Skip to content
Link copied to clipboard

Paul Perreault talks pros and cons of gene therapy

Maybe long term, gene therapy might derail a business like the one Paul Perreault leads in King of Prussia and Australia.

Paul Perreault has served as CEO of King of Prussia's CSL Behring since 2013.
Paul Perreault has served as CEO of King of Prussia's CSL Behring since 2013.Read moreMatthew Hall / For the Inquirer

Maybe long term, gene therapy might derail a business like the one Paul Perreault leads in King of Prussia and Australia.

After all, gene therapy, pursued by Philadelphia-based Spark Therapeutics and others, involves curing chronic conditions such as the bleeding disorder hemophilia by discovering and replacing the defective gene that causes it.

Perreault, 59, is chief executive officer and managing director of CSL Ltd., a $5.5 billion global bio-therapeutics company that, through its CSL Behring division, develops and manufactures products that treat - but don't cure - bleeding disorders, immune deficiencies, and other chronic and rare conditions.

The patient population for each condition that CSL Behring's products treat is relatively small, compared with, say, the number of people who use a drug for high blood pressure.

But they constitute a sizable repeat business, because CSL's customers tend to use its products - often infusions - for life, in order to stay alive.

Is gene therapy a concern?

While the concept of gene therapy seems simple - insert a correct copy of the defective or missing gene into the necessary cells - bringing it to reality is technically demanding, and many scientific obstacles still need to be overcome before it becomes a practical form of therapy for many diseases we treat today.

This has been a lifetime of work for many scientists, and although the human genome project launched a new scientific effort over a decade ago, the complexity of the human body, and even the slight mutations of a particular gene in patients with the same disease, has made the effort difficult.

So it's a long way off?

We don't see this as an immediate threat to our existing product portfolio or pipeline. We are also a global company, and there are still patients not being treated today around the world with current therapies. I think it will be a long time before gene therapy gets to all of these countries.

In August, CSL bought Novartis' flu vaccine business. Did this year's mild winter impact sales?

It's been a mild flu season this winter, and sales are down compared to expectations. However, in terms of next year, as we say in the flu vaccination business, if you have seen one flu season, you have seen one flu season.

It seems like a risky business. Flu strains can vary, and vaccines can end up covering wrong strains. Why get a flu shot?

You will get coverage for a number of those viruses. Is it always a hundred percent match? No. [But] we're trying to protect you from being a patient. The other thing is people have, I think, a misconception about influenza vaccine and the fact that you can also bring [the virus] home and not be sick yourself. You can be a carrier. If you get vaccinated, then you can ward off the virus.

Do you get a flu shot?

With all the time I spend in Australia, I get two shots a year, one for each hemisphere. At CSL, we make vaccines for each of the hemispheres, so we are developing and registering a new vaccine every six months. A very difficult task.

The pharmaceutical business requires so much patience, with drugs taking decades to develop. What keeps you motivated?

The patients are depending on us every day. I know it's hard, some days, to get up and come to work. For me, it's a privilege, because I know families and patients personally.

Fifteen years ago, there were actually shortages of some of these products. I had mothers calling me for product for their kids. I had congressmen calling me for constituents in their districts for product that I did not have, because we were short. I actually sourced product from competitors to help them find product, because these patients were getting sick and potentially dying. Today I get pictures of them at their graduations and at their weddings.

You have a grueling travel schedule - to Australia and 30 locations globally. Any advice for people embarking on a rough business travel career?

I have a separate bag already packed with all my toiletries. The main thing I try to do when I travel is try to get into the time zone that I'm going to as quickly as I can.

Interview questions and answers have been edited for space.

jvonbergen@phillynews.com

215-854-2769

@JaneVonBergen