July 7, 2025 • 32 mins
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This week's episode is one from the road, recorded in front of a live audience in Boston's Seaport neighborhood during the BIO conference (special thanks to MasterControl for making it happen). Amber Salzman, Ph.D., CEO of Epicrispr Biotechnologies (aka 'Epic Bio') explains how epigenetic editing is revolutionizing genetic medicine by controlling gene expression, without cutting DNA like traditional CRISPR technologies. Amber talks about FSHD, a progressive muscular dystrophy, how the company raised $68 million in Series B funding despite challenging market conditions, her partnership with Springbok Analytics for AI analysis of MRI images, working with a CDMO to manufacture a new treatment modality, and navigating the FDA during a time of disruption.
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Episode Transcript
Available transcripts are automatically generated. Complete accuracy is not guaranteed.
Ben Comer (00:00):
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(00:43):
Welcome back to the Business ofBiotech.
I'm your host, Ben Comer, chiefeditor at Life Science Leader
and today, thanks to MasterControl, we are recording from
Boston during the BIOconvention, in front of a live
studio audience.
Yeah, don't take my word for it.
Make some noise, thank you.
(01:04):
Joining me for this specialepisode taping is Amber Salzman,
Ph.
D,, CEO and President ofEpicrisper Biotechnologies, aka
Epic Bio, a company that's usingepigenetic modulators to
control gene expression andtreat complex diseases.
Amber is an experienced lifescience executive who was most
(01:25):
recently president and CEO atOhana Biosciences, working in
reproductive health, and beforethat she worked as president and
CEO at Adverium Biotechnologies, a company formed through the
merger of AvalancheBiotechnologies and Annapurna
SAS, the latter a gene therapycompany that she also led as CEO
(01:46):
.
Amber began her career at GSKand was a member of the R&D
executive team and was also aCEO at CardioKind, which was
sold to Cornerstone Therapeutics.
I'm not including all of herprofessional experiences here
for the sake of time, butsuffice it to say that Amber
knows what it takes to lead andbuild a successful startup from
(02:08):
the earliest stages.
Thank you so much for beinghere live and in person, Amber.
Amber Salzman (02:16):
Now.
Thank you for having me Reallyappreciate it.
Ben Comer (02:20):
We typically start off the business of biotech with
a little bit of background.
We typically start off thebusiness of biotech with a
little bit of background.
What initially interested youabout the biopharmaceutical
industry and maybe why youdecided to join Epic Bio as CEO?
Amber Salzman (02:34):
Sure, in some ways it's a little bit I got
lucky.
I mean, as we know, you have tobe lucky and flexible.
But I liked science andmedicine and ended up working at
GSK.
Well, I started at SmithKlinein French, ended up through
mergers becoming GSK and I wasreally surrounded by brilliant
drug developers and that's whereI really felt privileged to be
(02:58):
putting amazing medicines outfor patients.
I mean, whenever we would getthese letters from patients I
mean one that stood in my mindwas after 9-11, because you know
we did a lot in COPD After 9-11, we got a letter from a patient
saying that if it wasn't forour drug they wouldn't have been
able to escape.
So those kind of letters whenyou hear from families really
got me going.
(03:18):
Then, unfortunately, diseasesstruck my family in a pretty
harsh way, with a rareneurodegenerative disease and
having worked on all very commondiseases, I will admit that I
didn't fully appreciate thatthere was this whole other side
of drug development and raredisease that was different.
(03:39):
And when I learned that mynephew was going to pass away
and then we tested the familyand my son tested positive, as
did another nephew I realized,oh my God, I have a few years
before the bomb goes off, andthen we got to do something.
So that really launched me intolook, I have to do something, I
know I can do something,there's no other option and
(03:59):
really pushed me into drivingthings on my own and I ended up
leaving GSK and starting moresmall biotechs and really
feeling such excitement beingable to do something for so many
patients that are in need.
Ben Comer (04:14):
So what about Epic Bio?
Amber Salzman (04:15):
Yeah.
So when I talked about myfamily having a genetic disease,
I started working in geneticmedicine and this is a little
more than 20 years ago and therewere a lot of limitations in
what was available.
I mean not to put down what wasthere, but there were
limitations in what capabilitiesexisted.
And then when I interviewed, Iremember, with the scientific
(04:36):
founder Stanley Qi of Epicrispr,I still, when I think about it,
I'm not sure why they hired me.
I was like, yeah, but you can'tdo this, you can't do that.
And then still, when I thinkabout it, I'm not sure why they
(05:08):
hired me.
I was like, yeah, but you can'tdo this, you can't do that.
And they were kind of on my asssaying you did something for
your family's disease because wedid end up getting a product to
market.
Like what about us?
And it is actually anepigenetic disease by nature.
And when I interviewed with theteam at Epicrispr, there were a
few really great people thatwere there before I even joined.
They mentioned how they couldaddress FSHG and I was like, oh
(05:29):
my God, now I got to do that.
I mean, it was just, it was hadmy name on it, and I will say
the team was amazing.
I mean, we went from nothing tohaving an IND cleared in a new
modality in a little over threeyears.
So I'm I definitely made theright decision in joining the
team.
Ben Comer (05:47):
Right, I want to just let everyone know if you have a
question for Amber.
I'd love to get the audienceinvolved.
Raise your hand and I'll lookup periodically and call on you.
So let me know if you have aquestion.
You've been mentioningepigenetics.
You've been mentioningepigenetics.
I wonder if you could explainthe difference between
epigenetic editing and CRISPRediting, which people may be
(06:09):
more familiar with.
Amber Salzman (06:10):
Sure.
So first, just for the easyexplanation of the epigenome.
So I will say I have the sameDNA that I had when I was 18 and
I don't look 18.
I don't feel 18.
And that's because of theepigenetics that sit above the
DNA.
And the reason why I give thatexample is that you can really
(06:32):
control what your DNA doesthrough the epigenome, and
that's super, super powerful.
The original CRISPR applicationsthat came out used this
CRISPR-Cas molecule to bind tothe location of the DNA that
needed to be corrected and thenit would cut the DNA sometimes
double strand, sometimes singlestrand and that was an approach
(06:55):
to fix the DNA problems.
What epigenetic editing does?
It was we still use that Casmolecule to bind to the right
location in a very rapid andprecise way, but in epigenetics
we deactivate the nucleus ofthat Cas molecule so we no
longer cut the DNA and that'sreally important, and I'll get
(07:15):
to that in a minute and insteadwe fuse a gene regulator to the
Cas molecule so it tells the DNAwhat to do so it could say
don't express that protein or doexpress the protein at a higher
level.
So you're controlling what theDNA does rather than cutting it,
and the reason why that's superimportant is when you cut DNA,
(07:37):
you really risk genomicinstability.
So this is a safe way tocontrol what your DNA does and
it also gives you a lot ofprecise modulation.
So you don't have to just cutit out completely.
Sometimes you just have tolower the expression, not cut it
out.
Or you want to like for anhaploid-sufficient disease, you
(07:59):
want to double what the goodallele does and not more,
because it can be toxic becauseit can be toxic.
Ben Comer (08:05):
I want to switch over to funding, because I was in a
session yesterday on cancer andfunding for cancer therapies and
it was a panel of big pharmafolks BD development and they
essentially said we're notlooking at it if it's not phase
three or marketed product.
I don't need to tell theaudience of Business of Biotech
(08:28):
that it's a very challengingtime for funding early stage
companies.
However, epic Bio was able toraise $68 million in a Series B
last March with a novel modalityand despite this incredibly
hard funding funding environment, how did you do that, amber?
Amber Salzman (08:49):
Well, part of it was that while we were, we had
this wonderfully differentiatedplatform.
You know, a few years ago thatwas sort of in vogue, had this
wonderful huge platform that cando anything.
What we did and part of it isjust my drive to go after
patient needs.
So when I joined the company,I'm like great platform, what
are we doing for patients?
(09:10):
So it just worked out well thatwe were going to actually have
indications in the pipeline.
So investors really, inparticular last year and more
recently, they don't want tojust see the hype of what a
platform can do.
They want to see it applied toindications.
So that was one thing thatreally helped us that we had
(09:30):
really phenomenal preclinicaldata that we could talk about
and that we have otherindications behind it.
Also, we were super and aresuper capital efficient.
So there's other people in ourspace who raised earlier a lot
of money and it's not good thatthey're able to show that they
(09:52):
have the indications movingforward.
I mean there's other thingsthey're doing that is great.
It was just they got kind ofcaught in the crash.
But we have always, from thestart, been very capital
efficient and really focused onhow do we quickly and safely get
transformational medicine outthere.
So I think that that's whatreally helped.
Ben Comer (10:12):
How do you target investors for a company?
I mean, do you, do you haverelationships from previous
companies that you reach out tofirst?
Can you give us, I guess, justa sense of how you collect that
investor base?
Amber Salzman (10:27):
Yeah, I mean I always joke when I say if you
make an investor money, youcould be an expert or they think
you're great.
So I don't depend on thatthough, but I do so.
Clearly, investors that I'veworked with in the past reach
out to them and see if what thiscompany is doing fits their
investment profile.
So I will say, reaching out toinvestors, you know there's I
(10:51):
hate to say it the usualsuspects and talk to them, but I
will say we're in anenvironment where, as I say to
the team, after we are kind ofexhausted from all the pitching.
You got to kiss a lot of frogsin this environment and that's
how we did it.
You just talk to people and youknow, I'll be honest.
Sometimes you get the feedbackof like, wow, your data is
(11:11):
amazing, your platform isamazing, but I don't know, it's
not the right climate.
We want to only do publics now.
Like it's tough right now.
We have our own portfolios toworry about, but then you do get
investors who are looking at itand saying, wow, the data is
amazing, when you can be isamazing.
(11:31):
I want to get in now so thatwhen you have all the data come
out, I'm in with the winningcompany and it takes.
I mean, I don't, I'm not beingcritical of investors it takes a
lot of guts to, you know, notjust follow what everyone else
is doing and really say like,wow, I did the diligence, this
is amazing, I'm going to go withit.
So you, just as I said, kiss alot of frogs.
(11:54):
I don't want to say it in aderogatory way.
Look, there'll be other times.
We have relationships with themand a lot of them.
I know that as we move forwardat later stage we'll go back.
It's always a good engagementand they're excited about what
we're doing, so we'll use amiddle later funding round.
Ben Comer (12:12):
EPI 321 is EPIC's lead development candidate.
It targets FSHD, which youmentioned.
I was not familiar with thatdisease prior to learning about
Epic Bio.
I wonder if you could just Idon't know briefly tell us about
the disease and the unmet need.
Amber Salzman (12:30):
It's really a debilitating disease.
It starts as the name saysusually and there's a
variability in the face.
So you hear people who, as theystart to get worse, say oh, my
mom told me when I was a baby Inever closed my eyes or I
couldn't smile.
So it starts in the face, whereyou lose muscles there, and
then it works down to your upperbody, your scapula, and then in
(12:53):
many of the patients they endup non-ambulating and needing
wheelchairs.
But it's interesting because insome ways it can be an
invisible disability, like I wasat an FSHD meeting last week
and you know I would sit next toa few patients and just sitting
at the table, you're likethere's nothing wrong with them
and then you see how they haveto like drink a cup of water.
(13:13):
They can't just lift their arm,they need help.
They can't brush their hair.
Somebody's got to help them.
I mean, I know that sounds likea big deal, but it's really
tough.
It changes your whole qualityof life and how you think about
things.
And I can say in my family inparticular one of my
cousins-in-laws who really had awonderful career in NFL films
(13:36):
as a film director.
As he lost more and moreability, you know, they let him
work from home.
He was in a wheelchair, workedremotely and then he just he.
He had to stop working at areasonably young age, a career
that he loved.
So it's very debilitating, Imean as a wonderful wife who
does so much for him.
But it's hard to like be sodependent on your family to be
(13:59):
able to do things.
So it's variable, but it isreally affects your quality of
life.
Ben Comer (14:07):
I'm switching, I'm bouncing around here a little
bit.
I hope that's okay.
I did want to ask you aboutEpic's partner, springbok
Analytics.
This is something that juststuck out to me when I read
about it, because it's in, it'sa I think it's an AI partnership
and it's looking at I thinkit's analyzing MRI muscle
(14:28):
imaging.
But let me, let me let youexplain it.
Amber Salzman (14:30):
So, as I said, I kind of gave the typical
manifestation.
But some patients progressfaster in their legs than their
upper body and it varies.
So if you're running a clinicaltrial, imagine an endpoint that
measures how quickly they do a10-meter walk run.
Well, that may be a really goodendpoint for people who it
(14:50):
affects the lower body, but youmay not see anything in people
that it's more rapid in theirupper body.
Just use that as an example.
What's nice about head-bodyimaging is what Springbok has
done is they've made use ofnatural history data from FSHG
patients and they're able tolook overall for all of your
(15:13):
muscles how has the fat fractionchanged?
How's the inflammation, musclemass and then they correlated
that data with functionalmeasures not just 10 meter walk
(15:45):
run, but timed up and go a wholebunch of different measures to
see how does the fat fractionrelate to functional outcomes.
And where that's super powerfulfor us is that we can then have
quote unquote a digital twinfor the patients on treatment.
Now it's early days and theyhave to obviously spend more
(16:06):
time refining their model.
Good news is that there's moredata coming in that they can do
the machine learning so thatthey can even be more predictive
.
Do the machine learning so thatthey can even be more
predictive.
So that's where the machinelearning comes in to really
understand overall for this kindof profile of muscle.
How does that relate tofunctional?
Ben Comer (16:26):
measure.
Amber Salzman (16:26):
And our aspiration is to be able to use
that to go to regulatoryagencies and say this is how we
impacted fat fraction, forexample.
And you know, the agency wantsto know that you made somebody a
little longer feel better andaddress the disease in a
measurable way.
It's like how does this MRImean anything to a patient?
This does that kind of tie in.
(16:47):
Now we have other measures thatwe're looking at as biomarkers,
but that's just one of theapproaches to help us see what's
going on.
Ben Comer (16:56):
Have you had any initial conversations with
regulators about potentiallyusing that data?
Amber Salzman (17:03):
So we have not talked to them about using the
MRI data.
We have talked to them aboutusing methylation data.
So just to pull us back toepigenetics, so when I said you
want to change the way the DNAbehaves, well, when it comes to
FSHD, there's a certain regionthat expresses this Dux4 protein
(17:27):
, which has been known to betoxic to the muscles.
What we do is we go in andmethylate that region, which
means we tighten it up so thatthe Dux4 doesn't leak out.
What's known about this diseaseis that your disease severity
is linked to how muchmethylation you have.
So what we did when we spoke tothe and there's cohorts of
(17:49):
hundreds of patients wherethere's tight correlation.
This has been published in thelast 10, 15 years.
So when we spoke to theregulators, we said, given all
of this and we showed them thepublications if we increase
methylation, would you agreethat's a good biomarker and
predictive of clinical benefit?
And we got a reasonable answer.
(18:10):
They said, yeah, we buy thatmore methylation means less
disease severity and potentiallyno disease.
But nobody has shown us that ifyou change methylation it
changes the course of thedisease.
So we encourage you to use yourperson-to-human study to do
that.
So we did talk to I mean again,reasonable, since nobody has
(18:32):
shown it yet.
So that is one measure that wecan use with them.
We did not have the data inhand.
This was when we did ourpre-IND.
We did not have the data inhand.
This was when we did ourpre-IND.
We did not have the data inhand from Springbok.
Also, like I said, there's morework that needs to be done, but
we're very encouraged whereit's going.
Ben Comer (18:49):
What about the development process for
manufacturing?
This is something new.
Epigenetic editing.
Amber Salzman (19:07):
I don't know what that looks like.
Did you work with a CDMO?
Or first, if you don't mind,describe just the manufacturing
process early and we're tryingto move quickly.
We always have to design with.
You know quality by design.
So what she has cautioned mebecause I'm like don't you think
that's too much for just aphase one?
(19:28):
She's like no, because if youswitch anything later on you're
going to have to end up redoingthe trial.
So let's build in the qualitynow so that when we then
commercialize it we're set up.
So she really built thoseprinciples in and worked with
the CDMO, had a greatpartnership with them so that
when we went to the FDA we couldmake use of their CMC process
(19:52):
along with our approach.
We put together the differentassays for material release et
cetera.
But it was kind of like the endgoal in mind when we worked
with them and we had a goodpartner with the CDMO and I
think that's what led to areally good feedback from the
FDA.
Ben Comer (20:08):
And that was your process.
Development person, internalperson that you were mentioning.
What's her name?
Amber Salzman (20:13):
Dipali Patel, nobody ever go after.
She's amazing.
No, she's amazing, um, no,she's.
She's great, and and part of itwas also the partnership with
the cdmo, because we're, youknow, we're small.
I guess that we're capitalefficient.
We're 35 people, um, and she atthe time I mean since then
she's hired more people andblood in the bioreactors but she
didn't have the stuff to do so,but she had the idea, so she
(20:36):
worked really well with the cMOto make it.
Ben Comer (20:39):
And the CDMO helped get you to the IND.
It's interesting my colleagueled a panel on.
You know small versus largecompanies working with CDMOs.
Sometimes the small companiesfeel like they're shafted a
little bit, not getting equaltreatment.
I mean you, your process.
Amber Salzman (20:57):
Yeah, well, I will add.
Add, I've been around a longtime so I know a lot of people.
So, like I knew the ceo of thecompany and I'm not afraid to
pick up the phone and use it um,knew a lot of people there, we
and I also.
We also treated them like theywere part of the team so we
would talk about the patientsand really like patients are
(21:19):
waiting.
You know we always bringpatients and talk to the team
and really try to get the peopleon the team at the CDML working
with us.
So I totally hear what you'resaying, that you sometimes get
shafted, but I think part of itwas.
I give a lot of credit to Dipalibecause of the way she
basically I learned this fromher when she met with them.
She goes, yes, we want you tobe a good partner, but I want to
(21:41):
be a good partner, like thatwas her whole thing.
She goes how could?
Because a lot of times they'recomplaining about us just as
much as we're complaining aboutthem.
I mean, that's how it works.
So she's like you tell me how Ican be the best partner.
I want you to cite me as theperson that you want to work
with, so that I think just setthe stage that when you partner
and you know respect each otherand there were times where she's
(22:04):
like I don't think you shouldbe doing it, here's what I
recommend.
They were like this, veryskeptical, and then she would do
small scale stuff in-house andshow them the data and we had
results like nobody else hadworking with them really good
results.
Ben Comer (22:20):
Are you at a stage of scaling up at this point?
Amber Salzman (22:24):
Yeah, so we scale , I mean you do stuff smaller.
We went through the FIOSC allthe way up to 200 liter, then
500 liter and our clinical trialwe did 1,000 liter and it turns
out we didn't even have to goto 1,000 liter because our
titers were so good and then sofrom there we'll have to look to
(22:44):
see because she's getting.
He shows ideas on how toimprove the scale up as to
whether we'll stick with 1,000liter, but our CDMO also has
5,000 liter bioreactors.
Ben Comer (22:53):
And you'll likely stay with that CDMO.
Amber Salzman (22:57):
Right now everything's going well, so yeah
, With the 68 million Series B.
Ben Comer (23:02):
What kind of runway does that give you?
Amber Salzman (23:04):
We don't usually disclose a runway.
I mean I will say we will havealready early next year we'll
have our first clinical databecause we'll have the first
patient's muscle biopsies toshow that our product is doing
what it's supposed to.
So we're excited to be able toget the data.
Ben Comer (23:21):
Yeah, that's very exciting.
What advice would you give toother biotechs about engaging
with regulators, particularlythe FDA, around an IND for a
brand new drug modality?
Amber Salzman (23:35):
It's a great question.
I will say that we because wewere so small when we first
started, we didn't even have ourinternal regulatory person.
We went to some consultingcompanies and they Our team
knows what our product reallywell.
And they were like this is whatwe should be doing.
And I mean, I'll give the badexample to learn from.
(23:57):
And the consulting company waslike no, this is what the how
the FDA does it all the time,this is how you should do it.
And we're like but it doesn'tmake sense.
But we know in our heart ofhearts this is the right way to
show that it's safe.
We know in our heart of heartsthis is the right way to show
that it's safe.
And we basically went.
I say follow the data, followthe science, and if you in your
(24:18):
heart know that that's the rightway to do it, they can always
say no.
So engage with them.
We engage with them very earlyon.
I mean just to give you anexample.
So you know we have to go afterit.
It's a human construct.
We're going after human DNA.
So when you do a safety study,that gene sequence we're going
(24:39):
after doesn't exist.
So it's not exactly alike-for-like test.
So we were getting advice fromone firm who we'd ended up
working with oh, you have tocome up with a mouse construct
so that you can bind to theright location the mouse, and
I'm like, but then we're testinga drug that is not the drug
we're going to use in humans.
Like it just didn't feel right.
(25:00):
And you know I'm not aregulatory guru, I've been
around a long time, done a lot,but I just like scientifically,
I'm just logically it didn'tfeel right.
And they were insisting,insisting I think they were even
a former FDA person.
I'm like I just don't like that.
So the team, we all kind ofbrainstormed on it and we said
let's just talk to the FDA,let's put it forward.
If they tell us to use themouse construct, we'll do it,
(25:22):
but we just don't thinkscientifically that makes sense.
So we went with them with ourproposal.
So if you ask advice, I wouldsay do what you really believe
you can defend scientificallyand that if you were dosing your
family member, this is what youwould want done.
And if you feel that stronglyabout it, you could talk to the
agency.
I mean, there's smart peoplethere For the most part, most
(25:44):
people you talk to at the FDA.
They're there because they careabout patients.
They want to get safe andeffective medicine to patients,
so they'll heal you for the mostpart.
So I not that I mean, look,humans like the rest of us and
some are better, some are worse.
That's the way it is.
But for the most part there'sreally good people there.
And I say you know, if youfollow the science, follow the
(26:06):
data and do what you think isright and talk about it.
Ben Comer (26:11):
I mentioned.
We're a bio.
John Crowley, who's a currentexecutive chief executive of bio
, interviewed FDA CommissionerMartin McAree today.
We were chatting about it alittle bit just before we
started recording.
But what's your kind of senseabout the status of the FDA
currently?
Are you concerned?
(26:31):
Are you confident?
How do you feel about thestatus of the FDA currently?
Or are you concerned?
Are you confident?
How do you feel about thecurrent FDA?
Amber Salzman (26:34):
I will say right now I'm feeling rather
optimistic.
When I first saw some of thechanges and look, we all love
Peter Marks, like those kind ofchanges kind of got me unnerved.
But then when I saw wherethings landed and some of the
stuff McAbee is saying, I meanhe's definitely shaking stuff up
but I think in a constructivelike we got to be creative.
(26:55):
I mean he said things which I'mlike God, that's so obvious.
Why didn't they do it before?
He was talking about when yousubmit a drug for approval,
there's a lot of stuff that youhave in your hands like for a
while and then you're justwaiting for the study to end and
put the data in.
So he was saying like give usthe CMC section and a bunch of
(27:17):
other stuff early, becauseyou're just sitting on that, and
then you make us review it allin 30 days and we're like heads
down, you know, trying to wellfor a 90, but for, obviously,
for an approval it's much longer.
But those type of ideas wherehe's like let's do what makes
(27:40):
sense and try to be outside thebox, the way he said we should
be taking advantage of AI andthings like that, so that I
can't disagree with we haveengaged.
What can we do differently tospeed things up and do well by
patients combined with peoplewho've been at the FDA for a
long time.
They understand what you got todo to keep things safe and all.
I think they'll end up.
(28:01):
I mean, I think it will be ahealthy you shouldn't you
shouldn't always have groupthink.
So this will avoid groupthinkand I think it could ultimately
put us in a better place.
Look, there are clearly someplaces where I'm nervous, but at
least at a big picture and inthe spaces where we play right
now, I'm actually optimistic.
Ben Comer (28:24):
All right.
Well, before I get to my lastquestion, I wanted to ask you
here.
Anyone from the audience wantto ask Amber a question while
we've got her?
This is Ben Comer cutting inafter the taping at bio.
With an apology, I failed tocapture the audience questions
on microphone and they weren'taudible on the recording, so I'm
reading the questions andmyself.
(28:45):
Here's the first one Can yousafely multiplex with an
epigenetic editing therapy, orin other words, make more than
one edit or change with a singleconstruct?
Amber Salzman (28:57):
No, it's a really good question.
So when it comes to epigenetics, it's the same type of thing.
You'll target a specific locusthat you want to change and we
can do multiplexing and in a wayit's much safer than doing that
with cutting, because thenyou're cutting all over the
place.
In a way it's much safer thandoing that with cutting because
then you're cutting all over theplace.
So we can.
You know, if you want toupregulate one gene and
(29:18):
downregulate another, you canput that all in the same
construct.
I mean, it's not trivial.
I mean, obviously doing one ata time is easier, but you can do
that all at the same time.
Again, you have to cautionwhich cells do you need to make
the changes in?
So, for example, right now forFSHD, we're targeting skeletal
muscle cells.
If you wanted me to changeeverything in the skeletal
(29:39):
muscle, great.
But if you wanted me to changesomething in the skeletal muscle
and in I don't know hepatocytes, because it's a disease, it
gets a little bit trickier interms of your delivery and we'd
have to think about the best wayto do it.
But absolutely, you canmultiplex in a very safe way.
Ben Comer (29:56):
Amber, what are your top priorities for Epic Bio for
the second half of 2025?
Amber Salzman (30:02):
Yeah, we're super excited to be in the clinic and
to be able to deliver whatcould be a transformative
medicine to FSHD patients liverwhat could be a transformative
medicine to FSHD patients.
So we're very laser focused ongetting that trial, you know,
successfully moving forward.
You know we're alreadyscreening patients and we'll be
dosing shortly.
So really, laser focused onthat.
(30:25):
We have other indication in thepipeline.
We need to move forward andthen, I'll say it, you have to
keep kissing frogs, like youalways have to worry about, you
know, your next dollar.
So we'll be spending timemaking sure that we can raise
more.
I think we have an amazingstory.
We've heard that from so many.
So now's a good time to makesure that we can go forward
(30:49):
constructively and help patients.
Ben Comer (30:52):
All right, last call, anyone.
Have a question for Amber?
Before we wrap up here, onemore Sure Is there any concern
about target population size forepigenetic editing therapies in
terms of a ceiling formanufacturing quantity or
competition from differentmodalities?
Amber Salzman (31:08):
This hasn't been a challenge right now, in
particular because our leadindication is I mean, fshd is a
fairly common rare disease, soit's, you know, going to be
billions in commercialization.
So that hasn't been an issue.
I will say that when we werereviewing the pipeline it was a
consideration of how big thepopulation was and of course we
(31:30):
got to make sure the cost ofgoods stays pretty low so that
it's not going to be millions.
I will say there's more ofthose conversations now, but
we're in a good position.
I mean, in FSHD there's beenlike three deals in the last I
don't know nine, 12 months.
You know Sanofi put whatever itwas 80 million down up front on
a real gamble.
Ben Comer (31:54):
So there's been a lot going on in this indication
space, all right.
Well, I think we're going towrap up here.
Amber, thank you so much forbeing here.
Master Control, thank you forhosting us in this beautiful
space right down here in theseaport in Boston.
Amber Salzman (32:09):
Thank you.
Thank you for having me.
I appreciate everybody comingand attending.
Ben Comer (32:13):
We've been speaking with Amber Salzman, phd, and
that's a PhD in mathematics, Ibelieve.
Ceo at EpicrisperBiotechnologies, I'm Ben Comer
and you've just listened to theBusiness of Biotech live from
the Bio Conference.
Find us and subscribe anywhereyou listen to podcasts and be
sure to check out new weeklyvideo casts of these
(32:35):
conversations every Monday underthe Business of Biotech tab at
Life Science Leader.
We'll see you next week andthank you, as always, for
listening.
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(00:43):
Welcome back to the Business ofBiotech.
I'm your host, Ben Comer, chiefeditor at Life Science Leader
and today, thanks to MasterControl, we are recording from
Boston during the BIOconvention, in front of a live
studio audience.
Yeah, don't take my word for it.
Make some noise, thank you.
(01:04):
Joining me for this specialepisode taping is Amber Salzman,
Ph.
D,, CEO and President ofEpicrisper Biotechnologies, aka
Epic Bio, a company that's usingepigenetic modulators to
control gene expression andtreat complex diseases.
Amber is an experienced lifescience executive who was most
(01:25):
recently president and CEO atOhana Biosciences, working in
reproductive health, and beforethat she worked as president and
CEO at Adverium Biotechnologies, a company formed through the
merger of AvalancheBiotechnologies and Annapurna
SAS, the latter a gene therapycompany that she also led as CEO
(01:46):
.
Amber began her career at GSKand was a member of the R&D
executive team and was also aCEO at CardioKind, which was
sold to Cornerstone Therapeutics.
I'm not including all of herprofessional experiences here
for the sake of time, butsuffice it to say that Amber
knows what it takes to lead andbuild a successful startup from
(02:08):
the earliest stages.
Thank you so much for beinghere live and in person, Amber.
Amber Salzman (02:16):
Now.
Thank you for having me Reallyappreciate it.
Ben Comer (02:20):
We typically start off the business of biotech with
a little bit of background.
We typically start off thebusiness of biotech with a
little bit of background.
What initially interested youabout the biopharmaceutical
industry and maybe why youdecided to join Epic Bio as CEO?
Amber Salzman (02:34):
Sure, in some ways it's a little bit I got
lucky.
I mean, as we know, you have tobe lucky and flexible.
But I liked science andmedicine and ended up working at
GSK.
Well, I started at SmithKlinein French, ended up through
mergers becoming GSK and I wasreally surrounded by brilliant
drug developers and that's whereI really felt privileged to be
(02:58):
putting amazing medicines outfor patients.
I mean, whenever we would getthese letters from patients I
mean one that stood in my mindwas after 9-11, because you know
we did a lot in COPD After 9-11, we got a letter from a patient
saying that if it wasn't forour drug they wouldn't have been
able to escape.
So those kind of letters whenyou hear from families really
got me going.
(03:18):
Then, unfortunately, diseasesstruck my family in a pretty
harsh way, with a rareneurodegenerative disease and
having worked on all very commondiseases, I will admit that I
didn't fully appreciate thatthere was this whole other side
of drug development and raredisease that was different.
(03:39):
And when I learned that mynephew was going to pass away
and then we tested the familyand my son tested positive, as
did another nephew I realized,oh my God, I have a few years
before the bomb goes off, andthen we got to do something.
So that really launched me intolook, I have to do something, I
know I can do something,there's no other option and
(03:59):
really pushed me into drivingthings on my own and I ended up
leaving GSK and starting moresmall biotechs and really
feeling such excitement beingable to do something for so many
patients that are in need.
Ben Comer (04:14):
So what about Epic Bio?
Amber Salzman (04:15):
Yeah.
So when I talked about myfamily having a genetic disease,
I started working in geneticmedicine and this is a little
more than 20 years ago and therewere a lot of limitations in
what was available.
I mean not to put down what wasthere, but there were
limitations in what capabilitiesexisted.
And then when I interviewed, Iremember, with the scientific
(04:36):
founder Stanley Qi of Epicrispr,I still, when I think about it,
I'm not sure why they hired me.
I was like, yeah, but you can'tdo this, you can't do that.
And then still, when I thinkabout it, I'm not sure why they
(05:08):
hired me.
I was like, yeah, but you can'tdo this, you can't do that.
And they were kind of on my asssaying you did something for
your family's disease because wedid end up getting a product to
market.
Like what about us?
And it is actually anepigenetic disease by nature.
And when I interviewed with theteam at Epicrispr, there were a
few really great people thatwere there before I even joined.
They mentioned how they couldaddress FSHG and I was like, oh
(05:29):
my God, now I got to do that.
I mean, it was just, it was hadmy name on it, and I will say
the team was amazing.
I mean, we went from nothing tohaving an IND cleared in a new
modality in a little over threeyears.
So I'm I definitely made theright decision in joining the
team.
Ben Comer (05:47):
Right, I want to just let everyone know if you have a
question for Amber.
I'd love to get the audienceinvolved.
Raise your hand and I'll lookup periodically and call on you.
So let me know if you have aquestion.
You've been mentioningepigenetics.
You've been mentioningepigenetics.
I wonder if you could explainthe difference between
epigenetic editing and CRISPRediting, which people may be
(06:09):
more familiar with.
Amber Salzman (06:10):
Sure.
So first, just for the easyexplanation of the epigenome.
So I will say I have the sameDNA that I had when I was 18 and
I don't look 18.
I don't feel 18.
And that's because of theepigenetics that sit above the
DNA.
And the reason why I give thatexample is that you can really
(06:32):
control what your DNA doesthrough the epigenome, and
that's super, super powerful.
The original CRISPR applicationsthat came out used this
CRISPR-Cas molecule to bind tothe location of the DNA that
needed to be corrected and thenit would cut the DNA sometimes
double strand, sometimes singlestrand and that was an approach
(06:55):
to fix the DNA problems.
What epigenetic editing does?
It was we still use that Casmolecule to bind to the right
location in a very rapid andprecise way, but in epigenetics
we deactivate the nucleus ofthat Cas molecule so we no
longer cut the DNA and that'sreally important, and I'll get
(07:15):
to that in a minute and insteadwe fuse a gene regulator to the
Cas molecule so it tells the DNAwhat to do so it could say
don't express that protein or doexpress the protein at a higher
level.
So you're controlling what theDNA does rather than cutting it,
and the reason why that's superimportant is when you cut DNA,
(07:37):
you really risk genomicinstability.
So this is a safe way tocontrol what your DNA does and
it also gives you a lot ofprecise modulation.
So you don't have to just cutit out completely.
Sometimes you just have tolower the expression, not cut it
out.
Or you want to like for anhaploid-sufficient disease, you
(07:59):
want to double what the goodallele does and not more,
because it can be toxic becauseit can be toxic.
Ben Comer (08:05):
I want to switch over to funding, because I was in a
session yesterday on cancer andfunding for cancer therapies and
it was a panel of big pharmafolks BD development and they
essentially said we're notlooking at it if it's not phase
three or marketed product.
I don't need to tell theaudience of Business of Biotech
(08:28):
that it's a very challengingtime for funding early stage
companies.
However, epic Bio was able toraise $68 million in a Series B
last March with a novel modalityand despite this incredibly
hard funding funding environment, how did you do that, amber?
Amber Salzman (08:49):
Well, part of it was that while we were, we had
this wonderfully differentiatedplatform.
You know, a few years ago thatwas sort of in vogue, had this
wonderful huge platform that cando anything.
What we did and part of it isjust my drive to go after
patient needs.
So when I joined the company,I'm like great platform, what
are we doing for patients?
(09:10):
So it just worked out well thatwe were going to actually have
indications in the pipeline.
So investors really, inparticular last year and more
recently, they don't want tojust see the hype of what a
platform can do.
They want to see it applied toindications.
So that was one thing thatreally helped us that we had
(09:30):
really phenomenal preclinicaldata that we could talk about
and that we have otherindications behind it.
Also, we were super and aresuper capital efficient.
So there's other people in ourspace who raised earlier a lot
of money and it's not good thatthey're able to show that they
(09:52):
have the indications movingforward.
I mean there's other thingsthey're doing that is great.
It was just they got kind ofcaught in the crash.
But we have always, from thestart, been very capital
efficient and really focused onhow do we quickly and safely get
transformational medicine outthere.
So I think that that's whatreally helped.
Ben Comer (10:12):
How do you target investors for a company?
I mean, do you, do you haverelationships from previous
companies that you reach out tofirst?
Can you give us, I guess, justa sense of how you collect that
investor base?
Amber Salzman (10:27):
Yeah, I mean I always joke when I say if you
make an investor money, youcould be an expert or they think
you're great.
So I don't depend on thatthough, but I do so.
Clearly, investors that I'veworked with in the past reach
out to them and see if what thiscompany is doing fits their
investment profile.
So I will say, reaching out toinvestors, you know there's I
(10:51):
hate to say it the usualsuspects and talk to them, but I
will say we're in anenvironment where, as I say to
the team, after we are kind ofexhausted from all the pitching.
You got to kiss a lot of frogsin this environment and that's
how we did it.
You just talk to people and youknow, I'll be honest.
Sometimes you get the feedbackof like, wow, your data is
(11:11):
amazing, your platform isamazing, but I don't know, it's
not the right climate.
We want to only do publics now.
Like it's tough right now.
We have our own portfolios toworry about, but then you do get
investors who are looking at itand saying, wow, the data is
amazing, when you can be isamazing.
(11:31):
I want to get in now so thatwhen you have all the data come
out, I'm in with the winningcompany and it takes.
I mean, I don't, I'm not beingcritical of investors it takes a
lot of guts to, you know, notjust follow what everyone else
is doing and really say like,wow, I did the diligence, this
is amazing, I'm going to go withit.
So you, just as I said, kiss alot of frogs.
(11:54):
I don't want to say it in aderogatory way.
Look, there'll be other times.
We have relationships with themand a lot of them.
I know that as we move forwardat later stage we'll go back.
It's always a good engagementand they're excited about what
we're doing, so we'll use amiddle later funding round.
Ben Comer (12:12):
EPI 321 is EPIC's lead development candidate.
It targets FSHD, which youmentioned.
I was not familiar with thatdisease prior to learning about
Epic Bio.
I wonder if you could just Idon't know briefly tell us about
the disease and the unmet need.
Amber Salzman (12:30):
It's really a debilitating disease.
It starts as the name saysusually and there's a
variability in the face.
So you hear people who, as theystart to get worse, say oh, my
mom told me when I was a baby Inever closed my eyes or I
couldn't smile.
So it starts in the face, whereyou lose muscles there, and
then it works down to your upperbody, your scapula, and then in
(12:53):
many of the patients they endup non-ambulating and needing
wheelchairs.
But it's interesting because insome ways it can be an
invisible disability, like I wasat an FSHD meeting last week
and you know I would sit next toa few patients and just sitting
at the table, you're likethere's nothing wrong with them
and then you see how they haveto like drink a cup of water.
(13:13):
They can't just lift their arm,they need help.
They can't brush their hair.
Somebody's got to help them.
I mean, I know that sounds likea big deal, but it's really
tough.
It changes your whole qualityof life and how you think about
things.
And I can say in my family inparticular one of my
cousins-in-laws who really had awonderful career in NFL films
(13:36):
as a film director.
As he lost more and moreability, you know, they let him
work from home.
He was in a wheelchair, workedremotely and then he just he.
He had to stop working at areasonably young age, a career
that he loved.
So it's very debilitating, Imean as a wonderful wife who
does so much for him.
But it's hard to like be sodependent on your family to be
(13:59):
able to do things.
So it's variable, but it isreally affects your quality of
life.
Ben Comer (14:07):
I'm switching, I'm bouncing around here a little
bit.
I hope that's okay.
I did want to ask you aboutEpic's partner, springbok
Analytics.
This is something that juststuck out to me when I read
about it, because it's in, it'sa I think it's an AI partnership
and it's looking at I thinkit's analyzing MRI muscle
(14:28):
imaging.
But let me, let me let youexplain it.
Amber Salzman (14:30):
So, as I said, I kind of gave the typical
manifestation.
But some patients progressfaster in their legs than their
upper body and it varies.
So if you're running a clinicaltrial, imagine an endpoint that
measures how quickly they do a10-meter walk run.
Well, that may be a really goodendpoint for people who it
(14:50):
affects the lower body, but youmay not see anything in people
that it's more rapid in theirupper body.
Just use that as an example.
What's nice about head-bodyimaging is what Springbok has
done is they've made use ofnatural history data from FSHG
patients and they're able tolook overall for all of your
(15:13):
muscles how has the fat fractionchanged?
How's the inflammation, musclemass and then they correlated
that data with functionalmeasures not just 10 meter walk
(15:45):
run, but timed up and go a wholebunch of different measures to
see how does the fat fractionrelate to functional outcomes.
And where that's super powerfulfor us is that we can then have
quote unquote a digital twinfor the patients on treatment.
Now it's early days and theyhave to obviously spend more
(16:06):
time refining their model.
Good news is that there's moredata coming in that they can do
the machine learning so thatthey can even be more predictive
.
Do the machine learning so thatthey can even be more
predictive.
So that's where the machinelearning comes in to really
understand overall for this kindof profile of muscle.
How does that relate tofunctional?
Ben Comer (16:26):
measure.
Amber Salzman (16:26):
And our aspiration is to be able to use
that to go to regulatoryagencies and say this is how we
impacted fat fraction, forexample.
And you know, the agency wantsto know that you made somebody a
little longer feel better andaddress the disease in a
measurable way.
It's like how does this MRImean anything to a patient?
This does that kind of tie in.
(16:47):
Now we have other measures thatwe're looking at as biomarkers,
but that's just one of theapproaches to help us see what's
going on.
Ben Comer (16:56):
Have you had any initial conversations with
regulators about potentiallyusing that data?
Amber Salzman (17:03):
So we have not talked to them about using the
MRI data.
We have talked to them aboutusing methylation data.
So just to pull us back toepigenetics, so when I said you
want to change the way the DNAbehaves, well, when it comes to
FSHD, there's a certain regionthat expresses this Dux4 protein
(17:27):
, which has been known to betoxic to the muscles.
What we do is we go in andmethylate that region, which
means we tighten it up so thatthe Dux4 doesn't leak out.
What's known about this diseaseis that your disease severity
is linked to how muchmethylation you have.
So what we did when we spoke tothe and there's cohorts of
(17:49):
hundreds of patients wherethere's tight correlation.
This has been published in thelast 10, 15 years.
So when we spoke to theregulators, we said, given all
of this and we showed them thepublications if we increase
methylation, would you agreethat's a good biomarker and
predictive of clinical benefit?
And we got a reasonable answer.
(18:10):
They said, yeah, we buy thatmore methylation means less
disease severity and potentiallyno disease.
But nobody has shown us that ifyou change methylation it
changes the course of thedisease.
So we encourage you to use yourperson-to-human study to do
that.
So we did talk to I mean again,reasonable, since nobody has
(18:32):
shown it yet.
So that is one measure that wecan use with them.
We did not have the data inhand.
This was when we did ourpre-IND.
We did not have the data inhand.
This was when we did ourpre-IND.
We did not have the data inhand from Springbok.
Also, like I said, there's morework that needs to be done, but
we're very encouraged whereit's going.
Ben Comer (18:49):
What about the development process for
manufacturing?
This is something new.
Epigenetic editing.
Amber Salzman (19:07):
I don't know what that looks like.
Did you work with a CDMO?
Or first, if you don't mind,describe just the manufacturing
process early and we're tryingto move quickly.
We always have to design with.
You know quality by design.
So what she has cautioned mebecause I'm like don't you think
that's too much for just aphase one?
(19:28):
She's like no, because if youswitch anything later on you're
going to have to end up redoingthe trial.
So let's build in the qualitynow so that when we then
commercialize it we're set up.
So she really built thoseprinciples in and worked with
the CDMO, had a greatpartnership with them so that
when we went to the FDA we couldmake use of their CMC process
(19:52):
along with our approach.
We put together the differentassays for material release et
cetera.
But it was kind of like the endgoal in mind when we worked
with them and we had a goodpartner with the CDMO and I
think that's what led to areally good feedback from the
FDA.
Ben Comer (20:08):
And that was your process.
Development person, internalperson that you were mentioning.
What's her name?
Amber Salzman (20:13):
Dipali Patel, nobody ever go after.
She's amazing.
No, she's amazing, um, no,she's.
She's great, and and part of itwas also the partnership with
the cdmo, because we're, youknow, we're small.
I guess that we're capitalefficient.
We're 35 people, um, and she atthe time I mean since then
she's hired more people andblood in the bioreactors but she
didn't have the stuff to do so,but she had the idea, so she
(20:36):
worked really well with the cMOto make it.
Ben Comer (20:39):
And the CDMO helped get you to the IND.
It's interesting my colleagueled a panel on.
You know small versus largecompanies working with CDMOs.
Sometimes the small companiesfeel like they're shafted a
little bit, not getting equaltreatment.
I mean you, your process.
Amber Salzman (20:57):
Yeah, well, I will add.
Add, I've been around a longtime so I know a lot of people.
So, like I knew the ceo of thecompany and I'm not afraid to
pick up the phone and use it um,knew a lot of people there, we
and I also.
We also treated them like theywere part of the team so we
would talk about the patientsand really like patients are
(21:19):
waiting.
You know we always bringpatients and talk to the team
and really try to get the peopleon the team at the CDML working
with us.
So I totally hear what you'resaying, that you sometimes get
shafted, but I think part of itwas.
I give a lot of credit to Dipalibecause of the way she
basically I learned this fromher when she met with them.
She goes, yes, we want you tobe a good partner, but I want to
(21:41):
be a good partner, like thatwas her whole thing.
She goes how could?
Because a lot of times they'recomplaining about us just as
much as we're complaining aboutthem.
I mean, that's how it works.
So she's like you tell me how Ican be the best partner.
I want you to cite me as theperson that you want to work
with, so that I think just setthe stage that when you partner
and you know respect each otherand there were times where she's
(22:04):
like I don't think you shouldbe doing it, here's what I
recommend.
They were like this, veryskeptical, and then she would do
small scale stuff in-house andshow them the data and we had
results like nobody else hadworking with them really good
results.
Ben Comer (22:20):
Are you at a stage of scaling up at this point?
Amber Salzman (22:24):
Yeah, so we scale , I mean you do stuff smaller.
We went through the FIOSC allthe way up to 200 liter, then
500 liter and our clinical trialwe did 1,000 liter and it turns
out we didn't even have to goto 1,000 liter because our
titers were so good and then sofrom there we'll have to look to
(22:44):
see because she's getting.
He shows ideas on how toimprove the scale up as to
whether we'll stick with 1,000liter, but our CDMO also has
5,000 liter bioreactors.
Ben Comer (22:53):
And you'll likely stay with that CDMO.
Amber Salzman (22:57):
Right now everything's going well, so yeah
, With the 68 million Series B.
Ben Comer (23:02):
What kind of runway does that give you?
Amber Salzman (23:04):
We don't usually disclose a runway.
I mean I will say we will havealready early next year we'll
have our first clinical databecause we'll have the first
patient's muscle biopsies toshow that our product is doing
what it's supposed to.
So we're excited to be able toget the data.
Ben Comer (23:21):
Yeah, that's very exciting.
What advice would you give toother biotechs about engaging
with regulators, particularlythe FDA, around an IND for a
brand new drug modality?
Amber Salzman (23:35):
It's a great question.
I will say that we because wewere so small when we first
started, we didn't even have ourinternal regulatory person.
We went to some consultingcompanies and they Our team
knows what our product reallywell.
And they were like this is whatwe should be doing.
And I mean, I'll give the badexample to learn from.
(23:57):
And the consulting company waslike no, this is what the how
the FDA does it all the time,this is how you should do it.
And we're like but it doesn'tmake sense.
But we know in our heart ofhearts this is the right way to
show that it's safe.
We know in our heart of heartsthis is the right way to show
that it's safe.
And we basically went.
I say follow the data, followthe science, and if you in your
(24:18):
heart know that that's the rightway to do it, they can always
say no.
So engage with them.
We engage with them very earlyon.
I mean just to give you anexample.
So you know we have to go afterit.
It's a human construct.
We're going after human DNA.
So when you do a safety study,that gene sequence we're going
(24:39):
after doesn't exist.
So it's not exactly alike-for-like test.
So we were getting advice fromone firm who we'd ended up
working with oh, you have tocome up with a mouse construct
so that you can bind to theright location the mouse, and
I'm like, but then we're testinga drug that is not the drug
we're going to use in humans.
Like it just didn't feel right.
(25:00):
And you know I'm not aregulatory guru, I've been
around a long time, done a lot,but I just like scientifically,
I'm just logically it didn'tfeel right.
And they were insisting,insisting I think they were even
a former FDA person.
I'm like I just don't like that.
So the team, we all kind ofbrainstormed on it and we said
let's just talk to the FDA,let's put it forward.
If they tell us to use themouse construct, we'll do it,
(25:22):
but we just don't thinkscientifically that makes sense.
So we went with them with ourproposal.
So if you ask advice, I wouldsay do what you really believe
you can defend scientificallyand that if you were dosing your
family member, this is what youwould want done.
And if you feel that stronglyabout it, you could talk to the
agency.
I mean, there's smart peoplethere For the most part, most
(25:44):
people you talk to at the FDA.
They're there because they careabout patients.
They want to get safe andeffective medicine to patients,
so they'll heal you for the mostpart.
So I not that I mean, look,humans like the rest of us and
some are better, some are worse.
That's the way it is.
But for the most part there'sreally good people there.
And I say you know, if youfollow the science, follow the
(26:06):
data and do what you think isright and talk about it.
Ben Comer (26:11):
I mentioned.
We're a bio.
John Crowley, who's a currentexecutive chief executive of bio
, interviewed FDA CommissionerMartin McAree today.
We were chatting about it alittle bit just before we
started recording.
But what's your kind of senseabout the status of the FDA
currently?
Are you concerned?
(26:31):
Are you confident?
How do you feel about thestatus of the FDA currently?
Or are you concerned?
Are you confident?
How do you feel about thecurrent FDA?
Amber Salzman (26:34):
I will say right now I'm feeling rather
optimistic.
When I first saw some of thechanges and look, we all love
Peter Marks, like those kind ofchanges kind of got me unnerved.
But then when I saw wherethings landed and some of the
stuff McAbee is saying, I meanhe's definitely shaking stuff up
but I think in a constructivelike we got to be creative.
(26:55):
I mean he said things which I'mlike God, that's so obvious.
Why didn't they do it before?
He was talking about when yousubmit a drug for approval,
there's a lot of stuff that youhave in your hands like for a
while and then you're justwaiting for the study to end and
put the data in.
So he was saying like give usthe CMC section and a bunch of
(27:17):
other stuff early, becauseyou're just sitting on that, and
then you make us review it allin 30 days and we're like heads
down, you know, trying to wellfor a 90, but for, obviously,
for an approval it's much longer.
But those type of ideas wherehe's like let's do what makes
(27:40):
sense and try to be outside thebox, the way he said we should
be taking advantage of AI andthings like that, so that I
can't disagree with we haveengaged.
What can we do differently tospeed things up and do well by
patients combined with peoplewho've been at the FDA for a
long time.
They understand what you got todo to keep things safe and all.
I think they'll end up.
(28:01):
I mean, I think it will be ahealthy you shouldn't you
shouldn't always have groupthink.
So this will avoid groupthinkand I think it could ultimately
put us in a better place.
Look, there are clearly someplaces where I'm nervous, but at
least at a big picture and inthe spaces where we play right
now, I'm actually optimistic.
Ben Comer (28:24):
All right.
Well, before I get to my lastquestion, I wanted to ask you
here.
Anyone from the audience wantto ask Amber a question while
we've got her?
This is Ben Comer cutting inafter the taping at bio.
With an apology, I failed tocapture the audience questions
on microphone and they weren'taudible on the recording, so I'm
reading the questions andmyself.
(28:45):
Here's the first one Can yousafely multiplex with an
epigenetic editing therapy, orin other words, make more than
one edit or change with a singleconstruct?
Amber Salzman (28:57):
No, it's a really good question.
So when it comes to epigenetics, it's the same type of thing.
You'll target a specific locusthat you want to change and we
can do multiplexing and in a wayit's much safer than doing that
with cutting, because thenyou're cutting all over the
place.
In a way it's much safer thandoing that with cutting because
then you're cutting all over theplace.
So we can.
You know, if you want toupregulate one gene and
(29:18):
downregulate another, you canput that all in the same
construct.
I mean, it's not trivial.
I mean, obviously doing one ata time is easier, but you can do
that all at the same time.
Again, you have to cautionwhich cells do you need to make
the changes in?
So, for example, right now forFSHD, we're targeting skeletal
muscle cells.
If you wanted me to changeeverything in the skeletal
(29:39):
muscle, great.
But if you wanted me to changesomething in the skeletal muscle
and in I don't know hepatocytes, because it's a disease, it
gets a little bit trickier interms of your delivery and we'd
have to think about the best wayto do it.
But absolutely, you canmultiplex in a very safe way.
Ben Comer (29:56):
Amber, what are your top priorities for Epic Bio for
the second half of 2025?
Amber Salzman (30:02):
Yeah, we're super excited to be in the clinic and
to be able to deliver whatcould be a transformative
medicine to FSHD patients liverwhat could be a transformative
medicine to FSHD patients.
So we're very laser focused ongetting that trial, you know,
successfully moving forward.
You know we're alreadyscreening patients and we'll be
dosing shortly.
So really, laser focused onthat.
(30:25):
We have other indication in thepipeline.
We need to move forward andthen, I'll say it, you have to
keep kissing frogs, like youalways have to worry about, you
know, your next dollar.
So we'll be spending timemaking sure that we can raise
more.
I think we have an amazingstory.
We've heard that from so many.
So now's a good time to makesure that we can go forward
(30:49):
constructively and help patients.
Ben Comer (30:52):
All right, last call, anyone.
Have a question for Amber?
Before we wrap up here, onemore Sure Is there any concern
about target population size forepigenetic editing therapies in
terms of a ceiling formanufacturing quantity or
competition from differentmodalities?
Amber Salzman (31:08):
This hasn't been a challenge right now, in
particular because our leadindication is I mean, fshd is a
fairly common rare disease, soit's, you know, going to be
billions in commercialization.
So that hasn't been an issue.
I will say that when we werereviewing the pipeline it was a
consideration of how big thepopulation was and of course we
(31:30):
got to make sure the cost ofgoods stays pretty low so that
it's not going to be millions.
I will say there's more ofthose conversations now, but
we're in a good position.
I mean, in FSHD there's beenlike three deals in the last I
don't know nine, 12 months.
You know Sanofi put whatever itwas 80 million down up front on
a real gamble.
Ben Comer (31:54):
So there's been a lot going on in this indication
space, all right.
Well, I think we're going towrap up here.
Amber, thank you so much forbeing here.
Master Control, thank you forhosting us in this beautiful
space right down here in theseaport in Boston.
Amber Salzman (32:09):
Thank you.
Thank you for having me.
I appreciate everybody comingand attending.
Ben Comer (32:13):
We've been speaking with Amber Salzman, phd, and
that's a PhD in mathematics, Ibelieve.
Ceo at EpicrisperBiotechnologies, I'm Ben Comer
and you've just listened to theBusiness of Biotech live from
the Bio Conference.
Find us and subscribe anywhereyou listen to podcasts and be
sure to check out new weeklyvideo casts of these
(32:35):
conversations every Monday underthe Business of Biotech tab at
Life Science Leader.
We'll see you next week andthank you, as always, for
listening.
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