Gene Editing and Precision Medicine: The Future of Healthcare?

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Dr. Oscar Segurado, Chief Medical Officer at ASC Therapeutics, joined Natalie Yeadon in the latest episode of Impetus Digital‘s Fireside Chat series. Among other things, they discussed advanced gene therapies, gene editing, and personalized medicine. They also explored the many different hats he wears as CMO, consultant, and academic, and much more.

Here is a preview of our conversation:

Q: Can you speak a bit about the evolution of precision medicine as it applies to ASC Therapeutics and around these precise medicines in general?

A: I had the opportunity to learn clinical development of biologics in the 2000s while working with the Humira team at AbbVie. At that time, anti-TNF, an immunomodulatory cytokine, was a blockbuster, with no need for selecting or monitoring patients. So there was no need for biomarkers. Through my work over the last decade, focusing on gene and cell therapy programs, especially in rare diseases, I’ve realized how critical it is to apply all this knowledge about how to select patients and how to monitor patients through biomarkers. That is really going to help us have this kind of targeted, precision, personalized medicine. There’s no doubt that this is the future. Now, it’s going to take some time.

I can give you an example on Hemophilia A that we’re doing. I mentioned a moment ago that we have a different type of Factor VIII gene that we provide to the patients to start producing Factor VIII. When we provide this gene and put that around the nucleus, with a promoter, and start producing Factor VIII, we’re forcing the hepatocyte to do something that it is not used to doing, which is producing that type of product. Although the hepatocyte is a chemical factory, a biofactory, that produces hundreds and hundreds of different products, we need to see how the hepatocyte is in that single patient who receives our gene therapy. If that patient has any kind of pre-existing disease, that could be any kind of fibrosis, which is due to alcohol or non-alcoholic hepatitis, or because the patient had any kind of viral infection like Hepatitis B or C, the hepatocyte is not going to be able to produce Factor VIII as well compared to another patient who is young and has a very healthy liver.

We need to identify and select patients. We’re going to characterize those cells and look into functional ways to see how they are responding to our therapy. We’re also going to monitor them once they get the therapy. We are just incorporating our Phase I clinical trial request from the FDA, which is that the patients should get ultrasound every 6 months for 10 years. Why is that? Because some patients seem to potentially develop any kind of dysfunction in the liver. We have a number of tests, besides liver enzymes and so on, where we are going to be able to define how these patients are doing. We are collaborating, for instance, with Siemens, with a number of biomarkers in development. We’re also going to collaborate in looking at how these patients respond.

The use of biomarkers is absolutely critical in the way we combine all these new learnings on human biology and the way these patients are selected and monitored…

For more of our discussion, you can watch the whole Fireside Chat with Dr. Oscar Segurado, or listen to the podcast version, below.

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