April 16, 2025 • 12 mins
This week on BioTech Nation, Sean Ainsworth, CEO of ImmuSoft, explains how modifying the body’s own B cells to produce therapeutic proteins could revolutionize drug delivery. From improving treatment for rare diseases like Hurler syndrome to potentially replacing injections for autoimmune and metabolic conditions, this new approach opens the door to long-term, patient-specific solutions.
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Transcript
Episode Transcript
Available transcripts are automatically generated. Complete accuracy is not guaranteed.
Dr. Moira Gunn (00:11):
When you take your medicine, how you have to
take your prescribed drugs canbe hugely important. Is it a
simple pill, an injection, anasal spray, and very time
consuming, is it an infusionwhere you sit for several hours
with the vital therapeutic youneed being minutely introduced
(00:33):
into your bloodstream every fewweeks? Certainly any effort to
develop new ways to deliverdrugs is important, and it is
even more important when youhave a chronic condition where
you must have an essentialmedicine every day for the rest
of your life. Both a new way todeliver therapeutics and the
(00:54):
creation of the new therapeuticsthemselves is exactly what
ImmuSoft of Seattle is workingon. Sean Ainsworth is its CEO.
Sean, welcome to the program.
Sean Ainsworth (01:07):
Thanks so much, Moira. Thanks for having me on.
Dr. Moira Gunn (01:09):
We're all used to taking pills and injections
and even infusions into ourbloodstreams, and they all have
their advantages anddisadvantages. But who isn't
open to finding better ways todeliver drugs? And today, we're
talking about a new way todeliver drugs, and that's by
modifying something that occursnaturally in our bodies. They're
(01:33):
called B cells. What are Bcells?
Sean Ainsworth (01:36):
B cells are immune cells that are
responsible for producingantibodies. Antibodies against,
we can think of vaccines orviruses. And so that's what
gives us long lasting immunitythese different pathogens. And
we're taking these B cells andwe're adding the genes that
(01:56):
encode for proteins. And a lotof drugs are really just
proteins and so these aretherapeutic proteins that the B
cells then produce so when wegive them back to the patient,
they're naturally producing andreleasing those therapeutic
proteins into the bloodstream.
Dr. Moira Gunn (02:15):
Now obviously a lot of people take drugs all the
time. Does that mean all our Bcells are interchangeable?
Sean Ainsworth (02:24):
So they're going to be patient specific. In other
words, I can't take B cells frommyself and give them to somebody
else or vice versa. But we canindeed take a patient's B cells,
program those outside the body,and once they're producing those
(02:45):
therapeutic proteins, them backto that very same patient.
Dr. Moira Gunn (02:49):
Now importantly, while your specific B cells are
needed, ImmuSoft has beenworking on a general way to
modify B cells to make specificproteins. And before we get
there, the first medicaltreatment you've been working on
is Hurler syndrome. Tell usabout that.
Sean Ainsworth (03:08):
So Hurler syndrome is caused by a
defective gene called IDUA. Andwhen IDUA isn't working
properly, it leads to thingslike stiffness in your joints
amongst many other profoundimplications that follow these
(03:29):
patients really from birth andthrough to their entire lives if
not properly treated. So whenthese patients are affected by
that defective gene, it reallycauses a lot of issues from
joint stiffness that's quitesevere, that affects their
(03:49):
ability to walk. It also impactstheir cognitive function as well
as their heart, their liver,their spleen. It's really quite
profound, the impact on theseindividuals, and it starts in
childhood and follows themthrough their entire life.
Dr. Moira Gunn (04:09):
So for this treatment that ImmuSoft is
developing, you took B cellsfrom a particular first
candidate human here, and youtook them out and you made a
modification, to the B cell andyou gave it back to the human.
What was the modification?
Sean Ainsworth (04:26):
The modification was the addition of that fully
functioning IDUA gene.
Dr. Moira Gunn (04:32):
And that's it?
Sean Ainsworth (04:33):
That's it, yeah.
Dr. Moira Gunn (04:34):
You've just given them the gene, the ability
to produce the gene from those Bcells.
Sean Ainsworth (04:39):
Correct, correct. So at the cellular
level, that's the bigmodification. Once we give those
cells back to the patient, theymigrate to the bone marrow and
they begin producing thattherapeutic protein in mass
quantities and potentially formany, many years. And so I
(05:00):
believe you can imagine that 20receiving that therapeutic
protein will have a prettydramatic impact on the patient's
lives.
Dr. Moira Gunn (05:10):
So you're going slowly here as you must when you
make such a change to a humanbody. And I know you've started
in adults. In fact, you startedin just one adult over a year
ago and are about to start yoursecond adult, being very careful
here. What's been the experiencewith this first adult with
Hurler syndrome?
Sean Ainsworth (05:31):
So his experience has been quite
dramatic. And so we've seenimprovements in his ability to
walk. And to put some numbersaround it, we do what is called
a six minute walk test. And overthe course of six minutes, he
had a roughly two sixty yardimprovement, which is quite
(05:56):
extraordinary. Typically seeingsomewhere in the 30 plus minus
range is very impactful forthese patients.
He has also, reported being ableto do things like sit with his
legs crossed, he can reach hisback now, which he previously
couldn't do, he can betterstraighten his fingers. And so
(06:20):
these might not sound terriblydramatic, but you can imagine if
you've got a buildup ofmaterials in your joints that's
affecting your ability to movefreely, really does cause pain
and to remove some of thatmaterial and improve the
flexibility, improve the painprofile, it's quite impactful
(06:45):
for these patients.
Dr. Moira Gunn (06:46):
Now, it's very exciting to think, boy, that
gene is in there producingproteins that actually will they
will remove these, which they doevery day in most people. And
now they're removing even oldones, which is very exciting to
think about. But there's alsothe you get a little excited
(07:09):
when you say, Can you stop thecells from producing these
therapeutic proteins?
Sean Ainsworth (07:14):
We do believe that we can go in and remove the
cells if we had to. Sothankfully we haven't yet, but
there are drugs that aretargeted towards B cells, which
is actually another prettyunique aspect of what we do. So
other modalities are not quiteso easy to reverse, but we think
(07:35):
that we've got that opportunitywith our approach.
Dr. Moira Gunn (07:39):
And I know you're continuing with the
hurler syndrome and beingcareful with working with the
FDA here to make sure that wedon't get ahead of ourselves.
But as I said earlier, ImmuSofthas created a general way to
modify B cells. What else mightyou use this same B cell
modifying technology for?
Sean Ainsworth (07:59):
So we're hopeful that we can use it across a
broad number of differentprotein types. And at the outset
I mentioned that B cellsnaturally produce antibodies.
And so those comprise a prettybroad swath of different
therapeutics that are already inexistence. So if we think about,
(08:20):
for example, Humira and SKYRIZI,those are both very broadly used
in the autoimmune space and itcould potentially be delivered
from B cells. And then we canalso think of, for example, the
GLP-one approaches, which areOzempic, Wegovy, those are also
(08:46):
essentially therapeutic proteinswhich we can deliver from B
cells.
So there's really a broad numberof different areas we could
pursue with, with this B cellapproach.
Dr. Moira Gunn (09:00):
So in some ways, like what you're working on now
with hurlers, you are talkingabout inserting genes that would
modify a mutated, you know,problematic gene in someone's
body. And in in other ways, youwould actually be using it to
put genes in that would createbrand new drugs that literally
(09:23):
have a therapeutic effect.
Sean Ainsworth (09:25):
Indeed. Indeed, yes. And that's the beauty of
these gene therapeuticapproaches. They did start
typically with attempting tocorrect for defective genes, but
as the field has grown, theopportunity to add therapeutic
proteins that don't alreadyexist, so we can think of them
(09:48):
as kind of synthetic approaches,those are also opened up to
approaches like ours with the Bcell.
Dr. Moira Gunn (09:55):
Well, you're going one at a time slowly with
your first hurler patients here.Is it gonna be another year for
the second one? I mean, can wesee some faster progress
perhaps?
Sean Ainsworth (10:09):
I believe so indeed, yes. So we're planning
to dose that second patientimminently. We are actually
planning to re dose our firstpatient as well, which is super
exciting. That's something thatis not being done with a lot of
advanced therapeutics like this.And from here indeed, we expect
the cadence will pick uppotentially dosing several
(10:33):
patients this year.
Dr. Moira Gunn (10:34):
Now, this opens up the question of, can you tell
how well these B cells areworking?
Sean Ainsworth (10:41):
We can. So we can measure, aside from things
like a six minute walk test andflexibility, we can look at, we
call them molecular markers, andso we can detect those in the
blood and demonstrate that thebad stuff is going down. We can
do that in the blood, in theurine, and even the brain,
(11:06):
cerebrospinal fluid.
Dr. Moira Gunn (11:07):
We have to understand dosing is always the
issue. You know? Is it too much?Is it too little? Is it you
know?
What's really going on? How areyou monitoring people, and how
are you able to see that thisactually works other than the
fact that people are able towalk better and and, you know,
(11:29):
move their arms better andstraighten their fingers?
Sean Ainsworth (11:31):
So we can actually measure those long
chain sugars. We can pick themup in the urine, in the blood,
as well as other locales. We seethat those do get reduced over
time, as we expect, and that'sreally what's driving these
potential benefits in thepatients.
Dr. Moira Gunn (11:51):
Well, Sean, thank you so much. I hope you
come back. Keep us updated.
Sean Ainsworth (11:55):
All right. Well, you, Moira. It's been a
pleasure. And again, I reallyappreciate the opportunity to
speak to you here.
Dr. Moira Gunn (12:00):
Sean Ainsworth is the CEO of ImmuSoft. More
information is available on theweb at ImmuSoft.com. That's
immu,Immu, imu soft Com.
When you take your medicine, how you have to
take your prescribed drugs canbe hugely important. Is it a
simple pill, an injection, anasal spray, and very time
consuming, is it an infusionwhere you sit for several hours
with the vital therapeutic youneed being minutely introduced
(00:33):
into your bloodstream every fewweeks? Certainly any effort to
develop new ways to deliverdrugs is important, and it is
even more important when youhave a chronic condition where
you must have an essentialmedicine every day for the rest
of your life. Both a new way todeliver therapeutics and the
(00:54):
creation of the new therapeuticsthemselves is exactly what
ImmuSoft of Seattle is workingon. Sean Ainsworth is its CEO.
Sean, welcome to the program.
Sean Ainsworth (01:07):
Thanks so much, Moira. Thanks for having me on.
Dr. Moira Gunn (01:09):
We're all used to taking pills and injections
and even infusions into ourbloodstreams, and they all have
their advantages anddisadvantages. But who isn't
open to finding better ways todeliver drugs? And today, we're
talking about a new way todeliver drugs, and that's by
modifying something that occursnaturally in our bodies. They're
(01:33):
called B cells. What are Bcells?
Sean Ainsworth (01:36):
B cells are immune cells that are
responsible for producingantibodies. Antibodies against,
we can think of vaccines orviruses. And so that's what
gives us long lasting immunitythese different pathogens. And
we're taking these B cells andwe're adding the genes that
(01:56):
encode for proteins. And a lotof drugs are really just
proteins and so these aretherapeutic proteins that the B
cells then produce so when wegive them back to the patient,
they're naturally producing andreleasing those therapeutic
proteins into the bloodstream.
Dr. Moira Gunn (02:15):
Now obviously a lot of people take drugs all the
time. Does that mean all our Bcells are interchangeable?
Sean Ainsworth (02:24):
So they're going to be patient specific. In other
words, I can't take B cells frommyself and give them to somebody
else or vice versa. But we canindeed take a patient's B cells,
program those outside the body,and once they're producing those
(02:45):
therapeutic proteins, them backto that very same patient.
Dr. Moira Gunn (02:49):
Now importantly, while your specific B cells are
needed, ImmuSoft has beenworking on a general way to
modify B cells to make specificproteins. And before we get
there, the first medicaltreatment you've been working on
is Hurler syndrome. Tell usabout that.
Sean Ainsworth (03:08):
So Hurler syndrome is caused by a
defective gene called IDUA. Andwhen IDUA isn't working
properly, it leads to thingslike stiffness in your joints
amongst many other profoundimplications that follow these
(03:29):
patients really from birth andthrough to their entire lives if
not properly treated. So whenthese patients are affected by
that defective gene, it reallycauses a lot of issues from
joint stiffness that's quitesevere, that affects their
(03:49):
ability to walk. It also impactstheir cognitive function as well
as their heart, their liver,their spleen. It's really quite
profound, the impact on theseindividuals, and it starts in
childhood and follows themthrough their entire life.
Dr. Moira Gunn (04:09):
So for this treatment that ImmuSoft is
developing, you took B cellsfrom a particular first
candidate human here, and youtook them out and you made a
modification, to the B cell andyou gave it back to the human.
What was the modification?
Sean Ainsworth (04:26):
The modification was the addition of that fully
functioning IDUA gene.
Dr. Moira Gunn (04:32):
And that's it?
Sean Ainsworth (04:33):
That's it, yeah.
Dr. Moira Gunn (04:34):
You've just given them the gene, the ability
to produce the gene from those Bcells.
Sean Ainsworth (04:39):
Correct, correct. So at the cellular
level, that's the bigmodification. Once we give those
cells back to the patient, theymigrate to the bone marrow and
they begin producing thattherapeutic protein in mass
quantities and potentially formany, many years. And so I
(05:00):
believe you can imagine that 20receiving that therapeutic
protein will have a prettydramatic impact on the patient's
lives.
Dr. Moira Gunn (05:10):
So you're going slowly here as you must when you
make such a change to a humanbody. And I know you've started
in adults. In fact, you startedin just one adult over a year
ago and are about to start yoursecond adult, being very careful
here. What's been the experiencewith this first adult with
Hurler syndrome?
Sean Ainsworth (05:31):
So his experience has been quite
dramatic. And so we've seenimprovements in his ability to
walk. And to put some numbersaround it, we do what is called
a six minute walk test. And overthe course of six minutes, he
had a roughly two sixty yardimprovement, which is quite
(05:56):
extraordinary. Typically seeingsomewhere in the 30 plus minus
range is very impactful forthese patients.
He has also, reported being ableto do things like sit with his
legs crossed, he can reach hisback now, which he previously
couldn't do, he can betterstraighten his fingers. And so
(06:20):
these might not sound terriblydramatic, but you can imagine if
you've got a buildup ofmaterials in your joints that's
affecting your ability to movefreely, really does cause pain
and to remove some of thatmaterial and improve the
flexibility, improve the painprofile, it's quite impactful
(06:45):
for these patients.
Dr. Moira Gunn (06:46):
Now, it's very exciting to think, boy, that
gene is in there producingproteins that actually will they
will remove these, which they doevery day in most people. And
now they're removing even oldones, which is very exciting to
think about. But there's alsothe you get a little excited
(07:09):
when you say, Can you stop thecells from producing these
therapeutic proteins?
Sean Ainsworth (07:14):
We do believe that we can go in and remove the
cells if we had to. Sothankfully we haven't yet, but
there are drugs that aretargeted towards B cells, which
is actually another prettyunique aspect of what we do. So
other modalities are not quiteso easy to reverse, but we think
(07:35):
that we've got that opportunitywith our approach.
Dr. Moira Gunn (07:39):
And I know you're continuing with the
hurler syndrome and beingcareful with working with the
FDA here to make sure that wedon't get ahead of ourselves.
But as I said earlier, ImmuSofthas created a general way to
modify B cells. What else mightyou use this same B cell
modifying technology for?
Sean Ainsworth (07:59):
So we're hopeful that we can use it across a
broad number of differentprotein types. And at the outset
I mentioned that B cellsnaturally produce antibodies.
And so those comprise a prettybroad swath of different
therapeutics that are already inexistence. So if we think about,
(08:20):
for example, Humira and SKYRIZI,those are both very broadly used
in the autoimmune space and itcould potentially be delivered
from B cells. And then we canalso think of, for example, the
GLP-one approaches, which areOzempic, Wegovy, those are also
(08:46):
essentially therapeutic proteinswhich we can deliver from B
cells.
So there's really a broad numberof different areas we could
pursue with, with this B cellapproach.
Dr. Moira Gunn (09:00):
So in some ways, like what you're working on now
with hurlers, you are talkingabout inserting genes that would
modify a mutated, you know,problematic gene in someone's
body. And in in other ways, youwould actually be using it to
put genes in that would createbrand new drugs that literally
(09:23):
have a therapeutic effect.
Sean Ainsworth (09:25):
Indeed. Indeed, yes. And that's the beauty of
these gene therapeuticapproaches. They did start
typically with attempting tocorrect for defective genes, but
as the field has grown, theopportunity to add therapeutic
proteins that don't alreadyexist, so we can think of them
(09:48):
as kind of synthetic approaches,those are also opened up to
approaches like ours with the Bcell.
Dr. Moira Gunn (09:55):
Well, you're going one at a time slowly with
your first hurler patients here.Is it gonna be another year for
the second one? I mean, can wesee some faster progress
perhaps?
Sean Ainsworth (10:09):
I believe so indeed, yes. So we're planning
to dose that second patientimminently. We are actually
planning to re dose our firstpatient as well, which is super
exciting. That's something thatis not being done with a lot of
advanced therapeutics like this.And from here indeed, we expect
the cadence will pick uppotentially dosing several
(10:33):
patients this year.
Dr. Moira Gunn (10:34):
Now, this opens up the question of, can you tell
how well these B cells areworking?
Sean Ainsworth (10:41):
We can. So we can measure, aside from things
like a six minute walk test andflexibility, we can look at, we
call them molecular markers, andso we can detect those in the
blood and demonstrate that thebad stuff is going down. We can
do that in the blood, in theurine, and even the brain,
(11:06):
cerebrospinal fluid.
Dr. Moira Gunn (11:07):
We have to understand dosing is always the
issue. You know? Is it too much?Is it too little? Is it you
know?
What's really going on? How areyou monitoring people, and how
are you able to see that thisactually works other than the
fact that people are able towalk better and and, you know,
(11:29):
move their arms better andstraighten their fingers?
Sean Ainsworth (11:31):
So we can actually measure those long
chain sugars. We can pick themup in the urine, in the blood,
as well as other locales. We seethat those do get reduced over
time, as we expect, and that'sreally what's driving these
potential benefits in thepatients.
Dr. Moira Gunn (11:51):
Well, Sean, thank you so much. I hope you
come back. Keep us updated.
Sean Ainsworth (11:55):
All right. Well, you, Moira. It's been a
pleasure. And again, I reallyappreciate the opportunity to
speak to you here.
Dr. Moira Gunn (12:00):
Sean Ainsworth is the CEO of ImmuSoft. More
information is available on theweb at ImmuSoft.com. That's
immu,Immu, imu soft Com.
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