Chief Scientific Officer & Senior Vice President of Research and Early Development, Biomarin Pharmaceutical
Search every verified Kevin Eggan interview, podcast appearance, and on-the-record quote — each transcript cross-checked by AI and human review to confirm speaker identity. Kevin Eggan, Chief Scientific Officer and Senior Vice President of Research and Early Development at BioMarin Pharmaceutical, has described the company as having "an overwhelming track record of success" and "an incredible scientific discovery engine which is blazing the way in AAV gene therapies." He stated that the company aims to "take advantage of this revolution in genetics and genomics to really understand the cellular circuitry which controls diseases" and bring "transformative therapies to patients with severe genetic illness." Eggan has continued to discuss his research on amyotrophic lateral sclerosis (ALS), including work on the stathmin 2 protein. He reported that loss of stathmin 2 in mice leads to age-dependent motor decline and that oligonucleotides targeting a cryptic exon in the stathmin 2 gene have been identified that restore normal expression after TDP-43 knockdown, with a lead oligo currently in clinical study. Eggan also noted that levels of cryptic exon-containing RNA in blood from ALS patients are higher than in healthy controls, suggesting a potential biomarker. He previously co-founded Q-State Biosciences and described using stem cell and reprogramming technologies to model disease and identify potential therapeutics, including an anti-epileptic drug that was taken into the clinic.
“I'd like to introduce you to even a further concept which is the idea that we could use stem cell biology and reprogramming technologies to access classes of human cells that we've never been able to study in the laboratory, let alone use for therapeutic discovery.”
“This is an idea which I think really maximizes some of the unique properties of a specialty potent stem cells like human embryonic stem cells as really we can make a limitless supply of these cells for the first time.”
“The crucible in which we've been trying to employ this strategy is a devastating neurological condition called amyotrophic lateral sclerosis, which many of you may know is Lou Gehrig's Disease — this is a progressive and almost universally fatal neurological condition.”
“There's only one drug on the market for this condition which has a small statistical effect on lifespan; there are no disease-altering or curative therapeutics for ALS.”
“So far there have been hundreds of clinical trials which have come forward from therapeutics developed through that approach — there's been a 100 percent correlation between success in those animal models and failure in clinical trials, so clearly we need a new strategy.”
“One of the questions that we've been asking is whether or not we can model aspects of these electrophysiological changes actually in the tissue culture dish with a patient's own motor neurons, by taking easily obtained cells from patients like skin or blood and through reprogramming turn them into the types of cells th...”
“We've been able to show that indeed the cells of those patients in the dish manifest some aspects of those same changes that you see within their brain, we've been able to use those to identify potential therapeutics and excitingly we now are beginning to get some of the first clinical returns on those experiments.”
“The key discovery that we made implicated an already approved anti-epileptic drug as potentially being disease modifying in ALS — we actually were able to take that drug into the clinic; it's a GSK compound.”
“Three years ago along with Adam in the chemistry department we co-founded a company, Q-State Biosciences, which now has real collaborations using these types of technology in pain, epilepsy, cardio, and I think there are opportunities for using these technologies both for disease-oriented company generation as well as...”
“One of the things that was really attractive to me, it really drew me from Harvard to BioMarin was its overwhelming track record of success.”
“I believe that we've also built an incredible scientific discovery engine which is blazing the way in AAV gene therapies.”
“As we look ahead to the next 25 years and we ask ourselves what will it be that will ensure that BioMarin remains a success, it's that we will have taken advantage of this revolution in genetics and genomics to really understand the cellular circuitry which controls diseases.”
“With that in mind, we will have brought transformative therapies to patients with severe genetic illness.”
“We set out to carry out a fairly simple experiment at the time which was to take human embryonic stem cell lines that had been targeted to carry a motor neuron reporter gene and within those we differentiated them into spinal motor neurons and then exposed them to either control siRNAs or siRNAs that knock down TDP-43.”
“Because of the close association between the amount of TDP-43 alteration and the amount of stathmin 2, and because of its function which seemed reminiscent of the sort of thing that is altered in ALS, we began focusing first on trying to understand the underlying mechanisms that connected TDP-43 alterations to stathmin...”
Kevin Eggan, PhD Professor of Stem Cell and Regenerative Biology, Harvard University; Broad Institute of MIT and Harvard; ...
Kevin Eggan, Ph.D., joined BioMarin in October 2020 and serves as Group Vice President, Head of Research and Early ...
ALS ONE's 5th Annual ALS ONE Research Symposium, held Oct. 6, 7, & 11, 2022, hosted by our research team: Dr's Merit ...
Kevin Eggan, PhD Professor of Stem Cell and Regenerative Biology, Harvard University; Broad Institute of MIT and Harvard; Harvard Stem Cell Institute; scientific founder, Q-State Biosciences Part of the 2016 FUSION symposium at Harvard University. "FUSION: The Science and Business of Regenerative Medicine" was hosted by the Blavatnik Biomedical Accelerator (a program of Harvard's Office of Technology Development) and by the Blavatnik Fellowship in Life Science Entrepreneurship at Harvard Business School. For more information, visit http://otd.harvard.edu.
Kevin Eggan, Harvard Stem Cell Institute, USA, presentation at the Ask the Experts session in Sydney on 28 November 2011. The Ask the Experts session is held each year, in association with the International ALS/MND Symposium, to give people living with MND an opportunity to hear about the latest research from experts in the field. It is organised by the host association, MND Australia in 2011, and the International Alliance of ALS/MND Associations. It is supported by the ALS Hope Foundation.
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