[Applause] hello thank you very much it’s wonderful
hello thank you very much it’s wonderful
hello thank you very much it’s wonderful to be back at stage and I appreciate the
to be back at stage and I appreciate the
to be back at stage and I appreciate the opportunity to talk to you about
opportunity to talk to you about
opportunity to talk to you about something that is very near and dear and
something that is very near and dear and
something that is very near and dear and I’m very passionate about cure duchene
I’m very passionate about cure duchene
I’m very passionate about cure duchene tradition has been acknowledged over the
tradition has been acknowledged over the
tradition has been acknowledged over the last couple of years for creating a
last couple of years for creating a
last couple of years for creating a unique nonprofit business model the
unique nonprofit business model the
unique nonprofit business model the reality is I’d like to say that we set
reality is I’d like to say that we set
reality is I’d like to say that we set out to do this purposely but basically
out to do this purposely but basically
out to do this purposely but basically we just applied logic and common sense
we just applied logic and common sense
we just applied logic and common sense to a problem so a little bit of
to a problem so a little bit of
to a problem so a little bit of background I was raised in just a normal
background I was raised in just a normal
background I was raised in just a normal household I had an older brother and
household I had an older brother and
household I had an older brother and sister my mom used to always call me the
sister my mom used to always call me the
sister my mom used to always call me the pragmatists in the family and I because
pragmatists in the family and I because
pragmatists in the family and I because it was my mom telling me that I never
it was my mom telling me that I never
it was my mom telling me that I never bothered looking up the word to figure
bothered looking up the word to figure
bothered looking up the word to figure out what it meant and so many years
out what it meant and so many years
out what it meant and so many years later and I guess she would she would
later and I guess she would she would
later and I guess she would she would tell me that I was always able to shift
tell me that I was always able to shift
tell me that I was always able to shift from Planet a plan B pretty quickly
from Planet a plan B pretty quickly
from Planet a plan B pretty quickly without a lot of stress or anxiety and
without a lot of stress or anxiety and
without a lot of stress or anxiety and so I was also very rebellious when I was
so I was also very rebellious when I was
so I was also very rebellious when I was young I was especially my teenage years
young I was especially my teenage years
young I was especially my teenage years I was pretty much a rebel looking for a
I was pretty much a rebel looking for a
I was pretty much a rebel looking for a cause and I would go through very
cause and I would go through very
cause and I would go through very intense stages of whether it was horses
intense stages of whether it was horses
intense stages of whether it was horses or rock and roll or travel or whatever
or rock and roll or travel or whatever
or rock and roll or travel or whatever it was I was completely focused on what
it was I was completely focused on what
it was I was completely focused on what i was doing to the point that when I was
i was doing to the point that when I was
i was doing to the point that when I was in college my friend started calling me
in college my friend started calling me
in college my friend started calling me saying that I had monomania and I did
saying that I had monomania and I did
saying that I had monomania and I did look this word up and I wasn’t real
look this word up and I wasn’t real
look this word up and I wasn’t real pleased with the first definition about
pleased with the first definition about
pleased with the first definition about mental illness so I like to think that I
mental illness so I like to think that I
mental illness so I like to think that I attended more towards number two which
attended more towards number two which
attended more towards number two which was excessive concentration on a single
was excessive concentration on a single
was excessive concentration on a single subject and so we basically ended up
subject and so we basically ended up
subject and so we basically ended up having a huge huge problem our son
having a huge huge problem our son
having a huge huge problem our son Hawkin was born in 1997 when he was five
Hawkin was born in 1997 when he was five
Hawkin was born in 1997 when he was five years old he was diagnosed with Duchenne
years old he was diagnosed with Duchenne
years old he was diagnosed with Duchenne muscular dystrophy we had never heard of
muscular dystrophy we had never heard of
muscular dystrophy we had never heard of this word before and we really didn’t
this word before and we really didn’t
this word before and we really didn’t know what to do and there was a problem
know what to do and there was a problem
know what to do and there was a problem in my life that I honestly did not have
in my life that I honestly did not have
in my life that I honestly did not have a
a
a Lucien for and it was huge Duchenne
Lucien for and it was huge Duchenne
Lucien for and it was huge Duchenne muscular dystrophy is the most common
muscular dystrophy is the most common
muscular dystrophy is the most common form of muscular muscular dystrophy it
form of muscular muscular dystrophy it
form of muscular muscular dystrophy it affects boys it’s an excellent
affects boys it’s an excellent
affects boys it’s an excellent chromosome disease they’re usually
chromosome disease they’re usually
chromosome disease they’re usually diagnosed between three and five years
diagnosed between three and five years
diagnosed between three and five years old usually by the time they’re twelve
old usually by the time they’re twelve
old usually by the time they’re twelve years old they stop walking confined to
years old they stop walking confined to
years old they stop walking confined to a wheelchair later in teen years they
a wheelchair later in teen years they
a wheelchair later in teen years they need ventilation to breathe they’re
need ventilation to breathe they’re
need ventilation to breathe they’re unable to move their arms or even use a
unable to move their arms or even use a
unable to move their arms or even use a keyboard on a computer this is a
keyboard on a computer this is a
keyboard on a computer this is a terrible prognosis we researched we went
terrible prognosis we researched we went
terrible prognosis we researched we went to all the doctors we were basically
to all the doctors we were basically
to all the doctors we were basically told we’re told that there’s nothing you
told we’re told that there’s nothing you
told we’re told that there’s nothing you can do go home and love your child this
can do go home and love your child this
can do go home and love your child this disease affects all parts of the body it
disease affects all parts of the body it
disease affects all parts of the body it affects the brain there’s a lack of
affects the brain there’s a lack of
affects the brain there’s a lack of dystrophin which is a protein that’s in
dystrophin which is a protein that’s in
dystrophin which is a protein that’s in muscles and also in your brain so
muscles and also in your brain so
muscles and also in your brain so there’s cognitive issues many times of
there’s cognitive issues many times of
there’s cognitive issues many times of these kids your heart is a muscle and
these kids your heart is a muscle and
these kids your heart is a muscle and basically the primary cause of death is
basically the primary cause of death is
basically the primary cause of death is cardiac failure usually with with boys
cardiac failure usually with with boys
cardiac failure usually with with boys young men in their early to late 20s so
young men in their early to late 20s so
young men in their early to late 20s so it was a very serious problem we were
it was a very serious problem we were
it was a very serious problem we were basically told that our son we were
basically told that our son we were
basically told that our son we were going to lose him as most most parents
going to lose him as most most parents
going to lose him as most most parents were seeing their their sons graduate
were seeing their their sons graduate
were seeing their their sons graduate from high school so Dan pilota who
from high school so Dan pilota who
from high school so Dan pilota who actually was a TED talk several years
actually was a TED talk several years
actually was a TED talk several years ago that I heard and really inspired me
ago that I heard and really inspired me
ago that I heard and really inspired me I had already been running a non-profit
I had already been running a non-profit
I had already been running a non-profit for a few years and trying to grapple
for a few years and trying to grapple
for a few years and trying to grapple with just the regulation of the way
with just the regulation of the way
with just the regulation of the way things were done there were a couple
things were done there were a couple
things were done there were a couple muscular dystrophy organizations and
muscular dystrophy organizations and
muscular dystrophy organizations and Duchenne organizations and we tried to
Duchenne organizations and we tried to
Duchenne organizations and we tried to work with them and we really had no
work with them and we really had no
work with them and we really had no intention of going out and starting our
intention of going out and starting our
intention of going out and starting our own organization but what we heard was
own organization but what we heard was
own organization but what we heard was when we would come up with new ideas new
when we would come up with new ideas new
when we would come up with new ideas new ways of doing things that’s not the way
ways of doing things that’s not the way
ways of doing things that’s not the way we do things here and so we looked at
we do things here and so we looked at
we do things here and so we looked at this and thought well they don’t have a
this and thought well they don’t have a
this and thought well they don’t have a cure yet for a disease and they’re
cure yet for a disease and they’re
cure yet for a disease and they’re really not very far along so maybe we
really not very far along so maybe we
really not very far along so maybe we shouldn’t be doing it their way and so
shouldn’t be doing it their way and so
shouldn’t be doing it their way and so that was why we created the business
that was why we created the business
that was why we created the business model and started moving forward so Dan
model and started moving forward so Dan
model and started moving forward so Dan pilota was very articulate and talking
pilota was very articulate and talking
pilota was very articulate and talking about the dysfunction of how charities
about the dysfunction of how charities
about the dysfunction of how charities are
are
are and help what the public expects from a
and help what the public expects from a
and help what the public expects from a charity it seems that charities are
charity it seems that charities are
charity it seems that charities are supposed to operate with very little
supposed to operate with very little
supposed to operate with very little money and get big results and he felt
money and get big results and he felt
money and get big results and he felt and said basically that it’s it’s
and said basically that it’s it’s
and said basically that it’s it’s unrealistic to expect that that
unrealistic to expect that that
unrealistic to expect that that frugality is is a measure of success and
frugality is is a measure of success and
frugality is is a measure of success and morality when in essence we should be
morality when in essence we should be
morality when in essence we should be thinking big we should be thinking about
thinking big we should be thinking about
thinking big we should be thinking about problem solving and having setting and
problem solving and having setting and
problem solving and having setting and being rewarded for our accomplishments
being rewarded for our accomplishments
being rewarded for our accomplishments and for setting high goals and achieving
and for setting high goals and achieving
and for setting high goals and achieving them and not worrying about our expense
them and not worrying about our expense
them and not worrying about our expense ratio it’s like any business if you have
ratio it’s like any business if you have
ratio it’s like any business if you have to operate on an eighty-five percent
to operate on an eighty-five percent
to operate on an eighty-five percent profit margin you’re going to have a
profit margin you’re going to have a
profit margin you’re going to have a tough time and that’s what nonprofits
tough time and that’s what nonprofits
tough time and that’s what nonprofits are expected to do so he basically said
are expected to do so he basically said
are expected to do so he basically said let’s change the way we think about
let’s change the way we think about
let’s change the way we think about changing the world so here to Shen you
changing the world so here to Shen you
changing the world so here to Shen you know I think a lot of it has to do with
know I think a lot of it has to do with
know I think a lot of it has to do with the fact that both my husband and I came
the fact that both my husband and I came
the fact that both my husband and I came out of business and we started looking
out of business and we started looking
out of business and we started looking at traditional nonprofits and basically
at traditional nonprofits and basically
at traditional nonprofits and basically what you had is we would you know a
what you had is we would you know a
what you had is we would you know a non-profit goes out and works really
non-profit goes out and works really
non-profit goes out and works really really hard to raise money from people
really hard to raise money from people
really hard to raise money from people like you to to donate in in medical
like you to to donate in in medical
like you to to donate in in medical research what this means is you give a
research what this means is you give a
research what this means is you give a donation in the form of a grant to an
donation in the form of a grant to an
donation in the form of a grant to an academic institution and the scientists
academic institution and the scientists
academic institution and the scientists there you work on a sponsored research
there you work on a sponsored research
there you work on a sponsored research program and and you know if the if the
program and and you know if the if the
program and and you know if the if the science is successful and it’s promising
science is successful and it’s promising
science is successful and it’s promising a biotech or pharmaceutical company may
a biotech or pharmaceutical company may
a biotech or pharmaceutical company may come in and license that technology and
come in and license that technology and
come in and license that technology and if that drug is developed and it’s
if that drug is developed and it’s
if that drug is developed and it’s commercially successful then the biotech
commercially successful then the biotech
commercially successful then the biotech and the pharmaceutical company make a
and the pharmaceutical company make a
and the pharmaceutical company make a lot of money and you the nonprofit go
lot of money and you the nonprofit go
lot of money and you the nonprofit go back to your donors and ask for more
back to your donors and ask for more
back to your donors and ask for more money to do it all over again it’s very
money to do it all over again it’s very
money to do it all over again it’s very very hard raising money so what cured of
very hard raising money so what cured of
very hard raising money so what cured of shin did is we created what’s called the
shin did is we created what’s called the
shin did is we created what’s called the venture philanthropy model and actually
venture philanthropy model and actually
venture philanthropy model and actually we didn’t we didn’t create it it was was
we didn’t we didn’t create it it was was
we didn’t we didn’t create it it was was kind of an unknown phrase when we
kind of an unknown phrase when we
kind of an unknown phrase when we started this organization but a few
started this organization but a few
started this organization but a few organizations had already had successful
organizations had already had successful
organizations had already had successful business models
business models
business models namely actually one cystic fibrosis have
namely actually one cystic fibrosis have
namely actually one cystic fibrosis have done a fantastic job with this so
done a fantastic job with this so
done a fantastic job with this so basically here to shen we take our
basically here to shen we take our
basically here to shen we take our donations and we work with biotech and
donations and we work with biotech and
donations and we work with biotech and pharmaceutical companies as much as we
pharmaceutical companies as much as we
pharmaceutical companies as much as we can the difference between these two
can the difference between these two
can the difference between these two models and what what defines success is
models and what what defines success is
models and what what defines success is for an academic institution success
for an academic institution success
for an academic institution success means getting a paper published success
means getting a paper published success
means getting a paper published success for a pharmaceutical or biotech company
for a pharmaceutical or biotech company
for a pharmaceutical or biotech company means having a drug that’s working so
means having a drug that’s working so
means having a drug that’s working so cure Duchene knows that drug companies
cure Duchene knows that drug companies
cure Duchene knows that drug companies make drugs and so why not partner with
make drugs and so why not partner with
make drugs and so why not partner with them so we started partnering early with
them so we started partnering early with
them so we started partnering early with with drug companies where we would
with drug companies where we would
with drug companies where we would actually invest and and if there was
actually invest and and if there was
actually invest and and if there was commercial success then part of that
commercial success then part of that
commercial success then part of that return would come back to cure duchene
return would come back to cure duchene
return would come back to cure duchene that we could go and reinvest in further
that we could go and reinvest in further
that we could go and reinvest in further research and so we still have to go back
research and so we still have to go back
research and so we still have to go back to donors because as you’ll see in just
to donors because as you’ll see in just
to donors because as you’ll see in just a minute you know weary deploy the funds
a minute you know weary deploy the funds
a minute you know weary deploy the funds and if we’re going to move on to more
and if we’re going to move on to more
and if we’re going to move on to more projects we do need to continue to raise
projects we do need to continue to raise
projects we do need to continue to raise money but it’s not a one-way street this
money but it’s not a one-way street this
money but it’s not a one-way street this is a case study that actually is a very
is a case study that actually is a very
is a case study that actually is a very successful case study in 2004 cure
successful case study in 2004 cure
successful case study in 2004 cure duchene and over a couple years invested
duchene and over a couple years invested
duchene and over a couple years invested two million dollars into a little tiny
two million dollars into a little tiny
two million dollars into a little tiny biotech company called presenza and the
biotech company called presenza and the
biotech company called presenza and the netherlands and we went through ups and
netherlands and we went through ups and
netherlands and we went through ups and downs with with their drug development
downs with with their drug development
downs with with their drug development program and finally in 2015 a larger
program and finally in 2015 a larger
program and finally in 2015 a larger biotech company in northern california
biotech company in northern california
biotech company in northern california bought them called BioMarin and when
bought them called BioMarin and when
bought them called BioMarin and when that transaction happened are two
that transaction happened are two
that transaction happened are two million dollars we were able to get back
million dollars we were able to get back
million dollars we were able to get back about seven million dollars from that
about seven million dollars from that
about seven million dollars from that which we then redeployed into five
which we then redeployed into five
which we then redeployed into five additional research projects four of
additional research projects four of
additional research projects four of them being biotech companies one of them
them being biotech companies one of them
them being biotech companies one of them being an academic institution bamboo
being an academic institution bamboo
being an academic institution bamboo therapeutics is a gene therapy company
therapeutics is a gene therapy company
therapeutics is a gene therapy company that potentially has a cure by
that potentially has a cure by
that potentially has a cure by correcting the DNA and we invested money
correcting the DNA and we invested money
correcting the DNA and we invested money into that in two thousand
into that in two thousand
into that in two thousand January 2016 august 2016 Pfizer bought
January 2016 august 2016 Pfizer bought
January 2016 august 2016 Pfizer bought them in the future if that is successful
them in the future if that is successful
them in the future if that is successful and hopefully it will be you know at
and hopefully it will be you know at
and hopefully it will be you know at some point cure edition will be able to
some point cure edition will be able to
some point cure edition will be able to take those funds that we get from that
take those funds that we get from that
take those funds that we get from that and reinvested Capricorn era più tix
and reinvested Capricorn era più tix
and reinvested Capricorn era più tix has the heart stem-cell exosomes it’s
has the heart stem-cell exosomes it’s
has the heart stem-cell exosomes it’s delivered directly to the cardiac and it
delivered directly to the cardiac and it
delivered directly to the cardiac and it is in a phase 12 again we’re awaiting
is in a phase 12 again we’re awaiting
is in a phase 12 again we’re awaiting results for that miles Eric’s resurrect
results for that miles Eric’s resurrect
results for that miles Eric’s resurrect small companies that cure edition was
small companies that cure edition was
small companies that cure edition was the initial funder resurrects was right
the initial funder resurrects was right
the initial funder resurrects was right out of USC that we created a startup
out of USC that we created a startup
out of USC that we created a startup company and license at technology so
company and license at technology so
company and license at technology so it’s it’s really exciting to see that
it’s it’s really exciting to see that
it’s it’s really exciting to see that one one investment that started with
one one investment that started with
one one investment that started with people like you in the room here by
people like you in the room here by
people like you in the room here by trusting in an organization that cares
trusting in an organization that cares
trusting in an organization that cares so much about a disease and we have the
so much about a disease and we have the
so much about a disease and we have the best experts in the world you can see
best experts in the world you can see
best experts in the world you can see how it’s that the funds that donors give
how it’s that the funds that donors give
how it’s that the funds that donors give us are leveraged and able to grow so
us are leveraged and able to grow so
us are leveraged and able to grow so this is this is a business model that we
this is this is a business model that we
this is this is a business model that we would like to to work with we basically
would like to to work with we basically
would like to to work with we basically find the best science areas and another
find the best science areas and another
find the best science areas and another thing that sets cure edition apart from
thing that sets cure edition apart from
thing that sets cure edition apart from the traditional nonprofits most
the traditional nonprofits most
the traditional nonprofits most traditional nonprofits that fund medical
traditional nonprofits that fund medical
traditional nonprofits that fund medical research have you know very very expert
research have you know very very expert
research have you know very very expert Board of Advisors with the scientists
Board of Advisors with the scientists
Board of Advisors with the scientists and they once a year get together and
and they once a year get together and
and they once a year get together and they do a call for proposals and they
they do a call for proposals and they
they do a call for proposals and they meet and they score and it’s you know
meet and they score and it’s you know
meet and they score and it’s you know it’s a very high level meeting and they
it’s a very high level meeting and they
it’s a very high level meeting and they find the best science well that’s great
find the best science well that’s great
find the best science well that’s great but what if what if there’s certain
but what if what if there’s certain
but what if what if there’s certain parts of this disease that aren’t being
parts of this disease that aren’t being
parts of this disease that aren’t being addressed by the proposals that they
addressed by the proposals that they
addressed by the proposals that they happen to get and so we take a top-down
happen to get and so we take a top-down
happen to get and so we take a top-down view where we basically look at this
view where we basically look at this
view where we basically look at this disease and we realize that it’s cardiac
disease and we realize that it’s cardiac
disease and we realize that it’s cardiac its diaphragm muscles failure it’s
its diaphragm muscles failure it’s
its diaphragm muscles failure it’s brittle bones it’s inflammation and
brittle bones it’s inflammation and
brittle bones it’s inflammation and fibrosis and you know gene replacement
fibrosis and you know gene replacement
fibrosis and you know gene replacement and correcting the gene and all these
and correcting the gene and all these
and correcting the gene and all these different things that have to be taken
different things that have to be taken
different things that have to be taken care of so we look at it like from the
care of so we look at it like from the
care of so we look at it like from the top down like we have to impose our will
top down like we have to impose our will
top down like we have to impose our will on the science
on the science
on the science and so we basically go out and set the
and so we basically go out and set the
and so we basically go out and set the targets that we want to address and we
targets that we want to address and we
targets that we want to address and we find the best people to work on that and
find the best people to work on that and
find the best people to work on that and if nobody’s doing it we create that and
if nobody’s doing it we create that and
if nobody’s doing it we create that and it’s worked out to be very successful
it’s worked out to be very successful
it’s worked out to be very successful something else that distinguishes cure
something else that distinguishes cure
something else that distinguishes cure dash in from I think at least at the
dash in from I think at least at the
dash in from I think at least at the nonprofit’s that I’m familiar with in
nonprofit’s that I’m familiar with in
nonprofit’s that I’m familiar with in the muscular dystrophy space is when it
the muscular dystrophy space is when it
the muscular dystrophy space is when it came time to hire our team I did not go
came time to hire our team I did not go
came time to hire our team I did not go to academic institutions if drug
to academic institutions if drug
to academic institutions if drug companies make drugs then I wanted to
companies make drugs then I wanted to
companies make drugs then I wanted to hire a professional from the
hire a professional from the
hire a professional from the pharmaceutical business which we did
pharmaceutical business which we did
pharmaceutical business which we did with 30 we hired a scientist with 30
with 30 we hired a scientist with 30
with 30 we hired a scientist with 30 years biotech and pharmaceutical
years biotech and pharmaceutical
years biotech and pharmaceutical experience who can actually help us
experience who can actually help us
experience who can actually help us through the drug development process we
through the drug development process we
through the drug development process we hired somebody out of a venture capital
hired somebody out of a venture capital
hired somebody out of a venture capital firm who is in a Stanford trade Stanford
firm who is in a Stanford trade Stanford
firm who is in a Stanford trade Stanford trained and B that can help us design
trained and B that can help us design
trained and B that can help us design these deals and really work with the
these deals and really work with the
these deals and really work with the biotech companies and so we find the
biotech companies and so we find the
biotech companies and so we find the projects we find the best projects we
projects we find the best projects we
projects we find the best projects we fund them and we also foster them by
fund them and we also foster them by
fund them and we also foster them by offering our expertise the patient
offering our expertise the patient
offering our expertise the patient access the key opinion leaders and we’re
access the key opinion leaders and we’re
access the key opinion leaders and we’re able to foster these all the way through
able to foster these all the way through
able to foster these all the way through to the hopefully commercialization so
to the hopefully commercialization so
to the hopefully commercialization so venture philanthropy I I believe in my
venture philanthropy I I believe in my
venture philanthropy I I believe in my opinion is the way that nonprofits
opinion is the way that nonprofits
opinion is the way that nonprofits should be run if possible they’re
should be run if possible they’re
should be run if possible they’re self-sustaining again raising money is
self-sustaining again raising money is
self-sustaining again raising money is really really hard and will never stop
really really hard and will never stop
really really hard and will never stop raising money through our galas and our
raising money through our galas and our
raising money through our galas and our fundraising and that’s really the core
fundraising and that’s really the core
fundraising and that’s really the core but we are able to tell our donors that
but we are able to tell our donors that
but we are able to tell our donors that we’re going to leverage the money that
we’re going to leverage the money that
we’re going to leverage the money that you give us and grow it by being smart
you give us and grow it by being smart
you give us and grow it by being smart without money it’s replicable I’m on
without money it’s replicable I’m on
without money it’s replicable I’m on several panels talking to other
several panels talking to other
several panels talking to other nonprofit organizations especially in
nonprofit organizations especially in
nonprofit organizations especially in the rare disease space trying to help
the rare disease space trying to help
the rare disease space trying to help them understand you know different
them understand you know different
them understand you know different financing models whether its equity
financing models whether its equity
financing models whether its equity convertible notes royalties licensing
convertible notes royalties licensing
convertible notes royalties licensing agreements there’s a lot to learn and
agreements there’s a lot to learn and
agreements there’s a lot to learn and taken us will taking me personally a
taken us will taking me personally a
taken us will taking me personally a long time to learn that
long time to learn that
long time to learn that and and I think this is a model that is
and and I think this is a model that is
and and I think this is a model that is starting to be replicated a lot in
starting to be replicated a lot in
starting to be replicated a lot in nonprofits it’s it’s a way to
nonprofits it’s it’s a way to
nonprofits it’s it’s a way to revolutionize the the medical research
revolutionize the the medical research
revolutionize the the medical research and for me the most important part is
and for me the most important part is
and for me the most important part is this is a way to get meaningful funds
this is a way to get meaningful funds
this is a way to get meaningful funds and I’m not talking you know ten twenty
and I’m not talking you know ten twenty
and I’m not talking you know ten twenty thousand dollar grants we’re talking
thousand dollar grants we’re talking
thousand dollar grants we’re talking million or two million dollars where we
million or two million dollars where we
million or two million dollars where we can actually see the project and make it
can actually see the project and make it
can actually see the project and make it happen quickly so meaningful funds that
happen quickly so meaningful funds that
happen quickly so meaningful funds that come back to the people that care the
come back to the people that care the
come back to the people that care the most about what they’re going to do with
most about what they’re going to do with
most about what they’re going to do with the money so we care more than anybody
the money so we care more than anybody
the money so we care more than anybody we care more than the pharmaceutical or
we care more than the pharmaceutical or
we care more than the pharmaceutical or the biotech companies about the success
the biotech companies about the success
the biotech companies about the success of that particular drug and so when we
of that particular drug and so when we
of that particular drug and so when we have control over it and we have the say
have control over it and we have the say
have control over it and we have the say it goes a lot faster and again back to
it goes a lot faster and again back to
it goes a lot faster and again back to Dan pilotis point it’s like let’s let’s
Dan pilotis point it’s like let’s let’s
Dan pilotis point it’s like let’s let’s reward success and what is success in
reward success and what is success in
reward success and what is success in medical research it’s drugs that help
medical research it’s drugs that help
medical research it’s drugs that help patients not papers that are published
patients not papers that are published
patients not papers that are published in journals and so the whole venture
in journals and so the whole venture
in journals and so the whole venture philanthropy rewards success by actual
philanthropy rewards success by actual
philanthropy rewards success by actual results speaking of results last year we
results speaking of results last year we
results speaking of results last year we actually had the first drug approval for
actually had the first drug approval for
actually had the first drug approval for duchene it was a long and rocky road
duchene it was a long and rocky road
duchene it was a long and rocky road getting there we learned a lot on the
getting there we learned a lot on the
getting there we learned a lot on the way lots of mistakes and lots of ways
way lots of mistakes and lots of ways
way lots of mistakes and lots of ways that we would do things differently in
that we would do things differently in
that we would do things differently in the future but we do have one drug
the future but we do have one drug
the future but we do have one drug approval so we know we can do this this
approval so we know we can do this this
approval so we know we can do this this drug is not a cure we still have a long
drug is not a cure we still have a long
drug is not a cure we still have a long way to go it doesn’t treat the primary
way to go it doesn’t treat the primary
way to go it doesn’t treat the primary cause of death which is cardiac failure
cause of death which is cardiac failure
cause of death which is cardiac failure and it only treats about thirteen
and it only treats about thirteen
and it only treats about thirteen percent of the patients but it’s a start
percent of the patients but it’s a start
percent of the patients but it’s a start and my husband always uses the Roger
and my husband always uses the Roger
and my husband always uses the Roger Bannister the four-minute mile that
Bannister the four-minute mile that
Bannister the four-minute mile that until Roger Bannister round the
until Roger Bannister round the
until Roger Bannister round the four-minute mile nobody believed they
four-minute mile nobody believed they
four-minute mile nobody believed they could do it and after he did that lots
could do it and after he did that lots
could do it and after he did that lots of people made that record and so we
of people made that record and so we
of people made that record and so we believe that we are so close to being on
believe that we are so close to being on
believe that we are so close to being on the cusp of being able to treat many
the cusp of being able to treat many
the cusp of being able to treat many more boys in a much more meaningful way
more boys in a much more meaningful way
more boys in a much more meaningful way so these are few tiles of arns
so these are few tiles of arns
so these are few tiles of arns estimates and it shows basically you
estimates and it shows basically you
estimates and it shows basically you know a top-down view of treating the
know a top-down view of treating the
know a top-down view of treating the whole disease whether it’s replacing the
whole disease whether it’s replacing the
whole disease whether it’s replacing the dystrophin that’s missing treating the
dystrophin that’s missing treating the
dystrophin that’s missing treating the cardiac failure we funded an initiative
cardiac failure we funded an initiative
cardiac failure we funded an initiative to help companies better understand
to help companies better understand
to help companies better understand clinical trials to accelerate the
clinical trials to accelerate the
clinical trials to accelerate the process because the FDA was being very
process because the FDA was being very
process because the FDA was being very very difficult and so we had to learn
very difficult and so we had to learn
very difficult and so we had to learn how to work with the FDA to give them
how to work with the FDA to give them
how to work with the FDA to give them the information that they need it in a
the information that they need it in a
the information that they need it in a way that they would sign off on it
way that they would sign off on it
way that they would sign off on it basically i’ll come back to X onyx and
basically i’ll come back to X onyx and
basically i’ll come back to X onyx and these other these other ones I mentioned
these other these other ones I mentioned
these other these other ones I mentioned so just recently this week in fact on
so just recently this week in fact on
so just recently this week in fact on Monday we just announced the formation
Monday we just announced the formation
Monday we just announced the formation of a new company called X onyx and I
of a new company called X onyx and I
of a new company called X onyx and I don’t know if any of you have heard of
don’t know if any of you have heard of
don’t know if any of you have heard of gene editing or crisper cast nine but
gene editing or crisper cast nine but
gene editing or crisper cast nine but it’s taken to scientific world by storm
it’s taken to scientific world by storm
it’s taken to scientific world by storm it’s a means of going in and almost like
it’s a means of going in and almost like
it’s a means of going in and almost like using scissors to edit the gene and put
using scissors to edit the gene and put
using scissors to edit the gene and put it back together the way you want it to
it back together the way you want it to
it back together the way you want it to be and there is so much hope for this
be and there is so much hope for this
be and there is so much hope for this technology to cure so many diseases and
technology to cure so many diseases and
technology to cure so many diseases and make it not just a chronic issue where
make it not just a chronic issue where
make it not just a chronic issue where you have to have ongoing treatments but
you have to have ongoing treatments but
you have to have ongoing treatments but actually a cure and this is where we’re
actually a cure and this is where we’re
actually a cure and this is where we’re moving towards so talk about problem
moving towards so talk about problem
moving towards so talk about problem solving great opportunity the problem is
solving great opportunity the problem is
solving great opportunity the problem is is that there are three companies three
is that there are three companies three
is that there are three companies three public companies that are working on
public companies that are working on
public companies that are working on Chris Burke as 9 technology their public
Chris Burke as 9 technology their public
Chris Burke as 9 technology their public companies which means they’re their
companies which means they’re their
companies which means they’re their responsibilities to the shareholders and
responsibilities to the shareholders and
responsibilities to the shareholders and so they’re going to be working on the
so they’re going to be working on the
so they’re going to be working on the low-hanging fruit first which means
low-hanging fruit first which means
low-hanging fruit first which means they’re going to be working on diseases
they’re going to be working on diseases
they’re going to be working on diseases where there’s like a single organ so
where there’s like a single organ so
where there’s like a single organ so right now they’re working on for example
right now they’re working on for example
right now they’re working on for example I issues when you look at muscle we have
I issues when you look at muscle we have
I issues when you look at muscle we have a lot of muscle in our body and it
a lot of muscle in our body and it
a lot of muscle in our body and it spread all over the place so just
spread all over the place so just
spread all over the place so just looking at the expense of treating an
looking at the expense of treating an
looking at the expense of treating an eyeball versus the muscle throughout
eyeball versus the muscle throughout
eyeball versus the muscle throughout your body with a very very very
your body with a very very very
your body with a very very very expensive delivery system and drug is
expensive delivery system and drug is
expensive delivery system and drug is overwhelming not to mention how do you
overwhelming not to mention how do you
overwhelming not to mention how do you get it throughout the body it’s one
get it throughout the body it’s one
get it throughout the body it’s one thing to
thing to
thing to inject a needle into a specific organ
inject a needle into a specific organ
inject a needle into a specific organ and cure it it’s another thing to get it
and cure it it’s another thing to get it
and cure it it’s another thing to get it throughout your body so great technology
throughout your body so great technology
throughout your body so great technology big problem they don’t want to work on
big problem they don’t want to work on
big problem they don’t want to work on dish in right now and what it’s like to
dish in right now and what it’s like to
dish in right now and what it’s like to be a parent and to see a technology that
be a parent and to see a technology that
be a parent and to see a technology that could cure your kid and nobody wants to
could cure your kid and nobody wants to
could cure your kid and nobody wants to touch it because it doesn’t fit the
touch it because it doesn’t fit the
touch it because it doesn’t fit the business model at this particular point
business model at this particular point
business model at this particular point so what do you do you find a solution
so what do you do you find a solution
so what do you do you find a solution and our solution was to form a company
and our solution was to form a company
and our solution was to form a company called X onyx we licensed the technology
called X onyx we licensed the technology
called X onyx we licensed the technology from UT Southwestern dr. Eric Olson is a
from UT Southwestern dr. Eric Olson is a
from UT Southwestern dr. Eric Olson is a premier muscle biologist in in
premier muscle biologist in in
premier muscle biologist in in Christopher technology and we just had
Christopher technology and we just had
Christopher technology and we just had our announcement this week and we are
our announcement this week and we are
our announcement this week and we are very very excited this is another
very very excited this is another
very very excited this is another project that I’m extremely excited about
project that I’m extremely excited about
project that I’m extremely excited about so a company that had a drug that failed
so a company that had a drug that failed
so a company that had a drug that failed to get approval from the FDA and yet it
to get approval from the FDA and yet it
to get approval from the FDA and yet it was shown to be effective in kids and so
was shown to be effective in kids and so
was shown to be effective in kids and so here you’ve got a dichotomy between what
here you’ve got a dichotomy between what
here you’ve got a dichotomy between what the FDA is seen on paper and what the
the FDA is seen on paper and what the
the FDA is seen on paper and what the parents and the children are seen in
parents and the children are seen in
parents and the children are seen in their lives where they’re responding
their lives where they’re responding
their lives where they’re responding everywhere from stabilization of the of
everywhere from stabilization of the of
everywhere from stabilization of the of the disease which is a good thing
the disease which is a good thing
the disease which is a good thing stabilization is really good in duchene
stabilization is really good in duchene
stabilization is really good in duchene as well as some of the kids actually
as well as some of the kids actually
as well as some of the kids actually regaining function and having a better
regaining function and having a better
regaining function and having a better lifestyle and riding bikes or they never
lifestyle and riding bikes or they never
lifestyle and riding bikes or they never will be able to do that before and just
will be able to do that before and just
will be able to do that before and just improve quality of life and then the
improve quality of life and then the
improve quality of life and then the drug is told from them because the FDA
drug is told from them because the FDA
drug is told from them because the FDA could not see what the parents saw so
could not see what the parents saw so
could not see what the parents saw so this happened about nine months ago and
this happened about nine months ago and
this happened about nine months ago and the parents have been devastated and I’m
the parents have been devastated and I’m
the parents have been devastated and I’m very close with many of those and so we
very close with many of those and so we
very close with many of those and so we we worked really hard and again here’s a
we worked really hard and again here’s a
we worked really hard and again here’s a big problem a drug that works can’t get
big problem a drug that works can’t get
big problem a drug that works can’t get it to the kid so to make a very very
it to the kid so to make a very very
it to the kid so to make a very very long story short we created another
long story short we created another
long story short we created another nonprofit called CD access and we’ve
nonprofit called CD access and we’ve
nonprofit called CD access and we’ve negotiated with the company to transfer
negotiated with the company to transfer
negotiated with the company to transfer drug to this non-profit and we are now
drug to this non-profit and we are now
drug to this non-profit and we are now in the drug distribution and delivery
in the drug distribution and delivery
in the drug distribution and delivery business there’s always a way and is
business there’s always a way and is
business there’s always a way and is always a plan B
always a plan B
always a plan B and I encourage all nonprofits to think
and I encourage all nonprofits to think
and I encourage all nonprofits to think outside the box and figure it’s not
outside the box and figure it’s not
outside the box and figure it’s not about running an organization it’s about
about running an organization it’s about
about running an organization it’s about finding a cure so this is Hawkin he was
finding a cure so this is Hawkin he was
finding a cure so this is Hawkin he was number 88 on the sage football team he
number 88 on the sage football team he
number 88 on the sage football team he was talk about finding a solution he
was talk about finding a solution he
was talk about finding a solution he loved he loved football always and when
loved he loved football always and when
loved he loved football always and when he was diagnosed with Duchenne it was
he was diagnosed with Duchenne it was
he was diagnosed with Duchenne it was just devastating he always had a ball in
just devastating he always had a ball in
just devastating he always had a ball in his hand all the time when he was young
his hand all the time when he was young
his hand all the time when he was young and so thanks to the coaches here it’s
and so thanks to the coaches here it’s
and so thanks to the coaches here it’s aight ill and my husband being
aight ill and my husband being
aight ill and my husband being persistent Hawkin was able to be part of
persistent Hawkin was able to be part of
persistent Hawkin was able to be part of the football team and it’s been just a
the football team and it’s been just a
the football team and it’s been just a tremendous tremendous story and stage
tremendous tremendous story and stage
tremendous tremendous story and stage has been such a part of our life and
has been such a part of our life and
has been such a part of our life and it’s extremely satisfying to be here
it’s extremely satisfying to be here
it’s extremely satisfying to be here he’s now at USC in journalism and
he’s now at USC in journalism and
he’s now at USC in journalism and writing up a storm so I would just like
writing up a storm so I would just like
writing up a storm so I would just like to say thank you and I would like to if
to say thank you and I would like to if
to say thank you and I would like to if possible inspire people here especially
possible inspire people here especially
possible inspire people here especially the kids that if you have some quirks
the kids that if you have some quirks
the kids that if you have some quirks that people comment on go with it
that people comment on go with it
that people comment on go with it embrace it you know it’s God gives us
embrace it you know it’s God gives us
embrace it you know it’s God gives us gifts and sometimes they’re not easy
gifts and sometimes they’re not easy
gifts and sometimes they’re not easy ones and sometimes they make you feel
ones and sometimes they make you feel
ones and sometimes they make you feel uncomfortable and and you don’t fit in
uncomfortable and and you don’t fit in
uncomfortable and and you don’t fit in really well but there’s a reason why you
really well but there’s a reason why you
really well but there’s a reason why you have a gift and my my advice to you is
have a gift and my my advice to you is
have a gift and my my advice to you is let it let it play itself out and find a
let it let it play itself out and find a
let it let it play itself out and find a way that you can use that to help other
way that you can use that to help other
way that you can use that to help other people thank you
”
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