Most people know Dr. Leonard Schleifer as the CEO of Regeneron, one of the world’s largest biotech companies. But he is also the father of a child with a rare disease. Dr. Schleifer and his wife Harriet know the challenges of navigating an uncertain regulatory environment. They join the conversation to talk about the challenges even the most connected advocates face, and what’s needed to overcome hurdles to advance the science the rare disease community needs.
Dr. Leonard Schleifer, Regeneron CEO
Harriet Schleifer, Retired Attorney and Advocate
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I think there's one mistake that we're all making here today. Genes do not define individuals. Even people with genetic diseases, they identify individuals. But we are not the product of one gene. Even a gene that has a mistake in it that identifies the syndrome or the disease, but it's the whole constellation. And what we're finding even in TKT deficiency, and I'm sure it's true for everybody here who knows somebody with a genetic disease, that they're not identical twins to others who have. So we should remember to say we're identifying people, not defining them.
I think there has to be two ways we think about how we encourage investment in ultra rare diseases. First of all, when there are small companies doing it, there has to be a path to something for to get investors to do that. And if there isn't a path, the capital is not going to be there. When you're as large as Regeneron, we have 50 things in clinical development. Probably maybe 10 are for ultra rare diseases. We've even thought about a couple of them just giving them away. And that's okay. It's like the general practitioner in the old days, he could charge the people who could afford. And if somebody came in and couldn't afford, that's okay, too. If you're a large company like we are, you can do something like that. But you also learn so much from studying these rare diseases. The one that moves me the most is we've taken children who are profoundly deaf. You put a jet engine next to them and they hear nothing. And with gene therapy, they have gotten normal hearing. It's unbelievable. I mean, it really, anybody who watches the video of this, it makes you cry. And we're not going to make any money on this. Okay. But maybe we learn about how to deal with age related hearing loss or other conditions. Maybe we learn how that gene interacts. It's called otoerline in the inner ear, how it interacts in other hearing disorders. So, but there has to be a path because our system works based on these kinds of incentives.
Schleifer, L. (2026, March 5). The Limits of Power and Advocacy Dr. Leonard Schleifer and Retired Attorney Harriet Schleifer [Interview transcript]. CNBC Events. CEOInterviews.AI. https://ceointerviews.ai/interview/744903/
MLA
Leonard Schleifer. "The Limits of Power and Advocacy Dr. Leonard Schleifer and Retired Attorney Harriet Schleifer." CNBC Events, 5 Mar. 2026. Transcript, CEOInterviews.AI, https://ceointerviews.ai/interview/744903/.
BibTeX
@misc{schleifer2026_744903,
author = {Leonard Schleifer},
title = {The Limits of Power and Advocacy Dr. Leonard Schleifer and Retired Attorney Harriet Schleifer},
howpublished = {Interview transcript, CNBC Events. CEOInterviews.AI},
year = {2026},
month = {mar},
url = {https://ceointerviews.ai/interview/744903/},
note = {Speaker-attributed transcript with timestamps}
}
