Leadership

The promise of gene therapies

June 2024

It was exciting to see the news recently that the first patient – a 12-year-old boy – has begun a new gene therapy for sickle cell disease. It’s one of two recent gene therapies for this disease approved by the U.S. Food and Drug Administration.

Both treatments target the underlying cause of the disease – a faulty gene that the patient has inherited from their parents. Sickle cell disease is a debilitating and life-threatening blood disorder that affects about 100,000 people in the United States, alone, and these treatments have the potential to transform patients’ lives. 

Since 1990, when the first gene therapy was approved for a severe immunodeficiency disease, others have emerged for Duchenne muscular dystrophy, hemophilia and a retinal disorder that causes blindness, among other hereditary diseases. I’m excited to see the pace of discovery is accelerating.

 

While most of the progress has been in the realm of rare inherited diseases, gene therapy techniques – like CRSPR gene editing and lentiviral vector delivery – are being applied to development of potential treatments for various forms of cancer and other more common illnesses – even ways to address aging. It’s an incredibly promising frontier.

Quotation marks
"There are still major hurdles to overcome as more gene therapies emerge: treatment can be long and grueling for the patient, the therapies are costly and availability can be limited. That’s why Thermo Fisher is committed to doing everything we can to help our customers bring new treatments to market faster and with lower cost, so we can improve lives worldwide."
Marc Casper
President and CEO
Thermo Fisher Scientific

There are still major hurdles to overcome as more gene therapies emerge: treatment can be long and grueling for the patient, the therapies are costly and availability can be limited. That’s why Thermo Fisher is committed to doing everything we can to help our customers bring new treatments to market faster and with lower cost, so we can improve lives worldwide.

 

Our enabling technologies, services and expertise advance research, cutting-edge drug discovery and development, and our capabilities to support clinical trials and manufacture new therapies at scale help our customers accelerate development and commercialization. We continue to invest in these capabilities so we can help our customers move their breakthroughs forward and reach patients faster.

 

Thank you to all of our colleagues for the passion they bring to this work, and congratulations to our customers and all the innovators who are bringing new hope to patients everywhere. 

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