Genetic Medicines

Genetic medicines have the potential to transform human health in our lifetime.
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A photograph of a mother leaning over a bed playfully engaging with a laughing infant in morning light

Stopping disease at the root

Our scientists are boldly working to turn what was once thought to be impossible into possible. We’re reimagining a world where people could receive care tailored to their individual biology through new kinds of medicines made from a person’s own building blocks of genetic code. A world where people could live their lives without the risk of chronic illness. Where the very fabric of health care is transformed by potentially stopping devastating diseases before they start, through earlier diagnosis and preventative care, making medicines an ever-diminishing part of people’s lives.

Right now, genetic medicine is focused on curing disease, halting symptoms or stopping progression early in a person’s life. This is amazing and should be celebrated, but we’re only at the very beginning of the story.
Thanks to scientific advancements and dramatic progress in understanding the human genome, Lilly scientists have been able to unlock new hope for people living with chronic or currently untreatable illnesses.

We’ve already dedicated nearly a decade to solving these problems, and we’re only just getting started on our next legendary chapter. Today, more than a third of our pipeline is comprised of novel genetic medicines programs across multiple disease states where there remain significant unmet needs.
From discovering new potential treatment options for diseases with significant unmet needs, such as ALS (amyotrophic lateral sclerosis) and Parkinson's, to new approaches in cardiovascular diseases with the potential for once-a-year—or even once-per-lifetime—therapies, our scientists are making great progress in our focus areas.

Harnessing technology to change lives

This new class of medicines requires precision and accuracy in getting into the right cells and acting at the genetic level only on the intended specific target. Genetic medicines also require more advanced diagnostics that can help with earlier detection to stop a disease before it impacts someone’s health. While these are challenges to overcome, we’re advancing toward making previously “undruggable” targets now attainable and leaning on new tools and new technology to do it.
By combining machine learning with unmatched biological expertise, we’re redefining what’s possible, and pushing towards entirely new approaches.
Rapidly evolving technology, like artificial intelligence (AI), can help us decode and understand the vast amounts of genetic data before us. It is already helping us speed up our processes and identify patterns and correlations we couldn’t see before.

By combining machine learning with unmatched biological expertise, we’re redefining what’s possible, and pushing towards entirely new approaches. Our confidence lies in the knowledge that we are armed with the right people, the scientific capabilities and the technological expertise to boldly pioneer the discoveries genetic medicine has to offer.

Watch Bill's Parkinson's story

[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.]

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Waiting on a breakthrough
Through advancements in genetic medicines, we're working to give new hope to people—like Bill—living with Parkinson's disease.
Watch now