1 in 12 people has an orphan disease. 95% of orphan diseases have no treatment.
Each disease is caused
by a spectrum of genetic mutations.
Perlstein Lab studies each mutation
in model organisms to produce precision medicine.
Leave no mutation behind
PLab is the first Public Benefit Corporation accelerating precision drug discovery for 5,000+ rare genetic diseases.
Five months ago we introduced PERL101, one of our lead compounds for Niemann-Pick Type C (NPC) disease, in Part One. Here in Part Two we pick up the thread. On this journey we’ve progressed from nematodes to patient cells to mice with an unoptimized primary screening hit, bypassing hit-to-lead and shortening lead optimization. As we begin breeding up […]