Perlstein Lab PBC

Perlstein Lab PBC is building a fast, scalable and precise whole-animal screening platform that yields small-molecule orphan drug candidates. Our core technology combines: (1) patient genetic data, (2) chemical modifier screens using CRISPR-edited primordial disease models such as yeast, worms, fruit flies and zebrafish, and (3) IND-enabling studies. Validated orphan drug candidates will be sold or licensed to clinical-stage orphan drug developers. Perlstein Lab is initially focusing on single-gene Mendelian disease affecting basic cell biology. Our first two disease programs target the lysosomal storage disease Niemann-Pick C/NPC, and the newly diagnosed congenital glycosylation disorder NGLY1 deficiency.