January 22, 2024 • 25 mins
We are kickstarting 2024 with a Conversation in Drug Development with CEO and Founder of Boyds, Professor Alan Boyd. In this podcast episode, our host Harriet Edwards, and Professor Alan Boyd discuss trends from 2023 in the drug development space and its potential evolution in 2024. Join us as we explore the financial downturn affecting early-stage investment, the rise of cell and gene therapy approvals, and the use of artificial intelligence in various areas of drug discovery and development.
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(00:05):
Hello and a warm welcome to thelatest edition of Conversations
in Drug Development,brought to you by the team at Boyds.
This podcast is for our fellowcommunity of scientists and
clinicians working in thewonderful world of cell and gene
therapy and drug development.Thank you for tuning in and we
hope you enjoy the conversation.
(00:31):
Hello and welcome back to anotherinstallment of Conversations in
Drug Development.My name is Harriet Edwards,
and I'm one of the regulatoryteam here at Boyds and your
podcast host for today's episode.I am delighted to be joined by
Professor Alan Boyd on our podcasttoday, founder and CEO of Boyds.
Alan, welcome.Hopefully this is the first of
(00:51):
many appearances on the podcast.Thank you very much, Harriet,
and it's a pleasure to be heretoday and I'm looking forward to
our conversation. Absolutely.And I think it would be remiss if we
didn't have the first episode of 2024looking at all of the things we can
expect to come of this year, butalso notable trends and highlights
from 2023 that might inform usof things to look out for in the
(01:14):
drug development space this year.So we're going to kick it off with
a focus on advanced therapiesand cell and gene therapies.
We know they're in the news a lotat the moment, and I think we'd
be remiss if we didn't talk aboutone of the key highlights of or
lowlights, actually, of 2023.And something that continues to be
a big topic in drug development.And that's, of course,
(01:35):
the financial landscape.So I don't know if there's anything
that immediately springs to mind,Alan.
But there was a general downturnlast year,
and we're not sure whether that'sgoing to continue into this year.
If you got any thoughts on that.Well, it's a very important point
that you raise, Harriet, becauseof course, developing medicines
and devices is very expensive.And without the right sort of
(01:58):
finances and investment,we can't do anything. Now.
It's interesting during Covid,clearly industry and the
pharmaceutical industry played avery big part in particularly
getting the vaccines there.And so life science companies around
the world became the favorite ofthe stock market and investors.
And in 2021, something like $50billion were invested in life
(02:23):
science companies. Wow.That is probably the most money
that's ever been put in in onesingle year in terms of investment.
And the following year thatdropped down to about 25 billion,
which is still huge.Yes, it is,
but in 2023 it halved again. Wow.And I've seen figures from
standard and poor where, you know,up till about September,
(02:46):
only about, I don't know,something like 15 billion had gone
in which was a big reduction.And most of that was in the
United States.And that has been a sort of a general
increase over maybe the last decadeup to the point of the pandemic,
like you said,of investment in pharma and
particularly in the biotech space.Do you think it's just we've got
(03:07):
used to such a huge amount ofinvestment and things are now
normalizing after Covid,or is there something else going on?
Well, as you know, I've been inthe industry a long time,
well over 30 years.So I've seen downturns and
recessions.This is probably the fourth one
I've experienced during my career.So it is cyclical.
Now you ask me,how long is it going to last?
(03:29):
Well, if I could give you a precisedate, um, I wouldn't be sitting
here now, if you know what I mean.But it has been difficult,
particularly during 2023,particularly for seed funding.
This, you know, the money thatgoes into the very early stages of
drug development and companies.So that's been a real limit.
And in fact, even in our consultancywe've seen clients though they
(03:53):
they want us to do work,just haven't got the money.
Now fortunately though,there was there was a big conference
in San Francisco last week,the JP Morgan conference.
And coming out of that,it's where anybody who's anybody
in the pharma industry goes to.Clearly the financial situation
is improving.And that's the method it's starting
to and in fact, in America,they're even talking now about
(04:16):
flotations on the New York StockExchange of life science companies,
which is a great stimulus then,because once that gets going,
there's extra money there.People make money and reinvest it.
So hopefully we're going to see animprovement in 20 some positive news.
And I guess we keep our fingerscrossed for that.
I think it's notable to to mentionthat at the back half of last year
(04:38):
and particularly the last quarter,there were some positive
acquisitions, some positivereleases of funding into the the
gene therapy space, particularlywith I think there was one notable
acquisition by AstraZeneca,somebody that we've all heard of,
um, purchasing a company calledGray Cell, which was a cell therapy
company, autologous and allogeneicdeveloper for a massive $1.2 billion.
(05:02):
So the money is there.I think if, um, if people are willing
to put it into something that'spotentially a little bit more risky,
like the cell therapy developmentthat we see, but maybe it's it's
just a risk versus reward thing atthe moment. Yeah, I think it is.
And the other thing to think of,because of the downturn,
the valuation of companies hasactually dropped.
(05:22):
And so big Pharma can move in and buythem basically at a reduced rate
from where they were a year ago.Wow. And we've seen.
Quite a lot of that too now,which is good actually.
I mean, companies being taken over.But actually, if you're a major
stockholder in the in the smallcompany, you perhaps have not
raised it as much money as youthought you might have done.
So now, if you are a venturecapitalist or somebody with lots of
(05:45):
money in your pockets, it's a goodtime to snap up some bargains in
the biotech space. That's right.It's good to go out shopping,
basically. Absolutely. January sales.You heard that here first.
If if anybody's got a spare fewmillion or even billion to to spend.
But I think there is somecompetition and that's worth
(06:05):
mentioning where big pharma andinnovative medicines haven't
really had the competition before.There is now competition from
things like big tech and AI thatsort of swaying the market to
invest there as well.So that's maybe a factor with the the
slight downturn that we've seen.Yes, certainly.
Again, the money that has flowedin 2023 has made a large amount
(06:28):
of it has been into AI companies.So if you had AI in your title,
you, you know, stood a betterchance of getting investments,
let's put it that way.And we could talk about financials
for a long time, I'm sure.And everybody has an opinion there.
But since you mentioned I,I think it brings us nicely on
to maybe the next hot topic or atrend to watch out for in 2024.
And that's novel technology.So I mean, there's there's many novel
(06:51):
technologies we can talk about,but I think we first have to mention
that 2023, it wasn't all bad news.There might not have been as much
money in the the system for drugdevelopers, but it was a landmark
year for novel technologies,having our first ever gene edited
technology approved, and that wasby the UK regulator, the MHRA.
(07:11):
So back in November last year,we had an approval for a Crispr
edited technology to treat sicklecell disease, and that followed suit
with FDA approving later as well,and even other global territories
now following suit as well.So really positive and and maybe
something that will open thefloodgates for 2024 for these
type of technologies.Well,
(07:33):
you've raised a few points there.I mean, first of all, it was
great that the MHRA was the firstregulatory agency to approve it.
And if there are any benefits fromBrexit, then doing this sort of thing
by the MHRA, Brexit's allowed them todo that to be different, to look
at things in a slightly differentway and be on the front foot and
(07:57):
get these new products approved.So that's certainly happened.
And and of course, the FDA themselvesapproved six new cell and gene
therapy products last year, which.Is huge increase. Yes it is.
I mean, from the year before wehad a period, I think 2122 when
very few got approved.And so that's moving forward.
(08:19):
This year, the FDA are saying they'reexpecting to approve 17 selling gene
therapy products, which if they do,it's absolutely amazing.
And if I think back, you know,when I started, you know,
people often say I'm a pioneerin gene therapy. Perhaps I am.
But, you know, establishing one ofthe first companies in the early late
90s, early 2000 to think that 20,25 years later,
(08:43):
we now have 34 selling gene therapyproducts approved by the FDA.
And, you know,a similar number here in Europe.
So it really has mushroomed,and it's great to see it.
When I started out in the late 90s,people said I was crazy.
Nobody had ever done it.Why are you doing this after
having a huge job in in Zeneca?But perhaps, you know,
(09:05):
I wasn't that crazy after all.And it's really satisfying now to see
see what we've achieved actuallywith these treatments for patients.
Oh absolutely.And it makes us very proud,
I think, to to be part of theboys team and know that you have
that legacy behind you.And to see all of these sort of
gene therapies that we have theprivilege of working on,
some of them come to fruition.It's exciting for us as a as a team,
(09:28):
but also the wider sort of altruisticproperties of looking at how
well cell and gene therapy couldactually do for patients in need.
There is the ability to actuallycure diseases now,
and we have those products approvedon the market to cure something
rather than just to treat symptoms.It's it's a wonderful time to be
(09:49):
part of the drug development space.Yeah. No, I mean it's very exciting.
I've been qualified a long time now,and most of my professional
career treating patients and thendeveloping medicines was all about
trying to stop symptoms happening.But with gene therapy, actually,
and I don't particularly likethe word cure,
(10:09):
but that's actually what we're doing.We're actually changing and attacking
the root cause of the disease.Whereas all the other things
that we treat,we all take medicines, don't we?
At times we're just treatingsymptoms, not the root cause.
So that's what we're getting at.And and it's having us, you know,
when you see particularly some ofthe children that have now been.
(10:31):
Treated with things like her for theSMN disease, the inherited blindness.
This is a life changingtreatments not only for the,
you know, the patient, but alsofor the families. Oh, massively.
And actually, that's a reallynice way to put it, Alan.
I'm going to take that movingforward as treating the root
cause rather than a cure.I think that's a really great
way to to describe it.And you're right, with these gene
(10:52):
therapies that we're seeing, they aretreating some really awful diseases.
And a lot of them are specificallytargeting neurodegenerative diseases.
But in children.And to be able to offer that to
the the patient population thatcurrently don't have any options
is potentially life changing.So hopefully much more of that
(11:13):
in 2024. Yes.So it's interesting if you look at,
I've mentioned the number ofselling gene therapy products
that are approved.If we just take the gene therapy
side, two thirds of all the genetherapy studies and that have been
conducted across the world, and thereare now well over 4000 of them,
two thirds of those have focused ononcology. Wow. And cancer treatments.
(11:36):
Yet up till now,we've only got two approved gene
therapy products to treat cancer.And do you think there's a
reason for that?Is it that the problem with
development or is it postdevelopment where costs
potentially become prohibitive?I think I think what it is when
we're treating genetic mutations,that's a very specific target.
(11:59):
However, when we treat cancerpatients, we're actually using the
gene therapy itself to deliver aparticular protein, which is then
either attacking or supporting thecancer that's there or supporting
the immune response to that cancer.So it's I ought to think,
(12:21):
particularly when I'm teaching,when we're using gene therapy to
treat cancer, this is really thegene therapy is a sophisticated
way of delivering a protein.And I don't think people
recognize that.And of course,
then we have to make sure we've gotthe right amount of protein there.
And is it, you know,how it's it's acting.
And of course,that will be tried to be eliminated
by the immune system as well.I can sort of explain why things
(12:44):
have not happened yet,but the work goes on.
And do you think there's apotential for in in 2024?
And I think beyond this,this will continue the fight against
cancer and the use of gene therapiesto treat any oncology indication,
whether that's for pediatric,specifically a rare disease or
something more widespread.There is undoubtedly a focus
(13:06):
from the pharmaceutical industryto target oncology, isn't there?
Oh, definitely.But I think where the real
successes have come is with theCar T's and the immunotherapies.
All the Car T's are aimedpredominantly at at oncology
diseases.And we do use ex vivo transfection
now for things like the sickle celltreatments for immune deficiencies.
(13:30):
So we've got the technology.We just have to work it out.
And it's as you said we have thetechnology.
I think people now we're startingto get experience in developing
these types of medicines.There's the regulatory framework
that's starting to evolve and back upthe development of such novel and
individual types of medicine as well.It's not one size fits all anymore.
(13:53):
And that's, I guess,where I look at things.
I know you you look at them from acompletely different perspective,
having a medical background.But there are also some barriers,
even with all of that knowledge and,and access, that we have access
to those medicines, once we'vedemonstrated safety and efficacy
is a little bit tricky as well,particularly for countries that
(14:15):
maybe are less developed than, uh,sort of the ones that we would
expect to see approvals in the UK,the EU and the US, etc..
Do you think that procurement andpricing of these products, we go to
all of the effort of getting themapproved and and working on them,
developing them for people in need,and then we can't actually give
them to people in need.Do you think that's a problem?
(14:38):
Yes, I think it certainly is.I think at the moment I'm being
totally honest here.The selling gene therapies that
are being approved are for Westernsocieties, the wealthier nations,
because they are expensive,they're expensive to develop and
they're expensive to, you know,keep manufacturing and be out there.
And they're not really for themasses at the moment.
(15:02):
And I think that's something we needto really address particularly,
I mean, let's face it, you know,in places like Africa,
we have trouble delivering eventhe basic medicines to them,
let alone these, you know,these very complicated technical
delivery systems that are developing.And when you think about some of
these delivery systems and they'reactually personalized medicines,
(15:24):
aren't they.They're made per patient on,
on many occasions there might beissues with needing.
To cry out for the the medicine to.To have it in ultra cold
temperatures.And like you say, when we're looking
at something like a non developedcountry or continent like Africa that
maybe can't sustain that kind ofinfrastructure, that makes it a real
(15:46):
shame that actually the populationthat need it aren't going to have
access to it for some time. No.On the other side,
we all have to start somewhere. Yes.And and I think,
you know what we're doing.If you stand back and think we're
actually using DNA to treat andto say, you know, attack the
root cause of a disease now.And so it's just started.
(16:08):
We've only been going sort of 20,25 years. Seriously.
Most treatment modalities thathave been developed, you know,
Ace inhibitors, the antibody statinstook about 20 years from the first
ideas to actually making it to be amedicine. That's that's standard.
You know, you can look at all the,you know, the breakthroughs in
the last 50 years.So it's early days yet for cell
(16:31):
and gene therapy.And I think what we will do,
it will evolve.The manufacturer will get cheaper
and more straightforward.And so it probably won't happen
in my lifetime.But you know, we'll see say, in 30,
40 years, the technologies we havenow are much more widespread.
But I'd like to think so.And I agree with you.
(16:52):
I think it's something that everybodythat's working on these medicines
has at the forefront of their mind.They don't want these medicines
to be exclusively accessible forpeople that can afford it.
And so I think as long aseverybody is conscious of that,
there is going to be a move tomake things more accessible.
As you said, when when thetechnology and the scalability
(17:14):
is there to support lower costs,which is positive.
And I think you said before, we're inthe infancy of cell and gene therapy.
We've made great strides so far,and hopefully 2024 will be
another landmark year of notableapprovals and changes and maybe
some new regulations and guidanceto support such development.
But we are still at the verybeginning of the journey with
(17:37):
silent gene therapy.So overall positive. Yes, certainly.
Well, I'm a believer, you know that.And I'd like to finish this topic on
a positive note as well, becausethere was a general increase at the
end of last year in phase threetrials for sale and gene therapy,
particularly gene therapies.I think it was a 10% increase
was reported.So I think that could hopefully
(17:58):
mean we're looking at newapprovals in in 2024.
And as you said, I think there'sup to 17 predicted and maybe even
some more to come. That's right.So we've talked about gene
therapy and genome editing asone of the novel technologies.
But we can't get away fromartificial intelligence either.
Um,I know we talk about it a lot here.
And artificial intelligence withinthe drug development space can
(18:22):
mean so many different things.Um, for those people that maybe
haven't listened in,we do have a specific episode
about artificial intelligence,so we talk about it in much more
detail there and would encourageyou to to listen in to that.
But, Alan, do you have any thoughtson trends for the use of artificial
intelligence within drug developmentwithin salon gene therapy for 2024?
(18:43):
Well, there's quite a few areas.Yeah.
And these are already happening.I mean, we if you think about the
identification of molecules to treatdisease and drug development at that
level, drug discovery already and wehave used AI for a long time where
we take a particular structure,we can search for which bits of
(19:04):
that structure have previouslyknown to cause side effects and
adverse effects and things.So medicinal chemists can then
modify that structure.So that's already there.
The other place we're using AI is inimaging and the reading of images
with scans and X-rays and thingslike that. So that's already there.
There are also some companies nowusing AI in toxicology studies. Yes.
(19:28):
Because you'll be aware that thingthat takes the time is analyzing
and looking at all the slidesthat we take for the various
organs of animals that have beenexposed to potential treatments.
And all of that at the momentpretty much is manual, isn't it?
So absolutely.And there's thousands and
thousands of slides.But now there are ways of looking at,
(19:49):
should we say,the slides from the experiment
with slides from animals that havenot been exposed to the drug.
So it's an excellent way ofdoing that.
I know it's in its infancy,but that's happening. Yeah.
And the other thing that's happeningas well is it's in clinical.
We now can use AI to writeclinical protocols.
We can use AI to look at theliterature to find out, you know,
(20:13):
what's the right endpoint to usein a clinical trial as well.
So it it is there.I think one thing we have to be
careful of is using some of the wesay the things like chat PTC yes.
Um, and using that for creating.Documents and pulling
information from it.And certainly, as you know,
(20:35):
Harriet, I've more or less bannedthe use of that in writing our
regulatory documents.And the importance of that is that we
have to be totally accurate with ourregulatory documents, and if we don't
know the source of the information,then it could we could get in there.
And that could create a safetyproblem for patients. Absolutely.
And we did touch on that in the I,um, episode actually,
(20:59):
not just ChatGPT, but many of theseplatforms that are in their infancy
are fantastic in terms of their,the idea of them, but at the moment
they are only as good as the sourcedata that's input into them.
So the more they are used, the moredata is collected, the more accurate,
the more reliable they become.But as you said, for for using
things, for writing regulatorydocuments, for writing clinical
(21:23):
protocols based on previous data,etc. actually, it needs to be
used with extreme caution becausethere's only a certain amount of
sources that these platforms use,and they may be missing a complete
perspective because they haven't gotthat source data from everywhere.
So fantastic idea.I think it's something that will
definitely be used movingforward in drug development.
(21:45):
There are so many exciting areas,but it's still even more infant if
possible than genome editing and allof the other novel technology so
must be used with extreme caution.Yeah, no, I entirely agree with you.
And of course, you know, at themoment we haven't got any defined
regulations from the regulatoryagencies about the use of AI. Yes.
Or rather the correct use or theright use of it. And that will come.
(22:08):
I'm sure it will. Yeah.And we're all learning.
I think that's the key, isn't it?We're all learning the regulators
as well as the developers.And it's as with many of these
newer technologies,it's about being collaborative and
working together to make sure thatthat we build framework wise from a
regulatory perspective is usableand pragmatic and ensuring safety
at the end of the day as well.So it's a it's a tricky task for
(22:31):
regulators.I definitely don't envy the the
other side of the fence in termsof other topics and things that
we might look out for in 2024.I think we've we've covered the
obvious, Alan, but is there anythingelse that you think is a definite
trend to look out for this year? Yes.One area is antibody drug conjugates.
(22:51):
Now, those of you who've been aroundin industry for a while know we've
already been there in the past,probably in the sort of 90s into
But from what I'm seeing and
being at conferences, talking topeople and clients coming to,
to to ask for help, there is adefinite focus again on antibody
drug conjugate product or ADCs,as they're called it sort of moved
(23:14):
on from just connecting, say,a chemical entity to an antibody.
Now people are even puttinggenes attached to some of these,
these proteins and things like that.So they get the antibodies.
The ADC space is is kind ofrealizing its true potential.
Then I guess absolutely.And there's definitely a resurgence.
So I think it's something towatch out.
(23:36):
And the companies that are nowdoing this.
So yeah, again technology moves onand you know we're adding genes and
other things to these antibodies,which is great. It's amazing.
It's certainly an exciting time to bepart of um, of any drug development,
but particularly with the innovativetherapies that we see often at Boyds.
It's um, it's going to behopefully another exciting year.
(23:59):
Alan, it has been wonderful,as always, to to speak to you,
but to be able to bring you thepodcast and have everybody listening,
I think everyone will agree.It's been fascinating and we'll
definitely look forward tohaving you on another
installment before we end today.Are there any other hot topics
or points that you would like toto raise for people to look out
(24:19):
for in 2024?People who know me know I'm an
optimist.Um, and I think going forward,
once the money starts flowing again,I think again we're going to see
growth, perhaps not in the first halfof the year, but the second half.
I think that's where the impactwill come again and we'll all
get switched on.The money will start flowing,
more companies will be financedand we'll be looking at even
(24:43):
more products coming forward,which is very exciting because
after all, people often say,you know, I'm a businessman.
Well, yes, I am running a business,but I'm a doctor first.
And the most important thing to meis to get new medicines out there
for patients. That's why we do it.Absolutely. I couldn't agree more.
Alan, it has been wonderful tohave you on, and I look forward to
reviewing some of these trends,maybe at the end of the year as well,
(25:05):
to see if we were right or not.Okay. Thanks very much, Harriet.
I've really enjoyed the conversation.Thank you.
Thank you for listening toConversations in Drug Development,
the podcast series brought toyou by the team at boys.
Don't forget to follow us on theusual podcast platforms or visit our
(25:26):
website to ensure you don't missout on any of our future episodes.
Hello and a warm welcome to thelatest edition of Conversations
in Drug Development,brought to you by the team at Boyds.
This podcast is for our fellowcommunity of scientists and
clinicians working in thewonderful world of cell and gene
therapy and drug development.Thank you for tuning in and we
hope you enjoy the conversation.
(00:31):
Hello and welcome back to anotherinstallment of Conversations in
Drug Development.My name is Harriet Edwards,
and I'm one of the regulatoryteam here at Boyds and your
podcast host for today's episode.I am delighted to be joined by
Professor Alan Boyd on our podcasttoday, founder and CEO of Boyds.
Alan, welcome.Hopefully this is the first of
(00:51):
many appearances on the podcast.Thank you very much, Harriet,
and it's a pleasure to be heretoday and I'm looking forward to
our conversation. Absolutely.And I think it would be remiss if we
didn't have the first episode of 2024looking at all of the things we can
expect to come of this year, butalso notable trends and highlights
from 2023 that might inform usof things to look out for in the
(01:14):
drug development space this year.So we're going to kick it off with
a focus on advanced therapiesand cell and gene therapies.
We know they're in the news a lotat the moment, and I think we'd
be remiss if we didn't talk aboutone of the key highlights of or
lowlights, actually, of 2023.And something that continues to be
a big topic in drug development.And that's, of course,
(01:35):
the financial landscape.So I don't know if there's anything
that immediately springs to mind,Alan.
But there was a general downturnlast year,
and we're not sure whether that'sgoing to continue into this year.
If you got any thoughts on that.Well, it's a very important point
that you raise, Harriet, becauseof course, developing medicines
and devices is very expensive.And without the right sort of
(01:58):
finances and investment,we can't do anything. Now.
It's interesting during Covid,clearly industry and the
pharmaceutical industry played avery big part in particularly
getting the vaccines there.And so life science companies around
the world became the favorite ofthe stock market and investors.
And in 2021, something like $50billion were invested in life
(02:23):
science companies. Wow.That is probably the most money
that's ever been put in in onesingle year in terms of investment.
And the following year thatdropped down to about 25 billion,
which is still huge.Yes, it is,
but in 2023 it halved again. Wow.And I've seen figures from
standard and poor where, you know,up till about September,
(02:46):
only about, I don't know,something like 15 billion had gone
in which was a big reduction.And most of that was in the
United States.And that has been a sort of a general
increase over maybe the last decadeup to the point of the pandemic,
like you said,of investment in pharma and
particularly in the biotech space.Do you think it's just we've got
(03:07):
used to such a huge amount ofinvestment and things are now
normalizing after Covid,or is there something else going on?
Well, as you know, I've been inthe industry a long time,
well over 30 years.So I've seen downturns and
recessions.This is probably the fourth one
I've experienced during my career.So it is cyclical.
Now you ask me,how long is it going to last?
(03:29):
Well, if I could give you a precisedate, um, I wouldn't be sitting
here now, if you know what I mean.But it has been difficult,
particularly during 2023,particularly for seed funding.
This, you know, the money thatgoes into the very early stages of
drug development and companies.So that's been a real limit.
And in fact, even in our consultancywe've seen clients though they
(03:53):
they want us to do work,just haven't got the money.
Now fortunately though,there was there was a big conference
in San Francisco last week,the JP Morgan conference.
And coming out of that,it's where anybody who's anybody
in the pharma industry goes to.Clearly the financial situation
is improving.And that's the method it's starting
to and in fact, in America,they're even talking now about
(04:16):
flotations on the New York StockExchange of life science companies,
which is a great stimulus then,because once that gets going,
there's extra money there.People make money and reinvest it.
So hopefully we're going to see animprovement in 20 some positive news.
And I guess we keep our fingerscrossed for that.
I think it's notable to to mentionthat at the back half of last year
(04:38):
and particularly the last quarter,there were some positive
acquisitions, some positivereleases of funding into the the
gene therapy space, particularlywith I think there was one notable
acquisition by AstraZeneca,somebody that we've all heard of,
um, purchasing a company calledGray Cell, which was a cell therapy
company, autologous and allogeneicdeveloper for a massive $1.2 billion.
(05:02):
So the money is there.I think if, um, if people are willing
to put it into something that'spotentially a little bit more risky,
like the cell therapy developmentthat we see, but maybe it's it's
just a risk versus reward thing atthe moment. Yeah, I think it is.
And the other thing to think of,because of the downturn,
the valuation of companies hasactually dropped.
(05:22):
And so big Pharma can move in and buythem basically at a reduced rate
from where they were a year ago.Wow. And we've seen.
Quite a lot of that too now,which is good actually.
I mean, companies being taken over.But actually, if you're a major
stockholder in the in the smallcompany, you perhaps have not
raised it as much money as youthought you might have done.
So now, if you are a venturecapitalist or somebody with lots of
(05:45):
money in your pockets, it's a goodtime to snap up some bargains in
the biotech space. That's right.It's good to go out shopping,
basically. Absolutely. January sales.You heard that here first.
If if anybody's got a spare fewmillion or even billion to to spend.
But I think there is somecompetition and that's worth
(06:05):
mentioning where big pharma andinnovative medicines haven't
really had the competition before.There is now competition from
things like big tech and AI thatsort of swaying the market to
invest there as well.So that's maybe a factor with the the
slight downturn that we've seen.Yes, certainly.
Again, the money that has flowedin 2023 has made a large amount
(06:28):
of it has been into AI companies.So if you had AI in your title,
you, you know, stood a betterchance of getting investments,
let's put it that way.And we could talk about financials
for a long time, I'm sure.And everybody has an opinion there.
But since you mentioned I,I think it brings us nicely on
to maybe the next hot topic or atrend to watch out for in 2024.
And that's novel technology.So I mean, there's there's many novel
(06:51):
technologies we can talk about,but I think we first have to mention
that 2023, it wasn't all bad news.There might not have been as much
money in the the system for drugdevelopers, but it was a landmark
year for novel technologies,having our first ever gene edited
technology approved, and that wasby the UK regulator, the MHRA.
(07:11):
So back in November last year,we had an approval for a Crispr
edited technology to treat sicklecell disease, and that followed suit
with FDA approving later as well,and even other global territories
now following suit as well.So really positive and and maybe
something that will open thefloodgates for 2024 for these
type of technologies.Well,
(07:33):
you've raised a few points there.I mean, first of all, it was
great that the MHRA was the firstregulatory agency to approve it.
And if there are any benefits fromBrexit, then doing this sort of thing
by the MHRA, Brexit's allowed them todo that to be different, to look
at things in a slightly differentway and be on the front foot and
(07:57):
get these new products approved.So that's certainly happened.
And and of course, the FDA themselvesapproved six new cell and gene
therapy products last year, which.Is huge increase. Yes it is.
I mean, from the year before wehad a period, I think 2122 when
very few got approved.And so that's moving forward.
(08:19):
This year, the FDA are saying they'reexpecting to approve 17 selling gene
therapy products, which if they do,it's absolutely amazing.
And if I think back, you know,when I started, you know,
people often say I'm a pioneerin gene therapy. Perhaps I am.
But, you know, establishing one ofthe first companies in the early late
90s, early 2000 to think that 20,25 years later,
(08:43):
we now have 34 selling gene therapyproducts approved by the FDA.
And, you know,a similar number here in Europe.
So it really has mushroomed,and it's great to see it.
When I started out in the late 90s,people said I was crazy.
Nobody had ever done it.Why are you doing this after
having a huge job in in Zeneca?But perhaps, you know,
(09:05):
I wasn't that crazy after all.And it's really satisfying now to see
see what we've achieved actuallywith these treatments for patients.
Oh absolutely.And it makes us very proud,
I think, to to be part of theboys team and know that you have
that legacy behind you.And to see all of these sort of
gene therapies that we have theprivilege of working on,
some of them come to fruition.It's exciting for us as a as a team,
(09:28):
but also the wider sort of altruisticproperties of looking at how
well cell and gene therapy couldactually do for patients in need.
There is the ability to actuallycure diseases now,
and we have those products approvedon the market to cure something
rather than just to treat symptoms.It's it's a wonderful time to be
(09:49):
part of the drug development space.Yeah. No, I mean it's very exciting.
I've been qualified a long time now,and most of my professional
career treating patients and thendeveloping medicines was all about
trying to stop symptoms happening.But with gene therapy, actually,
and I don't particularly likethe word cure,
(10:09):
but that's actually what we're doing.We're actually changing and attacking
the root cause of the disease.Whereas all the other things
that we treat,we all take medicines, don't we?
At times we're just treatingsymptoms, not the root cause.
So that's what we're getting at.And and it's having us, you know,
when you see particularly some ofthe children that have now been.
(10:31):
Treated with things like her for theSMN disease, the inherited blindness.
This is a life changingtreatments not only for the,
you know, the patient, but alsofor the families. Oh, massively.
And actually, that's a reallynice way to put it, Alan.
I'm going to take that movingforward as treating the root
cause rather than a cure.I think that's a really great
way to to describe it.And you're right, with these gene
(10:52):
therapies that we're seeing, they aretreating some really awful diseases.
And a lot of them are specificallytargeting neurodegenerative diseases.
But in children.And to be able to offer that to
the the patient population thatcurrently don't have any options
is potentially life changing.So hopefully much more of that
(11:13):
in 2024. Yes.So it's interesting if you look at,
I've mentioned the number ofselling gene therapy products
that are approved.If we just take the gene therapy
side, two thirds of all the genetherapy studies and that have been
conducted across the world, and thereare now well over 4000 of them,
two thirds of those have focused ononcology. Wow. And cancer treatments.
(11:36):
Yet up till now,we've only got two approved gene
therapy products to treat cancer.And do you think there's a
reason for that?Is it that the problem with
development or is it postdevelopment where costs
potentially become prohibitive?I think I think what it is when
we're treating genetic mutations,that's a very specific target.
(11:59):
However, when we treat cancerpatients, we're actually using the
gene therapy itself to deliver aparticular protein, which is then
either attacking or supporting thecancer that's there or supporting
the immune response to that cancer.So it's I ought to think,
(12:21):
particularly when I'm teaching,when we're using gene therapy to
treat cancer, this is really thegene therapy is a sophisticated
way of delivering a protein.And I don't think people
recognize that.And of course,
then we have to make sure we've gotthe right amount of protein there.
And is it, you know,how it's it's acting.
And of course,that will be tried to be eliminated
by the immune system as well.I can sort of explain why things
(12:44):
have not happened yet,but the work goes on.
And do you think there's apotential for in in 2024?
And I think beyond this,this will continue the fight against
cancer and the use of gene therapiesto treat any oncology indication,
whether that's for pediatric,specifically a rare disease or
something more widespread.There is undoubtedly a focus
(13:06):
from the pharmaceutical industryto target oncology, isn't there?
Oh, definitely.But I think where the real
successes have come is with theCar T's and the immunotherapies.
All the Car T's are aimedpredominantly at at oncology
diseases.And we do use ex vivo transfection
now for things like the sickle celltreatments for immune deficiencies.
(13:30):
So we've got the technology.We just have to work it out.
And it's as you said we have thetechnology.
I think people now we're startingto get experience in developing
these types of medicines.There's the regulatory framework
that's starting to evolve and back upthe development of such novel and
individual types of medicine as well.It's not one size fits all anymore.
(13:53):
And that's, I guess,where I look at things.
I know you you look at them from acompletely different perspective,
having a medical background.But there are also some barriers,
even with all of that knowledge and,and access, that we have access
to those medicines, once we'vedemonstrated safety and efficacy
is a little bit tricky as well,particularly for countries that
(14:15):
maybe are less developed than, uh,sort of the ones that we would
expect to see approvals in the UK,the EU and the US, etc..
Do you think that procurement andpricing of these products, we go to
all of the effort of getting themapproved and and working on them,
developing them for people in need,and then we can't actually give
them to people in need.Do you think that's a problem?
(14:38):
Yes, I think it certainly is.I think at the moment I'm being
totally honest here.The selling gene therapies that
are being approved are for Westernsocieties, the wealthier nations,
because they are expensive,they're expensive to develop and
they're expensive to, you know,keep manufacturing and be out there.
And they're not really for themasses at the moment.
(15:02):
And I think that's something we needto really address particularly,
I mean, let's face it, you know,in places like Africa,
we have trouble delivering eventhe basic medicines to them,
let alone these, you know,these very complicated technical
delivery systems that are developing.And when you think about some of
these delivery systems and they'reactually personalized medicines,
(15:24):
aren't they.They're made per patient on,
on many occasions there might beissues with needing.
To cry out for the the medicine to.To have it in ultra cold
temperatures.And like you say, when we're looking
at something like a non developedcountry or continent like Africa that
maybe can't sustain that kind ofinfrastructure, that makes it a real
(15:46):
shame that actually the populationthat need it aren't going to have
access to it for some time. No.On the other side,
we all have to start somewhere. Yes.And and I think,
you know what we're doing.If you stand back and think we're
actually using DNA to treat andto say, you know, attack the
root cause of a disease now.And so it's just started.
(16:08):
We've only been going sort of 20,25 years. Seriously.
Most treatment modalities thathave been developed, you know,
Ace inhibitors, the antibody statinstook about 20 years from the first
ideas to actually making it to be amedicine. That's that's standard.
You know, you can look at all the,you know, the breakthroughs in
the last 50 years.So it's early days yet for cell
(16:31):
and gene therapy.And I think what we will do,
it will evolve.The manufacturer will get cheaper
and more straightforward.And so it probably won't happen
in my lifetime.But you know, we'll see say, in 30,
40 years, the technologies we havenow are much more widespread.
But I'd like to think so.And I agree with you.
(16:52):
I think it's something that everybodythat's working on these medicines
has at the forefront of their mind.They don't want these medicines
to be exclusively accessible forpeople that can afford it.
And so I think as long aseverybody is conscious of that,
there is going to be a move tomake things more accessible.
As you said, when when thetechnology and the scalability
(17:14):
is there to support lower costs,which is positive.
And I think you said before, we're inthe infancy of cell and gene therapy.
We've made great strides so far,and hopefully 2024 will be
another landmark year of notableapprovals and changes and maybe
some new regulations and guidanceto support such development.
But we are still at the verybeginning of the journey with
(17:37):
silent gene therapy.So overall positive. Yes, certainly.
Well, I'm a believer, you know that.And I'd like to finish this topic on
a positive note as well, becausethere was a general increase at the
end of last year in phase threetrials for sale and gene therapy,
particularly gene therapies.I think it was a 10% increase
was reported.So I think that could hopefully
(17:58):
mean we're looking at newapprovals in in 2024.
And as you said, I think there'sup to 17 predicted and maybe even
some more to come. That's right.So we've talked about gene
therapy and genome editing asone of the novel technologies.
But we can't get away fromartificial intelligence either.
Um,I know we talk about it a lot here.
And artificial intelligence withinthe drug development space can
(18:22):
mean so many different things.Um, for those people that maybe
haven't listened in,we do have a specific episode
about artificial intelligence,so we talk about it in much more
detail there and would encourageyou to to listen in to that.
But, Alan, do you have any thoughtson trends for the use of artificial
intelligence within drug developmentwithin salon gene therapy for 2024?
(18:43):
Well, there's quite a few areas.Yeah.
And these are already happening.I mean, we if you think about the
identification of molecules to treatdisease and drug development at that
level, drug discovery already and wehave used AI for a long time where
we take a particular structure,we can search for which bits of
(19:04):
that structure have previouslyknown to cause side effects and
adverse effects and things.So medicinal chemists can then
modify that structure.So that's already there.
The other place we're using AI is inimaging and the reading of images
with scans and X-rays and thingslike that. So that's already there.
There are also some companies nowusing AI in toxicology studies. Yes.
(19:28):
Because you'll be aware that thingthat takes the time is analyzing
and looking at all the slidesthat we take for the various
organs of animals that have beenexposed to potential treatments.
And all of that at the momentpretty much is manual, isn't it?
So absolutely.And there's thousands and
thousands of slides.But now there are ways of looking at,
(19:49):
should we say,the slides from the experiment
with slides from animals that havenot been exposed to the drug.
So it's an excellent way ofdoing that.
I know it's in its infancy,but that's happening. Yeah.
And the other thing that's happeningas well is it's in clinical.
We now can use AI to writeclinical protocols.
We can use AI to look at theliterature to find out, you know,
(20:13):
what's the right endpoint to usein a clinical trial as well.
So it it is there.I think one thing we have to be
careful of is using some of the wesay the things like chat PTC yes.
Um, and using that for creating.Documents and pulling
information from it.And certainly, as you know,
(20:35):
Harriet, I've more or less bannedthe use of that in writing our
regulatory documents.And the importance of that is that we
have to be totally accurate with ourregulatory documents, and if we don't
know the source of the information,then it could we could get in there.
And that could create a safetyproblem for patients. Absolutely.
And we did touch on that in the I,um, episode actually,
(20:59):
not just ChatGPT, but many of theseplatforms that are in their infancy
are fantastic in terms of their,the idea of them, but at the moment
they are only as good as the sourcedata that's input into them.
So the more they are used, the moredata is collected, the more accurate,
the more reliable they become.But as you said, for for using
things, for writing regulatorydocuments, for writing clinical
(21:23):
protocols based on previous data,etc. actually, it needs to be
used with extreme caution becausethere's only a certain amount of
sources that these platforms use,and they may be missing a complete
perspective because they haven't gotthat source data from everywhere.
So fantastic idea.I think it's something that will
definitely be used movingforward in drug development.
(21:45):
There are so many exciting areas,but it's still even more infant if
possible than genome editing and allof the other novel technology so
must be used with extreme caution.Yeah, no, I entirely agree with you.
And of course, you know, at themoment we haven't got any defined
regulations from the regulatoryagencies about the use of AI. Yes.
Or rather the correct use or theright use of it. And that will come.
(22:08):
I'm sure it will. Yeah.And we're all learning.
I think that's the key, isn't it?We're all learning the regulators
as well as the developers.And it's as with many of these
newer technologies,it's about being collaborative and
working together to make sure thatthat we build framework wise from a
regulatory perspective is usableand pragmatic and ensuring safety
at the end of the day as well.So it's a it's a tricky task for
(22:31):
regulators.I definitely don't envy the the
other side of the fence in termsof other topics and things that
we might look out for in 2024.I think we've we've covered the
obvious, Alan, but is there anythingelse that you think is a definite
trend to look out for this year? Yes.One area is antibody drug conjugates.
(22:51):
Now, those of you who've been aroundin industry for a while know we've
already been there in the past,probably in the sort of 90s into
But from what I'm seeing and
being at conferences, talking topeople and clients coming to,
to to ask for help, there is adefinite focus again on antibody
drug conjugate product or ADCs,as they're called it sort of moved
(23:14):
on from just connecting, say,a chemical entity to an antibody.
Now people are even puttinggenes attached to some of these,
these proteins and things like that.So they get the antibodies.
The ADC space is is kind ofrealizing its true potential.
Then I guess absolutely.And there's definitely a resurgence.
So I think it's something towatch out.
(23:36):
And the companies that are nowdoing this.
So yeah, again technology moves onand you know we're adding genes and
other things to these antibodies,which is great. It's amazing.
It's certainly an exciting time to bepart of um, of any drug development,
but particularly with the innovativetherapies that we see often at Boyds.
It's um, it's going to behopefully another exciting year.
(23:59):
Alan, it has been wonderful,as always, to to speak to you,
but to be able to bring you thepodcast and have everybody listening,
I think everyone will agree.It's been fascinating and we'll
definitely look forward tohaving you on another
installment before we end today.Are there any other hot topics
or points that you would like toto raise for people to look out
(24:19):
for in 2024?People who know me know I'm an
optimist.Um, and I think going forward,
once the money starts flowing again,I think again we're going to see
growth, perhaps not in the first halfof the year, but the second half.
I think that's where the impactwill come again and we'll all
get switched on.The money will start flowing,
more companies will be financedand we'll be looking at even
(24:43):
more products coming forward,which is very exciting because
after all, people often say,you know, I'm a businessman.
Well, yes, I am running a business,but I'm a doctor first.
And the most important thing to meis to get new medicines out there
for patients. That's why we do it.Absolutely. I couldn't agree more.
Alan, it has been wonderful tohave you on, and I look forward to
reviewing some of these trends,maybe at the end of the year as well,
(25:05):
to see if we were right or not.Okay. Thanks very much, Harriet.
I've really enjoyed the conversation.Thank you.
Thank you for listening toConversations in Drug Development,
the podcast series brought toyou by the team at boys.
Don't forget to follow us on theusual podcast platforms or visit our
(25:26):
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