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Bob Daniel Levine and this is Rarecast.
Cleafster Syndrome is a rare neurodevelopmental condition with significant unmet medical needs,
but a growing understanding of its underlying biology is creating new opportunities for therapeutic development.
Eric Sheef, Chief Scientific Officer of I Define and Parent of a Child with Cleafster Syndrome,
discusses the organization's progress in building the scientific foundation for a potential treatment,
the role of its patient community and the work ahead to translate scientific insights into meaningful therapies.
Eric, thanks for joining us.
Thanks, Danny, for having us.
We're going to talk about the ultra rare neurodevelopmental disorder Cleafster Syndrome.
I define and the organization's efforts to advance treatments and cures for the condition.
Let's start with Cleafster Syndrome, though, for listeners who may not be familiar with it, what is it?
Yeah, it's a rare neurodevelopmental disorder.
It's caused by damage to a single copy of a single gene called the HMT1.
So it's always caused by a spontaneous genetic change, either a mutation or a deletion of the gene.
So it doesn't run at families, it's equally distributed worldwide.
In terms of how many people have it, the best data, the best modeling we have says that's about one in 30,000 people.
And so that doesn't sound like a lot, but if you do the math, that's about 10,000 patients in the US.
And it's about a quarter of a million worldwide.
So while it's rare, it actually technically is an ultra rare.
It is very underdiagnosed as with many rare diseases.
And so we are actively working to find all the patients out there.
It's also worth mentioning that it's what's called the haphaline's efficiency.
So for those who aren't biologists, this is just a fancy word for one copy is not enough.
So if you recall your basic biology, for most genes, we have two copies of each gene.
One of those you got from your mom, one from your dad.
And in a lot of diseases, if you lose one copy, you're fine.
The other gene acts as a backup.
But in this condition, if you lose just one copy of the HMT1, you have the syndrome.
Now the good news is the function of the HMT1 is actually well known.
So effectively, to keep it at a high level, it functions as a master switch.
And it controls when many other genes are turned on and turned off.
And so this loss of control leads to many of the manifestations of the syndrome.
So I think the important thing to remember about the syndrome is that we know the gene,
we know what it does, and that opens past to getting to a treatment.
And how does the condition manifest itself and progress?
Yeah, so there's a huge unmet need.
So commonly it's seen within the first few months.
Obviously, you know, often what parents will be the first to notice this.
In the first few months of life that their baby isn't reaching developmental milestones.
You see things like low muscle tone, feeding difficulties.
And while this is primarily a disorder of the central nervous system,
there also sometimes can't be physical manifestations that are seen,
things like heart and kidney abnormalities.
And then later in childhood, it'll manifest as minimal or absence speech,
difficulty communicating intellectual disability.
That's usually in the moderate to severe range.
Most of our kids also have a diagnosis of autism.
And so we look at this as an important autism model,
because although there's a larger sphere of people who have autism,
where it's often not known,
in this case we totally understand the genetics of why these kids have autism,
it's because they have cholesterol syndrome that's triggering it.
Another key thing you see is that a lot of kids are sleep disturbance,
so they're up multiple times a night.
Obviously that's really tough for their parents and for the kid.
And about 30% of our kids will have seizures.
And then I think really important to mention is that there's a critical,
period of risk that comes on during adolescence.
And that's that there's a risk that the children will go through regression,
which means they start losing a lot of their hard-won skills.
That can be accompanied by psychosis and really severe sleep disturbance,
so like the kids won't sleep all night long.
And obviously this is just devastating.
The family and the child have been working for years, going to class,
going to therapies, and they just watch a lot of those skills go down the drain.
So we are in a race against the clock to get treatments that,
partially just to stave off that regression.
And if we had a treatment that just, you know, staved off regression,
and that was all it did, that would be a huge win for the community.
I should also mention in terms of the manifestation,
there's a lot of variability.
This is a syndrome that comes in many forms depending on the person.
What I'm telling you is the average.
And there are people with chaos who live semi-independently,
and then suddenly there are also a few have passed away from complications of the syndrome.
Well, what is the prognosis for someone who's diagnosed with the syndrome
and are there treatment options that physicians use?
Yes, so right now there is no targeted treatment for cleftar syndrome
that addresses the fundamental cause.
So the way it's treated is the supportive type methods,
like physical therapy, speech therapy,
ABA therapy for a child with a diagnosis of autism.
And these are really important.
And it is absolutely critical that the kids get that early intervention
and it does make a difference.
And then, of course, symptomatic treatment.
So if the child, for example, has seizures,
you treat those seizures with an anti-seizure med.
As I discussed, the prognosis is quite challenging,
particularly the potential of regression.
And so these days we're just managing symptoms,
but we really need a targeted treatment,
and that's why we're here.
I think one thing that's also really important to mention,
one hopeful thing about the prognosis,
is that this disorder is not degenerative.
So we've been contacted by families of 40-year-old patients
who just finally got tested and got an answer.
So we have reason to be encouraged.
There's no loss of neural tissue, let's say.
And so we have reason to be encouraged
that older patients can also potentially benefit,
at least a sudden degree from a treatment.
You have a doctorate in biomedical sciences,
you're a co-founder and chief scientific officer of I Defined,
but you're also the parent of a child with this condition.
How did your child come to be diagnosed with cleavage syndrome?
Yeah, so we had the typical experience
where we saw a lot of the early symptoms that I mentioned,
and we highlighted them to our pediatrician.
And we hear this story all the time from parents
that again it's them pushing.
And so if you're experiencing this with your baby push,
eventually the pediatricians got around to understanding
that there was something going on.
And we got the right genetic test,
and that took about two years.
And that is actually a pretty lucky trajectory.
Usually what we call the diagnostic odyssey can be a lot longer.
And as you know, there's been a huge push
in the broader rare community to shorten that odyssey,
to make genetic sequencing more of like a first-line test
when there's developmental questions.
And we've I Defined has tried to contribute to that effort
wherever we can to spread the word.
And I think that is starting to pay off.
I think a lot of the families we now hear from
who just got their diagnosis, got it in at six months or a year,
so much earlier than we did.
And how did that experience reshape your own career path?
Yeah, so when I first started training as a biologist,
I always expected I would become a drug developer.
That is what I thought I was going to do.
But when I was in graduate school,
I got really interested in the field of bioinformatics and genomics
and I ended up building my career in that space.
And so then when we found it, I defined my background
made it natural for me to take on leading the research program.
And so it's amazing I've come full circle,
and now I am working in drug development.
And so in a sense, when I first started planning out my career,
I was right, it's just funny how life will do that.
And then obviously, you know, my background puts a tent great position
to work with our partners as scientific peers,
and not just as advocates.
I mean, I can even get into the weeds with them
and we need help answering a question on KS,
or you know, what's known, what's available?
When you got this diagnosis,
you did have the background to perhaps understand it better
than most parents.
What was the state of understanding of this condition
when your child was diagnosed?
So it was known that the condition existed,
but Dr. Clevstone, her team,
had actually just identified the actual gene that caused it.
And so we literally got our diagnosis
under that old designation,
and then the syndrome was shortly thereafter
named Clevstone Syndrome in honor of her discovery
of that causative gene of EHMT1.
And in the subsequent years,
her team, many other researchers in the Netherlands,
and a lot of researchers elsewhere,
elsewhere.
have been investigating the cellular and clinical aspects
of the syndrome.
And so our foundational understanding
is never going to be complete of CAS.
But thanks to all that work, we think it's
more than sufficient now to really start seriously
working towards treatments.
So we think the primary bottleneck at this point
isn't really knowledge.
It's resourcing and execution.
What gap did you and other founding families
have I defined see that wasn't being addressed
by traditional research or advocacy structures?
Yeah, so traditionally advocacy is really centered
around awareness and support.
And that's important.
And we do plenty of that as well.
But we felt there wasn't enough work happening
to get cleaves to syndrome rapidly
to clinical trials for a targeted treatment.
And that we were the right ones to do it.
And similarly, traditional biological research,
it's generally based around better understanding
mechanisms and so on.
And that's also really critical, as I just discussed.
But we wanted to be the ones who pursued projects
and focus on projects that could get to the clinic
as soon as possible.
And in terms of our search for treatments,
we very much see ourselves as running
like an early stage biotech company,
but we just do it on a nonprofit basis.
So we structure our programs.
We negotiate our contracts like a biotech.
We just aren't seeking to make a profit.
We're looking to help our community.
How did you decide where to focus limited resources?
And how did you prioritize what to build first
versus what to wait?
Yeah, it's a really important question
because it's a nonprofit resources are always limited.
And what we did is we looked at the landscape holistically
together with our SAP, our Scientific Advisory Board.
And then we thought backwards from the final goal
of getting into clinical trials.
And we asked, you know, what's missing?
What would make the biggest impact?
And so one of the things we did early on
was create patient cell lines that can be, you know,
so-called IPSE cell lines that can be grown into neurons
in addition, other cells of interest.
And lines were out there, and there was a characterized model
for cleafster and neurons grown in dishes.
And so they have a known phenotype.
But those lines were siloed.
And so we realized we really just
needed to make our own lines.
And that's been a huge benefit because we've
had tons of requests.
There was clearly a pent-up demand.
And so that was a big step forward, I think, for the field.
And then we also, when we were looking at this overall road map,
was we looked at how long each thing
would take going backwards.
And so for example, if you need something
to show up in two years, and it takes two years to make it,
then you know that's a top priority.
And then I think also we didn't just look at the road map
as like, well, this is how we'll get from point A to point B.
But we asked at each phase, what could block us?
What could stop us?
And then how could we do something now to mitigate the risk?
And so an example of that was that there's
already a cleafster syndrome mouse model.
So we're not going to reinvent the wheel and make another mouse.
But we might need other models down the line.
And so there was models of cells in a dish.
There's the mouse.
But we wanted to add in other models in case the mouse didn't
turn out to be the right model or the cells in addition.
So we started organoid work at UC San Diego, partly
to learn more about CAS, but partly just to mitigate that risk.
So there would be another model.
And that model would be there available for researchers
in the future.
And those organoids are being made using our cell lines.
So if someone wants to recreate those organoids in the future,
they have access to lines they need.
What's been the biggest challenges in getting researchers
engaged in the condition that previously
had very little visibility?
So we've really rarely had any trouble getting researchers
engaged.
And I think there's really now a very large community
of researchers who are genuinely interested in rare.
And I think the great thing, too, is that they're
now starting to develop a lot of generalized technology
that can help us in other similar conditions.
So there's actually quite a bit to plug into.
So I think the biggest limitation for us
is always having just sufficient funds, sufficient resources
to do all the projects that we want to do.
And so it's not the limitation.
The limitation is not technology or interest
from researchers.
And as you work on developing resources,
are there things you're doing to ensure
they're broadly accessible to the research community
rather than siloed?
Yeah, we always keep this in mind.
We structure everything to whatever degree
we can to be as open as it possibly can be.
A great example.
Again, as the cell lines, we make available companies
as well, they're not just available to academics.
So if a company wants to start researching KS
and look for a treatment, then the lines are available.
And I should mention, if you're a researcher
and you want to look at what's available,
you can just go to our website, idefond.org.
There's a section there that'll get you started
with what resources are available for you.
We also, of course, we expect our researchers
to publish their work.
And we hold a yearly conference for everyone
that works in the KS space, whether they're funded by us
or not to share all their latest progress
and keep the whole field connected
and communicating and moving forward.
Natural history studies are often a turning point.
Did you learn anything that surprised you from yours?
Yeah, so first I would say I would split natural history
into two buckets and we're chasing both of those.
I would say that there's one group
which is the conventional prospective clinical studies
and those are, I think, the gold standard and really important.
But there's now also these digital approaches
that are more retrospective, like Rarex and Citizen Health.
We think both of those have a lot of value.
The prospective approach gives you really high quality data
and the retrospective approaches can really scale
because you can have a lot more patience
than you can in a prospective trial.
So we started our prospective natural history study
about a year ago.
So we're now a year in.
We're just about to start reading that out.
But we've already learned a lot
from that retrospective data that I mentioned.
So my biggest takeaway from that is that no matter
how much you think you know about the syndrome,
I have a child with the syndrome, I think I know it, right?
But there are aspects that will allude you
until you really study it comprehensively.
And so Rarex recently did an open science data challenge
to crunch the data that they have in Rarex.
That includes our data.
And when they did that, they came out with,
OK, here are the manifestations for each syndrome
that are high up in our list, but haven't really
been reported and what came out was constipation
as a very high burden.
If you look up the clinical descriptions
of chaos in the literature, it's really not prominent.
But it came up very high in Rarex.
But if you look online at the forums,
like on Facebook, people are mentioning it all the time.
So by being formal and comprehensive,
you really get insights, I think, into the syndrome
that you wouldn't, if you just say, well, we know it, right?
So you have to do that work.
I think that's what you learn.
And I think all this data together
puts us on a really great path to design
a clinical trial in the future, both the prospective data
and the retrospective data, and we expect to combine those.
Which makes me wonder, what role do families
play in contributing data?
And how do you maintain long-term engagement?
We always tell the families that we cannot do any of this
without them.
And in the case of these types of natural history studies,
we truly cannot do it without them.
And their response has been absolutely amazing.
So we do multiple things to basically stay
in touch with the community, gather information.
And one of those is we have a worldwide registry map.
So that's now got 1,100 patients that we can make contact
within the future.
And then we have more than 170 participants
to citizen health, more than 120 in rarex.
We're growing a presence in Simon Search Lite, which
is another one of these virtual natural history platforms.
And that's a lot, right?
That's a lot for all the families.
And it is challenging to keep everybody engaged.
And some of these resources involve filling
a lot of regular surveys.
And there is such a thing as a survey fatigue.
And that is real.
It's really been for me.
And so what we try to do is communicate
the benefits of all the work they're doing
and make sure they understand the impact they're having.
And now that we've got the data collected
as we're starting to read it out, we're really going
to make a strong effort to actively read out those wins.
Like I just mentioned in the rarex study
to the community, so they know that their work is really
creating a benefit.
I define recently announced to your research collaboration
with UT Southwestern Medical Center
to advance development above potential EMT1 gene
therapy for the disease.
The program's led by Stephen Gray, director of the UTSW gene
therapy program.
Someone will be familiar to many rarex listeners.
He's a leading CNS gene therapy researcher.
What's the case for an EHMT1 gene replacement therapy?
Yeah, as you mentioned, Steve doesn't really need
introduction.
And we were really honored that he
wanted to work with us on this project.
So the way I explain this for the nonbiologist
is using the analogy of a gene being like a factory.
So in a normal cell, there are two EHMT1 factories,
but in KS, remember, one of those factories
has basically broken down.
It's been shuttered.
So you're only getting 50% of the normal output of EHMT1.
And so with gene therapy, there's
the possibility of basically delivering
a brand new factory to the cell to make EHMT1
to replace the shuttered one.
And done right, you can add that back
and get back to 100% of the normal output.
And experiments have been done already
and mice with reactivating EHMT1 postnatally.
And those have been promising in terms
of improving the mouse phenotype, improving the cellular phenotype
in the brain.
And so we think it's really worth trying this
and seeing if we can do it in mice.
How big a milestone does this represent for I define?
Yeah, I always try to be cautious when
I talk about projects, but I think this one is really huge.
I think it puts a marker down that we are now positioned
as a community to run very quickly
towards potential treatments.
And I think it's also just really exciting
because gene therapy is not easy.
and I don't want anybody to think it is,
but I will say the foundational methods, I think.
you're getting more and more refined, and I think especially in the hands of someone like Steve,
I'm quite optimistic that this project will at least tell us that this is likely to be a promising
path for us, and that alone is really crucial information. Are there other therapeutic approaches
that I define as exploring or are considered promising for a cleavage just in general? Yeah,
absolutely. We're doing a lot. So again, we think like a biotech, and so our aim is to create a funnel
where we have multiple parallel projects. You don't want to do your project seriously, where you
try something it doesn't work, and then you try something else. We want to do this all in parallel
so that we can run them all side by side. Through a funnel over time, some are going to show promise,
some are going to fall by the wayside, but the idea is that one or more of those are going to make
it through the funnel and get progressed all the way to a trial. So, you know, going back to the
foundation of the syndrome being caused by HMT1, which is a master switch. So what we do is we split our
funnel into two big sections, and one of those is projects that will directly address the shortage
of VHMT1 in cells, like for example gene therapy, and then projects that will try to deal with all
the knock-on effects of the shortage of VHMT1. So an example of the first type of project, which
is addressing the shortage of VHMT1, is a project we started with Brian Dickinson's lab at the
University of Chicago. So he's pursued what's called synthetic biology. He's pioneered basically
stitching together different pieces of other genes to make these amplifiers that effectively turn
up the activity of a targeted gene. So going back to that factor analogy, remember that everyone with
KS still has a perfectly good factory. They have one good gene for HMT1, and so Brian's approach
tries to turbocharge that factory to make more HMT1. So hopefully get back to a hundred percent
expression, but just using the one copy that's there in, in every patient. And then going back
to your projects that try to deal with the knock-on effects. So a good example of that type of
project is that we're doing a repurposing project with unravel biosciences. So remember I mentioned
that HMT1 regulates a lot of other genes. So unravel technology is really well suited to us because
it looks at the dysregulation that's occurring in a rare disease, and then it builds a computational
model based on all the data they have in their database of existing drugs that are already available
on the market that could push those regulation patterns back towards what's seen in the control
individuals in the study. And so they can use the technology to screen tens of thousands of
molecules, generate a list of candidate compounds, and then we can potentially take those leads into
model systems and validate if they're going to work. And I should mention too, there were always
evaluating additional projects for both sides of the funnel. And so if you're listening and you
think you have something that we should add, then by all means reach out to us.
How do you think about prioritizing targets or mechanisms when the biology is still being
actively defined? Yeah, so we had the good fortune, as I mentioned before, that a lot of work
has been done over the years on the fundamental biology of KS. There are always questions remaining.
And so the way we look at this is we always ask what's the fastest way to determine if something
is going to work. Again, we're very treatment focused. So maybe there's an experiment that needs to be
done. Maybe there isn't. In the case of gene therapy, our answer when we looked at it was try it.
The best experiment is the direct experiment in this case. And I would also say it's not always
either or, again, talking about the organs I mentioned earlier. Our primary motivation for
having those created in phenotype was to establish them as another screenable system on our road map.
But you also will learn a lot of fundamental biology about KS by doing that work. And so I think
sometimes you don't have to choose. You helped establish a dedicated center of excellence at
Boston Children's. What is that enable that didn't exist before? It was one of the first things we
did as a foundation and it's still one of the most consequential. So Dr. Cleats Trust team had
already established a center of excellence in the Netherlands. But there just wasn't a similar
resource in the US. And we felt like it was essential to establish one of those for two big
reasons. I mean, one of them is we felt like families needed a place where they could go that
immediately knew the syndrome could take a multidisciplinary approach. Many rare families have this
experience. They get their diagnosis and the doctor literally says, you are the first one I've
ever seen. We wanted a better option for families. And then it also figured early and literally
puts KS on the map. When you have a COE in the US, it ensures visibility for KS. It means there's
a substantial wear control characterized cohort. So we now have 120 patients have been seen in our
COE at Boston Children's. So they've all been seen by the same team, right? They're all known to
that team. And that puts them in a great position to more easily take part in future studies. And
our natural history study, of course, is being run out of the COE at Boston Children's. And it
was easy to recruit for that study because there's already a lot of patients who are familiar with
that team and have visited the center before. Also, when you have a COE, I think you get a lot of
other halo benefits. So it was instrumental, for example, in getting ICD-10 code for KS. So KS
now has an ICD-10 code and it's a recognized medical condition in the US medical system.
How important is clinical standardization and preparing the field for clinical trials?
It's hugely important. It's off and a real rate limiter in terms of getting all the way through a
trial. And we've heard, you know, I think we've all heard horror stories of clinical trials where
there's the patients say it seemed to be working for them and then the trial reads out and they say
it failed. And what was what was the gap? And it looks like a lot of times the gap was the end
points were good. And that's exactly why, you know, we've started the COE early. We started
on the natural history work early. We want to make sure those end points are fully ready when
that therapies are ready to be tested. And how close are you to being clinical trial ready in
cleaves true? Yeah, we're on our way. We knew we need to start the prospective study right away
because again, it takes three years just to collect the full data set, you know, we're a year in
and we don't want to have something ready, like I said, and then ready to go to trial and then
start the study, right? So we're on track. And at this point, the biggest challenge is going to be
making the most of all the data that's being collected, locking in the best end points. And that's
will be actively chasing in the next couple of years. And I should add, we recently convened a
new clinical advisory board to help us build the projects and design the projects we need in our
road map to make sure those end points are ready. So this is something we're really actively
working on as we collect the data. So I define as part of a broader movement of patient
letter or parent led research, what do you think this model gets right that traditional systems
often miss? I think it's becoming clear that the patient led efforts can really be more efficient
faster than conventional pharma. And I would attribute it to a few factors. I think for us,
there's just genuine urgency. It is truly a passion project. We need to treat it in yesterday.
I think there's also a complete focus. We don't change tracks because, you know, to some other
condition because the market likes it better or there's some trend in the industry, we're just
all in for police to syndrome. And we can run also in a really lean way as a nonprofit. You know,
we're kind of forced to cut away the fat focus on the highest impact work. In our case, you're hearing
about the projects we've gone forward with, but we've reviewed others and we've determined the
probability of success was too low. And so we're very ruthless about chasing only the high
probability projects for getting to a treatment lead. And are there places where this model can run
into limitations? And so how do you navigate those? So scaling all the way to an approved
treatment, I think is still a huge challenge. So we're intentionally building each of our programs
in a way where we could hand it off to a commercial partner at the right time to take it the
rest of the way. And we see that handoff is a really important step because commercial
partners provide the scale and the financing necessary to get a treatment all the way to patients,
which is ultimately what we're what we're here for. And that said, I think it's interesting. There
are other groups who are similar to ours, but they're a little bit ahead of us and they're staying
on a longer than usual nonprofit track. And so we'll be monitoring how they do on that nonprofit
track and see if that looks viable versus the usual handoff to a commercial partner.
Are there things you've been able to do, things that would de-risk drug development for a commercial
partner that you think have have made it more inviting for companies to work with I define?
Yeah, I think, you know, everything we're doing is all about de-risking both for
commercial partners and for other and for researchers in general. I think it's about
making sure the field is seated in a way where everybody can get started. I already mentioned
the cell lines, you know, for I think a lot of times those cell lines are very hard to get for
companies, but in our case, it's very straightforward. If they need them, also we found, you know,
in the case of just researchers, they've leveraged our cell lines. For example, there's a team now,
UCLA, who are making KS organoids alongside the one at UC San Diego, and they're doing it simply
because they were able to get the lines. So it wasn't a project that we had to fund. And I think that
process is similar for companies. You know, we're doing work to validate these leads early on a
nonprofit basis and get them to the point where, as you said, you have to de-risk and they may be
willing to license that program in and take it on approval to commercialization.
And have you noted a shift in how industry views patient organizations over the past few years?
Absolutely. And here I would give credit to those who have come before us like the
Angelman and Red Syndrome communities. So they've really proven that the patient groups can do
work that leads to viable treatment candidates. So I think now the Saudi industry players
see patient groups as important potential partners in drug development.
and not just as helpful when you need to organize the community.
What advice would you offer other rare disease families who want to catalyze research in their own communities?
So I would say realize that as a rare disease community, no one is coming to save you,
but if you will do the work yourself, many, many people will help you.
And so what I would do is first start by finding your tribe, meaning the other families who have
someone in their family with the same condition.
If there's already a patient advocacy group, then reach out to them, figure out how to help them.
If there isn't an advocacy group already, then you're up.
Reach out to any other families you can find and start putting one together.
And you will be amazed how people will come out of the woodwork and how they will have skills
that will allow them from their day jobs to help contribute to your efforts.
Then the next thing I would do would be to attend one of the rare disease advocate boot camps
that are being hosted out of ultra genics and the rare epilepsy network.
Both of them are holding these boot camps and those are absolutely transformative.
We went to one of those at ultra genics early on in our development.
And it was it changed everything because they take you through the entire process all the way from
early research to approval so that you really understand that how you need to build that entire roadmap.
And then, you know, I would say finally reach out to the other rare groups who are a little bit of
head view because we've all been helped by those before us.
And so we're all interested in paying it forward by helping others.
So that's a good start just to start.
You'll have a lot more to do, but that'll get you going.
Eric, I define began it in 2020.
It's a remarkable pace of progress.
Anything else you'd add about what's allowed you to achieve what you've been able to so far
and and where people could go to learn more.
Yeah, I think, you know, the number one thing that's helped us to succeed is our community who have been amazing.
And I think the, with this exciting about Cleef's syndrome right now is that we understand the biology
well enough to really start acting.
The roadmap is ready, you know, we just need to, we just need to execute on that roadmap.
So I just wanted to make the invitation that, you know, if anything you've heard today,
makes you want to join us or help us on our journey, please reach out.
So we want to hear from you so you can reach us at iDefine.org.
That's the letter i and the word define.org.
Or you can simply email us at the word unlock at iDefine.org.
Eric chief, chief scientific officer of iDefine and parent of a child with Cleef's syndrome.
Eric, thanks so much for your time today.
Thanks, Danny, with my pleasure.
Thanks for listening.
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