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I am a scientist and a drug developer. I 00:10
develop gene therapy designed to fix a 00:15
single error, a typo in the human 00:18
generic code that could cause life 00:22
written diseases such as blindness. 00:25
totally 00:28
lost of a human vision. 00:31
But I truly understand the purpose 00:34
through a student as a rare disease 00:36
degenerated her retina. The tax in her 00:41
books dissolved. 00:45
She losed the ability to navigate campus 00:48
and had to leave from university. Her 00:51
word photo of potential 00:55
began to really close in. 00:58
Medicine calls her condition rare 01:02
because it affect fewer than one in 01:06
2,000 people. 01:10
But here's the truth that reshaped my 01:13
life's work. There are over 7,000 such 01:16
rare diseases. 01:20
At every story together, you will find 01:23
a patient population of 350 million 01:28
people worldwide. 01:32
That's a population of United States, 01:34
Canada, and Australia combined. 01:38
Now, one in 15 people affected globally. 01:42
My folks on one gene, one disease 01:48
is not a niche pursuit. It is one vital 01:53
piece of 350 million people puzzle. 01:56
We medicalized isolation by calling them 02:02
rare. 02:05
Solving this is how we build a future 02:07
that everyone counts. 02:11
Those 02:13
puzzles have a devastating human cost 02:15
defined by two crew gaps. 02:19
The first gap is a diagnostic gap. For 02:22
progressive diseases, 02:26
times equals loss function. Yet 02:29
not everyone can receive a genetic test 02:34
that tells you what's wrong on the DNA 02:37
before it become actionable. 02:41
Even you can find the answer 02:44
but often after the time or the best 02:47
window for intervention has closed. 02:50
The second is a treatment gap. 02:53
After that long search, a harder truth 02:56
emerges. 03:00
Only a few tiny percentage of the rare 03:02
disease have a proved therapy. For the 03:06
vast majority, the diagnosis is an 03:09
answer but not a cure. 03:13
Patients exchange 03:16
the wildness of the unknown for the 03:18
agony of the untreatable. 03:20
It takes 10 to 15 years to develop one 03:23
medicine from discovery to market by 03:26
following a historical approach. 03:30
The most heartbreaking result 03:35
disease causes about 30% 03:39
of the deaths in UNATO intensive care 03:43
ICU. 03:46
Those are babies whose puzzle we cannot 03:48
solve. 03:51
is either science or speed in time. 03:53
This is not a just a challenging. It's a 03:57
human emergency hiding in plain sight. 04:00
So how do we solve the puzzle? 04:05
My field gene therapy aims for one-time 04:09
treatment that correct the error at the 04:13
root. However, 04:17
no single group can tackling 7,000 04:19
diseases. This is a global mission. 04:23
We design and test one medicine in one 04:29
country, 04:32
trial cross nations, share data 04:33
worldwide. 04:37
We overcome 04:39
scientific challenging, 04:41
regulatory barriers, 04:43
finance hardship. 04:45
We leverage global resources, 04:47
cutting edge technology like AI to 04:51
accelerate this, 04:54
turning a search from years to months 04:56
into days. 05:01
our most crucial partner, the patient 05:04
and the family. 05:07
They're the heroes 05:10
sharing their personal story and the 05:12
data advocating for research and the 05:15
resources. Most importantly as true 05:19
trailblazer 05:22
participant in clinical trial to test 05:24
the safety and the efficacy of the 05:27
medicine experimental medicine before it 05:29
become available to others. Their true 05:33
architect co-architect of their solution 05:37
consider the most recent proof of what's 05:42
possible 05:45
at Children Hospital of Philadelphia. 05:47
A baby named KJ was born with a severe 05:50
rare disease. 05:55
His body cannot process a protein due to 05:57
a single error on his DNA. 06:00
He spent the first few months in the 06:05
hospital on extremely limited diet. 06:07
Then at just six months later, he become 06:12
the first child to receive a customized 06:16
gene editing therapy that fix that 06:20
single error on his DNA designed for 06:23
only him. 06:26
The medicine was not made by a single 06:29
person or single group. 06:31
It takes collective effort from 06:34
biologist, 06:37
doctors, 06:39
regulators, 06:41
drug developers, 06:42
and the family who refused to wait. 06:44
All working together cross discipline in 06:49
record time. 06:52
The treatment worked. Today, KJ is not 06:54
only alive, 06:58
he's living and growing, thriving 07:00
in a life his family never thought 07:05
possible. 07:08
This is a medicine for one person, one 07:11
era, and one cure. 07:14
Kudos to those who have worked so hard 07:17
to make this possible. 07:20
And now the regulatory agency has opened 07:23
a new path to make those personalized 07:27
treatment possible. 07:31
All because our community has show all 07:34
are possible when we work together. 07:39
What emerges when pieces connect? The 07:42
hope 07:45
revolutionize all medicine. The 07:47
experience we learned from ultra rare 07:50
disease namely N equal to one 07:53
for example for KJ personalized 07:57
treatment for more common ones 08:00
the collaborative model 08:03
become a blueprint for solving global 08:06
puzzle. 08:09
See rare 08:12
not as footprint 08:14
but as 350 08:17
million teachers showing us the limit of 08:20
our of our system and the bonelessness 08:23
of human resilience. 08:26
When we work together we build a world 08:29
that solve the puzzles. We build a world 08:32
that understand the fundamental truth 08:36
in health, 08:39
in humanity, everyone counts. 08:41
Most recently, I have heard the student 08:45
I mentioned at the beginning of my talk, 08:48
the one who dropped her from university 08:50
is now planning her return to to the 08:54
university overseas after receiving a 08:56
vision restoration gene therapy. 08:59
She's able to read the textbook again. 09:02
For her, a new life has been restored. 09:07
This is why we work 09:12
to accelerate this hope 09:16
and make it available for millions 09:21
waiting. 09:23
Because here's a common sense that go 09:26
far beyond the medicine. 09:30
Every single one of us has felt like a 09:32
puzzle piece that doesn't quite fit. 09:35
Whether from a hidden struggle 09:39
or m misunderstood differences or a 09:43
moment when the world moved too fast and 09:46
left us behind. 09:50
The 350 million people affected by rare 09:52
disease are not a separate category. 09:55
They're a mirror. Their fight 09:59
for a diagnosis 10:02
reflects our own long to be seen. Their 10:05
wait 10:09
for treatment echoes every time we have 10:11
prayed for a second chance in our life. 10:15
Solving this puzzle is not just an act 10:20
of medicine. It's a declaration that 10:23
no person should be considered too few 10:27
to matter, too different to help 10:30
or too small to count. 10:35
This is a scene that bind us all in the 10:40
world of billions. 10:44
Every single story 10:47
is a piece of the same human puzzle. 10:49
We cannot do this alone. 10:54
Amplify 10:58
the possibility and the promise from n = 11:00
1 11:04
to n = to 350 million and hopefully to 11:06
everyone. 11:11
It will take 11:15
yes all of us. 11:17

– English Lyrics

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Lyrics & Translation

[English]
I am a scientist and a drug developer. I
develop gene therapy designed to fix a
single error, a typo in the human
generic code that could cause life
written diseases such as blindness.
totally
lost of a human vision.
But I truly understand the purpose
through a student as a rare disease
degenerated her retina. The tax in her
books dissolved.
She losed the ability to navigate campus
and had to leave from university. Her
word photo of potential
began to really close in.
Medicine calls her condition rare
because it affect fewer than one in
2,000 people.
But here's the truth that reshaped my
life's work. There are over 7,000 such
rare diseases.
At every story together, you will find
a patient population of 350 million
people worldwide.
That's a population of United States,
Canada, and Australia combined.
Now, one in 15 people affected globally.
My folks on one gene, one disease
is not a niche pursuit. It is one vital
piece of 350 million people puzzle.
We medicalized isolation by calling them
rare.
Solving this is how we build a future
that everyone counts.
Those
puzzles have a devastating human cost
defined by two crew gaps.
The first gap is a diagnostic gap. For
progressive diseases,
times equals loss function. Yet
not everyone can receive a genetic test
that tells you what's wrong on the DNA
before it become actionable.
Even you can find the answer
but often after the time or the best
window for intervention has closed.
The second is a treatment gap.
After that long search, a harder truth
emerges.
Only a few tiny percentage of the rare
disease have a proved therapy. For the
vast majority, the diagnosis is an
answer but not a cure.
Patients exchange
the wildness of the unknown for the
agony of the untreatable.
It takes 10 to 15 years to develop one
medicine from discovery to market by
following a historical approach.
The most heartbreaking result
disease causes about 30%
of the deaths in UNATO intensive care
ICU.
Those are babies whose puzzle we cannot
solve.
is either science or speed in time.
This is not a just a challenging. It's a
human emergency hiding in plain sight.
So how do we solve the puzzle?
My field gene therapy aims for one-time
treatment that correct the error at the
root. However,
no single group can tackling 7,000
diseases. This is a global mission.
We design and test one medicine in one
country,
trial cross nations, share data
worldwide.
We overcome
scientific challenging,
regulatory barriers,
finance hardship.
We leverage global resources,
cutting edge technology like AI to
accelerate this,
turning a search from years to months
into days.
our most crucial partner, the patient
and the family.
They're the heroes
sharing their personal story and the
data advocating for research and the
resources. Most importantly as true
trailblazer
participant in clinical trial to test
the safety and the efficacy of the
medicine experimental medicine before it
become available to others. Their true
architect co-architect of their solution
consider the most recent proof of what's
possible
at Children Hospital of Philadelphia.
A baby named KJ was born with a severe
rare disease.
His body cannot process a protein due to
a single error on his DNA.
He spent the first few months in the
hospital on extremely limited diet.
Then at just six months later, he become
the first child to receive a customized
gene editing therapy that fix that
single error on his DNA designed for
only him.
The medicine was not made by a single
person or single group.
It takes collective effort from
biologist,
doctors,
regulators,
drug developers,
and the family who refused to wait.
All working together cross discipline in
record time.
The treatment worked. Today, KJ is not
only alive,
he's living and growing, thriving
in a life his family never thought
possible.
This is a medicine for one person, one
era, and one cure.
Kudos to those who have worked so hard
to make this possible.
And now the regulatory agency has opened
a new path to make those personalized
treatment possible.
All because our community has show all
are possible when we work together.
What emerges when pieces connect? The
hope
revolutionize all medicine. The
experience we learned from ultra rare
disease namely N equal to one
for example for KJ personalized
treatment for more common ones
the collaborative model
become a blueprint for solving global
puzzle.
See rare
not as footprint
but as 350
million teachers showing us the limit of
our of our system and the bonelessness
of human resilience.
When we work together we build a world
that solve the puzzles. We build a world
that understand the fundamental truth
in health,
in humanity, everyone counts.
Most recently, I have heard the student
I mentioned at the beginning of my talk,
the one who dropped her from university
is now planning her return to to the
university overseas after receiving a
vision restoration gene therapy.
She's able to read the textbook again.
For her, a new life has been restored.
This is why we work
to accelerate this hope
and make it available for millions
waiting.
Because here's a common sense that go
far beyond the medicine.
Every single one of us has felt like a
puzzle piece that doesn't quite fit.
Whether from a hidden struggle
or m misunderstood differences or a
moment when the world moved too fast and
left us behind.
The 350 million people affected by rare
disease are not a separate category.
They're a mirror. Their fight
for a diagnosis
reflects our own long to be seen. Their
wait
for treatment echoes every time we have
prayed for a second chance in our life.
Solving this puzzle is not just an act
of medicine. It's a declaration that
no person should be considered too few
to matter, too different to help
or too small to count.
This is a scene that bind us all in the
world of billions.
Every single story
is a piece of the same human puzzle.
We cannot do this alone.
Amplify
the possibility and the promise from n =
1
to n = to 350 million and hopefully to
everyone.
It will take
yes all of us.

Key Vocabulary

Start Practicing
Vocabulary Meanings

scientist

/ˈsaɪəntɪst/

B2
  • noun
  • - A person who is studying or has expert knowledge of one or more of the natural or physical sciences.

therapy

/ˈθɛrəpi/

B2
  • noun
  • - Treatment intended to relieve or heal a disorder.

blindness

/ˈblaɪndnəs/

B1
  • noun
  • - The state or condition of being unable to see.

navigate

/ˈnævɪɡeɪt/

B2
  • verb
  • - To find one's way over or through an area.

potential

/pəˈtɛnʃəl/

B2
  • noun
  • - Latent qualities or abilities that may be developed.

diagnostic

/ˌdaɪəɡˈnɒstɪk/

C1
  • adjective
  • - Concerned with the identification of the nature of an illness.

devastating

/ˈdɛvəsteɪtɪŋ/

B2
  • adjective
  • - Highly destructive or damaging.

intervention

/ˌɪntəˈvɛnʃən/

C1
  • noun
  • - The action of becoming involved in order to improve a situation.

agony

/ˈæɡəni/

C1
  • noun
  • - Extreme physical or mental suffering.

regulatory

/ˈrɛɡjʊlətəri/

C1
  • adjective
  • - Serving or intended to regulate something.

accelerate

/əkˈsɛləreɪt/

B2
  • verb
  • - To begin to move more quickly.

advocating

/ˈædvəkeɪtɪŋ/

C1
  • verb
  • - Publicly recommend or support.

trailblazer

/ˈtreɪlˌbleɪzər/

C2
  • noun
  • - A person who makes a new track through wild country; a pioneer.

efficacy

/ˈɛfɪkəsi/

C2
  • noun
  • - The ability to produce a desired or intended result.

thriving

/ˈθraɪvɪŋ/

B2
  • adjective
  • - Prosperous and growing; flourishing.

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Key Grammar Structures

  • A typo in the human genetic code that could cause life-threatening diseases such as blindness.

    ➔ Relative clause (defining)

    ➔ The clause 'that could cause...' modifies the noun 'typo', providing essential information.

  • She lost the ability to navigate campus and had to leave from university.

    ➔ Modal verb of obligation (past form)

    ➔ 'Had to' is used here to indicate an external obligation in the past.

  • Medicine calls her condition rare because it affects fewer than one in 2,000 people.

    ➔ Subordinating conjunction of reason

    ➔ 'Because' introduces the reason for calling the condition 'rare'.

  • Not everyone can receive a genetic test that tells you what's wrong on the DNA.

    ➔ Noun clause (as object)

    ➔ 'What's wrong on the DNA' functions as the object of the verb 'tells'.

  • It takes 10 to 15 years to develop one medicine.

    ➔ Cleft sentence structure (It + takes + time + to)

    ➔ 'It' is a dummy subject used here to express the duration of a process.

  • This is not just a challenge; it's a human emergency hiding in plain sight.

    ➔ Participial phrase

    ➔ 'Hiding' (present participle) modifies 'emergency', indicating its state.

  • The baby became the first child to receive a customized gene editing therapy.

    ➔ Infinitive as post-modifier

    ➔ 'To receive' modifies 'first child', specifying the action.

  • When we work together, we build a world that solves the puzzles.

    ➔ First conditional (real present/future condition)

    ➔ The 'When' clause sets the condition for the main action.

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