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Perlstein Lab Raises $2 Million For Orphan Drug Discovery

SAN FRANCISCO, Aug. 5, 2014 /PRNewswire/ -- Perlstein Lab PBC, a San Francisco-based public benefit corporation whose mission is precision orphan drug discovery, just closed a $2 million seed round. The round was co-led by the clinical-stage orphan drug developer Retrophin, Inc. and the Wilsey family, which is personally affected by an orphan disease.

One in 12 people suffer from an orphan disease, yet 95% of orphan diseases have no FDA-approved drugs. At the current rate of 10-20 new orphan drug approvals per year, it will take another 450 years before drugs are approved for all orphan diseases. Perlstein Lab's scalable platform generates patient-specific small-molecule orphan drug candidates by combining: (1) patient mutation data, (2) high-throughput screens of genome-edited primordial disease models, and (3) lead optimization and validation studies in genetically matched patient cells. The successful cystic fibrosis drug Kalydeco, which was developed by Vertex Pharmaceuticals with financial contributions from the Cystic Fibrosis Foundation, proves that specific mutations can be targeted and suppressed by small molecules.

First, Perlstein Lab customizes primordial disease models for orphan disease patients by editing the genomes of yeast cells, worms, fruit flies and zebrafish strains to express patient-derived mutations in the ancestral versions of human orphan disease genes. Although most preclinical research currently uses laboratory rodents, basic researchers have used non-mammalian "simple model organisms" for decades to elucidate fundamental aspects of human biology. Drug screening with genetically personalized fruit flies was key to the development of the approved thyroid cancer drug Caprelsa, which was developed by AstraZeneca with scientific contributions from the laboratory of Dr. Ross Cagan.

Next, Perlstein Lab will perform whole-organism phenotypic 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

Contact: Ethan Perlstein,

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SOURCE Perlstein Lab PBC
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