Kevin Eggan on disease-in-a-dish
From Kevin Eggan, PhD - Harvard FUSION Symposium 2016 · · Harvard OTD
“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.”
On , Kevin Eggan, Chief Scientific Officer & Senior Vice President of Research and Early Development at BIOMARIN PHARMACEUTICAL INC, spoke about disease-in-a-dish during Kevin Eggan, PhD - Harvard FUSION Symposium 2016 on Harvard OTD.
About Kevin Eggan
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.