Genetic medicines

00:00-00:02
[Close-up of a woman’s eyes, her face streaked by sun.]
00:02-00:04
[A flock of birds fly in front of a sunset.]
00:04-00:05
[A tree branch moves in the wind. Cut to a field of reeds moving in the wind.]
00:05-00:06 [A highway overpass through a window, partially obscured by a curtain. Cut to a traffic at night in an Asian city.]
00:06-00:09
[A large wave crashes, the camera goes underwater. Fade to black.]
00:10-00:14
[White text appears: “There is nothing wrong with your speakers.”]
00:15-00:19
[Text changes to: “The sounds you may - or may not - hear are deliberate.”]
00:19-00:23
[A man in a suit (Manny Simons) walks up to a piano. Cut to a close-up of hand his pushing down on a key.]
00:24-00:26
[Continuation of the previous shot. Cut to Manny narrating on screen.]
Manny: You can have a really rich, meaningful life
00:26-00:29
[A slightly wider shot of Manny narrating.]
Manny: without hearing, and people do every day,
00:29-00:31
[A close-up of an ear. The camera moves down the ear.]
Manny: but there are thousands of children born every year
0:31-00:34
[A continuation of the previous shot. Cut to Manny on screen.]
Manny: with a disabling hearing loss.
00:34-00:35
[Manny on screen again narrating. Lower third appears on screen: “Manny Simons, VP, Gene Therapy at Lilly, Founder & CEO at Akouos”.]
Manny: There are hundreds of millions of people around the world
00:35-00:38
[Manny continues speaking.]
Manny: who have disabling hearing loss of various forms.
00:38-00:40
[Manny continues speaking.]
Manny: There have been no medicines developed
00:40-00:43
[Manny continues speaking. Cut to Manny walking up to a grand piano. He places a finger onto a key.]
Manny: for any of these forms of hearing loss.
00:45-00:48
[Close-up inside the piano of a piano string being struck. Cut back to Manny standing in front of the piano.]
00:48-00:52
[Previous shot continues. Manny sits down at the piano.]
Manny: For me, hearing has been a central part of my life.
00:52-00:53
[Close-up of Manny’s hand playing the piano.]
00:53-00:57 [Contuation of previous shot. Cut to Manny’s face as he plays.]
Manny: Music was everywhere in our home growing up.
00:57-00:59
[Manny’s face comes into focus; he is not speaking.]
Manny: I was thinking about becoming a musician
00:59-01:02
[A different shot of Manny looking at the camera. Cut to Manny’s hands playing the piano.]
Manny: before I fell in love with science and medicine.
01:03-01:06
[Close-up inside the piano of the strings string being struck.]
Manny: When I started to think about what it would be like
01:06-01:09
[Manny on screen, speaking.]
Manny: for someone to be born without hearing
01:09-01:11
[Medium shot of Manny sitting at piano, playing.]
Manny: or lose their hearing, if there was a way to give that back
01:11-01:14
[Back to Manny speaking.]
Manny: or to make hearing available to those who wanted it,
01:14-01:17
[Zoomed in shot of Manny speaking. Jump cut of Manny smiling and laughing in the same spot.]
Manny: to me it seemed like a way that we could have
01:17-01:19
[Continuation of the previous shot. Cut to Manny’s hand striking a single key on the piano.]
Manny: a really dramatic impact on someone's life.
01:20-01:23
[Akouos sign and logo on top of blinds. Text under it reads: “A Wholly Owned Subsidiary of Eli Lilly and Company”. Cut to Manny’s face as he plays piano.]
Manny: Akouos is working to open the experience of hearing
01:23-01:25
[Close up of piano keys, Manny strikes one.]
Manny: to as many people as possible.
01:25-01:29
[A gloved hand holds a sample in a dish over a light. Cut to a female scientist looking into a microscope. Cut to her hands under the microscope, moving the sample with tweezers. Cut to hands, removing something from a small jar with tweezers. Cut to a close-up of the scientist’s eye looking into the microscope.]
Manny: You begin development of a new medicine with the expectation
01:29-01:30
[Manny’s hand strikes another key.]
Manny: that it takes a lot of time.
01:30-01:33
[A man in protective wear stands before a large barrel and pulls a handle on it. Cut to Manny playing the piano.]
Manny: It's a long process to make sure
01:33-01:35
[A large sealing lid is removed from the barrel.]
Manny: that what you're bringing forward
01:35-01:37
[A block is pulled out of the barrel as mist forms around it. Cut to the man’s face covered in a safety visor.]
Manny: is safe and as effective as possible.
01:38-01:41
[Manny on-screen speaking. Cut to a headline on the screen: “New York Times - Gene Therapy Allows an 11-Year-Old Boy to Hear for First Time.”]
Manny: In the case of this first clinical trial,
01:41-01:43
[Two new headlines replace the previous one. “Bioworld Med Tech - Hear here: Lilly’s Akouos gene therapy restores hearing loss in an 11-year-old” and “Genetic Engineering and Biotechnology News - Gene Therapy Restores hearing in 11-Year-Old After Just One Month.”]
Manny: we were very happy to see such a meaningful,
01:43-01:46
[New headline replaces the previous: “MedCity News - Eli Lilly Sounds off as Early Date Show How Gene Therapy Can Restore hearing. Then: “Precision Medicine Online - After Promising Early Efficacy, Eli Lilly Eager to Study Hearing Loss Gene Therapy in More Children”. then , “The Hearing Review - Clinical Trial for Gene Therapy for Genetic Hearing Loss Gets Positive Results.” Then “Clinical Trials Arena - Akouos reports positive data from gene therapy trial for hearing loss.”]
Manny: dramatic effect in the first participant.
01:46-01:50
[A boy walks up to a window. Cut to him looking out the window.]
01:50-01:52
[Manny on-screen speaking.]
Manny: It's an incredible motivator to keep pushing the boundaries
01:52-01:56
[Manny continues speaking. Cut to female scientist in lab, holding a device with a tube going into a bottle filled with liquid. Cut to a closer shot of the scientist’s face looking down.]
Manny: of what we can achieve to have a similar effect
01:56-01:59
[Cut to the same scientist operating a device in the lab. Cut to a different angle, as she is operating the same device. Cut to Manny at the piano. Cut to the inside of the piano, the strings being struck.]
Manny: in as many more people as we possibly can.
02:00-02:04
[Exterior front shot of the Lilly Seaport building. Cut to a wide shot of the whole building.]
Manny: The expansion of Lilly into the Seaport
02:04-02:07
[Continuation of the previous shot. Cut to an interior of the building, two people walk down a hall with the Lilly logo in view on a wall.]
Manny: and genetic medicine broadly into Boston over time
02:07-02:10
[Continuation of the previous shot. Cut to a close-up of the Lilly logo on the wall.]
Manny: provides an opportunity to bring together different groups
02:10-02:13
[A male scientist in a lab looks down at something. Cut to a screen displaying data that the scientist is looking at.]
Manny: with different expertise, different therapeutic areas
02:13-02:15
[Cut to a close-up of the data, with a visual depiction of the sample being analyzed. Cut to a small motor spinning inside of a glass bottle. Cut to the male scientist looking at the screen from below.]
Manny: coming together to develop the types of medicines
02:15-02:18
[The camera pans up as Manny plays the piano. Cut to a scientist operating a device. Cut to another scientist looking through a microscope. Cut to another scientist distributing liquid into a beaker.]
Manny: that we didn't think were possible five or ten years ago.
02:18-02:20
[Cut to a close-up of the Lilly logo on the exterior of the building.]
Manny: What we're doing is
02:20-02:24
[Manny on-screen speaking.]
Manny: working to take smart risks on bold science
02:24-02:26
[Manny continues speaking. Cut to gloved hand, placing vials into a holder.]
Manny: to make life-changing medicines for patients.
02:26-02:28
[Cut to a close-up of a scientist with safety glasses on.]
02:28-02:29
[Cut to a woman looking up at the sky; her hand casts a shadow on her face.]
02:29-02:30
[An exterior of the Seaport building, a plane passes by in the background.]
Manny: That is really exciting.
02:30-02:32
[A continuation of the previous shot. Cut to Manny on-screen speaking.]
Manny: You come to work every day not knowing exactly
02:32-02:34
[Continuation of the previous shot. Cut to a child running through a sprinkler. Cut to a wide shot of the child another child playing with her.]
Manny: what it's going to look like, but believing
02:34-02:37
[Three children chase a soccer ball on a field. Cut to a woman playing violin in a home library. Cut to a child on a swing. Cut to a mother holding a baby up to her face and touching their noses. Cut to a woman sitting on a surfboard. Cut to a child in a superhero costume running through a room.]
Manny: that it's going to ultimately have a real impact on people.
02:39-02:41
[Close-up of Manny’s face as he plays piano. Cut to his hands as he stops. He pulls them off the keys.]
02:43-02:47
[Lilly logo appears over a red background.]

[Music playing.]

[Red Lilly Logo animation appears on screen on a white background.]

[Black-and-white video shows a man walking in gym shoes on a basketball court. Scenes of men playing basketball.]

00:05-00:29
Bill McCleery: It's really interesting when I play basketball because I don't notice the symptoms when I'm on the court. Sometimes I'll limp into the gym and then get on the basketball court and feel totally normal as I'm running around, and then I'll get done playing and I'll limp out of the gym back to my vehicle. So I don't know if that makes that difference, but there's something that kicks in when you're just dribbling the ball, doing the things that you've done all your life to play that.
[Scenes of men playing basketball transition into a man sitting in his living room. The video transitions from black and white to color.]

00:29-00:57
Bill McCleery: It's just really therapeutic for me, and it just makes the disease not even seem like it's there at that moment. My name is Bill. I was diagnosed at age 50 with Parkinson's disease. Playing basketball as my primary therapy. Twice a week, half court with the guys. I'm the only one that has Parkinson's, and I can still fit into that peer group and play basketball with a group of older guys.


00:57-01:06
Bill McCleery: So I fear or have some trepidation about the time coming that I can't do those sorts of things. I was missing some outside shots.
[A man and woman sit in the stands in the gym watching the basketball game.]

01:06-01:07
Amy McCleery: Well I say good game.

01:07-01:20
Bill McCleery: A good game. All right. Thank you very much.
[A man sitting in his living room.]

They say the non-motor symptoms often come long before the motor symptoms. And I would say I did first experience anxiety and fatigue in the years leading up to my diagnosis and thought something was off.

[A woman is sitting in the living room talking. Video shows scenes of the couple walking and talking.]

01:20-01:36
Amy McCleery: We used to walk together. We would walk a couple of miles every night, and then it got to a point where his toes curl. And so we really got to the point we could not work together. Like that was kind of our time. At the end of the day, you know, it was kind of a grieving process of, we can't do this together.

01:37-01:39
Amy McCleery: But then we just started to find like other things that we could do.


01:39-01:45
Bill McCleery: We'll be holding hands and I squeeze a little bit too hard just without meaning to. And she'll be like, ouch. And loosen up a little bit on the hand-holding.
[Video shows scenes of the couple cooking together and eating dinner with their daughter.]

01:45-01:58
Amy McCleery: We were in a doctor's office and we saw someone in the later stages. The older gentleman was in a wheelchair. At some point, maybe that's or maybe that's in our future, I don't know. We'll figure that out when we get there.
[Video of a man using a computer]

01:58-02:08
Bill McCleery: You know, I'd love to think that there's some research happening somewhere that's going to lead to a great breakthrough. And I'm definitely interested in whether genetic science can be the premise of a cure for Parkinson's.
[Video transitions to a man in a blue suit coat being interviewed in an office setting. Scenes of the man working in the lab are shown.]

02:08-02:26
Andrew Adams: With these kinds of progressive diseases where your brain is degrading, we need to actually focus on what's the root cause and address the root cause of those diseases. And that's where genetic medicine comes in, because we don't want to do incremental things where we target symptoms. What we're looking to do here is make transformational medicines.
[Video transitions to a woman in a dark red jacket being interviewed. Video shows scenes of scientists working in a lab.]

02:26-02:50
Michelle Lynn Hall: Genetic medicine is basically doing everything that a lot of the medicines have done for years, but very intentionally focusing on treating and curing diseases at their foundational and root level. You come into your health care provider’s office, and we would give you a treatment that would get into your cells, get into the nucleus, and fundamentally correct a pathogenic mutation in your DNA.


02:50-03:04
Michelle Lynn Hall: And you would be done. I'm here because I think the most impactful thing I could do as a scientist in my career is create cures and treatments for debilitating neurodegenerative diseases like ALS, like Alzheimer's, like Parkinson's.
[Video transitions to a man on a porch swing talking with his daughter.]

03:04-03:06
Hannah McCleery: How have you been dealing with all the symptoms and everything?


03:07-03:18
Bill McCleery: I'm pretty good. Take my meds. like, my left hand acts up now and then. But I'm just glad that my left hand on my right. Yeah, that's not my shooting hand playing basketball. Yeah.


03:18-03:20
Hannah McCleery: To make the three pointers.
[Video transitions to show a man sitting in his living room. Video shows scenes of the man filling up a bird feeder in his yard, and we then see him back on the basketball court.]

03:20-03:45
Bill McCleery: That's right. With three daughters I do look forward to things like being part of their weddings. being part of grandkids someday. I don't think Parkinson's has to be a barrier or an impediment to that. I look to the future and plan on being there and being active and being part of things for years to come. One thing I've told my neurologist is that if she'd like to become famous by becoming the person to cure Parkinson's, I'll volunteer to be the person she cures it in.


03:45-03:54
Bill McCleery: I mean, I do hope there's a cure someday, whether it benefits me or generations down the road. So I'm hopeful. I hope to be able to continue that and to stay positive no matter what.
[Red Lilly logo appears.]

[Music fades in the background.]