.
Oh my god. I mean, guys, you know that's absolutely fucking illegal. But we had to do it because
It might be illegal, but
Unless I tried I mean so when she would start in school.
We would send up
right 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 interior insurance school.
And initially they didn't want to take her and they said, look, there's great surgical policy schools you could send her to. Like
Yeah, that'd be great, except she doesn't have cerebral palsy, 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 screen to a good
Please.
Most of you probably never read SMA. We certainly hadn't, and we're horrified that we weren't.
There's the most common genetic killer of infants and it's untreatable.
Incurable and eventual.
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, has to make kills in the first two years of life.
Well, that's your cases.
such as our daughters.
Depression could take many years.
So as the mind grows, the body steadily weakens.
eventually betrays it.
Obviously we were devastated and shattered.
Over the past year.
Our hearts have broken.
Every day.
As we've watched her become irresistibly adorable.
And I'm just saying.
Tell jokes, talk endlessly.
But also lose the ability to stand up.

I wouldn't walk.
The ability to even sit up by yourself.
And yeah, three realizations have helped us totally face the future.
First was truly learned and appreciated.
The importance of friends and community.
The comfort and love of friends.
has been a source of incredible strength for us.
and getting other parents with issues.
And learning from them.
And incredible shounders many of them do us.
That's been an incredible source of inspiration for us.
With reality there are no good diseases.
But they're many, many strong people.
And their courage is truly infectious.
Far more than they need to see.
More than ever.
We've come to realise how much deeper our reserves of strength are as part of community.
Than as individuals.
Second, the strength, comfort, inspiration of our friends, we've gained some perspective.
And be able to focus on finding the joy.
In the life that we have.
We slowly realized.
But if we just hold our heads in our hands.
We're accomplishing nothing.
Your daughter?
ourselves but
And first, it's hard not to be consumed by the shattered dreams.
I lost notions of what could have been.
Get over time.
We've come to focus on the many good things that
Can be.
And realise that there is
Join every day.
If anything.
We're now much more focused on appreciating the little things in life.
And today, despite all the issues.
Our daughters is 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 learn from our dog.
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 nettle of anger.
And sorrow into productive channels.
So as to perhaps have some good.
Some purpose, some meaning in all this.
And so joined us other parents of
in efforts to improve care.
And further research.
But this is me one day.
Eradicated.
In doing so
We found a productive outlet for our sorrow and our anger.
And also a real reason for hope.
And finally, let me just say how grateful we've been be part of such a wonderful community.
The strength and comfort offered by many of you has helped.
Let's focus on life.
No, I'm sorry.
After all.
In life.
So no doovers.
No retractions.
There's no appeals.
You either find joy in the life you have
We're living 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 the speech.
Wow.
Jesus.
But I 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 more 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 gonna get short.
under any circumstances. So
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 your 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 of weeks I'll get a call.
From someone
Saying look, I've got a friend or someone, etc. who has the 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 the parents and helping them think about what they can do, what can be done, what can't be done, et cetera.
I joined the board of Jackson Labs. They essentially invented mouse models and are still the leader of 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 instrument 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 then send the mice over all the
But I'm working with them and setting up actually a very use instant.
We're gonna 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 twenty 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 and sand.
They don't know how. Like in our case.
We had a lot of resources and a lot of connections and money and can essentially go and 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 goalers actually have they call it the Ghostbusters.
of air disease where
If you have a disease, you call us.
And we'll actually get some scientists to work on it to go and help you put together a planet.
Not carrying whether it's with us or not, but essentially helped be the brain trust for people to want to go and think about game plans for trying to solve disease that affects something that they care about.
How do you hope the
world of biotech.
evolves or changes given your incredible ground level experience
Not only just knowing how the system works and also
the extreme steps it took for you to speed up timelines.
But also knowing the potential impact can be so massive.
Twenty thousand kids, no evidence.
no symptoms in their life because of your work and everyone that you worked with in their work.
How do you hope with all that experience and knowledge that
The system itself evolves.
Well, 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 somebody over the head to get them to understand that urgencies can be
lucrative for them.
I mean SMA has been there aren'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 gonna be working 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 twenty 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 thought effort working with NIH FDA.
and disease groups.
to find ways to go and have a much more thought, practical early stage, 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 gonna go and spend the money and do it.
You don't have to get it to the ten yard line anymore. We still have to go to the midfield.
And so I think more novel structures would actually be great.
And I actually took back to that.
N H translational research program from way back when.
Well
It didn't end up making that much of a difference in SMA because we just spend up throwing the money out and going in ourselves.
It's a pretty cool idea.
And if one could do that.
Again, it's that Ghostbusters idea that I'm doing a Jackson Labs.
Because the problem is
We found in 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 let's say parallel processing or ruling things out. You don't get a grant by saying, I don't think this is gonna 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.
And so whether it's places like Jackson Labs, whether it's NH.
having these amped up translation research efforts where
People can set aside a little bit of money because honestly that early stage doesn't cost that much either.
it's later on that you really start going down bloody 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.
Well you think about obviously the the flavor of the day is Terra Garden Down.
Government did a pretty good job in some of the stuff.
And
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 here to be played that could be pretty thoughtful where
A few diseases were picked at a time.
And a modest amount of money is put in or coordinated so that you actually end up having a game plan. Cause 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.
And companies really don't like flattering 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 worldties and stuff like that. So having a third party that's sort of Switzerland.
And can be a bit of a honest broker on the stuff. It's 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 then had to bring their CFO in.
Because
Since Columbia was a major center, you could fill in Harvard or Stanford or whatever as well.
The S B drug cost.
eight hundred thousand dollars here 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
For example
There's a
Goldman Sachs analyst who is 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 rubs.
And of course then you gotta 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
It's a whole regulatory framework is crazy.
And so there are still kids dying of SA.
Even in cases in this case, for example, the family had gone around through supermarket stuff.
meaning cans and signs in town raise four hundred thousand dollars 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 show she was gonna die.
Or suffer.
Unnecessarily.
Because of crazy irrigatory bureaucratic stuff between countries.
We gotta figure out something on the cluster. Maybe it's
With these drugs having some amount of money get set aside.
When there were these windfall profits to go and help
charity care or things like that. But the notion that we develop these drugs and they're kids that are dying because they can't afford them is pretty insane.
It's hard for me to ask my traditional closing question in this particular case.
because of the story you've just told and
Knowing that
Contained in the story is so much kindness in so many directions.
that I'm sure it'll be hard to know where to begin.
But my question that I ask everybody is
For the kindest thing.
that anyone's ever done for them.
After a car is diagnosed.
People just giving me hugs.
Was about the best feeling on earth.
There's nothing more powerful than just
Compassion.
F
Friends, strangers.
Just uh
feel like you're all human together.
I think in life had our worst moments.
Just getting some of the
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 time.
I'm sure I speak for literally everyone listening when I say
Thank you for telling the story. You've imbued us, certainly me, with that same strength and determination
It's incredible what you've done already. I'm sure what you will continue to do.
I love hearing about
your daughter's current state and her wonderful marriage and
What a way to spend my day. Thank you so much for your time.
First.
Thanks.
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