September 30, 2025 • 30 mins
Rare diseases do not receive enough attention for the impact they have on patients and their families. This is what AlphaRose’s founder and CEO Casey McPherson learned after his daughter was diagnosed with a rare condition. In a conversation with host Ari Berman, McPherson shares how he switched from music to founding his own pharmaceutical company, what technologies AlphaRose is developing, and how his company is creating environments that allow innovation and iteration. He also shares his thoughts on embracing failure in order to make progress and emphasizes holding compassion and understanding for the patients and families that are impacted by rare diseases—because you never know what it’s like until it happens to you.
Bio-IT World’s Trends from the Trenches podcast delivers your insider’s look at the science, technology, and executive trends driving the life sciences through conversations with industry leaders.
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Episode Transcript
Available transcripts are automatically generated. Complete accuracy is not guaranteed.
Ari Berman (00:01):
Welcome to the Trends from the Trenches
Podcast, co-produced by Bioteamand BioIT World.
I'm your host, Ari Berman, CEOof BioTeam.
Today we have a trulyremarkable guest who has
navigated the worlds of musicand science with an incredible
purpose.
He's a guy I'm lucky to call afriend, and his story is one of
artistry, fatherhood, and arelentless fight for a cure.
(00:21):
You know him as a captivatingfrontman for two incredible
bands, the anthemic indie rockgroup Alpha Rev and the
progressive rock super groupFlying Colors.
He's brought us songs that havetopped the charts and filled
stadiums around the world withinstantly recognizable hits such
as New Morning and Sing Loud.
But beyond the stage andstudio, our guests' most
(00:42):
profound journey began at homewith his two daughters, Weston
and Rose.
When his youngest daughter,Rose, was diagnosed with a rare
debilitating genetic mutation,HNRNPH2, which encodes a protein
that belongs to theheterogeneous nuclearibro
protein family and plays a rolein RNA processing and transport,
his path changed forever.
What he discovered was aheartbreaking reality.
(01:04):
While there are over 10,000known rare diseases affecting
millions of people, as reportedby the National Center for
Advancing Transactional Science,most of them, including this
disease, have no treatment.
Refusing to accept that fate,he channeled the same passion
and creativity he poured intohis music into a new mission.
He founded Alpha RoseTherapeutics, a biotech company
dedicated to finding a treatmentfor Rhodes' rare disease.
(01:27):
And in doing so, created ascalable model to help thousands
of other families facingsimilar challenges utilizing
approaches like antisenceoligonucleotide-based
treatments.
He is a testament to the factthat a parent's love can move
mountains, or in this case,revolutionize our understanding
and ability to diagnose andtreat rare diseases, bringing
hope where there wasn't anybefore.
(01:49):
Please join me in welcomingwelcoming the one and only Casey
McPherson.
Hey, Casey.
Casey McPherson (01:54):
Hey, Ari.
I wish you were around.
That was the best intro I thinkI've ever had.
Ari Berman (01:59):
Oh, awesome.
I'll give it to you then.
Oh, welcome.
Um, but it's I've reallyenjoyed getting to know you over
the last few years, uh, asrandomly as that happened.
And story is one that I feltlike the Bio-IT community could
really grab onto, I think.
Um, and given that the missionand purpose of what you're doing
(02:20):
is largely about kids, aboutyour kid, um, but also kind of
about just pushing the envelopeof persistent medicine, which we
talk about in our field all thetime.
I thought it'd be fun to hearyour story.
So tell us a bit about yourjourney and how you came to
entirely switch fields fromrecord deals to running a
startup pharma company.
Casey McPherson (02:38):
Wow.
Well, um, you know, what whatdo they say that if you knew how
hard something was, youprobably would never do it, you
know?
And but that's been, you know,you look at the rare disease
metrics or pharma metrics or thesuccess of a drug metrics, and
actually they're better thanbeing a successful artist, those
metrics.
So I'm sort of used to the deckstacked against you, you know.
(03:01):
Uh, but I I think that therewere things in learning music
inherently is about creatingsomething out of nothing.
And if you've really been inthat environment for long
enough, you will find times whenyou make something that you
were just surprised it even cameout of you.
And then, but that's just thebeginning, and that's actually
(03:22):
the easy part.
The hard part is turning itinto a product that, you know,
can have uh some sort of globalreach.
And so this the songwriting,you know, in in some ways I
would account that topreclinical drug development for
genetic treatments.
We have the tools andtechnology now to to cure and
treat hundreds, maybe eventhousands of uh of these
(03:44):
diseases that have no treatmentsright now.
But but it's that next step ofturning it into a product and
getting it available.
And so, you know, when Rose wasdiagnosed, my experience was as
an entrepreneur, my experiencewas at a as a creator.
And uh when I saw that itwasn't a technology issue, it
was a business model problem, itwas a systems problem.
(04:05):
I thought, wow, maybe thissuper passionate, naive, like
I'll never give up, Scotch Irishkind of guy, like hit me one
more time, kind of person, anduh just see, just try to take me
down.
Um, that we have that incommon.
Yes, we do.
Uh blends for punishment.
Maybe we could look at some newmodels that uh would work for
(04:28):
these small population diseasesbecause it's a you know bigger
than cancer and AIDS combinedwhen you look at it as a whole.
But the way we've been lookingat it is these tiny diseases one
by one by one by one.
And a so, you know, when I sawthat ASOs in particular were
programmable medicines, um, itwas relatively safe to get into
(04:49):
the clinic if you did X, Y, andZ around safety and talks.
Um that that was it was sortof, you know, kind of calling
saying, hey, I might bescalable, you know, I might be
able to treat a lot of kids.
And so, you know, we made anASO, I I well, I recruited some
scientists, right, that had beendoing drug development um to
really teach me and train me onthe high-level side.
(05:12):
And and then ultragenics andsome other rare disease biotech
companies really took me undertheir wing um to mentor me.
But it was, you know, it wasreally scary at first to add a
record deal from Sony on my deskor go after a treatment for my
daughter.
I knew I couldn't do both, youknow.
And it's the first time in mylife that I chose that something
was more passionate for me thanthan making music.
(05:35):
And and uh I gotta say, man,making these making these
treatments is is pretty freakingrock and roll, you know, and
it's uh it's it's not there'sthere is uh it's not for the
faint of heart, you know.
And so I I feel strangely veryat home in in this environment.
Ari Berman (05:54):
That's uh awesome.
And it's funny because I don'tthink I've ever really thought
about like drug demand as rockand roll, I guess because I've
been exposed to it for so long,but I think you're right,
because it really is.
It's like, what have we notdone yet?
Let's try that, right?
Absolutely.
Casey McPherson (06:09):
You're you have the you have the gall to make
an assumption that you have anidea that has never been done
before, even within, even withinan assay or even within a
study, you know, like all theselittle points in time through
the development process,everybody's got to have a little
bit of a there is somethinginside me that's creative enough
(06:30):
and smart enough to be able toproduce something that has never
been produced before.
And it's a humbling thing, butit's also, I mean, that's what
rock and roll is.
Rock and roll is, you know,it's like it's like I am not
going to do what other peoplesay is the way this is just how
you're supposed to be or howyou're supposed to do it.
And, you know, I mean, maybeyou could argue that that we
(06:52):
haven't really seen thatmovement since the 70s.
It's an ideal, it's a it's atype of person.
And and now, now the leadsingers are the for me, are
those are the uh uh scientistsin the lab.
I'm just continually amazed atuh what people come up with.
Ari Berman (07:07):
Yeah, it's interesting.
I think I've told you thisbefore, but uh people often ask
me why scientists are nuts, andI sell them because science is
99% abject failure.
Um, and I guess rock and rollis too.
Casey McPherson (07:18):
Absolutely it is.
Yeah, it's like it's it Iremember at the height of my
career when I had a top 10 hitand a video on VH1, and I was
everybody's best friend.
You know, people were like,you're famous, you're like it
was as if you had never beenanything else.
But for me, all I could thinkof was the decades of failure
(07:39):
and bart, you know, empty roomsI played, um, broken down
vehicles, living under thepoverty line, you know, all the
labels that told me no or saidyou're not good enough, you
know, and and seeing how scienceworks, it's like you have to
like re-engineer how you thinkabout failure because failure is
(08:00):
is acting as if like you'velost.
And that's just not the casewith with music or with science.
Failure is like one stepfurther to the solution.
Ari Berman (08:09):
The truth is failure is never actually failure
unless you decide it is, right?
Because we don't learn withoutfailure.
No, we don't we don't we don'tbetter ourselves, we don't look
inward, we don't you have tofail to move forward.
You just there's no way to doit otherwise.
Otherwise, you think, oh, I'mgreat, everything's great.
Casey McPherson (08:25):
So yeah, I completely agree.
And it's almost as if, and andI'll tell you this this thing
with Rose, you know, the amountof suffering that these rare
diseases cause family, theamount of pain that she goes
through, you know, we often lookat things like failure and pain
as these things we try toavoid.
Ari Berman (08:45):
Yeah.
Casey McPherson (08:45):
And yet you cannot make anything great or
learn to be anything great orlearn to do anything great
without an immense amount offailure through that process.
And that I don't know for me, Ilook back at what we've
accomplished.
And without the pain that Rosiegoes through, without the pain
that I feel, I don't think Iwould have had the
(09:07):
self-discipline to learn thescience and the business and do
the amount of work and failuresto get, you know, even just to
the point that we're at now.
And so I, you know, I think itsort of turns everything on its
head of of sometimes it's bestto jump into all that because
it's it motivates you in a waythat nothing wrong with living
life on the beach, but livinglife on the beach doesn't really
(09:30):
encourage you to change a wholelot.
Ari Berman (09:32):
Not necessarily, no.
And there's nothing likeyou're, you know, being
connected to a problem like yourkid being the the you know,
having the problem, right?
So well, I mean, that's a greatsegue.
Why don't you tell me a littlebit about how Rose's mutation
has affected her and you know,what does she what does she deal
with?
Casey McPherson (09:50):
Yeah, so I'll start on the kind of micro and
then where the light bulb wentoff for me for alpha rose on the
macro level.
You know, so Rose lost herability to talk.
This gene starts expressing,you know, postnatally, and um,
and then she, you know, shereally struggles with walking.
She doesn't have any friends,she can't go to school.
(10:10):
You know, she's she is notpotty trained, and she's nine
now, nine years old.
And um and she's cute as canbe, by the way.
She is she's so cute.
I gotta say, um uh she andshe's even her even through all
these things, her personalitystill shines through where she
purposefully does something sheknows is gonna piss me off.
And it like makes her reallyhappy because she feels like she
(10:33):
was defiant, you know.
And that's a long line of uhfemales in my strong-willed
females in my family that thatsay, I'm gonna break the rules
and you're gonna watch me, youknow.
Um and so I love even thoughit's a pain in the butt
sometimes, I love seeing it.
Um, and she recently startedhaving seizures, you know.
This is it's been a year sinceshe had a seizure where her
(10:57):
heart stopped and she lost herbreathing.
And uh mom had to bring herback.
And so she's on seizure meds,but we don't know how long
that's gonna last.
And you know, what I learned,and so that's we don't go out to
eat.
We don't going on vacation is anightmare.
All these things that youtypically do as a family just
scratch them off, or know thatyou have an immense amount of
(11:19):
logistical planning ahead of youjust to do something very
simple.
And that's so where are allthese hundreds of millions of
families?
They're all at home survivingday to day.
And that's where like thismacro piece hit me, where these
families usually don't have theresources or the amount of
energy to do anything aboutthis.
And so consequently, they'rethey're sort of uh we call them
(11:41):
underserved.
Well, they're underheard,they're ignored, you know.
Ari Berman (11:45):
Absolutely.
Casey McPherson (11:46):
And and if if more children are dying, or they
say 30% of children with a raredisease die before their fifth
birthday, and they and there'sabout 200 million kiddos out
there with a rare disease, youcan imagine like we talk about
9-11, or we talk about the stuffhappening in the Middle East,
or like genocide.
(12:08):
This is the largest silentgenocide that I think we've seen
in you know our day and agethat is is solvable to some
degree, you know, not likecompletely curable, but we have
science.
And and and so that that reallydrove me to say I it can't just
be about rows.
(12:28):
Um, because I watched familiesdevelop these genetic
treatments, especially thosewith a little bit of means.
Ari Berman (12:34):
Yeah.
Casey McPherson (12:35):
You can go make an ASO or gene therapy for a
few million bucks and treattreat rows and in a clinical
trial and move on with money,which you may not know this, or
a lot of people may not knowthis, but there are families
doing that now.
Yeah.
And it's a thing.
And and they're saving theirkids' lives.
And but it's not scalable.
And no parent should ever haveto become a CEO of a biotech
(12:58):
company, you know.
So that's kind of what I want,I hope to prevent and I hope to
stop is the burden, thefinancial burden and the
educational burden, you know, onthese families so that those we
can get these treatments outand accessible.
Ari Berman (13:11):
Well, I think that last term is the most important
part there, accessible, becauseif it costs a couple million
bucks, there's not a lot offamilies that can do that.
Yeah, we're both CEOs, and ifneither of us can do it, right?
So that's that's why I had tolearn fundraising.
Casey McPherson (13:26):
And yeah, and it and and you know, there's
this huge misconception thatultra, you know, most rare
diseases genetic, most geneticdisease ultra-rare.
So that's that is true.
But there's this hugemisconception that ultra-rare
drugs do not make money.
And so it's been yeah largelyin the nonprofit world, which is
completely unscalable.
(13:47):
And you're just dump, you'rejust pouring money into a big
black hole, you know, withoutsome sort of sustainable plan.
And so that's why I started acompany eventually, you know,
for alpha rose, was that the thefinances work.
Maybe it's not a billion-dollardrug, but it certainly doesn't
cost me a billion dollars to getit to commercialize it.
You know, so it's it's like thedifference between like
(14:07):
building a residential house orbuilding a skyscraper.
We've been building skyscrapermedicines with these small
molecule programs, and thosehave like what a 90 to 95%
failure rate.
And genetic treatments, now noone's published on this, and I
hope I hope I can publish onthis at some point.
Genetic treatments have aexponentially higher chance of
(14:29):
success.
I'm watching dozens and dozensof these being developed by
people for the first time, andthey're working in the clinic,
you know.
Um so to me, it feels like thisis where medicine needs to go
if we're gonna start, you know,bringing down drug prices and
action making medicines to fix,you know, disease.
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Ari Berman (15:04):
Well, as we've discussed before, and you
brought it up, uh, the only wayto change anything in this world
is to figure out how to makemoney from it.
It's not because it's the rightthing to do.
If you can align those things,then uh something good could
happen, right?
Uh, but you gotta figure outhow to make money from it.
And the traditional pharmamodel of, you know, 12 years and
(15:25):
billions of dollars ofinvestment into this one small
molecule that has nine billionside effects, but it might
actually treat your symptoms.
Um, uh, and also it's gonnacost you $120,000 a pop, um, you
know, that's you know, that'sthe model.
Um and, you know, precisionmedicine, personalized medicine
has been something we've beenthrowing around for a long time.
And you're right, it ispartially there, but it's not
(15:46):
accessible.
And most people don't know thatit's there.
And yes, and since all thingson some level start with the
genetics, especially diseasesthat arise from genetic
mutations, uh, you know, is thissomething that can be knocked
down through some sort ofanti-sense, you know, thing uh,
you know, attaching to it?
It's uh really cool.
Casey McPherson (16:04):
That's I think, you know, it's it it's
interesting how accessible thiswas to me.
Like a lot of the companies andfamily foundations and families
that have sort of paved thisroad in some ways, you know, Eli
Lilly started a group calledChorus and they created this
drug development model.
Um, and if you if you look atwhat they published, um, this is
(16:27):
a huge, you know, pharmacorporation trying to figure out
how to create like lower riskand create efficiencies.
And I'm reading through it andsort of laughing because I'm
like, this is exactly whatfamilies are doing.
But they developed it, theydeveloped this model on their
own of what is enough, you know,when time equals life and and
you don't have a money treesitting next to you, then how do
(16:47):
you do efficient productdevelopment for a patient that's
gonna save their life, youknow?
And and to your point, youknow, I've never been motivated
by money solely, like that'snever been enough for me.
And but I think the it's it'sit's actually a fun part of this
job for me because I know thatproving to the investors when
(17:08):
they make their great returnswith lower risk off these
genetic medicines, it means thatthe investment community is
gonna start shifting to whatthey used to call orphan,
meaning like no home, to beinglike, oh man, I'd rather invest
in these because you know, Idon't invest in 20 of those
instead of one big drug becausethat they're gonna, you know,
(17:29):
most of them are gonna work.
And I I want to be a part ofthat shift.
Ari Berman (17:33):
Yes.
Uh well, and so all you need issome high profile thing out
there, and then people are gonnajump on it and you're out there
making lots of good noise inthe investor communities, which
tell us about Alpha Rose alittle bit.
Casey McPherson (17:45):
Yeah, well, so you know, the first thing that a
lot of venture capital don'tlike is we're a public benefit
corporation.
And so what blew my mind isthat healthcare companies as a
corporation are only required tobe fiduciarily responsible to
their shareholders.
That's correct.
So you can screw over as manypatients as you like, um, with
(18:08):
with sort of zero uh, you know,uh blowback into well, maybe in
the press, you know, but but sowhat I love about public benefit
corporations is more of abalance between your investors
and your patients.
And so you can't really screweither over, you know?
And and and I think inhealthcare, there's this unique
relationship between capital andpatients.
(18:30):
And it's it's like capitalism'sgreat when you get great
medicines out there thatpatients have access to.
Capitalism's not great when youhave shell drugs that are
working, but they, you know,aren't gonna make as much money.
And or when you have whenyou're choosing assets based on
revenue, not based on the impactit's gonna make on a patient.
(18:51):
So there's a ba— I thinkthere's a really nice balance.
And so Alpha Rose is sort offundamentally started with that
approach and co-founded byBelinda Tremir and Alan Waltz,
who, you know, if you'refamiliar with the Gen zyme
story, Henry Kermere was one ofthe most innovative CEOs for
rare disease, and Gen zyme soldfor 20 billion.
And and these two help buildthat company.
(19:13):
My presence Masoko Nakamura,she was also at Gen zyme,
commercialized over 20 rareproducts in her career.
So I have these reallyincredible folks on the
executive level that believethat if you put patients first,
you can build a very lucrativecompany um by focusing on
getting a treatment intopatients as quickly and safely
(19:35):
as possible and providing asmuch access as humanly possible.
And and they used to have abell they would ring every time
they treated a patient, youknow.
Oh, I love that globe.
And so that culture issomething I want to continue,
Henry's legacy in that a companydoesn't have to be a big, bad
company to make money and totreat patients.
(19:56):
And it's simply just where'syour focus?
Is your focus solely to makemoney?
And then, yeah, patients are apart of that, you know.
Well, then you become the big,bad, you know, healthcare
company.
But if you actually putpatients, you can do good and
make money.
It's possible, you know.
Ari Berman (20:12):
You can, yes.
It's harder.
Casey McPherson (20:14):
The goal, yeah.
It requires drawing some linesin the sand for sure.
Um, and it requires a differentkind of culture.
But you know, we've so forAlphaRose, we've we've had we're
we're sort of this integrationbetween ASO technology and
software, AI, eventually somerobotics, and and the idea that
(20:34):
these are programmablemedicines.
So the more we can layer onautomations through the
preclinical, clinical, andcommercialization process, the
more ability we will have toscale these treatments.
And what I realized in the lab,which was like the first
company I started, was thispreclinical lab, uh, is that so
many of these pieces arerepeatable.
(20:56):
And we're screening many, manycompounds to try to figure out
which one's toxic and whichone's not.
And I'm a musician, so youknow, is it crazy to think that
there might be some patterns inour biology to start to start
helping us make the argument forpredictive toxicity and
predictive interaction with thegene, you know, some predictive,
(21:16):
at least genetic efficacy.
Um, and and and so far nobody'stold me that's crazy.
And so we've been building somesome uh pilot systems to design
our ASOs around uh around this,and and I'm very excited about
it.
Ari Berman (21:31):
That's awesome.
Clearly, we're well aligned onthese things.
So yeah, uh, I I love thestory.
Um, so talk a little bit aboutsome of the technology to guys
are uh using that this audienceis both tech and science.
So yeah, what would that you'vetold me about this thing and I
I can't wait to go see it.
Casey McPherson (21:48):
So it's yeah, well, I mean, you know, there's
so many things that need to bebuilt.
Um, but but the way I've beenlooking at this is we have this
car, it kind of runs, and in thecurrent regulatory environment,
we can do this.
These little diseases willstill make some money, they'll
still be expensive drugs.
Yeah, but all of it works as anefficient if you create an
(22:10):
efficient pipeline with thecurrent technologies we have,
which are ASO therapeutics, youknow, using that chemistry set,
and then particular assays anduh animal studies that are
required to like make sure it'ssafe.
But then you start adding in umuh software, and ASOs are a
sequence of nucleotides.
And so so are genes, right?
(22:32):
Yeah, it's we quickly saw like,what if we have a database of
genetic mutations and we designsome uh automation, some machine
learning, and maybe somemagnetic AI to look at the
amenability of each mutation forour technology.
So building that database rightnow with neurodevelopmental and
(22:53):
just pulling from Omen,Plimbar, and I think, oh, this
sounds like a great idea.
Then the first problem we haveis that you know, Omen doesn't
communicate with Nomad and theyuse different.
And so I'm like, oh shit, it'slike Chinese and uh English and
Spanish all trying to to tocollaborate together.
So so that that becamesomething we started building
(23:14):
was this was that a stone to getthe the the gene databases
talking to each other.
And and then we built, which Iwe showed you, you know, and and
and you know, we've got a longway to go on this, but kind of
the first iteration of an ASOdesign tool that could look at
all of the factors that wouldcreate toxicity.
So usually you design these ina spreadsheet and you've got
(23:36):
thousands of designs justwalking down the gene.
But now there are certainthings like aspects around
what's the most active area ofthe gene where if we wanted to
uh uh downregulate or upregulatea gene, what are those most,
you know, best areas to do thatin?
So that's shortlists.
What are the type nucleotidesequences that could be
(23:58):
potentially more toxic?
We can shortlist around that.
And then there's there's somecalcium stuff that also, this is
where it gets a little bitlike, well, a lot over my head.
There's these these sort ofchemical and bioalgorithmic uh
assessments that we're able tomake now to even shortlist.
And so I think you know, wehave really great potential by
(24:20):
with this design tool, by themore data, the more quality data
we can feed it and order that.
The last thing that I'm excitedabout is we're taking, you
know, Rose's drug, Ros iphersen,is ready for the clinic.
And so we've been partneringwith some wearable companies
because the way they do clinicaltrials, like I don't know, I
didn't go to school for this,but when they told me this is
(24:41):
how you do a clinical trial, Iwas like, I'm sorry, but this
seems really dumb to me.
If you told me you're gonna seeefficacy based on the studies
that you're currently doing,you're not.
Like you're gonna themeasurements that they've come
up with were like made in the70s.
Like, think about how muchwe've developed here, and yet
(25:02):
our yet are yet the how we'recollecting data in our clinical
trials has not.
So we're using these wearableslike to track Rose and these
kids to establish a baseline andlike a three-dimensional
digital footprint unbiased thatwe can we can collect from
video, uh EEG and and motor andcognition at their home, doing
(25:25):
what they always do instead ofbeing at a clinician's office.
So I think where there's gonnabe a lot of really cool
innovation in that area toshorten a six-year trial down to
a year or two, you know.
Yeah.
Uh so that these are all youknow, technology pieces that now
are super, super important thatinterface really, really well
with this, with antisensalorganopotides.
Ari Berman (25:46):
I'm excited to see where that goes.
The uh wearable, the Internetof Things groups that have been
trying to figure out how to pullthis stuff in for a long time.
There's there's uh likepediatric moonshot is another
thing that's really interesting.
They they've been doing this,but wanting to take all that
data and process it on the edgeand then take the processed data
and pull it into the middle todo something cool with it,
(26:08):
right?
Um, but uh yeah.
Casey McPherson (26:11):
I mean, I think that's that's I'm excited about
um, you know, kind of the toolswe have now.
And it reminds me again, likebeing in a music studio where
you've got your analogsynthesizers, you got your great
compressors, you've got allthese great tools to work with.
The trick is, is if the focusis that time does equal life and
(26:32):
that patients are waiting forthese treatments, and we're in a
war that that kids and patientsare dying every minute right
now, that we have to be able tocreate an environment where we
can quickly innovate and iterateon these technologies because
it's like you're you're in aworld war and you're trying to
make a new gun or a new defensemech, you know, you don't have
(26:56):
unlimited time to to to do this,you know.
So I do feel this sense ofurgency and and like bringing
people in, like, let's work onthis, let's fix this.
You know, we can do it, it'spossible.
So, you know, that's excitingbut also frustrating.
Well, it's a short fuse, right?
Ari Berman (27:11):
Some people are like, uh, what are you talking
about?
You got the rest of your life,could be that long, right?
That's what people don'trealize.
And also people are like, eh,you know, it's a rare disease.
And well, you'll say that untilit happens to you, right?
Casey McPherson (27:22):
Yeah, you got a one in twenty chance of it
happening to you or familyunder.
And, you know, we love to, Imean, I'll tell you, when I saw
families with special needskids, I had zero desire to meet
them.
I sure am glad that isn't me.
Yeah.
And when it happens to you,you're, you know, it's like then
(27:43):
all of a sudden you see all theother families out there that
are struggling with this.
And, you know, it's uh it's avery different, different thing.
But it's, you know, there's thepeople that it hasn't happened
to that actually have thecapacity to do this stuff.
Those are the people I considertrue rock stars that are like,
all right, we got to changethis.
Let's fix it.
Where do we start?
Ari Berman (28:03):
Absolutely.
Absolutely.
Well, um, I guess the last bitI'll ask you is to wrap up is
how can people get involved andhelp?
Casey McPherson (28:10):
I mean, you know, we're uh always looking
for great minds that um havegifting that can help, you know,
participate and solve thisproblem.
We're also looking for moremission-driven investors.
I mean, you could easily turnour company into a large pharma
company.
Ari Berman (28:28):
Yep.
Casey McPherson (28:29):
So it's very important that our investors
align with the fact that we'reonly going to be commercializing
these ultra-rare products andwe're gonna be focused on
building the scale as opposed tojust being a traditional
company.
Um, and so, you know, we'rewe're running an equity
crowdfunding uh campaign whereanyone can invest.
Uh, you know, that's started indot com slash AlphaRose.
(28:51):
And then, you know, you canalways find us on our website
and on social media that that ifif someone's interested in
being a part of this in someway, um, you know, we're we're
looking for you.
You know, it's like the the uhthe US uh who was the old Uncle
Sam Uncle Sam Uncle Sam is like,you know, we want you because
at the end of the day, there'shundreds of millions of patients
(29:12):
waiting, and none of us can dothis alone.
So the more that we canpartner, band together, put our
resources together, Alpha Roseis merely a vessel to break open
this space.
I hope that many, many othercompanies do this like we're
doing it and and benefit from ittoo.
Ari Berman (29:28):
Awesome.
Well, I'm super excited to seewhere this goes, and I hope that
uh that I get to jump in andhelp too, because it's an
awesome, awesome effort.
So um too, Ari.
Casey McPherson (29:40):
Um we we needed many we we don't need many
excuses to work together becauseI have so much respect for you
and your team and what you guysdo.
Ari Berman (29:48):
Uh appreciate that.
Well, we're looking forward toit and thanks for being on the
podcast today.
Really appreciate it.
And uh uh I hope uh peoplereach out and get involved and
Uh realize, hey, precisionmedicine is closer than we
think.
And it's not just for theultra-rich.
Casey McPherson (30:05):
Yeah, absolutely.
Thanks for having me, Ari.
Ari Berman (30:08):
Thanks, Casey.
Take care.
Announcement (30:10):
Thank you for listening to Bio-IT World's
Trends from the Trenchespodcast.
Welcome to the Trends from the Trenches
Podcast, co-produced by Bioteamand BioIT World.
I'm your host, Ari Berman, CEOof BioTeam.
Today we have a trulyremarkable guest who has
navigated the worlds of musicand science with an incredible
purpose.
He's a guy I'm lucky to call afriend, and his story is one of
artistry, fatherhood, and arelentless fight for a cure.
(00:21):
You know him as a captivatingfrontman for two incredible
bands, the anthemic indie rockgroup Alpha Rev and the
progressive rock super groupFlying Colors.
He's brought us songs that havetopped the charts and filled
stadiums around the world withinstantly recognizable hits such
as New Morning and Sing Loud.
But beyond the stage andstudio, our guests' most
(00:42):
profound journey began at homewith his two daughters, Weston
and Rose.
When his youngest daughter,Rose, was diagnosed with a rare
debilitating genetic mutation,HNRNPH2, which encodes a protein
that belongs to theheterogeneous nuclearibro
protein family and plays a rolein RNA processing and transport,
his path changed forever.
What he discovered was aheartbreaking reality.
(01:04):
While there are over 10,000known rare diseases affecting
millions of people, as reportedby the National Center for
Advancing Transactional Science,most of them, including this
disease, have no treatment.
Refusing to accept that fate,he channeled the same passion
and creativity he poured intohis music into a new mission.
He founded Alpha RoseTherapeutics, a biotech company
dedicated to finding a treatmentfor Rhodes' rare disease.
(01:27):
And in doing so, created ascalable model to help thousands
of other families facingsimilar challenges utilizing
approaches like antisenceoligonucleotide-based
treatments.
He is a testament to the factthat a parent's love can move
mountains, or in this case,revolutionize our understanding
and ability to diagnose andtreat rare diseases, bringing
hope where there wasn't anybefore.
(01:49):
Please join me in welcomingwelcoming the one and only Casey
McPherson.
Hey, Casey.
Casey McPherson (01:54):
Hey, Ari.
I wish you were around.
That was the best intro I thinkI've ever had.
Ari Berman (01:59):
Oh, awesome.
I'll give it to you then.
Oh, welcome.
Um, but it's I've reallyenjoyed getting to know you over
the last few years, uh, asrandomly as that happened.
And story is one that I feltlike the Bio-IT community could
really grab onto, I think.
Um, and given that the missionand purpose of what you're doing
(02:20):
is largely about kids, aboutyour kid, um, but also kind of
about just pushing the envelopeof persistent medicine, which we
talk about in our field all thetime.
I thought it'd be fun to hearyour story.
So tell us a bit about yourjourney and how you came to
entirely switch fields fromrecord deals to running a
startup pharma company.
Casey McPherson (02:38):
Wow.
Well, um, you know, what whatdo they say that if you knew how
hard something was, youprobably would never do it, you
know?
And but that's been, you know,you look at the rare disease
metrics or pharma metrics or thesuccess of a drug metrics, and
actually they're better thanbeing a successful artist, those
metrics.
So I'm sort of used to the deckstacked against you, you know.
(03:01):
Uh, but I I think that therewere things in learning music
inherently is about creatingsomething out of nothing.
And if you've really been inthat environment for long
enough, you will find times whenyou make something that you
were just surprised it even cameout of you.
And then, but that's just thebeginning, and that's actually
(03:22):
the easy part.
The hard part is turning itinto a product that, you know,
can have uh some sort of globalreach.
And so this the songwriting,you know, in in some ways I
would account that topreclinical drug development for
genetic treatments.
We have the tools andtechnology now to to cure and
treat hundreds, maybe eventhousands of uh of these
(03:44):
diseases that have no treatmentsright now.
But but it's that next step ofturning it into a product and
getting it available.
And so, you know, when Rose wasdiagnosed, my experience was as
an entrepreneur, my experiencewas at a as a creator.
And uh when I saw that itwasn't a technology issue, it
was a business model problem, itwas a systems problem.
(04:05):
I thought, wow, maybe thissuper passionate, naive, like
I'll never give up, Scotch Irishkind of guy, like hit me one
more time, kind of person, anduh just see, just try to take me
down.
Um, that we have that incommon.
Yes, we do.
Uh blends for punishment.
Maybe we could look at some newmodels that uh would work for
(04:28):
these small population diseasesbecause it's a you know bigger
than cancer and AIDS combinedwhen you look at it as a whole.
But the way we've been lookingat it is these tiny diseases one
by one by one by one.
And a so, you know, when I sawthat ASOs in particular were
programmable medicines, um, itwas relatively safe to get into
(04:49):
the clinic if you did X, Y, andZ around safety and talks.
Um that that was it was sortof, you know, kind of calling
saying, hey, I might bescalable, you know, I might be
able to treat a lot of kids.
And so, you know, we made anASO, I I well, I recruited some
scientists, right, that had beendoing drug development um to
really teach me and train me onthe high-level side.
(05:12):
And and then ultragenics andsome other rare disease biotech
companies really took me undertheir wing um to mentor me.
But it was, you know, it wasreally scary at first to add a
record deal from Sony on my deskor go after a treatment for my
daughter.
I knew I couldn't do both, youknow.
And it's the first time in mylife that I chose that something
was more passionate for me thanthan making music.
(05:35):
And and uh I gotta say, man,making these making these
treatments is is pretty freakingrock and roll, you know, and
it's uh it's it's not there'sthere is uh it's not for the
faint of heart, you know.
And so I I feel strangely veryat home in in this environment.
Ari Berman (05:54):
That's uh awesome.
And it's funny because I don'tthink I've ever really thought
about like drug demand as rockand roll, I guess because I've
been exposed to it for so long,but I think you're right,
because it really is.
It's like, what have we notdone yet?
Let's try that, right?
Absolutely.
Casey McPherson (06:09):
You're you have the you have the gall to make
an assumption that you have anidea that has never been done
before, even within, even withinan assay or even within a
study, you know, like all theselittle points in time through
the development process,everybody's got to have a little
bit of a there is somethinginside me that's creative enough
(06:30):
and smart enough to be able toproduce something that has never
been produced before.
And it's a humbling thing, butit's also, I mean, that's what
rock and roll is.
Rock and roll is, you know,it's like it's like I am not
going to do what other peoplesay is the way this is just how
you're supposed to be or howyou're supposed to do it.
And, you know, I mean, maybeyou could argue that that we
(06:52):
haven't really seen thatmovement since the 70s.
It's an ideal, it's a it's atype of person.
And and now, now the leadsingers are the for me, are
those are the uh uh scientistsin the lab.
I'm just continually amazed atuh what people come up with.
Ari Berman (07:07):
Yeah, it's interesting.
I think I've told you thisbefore, but uh people often ask
me why scientists are nuts, andI sell them because science is
99% abject failure.
Um, and I guess rock and rollis too.
Casey McPherson (07:18):
Absolutely it is.
Yeah, it's like it's it Iremember at the height of my
career when I had a top 10 hitand a video on VH1, and I was
everybody's best friend.
You know, people were like,you're famous, you're like it
was as if you had never beenanything else.
But for me, all I could thinkof was the decades of failure
(07:39):
and bart, you know, empty roomsI played, um, broken down
vehicles, living under thepoverty line, you know, all the
labels that told me no or saidyou're not good enough, you
know, and and seeing how scienceworks, it's like you have to
like re-engineer how you thinkabout failure because failure is
(08:00):
is acting as if like you'velost.
And that's just not the casewith with music or with science.
Failure is like one stepfurther to the solution.
Ari Berman (08:09):
The truth is failure is never actually failure
unless you decide it is, right?
Because we don't learn withoutfailure.
No, we don't we don't we don'tbetter ourselves, we don't look
inward, we don't you have tofail to move forward.
You just there's no way to doit otherwise.
Otherwise, you think, oh, I'mgreat, everything's great.
Casey McPherson (08:25):
So yeah, I completely agree.
And it's almost as if, and andI'll tell you this this thing
with Rose, you know, the amountof suffering that these rare
diseases cause family, theamount of pain that she goes
through, you know, we often lookat things like failure and pain
as these things we try toavoid.
Ari Berman (08:45):
Yeah.
Casey McPherson (08:45):
And yet you cannot make anything great or
learn to be anything great orlearn to do anything great
without an immense amount offailure through that process.
And that I don't know for me, Ilook back at what we've
accomplished.
And without the pain that Rosiegoes through, without the pain
that I feel, I don't think Iwould have had the
(09:07):
self-discipline to learn thescience and the business and do
the amount of work and failuresto get, you know, even just to
the point that we're at now.
And so I, you know, I think itsort of turns everything on its
head of of sometimes it's bestto jump into all that because
it's it motivates you in a waythat nothing wrong with living
life on the beach, but livinglife on the beach doesn't really
(09:30):
encourage you to change a wholelot.
Ari Berman (09:32):
Not necessarily, no.
And there's nothing likeyou're, you know, being
connected to a problem like yourkid being the the you know,
having the problem, right?
So well, I mean, that's a greatsegue.
Why don't you tell me a littlebit about how Rose's mutation
has affected her and you know,what does she what does she deal
with?
Casey McPherson (09:50):
Yeah, so I'll start on the kind of micro and
then where the light bulb wentoff for me for alpha rose on the
macro level.
You know, so Rose lost herability to talk.
This gene starts expressing,you know, postnatally, and um,
and then she, you know, shereally struggles with walking.
She doesn't have any friends,she can't go to school.
(10:10):
You know, she's she is notpotty trained, and she's nine
now, nine years old.
And um and she's cute as canbe, by the way.
She is she's so cute.
I gotta say, um uh she andshe's even her even through all
these things, her personalitystill shines through where she
purposefully does something sheknows is gonna piss me off.
And it like makes her reallyhappy because she feels like she
(10:33):
was defiant, you know.
And that's a long line of uhfemales in my strong-willed
females in my family that thatsay, I'm gonna break the rules
and you're gonna watch me, youknow.
Um and so I love even thoughit's a pain in the butt
sometimes, I love seeing it.
Um, and she recently startedhaving seizures, you know.
This is it's been a year sinceshe had a seizure where her
(10:57):
heart stopped and she lost herbreathing.
And uh mom had to bring herback.
And so she's on seizure meds,but we don't know how long
that's gonna last.
And you know, what I learned,and so that's we don't go out to
eat.
We don't going on vacation is anightmare.
All these things that youtypically do as a family just
scratch them off, or know thatyou have an immense amount of
(11:19):
logistical planning ahead of youjust to do something very
simple.
And that's so where are allthese hundreds of millions of
families?
They're all at home survivingday to day.
And that's where like thismacro piece hit me, where these
families usually don't have theresources or the amount of
energy to do anything aboutthis.
And so consequently, they'rethey're sort of uh we call them
(11:41):
underserved.
Well, they're underheard,they're ignored, you know.
Ari Berman (11:45):
Absolutely.
Casey McPherson (11:46):
And and if if more children are dying, or they
say 30% of children with a raredisease die before their fifth
birthday, and they and there'sabout 200 million kiddos out
there with a rare disease, youcan imagine like we talk about
9-11, or we talk about the stuffhappening in the Middle East,
or like genocide.
(12:08):
This is the largest silentgenocide that I think we've seen
in you know our day and agethat is is solvable to some
degree, you know, not likecompletely curable, but we have
science.
And and and so that that reallydrove me to say I it can't just
be about rows.
(12:28):
Um, because I watched familiesdevelop these genetic
treatments, especially thosewith a little bit of means.
Ari Berman (12:34):
Yeah.
Casey McPherson (12:35):
You can go make an ASO or gene therapy for a
few million bucks and treattreat rows and in a clinical
trial and move on with money,which you may not know this, or
a lot of people may not knowthis, but there are families
doing that now.
Yeah.
And it's a thing.
And and they're saving theirkids' lives.
And but it's not scalable.
And no parent should ever haveto become a CEO of a biotech
(12:58):
company, you know.
So that's kind of what I want,I hope to prevent and I hope to
stop is the burden, thefinancial burden and the
educational burden, you know, onthese families so that those we
can get these treatments outand accessible.
Ari Berman (13:11):
Well, I think that last term is the most important
part there, accessible, becauseif it costs a couple million
bucks, there's not a lot offamilies that can do that.
Yeah, we're both CEOs, and ifneither of us can do it, right?
So that's that's why I had tolearn fundraising.
Casey McPherson (13:26):
And yeah, and it and and you know, there's
this huge misconception thatultra, you know, most rare
diseases genetic, most geneticdisease ultra-rare.
So that's that is true.
But there's this hugemisconception that ultra-rare
drugs do not make money.
And so it's been yeah largelyin the nonprofit world, which is
completely unscalable.
(13:47):
And you're just dump, you'rejust pouring money into a big
black hole, you know, withoutsome sort of sustainable plan.
And so that's why I started acompany eventually, you know,
for alpha rose, was that the thefinances work.
Maybe it's not a billion-dollardrug, but it certainly doesn't
cost me a billion dollars to getit to commercialize it.
You know, so it's it's like thedifference between like
(14:07):
building a residential house orbuilding a skyscraper.
We've been building skyscrapermedicines with these small
molecule programs, and thosehave like what a 90 to 95%
failure rate.
And genetic treatments, now noone's published on this, and I
hope I hope I can publish onthis at some point.
Genetic treatments have aexponentially higher chance of
(14:29):
success.
I'm watching dozens and dozensof these being developed by
people for the first time, andthey're working in the clinic,
you know.
Um so to me, it feels like thisis where medicine needs to go
if we're gonna start, you know,bringing down drug prices and
action making medicines to fix,you know, disease.
Announcement (14:48):
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We'd love to hear from you.
Please subscribe to the podcastand give us a rating.
It helps other people find andjoin the conversation.
If you've got speaker or topicideas, we'd love to hear those
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Ari Berman (15:04):
Well, as we've discussed before, and you
brought it up, uh, the only wayto change anything in this world
is to figure out how to makemoney from it.
It's not because it's the rightthing to do.
If you can align those things,then uh something good could
happen, right?
Uh, but you gotta figure outhow to make money from it.
And the traditional pharmamodel of, you know, 12 years and
(15:25):
billions of dollars ofinvestment into this one small
molecule that has nine billionside effects, but it might
actually treat your symptoms.
Um, uh, and also it's gonnacost you $120,000 a pop, um, you
know, that's you know, that'sthe model.
Um and, you know, precisionmedicine, personalized medicine
has been something we've beenthrowing around for a long time.
And you're right, it ispartially there, but it's not
(15:46):
accessible.
And most people don't know thatit's there.
And yes, and since all thingson some level start with the
genetics, especially diseasesthat arise from genetic
mutations, uh, you know, is thissomething that can be knocked
down through some sort ofanti-sense, you know, thing uh,
you know, attaching to it?
It's uh really cool.
Casey McPherson (16:04):
That's I think, you know, it's it it's
interesting how accessible thiswas to me.
Like a lot of the companies andfamily foundations and families
that have sort of paved thisroad in some ways, you know, Eli
Lilly started a group calledChorus and they created this
drug development model.
Um, and if you if you look atwhat they published, um, this is
(16:27):
a huge, you know, pharmacorporation trying to figure out
how to create like lower riskand create efficiencies.
And I'm reading through it andsort of laughing because I'm
like, this is exactly whatfamilies are doing.
But they developed it, theydeveloped this model on their
own of what is enough, you know,when time equals life and and
you don't have a money treesitting next to you, then how do
(16:47):
you do efficient productdevelopment for a patient that's
gonna save their life, youknow?
And and to your point, youknow, I've never been motivated
by money solely, like that'snever been enough for me.
And but I think the it's it'sit's actually a fun part of this
job for me because I know thatproving to the investors when
(17:08):
they make their great returnswith lower risk off these
genetic medicines, it means thatthe investment community is
gonna start shifting to whatthey used to call orphan,
meaning like no home, to beinglike, oh man, I'd rather invest
in these because you know, Idon't invest in 20 of those
instead of one big drug becausethat they're gonna, you know,
(17:29):
most of them are gonna work.
And I I want to be a part ofthat shift.
Ari Berman (17:33):
Yes.
Uh well, and so all you need issome high profile thing out
there, and then people are gonnajump on it and you're out there
making lots of good noise inthe investor communities, which
tell us about Alpha Rose alittle bit.
Casey McPherson (17:45):
Yeah, well, so you know, the first thing that a
lot of venture capital don'tlike is we're a public benefit
corporation.
And so what blew my mind isthat healthcare companies as a
corporation are only required tobe fiduciarily responsible to
their shareholders.
That's correct.
So you can screw over as manypatients as you like, um, with
(18:08):
with sort of zero uh, you know,uh blowback into well, maybe in
the press, you know, but but sowhat I love about public benefit
corporations is more of abalance between your investors
and your patients.
And so you can't really screweither over, you know?
And and and I think inhealthcare, there's this unique
relationship between capital andpatients.
(18:30):
And it's it's like capitalism'sgreat when you get great
medicines out there thatpatients have access to.
Capitalism's not great when youhave shell drugs that are
working, but they, you know,aren't gonna make as much money.
And or when you have whenyou're choosing assets based on
revenue, not based on the impactit's gonna make on a patient.
(18:51):
So there's a ba— I thinkthere's a really nice balance.
And so Alpha Rose is sort offundamentally started with that
approach and co-founded byBelinda Tremir and Alan Waltz,
who, you know, if you'refamiliar with the Gen zyme
story, Henry Kermere was one ofthe most innovative CEOs for
rare disease, and Gen zyme soldfor 20 billion.
And and these two help buildthat company.
(19:13):
My presence Masoko Nakamura,she was also at Gen zyme,
commercialized over 20 rareproducts in her career.
So I have these reallyincredible folks on the
executive level that believethat if you put patients first,
you can build a very lucrativecompany um by focusing on
getting a treatment intopatients as quickly and safely
(19:35):
as possible and providing asmuch access as humanly possible.
And and they used to have abell they would ring every time
they treated a patient, youknow.
Oh, I love that globe.
And so that culture issomething I want to continue,
Henry's legacy in that a companydoesn't have to be a big, bad
company to make money and totreat patients.
(19:56):
And it's simply just where'syour focus?
Is your focus solely to makemoney?
And then, yeah, patients are apart of that, you know.
Well, then you become the big,bad, you know, healthcare
company.
But if you actually putpatients, you can do good and
make money.
It's possible, you know.
Ari Berman (20:12):
You can, yes.
It's harder.
Casey McPherson (20:14):
The goal, yeah.
It requires drawing some linesin the sand for sure.
Um, and it requires a differentkind of culture.
But you know, we've so forAlphaRose, we've we've had we're
we're sort of this integrationbetween ASO technology and
software, AI, eventually somerobotics, and and the idea that
(20:34):
these are programmablemedicines.
So the more we can layer onautomations through the
preclinical, clinical, andcommercialization process, the
more ability we will have toscale these treatments.
And what I realized in the lab,which was like the first
company I started, was thispreclinical lab, uh, is that so
many of these pieces arerepeatable.
(20:56):
And we're screening many, manycompounds to try to figure out
which one's toxic and whichone's not.
And I'm a musician, so youknow, is it crazy to think that
there might be some patterns inour biology to start to start
helping us make the argument forpredictive toxicity and
predictive interaction with thegene, you know, some predictive,
(21:16):
at least genetic efficacy.
Um, and and and so far nobody'stold me that's crazy.
And so we've been building somesome uh pilot systems to design
our ASOs around uh around this,and and I'm very excited about
it.
Ari Berman (21:31):
That's awesome.
Clearly, we're well aligned onthese things.
So yeah, uh, I I love thestory.
Um, so talk a little bit aboutsome of the technology to guys
are uh using that this audienceis both tech and science.
So yeah, what would that you'vetold me about this thing and I
I can't wait to go see it.
Casey McPherson (21:48):
So it's yeah, well, I mean, you know, there's
so many things that need to bebuilt.
Um, but but the way I've beenlooking at this is we have this
car, it kind of runs, and in thecurrent regulatory environment,
we can do this.
These little diseases willstill make some money, they'll
still be expensive drugs.
Yeah, but all of it works as anefficient if you create an
(22:10):
efficient pipeline with thecurrent technologies we have,
which are ASO therapeutics, youknow, using that chemistry set,
and then particular assays anduh animal studies that are
required to like make sure it'ssafe.
But then you start adding in umuh software, and ASOs are a
sequence of nucleotides.
And so so are genes, right?
(22:32):
Yeah, it's we quickly saw like,what if we have a database of
genetic mutations and we designsome uh automation, some machine
learning, and maybe somemagnetic AI to look at the
amenability of each mutation forour technology.
So building that database rightnow with neurodevelopmental and
(22:53):
just pulling from Omen,Plimbar, and I think, oh, this
sounds like a great idea.
Then the first problem we haveis that you know, Omen doesn't
communicate with Nomad and theyuse different.
And so I'm like, oh shit, it'slike Chinese and uh English and
Spanish all trying to to tocollaborate together.
So so that that becamesomething we started building
(23:14):
was this was that a stone to getthe the the gene databases
talking to each other.
And and then we built, which Iwe showed you, you know, and and
and you know, we've got a longway to go on this, but kind of
the first iteration of an ASOdesign tool that could look at
all of the factors that wouldcreate toxicity.
So usually you design these ina spreadsheet and you've got
(23:36):
thousands of designs justwalking down the gene.
But now there are certainthings like aspects around
what's the most active area ofthe gene where if we wanted to
uh uh downregulate or upregulatea gene, what are those most,
you know, best areas to do thatin?
So that's shortlists.
What are the type nucleotidesequences that could be
(23:58):
potentially more toxic?
We can shortlist around that.
And then there's there's somecalcium stuff that also, this is
where it gets a little bitlike, well, a lot over my head.
There's these these sort ofchemical and bioalgorithmic uh
assessments that we're able tomake now to even shortlist.
And so I think you know, wehave really great potential by
(24:20):
with this design tool, by themore data, the more quality data
we can feed it and order that.
The last thing that I'm excitedabout is we're taking, you
know, Rose's drug, Ros iphersen,is ready for the clinic.
And so we've been partneringwith some wearable companies
because the way they do clinicaltrials, like I don't know, I
didn't go to school for this,but when they told me this is
(24:41):
how you do a clinical trial, Iwas like, I'm sorry, but this
seems really dumb to me.
If you told me you're gonna seeefficacy based on the studies
that you're currently doing,you're not.
Like you're gonna themeasurements that they've come
up with were like made in the70s.
Like, think about how muchwe've developed here, and yet
(25:02):
our yet are yet the how we'recollecting data in our clinical
trials has not.
So we're using these wearableslike to track Rose and these
kids to establish a baseline andlike a three-dimensional
digital footprint unbiased thatwe can we can collect from
video, uh EEG and and motor andcognition at their home, doing
(25:25):
what they always do instead ofbeing at a clinician's office.
So I think where there's gonnabe a lot of really cool
innovation in that area toshorten a six-year trial down to
a year or two, you know.
Yeah.
Uh so that these are all youknow, technology pieces that now
are super, super important thatinterface really, really well
with this, with antisensalorganopotides.
Ari Berman (25:46):
I'm excited to see where that goes.
The uh wearable, the Internetof Things groups that have been
trying to figure out how to pullthis stuff in for a long time.
There's there's uh likepediatric moonshot is another
thing that's really interesting.
They they've been doing this,but wanting to take all that
data and process it on the edgeand then take the processed data
and pull it into the middle todo something cool with it,
(26:08):
right?
Um, but uh yeah.
Casey McPherson (26:11):
I mean, I think that's that's I'm excited about
um, you know, kind of the toolswe have now.
And it reminds me again, likebeing in a music studio where
you've got your analogsynthesizers, you got your great
compressors, you've got allthese great tools to work with.
The trick is, is if the focusis that time does equal life and
(26:32):
that patients are waiting forthese treatments, and we're in a
war that that kids and patientsare dying every minute right
now, that we have to be able tocreate an environment where we
can quickly innovate and iterateon these technologies because
it's like you're you're in aworld war and you're trying to
make a new gun or a new defensemech, you know, you don't have
(26:56):
unlimited time to to to do this,you know.
So I do feel this sense ofurgency and and like bringing
people in, like, let's work onthis, let's fix this.
You know, we can do it, it'spossible.
So, you know, that's excitingbut also frustrating.
Well, it's a short fuse, right?
Ari Berman (27:11):
Some people are like, uh, what are you talking
about?
You got the rest of your life,could be that long, right?
That's what people don'trealize.
And also people are like, eh,you know, it's a rare disease.
And well, you'll say that untilit happens to you, right?
Casey McPherson (27:22):
Yeah, you got a one in twenty chance of it
happening to you or familyunder.
And, you know, we love to, Imean, I'll tell you, when I saw
families with special needskids, I had zero desire to meet
them.
I sure am glad that isn't me.
Yeah.
And when it happens to you,you're, you know, it's like then
(27:43):
all of a sudden you see all theother families out there that
are struggling with this.
And, you know, it's uh it's avery different, different thing.
But it's, you know, there's thepeople that it hasn't happened
to that actually have thecapacity to do this stuff.
Those are the people I considertrue rock stars that are like,
all right, we got to changethis.
Let's fix it.
Where do we start?
Ari Berman (28:03):
Absolutely.
Absolutely.
Well, um, I guess the last bitI'll ask you is to wrap up is
how can people get involved andhelp?
Casey McPherson (28:10):
I mean, you know, we're uh always looking
for great minds that um havegifting that can help, you know,
participate and solve thisproblem.
We're also looking for moremission-driven investors.
I mean, you could easily turnour company into a large pharma
company.
Ari Berman (28:28):
Yep.
Casey McPherson (28:29):
So it's very important that our investors
align with the fact that we'reonly going to be commercializing
these ultra-rare products andwe're gonna be focused on
building the scale as opposed tojust being a traditional
company.
Um, and so, you know, we'rewe're running an equity
crowdfunding uh campaign whereanyone can invest.
Uh, you know, that's started indot com slash AlphaRose.
(28:51):
And then, you know, you canalways find us on our website
and on social media that that ifif someone's interested in
being a part of this in someway, um, you know, we're we're
looking for you.
You know, it's like the the uhthe US uh who was the old Uncle
Sam Uncle Sam Uncle Sam is like,you know, we want you because
at the end of the day, there'shundreds of millions of patients
(29:12):
waiting, and none of us can dothis alone.
So the more that we canpartner, band together, put our
resources together, Alpha Roseis merely a vessel to break open
this space.
I hope that many, many othercompanies do this like we're
doing it and and benefit from ittoo.
Ari Berman (29:28):
Awesome.
Well, I'm super excited to seewhere this goes, and I hope that
uh that I get to jump in andhelp too, because it's an
awesome, awesome effort.
So um too, Ari.
Casey McPherson (29:40):
Um we we needed many we we don't need many
excuses to work together becauseI have so much respect for you
and your team and what you guysdo.
Ari Berman (29:48):
Uh appreciate that.
Well, we're looking forward toit and thanks for being on the
podcast today.
Really appreciate it.
And uh uh I hope uh peoplereach out and get involved and
Uh realize, hey, precisionmedicine is closer than we
think.
And it's not just for theultra-rich.
Casey McPherson (30:05):
Yeah, absolutely.
Thanks for having me, Ari.
Ari Berman (30:08):
Thanks, Casey.
Take care.
Announcement (30:10):
Thank you for listening to Bio-IT World's
Trends from the Trenchespodcast.
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