How to pronounce "wavered"
Transcript
[Music]
[Music]
[Music] [Applause]
what better way to
what better way to
what better way to start this is my son Charlie he's 11
start this is my son Charlie he's 11
start this is my son Charlie he's 11 years
years
years old and he's an hysterical quirky kid
old and he's an hysterical quirky kid
old and he's an hysterical quirky kid he loves life and he proudly marches to
he loves life and he proudly marches to
he loves life and he proudly marches to the beat of his own
the beat of his own
the beat of his own drum he loves Kobe Bryant and Dr dre as
drum he loves Kobe Bryant and Dr dre as
drum he loves Kobe Bryant and Dr dre as much as he loves writing risque mad libs
much as he loves writing risque mad libs
much as he loves writing risque mad libs or preparing brisket paninis on his own
or preparing brisket paninis on his own
or preparing brisket paninis on his own George Foreman girl
however when Charlie was three and a
however when Charlie was three and a
however when Charlie was three and a half years old the dark fell from the
half years old the dark fell from the
half years old the dark fell from the sky landed on my family and broke it
sky landed on my family and broke it
sky landed on my family and broke it into more pieces than could ever be
into more pieces than could ever be
into more pieces than could ever be imagined so many pieces in fact that
imagined so many pieces in fact that
imagined so many pieces in fact that life as we knew it was
life as we knew it was
life as we knew it was over you see my son Charlie was
over you see my son Charlie was
over you see my son Charlie was diagnosed with duchen muscular distrophe
diagnosed with duchen muscular distrophe
diagnosed with duchen muscular distrophe the most common and the most aggressive
the most common and the most aggressive
the most common and the most aggressive form of duchen mus of
form of duchen mus of
form of duchen mus of musculardrophe it's an infamous disease
musculardrophe it's an infamous disease
musculardrophe it's an infamous disease in that it's the number one genetic
in that it's the number one genetic
in that it's the number one genetic killer in the world of
killer in the world of
killer in the world of children children that suffer from
children children that suffer from
children children that suffer from duchen musculardrophe are missing a
duchen musculardrophe are missing a
duchen musculardrophe are missing a protein in their body called distroofen
protein in their body called distroofen
protein in their body called distroofen which is the largest protein in the body
which is the largest protein in the body
which is the largest protein in the body and it's essential for muscle strength
and it's essential for muscle strength
and it's essential for muscle strength and function an analogy that I often use
and function an analogy that I often use
and function an analogy that I often use is it's like the steel cables of a
is it's like the steel cables of a
is it's like the steel cables of a suspension bridge without the steel
suspension bridge without the steel
suspension bridge without the steel cables the bridge is unable to withstand
cables the bridge is unable to withstand
cables the bridge is unable to withstand any of mother nature's stresses kids
any of mother nature's stresses kids
any of mother nature's stresses kids that are missing this very important
that are missing this very important
that are missing this very important protein the muscles begin to deteriorate
protein the muscles begin to deteriorate
protein the muscles begin to deteriorate even with simple activities as getting
even with simple activities as getting
even with simple activities as getting up walking jumping uh for instance it
up walking jumping uh for instance it
up walking jumping uh for instance it affects one in every 3,500
affects one in every 3,500
affects one in every 3,500 boys it's an equal opportunity disease
boys it's an equal opportunity disease
boys it's an equal opportunity disease in that it knows no ethnic or geographic
in that it knows no ethnic or geographic
in that it knows no ethnic or geographic boundaries doesn't matter what country
boundaries doesn't matter what country
boundaries doesn't matter what country you're from doesn't matter what color
you're from doesn't matter what color
you're from doesn't matter what color your skin is one in every 3,500 boys
your skin is one in every 3,500 boys
your skin is one in every 3,500 boys throughout the world is born with this
throughout the world is born with this
throughout the world is born with this disease the prognosis is horrifying it's
disease the prognosis is horrifying it's
disease the prognosis is horrifying it's 100%
100%
100% fatal the disease is rapidly progressive
fatal the disease is rapidly progressive
fatal the disease is rapidly progressive and affects every muscle in a kid's body
and affects every muscle in a kid's body
and affects every muscle in a kid's body the their skeletal muscles the heart
the their skeletal muscles the heart
the their skeletal muscles the heart which is a muscle and the muscles of
which is a muscle and the muscles of
which is a muscle and the muscles of respiration
respiration
respiration by adolescence the kids are wheelchair
by adolescence the kids are wheelchair
by adolescence the kids are wheelchair bound and by their mid to late 20s they
bound and by their mid to late 20s they
bound and by their mid to late 20s they die this is the type of news that
die this is the type of news that
die this is the type of news that paralyzes
paralyzes
paralyzes most the sadness the anger and the fear
most the sadness the anger and the fear
most the sadness the anger and the fear is enough to stop anyone in their tracks
is enough to stop anyone in their tracks
is enough to stop anyone in their tracks for my wife and I we were determined to
for my wife and I we were determined to
for my wife and I we were determined to push through
this and to save our son's
this and to save our son's
this and to save our son's life in just four months after we
life in just four months after we
life in just four months after we received the tragic news that our son
received the tragic news that our son
received the tragic news that our son was diagnosed with duchen musculardrophe
was diagnosed with duchen musculardrophe
was diagnosed with duchen musculardrophe we decided to start Charlie's Fund a
we decided to start Charlie's Fund a
we decided to start Charlie's Fund a nonprofit
nonprofit
nonprofit organization that invests in
organization that invests in
organization that invests in therapeutics
therapeutics
therapeutics development for a therapy or a cure for
development for a therapy or a cure for
development for a therapy or a cure for duchen
duchen
duchen musculardrophe our mission is simple and
musculardrophe our mission is simple and
musculardrophe our mission is simple and crucial and we have never wavered from
crucial and we have never wavered from
crucial and we have never wavered from it from the first day we started our
it from the first day we started our
it from the first day we started our foundation we raise money and we direct
foundation we raise money and we direct
foundation we raise money and we direct it into the hands of those that we feel
it into the hands of those that we feel
it into the hands of those that we feel have the best shot on goal developing a
have the best shot on goal developing a
have the best shot on goal developing a therapy or a cure in time to save my son
therapy or a cure in time to save my son
therapy or a cure in time to save my son Charlie's life and the tens of thousands
Charlie's life and the tens of thousands
Charlie's life and the tens of thousands of boys who are needlessly suffering
of boys who are needlessly suffering
of boys who are needlessly suffering from this disease just like
him we are in a race against time my
him we are in a race against time my
him we are in a race against time my wife and I have been acutely aware of
wife and I have been acutely aware of
wife and I have been acutely aware of this fact since day one and
this fact since day one and
this fact since day one and unfortunately DMD was
unfortunately DMD was
unfortunately DMD was winning and progress was simply not
winning and progress was simply not
winning and progress was simply not moving fast enough for
moving fast enough for
moving fast enough for us there were no drugs and there
us there were no drugs and there
us there were no drugs and there currently are no drugs available for
currently are no drugs available for
currently are no drugs available for these kids to take in order to slow the
these kids to take in order to slow the
these kids to take in order to slow the progress of the disease with the
progress of the disease with the
progress of the disease with the exception of steroids which does very
exception of steroids which does very
exception of steroids which does very little to slow the
little to slow the
little to slow the devastation and worse it's associated
devastation and worse it's associated
devastation and worse it's associated with a host of terrible terrible side
with a host of terrible terrible side
with a host of terrible terrible side effects so bad in fact that many
effects so bad in fact that many
effects so bad in fact that many children are simply not able to take the
children are simply not able to take the
children are simply not able to take the drug
itself we knew we had to do things
itself we knew we had to do things
itself we knew we had to do things differently and we had to act
differently and we had to act
differently and we had to act quickly one thing that was very apparent
quickly one thing that was very apparent
quickly one thing that was very apparent to me was that we needed appropriate
to me was that we needed appropriate
to me was that we needed appropriate level of focus to be devoted towards
level of focus to be devoted towards
level of focus to be devoted towards this problem in order to make a
this problem in order to make a
this problem in order to make a difference in time for Charlie at the
difference in time for Charlie at the
difference in time for Charlie at the time we hired the world's only PhD level
time we hired the world's only PhD level
time we hired the world's only PhD level uh scientist who devoted a 100% of his
uh scientist who devoted a 100% of his
uh scientist who devoted a 100% of his efforts every single waking moment to
efforts every single waking moment to
efforts every single waking moment to help us realize a therapy or a cure for
help us realize a therapy or a cure for
help us realize a therapy or a cure for these boys it's amazing to think of
these boys it's amazing to think of
these boys it's amazing to think of something so simple as just devoting
something so simple as just devoting
something so simple as just devoting enough attention to a disease is what's
enough attention to a disease is what's
enough attention to a disease is what's needed and yet no one had done it before
needed and yet no one had done it before
needed and yet no one had done it before we did
we did
we did it another thing that was acutely that
it another thing that was acutely that
it another thing that was acutely that my wife and I were acutely aware of from
my wife and I were acutely aware of from
my wife and I were acutely aware of from the beginning was a sense of urgency
the beginning was a sense of urgency
the beginning was a sense of urgency time was not on our side the window of
time was not on our side the window of
time was not on our side the window of opportunity was
opportunity was
opportunity was narrow one of the annual meetings that
narrow one of the annual meetings that
narrow one of the annual meetings that my wife and I and all the key opinion
my wife and I and all the key opinion
my wife and I and all the key opinion leaders in the field for duchen
leaders in the field for duchen
leaders in the field for duchen musculardrophe
musculardrophe
musculardrophe uh one scientist uh was speaking and his
uh one scientist uh was speaking and his
uh one scientist uh was speaking and his words have always stuck with me he got
words have always stuck with me he got
words have always stuck with me he got up there and he spoke to an audience of
up there and he spoke to an audience of
up there and he spoke to an audience of several hundred people and he
several hundred people and he
several hundred people and he said "Myself included there are many
said "Myself included there are many
said "Myself included there are many scientists in this room who can cure
scientists in this room who can cure
scientists in this room who can cure duchen musculardrophe in a mouse it's
duchen musculardrophe in a mouse it's
duchen musculardrophe in a mouse it's time we move these drugs out of the
time we move these drugs out of the
time we move these drugs out of the animal models and into the kids before
animal models and into the kids before
animal models and into the kids before another generation is needlessly lost."
another generation is needlessly lost."
another generation is needlessly lost." I never forgot those words
I never forgot those words
I never forgot those words that evening when I came home I said to
that evening when I came home I said to
that evening when I came home I said to my wife I have an idea of what we're
my wife I have an idea of what we're
my wife I have an idea of what we're going to
going to
going to do we're going to screen every FDA
do we're going to screen every FDA
do we're going to screen every FDA approved drug in the country for other
approved drug in the country for other
approved drug in the country for other diseases and we're going to develop
diseases and we're going to develop
diseases and we're going to develop tests to see if there's any of these
tests to see if there's any of these
tests to see if there's any of these drugs that have any benefit in duchen
drugs that have any benefit in duchen
drugs that have any benefit in duchen musculardrophe so we developed these
musculardrophe so we developed these
musculardrophe so we developed these assays which are tests that are used to
assays which are tests that are used to
assays which are tests that are used to screen uh thousands tens of thousands
screen uh thousands tens of thousands
screen uh thousands tens of thousands sometimes millions of drugs in this case
sometimes millions of drugs in this case
sometimes millions of drugs in this case there was a finite number of drugs
there was a finite number of drugs
there was a finite number of drugs there's approximately 4,000 FDA approved
there's approximately 4,000 FDA approved
there's approximately 4,000 FDA approved drugs for other
drugs for other
drugs for other diseases just as I expected we got very
diseases just as I expected we got very
diseases just as I expected we got very promising hits from that screen and then
promising hits from that screen and then
promising hits from that screen and then we tested them in the animal model for
we tested them in the animal model for
we tested them in the animal model for this disease some of which showed great
this disease some of which showed great
this disease some of which showed great promise the results of this innovative
promise the results of this innovative
promise the results of this innovative initiative two clinical trials one of
initiative two clinical trials one of
initiative two clinical trials one of which is currently taking place at
which is currently taking place at
which is currently taking place at John's Hopkins University the other is
John's Hopkins University the other is
John's Hopkins University the other is taking place at Cincinnati Children's
taking place at Cincinnati Children's
taking place at Cincinnati Children's Hospital both of which are working on
Hospital both of which are working on
Hospital both of which are working on drugs that offer great promise in
drugs that offer great promise in
drugs that offer great promise in improving the strength and function of
improving the strength and function of
improving the strength and function of the skeletal muscles and the hearts of
the skeletal muscles and the hearts of
the skeletal muscles and the hearts of these kids which means that there is
these kids which means that there is
these kids which means that there is a good possibility that the quantity and
a good possibility that the quantity and
a good possibility that the quantity and quality of these children's lives for
quality of these children's lives for
quality of these children's lives for the first time in the history of this
the first time in the history of this
the first time in the history of this disease is going to be marketkedly
disease is going to be marketkedly
disease is going to be marketkedly improved
something else that we wanted to do that
something else that we wanted to do that
something else that we wanted to do that was needed and that would distinguish
was needed and that would distinguish
was needed and that would distinguish ourselves from the rest of the uh people
ourselves from the rest of the uh people
ourselves from the rest of the uh people and experts that were devoted to doing
and experts that were devoted to doing
and experts that were devoted to doing something about this disease was taking
something about this disease was taking
something about this disease was taking risks thinking outside the box being
risks thinking outside the box being
risks thinking outside the box being innovative in the early few months after
innovative in the early few months after
innovative in the early few months after we started Charlie's fund I would
we started Charlie's fund I would
we started Charlie's fund I would frantically scour the internet looking
frantically scour the internet looking
frantically scour the internet looking for all of the most promising research
for all of the most promising research
for all of the most promising research research that existed on this disease i
research that existed on this disease i
research that existed on this disease i picked up the phone and I called every
picked up the phone and I called every
picked up the phone and I called every scientist every clinician and every CEO
scientist every clinician and every CEO
scientist every clinician and every CEO of a biotech company that was doing
of a biotech company that was doing
of a biotech company that was doing anything that had to do with this
anything that had to do with this
anything that had to do with this disease
disease
disease within months I came up with a game plan
within months I came up with a game plan
within months I came up with a game plan with the help of uh my adviserss what we
with the help of uh my adviserss what we
with the help of uh my adviserss what we needed to do in this mining through the
needed to do in this mining through the
needed to do in this mining through the uh uh multitude of possibilities i came
uh uh multitude of possibilities i came
uh uh multitude of possibilities i came across this seedstage biotech company in
across this seedstage biotech company in
across this seedstage biotech company in the
the
the Netherlands they had less than 30 people
Netherlands they had less than 30 people
Netherlands they had less than 30 people at the time and they were working on
at the time and they were working on
at the time and they were working on proprietary technology that is something
proprietary technology that is something
proprietary technology that is something from Star Trek it's a type of modified
from Star Trek it's a type of modified
from Star Trek it's a type of modified gene therapy that tricks the muscles
gene therapy that tricks the muscles
gene therapy that tricks the muscles into once again making the missing
into once again making the missing
into once again making the missing protein I spoke of earlier called
protein I spoke of earlier called
protein I spoke of earlier called distroofen although making it in a
distroofen although making it in a
distroofen although making it in a shortened form with the hope that maybe
shortened form with the hope that maybe
shortened form with the hope that maybe there's still enough function in this
there's still enough function in this
there's still enough function in this protein that the kids quality of life
protein that the kids quality of life
protein that the kids quality of life that the the speed at which this uh
that the the speed at which this uh
that the the speed at which this uh muscle destruction was taking place
muscle destruction was taking place
muscle destruction was taking place would slow
down we also did something that was out
down we also did something that was out
down we also did something that was out of the box and innovative most
of the box and innovative most
of the box and innovative most nonprofits when they're providing funds
nonprofits when they're providing funds
nonprofits when they're providing funds or support for research do so in the
or support for research do so in the
or support for research do so in the form of a gift or sponsored research
form of a gift or sponsored research
form of a gift or sponsored research agreement where they roll out their
agreement where they roll out their
agreement where they roll out their funding incrementally with milestone
funding incrementally with milestone
funding incrementally with milestone achievement we decided to do something
achievement we decided to do something
achievement we decided to do something bigger knowing that drug development is
bigger knowing that drug development is
bigger knowing that drug development is expensive if these guys were on to
expensive if these guys were on to
expensive if these guys were on to something exciting they were going to
something exciting they were going to
something exciting they were going to need the money to demonstrate the proof
need the money to demonstrate the proof
need the money to demonstrate the proof of concept so that enough value could be
of concept so that enough value could be
of concept so that enough value could be built into this company that it would
built into this company that it would
built into this company that it would turn the attention of big biotech and
turn the attention of big biotech and
turn the attention of big biotech and big pharma the entities that have the
big pharma the entities that have the
big pharma the entities that have the sufficient funds to take this promising
sufficient funds to take this promising
sufficient funds to take this promising drug over the finish line we made an
drug over the finish line we made an
drug over the finish line we made an equity investment of
equity investment of
equity investment of $650,000 into this company the first
$650,000 into this company the first
$650,000 into this company the first $650,000 we raised went into this
$650,000 we raised went into this
$650,000 we raised went into this company
company
company the result they're in a phase three
the result they're in a phase three
the result they're in a phase three clinical trial in Europe in the United
clinical trial in Europe in the United
clinical trial in Europe in the United States in Canada and Asia and currently
States in Canada and Asia and currently
States in Canada and Asia and currently it is the most promising therapy in the
it is the most promising therapy in the
it is the most promising therapy in the world for duchen musculardrophe
world for duchen musculardrophe
world for duchen musculardrophe because of our support and others who
because of our support and others who
because of our support and others who were willing to take a risk in this
were willing to take a risk in this
were willing to take a risk in this company they have now recently uh
company they have now recently uh
company they have now recently uh partnered with Glacos Smith Klein the
partnered with Glacos Smith Klein the
partnered with Glacos Smith Klein the world's biggest pharmaceutical company
world's biggest pharmaceutical company
world's biggest pharmaceutical company in the world for a deal potentially
in the world for a deal potentially
in the world for a deal potentially worth over
worth over
worth over $720
$720
$720 million with upfronts and milestone
million with upfronts and milestone
million with upfronts and milestone achievements it's amazing what taking a
achievements it's amazing what taking a
achievements it's amazing what taking a risk an educated risk if you will can
risk an educated risk if you will can
risk an educated risk if you will can turn out if this company continues to be
turn out if this company continues to be
turn out if this company continues to be successful then Charlie's fund will be
successful then Charlie's fund will be
successful then Charlie's fund will be successful because of our equity
successful because of our equity
successful because of our equity investment the return on our investment
investment the return on our investment
investment the return on our investment will be used to refuel our efforts in
will be used to refuel our efforts in
will be used to refuel our efforts in other promising therapeutic strategies
other promising therapeutic strategies
other promising therapeutic strategies and I can once again place another
and I can once again place another
and I can once again place another educated bet hopefully on the next uh
educated bet hopefully on the next uh
educated bet hopefully on the next uh biotech company working on something for
biotech company working on something for
biotech company working on something for our
our
our children in summary our unprecedented
children in summary our unprecedented
children in summary our unprecedented progress in a short time had less to do
progress in a short time had less to do
progress in a short time had less to do about this groundbreaking scientific
about this groundbreaking scientific
about this groundbreaking scientific discovery and more to do with having the
discovery and more to do with having the
discovery and more to do with having the confidence and willingness to think
confidence and willingness to think
confidence and willingness to think outside the box
outside the box
outside the box on November
on November
on November 11th
11th
11th 1111 my son turned
11 this apicious numerological
11 this apicious numerological
11 this apicious numerological phenomenon occurs literally once every
phenomenon occurs literally once every
phenomenon occurs literally once every 10,000
10,000
10,000 years it's amazing i remember on
years it's amazing i remember on
years it's amazing i remember on Charlie's birthday I looked at my wife
Charlie's birthday I looked at my wife
Charlie's birthday I looked at my wife Tracy and I said to her "This is the
Tracy and I said to her "This is the
Tracy and I said to her "This is the year I'm going to deliver a devastating
year I'm going to deliver a devastating
year I'm going to deliver a devastating blow to DMD." And I want to look back at
blow to DMD." And I want to look back at
blow to DMD." And I want to look back at this year with no
this year with no
this year with no regrets it's easy to lose focus when
regrets it's easy to lose focus when
regrets it's easy to lose focus when you're doing something like we're trying
you're doing something like we're trying
you're doing something like we're trying to accomplish at Charlie's Fun there's
to accomplish at Charlie's Fun there's
to accomplish at Charlie's Fun there's so much at stake there are so many
so much at stake there are so many
so much at stake there are so many possibilities there's no room for
possibilities there's no room for
possibilities there's no room for error but moments like this are
error but moments like this are
error but moments like this are important and it always goes back to our
important and it always goes back to our
important and it always goes back to our mission and the one question are we
mission and the one question are we
mission and the one question are we doing everything we can to move things
doing everything we can to move things
doing everything we can to move things as fast as
as fast as
as fast as possible halo
possible halo
possible halo fugenone was a promising drug that I
fugenone was a promising drug that I
fugenone was a promising drug that I remember reading in a respected
remember reading in a respected
remember reading in a respected scientific journal that was being tested
scientific journal that was being tested
scientific journal that was being tested in the animal model for duchen
in the animal model for duchen
in the animal model for duchen musculardrophe a drug that was known to
musculardrophe a drug that was known to
musculardrophe a drug that was known to venture capitalists and scientists for
venture capitalists and scientists for
venture capitalists and scientists for other diseases that involved abnormal
other diseases that involved abnormal
other diseases that involved abnormal fibrosis but the venture capitalists
fibrosis but the venture capitalists
fibrosis but the venture capitalists lost faith after spending $80 million in
lost faith after spending $80 million in
lost faith after spending $80 million in this drug but I knew that there was
this drug but I knew that there was
this drug but I knew that there was still great promise in this drug for
still great promise in this drug for
still great promise in this drug for duchen musculardrophe after speaking
duchen musculardrophe after speaking
duchen musculardrophe after speaking with our scientific adviserss
with our scientific adviserss
with our scientific adviserss the problem was I didn't have the tools
the problem was I didn't have the tools
the problem was I didn't have the tools to figure out how to get the drug and
to figure out how to get the drug and
to figure out how to get the drug and how to push it forward so that it could
how to push it forward so that it could
how to push it forward so that it could potentially be given as a medicine to my
potentially be given as a medicine to my
potentially be given as a medicine to my son and kids like
son and kids like
son and kids like him i didn't turn my turn away from this
him i didn't turn my turn away from this
him i didn't turn my turn away from this opportunity instead we once again
opportunity instead we once again
opportunity instead we once again thought outside the box we decided to
thought outside the box we decided to
thought outside the box we decided to recruit a team of drug development
recruit a team of drug development
recruit a team of drug development experts people that represented the best
experts people that represented the best
experts people that represented the best and brightest in big pharma and
and brightest in big pharma and
and brightest in big pharma and biotech individuals with decades of
biotech individuals with decades of
biotech individuals with decades of experience in making drugs
experience in making drugs
experience in making drugs we did this in order to help us
we did this in order to help us
we did this in order to help us negotiate deals with the biotech
negotiate deals with the biotech
negotiate deals with the biotech companies to help us establish clinical
companies to help us establish clinical
companies to help us establish clinical trial networks with the key opinion
trial networks with the key opinion
trial networks with the key opinion leaders and the medical institutions
leaders and the medical institutions
leaders and the medical institutions that would be conducting the trials and
that would be conducting the trials and
that would be conducting the trials and to help us communicate with the
to help us communicate with the
to help us communicate with the regulatory authorities which is
regulatory authorities which is
regulatory authorities which is essential like the FDA in the United
essential like the FDA in the United
essential like the FDA in the United States and the EMEA which is the
States and the EMEA which is the
States and the EMEA which is the equivalent regulatory body in Europe
equivalent regulatory body in Europe
equivalent regulatory body in Europe incredibly in less than one year what
incredibly in less than one year what
incredibly in less than one year what have we accomplished with this team we
have we accomplished with this team we
have we accomplished with this team we acquired that promising drug from that
acquired that promising drug from that
acquired that promising drug from that dormant Israeli company and we started
dormant Israeli company and we started
dormant Israeli company and we started believe it or not our own biotech
believe it or not our own biotech
believe it or not our own biotech company called Halo Therapeutics in
company called Halo Therapeutics in
company called Halo Therapeutics in order to accelerate the development of
order to accelerate the development of
order to accelerate the development of this promising drug that we now call HT
this promising drug that we now call HT
this promising drug that we now call HT 100 and best of all we are on track and
100 and best of all we are on track and
100 and best of all we are on track and expect to begin a clinical trial this
expect to begin a clinical trial this
expect to begin a clinical trial this year
year
year as a result of the great promise of this
as a result of the great promise of this
as a result of the great promise of this drug and the expertise of the team that
drug and the expertise of the team that
drug and the expertise of the team that I
I
I assembled recently the United States and
assembled recently the United States and
assembled recently the United States and the European Union has granted Halo
the European Union has granted Halo
the European Union has granted Halo Therapeutics orphan drug
Therapeutics orphan drug
Therapeutics orphan drug status this distinction provides us with
status this distinction provides us with
status this distinction provides us with the regulatory and financial incentives
the regulatory and financial incentives
the regulatory and financial incentives to significantly compress the time and
to significantly compress the time and
to significantly compress the time and cost to get drugs into clinical trial
cost to get drugs into clinical trial
cost to get drugs into clinical trial and the marketplace again focusing on
and the marketplace again focusing on
and the marketplace again focusing on the
the
the urgency because of our bold
urgency because of our bold
urgency because of our bold efforts and the willingness and courage
efforts and the willingness and courage
efforts and the willingness and courage to take matters into our own hands we
to take matters into our own hands we
to take matters into our own hands we have now attracted the attention of the
have now attracted the attention of the
have now attracted the attention of the biotech
biotech
biotech industry in fact the Wall Street Journal
industry in fact the Wall Street Journal
industry in fact the Wall Street Journal has recently written an article about
has recently written an article about
has recently written an article about the groundbreaking innovation of
the groundbreaking innovation of
the groundbreaking innovation of Charlie's fund and halo therapeutics in
Charlie's fund and halo therapeutics in
Charlie's fund and halo therapeutics in the world of drug development and orphan
the world of drug development and orphan
the world of drug development and orphan diseases
i'm a Harvard trained physician who has
i'm a Harvard trained physician who has
i'm a Harvard trained physician who has an intimate understanding of the
an intimate understanding of the
an intimate understanding of the language of genetic
language of genetic
language of genetic medicine but my medical degree had
medicine but my medical degree had
medicine but my medical degree had nothing to do with getting us to the
nothing to do with getting us to the
nothing to do with getting us to the point of where we are right
point of where we are right
point of where we are right now instead it's been focus relentless
now instead it's been focus relentless
now instead it's been focus relentless determination and the courage to
determination and the courage to
determination and the courage to innovate
although for us the motivation was the
although for us the motivation was the
although for us the motivation was the need to save my son's life in
need to save my son's life in
need to save my son's life in reality these principles can be applied
reality these principles can be applied
reality these principles can be applied to any challenge that seemed to be
to any challenge that seemed to be
to any challenge that seemed to be insurmountable and it's these same
insurmountable and it's these same
insurmountable and it's these same principles that will help us rewrite the
principles that will help us rewrite the
principles that will help us rewrite the medical textbooks for this murderous
medical textbooks for this murderous
medical textbooks for this murderous disease and help us finally make duchen
disease and help us finally make duchen
disease and help us finally make duchen history thank you
history thank you
history thank you [Applause]
Phonetic Breakdown of "wavered"
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