Perlara

Perlara is a full-stack drug discovery and development company focused on inherited metabolic diseases, using a yeast-based platform and patient partnerships to advance therapies through IND-enabling studies and trials. They emphasize repurposing and a patient-partnered approach to accelerate progress in ultra-rare IMDs. Their flagship program is Maggie’s Pearl (epalrestat for PMM2-CDG) and they aim to scale programs from a dozen to several hundred over the decade.

The REMIT engine, a four-stage platform (repurposing with engineered models of inherited metabolic disease targets), builds a yeast avatar, screens a standardized compound library, validates findings in patient cells, and advances through IND-enabling steps to trials. The approach centers on four elements: yeast-based disease models, patient-cell validation, a unified 1-to-N timeline, and a patient/family-led partnership that anchors each program. The company hosts a data portal for pipeline and program data and highlights flagship and Cure Odysseys content as part of its platform narrative.

Who it’s for: Families and foundations supporting rare metabolic diseases, researchers and partner foundations, and pharmaceutical collaborators interested in leveraging yeast-based model systems and patient-centered pathways to accelerate therapy development for IMDs.

funding mentions (SBIR), active pipeline with Phase III trajectory, patient/family partnerships, and published pipeline data portal