Reversing symptoms with cell and gene therapies

Rewriting the Future of Healthcare

Health begins with understanding. And in recent decades, science has come to understand a lot about the human body. Particularly, our genetics. This has unlocked promising options for doctors and patients to treat some of the most debilitating diseases. Scientists are looking for answers where biology, chemistry and data science meet. 

 

Cell and gene therapies offer hope to millions of people living with genetic and some degenerative diseases. These new treatment options are a paradigm shift. They don’t just treat symptoms. They help the body repair itself from within. 

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This is Martin. His brain is capable of incredible things. It’s an organ made of tissue made of cells.

 

 

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But some of the cells in his brain aren’t functioning well. He is living with Parkinson’s disease.

 

And so are 10 million others worldwide.

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What Causes Parkinson’s?

When the cells responsible for producing dopamine begin to degenerate, the brain can’t effectively signal the body’s muscles into action. 

 

Although symptoms may vary, Parkinson’s often starts with tremors. And they can overtake the body as the disease progresses, making simple tasks like walking or holding a fork virtually impossible.

 

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For many decades, most treatments have focused on replacing dopamine with pills.

 

But scientists believe we can do better.

 

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Stem Cells

There are over 200 types of cells in the human body, and stem cells can transform into any of them. This remarkable potential means they could be used to replace poorly functioning cells in failing organs. In the case of Parkinson’s, that organ is the brain.

 

Bayer and the team at BlueRock Therapeutics are working to replace lost dopamine-producing neurons with fresh ones.

 

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Gene Therapies

The other class of therapies bringing hope to Parkinson’s patients focuses on specific genes inside the neuron, rather than replacing the entire cell.

 

We’re working with AskBio on technology that introduces a naturally occurring gene into failing cells. That gene — or string of instructions to the cell  — promotes survival. 

 

This approach is called gene therapy, and we’re looking to treat more than just Parkinson’s with it.

 

Erika
These are Erika’s muscles.

For those like Erika living with Pompe disease, an excess buildup of glycogen in their body’s cells degrades muscle growth. It’s an inherited disease which means genetic therapies might help. And that’s what we aim to do.

 

Along with AskBio we are working on a gene therapy treatment that could deliver a functional copy of the defective inherited gene, helping reverse symptoms.

 

Mateo
This is Mateo’s heart.

Using stem cell technology, along with BlueRock Therapeutics, we’re working to replace heart cells that are lost after a cardiac event like a heart attack. 

 

For those living with congestive heart failure we’re using gene therapy, alongside AskBio, to potentially help the heart heal, lowering the risk of adverse cardiac events. 

 

This approach would improve heart function by enhancing a naturally occurring protective gene.

 

Alex
These are Alex’s arms.

Limb-girdle muscular dystrophy type 2I/R9 is a genetic illness that often presents itself in childhood and weakens the body’s arms and legs. The symptoms gradually worsen over time, and people affected generally need a wheelchair about two years after onset.

 

We’re working with AskBio to create gene therapies that would provide a functioning copy of the defective inherited gene and potentially reverse symptoms.

 

Grandpa

These are only a few examples of genetic and degenerative conditions that we might be able to address with cell and gene therapies. We are working to go beyond managing symptoms to addressing the true cause of these diseases, with the potential to stop or even reverse them.

 

It’s all made possible through brilliant innovation, and open collaboration.