Perlstein Lab Information

Perlstein Lab solves the unmet medical needs of orphan disease patients with a scalable drug candidate generator that leaves no mutation behind. Our novel platform efficiently identifies personalized orphan drug candidates on a 12-18 month innovation cycle, combining: (1) patient genetic data and natural history, (2) high-throughput drug screening of genome-edited primordial animal models, and (3) safety and efficacy validation studies in patient-derived cells.