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Perlstein Lab Raises $2 Million For Orphan Drug Discovery
Date:8/5/2014

otypic screens to produce mutation-specific lead compounds faster, cheaper and more systematically than current approaches. Finally, Perlstein Lab will funnel optimized leads into toxicology and clinical biomarker studies in patient-derived cells. The end results will be IND-ready orphan drug candidates that will be co-developed with commercial partners via licensing deals. This genetically informed approach improves the likelihood of bringing personalized orphan drugs to market at scale, and with minimized use of lab mice and rats.

"We will initially focus on lysosomal storage diseases, which are not only de-risked scientifically, but also have some of the most well-organized patient advocacy groups," said CEO and founder Ethan O. Perlstein, Ph.D. Before leaving academia in 2012, Ethan Perlstein received a Ph.D. in molecular and cell biology from Harvard University and completed an independent postdoctoral fellowship at Princeton University. Perlstein Lab scientists include Dr. Nina DiPrimio, Dr. Tom Hartl, Dr. Sangeetha Iyer and Kiran Singh. Henry Perlstein is co-founder and CTO. Dr. Jun Axup is the Director of Outreach. Tom Ruginis, CEO of HappiLabs, is the virtual lab manager. Rounding out the core team are Dr. John Alan Tucker, a medicinal chemistry advisor, and Dr. Laura Shawver, a business development advisor. More about the team and the science can be found at www.perlsteinlab.com.

Contact: Ethan Perlstein, ethan@perlsteinlab.com

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