PERLARA

Perlara, PBC is the first Public Benefit Corporation accelerating precision drug discovery for 5,000+ rare genetic diseases. PLab uses a platform of CRISPR-engineered animal models (yeast, nematodes, fruit flies and zebrafish) in whole-organism phenotypic screens to generate disease-reversing orphan drug candidates much faster and cheaper than traditional in vitro and cell-based approaches. Their lead discovery programs are Niemann-Pick Type C, a lysosomal storage disorder first described nearly... a century ago, and NGLY1 Deficiency, a congenital disorder of glycosylation first diagnosed last year. Their initial pipeline includes, among others, additional lysosomal storage disorders, e.gs, Batten and Mucolipidosis IV, mitochondrial diseases, e.g., Leigh, and peroxisomal biogenesis disorders, e.g., Zellweger.More